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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT03715829
Registration number
NCT03715829
Ethics application status
Date submitted
19/10/2018
Date registered
23/10/2018
Date last updated
25/03/2022
Titles & IDs
Public title
A Phase 2b Study To Evaluate The Efficacy And Safety Profile Of PF-06651600 And PF-06700841 In Active Non-segmental Vitiligo Subjects
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Scientific title
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
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Secondary ID [1]
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2018-001271-20
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Secondary ID [2]
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B7981019
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Active Non-segmental Vitiligo
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Condition category
Condition code
Skin
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Dermatological conditions
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - PF-06651600
Treatment: Drugs - PF-06651600
Treatment: Drugs - PF-06651600
Treatment: Drugs - PF-06651600
Treatment: Drugs - PF-06651600
Treatment: Drugs - placebo
Treatment: Drugs - PF06700841
Treatment: Devices - narrow-band UVB phototherapy
Experimental: Cohort 1 - Induction dose 1 given once a day(QD) for 4 weeks followed by maintenance dose A given QD for 20 weeks
Experimental: Cohort 2 - Induction dose 2 given QD for 4 weeks followed by maintenance dose A given QD for 20 weeks
Experimental: Cohort 3 - Maintenance dose A given QD for 24 weeks
Experimental: Cohort 4 - Maintenance dose B given QD for 24 weeks
Experimental: Cohort 5 - Maintenance dose C given QD for 24 weeks
Placebo Comparator: Cohort 6 - Placebo given QD for 24 weeks
Experimental: Extension Cohort 1 - 4 week drug holiday (no drug given) followed by PF-06700841 oral tablet QD for 20 weeks
Experimental: Extension Cohort 2 - Induction dose 1 given QD for 4 weeks followed by maintenance dose A given QD for 20 weeks in conjunction with narrow band UVB phototherapy
Experimental: Extension Cohort 3 - Induction dose 1 given QD for 4 weeks followed by maintenance dose A given QD for 20 weeks
Experimental: Extension Cohort 4 - Maintenance dose A given QD for 24 weeks
Experimental: Extension Cohort 5 - Maintenance dose B given QD for 24 weeks
No Intervention: Extension Cohort 6 - Observation period for 24 weeks
Treatment: Drugs: PF-06651600
Induction dose 1. Oral tablet taken QD
Treatment: Drugs: PF-06651600
Induction dose 2. Oral tablet taken QD
Treatment: Drugs: PF-06651600
Maintenance dose A. Oral tablet taken QD
Treatment: Drugs: PF-06651600
Maintenance Dose B. Oral tablet taken QD
Treatment: Drugs: PF-06651600
Maintenance Dose C. Oral tablet taken QD
Treatment: Drugs: placebo
placebo
Treatment: Drugs: PF06700841
Oral tablet taken QD
Treatment: Devices: narrow-band UVB phototherapy
Phototherapy will be combined with PF-06651600
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Intervention code [1]
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Treatment: Drugs
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Intervention code [2]
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Treatment: Devices
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Percent Change From Baseline in Central Read Facial-Vitiligo Area Scoring Index (F-VASI) at Week 24 - Dose Ranging (DR) Period
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Assessment method [1]
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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)=? [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 central read F-VASI = ((post-baseline central read F-VASI - baseline central read F-VASI)/baseline central read F-VASI)×100. A negative percent change from baseline in central read F-VASI signified an improvement.
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Timepoint [1]
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Baseline, Week 24 (Baseline was defined as the last measurement prior to Study Day 18)
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Primary outcome [2]
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Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) up to Week 24 - DR Period
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Assessment method [2]
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Adverse Event (AE) was defined as any untoward medical occurrence in a study participant administered a product or medical device; the event did not necessarily need to have a causal relationship with the treatment or usage. 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. SAE was defined as 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.
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Timepoint [2]
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24 weeks
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Primary outcome [3]
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Number of Participants With the TEAEs of Anaemia, Neutropenia, Thrombocytopenia and Lymphopenia - DR Period
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Assessment method [3]
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An AE was any untoward medical occurrence in a study participant 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.
Baseline was defined as the last measurement prior to first dosing (Day 1).
