PF-06651600 for the Treatment of Alopecia Areata

Phase 2

Completed

Conditions: Alopecia Areata

Interventions: Drug: PF-06651600 Induction Dose
Drug: PF-06651600 Maintenance Dose #1
Drug: PF-06651600 Maintenance Dose #2
Drug: PF-06651600 Maintenance Dose #3
Drug: Placebo

Registration Number: NCT03732807

Lead Sponsor: Pfizer

Brief Summary: This is a global Phase 2b/3 study to evaluate the safety and effectiveness of an investigational study drug (called PF-06651600) in adults and adolescents (12 years and older) who have 50% or greater scalp hair loss. The study is placebo-controlled, meaning that some patients entering the study will not receive active study drug but will receive tablets with no active ingredients (a placebo). This is a dose-ranging study, investigating 5 different dosing regimens. It will be double-blinded, meaning that the sponsor, the study doctors, the staff, and the patients will not know whether a patient is on active study drug (or the dose) or placebo.

Detailed Description: Not available

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 718

Inclusion Criteria

Clinical diagnosis of alopecia areata with no other cause of hair loss
≥50% hair loss of the scalp, including alopecia totalis and alopecia universalis, without evidence of terminal hair regrowth within 6 months
Current episode of hair loss ≤10 years

Exclusion Criteria

Other types of alopecia or other diseases that can cause hair loss
Other scalp diseases that could interfere with assessment of hair loss/regrowth
Subjects with shaved heads must not enter the study until hair has grown back & is considered stable by the investigator
Any previous use of any Janus kinase (JAK) inhibitor

Study & Design

Study Type: INTERVENTIONAL

Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Sequence B	PF-06651600 Induction Dose	Induction dose given QD for 4 weeks followed by maintenance dose #2 given QD for 44 weeks
Sequence G	PF-06651600 Maintenance Dose #1	Placebo given QD for 24 weeks followed by maintenance dose #1 given QD for 24 weeks
Sequence G	Placebo	Placebo given QD for 24 weeks followed by maintenance dose #1 given QD for 24 weeks
Sequence D	PF-06651600 Maintenance Dose #2	Maintenance dose #2 given QD for 48 weeks
Sequence E	PF-06651600 Maintenance Dose #3	Maintenance dose #3 given QD for 48 weeks
Sequence B	PF-06651600 Maintenance Dose #2	Induction dose given QD for 4 weeks followed by maintenance dose #2 given QD for 44 weeks
Sequence C	PF-06651600 Maintenance Dose #1	Maintenance dose #1 given QD for 48 weeks
Sequence F	PF-06651600 Maintenance Dose #1	Placebo given QD for 24 weeks followed by induction dose given QD for 4 weeks then maintenance dose #1 given QD for 20 weeks
Sequence A	PF-06651600 Maintenance Dose #1	Induction dose given once daily (QD) for 4 weeks followed by maintenance dose #1 given QD for 44 weeks
Sequence F	Placebo	Placebo given QD for 24 weeks followed by induction dose given QD for 4 weeks then maintenance dose #1 given QD for 20 weeks
Sequence A	PF-06651600 Induction Dose	Induction dose given once daily (QD) for 4 weeks followed by maintenance dose #1 given QD for 44 weeks
Sequence F	PF-06651600 Induction Dose	Placebo given QD for 24 weeks followed by induction dose given QD for 4 weeks then maintenance dose #1 given QD for 20 weeks

Primary Outcome Measures

Name	Time	Method
Percentage of Participants With an Absolute Severity of Alopecia Tool (SALT) Score of Less Than or Equal to 20 at Week 24	Week 24	SALT is a quantitative assessment of AA severity based on the scalp hair loss. The SALT score can vary from 0 (normal) to 100 (severe), with higher scores representing increased severity of disease. In this outcome measure, percentage of participants with SALT score less than or equal to (\<=) 20 at week 24 were reported.

