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Clinical Trial Results:
A Phase 2b/3 Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Investigate the Efficacy and Safety of PF-06651600 in Adult and Adolescent Alopecia Areata (AA) Subjects With 50% or Greater Scalp Hair Loss

Summary
EudraCT number	2018-001714-14
Trial protocol	GB DE HU CZ PL ES
Global end of trial date	24 Jun 2021

Results information
Results version number	v1(current)
This version publication date	11 Jan 2022
First version publication date	11 Jan 2022
Other versions

Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information

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Trial information

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Sponsor protocol code

B7981015

ISRCTN number

US NCT number

NCT03732807

WHO universal trial number (UTN)

Sponsor organisation name

Pfizer Inc.

Sponsor organisation address

235 E 42nd Street, New York, United States, NY 10017

Public contact

Pfizer ClinicalTrials.gov Call Center, Pfizer Inc., 001 18007181021, ClinicalTrials.gov_Inquiries@pfizer.com

Scientific contact

Pfizer ClinicalTrials.gov Call Center, Pfizer Inc., 001 18007181021, ClinicalTrials.gov_Inquiries@pfizer.com

Is trial part of an agreed paediatric investigation plan (PIP)

Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?

Yes

Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?

Analysis stage

Final

Date of interim/final analysis

10 Jul 2021

Is this the analysis of the primary completion data?

Global end of trial reached?

Yes

Global end of trial date

24 Jun 2021

Was the trial ended prematurely?

Main objective of the trial

To evaluate the efficacy of PF-06651600 compared to placebo in adult and adolescent alopecia areata (AA) subjects with 50% or greater scalp hair loss on regrowth of lost hair (measured by an absolute severity of alopecia tool (SALT) Score ≤20) at week 24. To evaluate the efficacy of PF-06651600 compared to placebo in adult and adolescent AA subjects with 50% or greater scalp hair loss on regrowth of lost hair (as measured by an absolute SALT Score ≤10) at Week 24 (this objective was utilized as the primary objective for the European Medicines Agency and competent authorities in the Voluntary Harmonisation Procedure countries).

Protection of trial subjects

The study was in compliance with the ethical principles derived from the Declaration of Helsinki and in compliance with all International Council for Harmonization (ICH) Good Clinical Practice (GCP) Guidelines. All the local regulatory requirements pertinent to safety of trial subjects were followed.

Background therapy

Evidence for comparator

Actual start date of recruitment

03 Dec 2018

Long term follow-up planned

Independent data monitoring committee (IDMC) involvement?

Yes

Number of subjects enrolled per country

Country: Number of subjects enrolled

Argentina: 5

Country: Number of subjects enrolled

Australia: 67

Country: Number of subjects enrolled

Canada: 98

Country: Number of subjects enrolled

Chile: 44

Country: Number of subjects enrolled

China: 81

Country: Number of subjects enrolled

Colombia: 3

Country: Number of subjects enrolled

Czechia: 20

Country: Number of subjects enrolled

Denmark: 31

Country: Number of subjects enrolled

Spain: 17

Country: Number of subjects enrolled

Georgia: 23

Country: Number of subjects enrolled

Hungary: 1

Country: Number of subjects enrolled

Japan: 47

Country: Number of subjects enrolled

Korea, Republic of: 7

Country: Number of subjects enrolled

Mexico: 1

Country: Number of subjects enrolled

Poland: 53

Country: Number of subjects enrolled

Russian Federation: 14

Country: Number of subjects enrolled

Taiwan: 11

Country: Number of subjects enrolled

United States: 195

Worldwide total number of subjects

718

EEA total number of subjects

122

Number of subjects enrolled per age group

In utero

Preterm newborn - gestational age < 37 wk

Newborns (0-27 days)

Infants and toddlers (28 days-23 months)

Children (2-11 years)

Adolescents (12-17 years)

105

Adults (18-64 years)

593

From 65 to 84 years

85 years and over

Subject disposition

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Recruitment details

The study was conducted in 18 countries from 03 December 2018 to 24 June 2021. A total of 718 subjects were enrolled.

Screening details

Total 1097 subjects signed the informed consent form. Out of which 379 subjects were screen failures, 718 actually enrolled into the study and were assigned to study treatments.

Period 1 title

Treatment Phase (up to Week 24)

Is this the baseline period?

Yes

Allocation method

Randomised - controlled

Blinding used

Double blind

Roles blinded

Subject, Investigator

Are arms mutually exclusive

Yes

Ritlecitinib (PF-06651600) 200 mg then 50 mg

Arm description

Subjects aged 12 years or above with moderate to severe AA with greater than or equal to (>=) 50 percent (%) hair loss of the scalp were randomised to receive Ritlecitinib 4 of 50 milligram (mg) tablet once daily for 4 weeks (loading phase) and then 50 mg tablet once daily for next 20 weeks (maintenance phase). Subjects then entered into extension phase of 24 weeks and continued to receive 50 mg tablet once daily. Subjects were followed up to maximum of 5 weeks after the last dose of study drug .

Arm type

Experimental

Investigational medicinal product name

Ritlecitinib

Investigational medicinal product code

PF-06651600

Other name

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects received 4 of 50 mg tablet of Ritlecitinib once daily for 4 weeks and then 50 mg tablet once daily for next 44 weeks.

Ritlecitinib (PF-06651600) 200 mg then 30 mg

Arm description

Subjects aged 12 years or above with moderate to severe AA with >=50% hair loss of the scalp were randomised to receive Ritlecitinib 4 of 50 mg tablet once daily for 4 weeks (loading phase) and then 3 of 10 mg tablet once daily for next 20 weeks (maintenance phase). Subjects then entered into extension phase of 24 weeks and continued to receive 3 of 10 mg tablet once daily. Subjects were followed up to maximum of 5 weeks after the last dose of study drug.

Arm type

Experimental

Investigational medicinal product name

Ritlecitinib

Investigational medicinal product code

PF-06651600

Other name

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects received 4 of 50 mg tablet of Ritlecitinib once daily for 4 weeks and then 3 of 10 mg tablet once daily for next 44 weeks.

Ritlecitinib (PF-06651600) 50 mg

Arm description

Subjects aged 12 years or above with moderate to severe AA with >=50% hair loss of the scalp were randomised to receive Ritlecitinib 50 mg tablet once daily for 4 weeks (loading phase). Subjects then continued to receive 50 mg tablet once daily in maintenance phase of 20 weeks and extension phase of 24 weeks. Subjects were followed up to maximum of 5 weeks after the last dose of study drug.

Arm type

Experimental

Investigational medicinal product name

Ritlecitinib

Investigational medicinal product code

PF-06651600

Other name

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects received 50 mg tablet of Ritlecitinib once daily for 48 weeks.

Ritlecitinib (PF-06651600) 30 mg

Arm description

Subjects aged 12 years or above with moderate to severe AA with >=50% hair loss of the scalp were randomised to receive Ritlecitinib 3 of 10 mg tablet once daily for 4 weeks (loading phase). Subjects then continued to receive 3 of 10 mg tablet once daily in maintenance phase of 20 weeks and extension phase of 24 weeks. Subjects were followed up to maximum of 5 weeks after the last dose of study drug.

Arm type

Experimental

Investigational medicinal product name

Ritlecitinib

Investigational medicinal product code

PF-06651600

Other name

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects received 3 of 10 mg tablet of Ritlecitinib once daily for 48 weeks.

Ritlecitinib (PF-06651600) 10 mg

Arm description

Subjects aged 12 years or above with moderate to severe AA with >=50% hair loss of the scalp were randomised to receive Ritlecitinib 10 mg tablet once daily for 4 weeks (loading phase). Subjects then continued to receive 10 mg tablet once daily in maintenance phase of 20 weeks and extension phase of 24 weeks. Subjects were followed up to maximum of 5 weeks after the last dose of study drug.

Arm type

Experimental

Investigational medicinal product name

Ritlecitinib

Investigational medicinal product code

PF-06651600

Other name

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects received 10 mg tablet of Ritlecitinib once daily for 48 weeks.

Placebo, Ritlecitinib (PF-06651600) 200 mg then 50 mg

Arm description

Subjects aged 12 years or above with moderate to severe AA with >=50% hair loss of the scalp were randomised to receive placebo once daily for 4 weeks (loading phase) and then continued placebo once daily for next 20 weeks (maintenance phase). Subjects then entered into extension phase of 24 weeks and received 4 of 50 mg tablet once daily for 4 weeks and then 50 mg tablet once daily for 20 weeks. Subjects were followed up to maximum of 5 weeks after the last dose of study drug.

Arm type

Placebo

Investigational medicinal product name

Placebo, Ritlecitinib

Investigational medicinal product code

PF-06651600

Other name

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects received placebo once daily for 24 weeks and then 4 of 50 mg tablet of Ritlecitinib once daily for 4 weeks followed by 50 mg tablet of Ritlecitinib once daily for next 20 weeks.

Placebo, Ritlecitinib (PF-06651600) 50 mg

Arm description

Subjects aged 12 years or above with moderate to severe AA with >=50% hair loss of the scalp were randomised to receive placebo once daily for 4 weeks (loading phase) and then continued to receive placebo once daily for next 20 weeks (maintenance phase). Subjects then entered into extension phase of 24 weeks and receive 50 mg tablet once daily. Subjects were followed up to maximum of 5 weeks after the last dose of study drug.

Arm type

Placebo

Investigational medicinal product name

Placebo, Ritlecitinib

Investigational medicinal product code

PF-06651600

Other name

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects received placebo once daily for 24 weeks and then 50 mg tablet of Ritlecitinib once daily for next 24 weeks.

Number of subjects in period 1	Ritlecitinib (PF-06651600) 200 mg then 50 mg	Ritlecitinib (PF-06651600) 200 mg then 30 mg	Ritlecitinib (PF-06651600) 50 mg	Ritlecitinib (PF-06651600) 30 mg	Ritlecitinib (PF-06651600) 10 mg	Placebo, Ritlecitinib (PF-06651600) 200 mg then 50 mg	Placebo, Ritlecitinib (PF-06651600) 50 mg
Started	132	130	130	132	63	65	66
Completed	122	124	121	117	58	63	61
Not completed	10	6	9	15	5	2	5
Physician decision	-	1	2	5	-	-	1
Pregnancy	1	-	-	-	1	-	1
Adverse event	3	-	2	4	2	-	1
Lost to follow-up	1	-	1	2	1	-	-
Protocol deviation	1	1	-	-	-	-	-
Withdrawal by subject	4	4	4	4	1	1	2
Lack of efficacy	-	-	-	-	-	1	-

Period 2 title

Treatment Extension (Week 25 up to 48)

Is this the baseline period?

Allocation method

Randomised - controlled

Blinding used

Double blind

Roles blinded

Subject, Investigator

Are arms mutually exclusive

Yes

Ritlecitinib (PF-06651600) 200 mg then 50 mg

Arm description

Subjects aged 12 years or above with moderate to severe AA with >= 50 % hair loss of the scalp were randomised to receive Ritlecitinib 4 of 50 mg tablet once daily for 4 weeks (loading phase) and then 50 mg tablet once daily for next 20 weeks (maintenance phase). Subjects then entered into extension phase of 24 weeks and continued to receive 50 mg tablet once daily. Subjects were followed up to maximum of 5 weeks after the last dose of study drug .

Arm type

Experimental

Investigational medicinal product name

Ritlecitinib

Investigational medicinal product code

PF-06651600

Other name

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects received 4 of 50 mg tablet of Ritlecitinib once daily for 4 weeks and then 50 mg tablet once daily for next 44 weeks.

Ritlecitinib (PF-06651600) 200 mg then 30 mg

Arm description

Arm type

Experimental

Investigational medicinal product name

Ritlecitinib

Investigational medicinal product code

PF-06651600

Other name

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects received 4 of 50 mg tablet of Ritlecitinib once daily for 4 weeks and then 3 of 10 mg tablet once daily for next 44 weeks.

Ritlecitinib (PF-06651600) 50 mg

Arm description

Arm type

Experimental

Investigational medicinal product name

Ritlecitinib

Investigational medicinal product code

PF-06651600

Other name

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects received 50 mg tablet of Ritlecitinib once daily for 48 weeks.

Ritlecitinib (PF-06651600) 30 mg

Arm description

Arm type

Experimental

Investigational medicinal product name

Ritlecitinib

Investigational medicinal product code

PF-06651600

Other name

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects received 3 of 10 mg tablet of Ritlecitinib once daily for 48 weeks.

Ritlecitinib (PF-06651600) 10 mg

Arm description

Arm type

Experimental

Investigational medicinal product name

Ritlecitinib

Investigational medicinal product code

PF-06651600

Other name

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects received 10 mg tablet of Ritlecitinib once daily for 48 weeks.

Placebo, Ritlecitinib (PF-06651600) 200 mg then 50 mg

Arm description

Arm type

Placebo

Investigational medicinal product name

Placebo, Ritlecitinib

Investigational medicinal product code

PF-06651600

Other name

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects received placebo once daily for 24 weeks and then 4 of 50 mg tablet of Ritlecitinib once daily for 4 weeks followed by 50 mg tablet of Ritlecitinib once daily for next 20 weeks.

Placebo, Ritlecitinib (PF-06651600) 50 mg

Arm description

Subjects aged 12 years or above with moderate to severe AA with >=50% hair loss of the scalp were randomised to receive placebo once daily for 4 weeks (loading phase) and then continued to receive placebo once daily for next 20 weeks (maintenance phase). Subjects then entered into extension phase of 24 weeks and receive 50 mg tablet once daily. Subjects were followed up to maximum of 5 weeks after the last dose of study drug.

Arm type

Placebo

Investigational medicinal product name

Placebo, Ritlecitinib

Investigational medicinal product code

PF-06651600

Other name

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects received placebo once daily for 24 weeks and then 50 mg tablet of Ritlecitinib once daily for next 24 weeks.

Number of subjects in period 2	Ritlecitinib (PF-06651600) 200 mg then 50 mg	Ritlecitinib (PF-06651600) 200 mg then 30 mg	Ritlecitinib (PF-06651600) 50 mg	Ritlecitinib (PF-06651600) 30 mg	Ritlecitinib (PF-06651600) 10 mg	Placebo, Ritlecitinib (PF-06651600) 200 mg then 50 mg	Placebo, Ritlecitinib (PF-06651600) 50 mg
Started	122	124	121	117	58	63	61
Completed	126	119	125	123	58	62	62
Not completed	6	11	5	9	5	3	4
Physician decision	-	1	-	1	1	-	-
Adverse event	-	2	2	1	-	-	2
Unspecified	1	4	-	2	-	-	-
Lost to follow-up	-	-	2	1	1	2	-
Non-Compliant with study drug	-	1	-	-	-	-	-
Lack of efficacy	2	1	-	3	3	-	1
Withdrawal by subject	3	2	1	1	-	1	1
Joined	10	6	9	15	5	2	5
Other	10	6	9	15	5	2	5

Baseline characteristics

Top of page

Reporting group title

Ritlecitinib (PF-06651600) 200 mg then 50 mg

Reporting group description

Reporting group title

Ritlecitinib (PF-06651600) 200 mg then 30 mg

Reporting group description

Reporting group title

Ritlecitinib (PF-06651600) 50 mg

Reporting group description

Reporting group title

Ritlecitinib (PF-06651600) 30 mg

Reporting group description

Reporting group title

Ritlecitinib (PF-06651600) 10 mg

Reporting group description

Reporting group title

Placebo, Ritlecitinib (PF-06651600) 200 mg then 50 mg

Reporting group description

Reporting group title

Placebo, Ritlecitinib (PF-06651600) 50 mg

Reporting group description

Subjects aged 12 years or above with moderate to severe AA with >=50% hair loss of the scalp were randomised to receive placebo once daily for 4 weeks (loading phase) and then continued to receive placebo once daily for next 20 weeks (maintenance phase). Subjects then entered into extension phase of 24 weeks and receive 50 mg tablet once daily. Subjects were followed up to maximum of 5 weeks after the last dose of study drug.