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Timepoint [3]
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Baseline up to Week 24
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Primary outcome [4]
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Number of Participants With Clinically Meaningful Changes From Baseline in Lipid Profile up to Week 24 - DR Period
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Assessment method [4]
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Participants 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.
Baseline was defined as the last measurement prior to first dosing (Day 1).
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Timepoint [4]
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Baseline up to Week 24
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Primary outcome [5]
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Number of Participants With Liver Function Test Values Meeting the Protocol-Specified Discontinuation Criteria - DR Period
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Assessment method [5]
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Liver function tests included tests of alanine aminotransferase (ALT), aspartate aminotransferase (AST) and total bilirubin.
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Timepoint [5]
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24 weeks
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Primary outcome [6]
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Number of Participants With TEAEs and SAEs - Extension (Ext) Period
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Assessment method [6]
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AE was defined as any untoward medical occurrence in a study participant administered a product or medical device; the event did not necessarily need to have a causal relationship with the treatment or usage. 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. SAE was defined as any untoward medical occurrence at any dose that resulted in death; was life threatening (immediate risk of death); requires 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.
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Timepoint [6]
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24 weeks
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Primary outcome [7]
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Number of Participants With the TEAEs of Anaemia, Neutropenia, Thrombocytopenia and Lymphopenia - Ext Period
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Assessment method [7]
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An AE was any untoward medical occurrence in a study participant 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.
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Timepoint [7]
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24 weeks
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Primary outcome [8]
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Number of Participants With Clinically Meaningful Changes From Baseline in Lipid Profile - Ext Period
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Assessment method [8]
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Participants 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.
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Timepoint [8]
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24 Weeks
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Primary outcome [9]
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Number of Participants With Liver Function Test Values Meeting the Protocol-Specified Discontinuation Criteria - Ext Period
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Assessment method [9]
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Liver function tests included tests of alanine aminotransferase (ALT), aspartate aminotransferase (AST) and total bilirubin.
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Timepoint [9]
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24 weeks
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Secondary outcome [1]
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Percentage of Participants Achieving Central F-VASI75 at Week 24 - DR Period
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Assessment method [1]
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This outcome measure was the percentage of participants 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. Baseline was defined as the last measurement prior to study Day 18.
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Timepoint [1]
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Week 24
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Secondary outcome [2]
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Percentage of Participants Achieving T-VASI50 at Week 24 - DR Period
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Assessment method [2]
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Total body VASI (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= ?[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 (excluding hands), trunk, lower extremities (excluding feet), and feet. The extent of depigmentation was expressed by percentages: 0, 10%, 25%, 50%, 75%, 90% or 100%. The data below was the percentage of participants 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. Baseline was defined as the last measurement prior to first dosing (Day 1).
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Timepoint [2]
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Week 24
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Secondary outcome [3]
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Percent Change From Baseline in T-VASI at Designated Time Points - DR Period
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Assessment method [3]
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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 = ? [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 (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. Baseline was defined as the last measurement prior to first dosing (Day 1).
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Timepoint [3]
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Baseline, Weeks 4, 8, 12, 16, 20 and 24
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Secondary outcome [4]
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Percent Change From Baseline in Central Read F-VASI at Designated Time Points - DR Period
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Assessment method [4]
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The 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 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)=? [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.
Percent change from baseline in central read F-VASI = ((post-baseline central read F-VASI - baseline central read F-VASI)/baseline central read F-VASI)×100. Negative percent change from baseline in F-VASI signified an improvement. Baseline was defined as the last measurement prior to Study Day 18.
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Timepoint [4]
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0
Baseline, Weeks 4, 8, 16 and 24
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Secondary outcome [5]
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Percent Change From Baseline in Local F-VASI at Designated Time Points - DR Period
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Assessment method [5]
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The site assessment of the F-VASI was calculated using a formula that included contribution from face (possible range, 0.00 4.00): 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 participant'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 following percentages: 0, 10%, 25%, 50%, 75%, 90%, or 100%.
Percent change from baseline in Local F-VASI = ((post baseline Local F-VASI - baseline Local F-VASI)/baseline Local F-VASI)×100. Negative percent change from baseline in F-VASI signified an improvement. Baseline was defined as the last measurement prior to first dosing (Day 1).
