Clinical Trial Results:
A Phase 2b Randomized, Double‑Blind, Placebo‑Controlled, Multicenter, Dose‑Ranging Study to Evaluate the Efficacy and Safety Profile of PF‑06651600 With a Partially Blinded Extension Period to Evaluate the Efficacy and Safety of PF‑06651600 and PF‑06700841 in Subjects With Active Non‑Segmental Vitiligo

Clinical trials

Trial information

Subject disposition

Baseline characteristics

End points

Adverse events

More information

Trial information

Trial information

Subject disposition

Subject disposition

Baseline characteristics

Baseline characteristics

End points

End points

Adverse events

Adverse events

More information

Primary: Percent Change From Baseline in Central Read Facial-Vitiligo Area Scoring Index (F-VASI) at Week 24 - Dose Ranging (DR) Period

Primary: Number of Subjects With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) up to Week 24 - DR Period

Primary: Number of Subjects With the TEAEs of Anaemia, Neutropenia, Thrombocytopenia and Lymphopenia - DR Period

Primary: Number of Subjects With Clinically Meaningful Changes From Baseline in Lipid Profile up to Week 24 - DR Period

Primary: Number of Subjects With Liver Function Test Values Meeting the Protocol-Specified Discontinuation Criteria - DR Period

Primary: Number of Subjects With TEAEs and SAEs - Extension (Ext) Period

Primary: Number of Subjects With the TEAEs of Anaemia, Neutropenia, Thrombocytopenia and Lymphopenia - Ext Period

Primary: Number of Subjects With Clinically Meaningful Changes From Baseline in Lipid Profile - Ext Period

Primary: Number of Subjects With Liver Function Test Values Meeting the Protocol-Specified Discontinuation Criteria - Ext Period

Secondary: Proportion of Subjects Achieving Central F-VASI75 at Week 24 - DR Period

Secondary: Proportion of Subjects Achieving T-VASI50 at Week 24 - DR Period

Secondary: Percent Change From Baseline in T-VASI at Designated Time Points - DR Period

Secondary: Percent Change From Baseline in Central Read F-VASI at Designated Time Points - DR Period

Secondary: Percent Change From Baseline in Local F-VASI at Designated Time Points - DR Period

Secondary: Percent Change From Baseline in SA-VES at Designated Time Points - DR Period

Secondary: Absolute Change From Baseline in T-VASI at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving T-VASI50 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving T-VASI75 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving T-VASI90 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving T-VASI100 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving Central Read F-VASI50 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving Central Read F-VASI75 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving Central Read F-VASI90 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving Central Read F-VASI100 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving Local F-VASI50 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving Local F-VASI75 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving Local F-VASI90 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving Local F-VASI100 at Designated Time Points - DR Period

Secondary: Change From Baseline in Total Vitiligo-Specific Quality of Life (VitiQoL) Score at Designated Time Points - DR Period

Secondary: Change From Baseline in VitiQoL Participation Limitation Domain Score at Designated Time Points - DR Period

Secondary: Change From Baseline in VitiQoL Stigma Domain Score at Designated Time Points - DR Period

Secondary: Change From Baseline in VitiQoL Behaviors Domain Score at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving sIGA 0 or 1 and at Least a 2-Point Improvement at Week 24 - DR Period

Primary: Percent Change From Baseline in Central Read Facial-Vitiligo Area Scoring Index (F-VASI) at Week 24 - Dose Ranging (DR) Period

Primary: Percent Change From Baseline in Central Read Facial-Vitiligo Area Scoring Index (F-VASI) at Week 24 - Dose Ranging (DR) Period

Primary: Number of Subjects With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) up to Week 24 - DR Period

Primary: Number of Subjects With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) up to Week 24 - DR Period

Primary: Number of Subjects With the TEAEs of Anaemia, Neutropenia, Thrombocytopenia and Lymphopenia - DR Period

Primary: Number of Subjects With the TEAEs of Anaemia, Neutropenia, Thrombocytopenia and Lymphopenia - DR Period

Primary: Number of Subjects With Clinically Meaningful Changes From Baseline in Lipid Profile up to Week 24 - DR Period

Primary: Number of Subjects With Clinically Meaningful Changes From Baseline in Lipid Profile up to Week 24 - DR Period

Primary: Number of Subjects With Liver Function Test Values Meeting the Protocol-Specified Discontinuation Criteria - DR Period

Primary: Number of Subjects With Liver Function Test Values Meeting the Protocol-Specified Discontinuation Criteria - DR Period

Primary: Number of Subjects With TEAEs and SAEs - Extension (Ext) Period

Primary: Number of Subjects With TEAEs and SAEs - Extension (Ext) Period

Primary: Number of Subjects With the TEAEs of Anaemia, Neutropenia, Thrombocytopenia and Lymphopenia - Ext Period

Primary: Number of Subjects With the TEAEs of Anaemia, Neutropenia, Thrombocytopenia and Lymphopenia - Ext Period

Primary: Number of Subjects With Clinically Meaningful Changes From Baseline in Lipid Profile - Ext Period

Primary: Number of Subjects With Clinically Meaningful Changes From Baseline in Lipid Profile - Ext Period

Primary: Number of Subjects With Liver Function Test Values Meeting the Protocol-Specified Discontinuation Criteria - Ext Period

Primary: Number of Subjects With Liver Function Test Values Meeting the Protocol-Specified Discontinuation Criteria - Ext Period

Secondary: Proportion of Subjects Achieving Central F-VASI75 at Week 24 - DR Period

Secondary: Proportion of Subjects Achieving Central F-VASI75 at Week 24 - DR Period

Secondary: Proportion of Subjects Achieving T-VASI50 at Week 24 - DR Period

Secondary: Proportion of Subjects Achieving T-VASI50 at Week 24 - DR Period

Secondary: Percent Change From Baseline in T-VASI at Designated Time Points - DR Period

Secondary: Percent Change From Baseline in T-VASI at Designated Time Points - DR Period

Secondary: Percent Change From Baseline in Central Read F-VASI at Designated Time Points - DR Period

Secondary: Percent Change From Baseline in Central Read F-VASI at Designated Time Points - DR Period

Secondary: Percent Change From Baseline in Local F-VASI at Designated Time Points - DR Period

Secondary: Percent Change From Baseline in Local F-VASI at Designated Time Points - DR Period

Secondary: Percent Change From Baseline in SA-VES at Designated Time Points - DR Period

Secondary: Percent Change From Baseline in SA-VES at Designated Time Points - DR Period

Secondary: Absolute Change From Baseline in T-VASI at Designated Time Points - DR Period

Secondary: Absolute Change From Baseline in T-VASI at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving T-VASI50 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving T-VASI50 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving T-VASI75 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving T-VASI75 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving T-VASI90 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving T-VASI90 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving T-VASI100 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving T-VASI100 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving Central Read F-VASI50 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving Central Read F-VASI50 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving Central Read F-VASI75 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving Central Read F-VASI75 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving Central Read F-VASI90 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving Central Read F-VASI90 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving Central Read F-VASI100 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving Central Read F-VASI100 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving Local F-VASI50 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving Local F-VASI50 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving Local F-VASI75 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving Local F-VASI75 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving Local F-VASI90 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving Local F-VASI90 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving Local F-VASI100 at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving Local F-VASI100 at Designated Time Points - DR Period

Secondary: Change From Baseline in Total Vitiligo-Specific Quality of Life (VitiQoL) Score at Designated Time Points - DR Period

Secondary: Change From Baseline in Total Vitiligo-Specific Quality of Life (VitiQoL) Score at Designated Time Points - DR Period

Secondary: Change From Baseline in VitiQoL Participation Limitation Domain Score at Designated Time Points - DR Period

Secondary: Change From Baseline in VitiQoL Participation Limitation Domain Score at Designated Time Points - DR Period

Secondary: Change From Baseline in VitiQoL Stigma Domain Score at Designated Time Points - DR Period

Secondary: Change From Baseline in VitiQoL Stigma Domain Score at Designated Time Points - DR Period

Secondary: Change From Baseline in VitiQoL Behaviors Domain Score at Designated Time Points - DR Period

Secondary: Change From Baseline in VitiQoL Behaviors Domain Score at Designated Time Points - DR Period

Secondary: Proportion of Subjects Achieving sIGA 0 or 1 and at Least a 2-Point Improvement at Week 24 - DR Period

Secondary: Proportion of Subjects Achieving sIGA 0 or 1 and at Least a 2-Point Improvement at Week 24 - DR Period

Substantial protocol amendments (globally)

Interruptions (globally)

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Summary
EudraCT number	2018-001271-20
Trial protocol	DE BE ES IT
Global end of trial date	05 Feb 2021

Results information
Results version number	v1(current)
This version publication date	27 Jan 2022
First version publication date	27 Jan 2022
Other versions

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

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Sponsor protocol code

B7981019

ISRCTN number

US NCT number

NCT03715829

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

Final

Date of interim/final analysis

15 Sep 2021

Is this the analysis of the primary completion data?

Yes

Primary completion date

05 Feb 2021

Global end of trial reached?

Yes

Global end of trial date

05 Feb 2021

Was the trial ended prematurely?

Main objective of the trial

• To evaluate the efficacy of ritlecitinib dose/dosing regimens at Week 24 in adult participants with active non-segmental vitiligo. • To evaluate the safety and tolerability of ritlecitinib over time in adult participants with active non-segmental vitiligo. • To evaluate the safety and tolerability of ritlecitinib and brepocitinib in adult participants with active non-segmental vitiligo.

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 Conference on Harmonisation (ICH) Good Clinical Practice (GCP) Guidelines. In addition, all local regulatory requirements were followed, in particular, those affording greater protection to the safety of trial subjects.

Background therapy

Evidence for comparator

Actual start date of recruitment

26 Nov 2018

Long term follow-up planned

Independent data monitoring committee (IDMC) involvement?

Yes

Number of subjects enrolled per country

Country: Number of subjects enrolled

Australia: 21

Country: Number of subjects enrolled

Belgium: 8

Country: Number of subjects enrolled

Canada: 105

Country: Number of subjects enrolled

Germany: 21

Country: Number of subjects enrolled

Italy: 5

Country: Number of subjects enrolled

Japan: 23

Country: Number of subjects enrolled

Korea, Republic of: 16

Country: Number of subjects enrolled

Spain: 21

Country: Number of subjects enrolled

Taiwan: 7

Country: Number of subjects enrolled

United States: 137

Worldwide total number of subjects

364

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)

355

From 65 to 84 years

85 years and over

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Recruitment details

Screening details

A total of 578 subjects were screened for this study; 366 were randomized to treatment and 364 (99.5%) subjects received treatment.

Period 1 title

Dose Ranging Period

Is this the baseline period?

Yes

Allocation method

Randomised - controlled

Blinding used

Double blind

Roles blinded

Subject, Investigator, Monitor, Data analyst, Carer, Assessor

Are arms mutually exclusive

Yes

PF-06651600 200 mg - 50 mg QD

Arm description

Subjects were randomized to receive ritlecitinib (PF-06651600) induction dose of 200 mg QD for 4 weeks followed by ritlecitinib 50 mg QD for another 20 weeks.

Arm type

Experimental

Investigational medicinal product name

PF-06651600 200 mg

Investigational medicinal product code

Other name

ritlecitinib

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects swallowed the tablet once daily with ambient temperature water to a total volume of approximately 240 mL after breakfast whenever possible.

Investigational medicinal product name

PF-06651600 50 mg

Investigational medicinal product code

Other name

ritlecitinib

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects swallowed the tablet once daily with ambient temperature water to a total volume of approximately 240 mL after breakfast whenever possible.

PF-06651600 100 mg - 50 mg QD

Arm description

Subjects were randomized to receive ritlecitinib induction dose of 100 mg QD for 4 weeks followed by ritlecitinib 50 mg QD for another 20 weeks.

Arm type

Experimental

Investigational medicinal product name

PF06651600 50 mg

Investigational medicinal product code

Other name

ritlecitinib

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects swallowed the tablet once daily with ambient temperature water to a total volume of approximately 240 mL after breakfast whenever possible.

Investigational medicinal product name

PF-06651600 100 mg

Investigational medicinal product code

Other name

ritlecitinib

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects swallowed the tablet once daily with ambient temperature water to a total volume of approximately 240 mL after breakfast whenever possible.

PF-06651600 50 mg QD

Arm description

Subjects were randomized to receive ritlecitinib 50 mg QD for 24 weeks.

Arm type

Experimental

Investigational medicinal product name

PF-06651600 50 mg

Investigational medicinal product code

Other name

ritlecitinib

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects swallowed the tablet once daily with ambient temperature water to a total volume of approximately 240 mL after breakfast whenever possible.

PF-06651600 30 mg QD

Arm description

Subjects were randomized to receive ritlecitinib 30 mg QD for 24 weeks.

Arm type

Experimental

Investigational medicinal product name

PF-06651600 30 mg

Investigational medicinal product code

Other name

ritlecitinib

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects swallowed the tablet once daily with ambient temperature water to a total volume of approximately 240 mL after breakfast whenever possible.

PF-06651600 10 mg QD

Arm description

Subjects were randomized to receive ritlecitinib 10 mg QD for 24 weeks.

Arm type

Experimental

Investigational medicinal product name

PF-06651600 10 mg

Investigational medicinal product code

Other name

ritlecitinib

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects swallowed the tablet once daily with ambient temperature water to a total volume of approximately 240 mL after breakfast whenever possible.

Placebo

Arm description

Subjects were randomized to receive placebo for 24 weeks.

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

Subjects swallowed the tablet once daily with ambient temperature water to a total volume of approximately 240 mL after breakfast whenever possible.

