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Clinical Trial Results:
A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Study Investigating the Safety of Ritlecitinib (PF-06651600) in Adult Participants With Alopecia Areata

Summary
EudraCT number	2020-001509-21
Trial protocol	PL
Global end of trial date

Results information
Results version number	v2(current)
This version publication date	01 May 2024
First version publication date	02 Nov 2022
Other versions	v1
Version creation reason

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

B7981037

ISRCTN number

US NCT number

NCT04517864

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., +1 8007181021, ClinicalTrials.gov_Inquiries@pfizer.com

Scientific contact

Pfizer ClinicalTrials.gov Call Center, Pfizer Inc., +1 8007181021, 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?

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

Analysis stage

Interim

Date of interim/final analysis

13 Sep 2023

Is this the analysis of the primary completion data?

Yes

Primary completion date

04 Jan 2022

Global end of trial reached?

Main objective of the trial

This was a Phase 2a, randomized, double-blind, parallel group, placebo-controlled safety study designed to evaluate the safety and tolerability of ritlecitinib, including the assessments of brainstem auditory evoked potential (BAEP) and intraepidermal nerve fiber (IENF), in adults 18 to ≤50 years of age with ≥25% scalp hair loss due to alopecia areata (AA).

Protection of trial subjects

This study was conducted in compliance with the ethical principles originating in or derived from the Declaration of Helsinki and in compliance with all International Council for Harmonization (ICH) Good Clinical Practice (GCP) Guidelines. In addition, all local regulatory requirements were followed, in particular, those affording greater protection to the safety of subjects.

Background therapy

Evidence for comparator

Actual start date of recruitment

15 Sep 2020

Long term follow-up planned

Independent data monitoring committee (IDMC) involvement?

Yes

Number of subjects enrolled per country

Country: Number of subjects enrolled

Australia: 2

Country: Number of subjects enrolled

Canada: 8

Country: Number of subjects enrolled

Poland: 36

Country: Number of subjects enrolled

United States: 25

Worldwide total number of subjects

EEA total number of subjects

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)

Adults (18-64 years)

From 65 to 84 years

85 years and over

Subject disposition

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Recruitment details

A total of 71 subjects were enrolled and randomized to double-blind treatment and treated (36 in the Ritlecitinib 200/50 mg once daily [QD] arm and 35 in the Placebo -> Ritlecitinib 200/50 mg QD arm).

Screening details

A total of 131 subjects were screened for this study, among whom 71 subjects were randomized to double-blind treatment with 53 screen failures and 7 subjects were not screen failure but not randomized.

Period 1 title

Placebo-Controlled Phase

Is this the baseline period?

Yes

Allocation method

Randomised - controlled

Blinding used

Double blind

Roles blinded

Subject, Investigator

Are arms mutually exclusive

Yes

Ritlecitinib 200/50 mg QD

Arm description

In the 9-month Placebo-Controlled Phase, each subject received ritlecitinib 200 mg QD (50 mg/tablet ×4) during the initial 4-week period, and received 1 tablet of ritlecitinib 50 mg QD during the remainder of this treatment phase.

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

Ritlecitinib 200 mg QD (50 mg/tablet ×4) was received during the initial 4-week period, and 1 tablet of ritlecitinib 50 mg QD was received during the remainder of the Placebo-Controlled Phase.

Placebo

Arm description

Arm type

Placebo

Investigational medicinal product name

Placebo

Investigational medicinal product code

Other name

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

A total of 4 tablets of Placebo QD were received during the initial 4-week period, and 1 tablet of Placebo QD was received during the remainder of the Placebo-Controlled Phase.

Number of subjects in period 1	Ritlecitinib 200/50 mg QD	Placebo
Started	36	35
Completed	32	33
Not completed	4	2
Consent withdrawn by subject	1	1
Adverse event, non-fatal	1	-
Lost to follow-up	1	1
Protocol deviation	1	-

Period 2 title

Active Therapy Extension Phase

Is this the baseline period?

Allocation method

Non-randomised - controlled

Blinding used

Double blind

Roles blinded

Subject, Investigator

Are arms mutually exclusive

Yes

Ritlecitinib 50 mg QD

Arm description

In the 15-month Active Therapy Extension Phase, each subject received 1 tablet of ritlecitinib 50 mg QD and 3 tablets of placebo QD during the initial 4-week period, and then received 1 tablet of ritlecitinib 50 mg QD during the remainder of this treatment phase.

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

One tablet of ritlecitinib 50 mg QD and 3 tablets of placebo QD were received during the initial 4-week period, and 1 tablet of ritlecitinib 50 mg QD was received during the remainder of the Active Therapy Extension Phase.

Ritlecitinib 200/50 mg QD

Arm description

In the 15-month Active Therapy Extension Phase, each subject received ritlecitinib 200 mg QD (50 mg/tablet ×4) during the initial 4-week period, and then received 1 tablet of ritlecitinib 50 mg QD during the remainder of this treatment phase.

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

Ritlecitinib 200 mg QD (50 mg/tablet ×4) was received during the initial 4-week period, and 1 tablet of ritlecitinib 50 mg QD was received during the remainder of the Active Therapy Extension Phase.

Number of subjects in period 2	Ritlecitinib 50 mg QD	Ritlecitinib 200/50 mg QD
Started	32	33
Completed	1	0
Not completed	31	33
Physician decision	1	-
Consent withdrawn by subject	8	8
Remained Ongoing in Active Therapy Extension Phase	20	24
Adverse event, non-fatal	1	-
Lost to follow-up	-	1
Lack of efficacy	1	-

Baseline characteristics

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Reporting group title

Ritlecitinib 200/50 mg QD

Reporting group description

Reporting group title

Placebo

Reporting group description

Reporting group values	Ritlecitinib 200/50 mg QD	Placebo	Total
Number of subjects	36	35	71
Age categorical
Units: Subjects
Adults (18-64 years)	36	35	71
Age Continuous
Units: years
arithmetic mean (standard deviation)	35.1 ( 9.64 )	34.2 ( 8.95 )	-
Sex: Female, Male
Units: Subjects
Female	25	25	50
Male	11	10	21
Race (NIH/OMB)
Units: Subjects
Asian	3	0	3
Black or African American	7	4	11
White	26	28	54
More than one race	0	2	2
Unknown or Not Reported	0	1	1
Baseline Percentage of Nerve Fibers With Axonal Swelling
Units: Percentage of Nerve Fibers
arithmetic mean (standard deviation)	1.8 ( 2.48 )	1.8 ( 2.07 )	-
Baseline Severity of Alopecia Tool (SALT) Scores for Non-AT/AU Subjects
Baseline SALT score for non-alopecia totalis (AT)/alopecia universalis (AU) subjects: SALT was a quantitative assessment of alopecia severity based on scalp terminal hair loss performed by an experienced and qualified rater. Score parameters utilized a visual aid showing the division of the scalp hair into 4 quadrants (back, top of scalp, and both sides), with each of 4 quadrants given an accurate determination of the % of scalp surface area covered, representing 24%, 40%, 18%, and 18% of the total scalp surface area, respectively. Higher SALT score represents greater amount of hair loss.
Units: Units on a scale
arithmetic mean (standard deviation)	59.6 ( 30.31 )	53.7 ( 24.18 )	-
Baseline Intraepidermal Nerve Fiber Density (IENFD)
Units: fibers/mm
arithmetic mean (standard deviation)	10.2 ( 3.81 )	11.0 ( 3.95 )	-

Subject analysis set title

Ritlecitinib 200/50/50 mg QD

Subject analysis set type

Full analysis

Subject analysis set description

Subjects in 2 treatment periods: In the 9-month Placebo-Controlled Phase, each subject received ritlecitinib 200 mg QD (50 mg/tablet ×4) during the initial 4-week period, and received 1 tablet of ritlecitinib 50 mg QD during the remainder of this treatment phase. In the 15-month Active Therapy Extension Phase, each subject received 1 tablet of ritlecitinib 50 mg QD and 3 tablets of placebo QD during the initial 4-week period, and then received 1 tablet of ritlecitinib 50 mg QD during the remainder of this treatment phase.

Subject analysis set title

Placebo -> Ritlecitinib 200/50 mg QD

Subject analysis set type

Full analysis

Subject analysis set description

Subjects in 2 treatment periods: In the 9-month Placebo-Controlled Phase, each subject received a total of 4 tablets of placebo QD during the initial 4-week period, and received 1 tablet of placebo QD during the remainder of this treatment phase. In the 15-month Active Therapy Extension Phase, each subject received ritlecitinib 200 mg QD (50 mg/tablet ×4) during the initial 4-week period, and then received 1 tablet of ritlecitinib 50 mg QD during the remainder of this treatment phase.