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Timepoint [5]
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Baseline, Weeks 4, 8, 12, 16, 20 and 24
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Secondary outcome [6]
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Percent Change From Baseline in SA-VES at Designated Time Points - DR Period
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Assessment method [6]
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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. Baseline was defined as the last measurement prior to first dosing (Day 1).
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Timepoint [6]
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Baseline, Weeks 4, 16 and 24
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Secondary outcome [7]
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Absolute Change From Baseline in T-VASI at Designated Time Points - DR Period
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Assessment method [7]
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0
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 = ? [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 (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. Baseline was defined as the last measurement prior to first dosing (Day 1).
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Timepoint [7]
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0
Baseline, Weeks 4, 8, 12, 16, 20 and 24
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Secondary outcome [8]
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Percentage of Participants Achieving T-VASI50 at Designated Time Points - DR Period
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Assessment method [8]
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0
T-VASI was calculated by a formula that included contribution from 6 body regions (possible range, 0-100): T-VASI = ? [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 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%. The outcome measure was the percentage of participants achieving at least 50% improvement from baseline in T-VASI (T-VASI50). 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. Baseline was defined as the last measurement prior to first dosing (Day 1).
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Timepoint [8]
0
0
Baseline, Weeks 4, 8, 12, 16, 20 and 24
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Secondary outcome [9]
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Percentage of Participants Achieving T-VASI75 at Designated Time Points - DR Period
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Assessment method [9]
0
0
T-VASI was calculated by a formula that included contribution from 6 body regions (possible range, 0-100): T-VASI = ? [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 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%. The outcome measure was the percentage of participants achieving at least 75% improvement from baseline in T-VASI (T-VASI75). 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. Baseline was defined as the last measurement prior to first dosing (Day 1).
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Timepoint [9]
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0
Baseline, Weeks 4, 8, 12, 16, 20 and 24
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Secondary outcome [10]
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Percentage of Participants Achieving T-VASI90 at Designated Time Points - DR Period
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Assessment method [10]
0
0
T-VASI was calculated by a formula that included contribution from 6 body regions (possible range, 0-100): T-VASI = ? [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 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%. The outcome measure was the percentage of participants achieving at least 90% improvement from baseline in T-VASI (T-VASI90). 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. Baseline was defined as the last measurement prior to first dosing (Day 1).
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Timepoint [10]
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0
Baseline, Weeks 4, 8, 12, 16, 20 and 24
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Secondary outcome [11]
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Percentage of Participants Achieving T-VASI100 at Designated Time Points - DR Period
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Assessment method [11]
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0
T-VASI was calculated using a formula that included contribution from 6 body regions (possible range, 0-100): T-VASI = ? [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 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 outcome measure was the percentage of participants achieving 100% improvement from baseline in T-VASI (T-VASI100). 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. Baseline was defined as the last measurement prior to first dosing (Day 1).
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Timepoint [11]
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0
Baseline, Weeks 4, 8, 12, 16, 20 and 24
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Secondary outcome [12]
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Percentage of Participants Achieving Central Read F-VASI50 at Designated Time Points - DR Period
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Assessment method [12]
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0
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) with a modified method: F-VASI (central read)=? [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. Percent change from baseline in F-VASI = ((post-baseline F-VASI - baseline F-VASI)/baseline F-VASI)×100. This outcome measure was the percentage of participants 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. Baseline was defined as the last measurement prior to Study Day 18.
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Timepoint [12]
0
0
Baseline, Weeks 4, 8, 16 and 24
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Secondary outcome [13]
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0
Percentage of Participants Achieving Central Read F-VASI75 at Designated Time Points - DR Period
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Assessment method [13]
0
0
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) with a modified method: F-VASI (central read)=? [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. Percent change from baseline in F-VASI = ((post-baseline F-VASI - baseline F-VASI)/baseline F-VASI)×100. This outcome measure was the percentage of participants 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. Baseline was defined as the last measurement prior to Study Day 18.