Number of subjects in period 1	PF-06651600 200 mg - 50 mg QD	PF-06651600 100 mg - 50 mg QD	PF-06651600 50 mg QD	PF-06651600 30 mg QD	PF-06651600 10 mg QD	Placebo
Started	65	67	67	50	49	66
Completed	53	58	54	36	42	55
Not completed	12	9	13	14	7	11
Consent withdrawn by subject	8	4	3	9	2	3
Adverse event, non-fatal	2	4	5	2	3	3
Other	-	-	2	-	-	3
Medication Error Without Associated Adverse Event	-	-	1	-	-	-
Lost to follow-up	2	-	1	1	1	2
No Longer Meets Eligibility Criteria	-	-	1	-	1	-
Lack of efficacy	-	1	-	1	-	-
Protocol deviation	-	-	-	1	-	-

Period 2 title

Extension Period

Is this the baseline period?

Allocation method

Non-randomised - controlled

Blinding used

Double blind

Roles blinded

Subject, Investigator, Monitor, Data analyst, Carer, Assessor

Blinding implementation details

In the Extension (EXT) Period, the brepocitinib 60 mg - 30 mg QD group and ritlecitinib 200-50 mg QD+ nbUVB group were open-label; the rest of groups were double-blinded.

Are arms mutually exclusive

Yes

Extension (EXT) PF-06700841 60 mg - 30 mg QD

Arm description

After a 4-week drug holiday, subjects received induction dose of brepocitinib (PF-06700841) 60 mg QD for 4 weeks followed by brepocitinib 30 mg QD for 16 weeks. This arm is open label.

Arm type

Experimental

Investigational medicinal product name

PF-06700841 30 mg

Investigational medicinal product code

Other name

brepocitinib

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects swallowed the tablet once daily with ambient temperature water to a total volume of approximately 240 mL after breakfast whenever possible.

Investigational medicinal product name

PF-06700841 60 mg

Investigational medicinal product code

Other name

brepocitinib

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects swallowed the tablet once daily with ambient temperature water to a total volume of approximately 240 mL after breakfast whenever possible.

EXT PF-06651600 200 mg - 50 mg QD + nbUVB

Arm description

Induction dose of ritlecitinib 200 mg QD plus standardized narrow band UVB (nbUVB) add-on therapy for 4 weeks followed by ritlecitinib 50 mg QD plus standardized nbUVB add-on therapy for 20 weeks (only for subjects who provided nbUVB consent). Subjects who had <10% improvement in percent change in VASI at Extension Week 12 from the baseline value at Dose Ranging Period Week 24 were discontinued from the treatment and entered Follow-up Period. This arm is open label.

Arm type

Experimental

Investigational medicinal product name

PF-06651600 200 mg

Investigational medicinal product code

Other name

ritlecitinib

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects swallowed the tablet once daily with ambient temperature water to a total volume of approximately 240 mL after breakfast whenever possible.

Investigational medicinal product name

PF-06651600 50 mg

Investigational medicinal product code

Other name

ritlecitinib

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects swallowed the tablet once daily with ambient temperature water to a total volume of approximately 240 mL after breakfast whenever possible.

EXT PF-06651600 200 mg - 50 mg QD

Arm description

Induction dose of ritlecitinib 200 mg QD of for 4 weeks followed by ritlecitinib 50 mg QD for 20 weeks. This arm is double blinded.

Arm type

Experimental

Investigational medicinal product name

PF-06651600 50 mg

Investigational medicinal product code

Other name

ritlecitinib

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects swallowed the tablet once daily with ambient temperature water to a total volume of approximately 240 mL after breakfast whenever possible.

Investigational medicinal product name

PF-06651600 200 mg

Investigational medicinal product code

Other name

ritlecitinib

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects swallowed the tablet once daily with ambient temperature water to a total volume of approximately 240 mL after breakfast whenever possible.

EXT PF-06651600 50 mg QD

Arm description

Ritlecitinib 50 mg QD for 24 weeks. This arm is double blinded.

Arm type

Experimental

Investigational medicinal product name

PF-06651600 50 mg

Investigational medicinal product code

Other name

ritlecitinib

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects swallowed the tablet once daily with ambient temperature water to a total volume of approximately 240 mL after breakfast whenever possible.

EXT PF-06651600 30 mg QD

Arm description

Ritlecitinib 30 mg QD of for 24 weeks. This arm is double blinded.

Arm type

Experimental

Investigational medicinal product name

PF-06651600 30 mg

Investigational medicinal product code

Other name

ritlecitinib

Pharmaceutical forms

Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects swallowed the tablet once daily with ambient temperature water to a total volume of approximately 240 mL after breakfast whenever possible.

Number of subjects in period 2 ^[1]	Extension (EXT) PF-06700841 60 mg - 30 mg QD	EXT PF-06651600 200 mg - 50 mg QD + nbUVB	EXT PF-06651600 200 mg - 50 mg QD	EXT PF-06651600 50 mg QD	EXT PF-06651600 30 mg QD
Started	55	43	187	6	2
Completed	47	27	158	3	2
Not completed	8	16	29	3	0
Consent withdrawn by subject	3	3	12	3	-
Adverse event, non-fatal	3	1	6	-	-
Other	1	2	4	-	-
Medication Error Without Associated Adverse Event	-	1	1	-	-
Lost to follow-up	-	-	2	-	-
No Longer Meets Eligibility Criteria	-	-	1	-	-
Lack of efficacy	1	9	2	-	-
Protocol deviation	-	-	1	-	-

Notes
[1] - The number of subjects starting the period is not consistent with the number completing the preceding period. It is expected the number of subjects starting the subsequent period will be the same as the number completing the preceding period.
Justification: Not all of the participants completing the dose ranging period entered the extension period

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Reporting group title

PF-06651600 200 mg - 50 mg QD

Reporting group description

Subjects were randomized to receive ritlecitinib (PF-06651600) induction dose of 200 mg QD for 4 weeks followed by ritlecitinib 50 mg QD for another 20 weeks.

Reporting group title

PF-06651600 100 mg - 50 mg QD

Reporting group description

Subjects were randomized to receive ritlecitinib induction dose of 100 mg QD for 4 weeks followed by ritlecitinib 50 mg QD for another 20 weeks.

Reporting group title

PF-06651600 50 mg QD

Reporting group description

Subjects were randomized to receive ritlecitinib 50 mg QD for 24 weeks.

Reporting group title

PF-06651600 30 mg QD

Reporting group description

Subjects were randomized to receive ritlecitinib 30 mg QD for 24 weeks.

Reporting group title

PF-06651600 10 mg QD

Reporting group description

Subjects were randomized to receive ritlecitinib 10 mg QD for 24 weeks.

Reporting group title

Placebo

Reporting group description

Subjects were randomized to receive placebo for 24 weeks.

Reporting group values	PF-06651600 200 mg - 50 mg QD	PF-06651600 100 mg - 50 mg QD	PF-06651600 50 mg QD	PF-06651600 30 mg QD	PF-06651600 10 mg QD	Placebo	Total
Number of subjects	65	67	67	50	49	66	364
Age Categorical
Units: Subjects
< 18 years	0	0	0	0	0	0	0
18 - 44 years	33	29	33	20	16	30	161
45 - 64 years	30	37	34	30	31	32	194
≥ 65 years	2	1	0	0	2	4	9
Age continuous
Units: years
arithmetic mean (standard deviation)	45.37 ± 12.21	44.15 ± 11.15	43.34 ± 10.42	44.74 ± 13.51	46.57 ± 10.03	46.09 ± 11.51	-
Sex: Female, Male
Units: Subjects
Female	30	31	39	28	25	40	193
Male	35	36	28	22	24	26	171
Race (NIH/OMB)
Units: Subjects
American Indian or Alaska Native	0	0	0	1	0	0	1
Asian	15	17	17	5	11	21	86
Native Hawaiian or Other Pacific Islander	0	0	0	0	0	0	0
Black or African American	3	0	0	4	1	2	10
White	44	47	45	39	33	38	246
More than one race	0	1	1	0	2	1	5
Unknown or Not Reported	3	2	4	1	2	4	16
Ethnicity (NIH/OMB)
Units: Subjects
Hispanic or Latino	11	9	11	12	7	7	57
Not Hispanic or Latino	54	58	54	38	41	58	303
Unknown or Not Reported	0	0	2	0	1	1	4

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Reporting group title

PF-06651600 200 mg - 50 mg QD

Reporting group description

Subjects were randomized to receive ritlecitinib (PF-06651600) induction dose of 200 mg QD for 4 weeks followed by ritlecitinib 50 mg QD for another 20 weeks.

Reporting group title

PF-06651600 100 mg - 50 mg QD

Reporting group description

Subjects were randomized to receive ritlecitinib induction dose of 100 mg QD for 4 weeks followed by ritlecitinib 50 mg QD for another 20 weeks.

Reporting group title

PF-06651600 50 mg QD

Reporting group description

Subjects were randomized to receive ritlecitinib 50 mg QD for 24 weeks.

Reporting group title

PF-06651600 30 mg QD

Reporting group description

Subjects were randomized to receive ritlecitinib 30 mg QD for 24 weeks.

Reporting group title

PF-06651600 10 mg QD

Reporting group description

Subjects were randomized to receive ritlecitinib 10 mg QD for 24 weeks.

Reporting group title

Placebo

Reporting group description

Subjects were randomized to receive placebo for 24 weeks.

Reporting group title

Extension (EXT) PF-06700841 60 mg - 30 mg QD

Reporting group description

After a 4-week drug holiday, subjects received induction dose of brepocitinib (PF-06700841) 60 mg QD for 4 weeks followed by brepocitinib 30 mg QD for 16 weeks. This arm is open label.

Reporting group title

EXT PF-06651600 200 mg - 50 mg QD + nbUVB

Reporting group description

Reporting group title

EXT PF-06651600 200 mg - 50 mg QD

Reporting group description

Induction dose of ritlecitinib 200 mg QD of for 4 weeks followed by ritlecitinib 50 mg QD for 20 weeks. This arm is double blinded.

Reporting group title

EXT PF-06651600 50 mg QD

Reporting group description

Ritlecitinib 50 mg QD for 24 weeks. This arm is double blinded.

Reporting group title

EXT PF-06651600 30 mg QD

Reporting group description

Ritlecitinib 30 mg QD of for 24 weeks. This arm is double blinded.

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End point title

Percent Change From Baseline in Central Read Facial-Vitiligo Area Scoring Index (F-VASI) at Week 24 - Dose Ranging (DR) Period

End point description

Central read F-VASI was assessed based on the facial photographs taken at the site. The central read F-VASI was calculated using a formula that included contribution of affected facial surface areas showing 6 different depigmentation rates (0.1, 0.25, 0.5, 0.75, 0.9 and 1): F-VASI (central read) = Ʃ [Affected Facial Surface Area]×4×[Depigmentation Rates]. Face was defined as the area from the hairline on top of the forehead to the jawline at the bottom of the cheeks. F-VASI (central read) ranged from 0.000 to 4.000 by defining the affected Facial Surface Area (expressed as the value between 0.0 to 1.0) being 4% of total Body Surface Area. The higher score of F-VASI signified severer symptoms of non-segmental vitiligo. Percent change from baseline in F-VASI =((post-baseline F-VASI - baseline F-VASI)/baseline F-VASI)×100. The analysis population included all subjects who received at least 1 dose of randomized study medication and had a baseline and at least 1 post-baseline measurement.

End point type

Primary

End point timeframe

Baseline, Week 24. Baseline was defined as the last measurement prior to Study Day 18.

End point values	PF-06651600 200 mg - 50 mg QD	PF-06651600 100 mg - 50 mg QD	PF-06651600 50 mg QD	PF-06651600 30 mg QD	PF-06651600 10 mg QD	Placebo
Number of subjects analysed	62	63	59	45	48	57
Units: Percentage
least squares mean (standard error)	-21.2 ± 4.13	-21.2 ± 4.16	-18.5 ± 4.44	-14.6 ± 5.47	-3.0 ± 4.65	2.1 ± 4.06

PF-06651600 200mg - 50mg QD vs Placebo

Comparison groups

PF-06651600 200 mg - 50 mg QD v Placebo

Number of subjects included in analysis

119

Analysis specification

Pre-specified

Analysis type

P-value

< 0.0001 ^[1]

Method

ANCOVA

Parameter type

Mean difference (final values)

Point estimate

-23.2

Confidence interval

level

90%

sides

2-sided

lower limit

-32.53

upper limit

-13.96

Variability estimate

Standard error of the mean

Dispersion value

5.62

Notes
[1] - Hochberg’s step-up procedure was conducted to compare the ritlecitinib 200mg - 50mg QD dose group vs placebo using observed p-values. The familywise Type 1 error rate was controlled at one-sided 0.05. Hochberg adjusted p-value is presented here.

PF-06651600 100 mg - 50 mg QD vs Placebo

Comparison groups

PF-06651600 100 mg - 50 mg QD v Placebo

Number of subjects included in analysis

120

Analysis specification

Pre-specified

Analysis type

P-value

< 0.0001 ^[2]

Method

ANCOVA

Parameter type

Mean difference (final values)

Point estimate

-23.2

Confidence interval

level

90%

sides

2-sided

lower limit

-32.53

upper limit

-13.93

Variability estimate

Standard error of the mean

Dispersion value

5.63

Notes
[2] - Hochberg’s step-up procedure was conducted to compare the ritlecitinib 100mg - 50mg QD dose group vs placebo using observed p-values. The familywise Type 1 error rate was controlled at one-sided 0.05. Hochberg adjusted p-value is presented here.

PF-06651600 30 mg QD vs Placebo

Comparison groups

PF-06651600 30 mg QD v Placebo

Number of subjects included in analysis

102

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.0068 ^[3]

Method

ANCOVA

Parameter type

Mean difference (final values)

Point estimate

-16.7

Confidence interval

level

90%

sides

2-sided

lower limit

-27.77

upper limit

-5.61

Variability estimate

Standard error of the mean

Dispersion value

6.71

Notes
[3] - Hochberg’s step-up procedure was conducted to compare the ritlecitinib 30 mg QD dose group vs placebo using observed p-values. The familywise Type 1 error rate was controlled at one-sided 0.05. One-sided unadjusted p-value is presented here.