Secondary Outcome Measures

Name	Time	Method
Percentage of Participants With an Absolute SALT Score of Less Than or Equal to 10 at Week 24: Analysis 4	Week 24	SALT is a quantitative assessment of AA severity based on the scalp hair loss. The SALT score can vary from 0 (normal) to 100 (severe), with higher scores representing increased severity of disease. In this outcome measure, percentage of participants with SALT score \<= 10 at week 24 were reported.
Percentage of Participants With an Absolute SALT Score of Less Than or Equal to 10 at Week 24: Analysis 1	Week 24	SALT is a quantitative assessment of AA severity based on the scalp hair loss. The SALT score can vary from 0 (normal) to 100 (severe), with higher scores representing increased severity of disease. In this outcome measure, percentage of participants with SALT score \<= 10 at week 24 were reported.
Exposure Response of PF-06651600 on Regrowth of Lost Hair Based on Absolute SALT Score of Less Than or Equal to 20 at Week 24: Maximum Effect (Emax) Model	Week 24	The exposure response of Ritlecitinib (PF-06651600) on the regrowth of scalp hair was characterized using a Bayesian three-parameter hyperbolic Emax model for the SALT score \<=20 at Week 24 with an additional term for effect of loading dose. In Emax exposure-response model the response function was the log odds of the percentage of participants with response based on SALT \<=20 at Week 24, which was fit on the logistic scale and then back-transformed to percentage. The effect of loading dose is included as fixed factor in the model. The variable that represents loading dose has values of 1 for groups 200/50 mg once daily and 200/30 mg once daily and of 0 for the remaining groups. SALT is a quantitative assessment of AA severity based on the scalp hair loss. The SALT score can vary from 0 (normal) to 100 (severe), with higher scores representing increased severity of disease.
Percentage of Participants With at Least a 2 Grade Improvement From Baseline or a Score of 3 in Eyebrow Assessment (EBA) Score (Among Participants Without Normal EBA at Baseline) at Week 4, 8, 12, 18, 24, 28, 34, 40, and 48	Week 4, 8, 12, 18, 24, 28, 34, 40, and 48	EBA is a numeric rating scale developed to characterize eyebrow hair loss. The numeric rating scale ranges from 0 (none) to 3 (normal), where, 0= no eyebrow, 1=minimal eyebrow, 2=moderate eyebrow and 3= normal eyebrow, where higher scores represent less hair loss of eyebrows.
Percentage of Participants With at Least a 2 Grade Improvement From Baseline or a Score of 3 in Eyelash Assessment (ELA) Score (Among Participants Without Normal ELA at Baseline) at Week 4, 8, 12, 18, 24, 28, 34, 40, and 48	Week 4, 8, 12, 18, 24, 28, 34, 40, and 48	ELA is a numeric rating scale developed to characterize eyelash hair loss. The numeric rating scale ranges from 0 (none) to 3 (normal), where, 0=no eyelash, 1=minimal eyelash, 2=moderate eyelash and 3=normal eyelash, where higher scores represent less hair loss of eyelash.
Change From Baseline in Alopecia Areata Patient Priority Outcomes (AAPPO) Domain Scores at Week 4, 8, 12, 18, and 24: Emotional Symptoms and Activity Limitations	Baseline (Day 1), Week 4, 8, 12, 18, and 24	AAPPO scale is 11-item self-administered questionnaire that measured hair loss, emotional symptoms, and activity limitations over past week. Items 1-4 assessed current hair loss, eyebrow loss, eyelash loss and body hair loss and were analyzed separately on scale of 0-4, with 0 ='no hair loss' and 4='complete hair loss'. Items 5-8 assessed emotional symptoms. Response choices on these items were scored from 0 ='never' to 4='always'. Items 9-11 assessed activity limitations. Response choices on these items were scored from 0='not at all' to 4='completely'. Change from baseline in AAPPO emotional symptoms sub score were calculated as mean of items 5-8 and ranged from 0(never) to 4(always), where higher scores indicated more emotional symptoms. Change from baseline in AAPPO activity limitations sub score was calculated as mean of items 9-11 and ranged from 0(not at all) to 4(completely), where higher scores indicated more activity limitations. Baseline was defined as pre-dose on Day 1.
Percentage of Participants With Patient Global Impression of Change (PGI-C) Score of Moderately Improved or Greatly Improved at Week 24	Week 24	PGI-C is a self-administered questionnaire to evaluate the improvement or worsening of participant's AA as compared to the start of the study. PGI-C was assessed on a 7-point Likert scale ranged from 1 (greatly improved) to 7 (greatly worsened). Categories were defined based on the PGI-C scores as follows: 1=greatly improved, 2=moderately improved, 3=slightly improved, 4=not changed, 5=slightly worsened, 6=moderately worsened and 7=greatly worsened.
Exposure Response of PF-06651600 on Regrowth of Lost Hair Based on Absolute SALT Score of Less Than or Equal to 10 at Week 24: Maximum Effect (Emax) Model	Week 24	The exposure response of Ritlecitinib (PF-06651600) on the regrowth of scalp hair was characterized using a Bayesian three-parameter hyperbolic Emax model for the SALT score \<=10 at Week 24 with an additional term for effect of loading dose. In Emax exposure-response model the response function was the log odds of the percentage of participants with response based on SALT \<=10 at Week 24, which was fit on the logistic scale and then back-transformed to percentage. The effect of loading dose is included as fixed factor in the model. The variable that represents loading dose has values of 1 for groups 200/50 mg once daily and 200/30 mg once daily and of 0 for the remaining groups. SALT is a quantitative assessment of AA severity based on the scalp hair loss. The SALT score can vary from 0 (normal) to 100 (severe), with higher scores representing increased severity of disease.
