Study To Evaluate The Efficacy And Safety Profile Of PF-06651600 And PF-06700841 In Subjects With Alopecia Areata

A PHASE 2A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTICENTER STUDY TO EVALUATE THE EFFICACY AND SAFETY PROFILE OF PF-06651600 AND PF-06700841 IN SUBJECTS WITH MODERATE TO SEVERE ALOPECIA AREATA WITH A SINGLE-BLIND EXTENSION PERIOD AND A CROSS-OVER OPEN LABEL EXTENSION PERIOD

This is a Phase 2a, randomized, double blind, parallel group, multicenter study with an extension period. The study will have a maximum duration of approximately 113 weeks. This includes an up to 5 weeks Screening Period, a 24 week Treatment Period, a 4 week Drug Holiday (#1), an up to 12 month Single Blind (investigator open, sponsor open and subject blind) Extension Period, a 4 week drug holiday (#2), a 6 month Cross Over Open Label Extension Period and a 4 week Follow up Period.

Study Overview

• Status: Completed
• Conditions: Alopecia Areata
• Intervention / Treatment: Drug: Placebo; Drug: PF-06651600; Drug: PF-06700841

• Study Type: Interventional
• Enrollment (Actual): 142
• Phase: Phase 2

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Hurstville, New South Wales, Australia, 2220
      • Hearing Life
    • Kogarah, New South Wales, Australia, 2217
      • St George Dermatology and Skin Cancer Centre
    • Kogarah, New South Wales, Australia, 2217
      • Dr. Glen and Partners Medical Imaging
    • Kogarah, New South Wales, Australia, 2217
      • St. George Hearing and Balance Clinic
  • Queensland
    • Benowa, Queensland, Australia, 4217
      • The Skin Centre
    • Woolloongabba, Queensland, Australia, 4102
      • Veracity Clinical Research
  • Victoria
    • Carlton, Victoria, Australia, 3053
      • Skin & Cancer Foundation Inc.
    • East Melbourne, Victoria, Australia, 3002
      • Sinclair Dermatology
    • Richmond, Victoria, Australia, 3121
      • Bridge Road Imag ing
    • Richmond, Victoria, Australia, 3121
      • Richmond Audiology
• Canada
  • Manitoba
    • Winnipeg, Manitoba, Canada, R3M 3Z4
      • Wiseman Dermatology Research Inc.
  • Ontario
    • Markham, Ontario, Canada, L3P 1X2
      • Lynderm Research Inc.
    • Oakville, Ontario, Canada, L6J 7W5
      • Research by ICLS
    • Peterborough, Ontario, Canada, K9J 5K2
      • Skin Centre For Dermatology
    • Richmond Hill, Ontario, Canada, L4C 9M7
      • York Dermatology Center
    • Richmond Hill, Ontario, Canada, L4B 1A5
      • The Centre for Dermatology
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35233
      • University of Alabama at Birmingham, The Kirklin Clinic
    • Birmingham, Alabama, United States, 35233
      • University of Alabama at Birmingham, Dermatology at the Whitaker Clinic
  • California
    • Santa Ana, California, United States, 92701
      • Southern California Dermatology, Inc.
    • Santa Monica, California, United States, 90404
      • Clinical Science Institute
    • Santa Monica, California, United States, 90404
      • Office of F. Monte Purcelli
    • Santa Monica, California, United States, 90404
      • Tower Saint John's Imaging
  • Colorado
    • Aurora, Colorado, United States, 80045