PF-06651600 10 mg QD vs Placebo

Comparison groups

PF-06651600 10 mg QD v Placebo

Number of subjects included in analysis

105

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.2015 ^[4]

Method

ANCOVA

Parameter type

Mean difference (final values)

Point estimate

-5.1

Confidence interval

level

90%

sides

2-sided

lower limit

-15.02

upper limit

4.91

Variability estimate

Standard error of the mean

Dispersion value

6.03

Notes
[4] - Hochberg’s step-up procedure was conducted to compare the ritlecitinib 10 mg QD dose group vs placebo using observed p-values. The familywise Type 1 error rate was controlled at one-sided 0.05. One-sided unadjusted p-value is presented here.

PF-06651600 50 mg QD vs Placebo

Comparison groups

PF-06651600 50 mg QD v Placebo

Number of subjects included in analysis

116

Analysis specification

Pre-specified

Analysis type

P-value

= 0.0003 ^[5]

Method

ANCOVA

Parameter type

Mean difference (final values)

Point estimate

-20.6

Confidence interval

level

90%

sides

2-sided

lower limit

-30.23

upper limit

-10.93

Variability estimate

Standard error of the mean

Dispersion value

5.84

Notes
[5] - Hochberg’s step-up procedure was conducted to compare the ritlecitinib 50 mg QD dose group vs placebo using observed p-values. The familywise Type 1 error rate was controlled at one-sided 0.05. Hochberg adjusted p-value is presented here.

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End point title

Number of Subjects With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) up to Week 24 - DR Period ^[6]

End point description

AE was any untoward medical occurrence in a subject administered a product; the event did not need to have a causal relationship with the treatment. An AE was considered a TEAE if the event started during the effective duration of treatment. All events started on or after the first dosing day and time, if collected, but before the last dose plus the lag time were flagged as TEAEs. SAE was any untoward medical occurrence at any dose that resulted in death; was life threatening (immediate risk of death); required inpatient hospitalization or prolongation of existing hospitalization; resulted in persistent or significant disability/incapacity (substantial disruption of the ability to conduct normal life functions); resulted in congenital anomaly/birth defect; or that was considered to be an important medical event. Causality to study treatment was determined by the investigator. The safety analysis population included all subjects who received at least 1 dose of investigational product.

End point type

Primary

End point timeframe

24 weeks

Notes
[6] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No statistical analysis was planned for this endpoint

End point values	PF-06651600 200 mg - 50 mg QD	PF-06651600 100 mg - 50 mg QD	PF-06651600 50 mg QD	PF-06651600 30 mg QD	PF-06651600 10 mg QD	Placebo
Number of subjects analysed	65	67	67	50	49	66
Units: Subjects
Subjects With TEAEs (All-Causality)	56	45	54	30	40	52
Subjects With TEAEs (Treatment-Related)	32	19	20	17	18	20
Subjects With SAEs (All-Causality)	0	0	1	1	1	1
Subjects With SAEs (Treatment-Related)	0	0	0	0	0	0

No statistical analyses for this end point

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End point title

Number of Subjects With the TEAEs of Anaemia, Neutropenia, Thrombocytopenia and Lymphopenia - DR Period ^[7]

End point description

An AE was any untoward medical occurrence in a study subject administered a product or medical device; the event did not necessarily need to have a causal relationship with the treatment or usage. The abnormal test findings, clinically significant signs and symptoms of anaemia, neutropenia, thrombocytopenia and lymphopenia were reported as AEs. The clinical significance was determined by the investigator. An AE was considered a TEAE if the event started during the effective duration of treatment. All events that started on or after the first dosing day and time/start time, if collected, but before the last dose plus the lag time were flagged as TEAEs.

End point type

Primary

End point timeframe

Baseline up to Week 24 (Baseline was defined as the last measurement prior to first dosing [Day 1])

Notes
[7] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No statistical analysis was planned for this endpoint

End point values	PF-06651600 200 mg - 50 mg QD	PF-06651600 100 mg - 50 mg QD	PF-06651600 50 mg QD	PF-06651600 30 mg QD	PF-06651600 10 mg QD	Placebo
Number of subjects analysed	65	67	67	50	49	66
Units: Subjects
Subjects With Anaemia	0	0	0	0	0	0
Subjects With Neutropenia	0	1	0	0	1	1
Subjects With Thrombocytopenia	1	0	0	0	0	0
Subjects With Lymphopenia	1	0	0	0	0	0

No statistical analyses for this end point

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End point title

Number of Subjects With Clinically Meaningful Changes From Baseline in Lipid Profile up to Week 24 - DR Period ^[8]

End point description

Subjects had to abstain from all food and drink (except water and non-investigational products) for an 8-hour overnight fast prior to fasting lipid profile panel collection. Fasting lipid assessment included total cholesterol, low-density lipoprotein (LDL) cholesterol, high-density lipoprotein (HDL) cholesterol, and triglycerides. The clinical meaningfulness was determined by the investigator. The safety analysis population included all subjects who received at least 1 dose of investigational product.

End point type

Primary

End point timeframe

Baseline up to Week 24 (Baseline was defined as the last measurement prior to first dosing [Day 1])

Notes
[8] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No statistical analysis was planned for this endpoint

End point values	PF-06651600 200 mg - 50 mg QD	PF-06651600 100 mg - 50 mg QD	PF-06651600 50 mg QD	PF-06651600 30 mg QD	PF-06651600 10 mg QD	Placebo
Number of subjects analysed	65	67	67	50	49	66
Units: Subjects	0	0	0	0	0	0

No statistical analyses for this end point

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End point title

Number of Subjects With Liver Function Test Values Meeting the Protocol-Specified Discontinuation Criteria - DR Period ^[9]

End point description

Liver function tests included tests of alanine aminotransferase (ALT), aspartate aminotransferase (AST) and total bilirubin. The safety analysis population included all subjects who received at least 1 dose of investigational product.

End point type

Primary

End point timeframe

24 weeks

Notes
[9] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No statistical analysis was planned for this endpoint

No statistical analyses for this end point

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End point title

Number of Subjects With TEAEs and SAEs - Extension (Ext) Period ^[10]

End point description

End point type

Primary

End point timeframe

24 weeks

Notes
[10] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No statistical analysis was planned for this endpoint

End point values	Extension (EXT) PF-06700841 60 mg - 30 mg QD	EXT PF-06651600 200 mg - 50 mg QD + nbUVB	EXT PF-06651600 200 mg - 50 mg QD	EXT PF-06651600 50 mg QD	EXT PF-06651600 30 mg QD
Number of subjects analysed	55	43	187	6	2
Units: Subjects
Subjects With All-Causality TEAEs	38	32	119	3	2
Subjects With Treatment-Related TEAEs	20	12	36	0	0
Subjects With All-Causality SAEs	1	0	1	0	0
Subjects With Treatment-Related SAEs	1	0	0	0	0

No statistical analyses for this end point

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End point title

Number of Subjects With the TEAEs of Anaemia, Neutropenia, Thrombocytopenia and Lymphopenia - Ext Period ^[11]

End point description

End point type

Primary

End point timeframe

24 weeks

Notes
[11] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No statistical analysis was planned for this endpoint

End point values	Extension (EXT) PF-06700841 60 mg - 30 mg QD	EXT PF-06651600 200 mg - 50 mg QD + nbUVB	EXT PF-06651600 200 mg - 50 mg QD	EXT PF-06651600 50 mg QD	EXT PF-06651600 30 mg QD
Number of subjects analysed	55	43	187	6	2
Units: Subjects
Subjects With Anemia	0	0	0	1	0
Subjects With Neutropenia	1	0	1	0	0
Subjects With Thrombocytopenia	0	0	0	0	0
Subjects With Lymphopenia	0	0	0	0	0

No statistical analyses for this end point

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End point title

Number of Subjects With Clinically Meaningful Changes From Baseline in Lipid Profile - Ext Period ^[12]

End point description

Subjects had to abstain from all food and drink (except water and non-investigational products) for an 8-hour overnight fast prior to fasting lipid profile panel collection. Fasting lipid assessment included total cholesterol, LDL cholesterol, HDL cholesterol, and triglycerides. The clinical meaningfulness was determined by the investigator. The safety analysis population included all subjects who received at least 1 dose of investigational product.

End point type

Primary

End point timeframe

24 weeks

Notes
[12] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No statistical analysis was planned for this endpoint

End point values	Extension (EXT) PF-06700841 60 mg - 30 mg QD	EXT PF-06651600 200 mg - 50 mg QD + nbUVB	EXT PF-06651600 200 mg - 50 mg QD	EXT PF-06651600 50 mg QD	EXT PF-06651600 30 mg QD
Number of subjects analysed	55	43	187	6	2
Units: Subjects	0	0	0	0	0

No statistical analyses for this end point

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End point title

Number of Subjects With Liver Function Test Values Meeting the Protocol-Specified Discontinuation Criteria - Ext Period ^[13]

End point description

End point type

Primary

End point timeframe

24 weeks

Notes
[13] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No statistical analysis was planned for this endpoint

End point values	Extension (EXT) PF-06700841 60 mg - 30 mg QD	EXT PF-06651600 200 mg - 50 mg QD + nbUVB	EXT PF-06651600 200 mg - 50 mg QD	EXT PF-06651600 50 mg QD	EXT PF-06651600 30 mg QD
Number of subjects analysed	55	42	186	6	2
Units: Subjects
Bilirubin > 1.5 x ULN	0	0	0	0	0
AST > 2.5 x ULN	0	0	0	0	0
ALT > 2.5 x ULN	0	0	1	0	0

No statistical analyses for this end point

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End point title

Proportion of Subjects Achieving Central F-VASI75 at Week 24 - DR Period

End point description

This endpoint was the proportion of subjects achieving at least 75% improvement from baseline in central read F-VASI (F-VASI75) at Week 24. A negative percent change from baseline in central read F-VASI signified an improvement. The central read F-VASI75 response rate was analyzed by first treating the missing data (non-COVID-19 related) as non responders and then applying Chan and Zhang exact confidence interval (CI) method at Week 24. Central read F-VASI75=1 if percent change from baseline ≥75; central read F-VASI75=0 if percent change from baseline <75. Percent change from baseline in F-VASI=((post-baseline F-VASI - baseline F-VASI)/baseline F-VASI)×100. The analysis population included all subjects who received at least 1 dose of randomized study medication and had a baseline and at least 1 post-baseline measurement (after taking randomization study medication).

End point type

Secondary

End point timeframe

Baseline, Week 24. Baseline was defined as the last measurement prior to study Day 18.

End point values	PF-06651600 200 mg - 50 mg QD	PF-06651600 100 mg - 50 mg QD	PF-06651600 50 mg QD	PF-06651600 30 mg QD	PF-06651600 10 mg QD	Placebo
Number of subjects analysed	58	59	52	37	43	57
Units: Percentage of Subjects
number (not applicable)	12.1	8.5	7.7	2.7	2.3	0

No statistical analyses for this end point

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End point title

Proportion of Subjects Achieving T-VASI50 at Week 24 - DR Period

End point description

T-VASI was calculated using a formula that included contribution from 6 body regions (possible range, 0-100): T-VASI = Ʃ[Hand Units]×[Depigmentation]. One hand unit, which encompassed the palm plus the volar surface of all the digits, was approximately 1% of the total body surface area and was used as a guide to estimate the baseline percentage of vitiligo involvement of each body region. The body was divided into 6 separate and mutually exclusive regions: face/neck, hands, upper extremities, trunk, lower extremities, and feet. The extent of depigmentation was expressed by the percentages: 0, 10%, 25%, 50%, 75%, 90% or 100%. This endpoint was the proportion of subjects achieving at least 50% improvement from baseline in T-VASI (T-VASI50) at Week 24. Negative percent change from baseline in T-VASI signified an improvement. Analysis population included all subjects who received at least 1 dose of randomized study medication and had a baseline and at least 1 post-baseline measurement.

End point type

Secondary

End point timeframe

Baseline, Week 24. Baseline was defined as the last measurement prior to first dosing (Day 1).

End point values	PF-06651600 200 mg - 50 mg QD	PF-06651600 100 mg - 50 mg QD	PF-06651600 50 mg QD	PF-06651600 30 mg QD	PF-06651600 10 mg QD	Placebo
Number of subjects analysed	63	65	65	47	49	66
Units: Percentage of Subjects
number (not applicable)	7.9	4.6	4.6	10.6	4.1	9.1

No statistical analyses for this end point

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End point title

Percent Change From Baseline in T-VASI at Designated Time Points - DR Period

End point description

The total body VASI (T-VASI) was calculated using a formula that included contribution from all 6 body regions (possible range, 0-100) with a modified method: VASI = Ʃ [Hand Units] × [Depigmentation]. The body was divided into 6 separate and mutually exclusive regions: face/neck, hands, upper extremities (excluding hands), trunk, lower extremities (excluding the feet), and feet. The extent of depigmentation was expressed by the following percentages: 0, 10%, 25%, 50%, 75%, 90%, or 100%. Percent change from baseline in T-VASI = ((post-baseline T-VASI - baseline T-VASI)/baseline T-VASI)×100. Negative percent change from baseline in T-VASI signified an improvement. The analysis population included all subjects who received at least 1 dose of randomized study medication and had a baseline and at least 1 post-baseline measurement (after taking randomization study medication). The n in the parentheses below represents the number of evaluable subjects analyzed in each arm.

End point type

Secondary

End point timeframe

Baseline, Weeks 4, 8, 12, 16, 20 and 24. Baseline was defined as the last measurement prior to first dosing (Day 1).