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Timepoint [13]
0
0
Baseline, Weeks 4, 8, 16 and 24
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Secondary outcome [14]
0
0
Percentage of Participants Achieving Central Read F-VASI90 at Designated Time Points - DR Period
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Assessment method [14]
0
0
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) with a modified method: F-VASI (central read)=? [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. Percent change from baseline in F-VASI = ((post-baseline F-VASI - baseline F-VASI)/baseline F-VASI)×100. This outcome measure was the percentage of participants 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. Baseline was defined as the last measurement prior to Study Day 18.
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Timepoint [14]
0
0
Baseline, Weeks 4, 8, 16 and 24
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Secondary outcome [15]
0
0
Percentage of Participants Achieving Central Read F-VASI100 at Designated Time Points - DR Period
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Assessment method [15]
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0
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) with a modified method: F-VASI (central read)=? [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. Percent change from baseline in F-VASI = ((post-baseline F-VASI - baseline F-VASI)/baseline F-VASI)×100. This outcome measure was the percentage of participants 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. Baseline was defined as the last measurement prior to Study Day 18.
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Timepoint [15]
0
0
Baseline, Weeks 4, 8, 16 and 24
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Secondary outcome [16]
0
0
Percentage of Participants Achieving Local F-VASI50 at Designated Time Points - DR Period
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Assessment method [16]
0
0
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. Scalp, neck, eyebrows, eyelashes, and vermilion were excluded from this calculation. The volar surface of 1 digit (the participant'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 following percentages: 0, 10%, 25%, 50%, 75%, 90%, or 100%. Percent change from baseline in Local F-VASI = ((post baseline Local F-VASI - baseline Local F-VASI)/baseline Local F-VASI)×100. This outcome measure was the percentage of participants 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. Baseline was defined as the last measurement prior to first dosing (Day 1).
Query!
Timepoint [16]
0
0
Baseline, Weeks 4, 8, 12, 16, 20 and 24
Query!
Secondary outcome [17]
0
0
Percentage of Participants Achieving Local F-VASI75 at Designated Time Points - DR Period
Query!
Assessment method [17]
0
0
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. Scalp, neck, eyebrows, eyelashes, and vermilion were excluded from this calculation. The volar surface of 1 digit (the participant'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 following percentages: 0, 10%, 25%, 50%, 75%, 90%, or 100%. Percent change from baseline in Local F-VASI = ((post baseline Local F-VASI - baseline Local F-VASI)/baseline Local F-VASI)×100. This outcome measure was the percentage of participants 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. Baseline was defined as the last measurement prior to first dosing (Day 1).
Query!
Timepoint [17]
0
0
Baseline, Weeks 4, 8, 12, 16, 20 and 24
Query!
Secondary outcome [18]
0
0
Percentage of Participants Achieving Local F-VASI90 at Designated Time Points - DR Period
Query!
Assessment method [18]
0
0
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. Scalp, neck, eyebrows, eyelashes, and vermilion were excluded from this calculation. The volar surface of 1 digit (the participant'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 following percentages: 0, 10%, 25%, 50%, 75%, 90%, or 100%. Percent change from baseline in Local F-VASI = ((post baseline Local F-VASI - baseline Local F-VASI)/baseline Local F-VASI)×100. This outcome measure was the percentage of participants 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. Baseline was defined as the last measurement prior to first dosing (Day 1).
Query!
Timepoint [18]
0
0
Baseline, Weeks 4, 8, 12, 16, 20 and 24
Query!
Secondary outcome [19]
0
0
Percentage of Participants Achieving Local F-VASI100 at Designated Time Points - DR Period
Query!
Assessment method [19]
0
0
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. Scalp, neck, eyebrows, eyelashes, and vermilion were excluded from this calculation. The volar surface of 1 digit (the participant'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 following percentages: 0, 10%, 25%, 50%, 75%, 90%, or 100%. Percent change from baseline in Local F-VASI = ((post baseline Local F-VASI - baseline Local F-VASI)/baseline Local F-VASI)×100. This outcome measure was the percentage of participants achieving 100% improvement in site assessment F-VASI from baseline (F-VASI100). Negative percent change from baseline in F-VASI signified an improvement. Baseline was defined as the last measurement prior to first dosing (Day 1).
Query!
Timepoint [19]
0
0
Baseline, Weeks 4, 8, 12, 16, 20 and 24
Query!