Percentage of Participants With an Absolute SALT Score of Less Than or Equal to 10 at Week 4, 8, 12, 18, 28, 34, 40, and 48	Week 4, 8, 12, 18, 28, 34, 40, and 48	SALT is a quantitative assessment of AA severity based on the scalp hair loss. The SALT score can vary from 0 (normal) to 100 (severe), with higher scores representing increased severity of disease. In this outcome measure, percentage of participants with SALT score \<=10 were reported.
Percentage of Participants With at Least 75% Improvement in SALT Score (SALT75) From Baseline at Week 4, 8, 12, 18, 24, 28, 34, 40, and 48	Week 4, 8, 12, 18, 24, 28, 34, 40, and 48	SALT is a quantitative assessment of AA severity based on the scalp hair loss. The SALT score can vary from 0 (normal) to 100 (severe), with higher scores representing increased severity of disease. A SALT 75 response was a 75% or greater reduction from baseline in SALT score.
Change From Baseline in SALT Score at Week 4, 8, 12, 18, and 24	Baseline (Day 1), Week 4, 8, 12, 18, and 24	SALT is a quantitative assessment of AA severity based on the scalp hair loss. The SALT score can vary from 0 (normal) to 100 (severe), with higher scores representing increased severity of disease. Baseline was defined as pre-dose on Day 1.
Percentage of Participants With Patient Global Impression of Change (PGI-C) Score of Moderately Improved or Greatly Improved at Week 4, 8, 12, 18, 24, 34, 40, and 48	Week 4, 8, 12, 18, 24, 34, 40, and 48	PGI-C is a self-administered questionnaire to evaluate the improvement or worsening of participant's AA as compared to the start of the study. PGI-C was assessed on a 7-point Likert scale ranged from 1 (greatly improved) to 7 (greatly worsened). Categories were defined based on the PGI-C scores as follows: 1=greatly improved, 2=moderately improved, 3=slightly improved, 4=not changed, 5=slightly worsened, 6=moderately worsened and 7=greatly worsened.
Percentage of Participants With an Absolute SALT Score of Less Than or Equal to 20 at Week 4, 8, 12, 18, 28, 34, 40, and 48	Week 4, 8, 12, 18, 28, 34, 40, and 48	SALT is a quantitative assessment of AA severity based on the scalp hair loss. The SALT score can vary from 0 (normal) to 100 (severe), with higher scores representing increased severity of disease.
Change From Baseline in SALT Score at Week 28, 34, 40, and 48	Baseline (Day 1), Week 28, 34, 40, and 48	SALT is a quantitative assessment of AA severity based on the scalp hair loss. The SALT score can vary from 0 (normal) to 100 (severe), with higher scores representing increased severity of disease. Baseline was defined as pre-dose on Day 1.
Change From Baseline in Alopecia Areata Patient Priority Outcomes (AAPPO) Domain Scores at Week 34, 40, and 48: Emotional Symptoms and Activity Limitations	Baseline (Day 1), Week 34, 40, and 48	AAPPO scale is 11-item self-administered questionnaire that measured hair loss, emotional symptoms, and activity limitations over past week. Items 1-4 assessed current hair loss, eyebrow loss, eyelash loss and body hair loss and were analyzed separately on scale of 0-4, with 0 ='no hair loss' and 4='complete hair loss'. Items 5-8 assessed emotional symptoms. Response choices on these items were scored from 0 ='never' to 4='always'. Items 9-11 assessed activity limitations. Response choices on these items were scored from 0='not at all' to 4='completely'. Change from baseline in AAPPO emotional symptoms sub score were calculated as mean of items 5-8 and ranged from 0(never) to 4(always), where higher scores indicated more emotional symptoms. Change from baseline in AAPPO activity limitations sub score was calculated as mean of items 9-11 and ranged from 0(not at all) to 4(completely), where higher scores indicated more activity limitations. Baseline was defined as pre-dose on Day 1.
Percentage of Participants With Improvement From Baseline on Alopecia Areata Patient Priority Outcomes (AAPPO) Items 1-4 at Week 4, 8, 12, 18, 24, 34, 40, and 48	Week 4, 8, 12, 18, 24, 34, 40, and 48	AAPPO scale is a 11-item self-administered questionnaire that measured hair loss, emotional symptoms, and activity limitations over the past week. Items 1-4 were to assess the current hair loss, eyebrow loss, eyelash loss and body hair loss and were analyzed separately on a scale of 0-4, with 0 ='no hair loss' and 4='complete hair loss', where higher scores indicated more hair loss.

Trial Locations

Locations (153): The University of Alabama at Birmingham Hospital Outreach Lab
🇺🇸
Birmingham, Alabama, United States
The University of Alabama at Birmingham, Department of Dermatology
🇺🇸
Birmingham, Alabama, United States
Mosaic Dermatology
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Beverly Hills, California, United States
University of California, Irvine, Department of Dermatology, Dermatology Clinical Research Center
🇺🇸
Irvine, California, United States
Dermatology Specialists, Inc.
🇺🇸
Murrieta, California, United States
University of California, San Francisco
🇺🇸
San Francisco, California, United States
Kaiser Permanente Clinical Trials Unit
🇺🇸
San Francisco, California, United States
Southern California Dermatology, Inc.
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Santa Ana, California, United States
University of Colorado Anschutz Medical Campus
🇺🇸
Aurora, Colorado, United States
University of Colorado Hospital Clinical and Translational Research Center
🇺🇸
Aurora, Colorado, United States
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The University of Alabama at Birmingham Hospital Outreach Lab
🇺🇸Birmingham, Alabama, United States