End point values	PF-06651600 200 mg - 50 mg QD	PF-06651600 100 mg - 50 mg QD	PF-06651600 50 mg QD	PF-06651600 30 mg QD	PF-06651600 10 mg QD	Placebo
Number of subjects analysed	64	67	67	50	49	66
Units: Percentage
least squares mean (standard error)
Week 4 (n = 64, 66, 64, 50, 47 and 63)	-2.4 ± 1.51	-3.0 ± 1.50	-2.6 ± 1.50	-5.1 ± 1.72	-2.9 ± 1.76	-1.4 ± 1.52
Week 8 (n = 63, 65, 63, 48, 48, 64)	-6.2 ± 2.03	-5.4 ± 2.01	-5.1 ± 2.01	-6.6 ± 2.33	-3.0 ± 2.32	-5.8 ± 2.02
Week 12 (n = 59, 62, 60, 44, 46 and 61)	-7.2 ± 2.44	-11.9 ± 2.40	-9.1 ± 2.40	-11.4 ± 2.82	-4.9 ± 2.76	-8.0 ± 2.40
Week 16 (n = 56, 57, 55, 43, 43 and 58)	-10.0 ± 2.66	-13.1 ± 2.67	-12.6 ± 2.66	-11.9 ± 3.03	-5.7 ± 3.03	-12.0 ± 2.62
Week 20 (n = 52, 59, 53, 43, 41 and 55)	-13.8 ± 3.13	-16.0 ± 2.98	-14.2 ± 3.07	-12.0 ± 3.43	-9.2 ± 3.51	-13.3 ± 3.04
Week 24 (n = 52, 58, 52, 36, 41 and 56)	-14.7 ± 3.49	-19.2 ± 3.29	-14.7 ± 3.44	-14.0 ± 4.15	-12.1 ± 3.88	-11.0 ± 3.34

No statistical analyses for this end point

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End point title

Percent Change From Baseline in Central Read F-VASI at Designated Time Points - DR Period

End point description

Central read F-VASI was assessed based on the facial photographs taken at the site. Central read F-VASI was calculated using a formula that included contribution of affected facial surface areas showing all 6 different depigmentation rates (0.1, 0.25, 0.5, 0.75, 0.9 and 1) with a modified method: F-VASI (central read)=Ʃ [Affected Facial Surface Area]× 4 ×[Depigmentation Rates]. F-VASI (central read) ranged from 0.000 to 4.000 by defining the affected Facial Surface Area (expressed as the value between 0.0 to 1.0) being 4% of total Body Surface Area. Percent change from baseline in F-VASI = ((post-baseline F-VASI - baseline F-VASI)/baseline F-VASI)×100. Negative percent change from baseline in F-VASI signified an improvement. The analysis population included all subjects who received at least 1 dose of randomized study medication and had a baseline and at least 1 post-baseline measurement. The n in the parentheses below represents the number of evaluable subjects analyzed in each arm.

End point type

Secondary

End point timeframe

Baseline, Weeks 4, 8, 16 and 24. Baseline was defined as the last measurement prior to Study Day 18.

End point values	PF-06651600 200 mg - 50 mg QD	PF-06651600 100 mg - 50 mg QD	PF-06651600 50 mg QD	PF-06651600 30 mg QD	PF-06651600 10 mg QD	Placebo
Number of subjects analysed	62	63	59	45	48	57
Units: Percentage
least squares mean (standard error)
Week 4 (n = 61, 60, 55, 44, 47 and 56)	0.1 ± 0.80	0.4 ± 0.82	-0.2 ± 0.84	-1.2 ± 0.94	0.0 ± 0.91	0.6 ± 0.83
Week 8 (n = 57, 57, 52, 43, 47 and 55)	-7.0 ± 1.42	-5.3 ± 1.44	-1.8 ± 1.46	-1.4 ± 1.62	-0.1 ± 1.55	-0.2 ± 1.44
Week 16 (n = 51, 49, 49, 34, 37 and 47)	-17.0 ± 3.16	-16.4 ± 3.25	-10.7 ± 3.19	-13.3 ± 3.87	-5.6 ± 3.67	-0.2 ± 3.27
Week 24 (n = 49, 49, 41, 27, 37 and 50)	-21.2 ± 4.13	-21.2 ± 4.16	-18.5 ± 4.44	-14.6 ± 5.47	-3.0 ± 4.65	2.1 ± 4.06

No statistical analyses for this end point

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End point title

Percent Change From Baseline in Local F-VASI at Designated Time Points - DR Period

End point description

Site assessment of the F-VASI was calculated by a formula that included contribution from face (possible range 0 to 4): Local F-VASI=[Digit Units] × [Depigmentation]×0.1. Scalp, neck, eyebrows, eyelashes, and vermilion were excluded from this calculation. The volar surface of 1 digit (the subject’s thumb) was approximately 0.1% of the total body surface area and was used as a guide to estimate the baseline percentage of vitiligo involvement of face. The extent of depigmentation was expressed by the percentages of 0, 10%, 25%, 50%, 75%, 90%, or 100%. Percent change from baseline in F-VASI = ((post baseline F-VASI - baseline F-VASI)/baseline F-VASI)×100. Negative percent change from baseline in F-VASI signified an improvement. Analysis population included all participants who received at least 1 dose of randomized study medication and had a baseline and at least 1 post-baseline measurement. The n in the parentheses below represents the number of evaluable subjects analyzed in each arm.

End point type

Secondary

End point timeframe

Baseline, Weeks 4, 8, 12, 16, 20 and 24. Baseline was defined as the last measurement prior to first dosing (Day 1).

End point values	PF-06651600 200 mg - 50 mg QD	PF-06651600 100 mg - 50 mg QD	PF-06651600 50 mg QD	PF-06651600 30 mg QD	PF-06651600 10 mg QD	Placebo
Number of subjects analysed	64	67	67	50	49	66
Units: Percentage
least squares mean (standard error)
Week 4 (n = 64, 66, 64, 50, 47 and 63)	-4.9 ± 2.63	3.1 ± 2.61	-5.0 ± 2.60	0.4 ± 2.96	-5.3 ± 3.05	-6.3 ± 2.63
Week 8 (n = 63, 65, 63, 48, 48 and 64)	-11.3 ± 5.35	0.4 ± 5.32	-8.8 ± 5.30	10.1 ± 6.09	-7.3 ± 6.12	-7.5 ± 5.31
Week 12 (n = 59, 62, 60, 44, 46 and 61)	-15.9 ± 4.16	-6.5 ± 4.11	-12.2 ± 4.10	-7.1 ± 4.77	-9.8 ± 4.72	-11.4 ± 4.08
Week 16 (n = 56, 57, 56, 43, 44 and 58)	-19.4 ± 4.85	-6.2 ± 4.88	-19.7 ± 4.82	-6.4 ± 5.48	-10.3 ± 5.46	-13.3 ± 4.75
Week 20 (n = 52, 59, 53, 43, 41 and 55)	-21.9 ± 5.59	-12.9 ± 5.35	-23.2 ± 5.52	0 ± 6.11	-14.4 ± 6.30	-15.3 ± 5.44
Week 24 (n = 52, 58, 52, 36, 41 and 56)	-28.3 ± 5.70	-20.6 ± 5.38	-26.2 ± 5.61	-4.3 ± 6.70	-13.9 ± 6.32	-18.1 ± 5.43

No statistical analyses for this end point

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End point title

Percent Change From Baseline in SA-VES at Designated Time Points - DR Period

End point description

The Self-Assessment Vitiligo Extent Score (SA-VES) was a validated patient report outcome measurement instrument to provide information about disease extent. Vitiligo Extent Score (VES) was a measure to express the overall vitiligo involvement of the body (extent). Clinical illustrations for 19 separate body areas that reflected different degrees of involvement (1%, 5%, 10%, 25%, 50% and 75% depigmentation) were chosen to represent the participant’s skin lesions to get the total extent of the disease. VES was a sum of all surface measurement that was similar to VASI. The analysis population included all subjects who received at least 1 dose of randomized study medication and had a baseline and at least 1 post-baseline measurement (after taking randomization study medication). The n in the parentheses below represents the number of evaluable subjects analyzed in each arm.

End point type

Secondary

End point timeframe

Baseline, Weeks 4, 16 and 24. Baseline was defined as the last measurement prior to first dosing (Day 1).

End point values	PF-06651600 200 mg - 50 mg QD	PF-06651600 100 mg - 50 mg QD	PF-06651600 50 mg QD	PF-06651600 30 mg QD	PF-06651600 10 mg QD	Placebo
Number of subjects analysed	64	67	67	50	49	66
Units: Percentage
least squares mean (standard error)
Week 4 (n = 62, 66, 64, 50, 47 and 63)	2.7 ± 17.19	1.1 ± 16.71	-1.1 ± 16.74	4.9 ± 19.25	4.9 ± 19.84	50.4 ± 17.13
Week 16 (n = 54, 55, 56, 41, 44 and 58)	4.7 ± 13.82	-0.5 ± 13.48	-3.7 ± 13.45	6.9 ± 15.53	-0.5 ± 15.87	52.5 ± 13.72
Week 24 (n = 50, 56, 49, 32, 40 and 55)	0 ± 10.35	-3.5 ± 10.02	-4.3 ± 10.11	-4.4 ± 11.89	-1.6 ± 11.77	44.0 ± 10.16

No statistical analyses for this end point

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End point title

Absolute Change From Baseline in T-VASI at Designated Time Points - DR Period

End point description

The T-VASI was calculated using a formula that included contribution from 6 different body regions (possible range, 0-100) with a modified method: VASI = Ʃ [Hand Units] × [Depigmentation]. The body was divided into 6 separate and mutually exclusive regions: face/neck, hands, upper extremities (excluding hands), trunk, lower extremities (excluding the feet), and feet. The extent of depigmentation was expressed by the following percentages: 0, 10%, 25%, 50%, 75%, 90%, or 100%. The absolute change from baseline in T-VASI was analyzed using the ANCOVA analysis. Negative change from baseline in T-VASI signified an improvement. The analysis population included all participants who received at least 1 dose of randomized study medication and had a baseline and at least 1 post-baseline measurement. The n in the parentheses below represents the number of evaluable subjects analyzed in each arm.

End point type

Secondary

End point timeframe

Baseline, Weeks 4, 8, 12, 16, 20 and 24. Baseline was defined as the last measurement prior to first dosing (Day 1).

End point values	PF-06651600 200 mg - 50 mg QD	PF-06651600 100 mg - 50 mg QD	PF-06651600 50 mg QD	PF-06651600 30 mg QD	PF-06651600 10 mg QD	Placebo
Number of subjects analysed	64	67	67	50	49	66
Units: Units on a Scale
least squares mean (standard error)
Week 4 (n = 64, 67, 67, 50, 49 and 66)	-0.3 ± 0.25	-0.5 ± 0.25	-0.6 ± 0.25	-1.0 ± 0.28	-0.4 ± 0.29	-0.2 ± 0.25
Week 8 (n = 63, 65, 63, 48, 48 and 64)	-0.9 ± 0.32	-0.9 ± 0.32	-0.9 ± 0.32	-1.2 ± 0.37	-0.5 ± 0.36	-0.9 ± 0.32
Week 12 (n = 59, 62, 60, 44, 46 and 61)	-1.1 ± 0.41	-2.0 ± 0.40	-1.6 ± 0.40	-1.8 ± 0.47	-0.9 ± 0.46	-1.6 ± 0.40
Week 16 (n = 56, 57, 55, 43, 43 and 58)	-1.5 ± 0.46	-2.1 ± 0.46	-1.9 ± 0.46	-1.9 ± 0.53	-0.9 ± 0.53	-2.2 ± 0.46
Week 20 (n = 52, 59, 53, 43, 41 and 55)	-2.0 ± 0.55	-2.8 ± 0.53	-2.3 ± 0.54	-2.0 ± 0.61	-1.7 ± 0.62	-2.2 ± 0.54
Week 24 (n = 52, 58, 52, 36, 41 and 56)	-2.3 ± 0.62	-3.4 ± 0.59	-2.4 ± 0.61	-2.7 ± 0.74	-2.0 ± 0.69	-1.8 ± 0.60

No statistical analyses for this end point

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End point title

Proportion of Subjects Achieving T-VASI50 at Designated Time Points - DR Period

End point description

The T-VASI was calculated using a formula that included contribution from 6 different body regions (possible range, 0-100) with a modified method: T-VASI = Ʃ [Hand Units]×[Depigmentation]. The body was divided into 6 separate and mutually exclusive regions: face/neck, hands, upper extremities, trunk, lower extremities, and feet. The extent of depigmentation was expressed by the percentages: 0, 10%, 25%, 50%, 75%, 90% or 100%. This endpoint was the proportion of subjects achieving at least 50% improvement from baseline in T-VASI (T-VASI50). Negative percent change from baseline in T-VASI signified an improvement. Percent change from baseline in T-VASI = ((post-baseline T-VASI - baseline T-VASI)/baseline T-VASI)×100. The analysis population included all participants who received at least 1 dose of randomized study medication and had a baseline and at least 1 post-baseline measurement. The n in the parentheses below represents the number of evaluable subjects analyzed in each arm.

End point type

Secondary

End point timeframe

Baseline, Weeks 4, 8, 12, 16, 20 and 24. Baseline was defined as the last measurement prior to first dosing (Day 1).

End point values	PF-06651600 200 mg - 50 mg QD	PF-06651600 100 mg - 50 mg QD	PF-06651600 50 mg QD	PF-06651600 30 mg QD	PF-06651600 10 mg QD	Placebo
Number of subjects analysed	64	67	67	50	49	66
Units: Percentage of Subjects
number (not applicable)
Week 4 (n = 64, 67, 67, 50, 49 and 66)	1.6	1.5	0	2.0	0	0
Week 8 (n = 64, 67, 67, 50, 49 and 66)	1.6	1.5	1.5	4.0	0	1.5
Week 12 (n = 62, 66, 66, 49, 48 and 66)	1.6	1.5	1.5	4.1	0	4.5
Week 16 (n = 63, 62, 66, 47, 46 and 63)	0	3.2	0	2.1	0	6.3
Week 20 (n = 61, 67, 64, 49, 48 and 63)	4.9	1.5	1.6	6.1	2.1	9.5
Week 24 (n = 63, 65, 65, 47, 49 and 66)	7.9	4.6	4.6	10.6	4.1	9.1

No statistical analyses for this end point

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End point title

Proportion of Subjects Achieving T-VASI75 at Designated Time Points - DR Period

End point description

The T-VASI was calculated using a formula that included contribution from 6 different body regions (possible range, 0-100) with a modified method: T-VASI = Ʃ [Hand Units]×[Depigmentation]. The body was divided into 6 separate and mutually exclusive regions: face/neck, hands, upper extremities, trunk, lower extremities, and feet. The extent of depigmentation was expressed by the percentages: 0, 10%, 25%, 50%, 75%, 90% or 100%. This endpoint was the proportion of subjects achieving at least 75% improvement from baseline in T-VASI (T-VASI75). Negative percent change from baseline in T-VASI signified an improvement. Percent change from baseline in T-VASI = ((post-baseline T-VASI - baseline T-VASI)/baseline T-VASI)×100. The analysis population included all participants who received at least 1 dose of randomized study medication and had a baseline and at least 1 post-baseline measurement. The n in the parentheses below represents the number of evaluable subjects analyzed in each arm.