Secondary outcome [20]
0
0
Change From Baseline in Total VitiQoL Score at Designated Time Points - DR Period
Query!
Assessment method [20]
0
0
The Vitiligo-Specific Quality of Life (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 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 to 90, with higher scores representing greater burden. The 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. Baseline was defined as the last measurement prior to first dosing (Day 1).
Query!
Timepoint [20]
0
0
Baseline, Weeks 4, 16 and 24
Query!
Secondary outcome [21]
0
0
Change From Baseline in VitiQoL Participation Limitation Domain Score at Designated Time Points - DR Period
Query!
Assessment method [21]
0
0
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 to 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 and ranged from 0 to 42.
The change from baseline in VitiQoL Participation Limitation Domain Score was analyzed using the MMRM analysis. Baseline was defined as the last measurement prior to first dosing (Day 1).
Query!
Timepoint [21]
0
0
Baseline, Weeks 4, 16 and 24
Query!
Secondary outcome [22]
0
0
Change From Baseline in VitiQoL Stigma Domain Score at Designated Time Points - DR Period
Query!
Assessment method [22]
0
0
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 to 90, with higher scores representing greater burden. The VitiQoL Stigma domain score was the sum of items 1, 2, 5, 7 and 15, and ranged from 0 to 30.
The change from baseline in VitiQoL Stigma Domain Score was analyzed using the MMRM analysis. Baseline was defined as the last measurement prior to first dosing (Day 1).
Query!
Timepoint [22]
0
0
Baseline, Weeks 4, 16 and 24
Query!
Secondary outcome [23]
0
0
Change From Baseline in VitiQoL Behaviors Domain Score at Designated Time Points - DR Period
Query!
Assessment method [23]
0
0
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 to 90, with higher scores representing greater burden. The VitiQoL Behaviors domain score was the sum of items 8, 12 and 13, and ranged from 0 to 18.
The change from baseline in VitiQoL Behaviors Domain Score was analyzed using the MMRM analysis. Baseline was defined as the last measurement prior to first dosing (Day 1).
Query!
Timepoint [23]
0
0
Baseline, Weeks 4, 16 and 24
Query!
Secondary outcome [24]
0
0
Percentage of Participants Achieving sIGA 0 or 1 and at Least a 2-Point Improvement at Week 24 - DR Period
Query!
Assessment method [24]
0
0
The percentage 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.
Query!
Timepoint [24]
0
0
Week 24
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Eligibility
Key inclusion criteria
- Male or female subjects between 18-65 years of age, inclusive, at time of informed
consent.
- Must have moderate to severe active non-segmental vitiligo.
Query!
Minimum age
18
Years
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Query!
Maximum age
65
Years
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Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
Key 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)
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Query!
Query!
Query!
Intervention assignment
Parallel
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
26/11/2018
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Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
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Actual
5/02/2021
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Sample size
Target
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Accrual to date
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Final
366
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Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
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Recruitment hospital [1]
0
0
The Skin Hospital - Darlinghurst
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Recruitment hospital [2]
0
0
Premier Specialists Pty Ltd - Kogarah
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Recruitment hospital [3]
0
0
Veracity Clinical Research Pty Ltd - Woolloongabba
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Recruitment hospital [4]
0
0
Skin Health Institute - Carlton
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Recruitment hospital [5]
0
0
Sinclair Dermatology - East Melbourne
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Recruitment hospital [6]
0
0
The Royal Melbourne Hospital - Parkville
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Recruitment postcode(s) [1]
0
0
2010 - Darlinghurst
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Recruitment postcode(s) [2]
0
0
2217 - Kogarah
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Recruitment postcode(s) [3]
0
0
4102 - Woolloongabba
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Recruitment postcode(s) [4]
0
0
3053 - Carlton
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Recruitment postcode(s) [5]
0
0
3002 - East Melbourne
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Recruitment postcode(s) [6]
0
0
3050 - Parkville
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Recruitment postcode(s) [7]
0
0
3052 - Parkville
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Recruitment outside Australia
Country [1]
0
0
United States of America
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State/province [1]
0
0
California
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Country [2]
0
0
United States of America
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State/province [2]
0
0
Connecticut
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Country [3]
0
0
United States of America
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State/province [3]
0
0
Florida
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Country [4]
0
0
United States of America
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State/province [4]
0
0
Illinois
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Country [5]
0
0
United States of America
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State/province [5]
0
0
Maryland
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Country [6]
0
0
United States of America
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State/province [6]
0
0
Massachusetts
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Country [7]
0
0
United States of America
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State/province [7]
0
0
Michigan
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Country [8]
0
0
United States of America
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State/province [8]
0
0
Minnesota
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Country [9]
0
0
United States of America
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State/province [9]
0
0
New Jersey
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Country [10]
0
0
United States of America
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State/province [10]
0
0
New York
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Country [11]
0
0
United States of America
Query!