Subject analysis sets values	Ritlecitinib 200/50/50 mg QD	Placebo -> Ritlecitinib 200/50 mg QD
Number of subjects	36	35
Age categorical
Units: Subjects
Adults (18-64 years)	36	35
Age Continuous
Units: years
arithmetic mean (standard deviation)	35.1 ( 9.64 )	34.2 ( 8.95 )
Sex: Female, Male
Units: Subjects
Female	25	25
Male	11	10
Race (NIH/OMB)
Units: Subjects
Asian	3	0
Black or African American	7	4
White	26	28
More than one race	0	2
Unknown or Not Reported	0	1
Baseline Percentage of Nerve Fibers With Axonal Swelling
Units: Percentage of Nerve Fibers
arithmetic mean (standard deviation)	1.8 ( 2.48 )	1.8 ( 2.07 )
Baseline Severity of Alopecia Tool (SALT) Scores for Non-AT/AU Subjects
Baseline SALT score for non-alopecia totalis (AT)/alopecia universalis (AU) subjects: SALT was a quantitative assessment of alopecia severity based on scalp terminal hair loss performed by an experienced and qualified rater. Score parameters utilized a visual aid showing the division of the scalp hair into 4 quadrants (back, top of scalp, and both sides), with each of 4 quadrants given an accurate determination of the % of scalp surface area covered, representing 24%, 40%, 18%, and 18% of the total scalp surface area, respectively. Higher SALT score represents greater amount of hair loss.
Units: Units on a scale
arithmetic mean (standard deviation)	59.6 ( 30.31 )	53.7 ( 24.18 )
Baseline Intraepidermal Nerve Fiber Density (IENFD)
Units: fibers/mm
arithmetic mean (standard deviation)	10.2 ( 3.81 )	11.0 ( 3.95 )

End points

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Reporting group title

Ritlecitinib 200/50 mg QD

Reporting group description

Reporting group title

Placebo

Reporting group description

Reporting group title

Ritlecitinib 50 mg QD

Reporting group description

Reporting group title

Ritlecitinib 200/50 mg QD

Reporting group description

Subject analysis set title

Ritlecitinib 200/50/50 mg QD

Subject analysis set type

Full analysis

Subject analysis set description

Subject analysis set title

Placebo -> Ritlecitinib 200/50 mg QD

Subject analysis set type

Full analysis

Subject analysis set description

Primary: Change From Baseline in I-V Interwave Latency on Brainstem Auditory Evoked Potentials (BAEP) at a Stimulus Intensity of 80 dB From the Right Side at Month 9

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End point title

Change From Baseline in I-V Interwave Latency on Brainstem Auditory Evoked Potentials (BAEP) at a Stimulus Intensity of 80 dB From the Right Side at Month 9

End point description

BAEP interwave I-V latency was the primary endpoint for this study. High-intensity stimulation (80dB) was used. Subjects had BAEP evaluations performed at the same evaluation center, by the same audiology professional using the same equipment, during the study. Audiology and BAEP evaluations were done on the same day, with audiology assessment first. If they could not be done on the same day, assessments had to be within 7 days of each other. A central reader was used for BAEP to confirm that locally read BAEP waves were labelled appropriately and at their peak so that latency were accurate. Central reading also ensured consistency in BAEP interpretation. Analysis population included all subjects who received at least 1 dose of study intervention. Number of subjects analyzed signifies number of subjects who were evaluable for this endpoint.

End point type

Primary

End point timeframe

Baseline, Month 9 (Baseline was defined as the last non-missing measurement obtained before the first dose in the placebo-controlled phase)

End point values	Ritlecitinib 200/50 mg QD	Placebo
Number of subjects analysed	31	32
Units: Millisecond (ms)
least squares mean (confidence interval 95%)
Month 9	0.011 (-0.043 to 0.065)	-0.010 (-0.063 to 0.043)

LS Mean Difference (Ritlecitinib vs Placebo)

Statistical analysis description

Linear mixed-effects model with baseline, treatment group, visit (Month 6 and Month 9) and treatment group by visit interaction as fixed effects, and subject as a random effect with an unstructured covariance matrix assumption was used.

Comparison groups

Ritlecitinib 200/50 mg QD v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

Method

Mixed models analysis

Parameter type

Least Squares Mean Difference

Point estimate

0.021

Confidence interval

level

95%

sides

2-sided

lower limit

-0.056

upper limit

0.097

Variability estimate

Standard error of the mean

Dispersion value

0.0382

Primary: Change From Baseline in I-V Interwave Latency on BAEP at a Stimulus Intensity of 80 dB From the Left Side at Month 9

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End point title

Change From Baseline in I-V Interwave Latency on BAEP at a Stimulus Intensity of 80 dB From the Left Side at Month 9

End point description

End point type

Primary

End point timeframe

Baseline, Month 9 (Baseline was defined as the last non-missing measurement obtained before the first dose in the placebo-controlled phase)

End point values	Ritlecitinib 200/50 mg QD	Placebo
Number of subjects analysed	31	32
Units: ms
least squares mean (confidence interval 95%)
Month 9	0.031 (-0.012 to 0.075)	0.022 (-0.020 to 0.065)

LS Mean Difference (Ritlecitinib vs Placebo)

Statistical analysis description

Comparison groups

Ritlecitinib 200/50 mg QD v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

Method

Mixed models analysis

Parameter type

Least Squares Mean Difference

Point estimate

0.009

Confidence interval

level

95%

sides

2-sided

lower limit

-0.052

upper limit

0.07

Variability estimate

Standard error of the mean

Dispersion value

0.0307

Secondary: Change From Baseline in I-V Interwave Latency on BAEP at 80 dB From the Right Side at Month 6

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End point title

Change From Baseline in I-V Interwave Latency on BAEP at 80 dB From the Right Side at Month 6

End point description

High-intensity stimulation (80dB) was used. Subjects had BAEP evaluations performed at the same evaluation center, by the same audiology professional using the same equipment, during the study. Audiology and BAEP evaluations were done on the same day, with audiology assessment first. If they could not be done on the same day, assessments had to be within 7 days of each other. A central reader was used for BAEP to confirm that locally read BAEP waves were labelled appropriately and at their peak so that latency were accurate. Central reading also ensured consistency in BAEP interpretation. Analysis population included all subjects who received at least 1 dose of study intervention. Number of subjects analyzed signifies number of subjects who were evaluable for this endpoint.

End point type

Secondary

End point timeframe

Baseline, Month 6 (Baseline was defined as the last non-missing measurement obtained before the first dose in the placebo-controlled phase)

End point values	Ritlecitinib 200/50 mg QD	Placebo
Number of subjects analysed	34	34
Units: ms
least squares mean (confidence interval 95%)
Month 6	-0.030 (-0.072 to 0.011)	-0.024 (-0.065 to 0.017)

LS Mean Difference (Ritlecitinib vs Placebo)

Statistical analysis description

Comparison groups

Ritlecitinib 200/50 mg QD v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

Method

Mixed models analysis

Parameter type

Least Squares Mean Difference

Point estimate

-0.006

Confidence interval

level

95%

sides

2-sided

lower limit

-0.065

upper limit

0.053

Variability estimate

Standard error of the mean

Dispersion value

0.0297

Secondary: Change From Baseline in I-V Interwave Latency on BAEP at 80 dB From the Right Side at Month 9E and 15E

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End point title

Change From Baseline in I-V Interwave Latency on BAEP at 80 dB From the Right Side at Month 9E and 15E

End point description

End point type

Secondary

End point timeframe

Baseline, Months 9E and 15E (Month 9/15 in the Active Therapy Extension Phase). Baseline was defined as the last non-missing measurement obtained before the first dose in the Placebo-controlled Phase.

End point values	Ritlecitinib 200/50/50 mg QD	Placebo -> Ritlecitinib 200/50 mg QD
Number of subjects analysed	36	35
Units: millisecond (ms)
arithmetic mean (standard deviation)
Month 9E (n=25, 30)	0.010 ( 0.1233 )	-0.033 ( 0.2448 )
Month 15E (n=24, 28)	0.051 ( 0.1188 )	0.034 ( 0.2224 )

No statistical analyses for this end point

Secondary: Change From Baseline in I-V Interwave Latency on BAEP at 80 dB From the Left Side at Month 6

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End point title

Change From Baseline in I-V Interwave Latency on BAEP at 80 dB From the Left Side at Month 6

End point description

End point type

Secondary

End point timeframe

Baseline, Month 6 (Baseline was defined as the last non-missing measurement obtained before the first dose in the placebo-controlled phase)

End point values	Ritlecitinib 200/50 mg QD	Placebo
Number of subjects analysed	34	34
Units: ms
least squares mean (confidence interval 95%)
Month 6	0.021 (-0.011 to 0.054)	-0.020 (-0.053 to 0.012)

LS Mean Difference (Ritlecitinib vs Placebo)

Statistical analysis description

Comparison groups

Ritlecitinib 200/50 mg QD v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

Method

Mixed models analysis

Parameter type

Least Squares Mean Difference

Point estimate

0.042

Confidence interval

level

95%

sides

2-sided

lower limit

-0.005

upper limit

0.088

Variability estimate

Standard error of the mean

Dispersion value

0.0235

Secondary: Change From Baseline in I-V Interwave Latency on BAEP at 80 dB From the Left Side at Month 9E and 15E

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End point title

Change From Baseline in I-V Interwave Latency on BAEP at 80 dB From the Left Side at Month 9E and 15E

End point description

End point type

Secondary

End point timeframe

Baseline, Month 9E and 15E (Month 9/15 in the Active Therapy Extension Phase). Baseline was defined as the last non-missing measurement obtained before the first dose in the Placebo-controlled Phase.