End point type

Secondary

End point timeframe

Baseline, Weeks 4, 8, 12, 16, 20 and 24. Baseline was defined as the last measurement prior to first dosing (Day 1).

End point values	PF-06651600 200 mg - 50 mg QD	PF-06651600 100 mg - 50 mg QD	PF-06651600 50 mg QD	PF-06651600 30 mg QD	PF-06651600 10 mg QD	Placebo
Number of subjects analysed	64	67	67	50	49	66
Units: Percentage of Subjects
number (not applicable)
Week 4 (n = 64, 67, 67, 50, 49 and 66)	0	0	0	0	0	0
Week 8 (n = 64, 67, 67, 50, 49 and 66)	0	0	0	0	0	0
Week 12 (n = 62, 66, 66, 49, 48 and 66)	0	0	0	0	0	0
Week 16 (n = 63, 62, 66, 47, 46 and 63)	0	0	0	0	0	0
Week 20 (n = 61, 67, 64, 49, 48 and 63)	0	0	0	0	0	0
Week 24 (n = 63, 65, 65, 47, 49 and 66)	0	0	0	0	0	0

No statistical analyses for this end point

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End point title

Proportion of Subjects Achieving T-VASI90 at Designated Time Points - DR Period

End point description

The T-VASI was calculated using a formula that included contribution from 6 different body regions (possible range, 0-100) with a modified method: T-VASI = Ʃ [Hand Units]×[Depigmentation]. The body was divided into 6 separate and mutually exclusive regions: face/neck, hands, upper extremities, trunk, lower extremities, and feet. The extent of depigmentation was expressed by the percentages: 0, 10%, 25%, 50%, 75%, 90% or 100%. This endpoint was the proportion of subjects achieving at least 90% improvement from baseline in T-VASI (T-VASI90). Negative percent change from baseline in T-VASI signified an improvement. Percent change from baseline in T-VASI = ((post-baseline T-VASI - baseline T-VASI)/baseline T-VASI)×100. The analysis population included all participants who received at least 1 dose of randomized study medication and had a baseline and at least 1 post-baseline measurement. The n in the parentheses below represents the number of evaluable subjects analyzed in each arm.

End point type

Secondary

End point timeframe

Baseline, Weeks 4, 8, 12, 16, 20 and 24. Baseline was defined as the last measurement prior to first dosing (Day 1).

End point values	PF-06651600 200 mg - 50 mg QD	PF-06651600 100 mg - 50 mg QD	PF-06651600 50 mg QD	PF-06651600 30 mg QD	PF-06651600 10 mg QD	Placebo
Number of subjects analysed	64	67	67	50	49	66
Units: Percentage of Subjects
number (not applicable)
Week 4 (n = 64, 67, 67, 50, 49 and 66)	0	0	0	0	0	0
Week 8 (n = 64, 67, 67, 50, 49 and 66)	0	0	0	0	0	0
Week 12 (n = 62, 66, 66, 49, 48 and 66)	0	0	0	0	0	0
Week 16 (n = 63, 62, 66, 47, 46 and 63)	0	0	0	0	0	0
Week 20 (n = 61, 67, 64, 49, 48 and 63)	0	0	0	0	0	0
Week 24 (n = 63, 65, 65, 47, 49 and 66)	0	0	0	0	0	0

No statistical analyses for this end point

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End point title

Proportion of Subjects Achieving T-VASI100 at Designated Time Points - DR Period

End point description

The T-VASI was calculated using a formula that included contribution from 6 different body regions (possible range, 0-100) with a modified method: T-VASI = Ʃ [Hand Units]×[Depigmentation]. The body was divided into 6 separate and mutually exclusive regions: face/neck, hands, upper extremities, trunk, lower extremities, and feet. The extent of depigmentation was expressed by the percentages: 0, 10%, 25%, 50%, 75%, 90% or 100%. This endpoint was the proportion of subjects achieving at least 100% improvement from baseline in T-VASI (T-VASI100). Negative percent change from baseline in T-VASI signified an improvement. Percent change from baseline in T-VASI = ((post-baseline T-VASI - baseline T-VASI)/baseline T-VASI)×100. The analysis population included all participants who received at least 1 dose of randomized study medication and had a baseline and at least 1 post-baseline measurement. The n in the parentheses below represents the number of evaluable subjects analyzed in each arm.

End point type

Secondary

End point timeframe

Baseline, Weeks 4, 8, 12, 16, 20 and 24. Baseline was defined as the last measurement prior to first dosing (Day 1).

End point values	PF-06651600 200 mg - 50 mg QD	PF-06651600 100 mg - 50 mg QD	PF-06651600 50 mg QD	PF-06651600 30 mg QD	PF-06651600 10 mg QD	Placebo
Number of subjects analysed	64	67	67	50	49	66
Units: Percentage of Subjects
number (not applicable)
Week 4 (n = 64, 67, 67, 50, 49 and 66)	0	0	0	0	0	0
Week 8 (n = 64, 67, 67, 50, 49 and 66)	0	0	0	0	0	0
Week 12 (n = 62, 66, 66, 49, 48 and 66)	0	0	0	0	0	0
Week 16 (n = 63, 62, 66, 47, 46 and 63)	0	0	0	0	0	0
Week 20 (n = 61, 67, 64, 49, 48 and 63)	0	0	0	0	0	0
Week 24 (n = 63, 65, 65, 47, 49 and 66)	0	0	0	0	0	0

No statistical analyses for this end point

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End point title

Proportion of Subjects Achieving Central Read F-VASI50 at Designated Time Points - DR Period

End point description

The central read F-VASI was calculated using a formula that included contribution of affected facial surface areas showing all 6 different depigmentation rates (0.1, 0.25, 0.5, 0.75, 0.9 and 1): F-VASI (central read)=Ʃ [Affected Facial Surface Area] × 4 × [Depigmentation Rates]. F-VASI (central read) ranged from 0.000 to 4.000 by defining the affected Facial Surface Area (expressed as the value between 0.0 to 1.0) being 4% of total Body Surface Area. This endpoint was the proportion of subjects achieving at least 50% improvement in central read F-VASI from baseline (F-VASI50). Negative percent change from baseline in F-VASI signified an improvement. The analysis population included all subjects who received at least 1 dose of randomized study medication and had a baseline and at least 1 post-baseline measurement. The n in the parentheses below represents the number of evaluable subjects analyzed in each arm.

End point type

Secondary

End point timeframe

Baseline, Weeks 4, 8, 16 and 24. Baseline was defined as the last measurement prior to Study Day 18.

End point values	PF-06651600 200 mg - 50 mg QD	PF-06651600 100 mg - 50 mg QD	PF-06651600 50 mg QD	PF-06651600 30 mg QD	PF-06651600 10 mg QD	Placebo
Number of subjects analysed	62	63	59	45	48	57
Units: Percentage of Subjects
number (not applicable)
Week 4 (n = 62, 63, 59, 45, 48 and 57)	0	0	0	2.2	0	0
Week 8 (n = 62, 63, 59, 45, 48 and 57)	3.2	0	0	2.2	0	0
Week 16 (n = 61, 58, 58, 39, 42 and 52)	14.8	13.8	6.9	10.3	2.4	1.9
Week 24 (n = 58, 59, 52, 37, 43 and 57)	22.4	25.4	15.4	18.9	7.0	1.8

No statistical analyses for this end point

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End point title

Proportion of Subjects Achieving Central Read F-VASI75 at Designated Time Points - DR Period

End point description

The central read F-VASI was calculated using a formula that included contribution of affected facial surface areas showing all 6 different depigmentation rates (0.1, 0.25, 0.5, 0.75, 0.9 and 1): F-VASI (central read)=Ʃ [Affected Facial Surface Area] × 4 × [Depigmentation Rates]. F-VASI (central read) ranged from 0.000 to 4.000 by defining the affected Facial Surface Area (expressed as the value between 0.0 to 1.0) being 4% of total Body Surface Area. This endpoint was the proportion of subjects achieving at least 75% improvement in central read F-VASI from baseline (F-VASI75). Negative percent change from baseline in F-VASI signified an improvement. The analysis population included all subjects who received at least 1 dose of randomized study medication and had a baseline and at least 1 post-baseline measurement. The n in the parentheses below represents the number of evaluable subjects analyzed in each arm.

End point type

Secondary

End point timeframe

Baseline, Weeks 4, 8, 16 and 24. Baseline was defined as the last measurement prior to Study Day 18.

End point values	PF-06651600 200 mg - 50 mg QD	PF-06651600 100 mg - 50 mg QD	PF-06651600 50 mg QD	PF-06651600 30 mg QD	PF-06651600 10 mg QD	Placebo
Number of subjects analysed	62	63	59	45	48	57
Units: Percentage of Subjects
number (not applicable)
Week 4 (n = 62, 63, 59, 45, 48 and 57)	0	0	0	0	0	0
Week 8 (n = 62, 63, 59, 45, 48 and 57)	0	0	0	0	0	0
Week 16 (n = 61, 58, 58, 39, 42 and 52)	1.6	1.7	5.2	2.6	2.4	0
Week 24 (n = 58, 59, 52, 37, 43 and 57)	12.1	8.5	7.7	2.7	2.3	0

No statistical analyses for this end point

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End point title

Proportion of Subjects Achieving Central Read F-VASI90 at Designated Time Points - DR Period

End point description

The central read F-VASI was calculated using a formula that included contribution of affected facial surface areas showing all 6 different depigmentation rates (0.1, 0.25, 0.5, 0.75, 0.9 and 1): F-VASI (central read)=Ʃ [Affected Facial Surface Area] × 4 × [Depigmentation Rates]. F-VASI (central read) ranged from 0.000 to 4.000 by defining the affected Facial Surface Area (expressed as the value between 0.0 to 1.0) being 4% of total Body Surface Area. This endpoint was the proportion of subjects achieving at least 90% improvement in central read F-VASI from baseline (F-VASI90). Negative percent change from baseline in F-VASI signified an improvement. The analysis population included all subjects who received at least 1 dose of randomized study medication and had a baseline and at least 1 post-baseline measurement. The n in the parentheses below represents the number of evaluable subjects analyzed in each arm.

End point type

Secondary

End point timeframe

Baseline, Weeks 4, 8, 16 and 24. Baseline was defined as the last measurement prior to Study Day 18.

End point values	PF-06651600 200 mg - 50 mg QD	PF-06651600 100 mg - 50 mg QD	PF-06651600 50 mg QD	PF-06651600 30 mg QD	PF-06651600 10 mg QD	Placebo
Number of subjects analysed	62	63	59	45	48	57
Units: Percentage of Subjects
number (not applicable)
Week 4 (n = 62, 63, 59, 45, 48 and 57)	0	0	0	0	0	0
Week 8 (n = 62, 63, 59, 45, 48 and 57)	0	0	0	0	0	0
Week 16 (n = 61, 58, 58, 39, 42 and 52)	0	0	1.7	0	0	0
Week 24 (n = 58, 59, 52, 37, 43 and 57)	1.7	0	3.8	0	0	0

No statistical analyses for this end point

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End point title

Proportion of Subjects Achieving Central Read F-VASI100 at Designated Time Points - DR Period

End point description

The central read F-VASI was calculated using a formula that included contribution of affected facial surface areas showing all 6 different depigmentation rates (0.1, 0.25, 0.5, 0.75, 0.9 and 1): F-VASI (central read)=Ʃ [Affected Facial Surface Area] × 4 × [Depigmentation Rates]. F-VASI (central read) ranged from 0.000 to 4.000 by defining the affected Facial Surface Area (expressed as the value between 0.0 to 1.0) being 4% of total Body Surface Area. This endpoint was the proportion of subjects achieving at least 100% improvement in central read F-VASI from baseline (F-VASI100). Negative percent change from baseline in F-VASI signified an improvement. The analysis population included all subjects who received at least 1 dose of randomized study medication and had a baseline and at least 1 post-baseline measurement. The n in the parentheses below represents the number of evaluable subjects analyzed in each arm.

End point type

Secondary

End point timeframe

Baseline, Weeks 4, 8, 16 and 24. Baseline was defined as the last measurement prior to Study Day 18.

End point values	PF-06651600 200 mg - 50 mg QD	PF-06651600 100 mg - 50 mg QD	PF-06651600 50 mg QD	PF-06651600 30 mg QD	PF-06651600 10 mg QD	Placebo
Number of subjects analysed	62	63	59	45	48	57
Units: Percentage of Subjects
number (not applicable)
Week 4 (n = 62, 63, 59, 45, 48 and 57)	0	0	0	0	0	0
Week 8 (n = 62, 63, 59, 45, 48 and 57)	0	0	0	0	0	0
Week 16 (n = 61, 58, 58, 39, 42 and 52)	0	0	0	0	0	0
Week 24 (n = 58, 59, 52, 37, 43 and 57)	0	0	0	0	0	0

No statistical analyses for this end point

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End point title

Proportion of Subjects Achieving Local F-VASI50 at Designated Time Points - DR Period

End point description

The site assessment of the F-VASI was calculated using a formula that included contribution from face (possible range, 0-4): Local F-VASI = [Digit Units] × [Depigmentation] × 0.1. The volar surface of 1 digit (the subject’s thumb) was approximately 0.1% of the total body surface area and was used as a guide to estimate the baseline percentage of vitiligo involvement of face. The extent of depigmentation was expressed by the percentages: 0, 10%, 25%, 50%, 75%, 90%, or 100%. This endpoint was the proportion of subjects achieving at least 50% improvement in site assessment F-VASI from baseline (F-VASI50). Negative percent change from baseline in F-VASI signified an improvement. The analysis population included all subjects who received at least 1 dose of randomized study medication and had a baseline and at least 1 post-baseline measurement. The n in the parentheses below represents the number of evaluable subjects analyzed in each arm.