Reporting group values	Ritlecitinib (PF-06651600) 200 mg then 50 mg	Ritlecitinib (PF-06651600) 200 mg then 30 mg	Ritlecitinib (PF-06651600) 50 mg	Ritlecitinib (PF-06651600) 30 mg	Ritlecitinib (PF-06651600) 10 mg	Placebo, Ritlecitinib (PF-06651600) 200 mg then 50 mg	Placebo, Ritlecitinib (PF-06651600) 50 mg	Total
Number of subjects	132	130	130	132	63	65	66	718
Age categorical
Units: Subjects
In utero	0	0	0	0	0	0	0	0
Preterm newborn infants (gestational age < 37 wks)	0	0	0	0	0	0	0	0
Newborns (0-27 days)	0	0	0	0	0	0	0	0
Infants and toddlers (28 days-23 months)	0	0	0	0	0	0	0	0
Children (2-11 years)	0	0	0	0	0	0	0	0
Adolescents (12-17 years)	20	19	18	20	9	10	9	105
Adults (18-64 years)	108	110	109	105	54	53	54	593
From 65-84 years	4	1	3	7	0	2	3	20
85 years and over	0	0	0	0	0	0	0	0
Age Continuous
Units: Years
arithmetic mean (standard deviation)	34.5 ( 14.98 )	33.7 ( 13.75 )	32.4 ( 13.36 )	33.7 ( 14.83 )	34.3 ( 13.88 )	33.0 ( 14.01 )	35.0 ( 15.89 )	-
Sex: Female, Male
Units: Subjects
Female	81	85	71	80	43	46	40	446
Male	51	45	59	52	20	19	26	272
Race (NIH/OMB)
Units: Subjects
American Indian or Alaska Native	0	1	0	2	0	0	0	3
Asian	33	28	43	34	17	14	17	186
Native Hawaiian or Other Pacific Islander	0	0	0	0	1	0	0	1
Black or African American	6	7	5	3	2	2	2	27
White	92	90	79	91	42	47	47	488
More than one race	0	3	1	2	0	2	0	8
Unknown or Not Reported	1	1	2	0	1	0	0	5
Ethnicity (NIH/OMB)
Units: Subjects
Hispanic or Latino	18	16	11	23	8	7	4	87
Not Hispanic or Latino	113	114	116	109	55	58	61	626
Unknown or Not Reported	1	0	3	0	0	0	1	5

Subject analysis set title

Placebo

Subject analysis set type

Full analysis

Subject analysis set description

Subjects aged 12 years or above with moderate to severe AA with>=50% hair loss of the scalp were randomised to receive placebo once daily for 4 weeks (loading phase) and then continued placebo once daily for next 20 weeks (maintenance phase). Subjects then entered into extension phase of 24 weeks and received either 4 of 50 mg tablet once daily for 4 weeks followed by 50 mg tablet once daily for next 20 weeks or 50 mg tablet once daily for next 24 weeks. Subjects were followed up to maximum of 5 weeks after the last dose of study drug.

Subject analysis sets values	Placebo
Number of subjects	131
Age categorical
Units: Subjects
In utero	0
Preterm newborn infants (gestational age < 37 wks)	0
Newborns (0-27 days)	0
Infants and toddlers (28 days-23 months)	0
Children (2-11 years)	0
Adolescents (12-17 years)	19
Adults (18-64 years)	107
From 65-84 years	5
85 years and over	0
Age Continuous
Units: Years
arithmetic mean (standard deviation)	34.0 ( 14.96 )
Sex: Female, Male
Units: Subjects
Female	86
Male	45
Race (NIH/OMB)
Units: Subjects
American Indian or Alaska Native	0
Asian	31
Native Hawaiian or Other Pacific Islander	0
Black or African American	4
White	94
More than one race	2
Unknown or Not Reported	0
Ethnicity (NIH/OMB)
Units: Subjects
Hispanic or Latino	11
Not Hispanic or Latino	119
Unknown or Not Reported	1

End points

Top of page

Reporting group title

Ritlecitinib (PF-06651600) 200 mg then 50 mg

Reporting group description

Reporting group title

Ritlecitinib (PF-06651600) 200 mg then 30 mg

Reporting group description

Reporting group title

Ritlecitinib (PF-06651600) 50 mg

Reporting group description

Reporting group title

Ritlecitinib (PF-06651600) 30 mg

Reporting group description

Reporting group title

Ritlecitinib (PF-06651600) 10 mg

Reporting group description

Reporting group title

Placebo, Ritlecitinib (PF-06651600) 200 mg then 50 mg

Reporting group description

Reporting group title

Placebo, Ritlecitinib (PF-06651600) 50 mg

Reporting group description

Subjects aged 12 years or above with moderate to severe AA with >=50% hair loss of the scalp were randomised to receive placebo once daily for 4 weeks (loading phase) and then continued to receive placebo once daily for next 20 weeks (maintenance phase). Subjects then entered into extension phase of 24 weeks and receive 50 mg tablet once daily. Subjects were followed up to maximum of 5 weeks after the last dose of study drug.

Reporting group title

Ritlecitinib (PF-06651600) 200 mg then 50 mg

Reporting group description

Subjects aged 12 years or above with moderate to severe AA with >= 50 % hair loss of the scalp were randomised to receive Ritlecitinib 4 of 50 mg tablet once daily for 4 weeks (loading phase) and then 50 mg tablet once daily for next 20 weeks (maintenance phase). Subjects then entered into extension phase of 24 weeks and continued to receive 50 mg tablet once daily. Subjects were followed up to maximum of 5 weeks after the last dose of study drug .

Reporting group title

Ritlecitinib (PF-06651600) 200 mg then 30 mg

Reporting group description

Reporting group title

Ritlecitinib (PF-06651600) 50 mg

Reporting group description

Reporting group title

Ritlecitinib (PF-06651600) 30 mg

Reporting group description

Reporting group title

Ritlecitinib (PF-06651600) 10 mg

Reporting group description

Reporting group title

Placebo, Ritlecitinib (PF-06651600) 200 mg then 50 mg

Reporting group description

Reporting group title

Placebo, Ritlecitinib (PF-06651600) 50 mg

Reporting group description

Subjects aged 12 years or above with moderate to severe AA with >=50% hair loss of the scalp were randomised to receive placebo once daily for 4 weeks (loading phase) and then continued to receive placebo once daily for next 20 weeks (maintenance phase). Subjects then entered into extension phase of 24 weeks and receive 50 mg tablet once daily. Subjects were followed up to maximum of 5 weeks after the last dose of study drug.

Subject analysis set title

Placebo

Subject analysis set type

Full analysis

Subject analysis set description

Subjects aged 12 years or above with moderate to severe AA with>=50% hair loss of the scalp were randomised to receive placebo once daily for 4 weeks (loading phase) and then continued placebo once daily for next 20 weeks (maintenance phase). Subjects then entered into extension phase of 24 weeks and received either 4 of 50 mg tablet once daily for 4 weeks followed by 50 mg tablet once daily for next 20 weeks or 50 mg tablet once daily for next 24 weeks. Subjects were followed up to maximum of 5 weeks after the last dose of study drug.

Primary: Percentage of Subjects With an Absolute Severity of Alopecia Tool (SALT) Score of Less Than or Equal to 10 at Week 24: Analysis 4

Top of page

End point title

Percentage of Subjects With an Absolute Severity of Alopecia Tool (SALT) Score of Less Than or Equal to 10 at Week 24: Analysis 4 ^[1]

End point description

SALT is a quantitative assessment of AA severity based on the scalp hair loss. The SALT score can vary from 0 (normal) to 100 (severe), with higher scores representing increased severity of disease. In this endpoint, percentage of subjects with SALT score less than or equal to (<=) 10 at week 24 were reported. Full analysis set (FAS) included all subjects who were randomised, regardless of whether they received study medication. Here, “Number of Subjects Analysed” signifies subjects evaluable for this endpoint. Analysis 4: Data imputed by using a missing at random (MAR) mechanism for subjects with missing data due to COVID-19. Missing data due to reasons not related to COVID-19 were consider as non-responders.

End point type

Primary

End point timeframe

Week 24

Notes
[1] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: Statistics were planned only for the arms specified

End point values	Ritlecitinib (PF-06651600) 200 mg then 50 mg	Ritlecitinib (PF-06651600) 200 mg then 30 mg	Ritlecitinib (PF-06651600) 50 mg	Ritlecitinib (PF-06651600) 30 mg	Ritlecitinib (PF-06651600) 10 mg	Placebo
Number of subjects analysed	118	119	119	114	55	125
Units: Percentage of subjects
number (not applicable)	21.29	12.87	13.42	10.62	1.65	1.54

Ritlecitinib 200mg then 50mg versus Placebo

Statistical analysis description

A generalised linear mixed effect model without imputation using observed data up to Week 24 was used as the imputation model. A single complete imputed data set for Week 24 was analysed using the Miettinen and Nurminen method as the analysis model.

Comparison groups

Ritlecitinib (PF-06651600) 200 mg then 50 mg v Placebo

Number of subjects included in analysis

243

Analysis specification

Pre-specified

Analysis type

superiority

P-value

< 0.0001

Method

Miettinen and Nurminen method

Parameter type

Estimate of difference

Point estimate

19.75

Confidence interval

level

95%

sides

2-sided

lower limit

11.91

upper limit

27.59

Ritlecitinib 200mg then 30mg versus Placebo

Statistical analysis description

Comparison groups

Ritlecitinib (PF-06651600) 200 mg then 30 mg v Placebo

Number of subjects included in analysis

244

Analysis specification

Pre-specified

Analysis type

P-value

= 0.000526

Method

Miettinen and Nurminen method

Parameter type

Estimate of difference

Point estimate

11.33

Confidence interval

level

95%

sides

2-sided

lower limit

4.93

upper limit

17.74

Ritlecitinib 50 mg versus Placebo

Statistical analysis description

Comparison groups

Ritlecitinib (PF-06651600) 50 mg v Placebo

Number of subjects included in analysis

244

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.000311

Method

Miettinen and Nurminen method

Parameter type

Estimate of difference

Point estimate

11.88

Confidence interval

level

95%

sides

2-sided

lower limit

5.42

upper limit

18.33

Ritlecitinib 30 mg versus Placebo

Statistical analysis description

Comparison groups

Ritlecitinib (PF-06651600) 30 mg v Placebo

Number of subjects included in analysis

239

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.002922

Method

Miettinen and Nurminen method

Parameter type

Estimate of difference

Point estimate

9.09

Confidence interval

level

95%

sides

2-sided

lower limit

3.1

upper limit

15.07

Secondary: Percentage of Subjects With an Absolute SALT Score of Less Than or Equal to 20 at Week 24

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End point title

Percentage of Subjects With an Absolute SALT Score of Less Than or Equal to 20 at Week 24 ^[2]

End point description

SALT is a quantitative assessment of AA severity based on the scalp hair loss. The SALT score can vary from 0 (normal) to 100 (severe), with higher scores representing increased severity of disease. In this endpoint, percentage of subjects with SALT score <= 20 at week 24 were reported. subjects with missing SALT scores due to coronavirus disease -19 related reasons were excluded from this analysis, while subjects with missing data due to other reasons were considered as non-responders. FAS included all subjects who were randomised, regardless of whether they received study medication. Here, “Number of Subjects Analysed” signifies subjects evaluable for this endpoint.

End point type

Secondary

End point timeframe

Week 24

Notes
[2] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: Statistics were planned only for the arms specified

End point values	Ritlecitinib (PF-06651600) 200 mg then 50 mg	Ritlecitinib (PF-06651600) 200 mg then 30 mg	Ritlecitinib (PF-06651600) 50 mg	Ritlecitinib (PF-06651600) 30 mg	Ritlecitinib (PF-06651600) 10 mg	Placebo
Number of subjects analysed	124	121	124	119	59	130
Units: Percentage of Subjects
number (confidence interval 95%)	30.65 (22.53 to 38.76)	22.31 (14.90 to 29.73)	23.39 (15.94 to 30.84)	14.29 (8.00 to 20.57)	1.69 (0.00 to 4.99)	1.54 (0.00 to 3.65)

Ritlecitinib 200mg then 50mg versus Placebo

Comparison groups

Ritlecitinib (PF-06651600) 200 mg then 50 mg v Placebo

Number of subjects included in analysis

254

Analysis specification

Pre-specified

Analysis type

superiority

P-value

< 0.0001

Method

Miettinen and Nurminen method

Parameter type

Estimate of difference

Point estimate

29.11

Confidence interval

level

95%

sides

2-sided

lower limit

21.17

upper limit

37.91

Ritlecitinib 200mg then 30mg versus Placebo

Comparison groups

Ritlecitinib (PF-06651600) 200 mg then 30 mg v Placebo

Number of subjects included in analysis

251

Analysis specification

Pre-specified

Analysis type

superiority

P-value

< 0.0001

Method

Miettinen and Nurminen method

Parameter type

Estimate of difference

Point estimate

20.78

Confidence interval

level

95%

sides

2-sided

lower limit

13.65

upper limit

29.18

Ritlecitinib 50 mg versus Placebo

Comparison groups

Ritlecitinib (PF-06651600) 50 mg v Placebo

Number of subjects included in analysis

254

Analysis specification

Pre-specified

Analysis type

superiority

P-value

< 0.0001

Method

Miettinen and Nurminen method

Parameter type

Estimate of difference

Point estimate

21.85

Confidence interval

level

95%

sides

2-sided

lower limit

14.65

upper limit

30.23

Ritlecitinib 30 mg versus Placebo

Comparison groups

Ritlecitinib (PF-06651600) 30 mg v Placebo

Number of subjects included in analysis

249

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.000154

Method

Miettinen and Nurminen method

Parameter type

Estimate of difference

Point estimate

12.75

Confidence interval

level

95%

sides

2-sided

lower limit

6.69

upper limit

20.36

Secondary: Percentage of Subjects With an Absolute SALT Score of Less Than or Equal to 10 at Week 24: Analysis 1

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End point title

Percentage of Subjects With an Absolute SALT Score of Less Than or Equal to 10 at Week 24: Analysis 1 ^[3]

End point description

SALT is a quantitative assessment of AA severity based on the scalp hair loss. The SALT score can vary from 0 (normal) to 100 (severe), with higher scores representing increased severity of disease. In this endpoint, percentage of subjects with SALT score <= 10 at week 24 were reported. FAS included all subjects who were randomised, regardless of whether they received study medication. Here, “Number of Subjects Analysed” signifies subjects evaluable for this endpoint. Analysis 1: Subjects with missing SALT score at Week 24 due to COVID-19 related reasons excluded from analysis at that time point, subjects with missing SALT scores due to other reasons counted as non-responders at that time point.

End point type

Secondary

End point timeframe

Week 24

Notes
[3] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: Statistics were planned only for the arms specified

End point values	Ritlecitinib (PF-06651600) 200 mg then 50 mg	Ritlecitinib (PF-06651600) 200 mg then 30 mg	Ritlecitinib (PF-06651600) 50 mg	Ritlecitinib (PF-06651600) 30 mg	Ritlecitinib (PF-06651600) 10 mg	Placebo
Number of subjects analysed	124	121	124	119	59	130
Units: Percentage of subjects
number (confidence interval 95%)	21.77 (14.51 to 29.04)	13.22 (7.19 to 19.26)	13.71 (7.66 to 19.76)	10.92 (5.32 to 16.53)	1.69 (0.00 to 4.99)	1.54 (0.00 to 3.65)

Ritlecitinib 200mg then 50mg versus Placebo

Comparison groups

Ritlecitinib (PF-06651600) 200 mg then 50 mg v Placebo

Number of subjects included in analysis

254

Analysis specification

Pre-specified

Analysis type

superiority

P-value

< 0.0001

Method

Miettinen and Nurminen method

Parameter type

Estimate of difference

Point estimate

20.24

Confidence interval

level

95%

sides

2-sided

lower limit

13.23

upper limit

28.49

Ritlecitinib 200mg then 30mg versus Placebo

Comparison groups

Ritlecitinib (PF-06651600) 200 mg then 30 mg v Placebo

Number of subjects included in analysis

251

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.000337

Method

Miettinen and Nurminen method

Parameter type

Estimate of difference

Point estimate

11.68

Confidence interval

level

95%

sides

2-sided

lower limit

5.82

upper limit

19.07

Ritlecitinib 50 mg versus Placebo

Comparison groups

Ritlecitinib (PF-06651600) 50 mg v Placebo

Number of subjects included in analysis

254

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.000228

Method

Miettinen and Nurminen method

Parameter type

Estimate of difference

Point estimate

12.17

Confidence interval

level

95%

sides

2-sided

lower limit

6.27

upper limit

19.53

Ritlecitinib 30 mg versus Placebo

Comparison groups

Ritlecitinib (PF-06651600) 30 mg v Placebo

Number of subjects included in analysis

249

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.001875

Method

Miettinen and Nurminen method

Parameter type

Estimate of difference

Point estimate

9.39

Confidence interval

level

95%

sides

2-sided

lower limit

3.86

upper limit

16.46

Secondary: Percentage of Subjects With Patient Global Impression of Change (PGI-C) Score of Moderately Improved or Greatly Improved at Week 24

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End point title

Percentage of Subjects With Patient Global Impression of Change (PGI-C) Score of Moderately Improved or Greatly Improved at Week 24 ^[4]

End point description

PGI-C is a self-administered questionnaire to evaluate the improvement or worsening of subject’s AA as compared to the start of the study. PGI-C was assessed on a 7-point Likert scale ranged from 1 (greatly improved) to 7 (greatly worsened). Categories were defined based on the PGI-C scores as follows: 1=greatly improved, 2=moderately improved, 3=slightly improved, 4=not changed, 5=slightly worsened, 6=moderately worsened and 7=greatly worsened. FAS included all subjects who were randomised, regardless of whether they received study medication. Here, “Number of Subjects Analysed” signifies subjects evaluable for this endpoint.