End point values	Ritlecitinib 200/50/50 mg QD	Placebo -> Ritlecitinib 200/50 mg QD
Number of subjects analysed	36	35
Units: millisecond (ms)
arithmetic mean (standard deviation)
Month 9E (n=25, 30)	0.006 ( 0.1109 )	0.056 ( 0.1694 )
Month 15E (n=24, 28)	0.024 ( 0.1044 )	0.017 ( 0.2339 )

No statistical analyses for this end point

Secondary: Change From Baseline in Percentage of Intra-epidermal Nerve Fiber (IENF) With Axonal Swelling in Skin Punch Biopsies at Month 9

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End point title

Change From Baseline in Percentage of Intra-epidermal Nerve Fiber (IENF) With Axonal Swelling in Skin Punch Biopsies at Month 9

End point description

The endpoint “axonal dystrophy” referred to the percentage of IENF with axonal swellings. Axonal swellings were evaluated in peripheral skin punch biopsies from the distal part of lower extremities. Axonal swellings were counted by axon. Any IENF with single or multiple swellings was counted as a single event, ie, a single axon with axonal swellings. For each subject, data were reported as the percentage of IENF with any number of swellings. IENF was assessed at Day 1 and Month 9. Subjects who had entered the Active Therapy Extension Phase at Month 6 had a skin punch biopsy taken at Month 6 for IENF assessments instead of at Month 9. The skin biopsy must have been collected before the start of Active Therapy Extension Phase. Analysis population included all subjects who received at least 1 dose of study intervention. Number of subjects analyzed signifies number of subjects who were evaluable for this endpoint.

End point type

Secondary

End point timeframe

Baseline, Month 9 (Month 6 for the 2 subjects who entered the Active Therapy Extension Phase at Month 6). Baseline was defined as the last non-missing measurement obtained before the first dose in the placebo-controlled phase.

End point values	Ritlecitinib 200/50 mg QD	Placebo
Number of subjects analysed	32	33
Units: Percentage of Nerve Fibers
median (inter-quartile range (Q1-Q3))
End of Placebo-controlled Phase (EOP)	0.0 (-0.5 to 2.5)	0.0 (-2.0 to 1.0)

No statistical analyses for this end point

Secondary: Change From Baseline in Percentage of IENFs With Axonal Swelling in Skin Punch Biopsies at Month 15E

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End point title

Change From Baseline in Percentage of IENFs With Axonal Swelling in Skin Punch Biopsies at Month 15E

End point description

The endpoint “axonal dystrophy” referred to the percentage of IENF with axonal swellings. Axonal swellings were evaluated in peripheral skin punch biopsies from the distal part of lower extremities. Axonal swellings were counted by axon. Any IENF with single or multiple swellings was counted as a single event, ie, a single axon with axonal swellings. For each subject, data were reported as the percentage of IENF with any number of swellings. Analysis population included all subjects who received at least 1 dose of study intervention. Number of subjects analyzed signifies number of subjects who were evaluable for this endpoint. n = x, y in the following table represents the number of evaluable subjects in the Ritlecitinib 200/50/50 mg QD and Placebo->Ritlecitinib 200/50 mg QD arms, respectively.

End point type

Secondary

End point timeframe

Baseline, Month 15E (Month 15 in the Active Therapy Extension Phase). Baseline was defined as the last non-missing measurement obtained before the first dose in the placebo-controlled phase.

End point values	Ritlecitinib 200/50/50 mg QD	Placebo -> Ritlecitinib 200/50 mg QD
Number of subjects analysed	36	35
Units: Percentage of Nerve Fibers
median (inter-quartile range (Q1-Q3))
Month 15E (n=22, 25)	0 (-1.0 to 0)	0 (-1.0 to 0)

No statistical analyses for this end point

Secondary: Change From Baseline in Intraepidermal Nerve Fiber Density (IENFD) in Skin Punch Biopsies at Month 9

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End point title

Change From Baseline in Intraepidermal Nerve Fiber Density (IENFD) in Skin Punch Biopsies at Month 9

End point description

IENFD was evaluated in peripheral skin punch biopsies from the distal part of lower extremities. IENFD was measured by counting the number of fibers and fiber branches that independently crossed the dermal-epidermal barrier (basement membrane). Secondary branching was excluded from quantification and fragments were not counted. The length of the histology section was measured (mm) and the linear epidermal nerve fiber density was reported as number of intraepidermal nerve fibers/mm. Analysis population included all subjects who received at least 1 dose of study intervention. Number of subjects analyzed signifies number of subjects who were evaluable for this endpoint.

End point type

Secondary

End point timeframe

End point values	Ritlecitinib 200/50 mg QD	Placebo
Number of subjects analysed	32	33
Units: fibers/mm
arithmetic mean (standard deviation)
End of Placebo-controlled Phase (EOP)	-0.4 ( 3.90 )	-0.2 ( 2.72 )

No statistical analyses for this end point

Secondary: Change From Baseline in IENFD in Skin Punch Biopsies at Month 15E

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End point title

Change From Baseline in IENFD in Skin Punch Biopsies at Month 15E

End point description

End point type

Secondary

End point timeframe

Baseline, Month 15E (Month 15 in the Active Therapy Extension Phase). Baseline was defined as the last non-missing measurement obtained before the first dose in the placebo-controlled phase.

End point values	Ritlecitinib 200/50/50 mg QD	Placebo -> Ritlecitinib 200/50 mg QD
Number of subjects analysed	36	35
Units: fibers/mm
arithmetic mean (standard deviation)
Month 15E (n=22, 25)	0.2 ( 2.85 )	-1.1 ( 3.51 )

No statistical analyses for this end point

Secondary: Change From Baseline in Amplitude of Wave V on BAEP at a Stimulus Intensity of 80 dB From the Right Side at Month 6 and Month 9

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End point title

Change From Baseline in Amplitude of Wave V on BAEP at a Stimulus Intensity of 80 dB From the Right Side at Month 6 and Month 9

End point description

High-intensity stimulation (80dB) was used. Subjects had BAEP evaluations performed at the same evaluation center, by the same audiology professional using the same equipment, during the study. Audiology and BAEP evaluations were done on the same day, with audiology assessment first. If they could not be done on the same day, assessments had to be within 7 days of each other. A central reader was used for BAEP to confirm that locally read BAEP waves were labelled appropriately and at their peak so that amplitude data were accurate. Central reading also ensured consistency in BAEP interpretation. Analysis population included all subjects who received at least 1 dose of study intervention. Number of subjects analyzed signifies number of subjects who were evaluable for this endpoint. n = x, y in the following table represents the number of evaluable subjects in the Ritlecitinib 200/50 mg QD and Placebo arms, respectively.

End point type

Secondary

End point timeframe

Baseline, Month 6 and Month 9 (Baseline was defined as the last non-missing measurement obtained before the first dose in the placebo-controlled phase)

End point values	Ritlecitinib 200/50 mg QD	Placebo
Number of subjects analysed	34	34
Units: Microvolts (μV)
least squares mean (confidence interval 95%)
Month 6 (n = 34, 34)	-0.031 (-0.063 to 0)	-0.017 (-0.048 to 0.015)
Month 9 (n = 31, 32)	-0.051 (-0.085 to -0.018)	0.008 (-0.025 to 0.041)

LS Mean Difference (Ritlecitinib vs Placebo)

Statistical analysis description

Month 9

Comparison groups

Ritlecitinib 200/50 mg QD v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority ^[1]

Method

Mixed models analysis

Parameter type

Least Squares Mean Difference

Point estimate

-0.06

Confidence interval

level

95%

sides

2-sided

lower limit

-0.107

upper limit

-0.012

Variability estimate

Standard error of the mean

Dispersion value

0.0236

Notes
[1] - Linear mixed-effects model with baseline, treatment group, visit (Month 6 and Month 9) and treatment group by visit interaction as fixed effects, and subject as a random effect with an unstructured covariance matrix assumption was used.

LS Mean Difference (Ritlecitinib vs Placebo)

Statistical analysis description

Month 6

Comparison groups

Ritlecitinib 200/50 mg QD v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority ^[2]

Method

Mixed models analysis

Parameter type

Least Squares Mean Difference

Point estimate

-0.015

Confidence interval

level

95%

sides

2-sided

lower limit

-0.059

upper limit

0.03

Variability estimate

Standard error of the mean

Dispersion value

0.0224

Notes
[2] - Linear mixed-effects model with baseline, treatment group, visit (Month 6 and Month 9) and treatment group by visit interaction as fixed effects, and subject as a random effect with an unstructured covariance matrix assumption was used.

Secondary: Change From Baseline in Amplitude of Wave V on BAEP at a Stimulus Intensity of 80 dB From the Right Side at Month 9E and 15E

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End point title

Change From Baseline in Amplitude of Wave V on BAEP at a Stimulus Intensity of 80 dB From the Right Side at Month 9E and 15E

End point description

High-intensity stimulation (80dB) was used. Subjects had BAEP evaluations performed at the same evaluation center, by the same audiology professional using the same equipment, during the study. Audiology and BAEP evaluations were done on the same day, with audiology assessment first. If they could not be done on the same day, assessments had to be within 7 days of each other. A central reader was used for BAEP to confirm that locally read BAEP waves were labelled appropriately and at their peak so that amplitude data were accurate. Central reading also ensured consistency in BAEP interpretation. Analysis population included all subjects who received at least 1 dose of study intervention. Number of subjects analyzed signifies number of subjects who were evaluable for this endpoint. n = x, y in the following table represents the number of evaluable subjects in the Ritlecitinib 200/50/50 mg QD and Placebo->Ritlecitinib 200/50 mg QD arms, respectively.

End point type

Secondary

End point timeframe

Baseline, Month 9E and 15E (Month 9 and 15 in the Active Therapy Extension Phase). Baseline was defined as the last non-missing measurement obtained before the first dose in the placebo-controlled phase.