End point type

Secondary

End point timeframe

Baseline, Weeks 4, 8, 12, 16, 20 and 24. Baseline was defined as the last measurement prior to first dosing (Day 1).

End point values	PF-06651600 200 mg - 50 mg QD	PF-06651600 100 mg - 50 mg QD	PF-06651600 50 mg QD	PF-06651600 30 mg QD	PF-06651600 10 mg QD	Placebo
Number of subjects analysed	64	67	67	50	49	66
Units: Percentage of Subjects
number (not applicable)
Week 4 (n = 64, 67, 67, 50, 49 and 66)	3.1	3.0	1.5	2.0	0	3.0
Week 8 (n = 64, 67, 67, 50, 49 and 66)	9.4	3.0	4.5	2.0	4.1	4.5
Week 12 (n = 62, 66, 66, 49, 48 and 66)	16.1	9.1	9.1	8.2	8.3	9.1
Week 16 (n = 63, 62, 66, 47, 47 and 63)	14.3	9.7	13.6	8.5	10.6	14.3
Week 20 (n = 61, 67, 64, 49, 48, 63)	18.0	14.9	17.2	12.2	16.7	17.5
Week 24 (n = 63, 65, 65, 47, 49 and 66)	20.6	21.5	15.4	10.6	18.4	16.7

No statistical analyses for this end point

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End point title

Proportion of Subjects Achieving Local F-VASI75 at Designated Time Points - DR Period

End point description

The site assessment of the F-VASI was calculated using a formula that included contribution from face (possible range, 0-4): Local F-VASI = [Digit Units] × [Depigmentation] × 0.1. The volar surface of 1 digit (the subject’s thumb) was approximately 0.1% of the total body surface area and was used as a guide to estimate the baseline percentage of vitiligo involvement of face. The extent of depigmentation was expressed by the percentages: 0, 10%, 25%, 50%, 75%, 90%, or 100%. This endpoint was the proportion of subjects achieving at least 75% improvement in site assessment F-VASI from baseline (F-VASI75). Negative percent change from baseline in F-VASI signified an improvement. The analysis population included all subjects who received at least 1 dose of randomized study medication and had a baseline and at least 1 post-baseline measurement. The n in the parentheses below represents the number of evaluable subjects analyzed in each arm.

End point type

Secondary

End point timeframe

Baseline, Weeks 4, 8, 12, 16, 20 and 24. Baseline was defined as the last measurement prior to first dosing (Day 1).

End point values	PF-06651600 200 mg - 50 mg QD	PF-06651600 100 mg - 50 mg QD	PF-06651600 50 mg QD	PF-06651600 30 mg QD	PF-06651600 10 mg QD	Placebo
Number of subjects analysed	64	67	67	50	49	66
Units: Percentage of Subjects
number (not applicable)
Week 4 (n = 64, 67, 67, 50, 49 and 66)	0	0	0	0	0	1.5
Week 8 (n = 64, 67, 67, 50, 49 and 66)	0	0	3.0	2.0	0	0
Week 12 (n = 62, 66, 66, 49, 48 and 66)	1.6	3.0	4.5	6.1	2.1	1.5
Week 16 (n = 63, 62, 66, 47, 47 and 63)	3.2	0	4.5	4.3	4.3	7.9
Week 20 (n = 61, 67, 64, 49, 48 and 63)	6.6	1.5	9.4	6.1	6.3	11.1
Week 24 (n = 63, 65, 65, 47, 49 and 66)	11.1	4.6	6.2	4.3	4.1	13.6

No statistical analyses for this end point

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End point title

Proportion of Subjects Achieving Local F-VASI90 at Designated Time Points - DR Period

End point description

The site assessment of the F-VASI was calculated using a formula that included contribution from face (possible range, 0-4): Local F-VASI = [Digit Units] × [Depigmentation] × 0.1. The volar surface of 1 digit (the subject’s thumb) was approximately 0.1% of the total body surface area and was used as a guide to estimate the baseline percentage of vitiligo involvement of face. The extent of depigmentation was expressed by the percentages: 0, 10%, 25%, 50%, 75%, 90%, or 100%. This endpoint was the proportion of subjects achieving at least 90% improvement in site assessment F-VASI from baseline (F-VASI90). Negative percent change from baseline in F-VASI signified an improvement. The analysis population included all subjects who received at least 1 dose of randomized study medication and had a baseline and at least 1 post-baseline measurement. The n in the parentheses below represents the number of evaluable subjects analyzed in each arm.

End point type

Secondary

End point timeframe

Baseline, Weeks 4, 8, 12, 16, 20 and 24. Baseline was defined as the last measurement prior to first dosing (Day 1).

End point values	PF-06651600 200 mg - 50 mg QD	PF-06651600 100 mg - 50 mg QD	PF-06651600 50 mg QD	PF-06651600 30 mg QD	PF-06651600 10 mg QD	Placebo
Number of subjects analysed	64	67	67	50	49	66
Units: Percentage of Subjects
number (not applicable)
Week 4 (n = 64, 67, 67, 50, 49 and 66)	0	0	0	0	0	0
Week 8 (n = 64, 67, 67, 50, 49 and 66)	0	0	1.5	2.0	0	0
Week 12 (n = 62, 66, 66, 49, 48 and 66)	0	0	1.5	2.0	0	0
Week 16 (n = 63, 62, 66, 47, 47 and 63)	1.6	0	3.0	2.1	2.1	0
Week 20 (n = 61, 67, 64, 49, 48 and 63)	1.6	1.5	4.7	2.0	2.1	1.6
Week 24 (n = 63, 65, 65, 47, 49 and 66)	1.6	1.5	3.1	0	4.1	1.5

No statistical analyses for this end point

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End point title

Proportion of Subjects Achieving Local F-VASI100 at Designated Time Points - DR Period

End point description

The site assessment of the F-VASI was calculated using a formula that included contribution from face (possible range, 0-4): Local F-VASI = [Digit Units] × [Depigmentation] × 0.1. The volar surface of 1 digit (the subject’s thumb) was approximately 0.1% of the total body surface area and was used as a guide to estimate the baseline percentage of vitiligo involvement of face. The extent of depigmentation was expressed by the percentages: 0, 10%, 25%, 50%, 75%, 90%, or 100%. This endpoint was the proportion of subjects achieving at least 100% improvement in site assessment F-VASI from baseline (F-VASI100). Negative percent change from baseline in F-VASI signified an improvement. The analysis population included all subjects who received at least 1 dose of randomized study medication and had a baseline and at least 1 post-baseline measurement. The n in the parentheses below represents the number of evaluable subjects analyzed in each arm.

End point type

Secondary

End point timeframe

Baseline, Weeks 4, 8, 12, 16, 20 and 24. Baseline was defined as the last measurement prior to first dosing (Day 1).

End point values	PF-06651600 200 mg - 50 mg QD	PF-06651600 100 mg - 50 mg QD	PF-06651600 50 mg QD	PF-06651600 30 mg QD	PF-06651600 10 mg QD	Placebo
Number of subjects analysed	64	67	67	50	49	66
Units: Percentage of Subjects
number (not applicable)
Week 4 (n = 64, 67, 67, 50, 49 and 66)	0	0	0	0	0	0
Week 8 (n = 64, 67, 67, 50, 49 and 66)	0	0	0	2.0	0	0
Week 12 (n = 62, 66, 66, 49, 48 and 66)	0	0	0	0	0	0
Week 16 (n = 63, 62, 66, 47, 47 and 63)	0	0	0	0	0	0
Week 20 (n = 61, 67, 64, 49, 48 and 63)	0	0	1.6	0	0	0
Week 24 (n = 63, 65, 65, 47, 49 and 66)	0	0	1.5	0	0	0

No statistical analyses for this end point

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End point title

Change From Baseline in Total Vitiligo-Specific Quality of Life (VitiQoL) Score at Designated Time Points - DR Period

End point description

The VitiQoL instrument was a reliable and validated vitiligo disease-specific health-related quality of life (HRQoL) instrument which measured concepts relevant to vitiligo participants. The VitiQoL was a 15-item patient reported outcomes (PRO) measure which measured concepts of symptoms, daily activities, leisure activities, work, personal relationships and treatment. Responses ranged from “not at all” (scored 0) to “most of the time” (scored 6) and gave a minimum and maximum score from 0 90, with higher scores representing greater burden. VitiQoL total score was calculated as sum of items 1-15. The change from baseline in total VitiQoL score was analyzed using the mixed-effect models repeated measures (MMRM) analysis. The analysis population included all subjects who received at least 1 dose of randomized study medication and had a baseline and at least 1 post-baseline measurement. The n in the parentheses below represents the number of evaluable subjects analyzed in each arm.

End point type

Secondary

End point timeframe

Baseline, Weeks 4, 16 and 24. Baseline was defined as the last measurement prior to first dosing (Day 1).

End point values	PF-06651600 200 mg - 50 mg QD	PF-06651600 100 mg - 50 mg QD	PF-06651600 50 mg QD	PF-06651600 30 mg QD	PF-06651600 10 mg QD	Placebo
Number of subjects analysed	64	67	67	50	49	66
Units: Units on a Scale
least squares mean (standard error)
Week 4 (n = 62, 66, 64, 50, 47 and 63)	-3.9 ± 1.34	-2.9 ± 1.31	-4.4 ± 1.31	-4.4 ± 1.50	-3.5 ± 1.54	-5.1 ± 1.33
Week 16 (n = 54, 56, 56, 41, 44 and 59)	-6.5 ± 1.83	-4.9 ± 1.79	-6.3 ± 1.82	-4.8 ± 2.07	-10.5 ± 2.06	-7.1 ± 1.78
Week 24 (n = 50, 56, 50, 34, 41 and 56)	-6.5 ± 1.99	-3.6 ± 1.92	-7.7 ± 1.99	-7.0 ± 2.30	-9.7 ± 2.22	-6.4 ± 1.92

No statistical analyses for this end point

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End point title

Change From Baseline in VitiQoL Participation Limitation Domain Score at Designated Time Points - DR Period

End point description

The VitiQoL was a reliable and validated vitiligo disease specific health-related quality of life (HRQoL) instrument which measured concepts relevant to vitiligo participants. The VitiQoL was a 15-item PRO measure which measured concepts of symptoms, daily activities, leisure activities, work, personal relationships and treatment. Responses ranged from “not at all” (scored 0) to “most of the time” (scored 6) and gave a minimum and maximum score from 0 90, with higher scores representing greater burden. The VitiQoL Participation Limitation domain score was the sum of items 3, 4, 6, 9, 10, 11, 14. The change from baseline in VitiQoL Participation Limitation domain score was analyzed using the MMRM analysis. The analysis population included all subjects who received at least 1 dose of randomized study medication and had a baseline and at least 1 post-baseline measurement. The n in the parentheses below represents the number of evaluable subjects analyzed in each arm.

End point type

Secondary

End point timeframe

Baseline, Weeks 4, 16 and 24. Baseline was defined as the last measurement prior to first dosing (Day 1).

End point values	PF-06651600 200 mg - 50 mg QD	PF-06651600 100 mg - 50 mg QD	PF-06651600 50 mg QD	PF-06651600 30 mg QD	PF-06651600 10 mg QD	Placebo
Number of subjects analysed	64	67	67	50	49	66
Units: Units on a Scale
least squares mean (standard error)
Week 4 (n = 62, 66, 64, 50, 47 and 63)	-1.2 ± 0.71	-1.1 ± 0.69	-1.7 ± 0.69	-1.8 ± 0.79	-1.0 ± 0.81	-2.5 ± 0.70
Week 16 (n = 54, 56, 56, 41, 44 and 59)	-2.1 ± 0.88	-1.9 ± 0.86	-2.6 ± 0.87	-2.3 ± 1.00	-4.1 ± 0.99	-3.1 ± 0.85
Week 24 (n = 50, 56, 50, 34, 41 and 56)	-1.4 ± 0.98	-1.7 ± 0.94	-3.2 ± 0.97	-2.8 ± 1.14	-3.9 ± 1.09	-2.2 ± 0.94

No statistical analyses for this end point

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End point title

Change From Baseline in VitiQoL Stigma Domain Score at Designated Time Points - DR Period

End point description

The VitiQoL was a reliable and validated vitiligo disease specific HRQoL instrument which measured concepts relevant to vitiligo participants. The VitiQoL was a 15-item PRO measure which measured concepts of symptoms, daily activities, leisure activities, work, personal relationships and treatment. Responses ranged from “not at all” (scored 0) to “most of the time” (scored 6) and gave a minimum and maximum score from 0 90, with higher scores representing greater burden. The VitiQoL Stigma domain score was the sum of items 1, 2, 5, 7 and 15. The change from baseline in VitiQoL Stigma domain score was analyzed using the MMRM analysis. The analysis population included all subjects who received at least 1 dose of randomized study medication and had a baseline and at least 1 post-baseline measurement. The n in the parentheses below represents the number of evaluable subjects analyzed in each arm.

End point type

Secondary

End point timeframe

Baseline, Weeks 4, 16 and 24. Baseline was defined as the last measurement prior to first dosing (Day 1).

End point values	PF-06651600 200 mg - 50 mg QD	PF-06651600 100 mg - 50 mg QD	PF-06651600 50 mg QD	PF-06651600 30 mg QD	PF-06651600 10 mg QD	Placebo
Number of subjects analysed	64	67	67	50	49	66
Units: Units on a Scale
least squares mean (standard error)
Week 4 (n = 62, 66, 64, 50, 47 and 63)	-1.9 ± 0.56	-1.6 ± 0.55	-2.4 ± 0.55	-1.7 ± 0.62	-1.7 ± 0.64	-1.8 ± 0.56
Week 16 (n = 54, 56, 56, 41, 44 and 59)	-3.4 ± 0.71	-2.3 ± 0.70	-3.2 ± 0.71	-1.7 ± 0.80	-4.3 ± 0.80	-2.9 ± 0.69
Week 24 (n = 50, 56, 50, 34, 41 and 56)	-3.8 ± 0.76	-2.1 ± 0.73	-3.7 ± 0.76	-3.1 ± 0.88	-4.5 ± 0.84	-3.2 ± 0.73

No statistical analyses for this end point

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End point title

Change From Baseline in VitiQoL Behaviors Domain Score at Designated Time Points - DR Period

End point description

The VitiQoL was a reliable and validated vitiligo disease specific HRQoL instrument which measured concepts relevant to vitiligo participants. The VitiQoL was a 15-item PRO measure which measured concepts of symptoms, daily activities, leisure activities, work, personal relationships and treatment. Responses ranged from “not at all” (scored 0) to “most of the time” (scored 6) and gave a minimum and maximum score from 0 90, with higher scores representing greater burden. The VitiQoL Behaviors domain score was the sum of items 8, 12 and 13. The change from baseline in VitiQoL Behaviors domain score was analyzed using the MMRM analysis. The analysis population included all subjects who received at least 1 dose of randomized study medication and had a baseline and at least 1 post-baseline measurement. The n in the parentheses below represents the number of evaluable subjects analyzed in each arm.