End point type

Secondary

End point timeframe

Week 24

Notes
[4] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: Statistics were planned only for the arms specified

End point values	Ritlecitinib (PF-06651600) 200 mg then 50 mg	Ritlecitinib (PF-06651600) 200 mg then 30 mg	Ritlecitinib (PF-06651600) 50 mg	Ritlecitinib (PF-06651600) 30 mg	Ritlecitinib (PF-06651600) 10 mg	Placebo
Number of subjects analysed	120	119	120	116	55	125
Units: Percentage of subjects
number (not applicable)	52.19	45.40	49.17	41.95	11.36	9.23

Ritlecitinib 200 mg then 50 mg versus Placebo

Comparison groups

Ritlecitinib (PF-06651600) 200 mg then 50 mg v Placebo

Number of subjects included in analysis

245

Analysis specification

Pre-specified

Analysis type

superiority

P-value

< 0.0001

Method

Miettinen and Nurminen method

Parameter type

Estimate of difference

Point estimate

42.96

Confidence interval

level

95%

sides

2-sided

lower limit

31.68

upper limit

54.25

Ritlecitinib 200 mg then 30 mg versus Placebo

Comparison groups

Ritlecitinib (PF-06651600) 200 mg then 30 mg v Placebo

Number of subjects included in analysis

244

Analysis specification

Pre-specified

Analysis type

superiority

P-value

< 0.0001

Method

Miettinen and Nurminen method

Parameter type

Estimate of difference

Point estimate

36.18

Confidence interval

level

95%

sides

2-sided

lower limit

25.22

upper limit

47.14

Ritlecitinib 50 mg versus Placebo

Comparison groups

Ritlecitinib (PF-06651600) 50 mg v Placebo

Number of subjects included in analysis

245

Analysis specification

Pre-specified

Analysis type

superiority

P-value

< 0.0001

Method

Miettinen and Nurminen method

Parameter type

Estimate of difference

Point estimate

39.96

Confidence interval

level

95%

sides

2-sided

lower limit

28.85

upper limit

51.06

Ritlecitinib 30 mg versus Placebo

Comparison groups

Ritlecitinib (PF-06651600) 30 mg v Placebo

Number of subjects included in analysis

241

Analysis specification

Pre-specified

Analysis type

superiority

P-value

< 0.0001

Method

Miettinen and Nurminen method

Parameter type

Estimate of difference

Point estimate

32.72

Confidence interval

level

95%

sides

2-sided

lower limit

21.95

upper limit

43.5

Secondary: Exposure Response of PF-06651600 on Regrowth of Lost Hair Based on Absolute SALT Score of Less Than or Equal to 20 at Week 24: Maximum Effect (Emax) Model

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End point title

Exposure Response of PF-06651600 on Regrowth of Lost Hair Based on Absolute SALT Score of Less Than or Equal to 20 at Week 24: Maximum Effect (Emax) Model ^[5]

End point description

Exposure response of PF-06651600 on regrowth of scalp hair was characterised using Bayesian three-parameter hyperbolic Emax model for SALT score <=20 at Week 24 with additional term for effect of loading dose. In Emax exposure-response model response function was log odds of percentage of subjects with response based on SALT <=20 at Week 24, which was fit on logistic scale and then back-transformed to percentage. Effect of loading dose is included as fixed factor in model. Variable that represents loading dose has values of 1 for groups 200/50 mg once daily and 200/30 mg once daily and of 0 for remaining groups. SALT is quantitative assessment of AA severity based on scalp hair loss. SALT score can vary from 0 (normal) to 100 (severe), with higher scores representing increased severity of disease. FAS: all subjects who were randomised, regardless of whether they received study medication. Here, "Number of Subjects Analysed” signifies subjects evaluable for this endpoint.

End point type

Secondary

End point timeframe

Week 24

Notes
[5] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: Statistics were planned only for the arms specified

End point values	Ritlecitinib (PF-06651600) 200 mg then 50 mg	Ritlecitinib (PF-06651600) 200 mg then 30 mg	Ritlecitinib (PF-06651600) 50 mg	Ritlecitinib (PF-06651600) 30 mg	Ritlecitinib (PF-06651600) 10 mg	Placebo
Number of subjects analysed	124	121	124	119	59	130
Units: Percentage of subjects
number (not applicable)	32.37	20.57	22.52	13.58	4.81	1.63

No statistical analyses for this end point

Secondary: Exposure Response of PF-06651600 on Regrowth of Lost Hair Based on Absolute SALT Score of Less Than or Equal to 10 at Week 24: Maximum Effect (Emax) Model

Top of page

End point title

Exposure Response of PF-06651600 on Regrowth of Lost Hair Based on Absolute SALT Score of Less Than or Equal to 10 at Week 24: Maximum Effect (Emax) Model ^[6]

End point description

Exposure response of PF-06651600 on regrowth of scalp hair was characterised using Bayesian three-parameter hyperbolic Emax model for SALT score <=10 at Week 24 with additional term for effect of loading dose. In Emax exposure-response model response function was log odds of percentage of subjects with response based on SALT <=10 at Week 24, which was fit on logistic scale and then back-transformed to percentage. Effect of loading dose is included as fixed factor in model. Variable that represents loading dose has values of 1 for groups 200/50 mg once daily and 200/30 mg once daily and of 0 for remaining groups. SALT is quantitative assessment of AA severity based on scalp hair loss. SALT score can vary from 0 (normal) to 100 (severe), with higher scores representing increased severity of disease. FAS: all subjects who were randomised, regardless of whether they received study medication. Here, "Number of Subjects Analysed” signifies subjects evaluable for this endpoint.

End point type

Secondary

End point timeframe

Week 24

Notes
[6] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: Statistics were planned only for the arms specified

End point values	Ritlecitinib (PF-06651600) 200 mg then 50 mg	Ritlecitinib (PF-06651600) 200 mg then 30 mg	Ritlecitinib (PF-06651600) 50 mg	Ritlecitinib (PF-06651600) 30 mg	Ritlecitinib (PF-06651600) 10 mg	Placebo
Number of subjects analysed	124	121	124	119	59	130
Units: Percentage of subjects
number (not applicable)	21.29	13.76	14.43	9.02	3.88	1.66

No statistical analyses for this end point

Secondary: Percentage of Subjects With an Absolute SALT Score of Less Than or Equal to 20 at Week 4, 8, 12, 18, 28, 34, 40, and 48

Top of page

End point title

Percentage of Subjects With an Absolute SALT Score of Less Than or Equal to 20 at Week 4, 8, 12, 18, 28, 34, 40, and 48

End point description

SALT is a quantitative assessment of AA severity based on the scalp hair loss. The SALT score can vary from 0 (normal) to 100 (severe), with higher scores representing increased severity of disease. FAS included all subjects who were randomised, regardless of whether they received study medication. Here, “n” signifies subjects evaluable at specific time point. 99999 signifies that 95% confidence interval (CI) could not be calculated as there were no subjects with SALT score <=20.

End point type

Secondary

End point timeframe

Week 4, 8, 12, 18, 28, 34, 40, and 48

End point values	Ritlecitinib (PF-06651600) 200 mg then 50 mg	Ritlecitinib (PF-06651600) 200 mg then 30 mg	Ritlecitinib (PF-06651600) 50 mg	Ritlecitinib (PF-06651600) 30 mg	Ritlecitinib (PF-06651600) 10 mg	Placebo, Ritlecitinib (PF-06651600) 200 mg then 50 mg	Placebo, Ritlecitinib (PF-06651600) 50 mg
Number of subjects analysed	132	130	130	132	63	65	66
Units: Percentage of subjects
number (confidence interval 95%)
Week 4 (n=129, 124, 127, 127, 62, 63, 66)	0.78 (0.00 to 2.29)	0.00 (-99999 to 99999)	0.00 (-99999 to 99999)	0.00 (-99999 to 99999)	0.00 (-99999 to 99999)	0.00 (-99999 to 99999)	0.00 (-99999 to 99999)
Week 8 (n=128, 121, 122, 121, 62, 63, 64)	5.47 (1.53 to 9.41)	4.13 (0.59 to 7.68)	2.46 (0.00 to 5.21)	0.00 (-99999 to 99999)	0.00 (-99999 to 99999)	0.00 (-99999 to 99999)	0.00 (-99999 to 99999)
Week 12 (n= 126, 124, 126, 122, 59, 61, 63)	11.90 (6.25 to 17.56)	8.87 (3.87 to 13.88)	6.35 (2.09 to 10.61)	3.28 (0.12 to 6.44)	3.39 (0.00 to 8.01)	1.64 (0.00 to 4.83)	1.59 (0.00 to 4.67)
Week 18 (n= 121, 121, 122, 117, 60, 60, 62)	19.83 (12.73 to 26.94)	13.22 (7.19 to 19.26)	13.11 (7.12 to 19.10)	9.40 (4.11 to 14.69)	3.33 (0.00 to 7.88)	1.67 (0.00 to 4.91)	1.61 (0.00 to 4.75)
Week 28 (n= 120, 119, 119, 122, 60, 57, 63)	34.17 (25.68 to 42.65)	23.53 (15.91 to 31.15)	26.05 (18.16 to 33.94)	20.49 (13.33 to 27.65)	5.00 (0.00 to 10.51)	1.75 (0.00 to 5.16)	6.35 (0.33 to 12.37)
Week 34 (n= 125, 123, 124, 122, 59, 61,65)	38.40 (29.87 to 46.93)	27.64 (19.74 to 35.55)	33.87 (25.54 to 42.20)	28.69 (20.66 to 36.71)	5.08 (0.00 to 10.69)	19.67 (9.70 to 29.65)	9.23 (2.19 to 16.27)
Week 40 (n= 128, 123, 122, 120, 59, 65, 65)	39.06 (30.61 to 47.51)	33.33 (25.00 to 41.66)	39.34 (30.68 to 48.01)	30.00 (21.80 to 38.20)	6.78 (0.36 to 13.19)	23.08 (12.83 to 33.32)	15.38 (6.61 to 24.16)
Week 48 (n= 129, 122, 125, 122, 61, 65, 64)	39.53 (31.10 to 47.97)	34.43 (26.00 to 42.86)	43.20 (34.52 to 51.88)	31.15 (22.93 to 39.37)	9.84 (2.36 to 17.31)	33.85 (22.34 to 45.35)	18.75 (9.19 to 28.31)

No statistical analyses for this end point

Secondary: Percentage of Subjects With an Absolute SALT Score of Less Than or Equal to 10 at Week 4, 8, 12, 18, 28, 34, 40, and 48

Top of page

End point title

Percentage of Subjects With an Absolute SALT Score of Less Than or Equal to 10 at Week 4, 8, 12, 18, 28, 34, 40, and 48

End point description

SALT is a quantitative assessment of AA severity based on the scalp hair loss. The SALT score can vary from 0 (normal) to 100 (severe), with higher scores representing increased severity of disease. In this endpoint, percentage of subjects with SALT score <=10 were reported. FAS included all subjects who were randomised, regardless of whether they received study medication. Here, “n” signifies subjects evaluable at specific time point. 99999 signifies that 95% CI could not be calculated as there were no subjects with SALT score <=10.

End point type

Secondary

End point timeframe

Week 4, 8, 12, 18, 28, 34, 40, and 48

End point values	Ritlecitinib (PF-06651600) 200 mg then 50 mg	Ritlecitinib (PF-06651600) 200 mg then 30 mg	Ritlecitinib (PF-06651600) 50 mg	Ritlecitinib (PF-06651600) 30 mg	Ritlecitinib (PF-06651600) 10 mg	Placebo, Ritlecitinib (PF-06651600) 200 mg then 50 mg	Placebo, Ritlecitinib (PF-06651600) 50 mg
Number of subjects analysed	132	130	130	132	63	65	66
Units: Percentage of subjects
number (confidence interval 95%)
Week 4 (n=129, 124, 127, 127, 62, 63, 66)	0.78 (0.00 to 2.29)	0.00 (-99999 to 99999)	0.00 (-99999 to 99999)	0.00 (-99999 to 99999)	0.00 (-99999 to 99999)	0.00 (-99999 to 99999)	0.00 (-99999 to 99999)
Week 8 (n=128, 121, 122, 121, 62, 63, 64)	2.34 (0.00 to 4.96)	0.83 (0.00 to 2.44)	0.82 (0.00 to 2.42)	0.00 (-99999 to 99999)	0.00 (-99999 to 99999)	0.00 (-99999 to 99999)	0.00 (-99999 to 99999)
Week 12 (n=126, 124, 126, 122, 59, 61, 63)	6.35 (2.09 to 10.61)	5.65 (1.58 to 9.71)	5.56 (1.56 to 9.56)	0.82 (0.00 to 2.42)	1.69 (0.00 to 4.99)	0.00 (-99999 to 99999)	1.59 (0.00 to 4.67)
Week 18 (n=121, 121, 122, 117, 60, 60, 62)	12.40 (6.52 to 18.27)	7.44 (2.76 to 12.11)	6.56 (2.16 to 10.95)	5.13 (1.13 to 9.12)	1.67 (0.00 to 4.91)	1.67 (0.00 to 4.91)	1.61 (0.00 to 4.75)
Week 28 (n=120, 119, 119, 122, 60, 57, 63)	29.17 (21.03 to 37.30)	16.81 (10.09 to 23.53)	18.49 (11.51 to 25.46)	16.39 (9.82 to 22.96)	3.33 (0.00 to 7.88)	1.75 (0.00 to 5.16)	3.17 (0.00 to 7.50)
Week 34 (n=125, 123, 124, 122, 59, 61, 65)	30.40 (22.34 to 38.46)	18.70 (11.81 to 25.59)	23.39 (15.94 to 30.84)	22.13 (14.76 to 29.50)	3.39 (0.00 to 8.01)	13.11 (4.64 to 21.59)	4.62 (0.00 to 9.72)
Week 40 (n=128, 123, 122, 120, 59, 65, 65)	31.25 (23.22 to 39.28)	25.20 (17.53 to 32.88)	27.05 (19.17 to 34.93)	25.00 (17.25 to 32.75)	3.39 (0.00 to 8.01)	18.46 (9.03 to 27.89)	9.23 (2.19 to 16.27)
Week 48 (n=129, 122, 125, 122, 61, 65, 64)	33.33 (25.20 to 41.47)	27.87 (19.91 to 35.82)	31.20 (23.08 to 39.32)	25.41 (17.68 to 33.14)	6.56 (0.35 to 12.77)	24.62 (14.14 to 35.09)	14.06 (5.55 to 22.58)

No statistical analyses for this end point

Secondary: Percentage of Subjects With at Least 75% Improvement in SALT Score (SALT75) From Baseline at Week 4, 8, 12, 18, 24, 28, 34, 40, and 48

Top of page

End point title

Percentage of Subjects With at Least 75% Improvement in SALT Score (SALT75) From Baseline at Week 4, 8, 12, 18, 24, 28, 34, 40, and 48

End point description

SALT is a quantitative assessment of AA severity based on the scalp hair loss. The SALT score can vary from 0 (normal) to 100 (severe), with higher scores representing increased severity of disease. A SALT 75 response was a 75% or greater reduction from baseline in SALT score. FAS included all subjects who were randomised, regardless of whether they received study medication. Here, “n” signifies subjects evaluable at specific time point. 99999 signifies that CI could not be calculated as there were no subjects with at least 75% improvement in SALT score.