      • University of Colorado Hospital, Anschutz Cancer Pavilion
    • Aurora, Colorado, United States, 80045
      • University of Colorado Hospital Clinical and Translational Research Center, Inpatient Unit
    • Aurora, Colorado, United States, 80045
      • University of Colorado Hospital Clinical and Translational Research Center, Outpatient Clinic
  • Connecticut
    • New Haven, Connecticut, United States, 06519
      • Yale New Haven Hospital
    • New Haven, Connecticut, United States, 06510
      • Yale School of Medicine
    • New Haven, Connecticut, United States, 06519
      • Church Street Research Unit
    • New Haven, Connecticut, United States, 06511
      • Investigational Drug Services
    • New Haven, Connecticut, United States, 06519
      • Yale Hearing and Balance Center
  • Florida
    • Orange Park, Florida, United States, 32073
      • Park Avenue Dermatology
    • Orange Park, Florida, United States, 32073
      • Park Avenue Dermatology Administrative Annex
    • Tampa, Florida, United States, 33609
      • Olympian Clinical Research
    • Tampa, Florida, United States, 33624
      • Forward Clinical Trials, Inc
    • Tampa, Florida, United States, 33609
      • Rose Radiology
    • Tampa, Florida, United States, 33609
      • Edward B. Kampsen, MD
  • Georgia
    • Newnan, Georgia, United States, 30263
      • MedaPhase Inc.
  • Illinois
    • Skokie, Illinois, United States, 60077
      • NorthShore University HealthSystem Dermatology Clinical Trials Unit
  • Indiana
    • Indianapolis, Indiana, United States, 46256
      • Dawes Fretzin Clinical Research Group, LLC
    • Indianapolis, Indiana, United States, 46256
      • Dawes Fretzin Dermatology Group, LLC
  • Massachusetts
    • Boston, Massachusetts, United States, 02111
      • Tufts Medical Center
    • Boston, Massachusetts, United States, 02114
      • Massachusetts General Hospital Clinical Unit for Research Trials in Skin (CURTIS)
  • New York
    • New York, New York, United States, 10029
      • Icahn School of Medicine at Mount Sinai
    • New York, New York, United States, 10021
      • Weill Cornell Medicine
    • New York, New York, United States, 10065
      • Rockefeller University Hospital
    • New York, New York, United States, 10029
      • Mount Sinai Department of Otolaryngology
    • Rochester, New York, United States, 14642
      • University of Rochester Medical Center
    • Rochester, New York, United States, 14642
      • Investigational Drug Service
    • Rochester, New York, United States, 14642
      • University of Rochester Audiology
    • Rochester, New York, United States, 14642
      • University of Rochester Radiology
  • Oklahoma
    • Tulsa, Oklahoma, United States, 74136
      • Vital Prospects Clinical Research Institute, P.C.
  • South Dakota
    • Rapid City, South Dakota, United States, 57702
      • Health Concepts
  • Utah
    • Murray, Utah, United States, 84107
      • University of Utah MidValley Dermatology
    • Salt Lake City, Utah, United States, 84132
      • University of Utah Medical Center
  • Virginia
    • Norfolk, Virginia, United States, 23502
      • Virginia Clinical Research, Inc.