End point type

Secondary

End point timeframe

Baseline, Weeks 4, 16 and 24. Baseline was defined as the last measurement prior to first dosing (Day 1).

End point values	PF-06651600 200 mg - 50 mg QD	PF-06651600 100 mg - 50 mg QD	PF-06651600 50 mg QD	PF-06651600 30 mg QD	PF-06651600 10 mg QD	Placebo
Number of subjects analysed	64	67	67	50	49	66
Units: Units on a Scale
least squares mean (standard error)
Week 4 (n = 62, 66, 64, 50, 47 and 63)	-0.8 ± 0.37	-0.3 ± 0.36	-0.2 ± 0.36	-0.9 ± 0.41	-0.7 ± 0.42	-0.7 ± 0.37
Week 16 (n = 54, 56, 56, 41, 44 and 59)	-1.1 ± 0.50	-0.8 ± 0.49	-0.5 ± 0.50	-0.9 ± 0.57	-2.1 ± 0.56	-1.0 ± 0.49
Week 24 (n = 50, 56, 50, 34, 41 and 56)	-1.4 ± 0.53	0 ± 0.52	-0.7 ± 0.53	-1.3 ± 0.62	-1.3 ± 0.60	-0.9 ± 0.51

No statistical analyses for this end point

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End point title

Proportion of Subjects Achieving sIGA 0 or 1 and at Least a 2-Point Improvement at Week 24 - DR Period

End point description

The proportion of participants achieving a static Investigator Global Assessment (sIGA) Score 0/1 and sIGA ≥2-point improvement at Week 24 was presented in this outcome measure. The sIGA score ranged from 0 to 4. The sIGA Score 0 represented "Clear" with no signs of loss of pigmentation with natural light or with Woods lamp examination. The sIGA Score 1 represented "Almost Clear" with the following descriptors: • Faint, barely detectable loss of pigmentation mainly located on dorsal hands, feet, bony prominences, and/or limited areas. • Approximately 90% pigmentation within lesions. • No or rare signs of Koebner phenomenon, confetti like or trichrome lesions could be present. The sIGA Scores 2, 3 and 4 represented "Mild Vitiligo", "Moderate Vitiligo" and "Severe Vitiligo", respectively. The analysis population included all subjects who received at least 1 dose of randomized study medication and had a baseline and at least 1 post-baseline measurement.

End point type

Secondary

End point timeframe

Week 24

End point values	PF-06651600 200 mg - 50 mg QD	PF-06651600 100 mg - 50 mg QD	PF-06651600 50 mg QD	PF-06651600 30 mg QD	PF-06651600 10 mg QD	Placebo
Number of subjects analysed	62	64	65	46	48	65
Units: Percentage of Subjects
number (not applicable)	0	0	0	0	0	0

No statistical analyses for this end point

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Timeframe for reporting adverse events

48 weeks

Adverse event reporting additional description

The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized 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.

Assessment type

Non-systematic

Dictionary name

MedDRA

Dictionary version

24.0

Reporting group title

PF-06651600 200 mg - 50 mg QD

Reporting group description

Subjects were randomized to receive ritlecitinib induction dose (200 mg QD) for 4 weeks followed by maintenance dosing of 50 mg QD for 20 weeks

Reporting group title

PF-06651600 100 mg - 50 mg QD

Reporting group description

Subjects were randomized to receive ritlecitinib induction dose (100 mg QD) for 4 weeks followed by maintenance dosing of 50 mg QD for 20 weeks

Reporting group title

PF-06651600 50 mg QD

Reporting group description

Subjects were randomized to receive ritlecitinib 50 mg QD for 24 weeks

Reporting group title

PF-06651600 30 mg QD

Reporting group description

Subjects were randomized to receive ritlecitinib 30 mg QD for 24 weeks

Reporting group title

PF-06651600 10 mg QD

Reporting group description

Subjects were randomized to receive ritlecitinib 10 mg QD for 24 weeks

Reporting group title

Placebo

Reporting group description

Subjects were randomized to receive placebo for 24 weeks

Reporting group title

EXT PF-06700841 60 mg - 30 mg QD

Reporting group description

After a 4-week drug holiday (with no investigational product), subjects received induction dose of PF-06700841 60 mg QD for 4 weeks followed by maintenance dosing of PF-06700841 30 mg QD for 16 weeks. This arm is open label.

Reporting group title

EXT PF-06651600 200 mg - 50 mg QD + nbUVB

Reporting group description

Induction dose of PF-06651600 200 mg QD plus standardized narrow band UVB (nbUVB) add-on therapy for 4 weeks followed by maintenance dosing of PF-06651600 50 mg QD plus standardized nbUVB add-on therapy for 20 weeks (only for participants who provide nbUVB consent). Subjects who had <10% improvement in percent change in VASI at Extension Week 12 from the baseline value at Dose Ranging Period Week 24 were discontinued from the treatment and entered Follow-up Period. This arm is open label.

Reporting group title

EXT PF-06651600 200 mg - 50 mg QD

Reporting group description

Induction dose of 200 mg QD of PF 06651600 for 4 weeks followed by maintenance dosing of 50 mg QD of PF 06651600 for 20 weeks. This arm is double blinded.

Reporting group title

EXT PF-06651600 50 mg QD

Reporting group description

50 mg QD of PF 06651600 for 24 weeks. This arm is double blinded.

Reporting group title

EXT PF-06651600 30 mg QD

Reporting group description

30 mg QD of PF 06651600 for 24 weeks. This arm is double blinded.

Serious adverse events	PF-06651600 200 mg - 50 mg QD	PF-06651600 100 mg - 50 mg QD	PF-06651600 50 mg QD	PF-06651600 30 mg QD	PF-06651600 10 mg QD	Placebo	EXT PF-06700841 60 mg - 30 mg QD	EXT PF-06651600 200 mg - 50 mg QD + nbUVB	EXT PF-06651600 200 mg - 50 mg QD	EXT PF-06651600 50 mg QD	EXT PF-06651600 30 mg QD
Total subjects affected by serious adverse events
subjects affected / exposed	0 / 65 (0.00%)	0 / 67 (0.00%)	1 / 67 (1.49%)	1 / 50 (2.00%)	1 / 49 (2.04%)	1 / 66 (1.52%)	1 / 55 (1.82%)	0 / 43 (0.00%)	1 / 187 (0.53%)	0 / 6 (0.00%)	0 / 2 (0.00%)
number of deaths (all causes)	0	0	0	0	0	0	0	0	0	0	0
number of deaths resulting from adverse events	0	0	0	0	0	0	0	0	0	0	0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Uterine leiomyoma
subjects affected / exposed	0 / 65 (0.00%)	0 / 67 (0.00%)	0 / 67 (0.00%)	0 / 50 (0.00%)	0 / 49 (0.00%)	0 / 66 (0.00%)	0 / 55 (0.00%)	0 / 43 (0.00%)	1 / 187 (0.53%)	0 / 6 (0.00%)	0 / 2 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	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	0 / 0	0 / 0	0 / 0	0 / 0
Nervous system disorders
Migraine
subjects affected / exposed	0 / 65 (0.00%)	0 / 67 (0.00%)	1 / 67 (1.49%)	0 / 50 (0.00%)	1 / 49 (2.04%)	0 / 66 (0.00%)	0 / 55 (0.00%)	0 / 43 (0.00%)	0 / 187 (0.00%)	0 / 6 (0.00%)	0 / 2 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 1	0 / 0	0 / 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	0 / 0	0 / 0	0 / 0	0 / 0
Gastrointestinal disorders
Oesophageal spasm
subjects affected / exposed	0 / 65 (0.00%)	0 / 67 (0.00%)	0 / 67 (0.00%)	1 / 50 (2.00%)	0 / 49 (0.00%)	0 / 66 (0.00%)	0 / 55 (0.00%)	0 / 43 (0.00%)	0 / 187 (0.00%)	0 / 6 (0.00%)	0 / 2 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 1	0 / 0	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	0 / 0	0 / 0	0 / 0	0 / 0
Renal and urinary disorders
Neurogenic bladder
subjects affected / exposed	0 / 65 (0.00%)	0 / 67 (0.00%)	0 / 67 (0.00%)	0 / 50 (0.00%)	0 / 49 (0.00%)	1 / 66 (1.52%)	0 / 55 (0.00%)	0 / 43 (0.00%)	0 / 187 (0.00%)	0 / 6 (0.00%)	0 / 2 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	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	0 / 0	0 / 0	0 / 0	0 / 0
Infections and infestations
Disseminated varicella zoster virus infection
subjects affected / exposed	0 / 65 (0.00%)	0 / 67 (0.00%)	0 / 67 (0.00%)	0 / 50 (0.00%)	0 / 49 (0.00%)	0 / 66 (0.00%)	1 / 55 (1.82%)	0 / 43 (0.00%)	0 / 187 (0.00%)	0 / 6 (0.00%)	0 / 2 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	1 / 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	0 / 0	0 / 0	0 / 0	0 / 0