End point type

Secondary

End point timeframe

Week 4, 8, 12, 18, 24, 28, 34, 40, and 48

End point values	Ritlecitinib (PF-06651600) 200 mg then 50 mg	Ritlecitinib (PF-06651600) 200 mg then 30 mg	Ritlecitinib (PF-06651600) 50 mg	Ritlecitinib (PF-06651600) 30 mg	Ritlecitinib (PF-06651600) 10 mg	Placebo, Ritlecitinib (PF-06651600) 200 mg then 50 mg	Placebo, Ritlecitinib (PF-06651600) 50 mg
Number of subjects analysed	132	130	130	132	63	65	66
Units: Percentage of subjects
number (confidence interval 95%)
Week 4 (n=129, 124, 127, 127, 62, 63, 66)	0.78 (0.00 to 2.29)	0.00 (-99999 to 99999)	0.00 (-99999 to 99999)	0.00 (-99999 to 99999)	0.00 (-99999 to 99999)	0.00 (-99999 to 99999)	0.00 (-99999 to 99999)
Week 8 (n=128, 121, 122, 121, 62, 63, 64)	4.69 (1.03 to 8.35)	3.31 (0.12 to 6.49)	1.64 (0.00 to 3.89)	0.83 (0.00 to 2.44)	0.00 (-99999 to 99999)	1.59 (0.00 to 4.67)	0.00 (-99999 to 99999)
Week 12 (n=126, 124, 126, 122, 59, 61, 63)	11.90 (6.25 to 17.56)	8.06 (3.27 to 12.86)	6.35 (2.09 to 10.61)	2.46 (0.00 to 5.21)	1.69 (0.00 to 4.99)	1.64 (0.00 to 4.83)	1.59 (0.00 to 4.67)
Week 18 (n=121, 121, 122, 117, 60, 60, 62)	20.66 (13.45 to 27.88)	14.88 (8.54 to 21.22)	9.84 (4.55 to 15.12)	11.11 (5.42 to 16.81)	1.67 (0.00 to 4.91)	1.67 (0.00 to 4.91)	1.61 (0.00 to 4.75)
Week 24 (n=124, 121, 124, 119, 59, 65, 65)	31.45 (23.28 to 39.62)	20.66 (13.45 to 27.88)	22.58 (15.22 to 29.94)	13.45 (7.32 to 19.57)	1.69 (0.00 to 4.99)	1.54 (0.00 to 4.53)	3.08 (0.00 to 7.28)
Week 28 (n=120, 119, 119, 122, 60, 57, 63)	34.17 (25.68 to 42.65)	24.37 (16.66 to 32.08)	27.73 (19.69 to 35.77)	21.31 (14.04 to 28.58)	5.00 (0.00 to 10.51)	3.51 (0.00 to 8.29)	4.76 (0.00 to 10.02)
Week 34 (n=125, 123, 124, 122, 59, 61, 65)	38.40 (29.87 to 46.93)	32.52 (24.24 to 40.80)	38.71 (30.14 to 47.28)	28.69 (20.66 to 36.71)	5.08 (0.00 to 10.69)	21.31 (11.03 to 31.59)	10.77 (3.23 to 18.31)
Week 40 (n=128, 123, 122, 120, 59, 65, 65)	39.84 (31.36 to 48.33)	34.96 (26.53 to 43.39)	42.62 (33.85 to 51.40)	30.00 (21.80 to 38.20)	5.08 (0.00 to 10.69)	23.08 (12.83 to 33.32)	15.38 (6.61 to 24.16)
Week 48 (n=129, 122, 125, 122, 61, 65, 64)	39.53 (31.10 to 47.97)	36.07 (27.54 to 44.59)	46.40 (37.66 to 55.14)	31.15 (22.93 to 39.37)	9.84 (2.36 to 17.31)	32.31 (20.94 to 43.68)	21.88 (11.75 to 32.00)

No statistical analyses for this end point

Secondary: Change From Baseline in SALT Score at Week 4, 8, 12, 18, and 24

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End point title

Change From Baseline in SALT Score at Week 4, 8, 12, 18, and 24 ^[7]

End point description

SALT is a quantitative assessment of AA severity based on the scalp hair loss. The SALT score can vary from 0 (normal) to 100 (severe), with higher scores representing increased severity of disease. Baseline was defined as pre-dose on Day 1. FAS included all subjects who were randomised, regardless of whether they received study medication. Here, “n” signifies subjects evaluable at specific time point.

End point type

Secondary

End point timeframe

Baseline (Day 1), Week 4, 8, 12, 18, and 24

Notes
[7] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: Statistics were planned only for the arms specified

End point values	Ritlecitinib (PF-06651600) 200 mg then 50 mg	Ritlecitinib (PF-06651600) 200 mg then 30 mg	Ritlecitinib (PF-06651600) 50 mg	Ritlecitinib (PF-06651600) 30 mg	Ritlecitinib (PF-06651600) 10 mg	Placebo
Number of subjects analysed	132	130	130	132	63	131
Units: Units on a scale
least squares mean (confidence interval 95%)
Change at Week 4 (n= 127, 121, 124, 124, 58, 127)	-2.3 (-3.64 to -1.01)	-2.7 (-4.03 to -1.35)	-1.8 (-3.16 to -0.50)	-1.3 (-2.60 to 0.06)	-1.3 (-3.19 to 0.69)	-0.9 (-2.17 to 0.46)
Change at Week 8 (n= 124, 119, 117, 115, 57, 124)	-12.8 (-15.56 to -10.05)	-12.5 (-15.28 to -9.71)	-6.3 (-9.10 to -3.50)	-5.0 (-7.82 to -2.21)	-1.7 (-5.74 to 2.34)	-1.2 (-3.99 to 1.50)
Change at Week 12 (n= 121, 120, 121, 115, 56, 122)	-22.7 (-26.47 to -18.97)	-20.1 (-23.87 to -16.30)	-12.4 (-16.18 to -8.61)	-10.3 (-14.11 to -6.47)	-3.5 (-9.00 to 1.98)	-2.4 (-6.12 to 1.35)
Change at Week 18 (n= 117, 119, 117, 112, 56, 119)	-31.2 (-35.63 to -26.83)	-25.0 (-29.41 to -20.55)	-22.5 (-26.94 to -18.07)	-17.5 (-22.02 to -13.02)	-2.4 (-8.87 to 3.97)	-3.9 (-8.31 to 0.43)
Change at Week 24 (n= 118, 119, 119, 114, 55, 125)	-36.5 (-41.54 to -31.53)	-29.2 (-34.21 to -24.15)	-33.3 (-38.33 to -28.25)	-23.6 (-28.72 to -18.45)	-4.2 (-11.50 to 3.13)	-5.1 (-10.03 to -0.13)

No statistical analyses for this end point

Secondary: Change From Baseline in SALT Score at Week 28, 34, 40, and 48

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End point title

Change From Baseline in SALT Score at Week 28, 34, 40, and 48

End point description

SALT is a quantitative assessment of AA severity based on the scalp hair loss. The SALT score can vary from 0 (normal) to 100 (severe), with higher scores representing increased severity of disease. Baseline was defined as pre-dose on Day 1. FAS included all subjects who were randomised, regardless of whether they received study medication. Here, “n” signifies subjects evaluable at specific time point.

End point type

Secondary

End point timeframe

Baseline (Day 1), Week 28, 34, 40, and 48

End point values	Ritlecitinib (PF-06651600) 200 mg then 50 mg	Ritlecitinib (PF-06651600) 200 mg then 30 mg	Ritlecitinib (PF-06651600) 50 mg	Ritlecitinib (PF-06651600) 30 mg	Ritlecitinib (PF-06651600) 10 mg	Placebo, Ritlecitinib (PF-06651600) 200 mg then 50 mg	Placebo, Ritlecitinib (PF-06651600) 50 mg
Number of subjects analysed	132	130	130	132	63	65	66
Units: Units on a scale
arithmetic mean (standard deviation)
Baseline (n=132,130,130,132,63,65,66)	90.3 ( 15.05 )	90.5 ( 14.28 )	90.3 ( 14.69 )	90.0 ( 15.07 )	88.3 ( 16.87 )	94.4 ( 9.31 )	91.5 ( 13.22 )
Change at Week 28 (n=109,113,111,115,55,54,59)	-44.1 ( 36.35 )	-33.3 ( 33.85 )	-36.1 ( 33.42 )	-29.2 ( 32.56 )	-5.6 ( 23.15 )	-11.5 ( 22.75 )	-5.0 ( 18.51 )
Change at Week 34 (n=113,118,117,115,54,59,61)	-48.2 ( 36.16 )	-35.5 ( 35.50 )	-43.6 ( 34.35 )	-33.5 ( 35.01 )	-7.6 ( 23.61 )	-27.5 ( 32.78 )	-12.6 ( 24.47 )
Change at Week 40 (n= 116,118,113,109,54,61, 60)	-49.3 ( 36.11 )	-38.5 ( 37.14 )	-46.9 ( 35.68 )	-35.9 ( 35.90 )	-11.4 ( 26.01 )	-36.5 ( 34.56 )	-23.0 ( 30.64 )
Change at Week 48 (n=114,112,16,107,53,60,59)	-49.4 ( 36.09 )	-40.5 ( 37.27 )	-48.9 ( 36.63 )	-40.1 ( 35.87 )	-13.3 ( 30.13 )	-46.3 ( 35.51 )	-32.2 ( 32.39 )

No statistical analyses for this end point

Secondary: Percentage of Subjects With at Least a 2 Grade Improvement From Baseline or a Score of 3 in Eyebrow Assessment (EBA) Score (Among Subjects Without Normal EBA at Baseline) at Week 4, 8, 12, 18, 24, 28, 34, 40, and 48

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End point title

Percentage of Subjects With at Least a 2 Grade Improvement From Baseline or a Score of 3 in Eyebrow Assessment (EBA) Score (Among Subjects Without Normal EBA at Baseline) at Week 4, 8, 12, 18, 24, 28, 34, 40, and 48

End point description

EBA is a numeric rating scale developed to characterize eyebrow hair loss. The numeric rating scale ranges from 0 (none) to 3 (normal), where, 0= no eyebrow, 1=minimal eyebrow, 2=moderate eyebrow and 3= normal eyebrow, where higher scores represent less hair loss of eyebrows. FAS included all subjects who were randomised, regardless of whether they received study medication. Here, “n” signifies subjects evaluable at specific time point. 99999 signifies that 95% CI could not be calculated as there were no subjects with at least a 2-grade improvement or a score of 3 in EBA.

End point type

Secondary

End point timeframe

Week 4, 8, 12, 18, 24, 28, 34, 40, and 48

End point values	Ritlecitinib (PF-06651600) 200 mg then 50 mg	Ritlecitinib (PF-06651600) 200 mg then 30 mg	Ritlecitinib (PF-06651600) 50 mg	Ritlecitinib (PF-06651600) 30 mg	Ritlecitinib (PF-06651600) 10 mg	Placebo, Ritlecitinib (PF-06651600) 200 mg then 50 mg	Placebo, Ritlecitinib (PF-06651600) 50 mg
Number of subjects analysed	132	130	130	132	63	65	66
Units: Percentage of subjects
number (confidence interval 95%)
Week 4 (n= 107, 104, 104, 107, 51, 53, 52)	1.87 (0.00 to 4.44)	2.88 (0.00 to 6.10)	0.00 (-99999 to 99999)	0.93 (0.00 to 2.76)	1.96 (0.00 to 5.77)	0.00 (-99999 to 99999)	3.85 (0.00 to 9.07)
Week 8 (n= 107, 101, 101, 102, 52, 53, 51)	10.28 (4.53 to 16.03)	13.86 (7.12 to 20.60)	2.97 (0.00 to 6.28)	4.90 (0.71 to 9.09)	3.85 (0.00 to 9.07)	3.77 (0.00 to 8.90)	0.00 (-99999 to 99999)
Week 12 (n= 105, 103, 104, 105, 48, 51, 50)	22.86 (14.83 to 30.89)	17.48 (10.14 to 24.81)	9.62 (3.95 to 15.28)	7.62 (2.54 to 12.69)	6.25 (0.00 to 13.10)	0.00 (-99999 to 99999)	4.00 (0.00 to 9.43)
Week 18 (n= 101, 101, 101, 101, 49, 50, 49)	29.70 (20.79 to 38.61)	18.81 (11.19 to 26.43)	17.82 (10.36 to 25.29)	12.87 (6.34 to 19.40)	10.20 (1.73 to 18.68)	0.00 (-99999 to 99999)	8.16 (0.50 to 15.83)
Week 24 (n= 103, 102, 100, 102, 48, 55, 52)	33.98 (24.83 to 43.13)	25.49 (17.03 to 33.95)	29.00 (20.11 to 37.89)	16.67 (9.43 to 23.90)	8.33 (0.51 to 16.15)	3.64 (0.00 to 8.58)	5.77 (0.00 to 12.11)
Week 28 (n= 100, 100, 99, 105, 49, 48, 50)	37.00 (27.54 to 46.46)	29.00 (20.11 to 37.89)	27.27 (18.50 to 36.05)	24.76 (16.51 to 33.02)	12.24 (3.07 to 21.42)	4.17 (0.00 to 9.82)	6.00 (0.00 to 12.58)
Week 34 (n= 104, 104, 101, 105, 48, 51, 52)	41.35 (31.88 to 50.81)	30.77 (21.90 to 39.64)	34.65 (25.37 to 43.93)	31.43 (22.55 to 40.31)	14.58 (4.60 to 24.57)	15.69 (5.71 to 25.67)	13.46 (4.18 to 22.74)
Week 40 (n= 106, 102, 100, 104, 50, 55, 52)	43.40 (33.96 to 52.83)	30.39 (21.47 to 39.32)	39.00 (29.44 to 48.56)	33.65 (24.57 to 42.74)	16.00 (5.84 to 26.16)	20.00 (9.43 to 30.57)	26.92 (14.87 to 38.98)
Week 48 (n= 107, 101, 101, 105, 50, 55, 51)	42.99 (33.61 to 52.37)	32.67 (23.53 to 41.82)	43.56 (33.89 to 53.23)	33.33 (24.32 to 42.35)	16.00 (5.84 to 26.16)	30.91 (18.70 to 43.12)	31.37 (18.64 to 44.11)

No statistical analyses for this end point

Secondary: Percentage of Subjects With at Least a 2 Grade Improvement From Baseline or a Score of 3 in Eyelash Assessment (ELA) Score (Among Subjects Without Normal ELA at Baseline) at Week 4, 8, 12, 18, 24, 28, 34, 40, and 48

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End point title

Percentage of Subjects With at Least a 2 Grade Improvement From Baseline or a Score of 3 in Eyelash Assessment (ELA) Score (Among Subjects Without Normal ELA at Baseline) at Week 4, 8, 12, 18, 24, 28, 34, 40, and 48

End point description

ELA is a numeric rating scale developed to characterize eyelash hair loss. The numeric rating scale ranges from 0 (none) to 3 (normal), where, 0=no eyelash, 1=minimal eyelash, 2=moderate eyelash and 3=normal eyelash, where higher scores represent less hair loss of eyelash. FAS included all subjects who were randomised, regardless of whether they received study medication. Here, “n” signifies subjects evaluable at specific time point. 99999 signifies that 95% CI could not be calculated as there were no subjects with at least a 2-grade improvement or a score of 3 in ELA.

End point type

Secondary

End point timeframe

Week 4, 8, 12, 18, 24, 28, 34, 40, and 48

End point values	Ritlecitinib (PF-06651600) 200 mg then 50 mg	Ritlecitinib (PF-06651600) 200 mg then 30 mg	Ritlecitinib (PF-06651600) 50 mg	Ritlecitinib (PF-06651600) 30 mg	Ritlecitinib (PF-06651600) 10 mg	Placebo, Ritlecitinib (PF-06651600) 200 mg then 50 mg	Placebo, Ritlecitinib (PF-06651600) 50 mg
Number of subjects analysed	132	130	130	132	63	65	66
Units: Percentage of subjects
number (confidence interval 95%)
Week 4 (n= 99, 93, 93, 97, 44, 49, 46)	3.03 (0.00 to 6.41)	1.08 (0.00 to 3.17)	5.38 (0.79 to 9.96)	1.03 (0.00 to 3.04)	4.55 (0.00 to 10.70)	2.04 (0.00 to 6.00)	0.00 (-99999 to 99999)
Week 8 (n= 99, 90, 91, 92, 45, 49, 45)	9.09 (3.43 to 14.75)	4.44 (0.19 to 8.70)	8.79 (2.97 to 14.61)	4.35 (0.18 to 8.51)	2.22 (0.00 to 6.53)	2.04 (0.00 to 6.00)	0.00 (-99999 to 99999)
Week 12 (n= 97, 91, 94, 95, 42, 47, 44)	16.49 (9.11 to 23.88)	10.99 (4.56 to 17.41)	11.70 (5.20 to 18.20)	7.37 (2.11 to 12.62)	4.76 (0.00 to 11.20)	0.00 (-99999 to 99999)	0.00 (-99999 to 99999)
Week 18 (n= 94, 90, 90, 91, 43, 46, 43)	25.53 (16.72 to 34.35)	20.00 (11.74 to 28.26)	20.00 (11.74 to 28.26)	12.09 (5.39 to 18.79)	6.98 (0.00 to 14.59)	2.17 (0.00 to 6.39)	0.00 (-99999 to 99999)
Week 24 (n= 96, 89, 90, 92, 41, 51, 46)	30.21 (21.02 to 39.39)	21.35 (12.84 to 29.86)	28.89 (19.52 to 38.25)	26.09 (17.11 to 35.06)	4.88 (0.00 to 11.47)	9.80 (1.64 to 17.97)	0.00 (-99999 to 99999)
Week 28 (n= 94, 88, 87, 95, 42, 44, 44)	29.79 (20.54 to 39.03)	25.00 (15.95 to 34.05)	36.78 (26.65 to 46.91)	24.21 (15.60 to 32.82)	4.76 (0.00 to 11.20)	6.82 (0.00 to 14.27)	4.55 (0.00 to 10.70)
Week 34 (n= 97, 91, 90, 95, 41, 48, 46)	34.02 (24.59 to 43.45)	26.37 (17.32 to 35.43)	38.89 (28.82 to 48.96)	26.32 (17.46 to 35.17)	7.32 (0.00 to 15.29)	16.67 (6.12 to 27.21)	6.52 (0.00 to 13.66)
Week 40 (n= 98, 89, 89, 94, 43, 51, 46)	39.80 (30.10 to 49.49)	28.09 (18.75 to 37.43)	38.20 (28.11 to 48.30)	27.66 (18.62 to 36.70)	18.60 (6.97 to 30.24)	25.49 (13.53 to 37.45)	17.39 (6.44 to 28.34)
Week 48 (n= 99, 88, 90, 95, 43, 51, 45)	38.38 (28.80 to 47.96)	29.55 (20.01 to 39.08)	40.00 (29.88 to 50.12)	30.53 (21.27 to 39.79)	20.93 (8.77 to 33.09)	37.25 (23.99 to 50.52)	35.56 (21.57 to 49.54)