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 75 years (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Male or female subjects between 18 75 years of age, inclusive, at time of informed consent.
• Must have moderate to severe alopecia areata:

Exclusion Criteria:

• History of human immunodeficiency virus (HIV) or positive HIV serology at screening,
• Infected with hepatitis B or hepatitis C viruses.
• Have evidence of active or latent or inadequately treated infection with Mycobacterium tuberculosis (TB)
• Have received any of the following treatment regiments specified in the timeframes outlined below:

Within 6 months of first dose of study drug: Any cell depleting agents Within 12 weeks of first dose of study drug: Any studies with JAK inhibitors; Other biologics Within 8 weeks of first dose of study drug: Participation in other studies involving investigational drug(s) Within 6 weeks of first dose of study drug: Have been vaccinated with live or attenuated live vaccine.

Within 4 weeks of first dose of study drug: Use of oral immune suppressants; Phototherapy (NB UVB) or broad band phototherapy; Regular use (more than 2 visits per week) of a tanning booth/parlor.

Within 2 week of first dose of study drug: Topical treatments that could affect AA; Herbal medications with unknown properties or known beneficial effects for AA.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: RANDOMIZED
• Masking: TRIPLE

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: Cohort 1; PF-06651600	Drug: PF-06651600; 200 mg QD during induction and 50 mg QD during Maintenance
EXPERIMENTAL: Cohort 2; PF-06700841	Drug: PF-06700841; 60 mg QD during induction and 30 mg QD during maintenance
PLACEBO_COMPARATOR: Cohort placebo; placebo	Drug: Placebo; Placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change From Baseline in Severity of Alopecia Tool (SALT) Score at Week 24	SALT is a quantitative assessment of alopecia areata (AA) severity based on the scalp hair loss. Score range: 0-100%. Higher score indicates more severe disease. Change from baseline is defined as the baseline value minus the value at a specific visit. Positive change from baseline signifies an improvement. Baseline is defined as the last measurement prior to first dosing (Day 1).	Baseline, Week24
Number of Participants With Treatment-emergent Adverse Events (All-causality and Treatment-related) - Single-Blind Extension (SBE) Period	An AE (non-serious and serious) was any untoward medical occurrence in a participant who received study treatment without regard to possibility of causal relationship. Any such events with initial onset or increasing in severity after the first dose of study treatment were counted as treatment-emergent Adverse Event (TEAE). Treatment-related TEAE were determined by investigators. Arms end with "withdrawal Segment" and "retreatment segment" described the same population while in different treatment segment .The reason why count on PF-06700841 differ by 1 participant is that 1 responder directly entered the retreatment segment and skipped the withdrawal segment.	Week 28 up to Week 52
Number of Participants With Treatment-emergent Adverse Events (All-causality and Treatment-related) - Cross-Over Extension (COE) Period	An AE (non-serious and serious) was any untoward medical occurrence in a participant who received study treatment without regard to possibility of causal relationship. Any such events with initial onset or increasing in severity after the first dose of study treatment were counted as treatment-emergent Adverse Event (TEAE). Treatment-related TEAE were determined by investigators.	COE day 1 up to end of study
Number of Participants With Laboratory Abnormalities During SBE Period	Following laboratory parameters were assessed against pre-defined abnormality criteria: hematology(Hemoglobin, Hematocrit, RBC count, Reticulocyte count, Platelet count, WBC count with differential, Total neutrophils, Eosinophils, Monocytes, Basophils, Lymphocytes); serum chemistry (BUN and Creatinine, Cystatin C, Creatine Phosphokinase, Glucose , Na+, K+, Cl ,Ca++, Total CO2, AST, ALT, Total Indirect & Direct Bilirubin, Alkaline phosphatase, Uric acid, Albumin,Total protein, Fasting lipid Profile Panel; urinalysis(pH, Glucose, Protein, Nitrites, Leukocyte esterase, Microscopy culture);Other(HIV, HBsAg, HBcAb, HepB reflex (HbsAB), if applicable, HCVAb, Serum pregnancy test, Urine pregnancy test, FSH, QFT G or other IGRA, or PPD, EBV, CMV, HSV1, HSV2, VZV, Skin swab for herpetiform rash, Skin swab for potential drug related rash).Retest/discontinuation criteria are defined in Protocol Appendix 6.1 and 6.2 respectively.	Week 28 up to Week 52 for non-responders and responders in the withdrawal segment, AT day 1 up to AT Week 24 for retreatment segment (AT=active treatment)
Numbers of Participants With Specific Clinical Laboratory Abnormalities During COE Period	Following laboratory parameters were assessed against pre-defined abnormality criteria: hematology(Hemoglobin, Hematocrit, RBC count, Reticulocyte count, Platelet count, WBC count with differential, Total neutrophils, Eosinophils, Monocytes, Basophils, Lymphocytes); serum chemistry (BUN and Creatinine, Cystatin C, Creatine Phosphokinase, Glucose , Na+, K+, Cl ,Ca++, Total CO2, AST, ALT, Total Indirect & Direct Bilirubin, Alkaline phosphatase, Uric acid, Albumin,Total protein, Fasting lipid Profile Panel; urinalysis(pH, Glucose, Protein, Nitrites, Leukocyte esterase, Microscopy culture);Other(HIV, HBsAg, HBcAb, HepB reflex (HbsAB), if applicable, HCVAb, Serum pregnancy test, Urine pregnancy test, FSH, QFT G or other IGRA, or PPD, EBV, CMV, HSV1, HSV2, VZV, Skin swab for herpetiform rash, Skin swab for potential drug related rash).Retest/discontinuation criteria are defined in Protocol Appendix 6.1 and 6.2 respectively.	COE day 1 up to end of study

