Active Non-segmental Vitiligo Clinical Trial
Official 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
| Verified date | March 2022 |
| Source | Pfizer |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
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.
| Status | Completed |
| Enrollment | 366 |
| Est. completion date | February 5, 2021 |
| Est. primary completion date | February 5, 2021 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 18 Years to 65 Years |
| Eligibility | 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. 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) |
| Country | Name | City | State |
|---|---|---|---|
| Australia | Skin Health Institute | Carlton | Victoria |
| Australia | The Skin Hospital | Darlinghurst | New South Wales |
| Australia | Sinclair Dermatology | East Melbourne | Victoria |
| Australia | Premier Specialists Pty Ltd | Kogarah | New South Wales |
| Australia | The Royal Melbourne Hospital | Parkville | Victoria |
| Australia | The Royal Melbourne Hospital | Parkville | Victoria |
| Australia | Veracity Clinical Research Pty Ltd | Woolloongabba | Queensland |
| Belgium | UZ Brussel - Dermatology | Brussel | |
| Belgium | Hôpital Erasme Dermatology | Brussels | |
| Belgium | UZ Gent - Dermatology | Gent | |
| Canada | CCA Medical Research | Ajax | Ontario |
| Canada | Guenther Research Inc. | London | Ontario |
| Canada | Lynderm Research Inc. | Markham | Ontario |
| Canada | DermEdge Research | Mississauga | Ontario |
| Canada | Innovaderm Research Inc. | Montreal | Quebec |
| Canada | North York Research Inc. | North York | Ontario |
| Canada | The Centre for Clinical Trials | Oakville | Ontario |
| Canada | Centre de Recherche Dermatologique du Quebec metropolitain (CRDQ) | Quebec | |
| Canada | Centre de Recherche Saint-Louis | Quebec | |
| Canada | Centre de Recherche Saint-Louis | Quebec | |
| Canada | The Centre for Dermatology | Richmond Hill | Ontario |
| Canada | Diex Research Sherbrooke Inc. | Sherbrooke | Quebec |
| Canada | Dr. Chih-ho Hong Medical Inc. | Surrey | British Columbia |
| Canada | Enverus Medical Research | Surrey | British Columbia |
| Canada | University of British Columbia | Vancouver | British Columbia |
| Canada | K.Papp Clinical Research | Waterloo | Ontario |
| Canada | Wiseman Dermatology Research Inc. | Winnipeg | Manitoba |
| Germany | Fachklinik Bad Bentheim, Fachbereich Dermatologie und Allergologie, Dermatologische Ambulanz | Bad Bentheim | |
| Germany | Universitaetsklinikum Erlangen Hautklinik Studienambulanz | Erlangen | |
| Germany | Universitätsklinikum Frankfurt, Klinik für Dermatologie, Venerologie und Allergologie | Frankfurt am Main | |
| Germany | Universitaetsklinikum Schleswig-Holstein, Campus Luebeck CCIM | Luebeck | |
| Germany | Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz | Mainz | |
| Germany | Universitaetsklinikum Muenster | Muenster | |
| Italy | Istituti Fisioterapici Ospitalieri, Istituto di Ricovero e Cura a Carattere Scientifico, | Roma | RM |
| Japan | Nippon Medical School Hospital | Bunkyo-ku | Tokyo |
| Japan | Yamanashi Prefectural Central Hospital | Kofu | Yamanashi |
| Japan | Nagoya City University Hospital - Dermatology | Nagoya | Aichi |
| Japan | Tohoku University Hospital | Sendai | Miyagi |
| Japan | Tokyo Medical University Hospital | Shinjuku-ku | Tokyo |
| Korea, Republic of | Dongguk University Ilsan Hospital | Goyang-si | Gyeonggi-do |
| Korea, Republic of | Inha University Hospital | Incheon | |
| Korea, Republic of | Severance Hospital, Yonsei University Health System | Seoul | |
| Korea, Republic of | Ajou University Hospital | Suwon | Gyeonggi-do |
| Korea, Republic of | The Catholic University of Korea, St. Vincent's Hospital | Suwon-si | Gyeonggi-do |
| Spain | Hospital de la Santa Creu i Sant Pau | Barcelona | |
| Spain | Hospital Universitario Reina Sofia | Cordoba | |
| Spain | Hospital Universitario La Paz | Madrid | |
| Spain | Hospital Universitario Ramón y Cajal | Madrid | |
| Spain | Hospital Universitari i Politecnic La Fe | Valencia | |
| Taiwan | Chang Gung Medical Foundation - Kaohsiung Chang Gung Memorial Hospital | Kaohsiung | |
| Taiwan | National Cheng Kung University Hospital | Tainan | |
| Taiwan | National Taiwan University Hospital | Taipei | |
| United States | University Physicians Group | Austin | Texas |
| United States | Tobias & Battite, Inc. | Boston | Massachusetts |
| United States | Tufts Medical Center | Boston | Massachusetts |
| United States | Brookside Dermatology Associates | Bridgeport | Connecticut |
| United States | New England Research Associates, LLC | Bridgeport | Connecticut |
| United States | Chevy Chase Dermatology Center (TrialSpark, Inc.) | Chevy Chase | Maryland |
| United States | TrialSpark - Samantha Toerge, MD | Chevy Chase | Maryland |