No statistical analyses for this end point

Secondary: Percentage of Subjects With Patient Global Impression of Change (PGI-C) Score of Moderately Improved or Greatly Improved at Week 4, 8, 12, 18, 24, 34, 40, and 48

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End point title

Percentage of Subjects With Patient Global Impression of Change (PGI-C) Score of Moderately Improved or Greatly Improved at Week 4, 8, 12, 18, 24, 34, 40, and 48

End point description

End point type

Secondary

End point timeframe

Week 4, 8, 12, 18, 24, 34, 40, and 48

End point values	Ritlecitinib (PF-06651600) 200 mg then 50 mg	Ritlecitinib (PF-06651600) 200 mg then 30 mg	Ritlecitinib (PF-06651600) 50 mg	Ritlecitinib (PF-06651600) 30 mg	Ritlecitinib (PF-06651600) 10 mg	Placebo, Ritlecitinib (PF-06651600) 200 mg then 50 mg	Placebo, Ritlecitinib (PF-06651600) 50 mg
Number of subjects analysed	132	130	130	132	63	65	66
Units: Percentage of subjects
number (not applicable)
W4:greatly improved(n=129,124,127,127,62,63,65)	5.43	4.03	2.36	0	0	0	0
W4:moderatelyimproved(n=129,124,127,127,62,63,65)	3.10	11.29	4.72	7.87	1.61	1.59	1.54
W8:greatly improved(n=128,121,123,121,61,63,64)	17.19	14.88	8.13	4.13	1.64	1.59	1.56
W8:moderatelyimproved(n=128,121,123,121,61,63,64)	13.28	21.49	7.32	16.53	4.92	3.17	4.69
W12:greatly improved(n=127,124,125,123,59,60,63)	22.83	22.58	13.60	12.20	3.39	0	3.17
W12:moderatelyimproved(n=127,124,125,123,59,60,63)	18.11	19.35	14.40	14.63	3.39	10.00	7.94
W18:greatly improved(n=123,121,122,119,61,60,61)	27.64	22.31	18.03	19.33	1.64	1.67	3.28
W18:moderatelyimproved(n=123,121,22,119,61,60,61)	23.58	23.97	27.87	15.13	6.56	10.00	4.92
W24:greatly improved(n=126,121,125,121,60,65,65)	36.51	26.45	28.80	28.93	1.67	1.54	4.62
W24:moderatelyimproved(n=126,121,125,121,60,65,65)	16.67	20.66	20.80	13.22	10.00	9.23	3.08
W34:greatly improved(n=125,121,123,122,59,59,64)	43.20	35.54	37.40	31.97	5.08	18.64	7.81
W34:moderatelyimproved(n=125,121,123,122,59,59,64)	12.00	17.36	15.45	15.57	6.78	22.03	20.31
W40:greatly improved(n=128,124,122,120,59,65,65)	43.75	34.68	37.70	30.00	6.78	30.77	13.85
W40:moderatelyimproved(n=128,124,122,120,59,65,65)	15.63	14.52	14.75	19.17	11.86	24.62	15.38
W48:greatly improved(n=129,123,125,122,61,64,64)	44.96	33.33	42.40	31.15	6.56	42.19	26.56
W48:moderatelyimproved(n=129,123,125,122,61,64,64)	13.18	18.70	13.60	18.03	9.84	17.19	17.19

No statistical analyses for this end point

Secondary: Change From Baseline in Alopecia Areata Patient Priority Outcomes (AAPPO) Domain Scores at Week 4, 8, 12, 18, and 24: Emotional Symptoms (ES) and Activity Limitations (AL)

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End point title

Change From Baseline in Alopecia Areata Patient Priority Outcomes (AAPPO) Domain Scores at Week 4, 8, 12, 18, and 24: Emotional Symptoms (ES) and Activity Limitations (AL) ^[8]

End point description

AAPPO scale:11-item self-administered questionnaire that measured hair loss, emotional symptoms, and activity limitations over past week. Items 1-4 assessed current hair loss, eyebrow loss, eyelash loss and body hair loss and analysed separately on scale 0-4(0=no hair loss and 4=complete hair loss). Items 5-8 assessed emotional symptoms. Response were scored from 0=never to 4=always. Items 9-11 assessed activity limitations. Response were scored from 0=not at all to 4=completely. Change from baseline in AAPPO emotional symptoms sub score was calculated as mean of items 5-8(0=never to 4=always),higher scores:more emotional symptoms; Change from baseline in AAPPO activity limitations sub score was calculated as mean of items 9-11(0=not at all to 4=completely),higher scores:more activity limitations. Baseline was defined as pre-dose on Day 1. FAS:subjects randomised, regardless of whether received study medication. Here, “n” signifies subjects evaluable at specific time point.

End point type

Secondary

End point timeframe

Baseline (Day 1), Week 4, 8, 12, 18, and 24

Notes
[8] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: Statistics were planned only for the arms specified

End point values	Ritlecitinib (PF-06651600) 200 mg then 50 mg	Ritlecitinib (PF-06651600) 200 mg then 30 mg	Ritlecitinib (PF-06651600) 50 mg	Ritlecitinib (PF-06651600) 30 mg	Ritlecitinib (PF-06651600) 10 mg	Placebo
Number of subjects analysed	132	130	130	132	63	131
Units: Units on a scale
least squares mean (confidence interval 95%)
Change at Week 4: ES (n=127,121,123,122,58,124)	-0.37 (-0.49 to -0.25)	-0.22 (-0.34 to -0.10)	-0.33 (-0.45 to -0.21)	-0.31 (-0.44 to -0.19)	-0.29 (-0.47 to -0.11)	-0.28 (-0.40 to -0.16)
Change at Week 8: ES (n= 122,119,117,115,56,123)	-0.50 (-0.62 to -0.38)	-0.46 (-0.58 to -0.34)	-0.50 (-0.62 to -0.37)	-0.36 (-0.48 to -0.23)	-0.45 (-0.62 to -0.27)	-0.39 (-0.51 to -0.27)
Change at Week 12: ES (n=122,120,119,116,56,121)	-0.53 (-0.66 to -0.40)	-0.54 (-0.67 to -0.41)	-0.48 (-0.61 to -0.34)	-0.49 (-0.63 to -0.36)	-0.50 (-0.69 to -0.31)	-0.36 (-0.49 to -0.23)
Change at Week 18: ES (n=118,119,116,114,57,118)	-0.64 (-0.78 to -0.51)	-0.57 (-0.71 to -0.44)	-0.60 (-0.74 to -0.46)	-0.54 (-0.68 to -0.40)	-0.44 (-0.63 to -0.24)	-0.43 (-0.56 to -0.29)
Change at Week 24: ES (n=120,118,119,116,55,125)	-0.61 (-0.75 to -0.46)	-0.61 (-0.76 to -0.47)	-0.69 (-0.83 to -0.54)	-0.58 (-0.72 to -0.43)	-0.49 (-0.70 to -0.28)	-0.47 (-0.61 to -0.33)
Change at Week 4: AL (n=127,121,123,122,58,125)	-0.19 (-0.29 to -0.08)	-0.04 (-0.15 to 0.07)	-0.16 (-0.27 to -0.05)	-0.13 (-0.24 to -0.02)	-0.20 (-0.36 to -0.04)	-0.18 (-0.29 to -0.07)
Change at Week 8: AL (n=122,119,117,115,56,123)	-0.24 (-0.34 to -0.13)	-0.25 (-0.36 to -0.14)	-0.18 (-0.29 to -0.07)	-0.09 (-0.20 to 0.02)	-0.21 (-0.37 to -0.05)	-0.20 (-0.31 to -0.09)
Change at Week 12: AL (n=122,120,119,116,56,121)	-0.27 (-0.38 to -0.17)	-0.22 (-0.32 to -0.11)	-0.21 (-0.32 to -0.11)	-0.24 (-0.35 to -0.13)	-0.27 (-0.42 to -0.11)	-0.21 (-0.32 to -0.11)
Change at Week 18: AL (n=118,119,116,114,57,118)	-0.35 (-0.45 to -0.24)	-0.26 (-0.36 to -0.15)	-0.23 (-0.34 to -0.12)	-0.23 (-0.34 to -0.12)	-0.28 (-0.44 to -0.13)	-0.26 (-0.36 to -0.15)
Change at Week 24: AL (n=120,118,119,115,55,125)	-0.30 (-0.40 to -0.20)	-0.30 (-0.40 to -0.20)	-0.31 (-0.41 to -0.21)	-0.28 (-0.38 to -0.18)	-0.31 (-0.45 to -0.16)	-0.29 (-0.39 to -0.19)

No statistical analyses for this end point

Secondary: Change From Baseline in Alopecia Areata Patient Priority Outcomes (AAPPO) Domain Scores at Week 34, 40, and 48: Emotional Symptoms (ES) and Activity Limitations (AL)

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End point title

Change From Baseline in Alopecia Areata Patient Priority Outcomes (AAPPO) Domain Scores at Week 34, 40, and 48: Emotional Symptoms (ES) and Activity Limitations (AL)

End point description

End point type

Secondary

End point timeframe

Baseline (Day 1), Week 34, 40, and 48

End point values	Ritlecitinib (PF-06651600) 200 mg then 50 mg	Ritlecitinib (PF-06651600) 200 mg then 30 mg	Ritlecitinib (PF-06651600) 50 mg	Ritlecitinib (PF-06651600) 30 mg	Ritlecitinib (PF-06651600) 10 mg	Placebo, Ritlecitinib (PF-06651600) 200 mg then 50 mg	Placebo, Ritlecitinib (PF-06651600) 50 mg
Number of subjects analysed	132	130	130	132	63	65	66
Units: Units on a scale
arithmetic mean (standard deviation)
Change at Week 34:ES (n=112,116,114,113,53,57,60)	-0.78 ( 0.95 )	-0.72 ( 1.00 )	-0.72 ( 0.93 )	-0.71 ( 1.06 )	-0.62 ( 0.83 )	-0.77 ( 0.92 )	-0.64 ( 0.91 )
Change at Week 40:ES (n= 115,119,112,110,54,62,60)	-0.84 ( 1.04 )	-0.80 ( 1.02 )	-0.81 ( 0.98 )	-0.83 ( 1.11 )	-0.62 ( 0.96 )	-0.83 ( 1.01 )	-0.66 ( 1.00 )
Change at Week 48:ES (n=114,112,115,107,53,60,59)	-0.96 ( 0.99 )	-0.84 ( 1.07 )	-0.85 ( 1.04 )	-0.72 ( 1.15 )	-0.50 ( 0.89 )	-0.98 ( 1.05 )	-0.68 ( 1.03 )
Change at Week 34:AL (n=112,116,114,115,53,57,60)	-0.37 ( 0.71 )	-0.31 ( 0.78 )	-0.26 ( 0.73 )	-0.37 ( 0.78 )	-0.33 ( 0.76 )	-0.36 ( 0.59 )	-0.42 ( 0.78 )
Change at Week 40:AL (n=115,119,112,110,54,62,60)	-0.44 ( 0.76 )	-0.33 ( 0.84 )	-0.25 ( 0.68 )	-0.42 ( 0.81 )	-0.23 ( 0.71 )	-0.40 ( 0.66 )	-0.39 ( 0.84 )
Change at Week 48:AL (n=114,112,115,107,53,60,59)	-0.43 ( 0.79 )	-0.39 ( 0.84 )	-0.29 ( 0.76 )	-0.36 ( 0.85 )	-0.26 ( 0.90 )	-0.44 ( 0.71 )	-0.36 ( 0.81 )

No statistical analyses for this end point

Secondary: Percentage of Subjects With Improvement From Baseline on Alopecia Areata Patient Priority Outcomes (AAPPO) Items 1-4 at Week 4, 8, 12, 18, 24, 34, 40, and 48

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End point title

Percentage of Subjects With Improvement From Baseline on Alopecia Areata Patient Priority Outcomes (AAPPO) Items 1-4 at Week 4, 8, 12, 18, 24, 34, 40, and 48

End point description

AAPPO scale is a 11-item self-administered questionnaire that measured hair loss, emotional symptoms, and activity limitations over the past week. Items 1-4 were to assess the current hair loss (CHL), eyebrow loss, eyelash loss and body hair loss and were analysed separately on a scale of 0-4, with 0 =‘no hair loss’ and 4=‘complete hair loss’, where higher scores indicated more hair loss. FAS included all subjects who were randomised, regardless of whether they received study medication. Here, “n” signifies subjects evaluable at specific time point. W=week