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change From Baseline in Severity of Alopecia Tool (SALT) Score at Week 24 -AT/AU Participants	SALT is a quantitative assessment of alopecia areata (AA) severity based on the scalp hair loss. The SALT score can vary from 0 to 100%, with higher scores representing increasing severity of disease. Change from baseline is defined as the baseline value minus the value at a specific visit. Positive change from baseline implies an improvement. Alopecia totalis (AT): derived as SALT score = 100% at baseline only. Alopecia universalis (AU): derived as SALT score = 100% and both eyelash and eyebrow assessments were "none" at baseline.	Baseline, Week 24
Percentage of Participants Achieving SALT 30 at Week 24	SALT is a quantitative assessment of alopecia areata (AA) severity based on the scalp hair loss. A SALT 30 response is a 30% or greater reduction from baseline in SALT score. The SALT score can vary from 0 to 100%, with higher scores representing increasing severity of disease. The analysis was done using FAS (full analysis set) based on Non-responder imputation (ie. set missing values to be non-responsive) data. The 90% CI was calculated using Chan and Zhang method.	Baseline, Week 24
Change From Baseline in Severity of Alopecia Tool (SALT) Across Time (Treatment Period)	SALT is a quantitative assessment of alopecia areata (AA) severity based on the scalp hair loss. The SALT score can vary from 0 to 100% with higher score indicates more severe disease. Baseline is defined as the last measurement prior to first dosing (Day 1). Change from baseline is defined as the baseline value minus the value at a specific visit. Positive change from baseline implies an improvement. Least Square Mean and 90% Confidence Interval of Arms (PF-06651600 and PF-06700841) are the Least Square Mean and 90% Confidence Interval for difference from placebo respectively.	Baseline, Weeks 2,4,6,8,12,16,20,24
Percent Change From Baseline in Severity of Alopecia Tool (SALT) Across Time (Treatment Period)	SALT is a quantitative assessment of alopecia areata (AA) severity based on the scalp hair loss. The SALT score can vary from 0 to 100% with higher score indicates more severe disease. Baseline is defined as the last measurement prior to first dosing (Day 1). Percent change from baseline is defined as SALT baseline value minus SALT value at a specific visit divided by baseline and multiplying by 100. Positive change from baseline implies an improvement. Least Square Mean and 90% Confidence Interval of Arms (PF-06651600 and PF-06700841) are the Least Square Mean and 90% Confidence Interval for difference from placebo respectively.	Baseline, Weeks 2,4,6,8,12,16,20,24
Percentage of Participants Achieving SALT 30 Across Time (Treatment Period)	SALT is a quantitative assessment of alopecia areata (AA) severity based on the scalp hair loss. A SALT 30 response is a 30% or greater reduction from baseline in SALT score. The SALT score can vary from 0 to 100%, with higher scores representing increasing severity of disease. The analysis was done using FAS (full analysis set) based on Non-responder imputation (ie. set missing values to be non-responsive) data. The percentage(Number) and 90% confidence interval (CI) of Arms(PF-06651600 and PF-06700841) in this outcome measurement is the percentage and 90% CI for difference from placebo. The 90% CI was calculated using Chan and Zhang method.	Baseline, Weeks 2,4,6,8,12,16,20,24
Percentage of Participants Achieving SALT 50 Across Time (Treatment Period)	SALT is a quantitative assessment of alopecia areata (AA) severity based on the scalp hair loss. A SALT 50 response is a 50% or greater reduction from baseline in SALT score. The SALT score can vary from 0 to 100%, with higher scores representing increasing severity of disease. The analysis was done using FAS (full analysis set) based on Non-responder imputation (ie. set missing values to be non-responsive) data. The percentage(Number) and 90% confidence interval (CI) of Arms(PF-06651600 and PF-06700841) in this outcome measurement is the percentage and 90% CI for difference from placebo. The 90% CI was calculated using Chan and Zhang method.	Baseline, Weeks 2,4,6,8,12,16,20,24
Percentage of Participants Achieving SALT 75 Across Time (Treatment Period)	SALT is a quantitative assessment of alopecia areata (AA) severity based on the scalp hair loss. A SALT 75 response is a 75% or greater reduction from baseline in SALT score. The SALT score can vary from 0 to 100%, with higher scores representing increasing severity of disease. The analysis was done using FAS (full analysis set) based on Non-responder imputation (ie. set missing values to be non-responsive) data. The percentage(Number) and 90% confidence interval (CI) of Arms(PF-06651600 and PF-06700841) in this outcome measurement is the percentage and 90% CI for difference from placebo. The 90% CI was calculated using Chan and Zhang method.	Baseline, Weeks 2,4,6,8,12,16,20,24