End point values	Ritlecitinib 200/50/50 mg QD	Placebo -> Ritlecitinib 200/50 mg QD
Number of subjects analysed	36	35
Units: μV
arithmetic mean (standard deviation)
Month 9E (n = 25, 30)	-0.052 ( 0.1109 )	-0.025 ( 0.1649 )
Month 15E (n = 24, 28)	-0.065 ( 0.1513 )	-0.042 ( 0.1214 )

No statistical analyses for this end point

Secondary: Change From Baseline in Amplitude of Wave V on BAEP at a Stimulus Intensity of 80 dB From the Left Side at Month 6 and Month 9

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End point title

Change From Baseline in Amplitude of Wave V on BAEP at a Stimulus Intensity of 80 dB From the Left Side at Month 6 and Month 9

End point description

High-intensity stimulation (80dB) was used. Subjects had BAEP evaluations performed at the same evaluation center, by the same audiology professional using the same equipment, during the study. Audiology and BAEP evaluations were done on the same day, with audiology assessment first. If they could not be done on the same day, assessments had to be within 7 days of each other. A central reader was used for BAEP to confirm that locally read BAEP waves were labelled appropriately and at their peak so that amplitude data were accurate. Central reading also ensured consistency in BAEP interpretation. Analysis population included all subjects who received at least 1 dose of study intervention. Number of subjects analyzed signifies number of subjects who were evaluable for this endpoint. n = x, y in the following table represents the number of evaluable subjects in the Ritlecitinib 200/50 mg QD and Placebo arms, respectively.

End point type

Secondary

End point timeframe

Baseline, Month 6 and Month 9 (Baseline was defined as the last non-missing measurement obtained before the first dose in the placebo-controlled phase)

End point values	Ritlecitinib 200/50 mg QD	Placebo
Number of subjects analysed	34	34
Units: Microvolts (μV)
least squares mean (confidence interval 95%)
Month 6 (n = 34, 34)	-0.047 (-0.082 to -0.013)	-0.019 (-0.053 to 0.015)
Month 9 (n = 31, 32)	-0.045 (-0.082 to -0.008)	-0.049 (-0.085 to -0.012)

LS Mean Difference (Ritlecitinib vs Placebo)

Statistical analysis description

Month 9

Comparison groups

Ritlecitinib 200/50 mg QD v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

^[3]

Method

Mixed models analysis

Parameter type

Least Squares Mean Difference

Point estimate

0.003

Confidence interval

level

95%

sides

2-sided

lower limit

-0.049

upper limit

0.056

Variability estimate

Standard error of the mean

Dispersion value

0.0261

Notes
[3] - Linear mixed-effects model with baseline, treatment group, visit (Month 6 and Month 9) and treatment group by visit interaction as fixed effects, and subject as a random effect with an unstructured covariance matrix assumption was used.

LS Mean Difference (Ritlecitinib vs Placebo)

Statistical analysis description

Month 6

Comparison groups

Ritlecitinib 200/50 mg QD v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

^[4]

Method

Mixed models analysis

Parameter type

Least Squares Mean Difference

Point estimate

-0.028

Confidence interval

level

95%

sides

2-sided

lower limit

-0.076

upper limit

0.02

Variability estimate

Standard error of the mean

Dispersion value

0.0242

Notes
[4] - Linear mixed-effects model with baseline, treatment group, visit (Month 6 and Month 9) and treatment group by visit interaction as fixed effects, and subject as a random effect with an unstructured covariance matrix assumption was used.

Secondary: Change From Baseline in Amplitude of Wave V on BAEP at a Stimulus Intensity of 80 dB From the Left Side at Month 9E and 15E

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End point title

Change From Baseline in Amplitude of Wave V on BAEP at a Stimulus Intensity of 80 dB From the Left Side at Month 9E and 15E

End point description

High-intensity stimulation (80dB) was used. Subjects had BAEP evaluations performed at the same evaluation center, by the same audiology professional using the same equipment, during the study. Audiology and BAEP evaluations were done on the same day, with audiology assessment first. If they could not be done on the same day, assessments had to be within 7 days of each other. A central reader was used for BAEP to confirm that locally read BAEP waves were labelled appropriately and at their peak so that amplitude data were accurate. Central reading also ensured consistency in BAEP interpretation. Analysis population included all subjects who received at least 1 dose of study intervention. Number of subjects analyzed signifies number of subjects who were evaluable for this endpoint. n = x, y in the following table represents the number of evaluable subjects in the Ritlecitinib 200/50/50 mg QD and Placebo->Ritlecitinib 200/50 mg QD arms, respectively.

End point type

Secondary

End point timeframe

End point values	Ritlecitinib 200/50/50 mg QD	Placebo -> Ritlecitinib 200/50 mg QD
Number of subjects analysed	36	35
Units: μV
arithmetic mean (standard deviation)
Month 9E (25, 30)	-0.058 ( 0.1209 )	-0.025 ( 0.1307 )
Month 15E (n = 24, 28)	-0.047 ( 0.1314 )	-0.023 ( 0.1217 )

No statistical analyses for this end point

Secondary: Number of Subjects With Absence of Wave V on BAEP at Stimulus Intensities Ranging From 80 dB to 40 dB From Right Side up to Month 9

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End point title

Number of Subjects With Absence of Wave V on BAEP at Stimulus Intensities Ranging From 80 dB to 40 dB From Right Side up to Month 9

End point description

Absence of wave V on BAEP at stimulus intensities ranging from 80dB to 40dB were summarized descriptively using number of subjects by treatment group at each intensity level. Analysis population included all subjects who received at least 1 dose of study intervention. Number of subjects analyzed signifies number of subjects who were evaluable for this endpoint. n = x, y in the following table represents the number of subjects at each visit in the Ritlecitinib 200/50 mg QD and Placebo arms, respectively. One subject had fluctuating absence of Wave V at lower intensities. Hearing sensitivity remained normal from screening through Month 9. The case was reviewed by a panel of neuroaudiology experts who concluded that there was no evidence of neural transmission abnormality in the auditory nerve or auditory brainstem and that the likely explanation for the absence of Wave V was that the evoked response amplitude was too small for it to be identified within the electroencephalogram (EEG).

End point type

Secondary

End point timeframe

Baseline, Month 6 and Month 9

End point values	Ritlecitinib 200/50 mg QD	Placebo
Number of subjects analysed	36	35
Units: Subjects
Baseline-80dB normal hearing level (nHL) (n=36,35)	0	0
Baseline - 70 dB nHL (n = 36, 35)	0	0
Baseline - 60 dB nHL (n = 36, 35)	0	0
Baseline - 50 dB nHL (n = 36, 35)	0	0
Baseline - 40 dB nHL (n = 36, 35)	0	0
Month 6 - 80 dB nHL (n = 34, 34)	0	0
Month 6 - 70 dB nHL (n = 34, 34)	0	0
Month 6 - 60 dB nHL (n = 34, 34)	0	0
Month 6 - 50 dB nHL (n = 34, 34)	0	0
Month 6 - 40 dB nHL (n = 34, 34)	0	0
Month 9 - 80 dB nHL (n = 31, 32)	0	0
Month 9 - 70 dB nHL (n = 31, 32)	0	0
Month 9 - 60 dB nHL (n = 31, 32)	0	0
Month 9 - 50 dB nHL (n = 31, 32)	0	0
Month 9 - 40 dB nHL (n = 31, 32)	1	0

No statistical analyses for this end point

Secondary: Number of Subjects With Absence of Wave V on BAEP at Stimulus Intensities Ranging From 80 dB to 40 dB From Right Side at Baseline, Month 3E, 6E, 9E and 15E

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End point title

Number of Subjects With Absence of Wave V on BAEP at Stimulus Intensities Ranging From 80 dB to 40 dB From Right Side at Baseline, Month 3E, 6E, 9E and 15E

End point description

Absence of wave V on BAEP at stimulus intensities ranging from 80dB to 40dB were summarized using number of subjects by treatment group at each intensity level. All subjects had Wave V on BAEP present at stimulus intensities ranging from 80 dB to 40 dB up to Month 15E except for 1 subject. Hearing sensitivity remained normal from screening through Month 15E bilaterally. Analysis population included all subjects who received at least 1 dose of study intervention. Number of subjects analyzed signifies number of subjects who were evaluable for this endpoint. n = x, y in the following table represents the number of evaluable subjects in the Ritlecitinib 200/50/50 mg QD and Placebo->Ritlecitinib 200/50 mg QD arms, respectively.

End point type

Secondary

End point timeframe

Baseline, Month 3E, 6E, 9E and 15E (Month 3/6/9/15 in the Active Therapy Extension Phase). Baseline was defined as the last non-missing measurement obtained before the first dose in the Placebo-Controlled Phase.