End point type

Secondary

End point timeframe

Week 4, 8, 12, 18, 24, 34, 40, and 48

End point values	Ritlecitinib (PF-06651600) 200 mg then 50 mg	Ritlecitinib (PF-06651600) 200 mg then 30 mg	Ritlecitinib (PF-06651600) 50 mg	Ritlecitinib (PF-06651600) 30 mg	Ritlecitinib (PF-06651600) 10 mg	Placebo, Ritlecitinib (PF-06651600) 200 mg then 50 mg	Placebo, Ritlecitinib (PF-06651600) 50 mg
Number of subjects analysed	132	130	130	132	63	65	66
Units: Percentage of subjects
number (confidence interval 95%)
W4:CHL on scalp (n=123,123,120,124,59,63,63)	11.38 (5.77 to 16.99)	9.76 (4.51 to 15.00)	4.17 (0.59 to 7.74)	4.84 (1.06 to 8.62)	1.69 (0.00 to 4.99)	6.35 (0.33 to 12.37)	3.17 (0.00 to 7.50)
W8:CHL on scalp (n=122,120,116,118,58,63,62)	11.48 (5.82 to 17.13)	16.67 (10.00 to 23.33)	7.76 (2.89 to 12.63)	8.47 (3.45 to 13.50)	1.72 (0.00 to 5.07)	4.76 (0.00 to 10.02)	8.06 (1.29 to 14.84)
W12: CHL on scalp (n=121,123,118,120,56,60,61)	20.66 (13.45 to 27.88)	19.51 (12.51 to 26.52)	12.71 (6.70 to 18.72)	13.33 (7.25 to 19.42)	3.57 (0.00 to 8.43)	6.67 (0.35 to 12.98)	4.92 (0.00 to 10.34)
W18: CHL on scalp (n=117,120,116,116,58,60,59)	27.35 (19.27 to 35.43)	25.00 (17.25 to 32.75)	13.79 (7.52 to 20.07)	16.38 (9.64 to 23.11)	8.62 (1.40 to 15.84)	8.33 (1.34 to 15.33)	6.78 (0.36 to 13.19)
W24: CHL on scalp (n=120,120,118,118,57,65,63)	35.83 (27.25 to 44.41)	30.00 (21.80 to 38.20)	26.27 (18.33 to 34.21)	24.58 (16.81 to 32.34)	5.26 (0.00 to 11.06)	10.77 (3.23 to 18.31)	6.35 (0.33 to 12.37)
W34: CHL on scalp (n=119,119,116,119,56,59,62)	42.02 (33.15 to 50.89)	34.45 (25.92 to 42.99)	29.31 (21.03 to 37.59)	30.25 (22.00 to 38.51)	7.14 (0.40 to 13.89)	22.03 (11.46 to 32.61)	11.29 (3.41 to 19.17)
W40: CHL on scalp (n=122,123,115,117,56,65,63)	41.80 (33.05 to 50.56)	36.59 (28.07 to 45.10)	35.65 (26.90 to 44.41)	32.48 (23.99 to 40.96)	8.93 (1.46 to 16.40)	29.23 (18.17 to 40.29)	19.05 (9.35 to 28.74)
W48: CHL on scalp (n=123,122,118,119,58,65,62)	42.28 (33.55 to 51.01)	33.61 (25.22 to 41.99)	42.37 (33.46 to 51.29)	32.77 (24.34 to 41.21)	8.62 (1.40 to 15.84)	33.85 (22.34 to 45.35)	20.97 (10.83 to 31.10)
W4: CHL on eyebrows (n=99,98,94,96,46,48,46)	9.09 (3.43 to 14.75)	7.14 (2.04 to 12.24)	7.45 (2.14 to 12.75)	4.17 (0.17 to 8.16)	4.35 (0.00 to 10.24)	6.25 (0.00 to 13.10)	8.70 (0.55 to 16.84)
W8:CHL on eyebrows (n=98,95,92,91,46,48,45)	17.35 (9.85 to 24.84)	21.05 (12.85 to 29.25)	11.96 (5.33 to 18.59)	10.99 (4.56 to 17.41)	6.52 (0.00 to 13.66)	4.17 (0.00 to 9.82)	11.11 (1.93 to 20.29)
W12: CHL on eyebrows (n=97,97,94,94,43,45,44)	27.84 (18.92 to 36.75)	27.84 (18.92 to 36.75)	18.09 (10.30 to 25.87)	15.96 (8.55 to 23.36)	11.63 (2.05 to 21.21)	11.11 (1.93 to 20.29)	6.82 (0.00 to 14.27)
W18: CHL on eyebrows (n=94,95,91,91,45,45,42)	27.66 (18.62 to 36.70)	33.68 (24.18 to 43.19)	27.47 (18.30 to 36.64)	19.78 (11.60 to 27.96)	13.33 (3.40 to 23.27)	11.11 (1.93 to 20.29)	7.14 (0.00 to 14.93)
W24: CHL on eyebrows (n=96,94,92,92,44,50,46)	32.29 (22.94 to 41.65)	32.98 (23.47 to 42.48)	30.43 (21.03 to 39.84)	29.35 (20.04 to 38.65)	6.82 (0.00 to 14.27)	12.00 (2.99 to 21.01)	10.87 (1.87 to 19.86)
W34: CHL on eyebrows (n=96,93,90,94,43,44,45)	42.71 (32.81 to 52.60)	38.71 (28.81 to 48.61)	38.89 (28.82 to 48.96)	31.91 (22.49 to 41.34)	16.28 (5.24 to 27.31)	22.73 (10.34 to 35.11)	13.33 (3.40 to 23.27)
W40: CHL on eyebrows (n=97,95,90,92,45,50,46)	42.27 (32.44 to 52.10)	36.84 (27.14 to 46.54)	38.89 (28.82 to 48.96)	38.04 (28.12 to 47.96)	20.00 (8.31 to 31.69)	32.00 (19.07 to 44.93)	23.91 (11.59 to 36.24)
W48: CHL on eyebrows (n=98,94,91,94,45,50,45)	44.90 (35.05 to 54.75)	34.04 (24.46 to 43.62)	43.96 (33.76 to 54.15)	39.36 (29.49 to 49.24)	15.56 (4.97 to 26.14)	38.00 (24.55 to 51.45)	33.33 (19.56 to 47.11)
W4: CHL on eyelashes (n=87,87,79,86,38,45,38)	11.49 (4.79 to 18.20)	4.60 (0.20 to 9.00)	7.59 (1.75 to 13.44)	9.30 (3.16 to 15.44)	5.26 (0.00 to 12.36)	8.89 (0.57 to 17.20)	5.26 (0.00 to 12.36)
W8: CHL on eyelashes (n=86,85,78,81,38,45,37)	17.44 (9.42 to 25.46)	17.65 (9.54 to 25.75)	12.82 (5.40 to 20.24)	14.81 (7.08 to 22.55)	7.89 (0.00 to 16.47)	6.67 (0.00 to 13.95)	8.11 (0.00 to 16.90)
W12: CHL on eyelashes (n=85,86,79,84,36,41,36)	29.41 (19.73 to 39.10)	26.74 (17.39 to 36.10)	16.46 (8.28 to 24.63)	19.05 (10.65 to 27.44)	8.33 (0.00 to 17.36)	14.63 (3.82 to 25.45)	8.33 (0.00 to 17.36)
W18:CHL on eyelashes (n=83,85,76,81,38,41,34)	31.33 (21.35 to 41.30)	37.65 (27.35 to 47.95)	21.05 (11.89 to 30.22)	22.22 (13.17 to 31.28)	7.89 (0.00 to 16.47)	12.20 (2.18 to 22.21)	2.94 (0.00 to 8.62)
W24: CHL on eyelashes (n=86,83,77,82,36,46,38)	31.40 (21.59 to 41.20)	42.17 (31.54 to 52.79)	31.17 (20.82 to 41.51)	28.05 (18.33 to 37.77)	8.33 (0.00 to 17.36)	13.04 (3.31 to 22.78)	2.63 (0.00 to 7.72)
W34: CHL on eyelashes (n=85,82,75,84,35,42,37)	40.00 (29.59 to 50.41)	36.59 (26.16 to 47.01)	37.33 (26.39 to 48.28)	34.52 (24.36 to 44.69)	11.43 (0.89 to 21.97)	21.43 (9.02 to 33.84)	13.51 (2.50 to 24.53)
W40: CHL on eyelashes (n=86,84,76,82,37,46,38)	43.02 (32.56 to 53.49)	39.29 (28.84 to 49.73)	32.89 (22.33 to 43.46)	36.59 (26.16 to 47.01)	10.81 (0.81 to 20.82)	28.26 (15.25 to 41.27)	13.16 (2.41 to 23.91)
W48: CHL on eyelashes (n=86,83,76,84,37,46,38)	41.86 (31.43 to 52.29)	37.35 (26.94 to 47.76)	38.16 (27.24 to 49.08)	34.52 (24.36 to 44.69)	8.11 (0.00 to 16.90)	39.13 (25.03 to 53.23)	28.95 (14.53 to 43.37)
W4: CHL on body (n=96,97,96,94,47,47,44)	11.46 (5.09 to 17.83)	9.28 (3.50 to 15.05)	10.42 (4.31 to 16.53)	6.38 (1.44 to 11.32)	2.13 (0.00 to 6.25)	4.26 (0.00 to 10.03)	11.36 (1.99 to 20.74)
W8:CHL on body (n=95,94,93,89,46,47,43)	14.74 (7.61 to 21.86)	15.96 (8.55 to 23.36)	13.98 (6.93 to 21.03)	10.11 (3.85 to 16.38)	2.17 (0.00 to 6.39)	6.38 (0.00 to 13.37)	11.63 (2.05 to 21.21)
W12: CHL on body (n=94,94,94,91,45,44,42)	17.02 (9.42 to 24.62)	19.15 (11.19 to 27.10)	14.89 (7.70 to 22.09)	14.29 (7.10 to 21.48)	4.44 (0.00 to 10.47)	11.36 (1.99 to 20.74)	7.14 (0.00 to 14.93)
W18: CHL on body (n=91,92,93,88,47,44,40)	19.78 (11.60 to 27.96)	25.00 (16.15 to 33.85)	15.05 (7.79 to 22.32)	15.91 (8.27 to 23.55)	10.64 (1.82 to 19.45)	9.09 (0.60 to 17.59)	7.50 (0.00 to 15.66)
W24: CHL on body (n=93,93,93,90,45,49,44)	25.81 (16.91 to 34.70)	27.96 (18.84 to 37.08)	20.43 (12.24 to 28.62)	22.22 (13.63 to 30.81)	6.67 (0.00 to 13.95)	16.33 (5.98 to 26.68)	11.36 (1.99 to 20.74)
W34: CHL on body (n=93,92,92,92,44,44,43)	33.33 (23.75 to 42.91)	33.70 (24.04 to 43.35)	31.52 (22.03 to 41.02)	26.09 (17.11 to 35.06)	11.36 (1.99 to 20.74)	22.73 (10.34 to 35.11)	9.30 (0.62 to 17.98)
W40: CHL on body (n=94,94,91,90,46,49,44)	35.11 (25.46 to 44.76)	36.17 (26.46 to 45.88)	30.77 (21.29 to 40.25)	25.56 (16.54 to 34.57)	8.70 (0.55 to 16.84)	22.45 (10.77 to 34.13)	11.36 (1.99 to 20.74)
W48: CHL on body (n=95,93,93,92,46,49,44)	34.74 (25.16 to 44.31)	34.41 (24.75 to 44.06)	36.56 (26.77 to 46.35)	30.43 (21.03 to 39.84)	10.87 (1.87 to 19.86)	34.69 (21.37 to 48.02)	20.45 (8.54 to 32.37)

No statistical analyses for this end point

Other pre-specified: Change From Baseline in Depression Subscale Score of Hospital Anxiety and Depression Scale (HADS) at Weeks 4, 8, 12, and 24

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End point title

Change From Baseline in Depression Subscale Score of Hospital Anxiety and Depression Scale (HADS) at Weeks 4, 8, 12, and 24 ^[9]

End point description

HADS: validated 14-item PRO measure assess states of anxiety and depression over past week. Items rated on 4-point severity scale. HADS produces 2 scales, 1 each for anxiety(HADS–A) and depression(HADS–D), differentiating two states with established normal score cut-offs. Instrument have been validated for use by adolescents >=12 years. Each subscale comprised of 7 items and subject responds how each item applies to him/her over past week prior to baseline visit, on 4point response scale. Separate scores calculated for anxiety and depression with score ranges from 0 (no presence of anxiety/depression) to 3 (severe feeling of anxiety/depression). Total score range was from 0-21 for depression subscale; higher score indicating greater severity of depression symptoms. Baseline was defined as pre-dose on Day 1. FAS included all subjects who were randomised, regardless of whether they received study medication. Here, “n” signifies subjects evaluable at specific time point.

End point type

Other pre-specified

End point timeframe

Baseline (Day 1), Week 4, 8, 12, and 24

Notes
[9] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: Statistics were planned only for the arms specified

End point values	Ritlecitinib (PF-06651600) 200 mg then 50 mg	Ritlecitinib (PF-06651600) 200 mg then 30 mg	Ritlecitinib (PF-06651600) 50 mg	Ritlecitinib (PF-06651600) 30 mg	Ritlecitinib (PF-06651600) 10 mg	Placebo
Number of subjects analysed	132	130	130	132	63	131
Units: Units on a scale
least squares mean (confidence interval 95%)
Change at Week 4 (n= 126, 120, 123, 121, 58, 125)	-0.1 (-0.54 to 0.26)	-0.4 (-0.82 to -0.01)	-0.1 (-0.48 to 0.33)	-0.1 (-0.48 to 0.33)	-0.3 (-0.86 to 0.32)	-0.3 (-0.73 to 0.07)
Change at Week 8 (n= 124, 119, 118, 114, 56, 124)	-0.5 (-0.89 to -0.06)	-0.3 (-0.71 to 0.12)	-0.2 (-0.58 to 0.26)	0.2 (-0.25 to 0.59)	-0.2 (-0.79 to 0.43)	-0.3 (-0.73 to 0.10)
Change at Week 12 (n= 121, 119, 119, 114, 56, 120)	-0.6 (-1.04 to -0.21)	-0.4 (-0.81 to 0.03)	0.0 (-0.45 to 0.39)	0.1 (-0.32 to 0.53)	-0.5 (-1.07 to 0.15)	-0.1 (-0.53 to 0.30)
Change at Week 24 (n= 120, 118, 120, 116, 56, 124)	-0.4 (-0.79 to 0.07)	-0.8 (-1.21 to -0.35)	-0.3 (-0.70 to 0.16)	-0.2 (-0.66 to 0.21)	-0.4 (-1.04 to 0.21)	0.0 (-0.46 to 0.39)

No statistical analyses for this end point

Other pre-specified: Change From Baseline in Depression Subscale Score of Hospital Anxiety and Depression Scale (HADS) at Weeks 48

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End point title

Change From Baseline in Depression Subscale Score of Hospital Anxiety and Depression Scale (HADS) at Weeks 48

End point description

HADS: validated 14-item PRO measure assess states of anxiety and depression over past week. Items rated on 4-point severity scale. HADS produces 2 scales, 1 each for anxiety(HADS–A) and depression(HADS–D), differentiating two states with established normal score cut-offs. Instrument have been validated for use by adolescents >=12 years. Each subscale comprised of 7 items and subject responds how each item applies to him/her over past week prior to baseline visit, on 4-point response scale. Separate scores calculated for anxiety and depression with score ranges from 0 (no presence of anxiety/depression) to 3 (severe feeling of anxiety/depression). Total score range was from 0-21 for depression subscale; higher score indicating greater severity of depression symptoms. Baseline was defined as pre-dose on Day 1. FAS included all subjects who were randomised, regardless of whether they received study medication. Here, “n” signifies subjects evaluable at specific time point.

End point type

Other pre-specified

End point timeframe

Baseline (Day 1), Week 48

End point values	Ritlecitinib (PF-06651600) 200 mg then 50 mg	Ritlecitinib (PF-06651600) 200 mg then 30 mg	Ritlecitinib (PF-06651600) 50 mg	Ritlecitinib (PF-06651600) 30 mg	Ritlecitinib (PF-06651600) 10 mg	Placebo, Ritlecitinib (PF-06651600) 200 mg then 50 mg	Placebo, Ritlecitinib (PF-06651600) 50 mg
Number of subjects analysed	132	130	130	132	63	65	66
Units: Units on a scale
arithmetic mean (standard deviation)
Baseline (n= 131,130, 130, 131, 63, 65, 66)	2.7 ( 2.60 )	3.0 ( 3.16 )	2.9 ( 3.00 )	2.8 ( 2.99 )	2.8 ( 2.80 )	3.2 ( 3.36 )	3.3 ( 3.54 )
Change at Week 48(n=118,118,117,111,55,59,60)	-0.7 ( 2.13 )	-0.4 ( 3.15 )	-0.3 ( 2.67 )	-0.6 ( 2.44 )	-0.5 ( 2.53 )	-0.9 ( 3.25 )	-0.4 ( 3.28 )

No statistical analyses for this end point

Other pre-specified: Change From Baseline in Anxiety Subscale Score of Hospital Anxiety and Depression Scale (HADS) at Weeks 4, 8, 12, and 24

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End point title

Change From Baseline in Anxiety Subscale Score of Hospital Anxiety and Depression Scale (HADS) at Weeks 4, 8, 12, and 24 ^[10]

End point description

HADS: validated 14-item PRO measure assess states of anxiety and depression over past week. Items rated on 4-point severity scale. HADS produces 2 scales, 1 each for anxiety(HADS–A) and depression(HADS–D), differentiating two states with established normal score cut-offs. Instrument have been validated for use by adolescents >=12 years. Each subscale comprised of 7 items and subject responds how each item applies to him/her over past week prior to baseline visit, on 4-point response scale. Separate scores calculated for anxiety and depression with score ranges from 0 (no presence of anxiety/depression) to 3 (severe feeling of anxiety/depression). Total score range was from 0-21 for anxiety subscale; higher score indicating greater severity of anxiety symptoms. Baseline was defined as pre-dose on Day 1. FAS included all subjects who were randomised, regardless of whether they received study medication. Here, “n” signifies subjects evaluable at specific time point.

End point type

Other pre-specified

End point timeframe

Baseline (Day 1), Week 4, 8, 12, and 24

Notes
[10] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: Statistics were planned only for the arms specified

End point values	Ritlecitinib (PF-06651600) 200 mg then 50 mg	Ritlecitinib (PF-06651600) 200 mg then 30 mg	Ritlecitinib (PF-06651600) 50 mg	Ritlecitinib (PF-06651600) 30 mg	Ritlecitinib (PF-06651600) 10 mg	Placebo
Number of subjects analysed	132	130	130	132	63	131
Units: Units on a scale
least squares mean (confidence interval 95%)
Change at Week 4 (n= 125, 119, 123, 121, 58, 125)	-0.3 (-0.78 to 0.08)	-0.4 (-0.79 to 0.08)	-0.4 (-0.79 to 0.08)	-0.5 (-0.97 to -0.10)	-0.6 (-1.25 to 0.02)	-0.1 (-0.54 to 0.32)
Change at Week 8 (n= 123, 119, 118, 114, 56, 123)	-0.8 (-1.25 to -0.31)	-0.4 (-0.88 to 0.07)	-0.8 (-1.27 to -0.31)	-0.3 (-0.79 to 0.17)	-0.9 (-1.59 to -0.20)	-0.5 (-0.96 to -0.03)
Change at Week 12 (n= 120, 119, 119, 114, 56, 121)	-0.7 (-1.22 to -0.28)	-0.9 (-1.40 to -0.46)	-0.7 (-1.22 to -0.27)	-0.3 (-0.77 to 0.19)	-0.9 (-1.55 to -0.17)	-0.4 (-0.84 to 0.09)
Change at Week 24 (n= 119, 118, 120, 115, 56, 124)	-0.8 (-1.26 to -0.25)	-0.7 (-1.19 to -0.18)	-0.8 (-1.28 to -0.27)	-0.3 (-0.76 to 0.26)	-1.0 (-1.69 to -0.21)	-0.6 (-1.06 to -0.07)

No statistical analyses for this end point

Other pre-specified: Change From Baseline in Anxiety Subscale Score of Hospital Anxiety and Depression Scale (HADS) at Week 48

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End point title

Change From Baseline in Anxiety Subscale Score of Hospital Anxiety and Depression Scale (HADS) at Week 48

End point description

HADS: validated 14-item PRO measure assess states of anxiety and depression over past week. Items rated on 4-point severity scale. HADS produces 2 scales, 1 each for anxiety(HADS–A) and depression(HADS–D), differentiating two states with established normal score cut-offs. Instrument have been validated for use by adolescents >=12 years. Each subscale comprised of 7 items and subject responds how each item applies to him/her over past week prior to baseline visit, on 4-point response scale. Separate scores calculated for anxiety and depression with score ranges from 0 (no presence of anxiety/depression) to 3 (severe feeling of anxiety/depression). Total score range was from 0-21 for anxiety subscale; higher score indicating greater severity of anxiety symptoms. Baseline was defined as pre-dose on Day 1. FAS included all subjects who were randomised, regardless of whether they received study medication. Here, “n” signifies subjects evaluable at specific time point.