Percentage of Participants Achieving SALT 90 Across Time (Treatment Period)	SALT is a quantitative assessment of alopecia areata (AA) severity based on the scalp hair loss. A SALT 90 response is a 90% or greater reduction from baseline in SALT score. The SALT score can vary from 0 to 100%, with higher scores representing increasing severity of disease. The analysis was done using FAS (full analysis set) based on Non-responder imputation (ie. set missing values to be non-responsive) data. The percentage(Number) and 90% confidence interval (CI) of Arms(PF-06651600 and PF-06700841) in this outcome measurement is the percentage and 90% CI for difference from placebo. The 90% CI was calculated using Chan and Zhang method.	Baseline, Weeks 2,4,6,8,12,16,20,24
Percentage of Participants Achieving SALT 100 Across Time (Treatment Period)	SALT is a quantitative assessment of alopecia areata (AA) severity based on the scalp hair loss. A SALT 100 response is a 100% or greater reduction from baseline in SALT score. The SALT score can vary from 0 to 100%, with higher scores representing increasing severity of disease. The analysis was done using FAS (full analysis set) based on Non-responder imputation (ie. set missing values to be non-responsive) data. The percentage(Number) and 90% confidence interval (CI) of Arms(PF-06651600 and PF-06700841) in this outcome measurement is the percentage and 90% CI for difference from placebo. The 90% CI was calculated using Chan and Zhang method.	Baseline, Weeks 2,4,6,8,12,16,20,24
Number of Participants With the IGA Score Change (Treatment Period)	The clinical evaluator of alopecia areata (AA) will perform an assessment of the overall improvement of AA and assign an Investigator Global Assessment (IGA) score(ranging from 0 to 5) with higher score representing higher regrowth rate. Baseline is defined as the last measurement prior to first dosing (Day 1).	baseline, Week 2,4,6,8,12,16,20,24
Number of Participants With Treatment-emergent Adverse Events (All-causality and Treatment-related) - Treatment Period	An AE (non-serious and serious) was any untoward medical occurrence in a participant who received study treatment without regard to possibility of causal relationship. Any such events with initial onset or increasing in severity after the first dose of study treatment were counted as treatment-emergent Adverse Event (TEAE). Treatment-related TEAE were determined by investigators.	baseline up to Week 24
Number of Participants With Laboratory Abnormalities During Treatment Period	Following laboratory parameters were assessed against pre-defined abnormality criteria: hematology(Hemoglobin, Hematocrit, RBC count, Reticulocyte count, Platelet count, WBC count with differential, Total neutrophils, Eosinophils, Monocytes, Basophils, Lymphocytes); serum chemistry (BUN and Creatinine, Cystatin C, Creatine Phosphokinase, Glucose , Na+, K+, Cl ,Ca++, Total CO2, AST, ALT, Total Indirect & Direct Bilirubin, Alkaline phosphatase, Uric acid, Albumin,Total protein, Fasting lipid Profile Panel; urinalysis(pH, Glucose, Protein, Nitrites, Leukocyte esterase, Microscopy culture);Other(HIV, HBsAg, HBcAb, HepB reflex (HbsAB), if applicable, HCVAb, Serum pregnancy test, Urine pregnancy test, FSH, QFT G or other IGRA, or PPD, EBV, CMV, HSV1, HSV2, VZV, Skin swab for herpetiform rash, Skin swab for potential drug related rash).Retest/discontinuation criteria are defined in Protocol Appendix 6.1 and 6.2 respectively.	Baseline up to Week 24
Time to Achieve the Retreatment Criteria During the Withdrawal/Retreatment Part of the Extension Period Among Subjects Who Achieved Primary Endpoint at Week 24 (SBE Period)	Time to re-treatment (weeks) = (date of re-treatment criteria met - date at Week 24 +1)/7. The calendar time to retreatment was calculated. Baseline is defined as Week 24 measurement. The duration in Drug Holiday #1 (ranging from 2-6 weeks) is counted in the Kaplan-Meier analysis. One subject directly entered the re-treatment period and hence is censored at baseline in the Kaplan-Meier analysis.	Week 24 up to Week 52