End point values	Ritlecitinib 200/50/50 mg QD	Placebo -> Ritlecitinib 200/50 mg QD
Number of subjects analysed	36	35
Units: Subjects
Baseline - 80 dB nHL (n=36, 35)	0	0
Baseline - 70 dB nHL (n=36, 35)	0	0
Baseline - 60 dB nHL (n=36, 35)	0	0
Baseline - 50 dB nHL (n=36, 35)	0	0
Baseline - 40 dB nHL (n=36, 35)	0	0
Month 3E - 80 dB nHL (n=1, 0)	0	0
Month 3E - 70 dB nHL (n=1, 0)	0	0
Month 3E - 60 dB nHL (n=1, 0)	0	0
Month 3E - 50 dB nHL (n=1, 0)	0	0
Month 3E - 40 dB nHL (n=1, 0)	0	0
Month 6E - 80 dB nHL (n=1, 1)	0	0
Month 6E - 70 dB nHL (n=1, 1)	0	0
Month 6E - 60 dB nHL (n=1, 1)	0	0
Month 6E - 50 dB nHL (n=1, 1)	0	0
Month 6E - 40 dB nHL (n=1, 1)	0	0
Month 9E - 80 dB nHL (n=25, 30)	0	0
Month 9E - 70 dB nHL (n=25, 30)	0	0
Month 9E - 60 dB nHL (n=25, 30)	0	0
Month 9E - 50 dB nHL (n=25, 30)	1	0
Month 9E - 40 dB nHL (n=25, 30)	1	0
Month 15E - 80 dB nHL (n=24, 28)	0	0
Month 15E - 70 dB nHL (n=24, 28)	0	0
Month 15E - 60 dB nHL (n=24, 28)	0	0
Month 15E - 50 dB nHL (n=24, 28)	0	0
Month 15E - 40 dB nHL (n=24, 28)	0	0

No statistical analyses for this end point

Secondary: Number of Subjects With Absence of Wave V on BAEP at Stimulus Intensities Ranging From 80 dB to 40 dB From Left Side up to Month 9

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End point title

Number of Subjects With Absence of Wave V on BAEP at Stimulus Intensities Ranging From 80 dB to 40 dB From Left Side up to Month 9

End point description

End point type

Secondary

End point timeframe

Baseline, Month 6 and Month 9

End point values	Ritlecitinib 200/50 mg QD	Placebo
Number of subjects analysed	36	35
Units: Subjects
Baseline - 80 dB nHL (n = 36, 35)	0	0
Baseline - 70 dB nHL (n = 36, 35)	0	0
Baseline - 60 dB nHL (n = 36, 35)	0	0
Baseline - 50 dB nHL (n = 36, 35)	0	0
Baseline - 40 dB nHL (n = 36, 35)	0	0
Month 6 - 80 dB nHL (n = 34, 34)	0	0
Month 6 - 70 dB nHL (n = 34, 34)	0	0
Month 6 - 60 dB nHL (n = 34, 34)	0	0
Month 6 - 50 dB nHL (n = 34, 34)	0	0
Month 6 - 40 dB nHL (n = 34, 34)	0	0
Month 9 - 80 dB nHL (n = 31, 32)	0	0
Month 9 - 70 dB nHL (n = 31, 32)	0	0
Month 9 - 60 dB nHL (n = 31, 32)	0	0
Month 9 - 50 dB nHL (n = 31, 32)	0	0
Month 9 - 40 dB nHL (n = 31, 32)	0	0

No statistical analyses for this end point

Secondary: Number of Subjects With Absence of Wave V on BAEP at Stimulus Intensities Ranging From 80 dB to 40 dB From Left Side at Baseline, Month 3E, 6E, 9E and 15E

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End point title

Number of Subjects With Absence of Wave V on BAEP at Stimulus Intensities Ranging From 80 dB to 40 dB From Left Side at Baseline, Month 3E, 6E, 9E and 15E

End point description

Absence of wave V on BAEP at stimulus intensities ranging from 80dB to 40dB were summarized descriptively using number of subjects by treatment group at each intensity level. All participants had Wave V on BAEP present at stimulus intensities ranging from 80 dB to 40 dB up to Month 15E except for 1 subject. Hearing sensitivity remained normal from screening through Month 15E bilaterally. Analysis population included all subjects who received at least 1 dose of study intervention. Number of subjects analyzed signifies number of subjects who were evaluable for this endpoint. n = x, y in the following table represents the number of evaluable subjects in the Ritlecitinib 200/50/50 mg QD and Placebo->Ritlecitinib 200/50 mg QD arms, respectively.

End point type

Secondary

End point timeframe

End point values	Ritlecitinib 200/50/50 mg QD	Placebo -> Ritlecitinib 200/50 mg QD
Number of subjects analysed	36	35
Units: Subjects
Baseline - 80 dB nHL (n=36, 35)	0	0
Baseline - 70 dB nHL (n=36, 35)	0	0
Baseline - 60 dB nHL (n=36, 35)	0	0
Baseline - 50 dB nHL (n=36, 35)	0	0
Baseline - 40 dB nHL (n=36, 35)	0	0
Month 3E - 80 dB nHL (n=1, 0)	0	0
Month 3E - 70 dB nHL (n=1, 0)	0	0
Month 3E - 60 dB nHL (n=1, 0)	0	0
Month 3E - 50 dB nHL (n=1, 0)	0	0
Month 3E - 40 dB nHL (n=1, 0)	0	0
Month 6E - 80 dB nHL (n=1, 1)	0	0
Month 6E - 70 dB nHL (n=1, 1)	0	0
Month 6E - 60 dB nHL (n=1, 1)	0	0
Month 6E - 50 dB nHL (n=1, 1)	0	0
Month 6E - 40 dB nHL (n=1, 1)	0	0
Month 9E - 80 dB nHL (n=25, 30)	0	0
Month 9E - 70 dB nHL (n=25, 30)	0	0
Month 9E - 60 dB nHL (n=25, 30)	0	0
Month 9E - 50 dB nHL (n=25, 30)	0	0
Month 9E - 40 dB nHL (n=25, 30)	0	0
Month 15E - 80 dB nHL (n=24, 28)	0	0
Month 15E - 70 dB nHL (n=24, 28)	0	0
Month 15E - 60 dB nHL (n=24, 28)	0	0
Month 15E - 50 dB nHL (n=24, 28)	0	0
Month 15E - 40 dB nHL (n=24, 28)	0	0

No statistical analyses for this end point

Secondary: Number of Subjects With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse events (TESAEs)

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End point title

Number of Subjects With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse events (TESAEs)

End point description

An adverse event (AE) was any untoward medical occurrence in a subject, temporally associated with the use of study intervention, whether or not considered related to the study intervention. Seriousness of an AE was assessed under the criteria of serious adverse event (SAE). An SAE was defined as any untoward medical occurrence that, at any dose: resulted in death; was life-threatening; required inpatient hospitalization or prolongation of existing hospitalization; resulted in persistent disability/incapacity; was a congenital anomaly/birth defect, etc. Treatment-emergent events were with onset date occurring during the on-treatment period. Relatedness to study treatment was determined by the investigator. Analysis population included all subjects taking at least 1 dose of study intervention.

End point type

Secondary

End point timeframe

Approximately 16 months

End point values	Ritlecitinib 200/50 mg QD	Ritlecitinib 50 mg QD	Placebo	Ritlecitinib 200/50 mg QD
Number of subjects analysed	36	32	35	31
Units: Subjects
TEAE (All-Causality)	29	3	22	3
TEAE (Treatment-Related)	9	0	5	1
TESAE (All-Causality)	0	1	1	0
TESAE (Treatment-Related)	0	0	0	0

No statistical analyses for this end point

Secondary: Number of Subjects Who Discontinued From Study Due to Adverse Event (AEs)

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End point title

Number of Subjects Who Discontinued From Study Due to Adverse Event (AEs)

End point description

An AE was any untoward medical occurrence in a subject, temporally associated with the use of study intervention, whether or not considered related to the study intervention. Subjects who had an AE record that indicated that the AE caused the subject to be discontinued from the study. Relatedness to study treatment was determined by the investigator. Analysis population included all subjects taking at least 1 dose of study intervention.

End point type

Secondary

End point timeframe

Approximately 16 months

End point values	Ritlecitinib 200/50 mg QD	Ritlecitinib 50 mg QD	Placebo	Ritlecitinib 200/50 mg QD
Number of subjects analysed	36	32	35	31
Units: Subjects
Due to All-Causality AEs	0	1	0	0
Due to Treatment-Related AEs	0	0	0	0

No statistical analyses for this end point

Secondary: Number of Subjects Who Discontinued Study Drug Due to AE and Continued Study

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End point title

Number of Subjects Who Discontinued Study Drug Due to AE and Continued Study

End point description

An AE was any untoward medical occurrence in a subject, temporally associated with the use of study intervention, whether or not considered related to the study intervention. This Outcome Measures presented the number of subjects who had an AE record that indicated that action taken with study treatment was drug withdrawn but AE did not cause the subject to be discontinued from study. Relatedness to study treatment was determined by the investigator. Analysis population included all subjects taking at least 1 dose of study intervention.

End point type

Secondary

End point timeframe

Approximately 16 months

End point values	Ritlecitinib 200/50 mg QD	Ritlecitinib 50 mg QD	Placebo	Ritlecitinib 200/50 mg QD
Number of subjects analysed	36	32	35	31
Units: Subjects
Due to All-Causality AEs	1	0	0	0
Due to Treatment-Related AEs	0	0	0	0

No statistical analyses for this end point

Secondary: Number of Subjects With Temporary Drug Discontinuation Due to AEs

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End point title

Number of Subjects With Temporary Drug Discontinuation Due to AEs

End point description

An AE was any untoward medical occurrence in a subject, temporally associated with the use of study intervention, whether or not considered related to the study intervention. The number of subjects with temporary drug discontinuation due to both all-causality and treatment-related AEs are presented below. Relatedness to study treatment was determined by the investigator. Analysis population included all subjects taking at least 1 dose of study intervention.