End point type

Other pre-specified

End point timeframe

Baseline (Day 1), Week 48

End point values	Ritlecitinib (PF-06651600) 200 mg then 50 mg	Ritlecitinib (PF-06651600) 200 mg then 30 mg	Ritlecitinib (PF-06651600) 50 mg	Ritlecitinib (PF-06651600) 30 mg	Ritlecitinib (PF-06651600) 10 mg	Placebo, Ritlecitinib (PF-06651600) 200 mg then 50 mg	Placebo, Ritlecitinib (PF-06651600) 50 mg
Number of subjects analysed	132	130	130	132	63	65	66
Units: Units on a scale
arithmetic mean (standard deviation)
Baseline (n= 130, 130, 130, 131, 63, 65, 66)	4.6 ( 3.84 )	4.5 ( 3.18 )	4.9 ( 3.31 )	4.3 ( 3.42 )	5.2 ( 3.38 )	5.3 ( 4.20 )	5.3 ( 3.70 )
Change at Week 48(n=117,118,117,111,55,59,60)	-1.0 ( 2.92 )	-0.8 ( 3.10 )	-0.8 ( 3.09 )	-0.5 ( 3.40 )	-1.2 ( 2.89 )	-1.3 ( 3.31 )	-0.5 ( 4.53 )

No statistical analyses for this end point

Other pre-specified: Percentage of Subjects With a Baseline Score Indicative of Depression Achieving Normal Depression Subscale Score of HADS at Week 4, 8, 12, 24, and 48

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End point title

Percentage of Subjects With a Baseline Score Indicative of Depression Achieving Normal Depression Subscale Score of HADS at Week 4, 8, 12, 24, and 48

End point description

End point type

Other pre-specified

End point timeframe

Week 4, 8, 12, 24 and 48

End point values	Ritlecitinib (PF-06651600) 200 mg then 50 mg	Ritlecitinib (PF-06651600) 200 mg then 30 mg	Ritlecitinib (PF-06651600) 50 mg	Ritlecitinib (PF-06651600) 30 mg	Ritlecitinib (PF-06651600) 10 mg	Placebo, Ritlecitinib (PF-06651600) 200 mg then 50 mg	Placebo, Ritlecitinib (PF-06651600) 50 mg
Number of subjects analysed	132	130	130	132	63	65	66
Units: Percentage of subjects
number (confidence interval 95%)
Week 4 (n= 7, 10, 13, 14, 5, 5, 10)	42.86 (6.20 to 79.52)	60.00 (29.64 to 90.36)	53.85 (26.75 to 80.95)	42.86 (16.93 to 68.78)	40.00 (0.00 to 82.94)	60.00 (17.06 to 100.00)	50.00 (19.01 to 80.99)
Week 8 (n= 6, 11, 12, 12, 5, 4, 10)	66.67 (28.95 to 100.00)	45.45 (16.03 to 74.88)	58.33 (30.44 to 86.23)	33.33 (6.66 to 60.01)	20.00 (0.00 to 55.06)	50.00 (1.00 to 99.00)	40.00 (9.64 to 70.36)
Week 12 (n= 6, 11, 12, 13, 5, 5, 8)	50.00 (9.99 to 90.01)	63.64 (35.21 to 92.06)	66.67 (39.99 to 93.34)	30.77 (5.68 to 55.86)	20.00 (0.00 to 55.06)	60.00 (17.06 to 100.00)	62.50 (28.95 to 96.05)
Week 24 (n= 7, 11, 12, 14, 4, 4, 10)	71.43 (37.96 to 100.00)	63.64 (35.21 to 92.06)	41.67 (13.77 to 69.56)	42.86 (16.93 to 68.78)	50.00 (1.00 to 99.00)	50.00 (1.00 to 99.00)	50.00 (19.01 to 80.99)
Week 48 (n= 6, 11, 12, 13, 5, 5, 10)	50.00 (9.99 to 90.01)	63.64 (35.21 to 92.06)	58.33 (30.44 to 86.23)	46.15 (19.05 to 73.25)	80.00 (44.94 to 100.00)	60.00 (17.06 to 100.00)	60.00 (29.64 to 90.36)

No statistical analyses for this end point

Other pre-specified: Percentage of Subjects With a Baseline Score Indicative of Anxiety Achieving Normal Anxiety Subscale Score of HADS at Week 4, 8, 12, 24, and 48

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End point title

Percentage of Subjects With a Baseline Score Indicative of Anxiety Achieving Normal Anxiety Subscale Score of HADS at Week 4, 8, 12, 24, and 48

End point description

HADS: validated 14-item PRO measure assess states of anxiety and depression over past week. Items rated on 4-point severity scale. HADS produces 2 scales, 1 each for anxiety(HADS–A) and depression(HADS–D), differentiating two states with established normal score cut-offs. Instrument have been validated for use by adolescents >=12 years. Each subscale comprised of 7 items and subject responds how each item applies to him/her over past week prior to baseline visit, on 4-point response scale. Separate scores calculated for anxiety and depression with score ranges from 0 (no presence of anxiety/depression) to 3 (severe feeling of anxiety/depression). Total score range was from 0-21 for anxiety subscale; higher score indicating greater severity of anxiety symptoms. Baseline was defined as pre-dose on Day 1. FAS included all subjects who were randomised, regardless of whether they received study medication. Here, “n” signifies subjects evaluable at specific time point.

End point type

Other pre-specified

End point timeframe

Week 4, 8, 12, 24 and 48

End point values	Ritlecitinib (PF-06651600) 200 mg then 50 mg	Ritlecitinib (PF-06651600) 200 mg then 30 mg	Ritlecitinib (PF-06651600) 50 mg	Ritlecitinib (PF-06651600) 30 mg	Ritlecitinib (PF-06651600) 10 mg	Placebo, Ritlecitinib (PF-06651600) 200 mg then 50 mg	Placebo, Ritlecitinib (PF-06651600) 50 mg
Number of subjects analysed	132	130	130	132	63	65	66
Units: Percentage of subjects
number (confidence interval 95%)
Week 4 (29, 22, 26, 16, 15, 19, 13)	51.72 (33.54 to 69.91)	45.45 (24.65 to 66.26)	34.62 (16.33 to 52.90)	37.50 (13.78 to 61.22)	40.00 (15.21 to 64.79)	31.58 (10.68 to 52.48)	30.77 (5.68 to 55.86)
Week 8 (n= 28, 20, 25, 12, 14, 18,13)	46.43 (27.96 to 64.90)	50.00 (28.09 to 71.91)	68.00 (49.71 to 86.29)	41.67 (13.77 to 69.56)	71.43 (47.76 to 95.09)	33.33 (11.56 to 55.11)	46.15 (19.05 to 73.25)
Week 12 (n= 28, 19, 26, 16, 14, 19, 12)	50.00 (31.48 to 68.52)	57.89 (35.69 to 80.10)	38.46 (19.76 to 57.16)	56.25 (31.94 to 80.56)	42.86 (16.93 to 68.78)	47.37 (24.92 to 69.82)	41.67 (13.77 to 69.56)
Week 24 (n= 27, 20, 26, 15, 14, 18, 13)	51.85 (33.01 to 70.70)	65.00 (44.10 to 85.90)	50.00 (30.78 to 69.22)	46.67 (21.42 to 71.91)	57.14 (31.22 to 83.07)	44.44 (21.49 to 67.40)	38.46 (12.02 to 64.91)
Week 48 (n= 26, 18, 25, 14, 13, 19, 12)	53.85 (34.68 to 73.01)	66.67 (44.89 to 88.44)	60.00 (40.80 to 79.20)	50.00 (23.81 to 76.19)	61.54 (35.09 to 87.98)	57.89 (35.69 to 80.10)	58.33 (30.44 to 86.23)

No statistical analyses for this end point

Adverse events

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Timeframe for reporting adverse events

Baseline up to 5 weeks after last dose of study drug (maximum up to Week 53)

Adverse event reporting additional description

Same event may appear as both an adverse event(AE) and serious adverse event(SAE). An event may be categorised as serious in one subject and as non-serious in another subject, or one subject may have experienced both a serious and non-serious event during the study. Safety analysis set: all subjects who received at least 1 dose of study medication.

Assessment type

Non-systematic

Dictionary name

MedDRA

Dictionary version

24.0

Reporting group title

Ritlecitinib (PF-06651600) 200 mg then 50 mg

Reporting group description

Subjects aged 12 years or above with moderate to severe AA with >=50% hair loss of the scalp were randomised to receive Ritlecitinib 200 mg tablet once daily for 4 weeks (loading phase) and then 50 mg tablet once daily for next 20 weeks (maintenance phase). Subjects then entered into extension phase of 24 weeks and continued to receive 50 mg tablet once daily. Subjects were followed up to maximum of 5 weeks after the last dose of study drug .

Reporting group title

Ritlecitinib (PF-06651600) 50 mg

Reporting group description

Reporting group title

Ritlecitinib (PF-06651600) 200 mg then 30 mg

Reporting group description

Subjects aged 12 years or above with moderate to severe AA with >=50% hair loss of the scalp were randomised to receive Ritlecitinib 200 mg tablet once daily for 4 weeks (loading phase) and then 30 mg tablet once daily for next 20 weeks (maintenance phase). Subjects then entered into extension phase of 24 weeks and continued to receive 30 mg tablet once daily. Subjects were followed up to maximum of 5 weeks after the last dose of study drug.

Reporting group title

Ritlecitinib (PF-06651600) 30 mg

Reporting group description

Subjects aged 12 years or above with moderate to severe AA with >=50% hair loss of the scalp were randomised to receive Ritlecitinib 30 mg tablet once daily for 4 weeks (loading phase). Subjects then continued to receive 30 mg tablet once daily in maintenance phase of 20 weeks and extension phase of 24 weeks. Subjects were followed up to maximum of 5 weeks after the last dose of study drug.

Reporting group title

Ritlecitinib (PF-06651600) 10 mg

Reporting group description

Reporting group title

Placebo, Ritlecitinib (PF-06651600) 200 mg then 50 mg

Reporting group description

Subjects aged 12 years or above with moderate to severe AA with >=50% hair loss of the scalp were randomised to receive placebo once daily for 4 weeks (loading phase) and then continued placebo once daily for next 20 weeks (maintenance phase). Subjects then entered into extension phase of 24 weeks and received 200 mg tablet once daily for 4 weeks and then 50 mg tablet once daily for 20 weeks. Subjects were followed up to maximum of 5 weeks after the last dose of study drug.

Reporting group title

Placebo, Ritlecitinib (PF-06651600) 50 mg

Reporting group description

Subjects aged 12 years or above with moderate to severe AA with >=50% hair loss of the scalp were randomised to receive placebo once daily for 4 weeks (loading phase) and then continued to receive placebo once daily for next 20 weeks (maintenance phase). Subjects then entered into extension phase of 24 weeks and receive 50 mg tablet once daily. Subjects were followed up to maximum of 5 weeks after the last dose of study drug.

Serious adverse events	Ritlecitinib (PF-06651600) 200 mg then 50 mg	Ritlecitinib (PF-06651600) 50 mg	Ritlecitinib (PF-06651600) 200 mg then 30 mg	Ritlecitinib (PF-06651600) 30 mg	Ritlecitinib (PF-06651600) 10 mg	Placebo, Ritlecitinib (PF-06651600) 200 mg then 50 mg	Placebo, Ritlecitinib (PF-06651600) 50 mg
Total subjects affected by serious adverse events
subjects affected / exposed	4 / 131 (3.05%)	2 / 130 (1.54%)	2 / 129 (1.55%)	1 / 132 (0.76%)	2 / 62 (3.23%)	0 / 65 (0.00%)	3 / 66 (4.55%)
number of deaths (all causes)	0	0	0	0	0	0	0
number of deaths resulting from adverse events
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Breast cancer
subjects affected / exposed	0 / 131 (0.00%)	1 / 130 (0.77%)	0 / 129 (0.00%)	0 / 132 (0.00%)	0 / 62 (0.00%)	0 / 65 (0.00%)	0 / 66 (0.00%)
occurrences causally related to treatment / all	0 / 0	1 / 1	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Invasive lobular breast carcinoma
subjects affected / exposed	1 / 131 (0.76%)	0 / 130 (0.00%)	0 / 129 (0.00%)	0 / 132 (0.00%)	0 / 62 (0.00%)	0 / 65 (0.00%)	0 / 66 (0.00%)
occurrences causally related to treatment / all	1 / 1	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Injury, poisoning and procedural complications
Chemical poisoning
subjects affected / exposed	0 / 131 (0.00%)	0 / 130 (0.00%)	1 / 129 (0.78%)	0 / 132 (0.00%)	0 / 62 (0.00%)	0 / 65 (0.00%)	0 / 66 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 1	0 / 0	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Pregnancy, puerperium and perinatal conditions
Abortion spontaneous
subjects affected / exposed	1 / 131 (0.76%)	0 / 130 (0.00%)	0 / 129 (0.00%)	0 / 132 (0.00%)	0 / 62 (0.00%)	0 / 65 (0.00%)	1 / 66 (1.52%)
occurrences causally related to treatment / all	0 / 1	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Reproductive system and breast disorders
Heavy menstrual bleeding
subjects affected / exposed	0 / 131 (0.00%)	0 / 130 (0.00%)	0 / 129 (0.00%)	0 / 132 (0.00%)	0 / 62 (0.00%)	0 / 65 (0.00%)	1 / 66 (1.52%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
subjects affected / exposed	0 / 131 (0.00%)	1 / 130 (0.77%)	0 / 129 (0.00%)	0 / 132 (0.00%)	0 / 62 (0.00%)	0 / 65 (0.00%)	0 / 66 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 1	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Skin and subcutaneous tissue disorders
Eczema
subjects affected / exposed	0 / 131 (0.00%)	0 / 130 (0.00%)	0 / 129 (0.00%)	0 / 132 (0.00%)	1 / 62 (1.61%)	0 / 65 (0.00%)	0 / 66 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 1	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Psychiatric disorders
Conversion disorder
subjects affected / exposed	0 / 131 (0.00%)	0 / 130 (0.00%)	0 / 129 (0.00%)	0 / 132 (0.00%)	0 / 62 (0.00%)	0 / 65 (0.00%)	1 / 66 (1.52%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Suicidal behaviour
subjects affected / exposed	0 / 131 (0.00%)	0 / 130 (0.00%)	1 / 129 (0.78%)	0 / 132 (0.00%)	1 / 62 (1.61%)	0 / 65 (0.00%)	0 / 66 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 1	0 / 0	0 / 1	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Infections and infestations
Appendicitis
subjects affected / exposed	1 / 131 (0.76%)	0 / 130 (0.00%)	1 / 129 (0.78%)	0 / 132 (0.00%)	0 / 62 (0.00%)	0 / 65 (0.00%)	0 / 66 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0	0 / 1	0 / 0	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Diverticulitis
subjects affected / exposed	0 / 131 (0.00%)	0 / 130 (0.00%)	0 / 129 (0.00%)	1 / 132 (0.76%)	0 / 62 (0.00%)	0 / 65 (0.00%)	0 / 66 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 1	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Empyema
subjects affected / exposed	1 / 131 (0.76%)	0 / 130 (0.00%)	0 / 129 (0.00%)	0 / 132 (0.00%)	0 / 62 (0.00%)	0 / 65 (0.00%)	0 / 66 (0.00%)
occurrences causally related to treatment / all	1 / 1	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Sepsis
subjects affected / exposed	1 / 131 (0.76%)	0 / 130 (0.00%)	0 / 129 (0.00%)	0 / 132 (0.00%)	0 / 62 (0.00%)	0 / 65 (0.00%)	0 / 66 (0.00%)
occurrences causally related to treatment / all	1 / 1	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0