Change From Baseline in SALT Across Time (SBE Period)	SALT is a quantitative assessment of alopecia areata (AA) severity based on the scalp hair loss. The SALT score can vary from 0 to 100% with higher score indicates more severe disease. Baseline is defined as the last measurement prior to first dosing (Day 1). Change from baseline is defined as the baseline value minus the value at a specific visit. Positive change from baseline implies an improvement. Least Square Mean and 90% confidence interval in this outcome measurement is the LSM and 90%CI for difference from initial 24-week treatment period placebo respectively.	Weeks 30, 32, 34, 36, 40, 44, 48, 52 for non-responders, and AT Weeks 2, 4, 6, 8, 12, 16, 20, 24 for retreated responders.(AT=active treatment)
Percentage of Participants Achieving SALT 30 Across Time (SBE Period)	SALT is a quantitative assessment of alopecia areata (AA) severity based on the scalp hair loss. A SALT 30 response is a 30% or greater reduction from baseline in SALT score. The SALT score can vary from 0 to 100%, with higher scores representing increasing severity of disease. The analysis was done using FAS (full analysis set) based on Non-responder imputation (ie. set missing values to be non-responsive) data. The percentage(Number) and 90% confidence interval (CI) in this outcome measurement is the percentage and 90% CI for difference from initial 24-week treatment period placebo. The 90% CI was calculated using Chan and Zhang method.	Weeks 30, 32, 34, 36, 40, 44, 48, 52 for non-responders, and AT Weeks 2, 4, 6, 8, 12, 16, 20, 24 for retreated responders.(AT=active treatment)
Percentage of Participants Achieving SALT 50 Across Time (SBE Period)	SALT is a quantitative assessment of alopecia areata (AA) severity based on the scalp hair loss. A SALT 50 response is a 50% or greater reduction from baseline in SALT score. The SALT score can vary from 0 to 100%, with higher scores representing increasing severity of disease. The analysis was done using FAS (full analysis set) based on Non-responder imputation (ie. set missing values to be non-responsive) data. The percentage(Number) and 90% confidence interval (CI) in this outcome measurement is the percentage and 90% CI for difference from initial 24-week treatment period placebo. The 90% CI was calculated using Chan and Zhang method.	Weeks 30, 32, 34, 36, 40, 44, 48, 52 for non-responders, and AT Weeks 2, 4, 6, 8, 12, 16, 20, 24 for retreated responders.(AT=active treatment)
Percentage of Participants Achieving SALT 75 Across Time (SBE Period)	SALT is a quantitative assessment of alopecia areata (AA) severity based on the scalp hair loss. A SALT 75 response is a 75% or greater reduction from baseline in SALT score. The SALT score can vary from 0 to 100%, with higher scores representing increasing severity of disease. The analysis was done using FAS (full analysis set) based on Non-responder imputation (ie. set missing values to be non-responsive) data. The percentage(Number) and 90% confidence interval (CI) in this outcome measurement is the percentage and 90% CI for difference from initial 24-week treatment period placebo. The 90% CI was calculated using Chan and Zhang method.	Weeks 30, 32, 34, 36, 40, 44, 48, 52 for non-responders, and AT Weeks 2, 4, 6, 8, 12, 16, 20, 24 for retreated responders.(AT=active treatment)
Percentage of Participants Achieving SALT 90 Across Time (SBE Period)	SALT is a quantitative assessment of alopecia areata (AA) severity based on the scalp hair loss. A SALT 90 response is a 90% or greater reduction from baseline in SALT score. The SALT score can vary from 0 to 100%, with higher scores representing increasing severity of disease. The analysis was done using FAS (full analysis set) based on Non-responder imputation (ie. set missing values to be non-responsive) data. The percentage(Number) and 90% confidence interval (CI) in this outcome measurement is the percentage and 90% CI for difference from initial 24-week treatment period placebo. The 90% CI was calculated using Chan and Zhang method.	Weeks 30, 32, 34, 36, 40, 44, 48, 52 for non-responders, and AT Weeks 2, 4, 6, 8, 12, 16, 20, 24 for retreated responders.(AT=active treatment)
Percentage of Participants Achieving SALT 100 Across Time (SBE Period)	SALT is a quantitative assessment of alopecia areata (AA) severity based on the scalp hair loss. A SALT 100 response is a 100% or greater reduction from baseline in SALT score. The SALT score can vary from 0 to 100%, with higher scores representing increasing severity of disease. The analysis was done using FAS (full analysis set) based on Non-responder imputation (ie. set missing values to be non-responsive) data. The percentage(Number) and 90% confidence interval (CI) in this outcome measurement is the percentage and 90% CI for difference from initial 24-week treatment period placebo. The 90% CI was calculated using Chan and Zhang method.	Weeks 30, 32, 34, 36, 40, 44, 48, 52 for non-responders, and AT Weeks 2, 4, 6, 8, 12, 16, 20, 24 for retreated responders.(AT=active treatment)