| United States | Advanced Skin and MOHS Surgery Center, c/o TrialSpark, Inc. | Chicago | Illinois |
| United States | ForeFront Dermatology | Columbus | Ohio |
| United States | Remington-Davis, Inc. Clinical Research | Columbus | Ohio |
| United States | University of Texas Southwestern Medical Center | Dallas | Texas |
| United States | Henry Ford Hospital Department of Dermatology | Detroit | Michigan |
| United States | Pickens Academic Tower | Houston | Texas |
| United States | The University of Texas Health Science Center at Houston | Houston | Texas |
| United States | The University of Texas MD Anderson Cancer Center | Houston | Texas |
| United States | Marvel Research, LLC | Huntington Beach | California |
| United States | University of California, Irvine, Dermatology Clinical Research Center | Irvine | California |
| United States | Vitiligo and Pigmentation Institute Of Southern California | Los Angeles | California |
| United States | New Horizon Research Center | Miami | Florida |
| United States | University of Minnesota Department of Dermatology | Minneapolis | Minnesota |
| United States | Dermatology Specialist, Inc. | Murrieta | California |
| United States | Icahn School of Medicine at Mount Sinai | New York | New York |
| United States | MDCS: Medical Dermatology & Cosmetic Surgery (TrialSpark, Inc.) | New York | New York |
| United States | Upper West Side Dermatology c/o TrialSpark, Inc | New York | New York |
| United States | Virginia Clinical Research, Inc | Norfolk | Virginia |
| United States | Dermatology Specialists, Inc. | Oceanside | California |
| United States | South Nassau Dermatology | Oceanside | New York |
| United States | TrialSpark, Inc. - Russell W. Cohen, MD | Oceanside | New York |
| United States | Park Avenue Dermatology | Orange Park | Florida |
| United States | TrialSpark - Ronald Shore, MD | Rockville | Maryland |
| United States | UC Davis Health | Sacramento | California |
| United States | ForCare Clinical Research | Tampa | Florida |
| United States | Vital Prospects Clinical Research Institute, PC | Tulsa | Oklahoma |
| United States | The Dermatology Group, P.C. | Verona | New Jersey |
| United States | Tamjidi Skin Institute (TrialSpark, Inc.) | Vienna | Virginia |
| United States | PMG Research of Wilmington, LLC | Wilmington | North Carolina |
| United States | Investigational Drug Service Pharmacy | Worcester | Massachusetts |
| United States | UMass Memorial Medical Center Ear Nose and Throat | Worcester | Massachusetts |
| United States | UMass Memorial Medical Center, Hahnemann Campus | Worcester | Massachusetts |
| United States | University of Massachusetts Medical School | Worcester | Massachusetts |
| Lead Sponsor | Collaborator |
|---|---|
| Pfizer |
United States, Australia, Belgium, Canada, Germany, Italy, Japan, Korea, Republic of, Spain, Taiwan,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Percent Change From Baseline in Central Read Facial-Vitiligo Area Scoring Index (F-VASI) at Week 24 - Dose Ranging (DR) Period | 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. | Baseline, Week 24 (Baseline was defined as the last measurement prior to Study Day 18) | |
| Primary | Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) up to Week 24 - DR Period | 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. | 24 weeks | |
| Primary | Number of Participants With the TEAEs of Anaemia, Neutropenia, Thrombocytopenia and Lymphopenia - DR Period | 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). |
Baseline up to Week 24 | |
| Primary | Number of Participants With Clinically Meaningful Changes From Baseline in Lipid Profile up to Week 24 - DR Period | 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). |
Baseline up to Week 24 | |
| Primary | Number of Participants With Liver Function Test Values Meeting the Protocol-Specified Discontinuation Criteria - DR Period | Liver function tests included tests of alanine aminotransferase (ALT), aspartate aminotransferase (AST) and total bilirubin. | 24 weeks | |
| Primary | Number of Participants With TEAEs and SAEs - Extension (Ext) Period | 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. | 24 weeks | |
| Primary | Number of Participants With the TEAEs of Anaemia, Neutropenia, Thrombocytopenia and Lymphopenia - Ext Period | 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. |
24 weeks | |
| Primary | Number of Participants With Clinically Meaningful Changes From Baseline in Lipid Profile - Ext Period | 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. | 24 Weeks | |
| Primary | Number of Participants With Liver Function Test Values Meeting the Protocol-Specified Discontinuation Criteria - Ext Period | Liver function tests included tests of alanine aminotransferase (ALT), aspartate aminotransferase (AST) and total bilirubin. | 24 weeks | |