Frequency threshold for reporting non-serious adverse events: 5%

Non-serious adverse events	Ritlecitinib (PF-06651600) 200 mg then 50 mg	Ritlecitinib (PF-06651600) 50 mg	Ritlecitinib (PF-06651600) 200 mg then 30 mg	Ritlecitinib (PF-06651600) 30 mg	Ritlecitinib (PF-06651600) 10 mg	Placebo, Ritlecitinib (PF-06651600) 200 mg then 50 mg	Placebo, Ritlecitinib (PF-06651600) 50 mg
Total subjects affected by non serious adverse events
subjects affected / exposed	84 / 131 (64.12%)	81 / 130 (62.31%)	76 / 129 (58.91%)	77 / 132 (58.33%)	32 / 62 (51.61%)	39 / 65 (60.00%)	39 / 66 (59.09%)
Nervous system disorders
Dizziness
subjects affected / exposed	9 / 131 (6.87%)	4 / 130 (3.08%)	8 / 129 (6.20%)	8 / 132 (6.06%)	1 / 62 (1.61%)	0 / 65 (0.00%)	2 / 66 (3.03%)
occurrences all number	10	4	8	9	2	0	3
Headache
subjects affected / exposed	17 / 131 (12.98%)	16 / 130 (12.31%)	14 / 129 (10.85%)	24 / 132 (18.18%)	12 / 62 (19.35%)	8 / 65 (12.31%)	8 / 66 (12.12%)
occurrences all number	25	21	18	40	17	25	13
General disorders and administration site conditions
Fatigue
subjects affected / exposed	4 / 131 (3.05%)	6 / 130 (4.62%)	6 / 129 (4.65%)	6 / 132 (4.55%)	4 / 62 (6.45%)	3 / 65 (4.62%)	2 / 66 (3.03%)
occurrences all number	4	8	6	7	4	3	2
Gastrointestinal disorders
Abdominal pain upper
subjects affected / exposed	1 / 131 (0.76%)	5 / 130 (3.85%)	5 / 129 (3.88%)	3 / 132 (2.27%)	0 / 62 (0.00%)	4 / 65 (6.15%)	0 / 66 (0.00%)
occurrences all number	1	6	5	3	0	4	0
Constipation
subjects affected / exposed	1 / 131 (0.76%)	1 / 130 (0.77%)	0 / 129 (0.00%)	7 / 132 (5.30%)	1 / 62 (1.61%)	1 / 65 (1.54%)	0 / 66 (0.00%)
occurrences all number	1	1	0	7	1	1	0
Diarrhoea
subjects affected / exposed	9 / 131 (6.87%)	12 / 130 (9.23%)	4 / 129 (3.10%)	8 / 132 (6.06%)	0 / 62 (0.00%)	4 / 65 (6.15%)	1 / 66 (1.52%)
occurrences all number	9	13	4	8	0	5	1
Nausea
subjects affected / exposed	11 / 131 (8.40%)	3 / 130 (2.31%)	3 / 129 (2.33%)	12 / 132 (9.09%)	3 / 62 (4.84%)	8 / 65 (12.31%)	1 / 66 (1.52%)
occurrences all number	14	5	3	15	6	10	1
Vomiting
subjects affected / exposed	6 / 131 (4.58%)	2 / 130 (1.54%)	7 / 129 (5.43%)	5 / 132 (3.79%)	1 / 62 (1.61%)	2 / 65 (3.08%)	3 / 66 (4.55%)
occurrences all number	7	3	8	8	1	2	3
Respiratory, thoracic and mediastinal disorders
Cough
subjects affected / exposed	6 / 131 (4.58%)	3 / 130 (2.31%)	0 / 129 (0.00%)	3 / 132 (2.27%)	0 / 62 (0.00%)	4 / 65 (6.15%)	2 / 66 (3.03%)
occurrences all number	6	4	0	3	0	4	2
Nasal congestion
subjects affected / exposed	1 / 131 (0.76%)	2 / 130 (1.54%)	1 / 129 (0.78%)	3 / 132 (2.27%)	4 / 62 (6.45%)	1 / 65 (1.54%)	1 / 66 (1.52%)
occurrences all number	1	2	1	3	4	2	1
Oropharyngeal pain
subjects affected / exposed	4 / 131 (3.05%)	6 / 130 (4.62%)	6 / 129 (4.65%)	1 / 132 (0.76%)	0 / 62 (0.00%)	2 / 65 (3.08%)	5 / 66 (7.58%)
occurrences all number	6	6	6	1	0	3	9
Skin and subcutaneous tissue disorders
Acne
subjects affected / exposed	6 / 131 (4.58%)	12 / 130 (9.23%)	10 / 129 (7.75%)	12 / 132 (9.09%)	3 / 62 (4.84%)	5 / 65 (7.69%)	8 / 66 (12.12%)
occurrences all number	9	13	13	12	4	5	9
Pruritus
subjects affected / exposed	4 / 131 (3.05%)	1 / 130 (0.77%)	7 / 129 (5.43%)	3 / 132 (2.27%)	1 / 62 (1.61%)	1 / 65 (1.54%)	1 / 66 (1.52%)
occurrences all number	4	1	16	3	1	1	1
Rash
subjects affected / exposed	5 / 131 (3.82%)	7 / 130 (5.38%)	3 / 129 (2.33%)	1 / 132 (0.76%)	0 / 62 (0.00%)	1 / 65 (1.54%)	1 / 66 (1.52%)
occurrences all number	7	7	3	2	0	1	1
Urticaria
subjects affected / exposed	9 / 131 (6.87%)	7 / 130 (5.38%)	9 / 129 (6.98%)	5 / 132 (3.79%)	1 / 62 (1.61%)	4 / 65 (6.15%)	4 / 66 (6.06%)
occurrences all number	12	13	11	5	1	12	4
Psychiatric disorders
Insomnia
subjects affected / exposed	3 / 131 (2.29%)	2 / 130 (1.54%)	0 / 129 (0.00%)	1 / 132 (0.76%)	1 / 62 (1.61%)	1 / 65 (1.54%)	4 / 66 (6.06%)
occurrences all number	3	2	0	1	1	1	4
Musculoskeletal and connective tissue disorders
Arthralgia
subjects affected / exposed	4 / 131 (3.05%)	2 / 130 (1.54%)	5 / 129 (3.88%)	4 / 132 (3.03%)	2 / 62 (3.23%)	2 / 65 (3.08%)	6 / 66 (9.09%)
occurrences all number	4	2	5	4	2	3	6
Myalgia
subjects affected / exposed	6 / 131 (4.58%)	3 / 130 (2.31%)	3 / 129 (2.33%)	5 / 132 (3.79%)	6 / 62 (9.68%)	0 / 65 (0.00%)	1 / 66 (1.52%)
occurrences all number	6	4	3	7	8	0	1
Infections and infestations
Folliculitis
subjects affected / exposed	11 / 131 (8.40%)	8 / 130 (6.15%)	11 / 129 (8.53%)	5 / 132 (3.79%)	4 / 62 (6.45%)	4 / 65 (6.15%)	4 / 66 (6.06%)
occurrences all number	13	8	13	5	4	4	5
Influenza
subjects affected / exposed	8 / 131 (6.11%)	3 / 130 (2.31%)	1 / 129 (0.78%)	3 / 132 (2.27%)	3 / 62 (4.84%)	1 / 65 (1.54%)	0 / 66 (0.00%)
occurrences all number	8	3	1	3	3	1	0
Nasopharyngitis
subjects affected / exposed	19 / 131 (14.50%)	18 / 130 (13.85%)	21 / 129 (16.28%)	21 / 132 (15.91%)	7 / 62 (11.29%)	7 / 65 (10.77%)	4 / 66 (6.06%)
occurrences all number	22	21	27	29	13	8	4
Upper respiratory tract infection
subjects affected / exposed	18 / 131 (13.74%)	11 / 130 (8.46%)	12 / 129 (9.30%)	16 / 132 (12.12%)	2 / 62 (3.23%)	7 / 65 (10.77%)	6 / 66 (9.09%)
occurrences all number	21	14	16	20	2	8	7
Urinary tract infection
subjects affected / exposed	11 / 131 (8.40%)	1 / 130 (0.77%)	3 / 129 (2.33%)	5 / 132 (3.79%)	0 / 62 (0.00%)	4 / 65 (6.15%)	2 / 66 (3.03%)
occurrences all number	21	1	5	6	0	4	2

More information

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Were there any global substantial amendments to the protocol? No

Were there any global interruptions to the trial? No

Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.

None reported

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Clinical trials

Clinical Trial Results: A Phase 2b/3 Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Investigate the Efficacy and Safety of PF-06651600 in Adult and Adolescent Alopecia Areata (AA) Subjects With 50% or Greater Scalp Hair Loss

Trial information

Trial information

Subject disposition

Subject disposition

Baseline characteristics

Baseline characteristics

End points

End points

Primary: Percentage of Subjects With an Absolute Severity of Alopecia Tool (SALT) Score of Less Than or Equal to 10 at Week 24: Analysis 4

Primary: Percentage of Subjects With an Absolute Severity of Alopecia Tool (SALT) Score of Less Than or Equal to 10 at Week 24: Analysis 4

Secondary: Percentage of Subjects With an Absolute SALT Score of Less Than or Equal to 20 at Week 24

Secondary: Percentage of Subjects With an Absolute SALT Score of Less Than or Equal to 20 at Week 24

Secondary: Percentage of Subjects With an Absolute SALT Score of Less Than or Equal to 10 at Week 24: Analysis 1

Secondary: Percentage of Subjects With an Absolute SALT Score of Less Than or Equal to 10 at Week 24: Analysis 1

Secondary: Percentage of Subjects With Patient Global Impression of Change (PGI-C) Score of Moderately Improved or Greatly Improved at Week 24

Secondary: Percentage of Subjects With Patient Global Impression of Change (PGI-C) Score of Moderately Improved or Greatly Improved at Week 24

Secondary: Exposure Response of PF-06651600 on Regrowth of Lost Hair Based on Absolute SALT Score of Less Than or Equal to 20 at Week 24: Maximum Effect (Emax) Model

Secondary: Exposure Response of PF-06651600 on Regrowth of Lost Hair Based on Absolute SALT Score of Less Than or Equal to 20 at Week 24: Maximum Effect (Emax) Model

Secondary: Exposure Response of PF-06651600 on Regrowth of Lost Hair Based on Absolute SALT Score of Less Than or Equal to 10 at Week 24: Maximum Effect (Emax) Model

Secondary: Exposure Response of PF-06651600 on Regrowth of Lost Hair Based on Absolute SALT Score of Less Than or Equal to 10 at Week 24: Maximum Effect (Emax) Model

Secondary: Percentage of Subjects With an Absolute SALT Score of Less Than or Equal to 20 at Week 4, 8, 12, 18, 28, 34, 40, and 48

Secondary: Percentage of Subjects With an Absolute SALT Score of Less Than or Equal to 20 at Week 4, 8, 12, 18, 28, 34, 40, and 48

Secondary: Percentage of Subjects With an Absolute SALT Score of Less Than or Equal to 10 at Week 4, 8, 12, 18, 28, 34, 40, and 48

Secondary: Percentage of Subjects With an Absolute SALT Score of Less Than or Equal to 10 at Week 4, 8, 12, 18, 28, 34, 40, and 48

Secondary: Percentage of Subjects With at Least 75% Improvement in SALT Score (SALT75) From Baseline at Week 4, 8, 12, 18, 24, 28, 34, 40, and 48

Secondary: Percentage of Subjects With at Least 75% Improvement in SALT Score (SALT75) From Baseline at Week 4, 8, 12, 18, 24, 28, 34, 40, and 48

Secondary: Change From Baseline in SALT Score at Week 4, 8, 12, 18, and 24

Secondary: Change From Baseline in SALT Score at Week 4, 8, 12, 18, and 24

Secondary: Change From Baseline in SALT Score at Week 28, 34, 40, and 48

Secondary: Change From Baseline in SALT Score at Week 28, 34, 40, and 48

Secondary: Percentage of Subjects With at Least a 2 Grade Improvement From Baseline or a Score of 3 in Eyebrow Assessment (EBA) Score (Among Subjects Without Normal EBA at Baseline) at Week 4, 8, 12, 18, 24, 28, 34, 40, and 48

Secondary: Percentage of Subjects With at Least a 2 Grade Improvement From Baseline or a Score of 3 in Eyebrow Assessment (EBA) Score (Among Subjects Without Normal EBA at Baseline) at Week 4, 8, 12, 18, 24, 28, 34, 40, and 48

Secondary: Percentage of Subjects With at Least a 2 Grade Improvement From Baseline or a Score of 3 in Eyelash Assessment (ELA) Score (Among Subjects Without Normal ELA at Baseline) at Week 4, 8, 12, 18, 24, 28, 34, 40, and 48

Secondary: Percentage of Subjects With at Least a 2 Grade Improvement From Baseline or a Score of 3 in Eyelash Assessment (ELA) Score (Among Subjects Without Normal ELA at Baseline) at Week 4, 8, 12, 18, 24, 28, 34, 40, and 48

Secondary: Percentage of Subjects With Patient Global Impression of Change (PGI-C) Score of Moderately Improved or Greatly Improved at Week 4, 8, 12, 18, 24, 34, 40, and 48

Secondary: Percentage of Subjects With Patient Global Impression of Change (PGI-C) Score of Moderately Improved or Greatly Improved at Week 4, 8, 12, 18, 24, 34, 40, and 48

Secondary: Change From Baseline in Alopecia Areata Patient Priority Outcomes (AAPPO) Domain Scores at Week 4, 8, 12, 18, and 24: Emotional Symptoms (ES) and Activity Limitations (AL)

Secondary: Change From Baseline in Alopecia Areata Patient Priority Outcomes (AAPPO) Domain Scores at Week 4, 8, 12, 18, and 24: Emotional Symptoms (ES) and Activity Limitations (AL)

Secondary: Change From Baseline in Alopecia Areata Patient Priority Outcomes (AAPPO) Domain Scores at Week 34, 40, and 48: Emotional Symptoms (ES) and Activity Limitations (AL)

Secondary: Change From Baseline in Alopecia Areata Patient Priority Outcomes (AAPPO) Domain Scores at Week 34, 40, and 48: Emotional Symptoms (ES) and Activity Limitations (AL)

Secondary: Percentage of Subjects With Improvement From Baseline on Alopecia Areata Patient Priority Outcomes (AAPPO) Items 1-4 at Week 4, 8, 12, 18, 24, 34, 40, and 48

Secondary: Percentage of Subjects With Improvement From Baseline on Alopecia Areata Patient Priority Outcomes (AAPPO) Items 1-4 at Week 4, 8, 12, 18, 24, 34, 40, and 48

Other pre-specified: Change From Baseline in Depression Subscale Score of Hospital Anxiety and Depression Scale (HADS) at Weeks 4, 8, 12, and 24

Other pre-specified: Change From Baseline in Depression Subscale Score of Hospital Anxiety and Depression Scale (HADS) at Weeks 4, 8, 12, and 24

Other pre-specified: Change From Baseline in Depression Subscale Score of Hospital Anxiety and Depression Scale (HADS) at Weeks 48

Other pre-specified: Change From Baseline in Depression Subscale Score of Hospital Anxiety and Depression Scale (HADS) at Weeks 48

Other pre-specified: Change From Baseline in Anxiety Subscale Score of Hospital Anxiety and Depression Scale (HADS) at Weeks 4, 8, 12, and 24

Other pre-specified: Change From Baseline in Anxiety Subscale Score of Hospital Anxiety and Depression Scale (HADS) at Weeks 4, 8, 12, and 24

Other pre-specified: Change From Baseline in Anxiety Subscale Score of Hospital Anxiety and Depression Scale (HADS) at Week 48

Other pre-specified: Change From Baseline in Anxiety Subscale Score of Hospital Anxiety and Depression Scale (HADS) at Week 48

Other pre-specified: Percentage of Subjects With a Baseline Score Indicative of Depression Achieving Normal Depression Subscale Score of HADS at Week 4, 8, 12, 24, and 48

Other pre-specified: Percentage of Subjects With a Baseline Score Indicative of Depression Achieving Normal Depression Subscale Score of HADS at Week 4, 8, 12, 24, and 48

Other pre-specified: Percentage of Subjects With a Baseline Score Indicative of Anxiety Achieving Normal Anxiety Subscale Score of HADS at Week 4, 8, 12, 24, and 48

Other pre-specified: Percentage of Subjects With a Baseline Score Indicative of Anxiety Achieving Normal Anxiety Subscale Score of HADS at Week 4, 8, 12, 24, and 48

Adverse events

Adverse events

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Substantial protocol amendments (globally)

Interruptions (globally)

Limitations and caveats

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Clinical Trial Results:
A Phase 2b/3 Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Investigate the Efficacy and Safety of PF-06651600 in Adult and Adolescent Alopecia Areata (AA) Subjects With 50% or Greater Scalp Hair Loss