Collaborators and Investigators

• Sponsor: Pfizer

Publications and helpful links

General Publications

• Hughes JH, Qiu R, Banfield C, Dowty ME, Nicholas T. Population Pharmacokinetics of Oral Brepocitinib in Healthy Volunteers and Patients. Clin Pharmacol Drug Dev. 2022 Dec;11(12):1447-1456. doi: 10.1002/cpdd.1163. Epub 2022 Aug 31.
• Fensome A, Ambler CM, Arnold E, Banker ME, Brown MF, Chrencik J, Clark JD, Dowty ME, Efremov IV, Flick A, Gerstenberger BS, Gopalsamy A, Hayward MM, Hegen M, Hollingshead BD, Jussif J, Knafels JD, Limburg DC, Lin D, Lin TH, Pierce BS, Saiah E, Sharma R, Symanowicz PT, Telliez JB, Trujillo JI, Vajdos FF, Vincent F, Wan ZK, Xing L, Yang X, Yang X, Zhang L. Dual Inhibition of TYK2 and JAK1 for the Treatment of Autoimmune Diseases: Discovery of (( S)-2,2-Difluorocyclopropyl)((1 R,5 S)-3-(2-((1-methyl-1 H-pyrazol-4-yl)amino)pyrimidin-4-yl)-3,8-diazabicyclo[3.2.1]octan-8-yl)methanone (PF-06700841). J Med Chem. 2018 Oct 11;61(19):8597-8612. doi: 10.1021/acs.jmedchem.8b00917. Epub 2018 Aug 16.
• Peeva E, Guttman-Yassky E, Banerjee A, Sinclair R, Cox LA, Zhu L, Zhu H, Vincent M, King B. Maintenance, withdrawal, and re-treatment with ritlecitinib and brepocitinib in patients with alopecia areata in a single-blind extension of a phase 2a randomized clinical trial. J Am Acad Dermatol. 2022 Aug;87(2):390-393. doi: 10.1016/j.jaad.2021.12.008. Epub 2021 Dec 13. No abstract available.
• Guttman-Yassky E, Pavel AB, Diaz A, Zhang N, Del Duca E, Estrada Y, King B, Banerjee A, Banfield C, Cox LA, Dowty ME, Page K, Vincent MS, Zhang W, Zhu L, Peeva E. Ritlecitinib and brepocitinib demonstrate significant improvement in scalp alopecia areata biomarkers. J Allergy Clin Immunol. 2022 Apr;149(4):1318-1328. doi: 10.1016/j.jaci.2021.10.036. Epub 2021 Dec 1.

Helpful Links

• To obtain contact information for a study center near you, click here.

Study record dates

Study Major Dates

• Study Start (ACTUAL): December 15, 2016
• Primary Completion (ACTUAL): May 15, 2019
• Study Completion (ACTUAL): May 15, 2019

Study Registration Dates

• First Submitted: November 23, 2016
• First Submitted That Met QC Criteria: November 23, 2016
• First Posted (ESTIMATE): November 29, 2016

Study Record Updates

• Last Update Posted (ACTUAL): May 26, 2020
• Last Update Submitted That Met QC Criteria: May 6, 2020
• Last Verified: May 1, 2020

More Information

Terms related to this study

• Keywords: safety; placebo; efficacy; JAK; janus kinase; severe; moderate; randomized; double-blind; Phase 2; alopecia areata
• Additional Relevant MeSH Terms: Skin Diseases; Pathological Conditions, Anatomical; Hypotrichosis; Hair Diseases; Alopecia; Alopecia Areata; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Protein Kinase Inhibitors; PF-06700841
• Other Study ID Numbers: B7931005; 2016-004048-13 (EUDRACT_NUMBER); Allegro (Alias Study Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No