End point type

Secondary

End point timeframe

Approximately 16 months

End point values	Ritlecitinib 200/50 mg QD	Ritlecitinib 50 mg QD	Placebo	Ritlecitinib 200/50 mg QD
Number of subjects analysed	36	32	35	31
Units: Subjects
Due to All-Causality AEs	6	0	1	0
Due to Treatment-Related AEs	3	0	0	0

No statistical analyses for this end point

Secondary: Number of Subjects With Clinically Significant Abnormalities in Vital Signs

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End point title

Number of Subjects With Clinically Significant Abnormalities in Vital Signs

End point description

Oral, tympanic, or axillary temperature, pulse rate, respiratory rate, and blood pressure (BP) were assessed. BP and pulse measurements were assessed in a chair, back supported and arms bared (free of restrictions such as rolled-up sleeves, etc) and supported at heart level. Measurements were taken on the same arm at each visit (preferably non-dominant) with a completely automated device. Pulse rate was measured at approximately the same time as BP for a minimum of 30 seconds. BP and pulse measurements should be preceded by at least 5 minutes of rest for the subject in a quiet setting without distractions (eg, television, cell phones). Subjects refrained from smoking or ingesting caffeine during the 30 minutes preceding the measurements. The clinical significance was determined by the investigator. Analysis population included all subjects taking at least 1 dose of study intervention.

End point type

Secondary

End point timeframe

Approximately 16 months

End point values	Ritlecitinib 200/50 mg QD	Ritlecitinib 50 mg QD	Placebo	Ritlecitinib 200/50 mg QD
Number of subjects analysed	36	32	35	31
Units: Subjects	0	0	0	0

No statistical analyses for this end point

Secondary: Number of Subjects With Clinically Significant Abnormalities in Clinical Laboratory Values

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End point title

Number of Subjects With Clinically Significant Abnormalities in Clinical Laboratory Values

End point description

Safety laboratory assessments included the categories of Hematology, Chemistry, Urinalysis and other tests. The clinical significance was determined by the investigator. Analysis population included all subjects taking at least 1 dose of study intervention.

End point type

Secondary

End point timeframe

Approximately 16 months

End point values	Ritlecitinib 200/50 mg QD	Ritlecitinib 50 mg QD	Placebo	Ritlecitinib 200/50 mg QD
Number of subjects analysed	36	32	35	31
Units: Subjects	0	0	0	0

No statistical analyses for this end point

Secondary: Change From Baseline in Overall Severity of Alopecia Tool (SALT) Scores up to Month 9

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End point title

Change From Baseline in Overall Severity of Alopecia Tool (SALT) Scores up to Month 9

End point description

SALT is a quantitative assessment of AA severity based on scalp terminal hair loss. The overall SALT score included hair loss regardless of etiology (ie, scalp hair loss due to both non-AA and AA) and was collected at study visits. The Overall SALT Score ranged from 0 to 100%, with higher scores representing greater amount of hair loss. Analysis population included all randomized subjects taking at least 1 dose of study intervention. Number of subjects analyzed signifies number of subjects who were evaluable for this endpoint.

End point type

Secondary

End point timeframe

Baseline, Months 3, 6 and 9 (Baseline was defined as the last non-missing measurement obtained before the first dose in the placebo-controlled phase)

End point values	Ritlecitinib 200/50 mg QD	Placebo
Number of subjects analysed	35	35
Units: Units on a scale
least squares mean (confidence interval 95%)
Month 3	-23.0 (-29.70 to -16.25)	-2.7 (-9.39 to 4.06)
Month 6	-35.2 (-44.55 to -25.81)	-5.1 (-14.44 to 4.18)
Month 9	-38.2 (-47.46 to -28.86)	-6.8 (-16.08 to 2.40)

No statistical analyses for this end point

Secondary: Change From Baseline in Overall SALT Scores at Month 3E, 6E, 9E, 12E and 15E

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End point title

Change From Baseline in Overall SALT Scores at Month 3E, 6E, 9E, 12E and 15E

End point description

End point type

Secondary

End point timeframe

Baseline, Month 3E, 6E, 9E, 12E and 15E (Month 3/6/9/12/15 in the Active Therapy Extension Phase). Baseline was defined as the last non-missing measurement obtained before the first dose in the Placebo-Controlled Phase.

End point values	Ritlecitinib 200/50/50 mg QD	Placebo -> Ritlecitinib 200/50 mg QD
Number of subjects analysed	36	35
Units: Units on a scale
arithmetic mean (standard deviation)
Month 3E (n = 32, 33)	-41.7 ( 33.98 )	-38.7 ( 33.49 )
Month 6E (n = 29, 30)	-38.0 ( 29.88 )	-43.4 ( 34.13 )
Month 9E (n = 26, 30)	-38.7 ( 29.90 )	-46.6 ( 34.14 )
Month 12E (n = 25, 29)	-41.6 ( 29.67 )	-48.9 ( 34.77 )
Month 15E (n = 24, 28)	-44.6 ( 28.51 )	-49.9 ( 34.40 )

No statistical analyses for this end point

Secondary: Change From Baseline in Alopecia Areata - Severity of Alopecia Tool (AA-SALT) Score up to Month 9

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End point title

Change From Baseline in Alopecia Areata - Severity of Alopecia Tool (AA-SALT) Score up to Month 9

End point description

SALT is a quantitative assessment of AA severity based on scalp terminal hair loss. AA-SALT is amount of scalp hair loss due to AA. AA-SALT score in Placebo-Controlled Phase = overall SALT score – non-AA SALT score at Month 6 (for those subjects who entered Active Therapy Extension Phase at Month 6) or Month 9 (non-AA SALT: scalp hair loss other than that due to AA). AA-SALT Score ranged from 0 to 100%, with higher scores representing greater amount of hair loss. Analysis population included all randomized subjects taking at least 1 dose of study intervention. Number of subjects analyzed signifies number of subjects who were evaluable for this endpoint. n = x, y below represents the number of evaluable subjects in Ritlecitinib 200/50 mg and Placebo arms, respectively.

End point type

Secondary

End point timeframe

Baseline, Months 3, 6 and 9 (Baseline was defined as the last non-missing measurement obtained before the first dose in the placebo-controlled phase)

End point values	Ritlecitinib 200/50 mg QD	Placebo
Number of subjects analysed	35	35
Units: Units on a scale
least squares mean (confidence interval 95%)
Month 3 (n = 35, 35)	-23.0 (-29.70 to -16.25)	-2.7 (-9.39 to 4.06)
Month 6 (n = 34, 35)	-35.2 (-44.55 to -25.81)	-5.1 (-14.44 to 4.18)
Month 9 (n = 32, 31)	-38.2 (-47.46 to -28.87)	-6.8 (-16.08 to 2.40)

No statistical analyses for this end point

Secondary: Change From Baseline in AA-SALT Score at Month 3E, 6E, 9E, 12E and 15E

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End point title

Change From Baseline in AA-SALT Score at Month 3E, 6E, 9E, 12E and 15E

End point description

SALT is a quantitative assessment of AA severity based on scalp terminal hair loss. AA-SALT is the amount of scalp hair loss due to AA. AA SALT score in Placebo-Controlled Phase = overall SALT score – non-AA SALT score (The non-AA SALT score only took into account scalp hair loss other than that due to AA and was required to be assessed only at Month 9 [or Month 6 for those who entered the Active Therapy Extension Phase at Month 6] in Placebo-Controlled Phase). The AA-SALT Score ranged from 0 to 100%, with higher scores representing greater amount of hair loss. Analysis population included all randomized subjects taking at least 1 dose of study intervention. Number of subjects analyzed signifies number of subjects who were evaluable for this endpoint. n = x, y in the following table represents the number of evaluable subjects in the Ritlecitinib 200/50/50 mg QD and Placebo->Ritlecitinib 200/50 mg QD arms, respectively.

End point type

Secondary

End point timeframe

End point values	Ritlecitinib 200/50/50 mg QD	Placebo -> Ritlecitinib 200/50 mg QD
Number of subjects analysed	36	35
Units: Units on a scale
arithmetic mean (standard deviation)
Month 3E (n = 32, 33)	-41.7 ( 33.97 )	-39.4 ( 32.89 )
Month 6E (n = 29, 30)	-38.0 ( 29.87 )	-44.2 ( 33.37 )
Month 9E (n = 26, 30)	-38.7 ( 29.90 )	-47.4 ( 33.36 )
Month 12E (n = 25, 29)	-41.6 ( 29.70 )	-49.7 ( 33.92 )
Month 15E (n = 24, 28)	-44.8 ( 28.37 )	-50.8 ( 33.49 )

No statistical analyses for this end point

Secondary: Number of Subjects With Patient's Global Impression of Change (PGI-C) Response up to Month 9

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End point title

Number of Subjects With Patient's Global Impression of Change (PGI-C) Response up to Month 9

End point description

The PGI-C asked the subjects to evaluate the improvement or worsening of their AA as compared to the start of the study using a single item, “Since the start of the study, my alopecia areata has: …”. The subjects selected one of seven responses ranging from "greatly improved" to "greatly worsened". This Outcome Measure presented the number of subjects with PGI-C response which was defined as "greatly improved" or "moderately improved". Subjects with missing PGI-C scores were considered as non-responders. Analysis population included all randomized subjects taking at least 1 dose of study intervention.