State/province [11]
0
0
North Carolina
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Country [12]
0
0
United States of America
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State/province [12]
0
0
Ohio
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Country [13]
0
0
United States of America
Query!
State/province [13]
0
0
Oklahoma
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Country [14]
0
0
United States of America
Query!
State/province [14]
0
0
Texas
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Country [15]
0
0
United States of America
Query!
State/province [15]
0
0
Virginia
Query!
Country [16]
0
0
Belgium
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State/province [16]
0
0
Brussels
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Country [17]
0
0
Belgium
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State/province [17]
0
0
Brussel
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Country [18]
0
0
Belgium
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State/province [18]
0
0
Gent
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Country [19]
0
0
Canada
Query!
State/province [19]
0
0
British Columbia
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Country [20]
0
0
Canada
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State/province [20]
0
0
Manitoba
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Country [21]
0
0
Canada
Query!
State/province [21]
0
0
Ontario
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Country [22]
0
0
Canada
Query!
State/province [22]
0
0
Quebec
Query!
Country [23]
0
0
Germany
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State/province [23]
0
0
Bad Bentheim
Query!
Country [24]
0
0
Germany
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State/province [24]
0
0
Erlangen
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Country [25]
0
0
Germany
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State/province [25]
0
0
Frankfurt am Main
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Country [26]
0
0
Germany
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State/province [26]
0
0
Luebeck
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Country [27]
0
0
Germany
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State/province [27]
0
0
Mainz
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Country [28]
0
0
Germany
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State/province [28]
0
0
Muenster
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Country [29]
0
0
Italy
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State/province [29]
0
0
RM
Query!
Country [30]
0
0
Japan
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State/province [30]
0
0
Aichi
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Country [31]
0
0
Japan
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State/province [31]
0
0
Miyagi
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Country [32]
0
0
Japan
Query!
State/province [32]
0
0
Tokyo
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Country [33]
0
0
Japan
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State/province [33]
0
0
Yamanashi
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Country [34]
0
0
Korea, Republic of
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State/province [34]
0
0
Gyeonggi-do
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Country [35]
0
0
Korea, Republic of
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State/province [35]
0
0
Incheon
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Country [36]
0
0
Korea, Republic of
Query!
State/province [36]
0
0
Seoul
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Country [37]
0
0
Spain
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State/province [37]
0
0
Barcelona
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Country [38]
0
0
Spain
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State/province [38]
0
0
Cordoba
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Country [39]
0
0
Spain
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State/province [39]
0
0
Madrid
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Country [40]
0
0
Spain
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State/province [40]
0
0
Valencia
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Country [41]
0
0
Taiwan
Query!
State/province [41]
0
0
Kaohsiung
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Country [42]
0
0
Taiwan
Query!
State/province [42]
0
0
Tainan
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Country [43]
0
0
Taiwan
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State/province [43]
0
0
Taipei
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Pfizer
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
This is a Phase 2b, randomized, double blind, parallel group, multicenter study with an
extension period. The study will have a maximum duration of approximately 60 weeks. This
includes an up to 4 weeks Screening Period, a 24 week dose ranging period, an up to 24 week
extension period and a 8 week Follow up Period.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT03715829
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
0
0
Pfizer CT.gov Call Center
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Address
0
0
Pfizer
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Country
0
0
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Phone
0
0
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Fax
0
0
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Email
0
0
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Contact person for public queries
Name
0
0
Query!
Address
0
0
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Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT03715829
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