| Secondary | Percentage of Participants Achieving Central F-VASI75 at Week 24 - DR Period | 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. |
Week 24 | |
| Secondary | Percentage of Participants Achieving T-VASI50 at Week 24 - DR Period | 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). | Week 24 | |
| Secondary | Percent Change From Baseline in T-VASI at Designated Time Points - DR Period | 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). | Baseline, Weeks 4, 8, 12, 16, 20 and 24 | |
| Secondary | Percent Change From Baseline in Central Read F-VASI at Designated Time Points - DR Period | 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. |
Baseline, Weeks 4, 8, 16 and 24 | |
| Secondary | Percent Change From Baseline in Local F-VASI at Designated Time Points - DR Period | 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). |
Baseline, Weeks 4, 8, 12, 16, 20 and 24 | |
| Secondary | Percent Change From Baseline in SA-VES at Designated Time Points - DR Period | 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). | Baseline, Weeks 4, 16 and 24 | |
| Secondary | Absolute Change From Baseline in T-VASI at Designated Time Points - DR Period | 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). |
Baseline, Weeks 4, 8, 12, 16, 20 and 24 | |
| Secondary | Percentage of Participants Achieving T-VASI50 at Designated Time Points - DR Period | 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). | Baseline, Weeks 4, 8, 12, 16, 20 and 24 | |
| Secondary | Percentage of Participants Achieving T-VASI75 at Designated Time Points - DR Period | 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). | Baseline, Weeks 4, 8, 12, 16, 20 and 24 | |
| Secondary | Percentage of Participants Achieving T-VASI90 at Designated Time Points - DR Period | 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). | Baseline, Weeks 4, 8, 12, 16, 20 and 24 | |
| Secondary | Percentage of Participants Achieving T-VASI100 at Designated Time Points - DR Period | 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). | Baseline, Weeks 4, 8, 12, 16, 20 and 24 | |
| Secondary | Percentage of Participants Achieving Central Read F-VASI50 at Designated Time Points - DR Period | 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. | Baseline, Weeks 4, 8, 16 and 24 | |
| Secondary | Percentage of Participants Achieving Central Read F-VASI75 at Designated Time Points - DR Period | 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. | Baseline, Weeks 4, 8, 16 and 24 | |
| Secondary | Percentage of Participants Achieving Central Read F-VASI90 at Designated Time Points - DR Period | 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. | Baseline, Weeks 4, 8, 16 and 24 | |
| Secondary | Percentage of Participants Achieving Central Read F-VASI100 at Designated Time Points - DR Period | 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. | Baseline, Weeks 4, 8, 16 and 24 | |
| Secondary | Percentage of Participants Achieving Local F-VASI50 at Designated Time Points - DR Period | 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). | Baseline, Weeks 4, 8, 12, 16, 20 and 24 | |
| Secondary | Percentage of Participants Achieving Local F-VASI75 at Designated Time Points - DR Period | 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). | Baseline, Weeks 4, 8, 12, 16, 20 and 24 | |
| Secondary | Percentage of Participants Achieving Local F-VASI90 at Designated Time Points - DR Period | 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). | Baseline, Weeks 4, 8, 12, 16, 20 and 24 | |
| Secondary | Percentage of Participants Achieving Local F-VASI100 at Designated Time Points - DR Period | 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). | Baseline, Weeks 4, 8, 12, 16, 20 and 24 | |
| Secondary | Change From Baseline in Total VitiQoL Score at Designated Time Points - DR Period | 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). |
Baseline, Weeks 4, 16 and 24 | |
| Secondary | Change From Baseline in VitiQoL Participation Limitation Domain Score at Designated Time Points - DR Period | 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). |
Baseline, Weeks 4, 16 and 24 | |
| Secondary | Change From Baseline in VitiQoL Stigma Domain Score at Designated Time Points - DR Period | 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). |
Baseline, Weeks 4, 16 and 24 | |
| Secondary | Change From Baseline in VitiQoL Behaviors Domain Score at Designated Time Points - DR Period | 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). |
Baseline, Weeks 4, 16 and 24 | |
| Secondary | Percentage of Participants Achieving sIGA 0 or 1 and at Least a 2-Point Improvement at Week 24 - DR Period | 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. |
Week 24 |