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

Non-serious adverse events	PF-06651600 200 mg - 50 mg QD	PF-06651600 100 mg - 50 mg QD	PF-06651600 50 mg QD	PF-06651600 30 mg QD	PF-06651600 10 mg QD	Placebo	EXT PF-06700841 60 mg - 30 mg QD	EXT PF-06651600 200 mg - 50 mg QD + nbUVB	EXT PF-06651600 200 mg - 50 mg QD	EXT PF-06651600 50 mg QD	EXT PF-06651600 30 mg QD
Total subjects affected by non serious adverse events
subjects affected / exposed	31 / 65 (47.69%)	35 / 67 (52.24%)	32 / 67 (47.76%)	23 / 50 (46.00%)	22 / 49 (44.90%)	37 / 66 (56.06%)	17 / 55 (30.91%)	14 / 43 (32.56%)	46 / 187 (24.60%)	3 / 6 (50.00%)	2 / 2 (100.00%)
Vascular disorders
Hypertension
subjects affected / exposed	0 / 65 (0.00%)	0 / 67 (0.00%)	0 / 67 (0.00%)	0 / 50 (0.00%)	0 / 49 (0.00%)	0 / 66 (0.00%)	1 / 55 (1.82%)	0 / 43 (0.00%)	2 / 187 (1.07%)	1 / 6 (16.67%)	0 / 2 (0.00%)
occurrences all number	0	0	0	0	0	0	1	0	2	1	0
General disorders and administration site conditions
Fatigue
subjects affected / exposed	6 / 65 (9.23%)	1 / 67 (1.49%)	2 / 67 (2.99%)	0 / 50 (0.00%)	1 / 49 (2.04%)	1 / 66 (1.52%)	0 / 55 (0.00%)	0 / 43 (0.00%)	0 / 187 (0.00%)	0 / 6 (0.00%)	0 / 2 (0.00%)
occurrences all number	6	1	2	0	1	1	0	0	0	0	0
Influenza like illness
subjects affected / exposed	0 / 65 (0.00%)	0 / 67 (0.00%)	0 / 67 (0.00%)	0 / 50 (0.00%)	0 / 49 (0.00%)	0 / 66 (0.00%)	0 / 55 (0.00%)	0 / 43 (0.00%)	0 / 187 (0.00%)	1 / 6 (16.67%)	0 / 2 (0.00%)
occurrences all number	0	0	0	0	0	0	0	0	0	1	0
Respiratory, thoracic and mediastinal disorders
Cough
subjects affected / exposed	0 / 65 (0.00%)	0 / 67 (0.00%)	0 / 67 (0.00%)	0 / 50 (0.00%)	0 / 49 (0.00%)	0 / 66 (0.00%)	3 / 55 (5.45%)	2 / 43 (4.65%)	3 / 187 (1.60%)	0 / 6 (0.00%)	0 / 2 (0.00%)
occurrences all number	0	0	0	0	0	0	3	2	3	0	0
Epistaxis
subjects affected / exposed	0 / 65 (0.00%)	0 / 67 (0.00%)	0 / 67 (0.00%)	0 / 50 (0.00%)	0 / 49 (0.00%)	0 / 66 (0.00%)	0 / 55 (0.00%)	0 / 43 (0.00%)	0 / 187 (0.00%)	1 / 6 (16.67%)	0 / 2 (0.00%)
occurrences all number	0	0	0	0	0	0	0	0	0	1	0
Psychiatric disorders
Insomnia
subjects affected / exposed	1 / 65 (1.54%)	0 / 67 (0.00%)	1 / 67 (1.49%)	3 / 50 (6.00%)	0 / 49 (0.00%)	1 / 66 (1.52%)	0 / 55 (0.00%)	0 / 43 (0.00%)	0 / 187 (0.00%)	0 / 6 (0.00%)	0 / 2 (0.00%)
occurrences all number	1	0	1	3	0	1	0	0	0	0	0
Investigations
Blood creatine phosphokinase increased
subjects affected / exposed	0 / 65 (0.00%)	0 / 67 (0.00%)	0 / 67 (0.00%)	0 / 50 (0.00%)	0 / 49 (0.00%)	0 / 66 (0.00%)	3 / 55 (5.45%)	4 / 43 (9.30%)	3 / 187 (1.60%)	0 / 6 (0.00%)	0 / 2 (0.00%)
occurrences all number	0	0	0	0	0	0	3	5	3	0	0
Nervous system disorders
Headache
subjects affected / exposed	4 / 65 (6.15%)	7 / 67 (10.45%)	8 / 67 (11.94%)	1 / 50 (2.00%)	4 / 49 (8.16%)	8 / 66 (12.12%)	4 / 55 (7.27%)	3 / 43 (6.98%)	8 / 187 (4.28%)	0 / 6 (0.00%)	0 / 2 (0.00%)
occurrences all number	4	9	8	2	5	10	4	3	8	0	0
Blood and lymphatic system disorders
Anaemia
subjects affected / exposed	0 / 65 (0.00%)	0 / 67 (0.00%)	0 / 67 (0.00%)	0 / 50 (0.00%)	0 / 49 (0.00%)	0 / 66 (0.00%)	0 / 55 (0.00%)	0 / 43 (0.00%)	0 / 187 (0.00%)	1 / 6 (16.67%)	0 / 2 (0.00%)
occurrences all number	0	0	0	0	0	0	0	0	0	1	0
Ear and labyrinth disorders
Ear pain
subjects affected / exposed	0 / 65 (0.00%)	0 / 67 (0.00%)	0 / 67 (0.00%)	0 / 50 (0.00%)	0 / 49 (0.00%)	0 / 66 (0.00%)	0 / 55 (0.00%)	0 / 43 (0.00%)	0 / 187 (0.00%)	1 / 6 (16.67%)	0 / 2 (0.00%)
occurrences all number	0	0	0	0	0	0	0	0	0	1	0
Gastrointestinal disorders
Abdominal pain
subjects affected / exposed	0 / 65 (0.00%)	1 / 67 (1.49%)	2 / 67 (2.99%)	0 / 50 (0.00%)	1 / 49 (2.04%)	4 / 66 (6.06%)	0 / 55 (0.00%)	0 / 43 (0.00%)	0 / 187 (0.00%)	0 / 6 (0.00%)	0 / 2 (0.00%)
occurrences all number	0	1	3	0	1	4	0	0	0	0	0
Diarrhoea
subjects affected / exposed	0 / 65 (0.00%)	5 / 67 (7.46%)	2 / 67 (2.99%)	4 / 50 (8.00%)	1 / 49 (2.04%)	1 / 66 (1.52%)	0 / 55 (0.00%)	0 / 43 (0.00%)	0 / 187 (0.00%)	0 / 6 (0.00%)	0 / 2 (0.00%)
occurrences all number	0	7	2	5	1	2	0	0	0	0	0
Dyspepsia
subjects affected / exposed	1 / 65 (1.54%)	0 / 67 (0.00%)	0 / 67 (0.00%)	0 / 50 (0.00%)	3 / 49 (6.12%)	3 / 66 (4.55%)	0 / 55 (0.00%)	0 / 43 (0.00%)	0 / 187 (0.00%)	0 / 6 (0.00%)	0 / 2 (0.00%)
occurrences all number	1	0	0	0	4	3	0	0	0	0	0
Skin and subcutaneous tissue disorders
Acne
subjects affected / exposed	4 / 65 (6.15%)	1 / 67 (1.49%)	4 / 67 (5.97%)	1 / 50 (2.00%)	0 / 49 (0.00%)	0 / 66 (0.00%)	0 / 55 (0.00%)	0 / 43 (0.00%)	0 / 187 (0.00%)	0 / 6 (0.00%)	0 / 2 (0.00%)
occurrences all number	4	1	4	1	0	0	0	0	0	0	0
Dry skin
subjects affected / exposed	1 / 65 (1.54%)	4 / 67 (5.97%)	0 / 67 (0.00%)	2 / 50 (4.00%)	1 / 49 (2.04%)	2 / 66 (3.03%)	0 / 55 (0.00%)	0 / 43 (0.00%)	0 / 187 (0.00%)	0 / 6 (0.00%)	0 / 2 (0.00%)
occurrences all number	1	4	0	2	1	2	0	0	0	0	0
Pruritus
subjects affected / exposed	3 / 65 (4.62%)	2 / 67 (2.99%)	2 / 67 (2.99%)	1 / 50 (2.00%)	2 / 49 (4.08%)	5 / 66 (7.58%)	0 / 55 (0.00%)	3 / 43 (6.98%)	5 / 187 (2.67%)	0 / 6 (0.00%)	0 / 2 (0.00%)
occurrences all number	3	2	2	1	2	5	0	3	6	0	0
Urticaria
subjects affected / exposed	2 / 65 (3.08%)	3 / 67 (4.48%)	1 / 67 (1.49%)	0 / 50 (0.00%)	3 / 49 (6.12%)	0 / 66 (0.00%)	0 / 55 (0.00%)	0 / 43 (0.00%)	0 / 187 (0.00%)	0 / 6 (0.00%)	0 / 2 (0.00%)
occurrences all number	3	6	1	0	4	0	0	0	0	0	0
Photosensitivity reaction
subjects affected / exposed	0 / 65 (0.00%)	0 / 67 (0.00%)	0 / 67 (0.00%)	0 / 50 (0.00%)	0 / 49 (0.00%)	0 / 66 (0.00%)	0 / 55 (0.00%)	3 / 43 (6.98%)	0 / 187 (0.00%)	0 / 6 (0.00%)	0 / 2 (0.00%)
occurrences all number	0	0	0	0	0	0	0	4	0	0	0
Rash
subjects affected / exposed	0 / 65 (0.00%)	0 / 67 (0.00%)	0 / 67 (0.00%)	0 / 50 (0.00%)	0 / 49 (0.00%)	0 / 66 (0.00%)	0 / 55 (0.00%)	1 / 43 (2.33%)	1 / 187 (0.53%)	0 / 6 (0.00%)	1 / 2 (50.00%)
occurrences all number	0	0	0	0	0	0	0	1	3	0	1
Musculoskeletal and connective tissue disorders
Back pain
subjects affected / exposed	3 / 65 (4.62%)	3 / 67 (4.48%)	6 / 67 (8.96%)	0 / 50 (0.00%)	1 / 49 (2.04%)	3 / 66 (4.55%)	0 / 55 (0.00%)	0 / 43 (0.00%)	0 / 187 (0.00%)	0 / 6 (0.00%)	0 / 2 (0.00%)
occurrences all number	3	3	8	0	1	3	0	0	0	0	0
Spinal segmental dysfunction
subjects affected / exposed	0 / 65 (0.00%)	0 / 67 (0.00%)	0 / 67 (0.00%)	0 / 50 (0.00%)	0 / 49 (0.00%)	0 / 66 (0.00%)	0 / 55 (0.00%)	0 / 43 (0.00%)	0 / 187 (0.00%)	1 / 6 (16.67%)	0 / 2 (0.00%)
occurrences all number	0	0	0	0	0	0	0	0	0	1	0
Infections and infestations
Bronchitis
subjects affected / exposed	0 / 65 (0.00%)	1 / 67 (1.49%)	1 / 67 (1.49%)	3 / 50 (6.00%)	0 / 49 (0.00%)	0 / 66 (0.00%)	1 / 55 (1.82%)	0 / 43 (0.00%)	0 / 187 (0.00%)	0 / 6 (0.00%)	1 / 2 (50.00%)
occurrences all number	0	1	1	6	0	0	1	0	0	0	1
Folliculitis
subjects affected / exposed	0 / 65 (0.00%)	1 / 67 (1.49%)	1 / 67 (1.49%)	1 / 50 (2.00%)	0 / 49 (0.00%)	4 / 66 (6.06%)	0 / 55 (0.00%)	0 / 43 (0.00%)	0 / 187 (0.00%)	0 / 6 (0.00%)	0 / 2 (0.00%)
occurrences all number	0	2	1	1	0	4	0	0	0	0	0
Gastroenteritis
subjects affected / exposed	2 / 65 (3.08%)	0 / 67 (0.00%)	1 / 67 (1.49%)	3 / 50 (6.00%)	1 / 49 (2.04%)	1 / 66 (1.52%)	0 / 55 (0.00%)	0 / 43 (0.00%)	0 / 187 (0.00%)	0 / 6 (0.00%)	0 / 2 (0.00%)
occurrences all number	2	0	1	3	1	1	0	0	0	0	0
Nasopharyngitis
subjects affected / exposed	8 / 65 (12.31%)	10 / 67 (14.93%)	16 / 67 (23.88%)	5 / 50 (10.00%)	5 / 49 (10.20%)	14 / 66 (21.21%)	3 / 55 (5.45%)	0 / 43 (0.00%)	8 / 187 (4.28%)	0 / 6 (0.00%)	0 / 2 (0.00%)
occurrences all number	9	11	20	8	5	20	3	0	10	0	0
Upper respiratory tract infection
subjects affected / exposed	5 / 65 (7.69%)	10 / 67 (14.93%)	5 / 67 (7.46%)	8 / 50 (16.00%)	6 / 49 (12.24%)	8 / 66 (12.12%)	4 / 55 (7.27%)	2 / 43 (4.65%)	9 / 187 (4.81%)	0 / 6 (0.00%)	0 / 2 (0.00%)
occurrences all number	6	11	8	8	7	8	4	2	10	0	0
Urinary tract infection
subjects affected / exposed	4 / 65 (6.15%)	4 / 67 (5.97%)	2 / 67 (2.99%)	2 / 50 (4.00%)	3 / 49 (6.12%)	3 / 66 (4.55%)	1 / 55 (1.82%)	3 / 43 (6.98%)	12 / 187 (6.42%)	1 / 6 (16.67%)	1 / 2 (50.00%)
occurrences all number	5	6	2	2	3	3	2	4	16	1	1
COVID-19
subjects affected / exposed	0 / 65 (0.00%)	0 / 67 (0.00%)	0 / 67 (0.00%)	0 / 50 (0.00%)	0 / 49 (0.00%)	0 / 66 (0.00%)	0 / 55 (0.00%)	0 / 43 (0.00%)	2 / 187 (1.07%)	0 / 6 (0.00%)	1 / 2 (50.00%)
occurrences all number	0	0	0	0	0	0	0	0	2	0	1
Influenza
subjects affected / exposed	0 / 65 (0.00%)	0 / 67 (0.00%)	0 / 67 (0.00%)	0 / 50 (0.00%)	0 / 49 (0.00%)	0 / 66 (0.00%)	3 / 55 (5.45%)	0 / 43 (0.00%)	2 / 187 (1.07%)	0 / 6 (0.00%)	0 / 2 (0.00%)
occurrences all number	0	0	0	0	0	0	3	0	2	0	0
Onychomycosis
subjects affected / exposed	0 / 65 (0.00%)	0 / 67 (0.00%)	0 / 67 (0.00%)	0 / 50 (0.00%)	0 / 49 (0.00%)	0 / 66 (0.00%)	0 / 55 (0.00%)	0 / 43 (0.00%)	0 / 187 (0.00%)	0 / 6 (0.00%)	1 / 2 (50.00%)
occurrences all number	0	0	0	0	0	0	0	0	0	0	1
Metabolism and nutrition disorders
Hypercholesterolaemia
subjects affected / exposed	0 / 65 (0.00%)	0 / 67 (0.00%)	0 / 67 (0.00%)	0 / 50 (0.00%)	0 / 49 (0.00%)	0 / 66 (0.00%)	0 / 55 (0.00%)	0 / 43 (0.00%)	0 / 187 (0.00%)	1 / 6 (16.67%)	0 / 2 (0.00%)
occurrences all number	0	0	0	0	0	0	0	0	0	1	0

Top of page

Were there any global substantial amendments to the protocol? Yes

04 Mar 2019

Updated Schedule of Activities; updated exploratory endpoints; added new non-clinical safety information to Section 1.2.3.1 Nonclinical Safety; emerging data was added demonstrating a drug interaction (DDI) with oral contraceptives containing ethinyl estradiol (EE) in Section 1.2.3.2 Clinical Experience; Section 4.4.1 Contraception Requirements were modified; updated Section 1.2.4 Study Rationale, Section 4 Subject Eligibility Criteria, Section 4.4.1 Contraception Requirements, and Section 4.4 Lifestyle Requirements; updated description of static investigator global assessment (sIGA) in Section 7.4 Clinical Efficacy Assessments; safety adjudication committees were added to Section 9 Data Analysis/Statistical Methods; updated Appendix 10 Country Specific Requirements; minor administrative changes and sentence revisions made throughout the document.

14 Jun 2019

updated Schedule of Activities; updated Section 1.2.4.1 PF-06651600 Dose Rationale and Section 4.2 Exclusion Criteria; added definitions of adverse reaction (AR), serious adverse reaction (SAR), and suspected unexpected serious adverse reactions (SUSAR) to Section 8 Adverse Event Reporting; updated Appendix 2 Prohibited Concomitant Medications; minor administrative changes and sentence revisions made throughout the document.

02 Jul 2019

Added risk-benefit assessment to Section 1.2.5 Summary of Benefits and Risks; updated Section 4.2 Exclusion Criteria.

04 Nov 2019

Added IND number for PF-06700841 to cover page; updated Schedule of Activities; added B7931019 study data and safety information from phase 2 studies to Section 1.2.3.2 Clinical Experience; updated exploratory endpoints; details of Section 7.11.1 Skin Biopsies have been modified; updated Section 7 Assessments and Appendix 2; minor administrative changes and sentence revisions made throughout the document.

26 Jun 2020

Updated Schedule of Activities; updated exploratory endpoints; clarification that the site assessment F-VASI will be performed by the investigator(s) was added to Section 7.4.3 Facial Vitiligo Area Scoring Index Site Assessment; the sample size rationale was updated in Section 9.1; the analysis of the primary endpoint and secondary endpoints were updated in Section 9.2; the detail of the analysis was removed from Section 9.5 Safety Analysis during the Dose Ranging Period; added Alternative Measures During Public Emergencies to Appendix 11; minor administrative changes and sentence revisions made throughout the document.

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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End point values

PF-06651600 200 mg - 50 mg QD

PF-06651600 100 mg - 50 mg QD

PF-06651600 50 mg QD

PF-06651600 30 mg QD

PF-06651600 10 mg QD

Number of subjects analysed

Units: Subjects

Bilirubin > 1.5 x upper limit of normal (ULN)

AST > 2.5 x ULN

ALT > 2.5 x ULN