End point type

Secondary

End point timeframe

Months 1, 3, 6 and 9

End point values	Ritlecitinib 200/50 mg QD	Placebo
Number of subjects analysed	36	35
Units: Subjects
Month 1	4	3
Month 3	20	6
Month 6	21	10
Month 9	19	6

No statistical analyses for this end point

Secondary: Number of Subjects With PGI-C Response at Month 3E, 6E, 9E, 12E and 15E

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End point title

Number of Subjects With PGI-C Response at Month 3E, 6E, 9E, 12E and 15E

End point description

End point type

Secondary

End point timeframe

Month 3E, 6E, 9E, 12E and 15E (Month 3/6/9/12/15 in the Active Therapy Extension Phase)

End point values	Ritlecitinib 200/50/50 mg QD	Placebo -> Ritlecitinib 200/50 mg QD
Number of subjects analysed	36	35
Units: Subjects
Month 3E	22	22
Month 6E	16	20
Month 9E	20	18
Month 12E	17	18
Month 15E	17	18

No statistical analyses for this end point

Adverse events

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Timeframe for reporting adverse events

24 months

Adverse event reporting additional description

Same event may appear as both an AE and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 subject and as non-serious in another subject, or 1 subject may have experienced both serious and non-serious event during the study.

Assessment type

Non-systematic

Dictionary name

MedDRA

Dictionary version

26.0

Reporting group title

Placebo -> Ritlecitinib 200/50 mg QD

Reporting group description

In the 9-month Placebo-Controlled Phase, each subject received a total of 4 tablets of placebo QD during the initial 4-week period, and received 1 tablet of placebo QD during the remainder of this treatment phase. In the 15-month Active Therapy Extension Phase, each subject received ritlecitinib 200 mg QD (50 mg/tablet ×4) during the initial 4-week period, and then received 1 tablet of ritlecitinib 50 mg QD during the remainder of this treatment phase.

Reporting group title

Ritlecitinib 200/50/50 mg QD

Reporting group description

In the 9-Month Placebo-Controlled Phase, each subject received ritlecitinib 200 mg QD (50 mg/tablet ×4) during the initial 4-week period, and received 1 tablet of ritlecitinib 50 mg QD during the remainder of this treatment phase. In the 15-Month Active Therapy Extension Phase, each subject received 1 tablet of ritlecitinib 50 mg QD and 3 tablets of placebo QD during the initial 4-week period, and then received 1 tablet of ritlecitinib 50 mg QD during the remainder of this phase.

Serious adverse events	Placebo -> Ritlecitinib 200/50 mg QD	Ritlecitinib 200/50/50 mg QD
Total subjects affected by serious adverse events
subjects affected / exposed	2 / 35 (5.71%)	3 / 36 (8.33%)
number of deaths (all causes)	0	0
number of deaths resulting from adverse events	0	0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Intraductal proliferative breast lesion
subjects affected / exposed	0 / 35 (0.00%)	1 / 36 (2.78%)
occurrences causally related to treatment / all	0 / 0	1 / 1
deaths causally related to treatment / all	0 / 0	0 / 0
Injury, poisoning and procedural complications
Humerus fracture
subjects affected / exposed	1 / 35 (2.86%)	0 / 36 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Vascular disorders
Takayasu's arteritis
subjects affected / exposed	0 / 35 (0.00%)	1 / 36 (2.78%)
occurrences causally related to treatment / all	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0
Hepatobiliary disorders
Biliary colic
subjects affected / exposed	0 / 35 (0.00%)	1 / 36 (2.78%)
occurrences causally related to treatment / all	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0
Skin and subcutaneous tissue disorders
Chronic pigmented purpura
subjects affected / exposed	1 / 35 (2.86%)	0 / 36 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0

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

Non-serious adverse events	Placebo -> Ritlecitinib 200/50 mg QD	Ritlecitinib 200/50/50 mg QD
Total subjects affected by non serious adverse events
subjects affected / exposed	24 / 35 (68.57%)	27 / 36 (75.00%)
Investigations
Blood creatine phosphokinase increased
subjects affected / exposed	0 / 35 (0.00%)	2 / 36 (5.56%)
occurrences all number	0	2
Injury, poisoning and procedural complications
Limb injury
subjects affected / exposed	2 / 35 (5.71%)	0 / 36 (0.00%)
occurrences all number	2	0
Joint dislocation
subjects affected / exposed	2 / 35 (5.71%)	0 / 36 (0.00%)
occurrences all number	2	0
Vascular disorders
Hypertension
subjects affected / exposed	0 / 35 (0.00%)	3 / 36 (8.33%)
occurrences all number	0	3
Nervous system disorders
Dizziness
subjects affected / exposed	1 / 35 (2.86%)	3 / 36 (8.33%)
occurrences all number	1	3
Headache
subjects affected / exposed	4 / 35 (11.43%)	4 / 36 (11.11%)
occurrences all number	4	5
Hypoaesthesia
subjects affected / exposed	3 / 35 (8.57%)	2 / 36 (5.56%)
occurrences all number	4	4
Paraesthesia
subjects affected / exposed	1 / 35 (2.86%)	4 / 36 (11.11%)
occurrences all number	1	4
Blood and lymphatic system disorders
Lymphadenopathy
subjects affected / exposed	2 / 35 (5.71%)	0 / 36 (0.00%)
occurrences all number	2	0
Ear and labyrinth disorders
Tinnitus
subjects affected / exposed	0 / 35 (0.00%)	2 / 36 (5.56%)
occurrences all number	0	2
Vertigo
subjects affected / exposed	2 / 35 (5.71%)	0 / 36 (0.00%)
occurrences all number	2	0
Gastrointestinal disorders
Vomiting
subjects affected / exposed	1 / 35 (2.86%)	4 / 36 (11.11%)
occurrences all number	1	4
Nausea
subjects affected / exposed	3 / 35 (8.57%)	1 / 36 (2.78%)
occurrences all number	4	1
Diarrhoea
subjects affected / exposed	0 / 35 (0.00%)	3 / 36 (8.33%)
occurrences all number	0	4
Respiratory, thoracic and mediastinal disorders
Nasal congestion
subjects affected / exposed	1 / 35 (2.86%)	2 / 36 (5.56%)
occurrences all number	1	2
Rhinitis allergic
subjects affected / exposed	0 / 35 (0.00%)	2 / 36 (5.56%)
occurrences all number	0	2
Skin and subcutaneous tissue disorders
Acne
subjects affected / exposed	3 / 35 (8.57%)	2 / 36 (5.56%)
occurrences all number	3	2
Pruritus
subjects affected / exposed	2 / 35 (5.71%)	0 / 36 (0.00%)
occurrences all number	4	0
Psychiatric disorders
Insomnia
subjects affected / exposed	0 / 35 (0.00%)	2 / 36 (5.56%)
occurrences all number	0	2
Musculoskeletal and connective tissue disorders
Arthralgia
subjects affected / exposed	1 / 35 (2.86%)	2 / 36 (5.56%)
occurrences all number	1	2
Back pain
subjects affected / exposed	1 / 35 (2.86%)	2 / 36 (5.56%)
occurrences all number	1	2
Infections and infestations
COVID-19
subjects affected / exposed	7 / 35 (20.00%)	8 / 36 (22.22%)
occurrences all number	8	8
Acne pustular
subjects affected / exposed	0 / 35 (0.00%)	4 / 36 (11.11%)
occurrences all number	0	5
Nasopharyngitis
subjects affected / exposed	3 / 35 (8.57%)	4 / 36 (11.11%)
occurrences all number	4	6
Urinary tract infection
subjects affected / exposed	3 / 35 (8.57%)	2 / 36 (5.56%)
occurrences all number	4	2
Upper respiratory tract infection
subjects affected / exposed	3 / 35 (8.57%)	3 / 36 (8.33%)
occurrences all number	4	3
Tonsillitis
subjects affected / exposed	0 / 35 (0.00%)	3 / 36 (8.33%)
occurrences all number	0	3
Sinusitis
subjects affected / exposed	2 / 35 (5.71%)	2 / 36 (5.56%)
occurrences all number	2	2
Gastroenteritis
subjects affected / exposed	0 / 35 (0.00%)	2 / 36 (5.56%)
occurrences all number	0	2
Pharyngitis
subjects affected / exposed	0 / 35 (0.00%)	3 / 36 (8.33%)
occurrences all number	0	3
Laryngitis
subjects affected / exposed	2 / 35 (5.71%)	0 / 36 (0.00%)
occurrences all number	2	0

More information

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Were there any global substantial amendments to the protocol? Yes

20 Apr 2021

Updated Schedule of Activities and Section 4.1 Study Design; revised Inclusion and Exclusion Criteria; Section 6.2.1 Preparation and Dispensing was updated to add that at the EOP visit; updated Section 6.5.3 Vaccinations, Section 8.2.6 Audiological Evaluations, Section 8.2.7 Brainstem Auditory Evoked Potential Evaluations, Section 8.2.10.1.3 Subsequent Tuberculosis Testing, Section 8.2.11.1. Columbia Suicide Severity Rating Scale (C-SSRS); Section 8.2.9.2.2 Canfield Rash Manual was updated to Canfield Photography User Manual; In Section 8.2.14 The name of the ‘Rater Qualifications Manual’ was updated to ‘Rater Assessment Manual’; Section 8.3.6 Cardiovascular and Death Events, was updated to reflect the adjudication process; Section 8.3.8 Adverse Events of Special Interest, was updated to add a reference to the section with the definition and the reporting process; deleted Section 8.10 Health Economics; updated Appendix 4, 6, 7, 9.

28 Apr 2022

In order to allow study subjects continued access to study intervention with collection of additional long-term safety and efficacy data, study duration was extended up to 60 months (or until availability of commercial product in the country or until the sponsor terminates the study in that country). The endpoints were updated as a result of the study extension. Added ECG analysis under safety endpoints at TP1 and TP2 phases only. Added detail on permanent discontinuation and defined the Observation Period. Update was made in alignment with Investigator Brochure version 8.0 December 2021, and also to clarify the safety of PF-06651600. Update was made to clarify the safety of PF-06651600. Mean Clinical AUC and Calculated Safety Margin updated. Added additional clarifications on the contraception check. Updated the blinding approach in the study. Updated the amount of blood to be collected from each subject. Clarified when an interim analysis may be needed. Updated language regarding events requiring submission to an adjudication/review committee, and text regarding confirmation of post-menopausal status during the study. Updated hemoglobin values requiring re-testing in Table 3.

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.

The study is still ongoing. This report reflects data collected up to the data-cut on 19 May 2023, and will be updated after completion of the whole study.

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Clinical trials

Clinical Trial Results: A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Study Investigating the Safety of Ritlecitinib (PF-06651600) in Adult Participants With Alopecia Areata

Trial information

Trial information

Subject disposition

Subject disposition

Baseline characteristics

Baseline characteristics

End points

End points

Primary: Change From Baseline in I-V Interwave Latency on Brainstem Auditory Evoked Potentials (BAEP) at a Stimulus Intensity of 80 dB From the Right Side at Month 9

Primary: Change From Baseline in I-V Interwave Latency on Brainstem Auditory Evoked Potentials (BAEP) at a Stimulus Intensity of 80 dB From the Right Side at Month 9

Primary: Change From Baseline in I-V Interwave Latency on BAEP at a Stimulus Intensity of 80 dB From the Left Side at Month 9

Primary: Change From Baseline in I-V Interwave Latency on BAEP at a Stimulus Intensity of 80 dB From the Left Side at Month 9

Secondary: Change From Baseline in I-V Interwave Latency on BAEP at 80 dB From the Right Side at Month 6

Secondary: Change From Baseline in I-V Interwave Latency on BAEP at 80 dB From the Right Side at Month 6

Secondary: Change From Baseline in I-V Interwave Latency on BAEP at 80 dB From the Right Side at Month 9E and 15E

Secondary: Change From Baseline in I-V Interwave Latency on BAEP at 80 dB From the Right Side at Month 9E and 15E

Secondary: Change From Baseline in I-V Interwave Latency on BAEP at 80 dB From the Left Side at Month 6

Secondary: Change From Baseline in I-V Interwave Latency on BAEP at 80 dB From the Left Side at Month 6

Secondary: Change From Baseline in I-V Interwave Latency on BAEP at 80 dB From the Left Side at Month 9E and 15E

Secondary: Change From Baseline in I-V Interwave Latency on BAEP at 80 dB From the Left Side at Month 9E and 15E

Secondary: Change From Baseline in Percentage of Intra-epidermal Nerve Fiber (IENF) With Axonal Swelling in Skin Punch Biopsies at Month 9

Secondary: Change From Baseline in Percentage of Intra-epidermal Nerve Fiber (IENF) With Axonal Swelling in Skin Punch Biopsies at Month 9

Secondary: Change From Baseline in Percentage of IENFs With Axonal Swelling in Skin Punch Biopsies at Month 15E

Secondary: Change From Baseline in Percentage of IENFs With Axonal Swelling in Skin Punch Biopsies at Month 15E

Secondary: Change From Baseline in Intraepidermal Nerve Fiber Density (IENFD) in Skin Punch Biopsies at Month 9

Secondary: Change From Baseline in Intraepidermal Nerve Fiber Density (IENFD) in Skin Punch Biopsies at Month 9

Secondary: Change From Baseline in IENFD in Skin Punch Biopsies at Month 15E

Secondary: Change From Baseline in IENFD in Skin Punch Biopsies at Month 15E

Secondary: Change From Baseline in Amplitude of Wave V on BAEP at a Stimulus Intensity of 80 dB From the Right Side at Month 6 and Month 9

Secondary: Change From Baseline in Amplitude of Wave V on BAEP at a Stimulus Intensity of 80 dB From the Right Side at Month 6 and Month 9

Secondary: Change From Baseline in Amplitude of Wave V on BAEP at a Stimulus Intensity of 80 dB From the Right Side at Month 9E and 15E

Secondary: Change From Baseline in Amplitude of Wave V on BAEP at a Stimulus Intensity of 80 dB From the Right Side at Month 9E and 15E

Secondary: Change From Baseline in Amplitude of Wave V on BAEP at a Stimulus Intensity of 80 dB From the Left Side at Month 6 and Month 9

Secondary: Change From Baseline in Amplitude of Wave V on BAEP at a Stimulus Intensity of 80 dB From the Left Side at Month 6 and Month 9

Secondary: Change From Baseline in Amplitude of Wave V on BAEP at a Stimulus Intensity of 80 dB From the Left Side at Month 9E and 15E

Secondary: Change From Baseline in Amplitude of Wave V on BAEP at a Stimulus Intensity of 80 dB From the Left Side at Month 9E and 15E

Secondary: Number of Subjects With Absence of Wave V on BAEP at Stimulus Intensities Ranging From 80 dB to 40 dB From Right Side up to Month 9

Secondary: Number of Subjects With Absence of Wave V on BAEP at Stimulus Intensities Ranging From 80 dB to 40 dB From Right Side up to Month 9

Secondary: Number of Subjects With Absence of Wave V on BAEP at Stimulus Intensities Ranging From 80 dB to 40 dB From Right Side at Baseline, Month 3E, 6E, 9E and 15E

Secondary: Number of Subjects With Absence of Wave V on BAEP at Stimulus Intensities Ranging From 80 dB to 40 dB From Right Side at Baseline, Month 3E, 6E, 9E and 15E

Secondary: Number of Subjects With Absence of Wave V on BAEP at Stimulus Intensities Ranging From 80 dB to 40 dB From Left Side up to Month 9

Secondary: Number of Subjects With Absence of Wave V on BAEP at Stimulus Intensities Ranging From 80 dB to 40 dB From Left Side up to Month 9

Secondary: Number of Subjects With Absence of Wave V on BAEP at Stimulus Intensities Ranging From 80 dB to 40 dB From Left Side at Baseline, Month 3E, 6E, 9E and 15E

Secondary: Number of Subjects With Absence of Wave V on BAEP at Stimulus Intensities Ranging From 80 dB to 40 dB From Left Side at Baseline, Month 3E, 6E, 9E and 15E

Secondary: Number of Subjects With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse events (TESAEs)

Secondary: Number of Subjects With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse events (TESAEs)

Secondary: Number of Subjects Who Discontinued From Study Due to Adverse Event (AEs)

Secondary: Number of Subjects Who Discontinued From Study Due to Adverse Event (AEs)

Secondary: Number of Subjects Who Discontinued Study Drug Due to AE and Continued Study

Secondary: Number of Subjects Who Discontinued Study Drug Due to AE and Continued Study

Secondary: Number of Subjects With Temporary Drug Discontinuation Due to AEs

Secondary: Number of Subjects With Temporary Drug Discontinuation Due to AEs

Secondary: Number of Subjects With Clinically Significant Abnormalities in Vital Signs

Secondary: Number of Subjects With Clinically Significant Abnormalities in Vital Signs

Secondary: Number of Subjects With Clinically Significant Abnormalities in Clinical Laboratory Values

Secondary: Number of Subjects With Clinically Significant Abnormalities in Clinical Laboratory Values

Secondary: Change From Baseline in Overall Severity of Alopecia Tool (SALT) Scores up to Month 9

Secondary: Change From Baseline in Overall Severity of Alopecia Tool (SALT) Scores up to Month 9

Secondary: Change From Baseline in Overall SALT Scores at Month 3E, 6E, 9E, 12E and 15E

Secondary: Change From Baseline in Overall SALT Scores at Month 3E, 6E, 9E, 12E and 15E

Secondary: Change From Baseline in Alopecia Areata - Severity of Alopecia Tool (AA-SALT) Score up to Month 9

Secondary: Change From Baseline in Alopecia Areata - Severity of Alopecia Tool (AA-SALT) Score up to Month 9

Secondary: Change From Baseline in AA-SALT Score at Month 3E, 6E, 9E, 12E and 15E

Secondary: Change From Baseline in AA-SALT Score at Month 3E, 6E, 9E, 12E and 15E

Secondary: Number of Subjects With Patient's Global Impression of Change (PGI-C) Response up to Month 9

Secondary: Number of Subjects With Patient's Global Impression of Change (PGI-C) Response up to Month 9

Secondary: Number of Subjects With PGI-C Response at Month 3E, 6E, 9E, 12E and 15E

Secondary: Number of Subjects With PGI-C Response at Month 3E, 6E, 9E, 12E and 15E

Adverse events

Adverse events

More information

Substantial protocol amendments (globally)

Interruptions (globally)

Limitations and caveats

More information

Clinical Trial Results:
A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Study Investigating the Safety of Ritlecitinib (PF-06651600) in Adult Participants With Alopecia Areata