Topical Ruxolitinib Evaluation in Vitiligo Study 2 (TRuE-V2): A Phase 3, Double-Blind, Randomized, Vehicle-Controlled, Efficacy and Safety Study of Ruxolitinib Cream Followed by an Extension Period in Participants With Vitiligo
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 344
- 试验地点
- 72
- 主要终点
- Percentage of Participants Achieving a ≥ 75% Improvement From Baseline in the Face Vitiligo Area Scoring Index (F-VASI75) Score at Week 24
研究概览
简要总结
The purpose of this study is to evaluate the efficacy and safety of ruxolitinib cream in adolescent and adult participants with non-segmental vitiligo for whom total body involved vitiligo area (facial and nonfacial) does not exceed 10% body surface area (BSA).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical diagnosis of non-segmental vitiligo with depigmented area including ≥ 0.5% BSA on the face, ≥ 0.5 F-VASI, ≥ 3% BSA on nonfacial areas, ≥ 3 T-VASI, and total body vitiligo area (facial and nonfacial) not exceeding 10% BSA.
- •Agree to discontinue all agents used to treat vitiligo from screening through the final safety follow-up visit. Over-the-counter preparations deemed acceptable by the investigator and camouflage makeups are permitted.
- •Must be willing to take appropriate contraceptive measures to avoid pregnancy or fathering a child for the duration of study participation.
排除标准
- •No pigmented hair within any of the vitiligo areas on the face.
- •Other forms of vitiligo (eg, segmental) or other differential diagnosis of vitiligo or other skin depigmentation disorders (eg, piebaldism, pityriasis alba, leprosy, postinflammatory hypopigmentation, progressive macule hypomelanosis, nevus anemicus, chemical leukoderma, and tinea versicolor).
- •Have used depigmentation treatments (eg, monobenzone) for past treatment of vitiligo or other pigmented areas.
- •Use of protocol-defined treatments within the indicated washout period before baseline.
研究组 & 干预措施
Double-Blind Period: Ruxolitinib cream 1.5% BID
Participants applied ruxolitinib 1.5% cream twice daily (BID) for 24 weeks.
干预措施: Ruxolitinib cream (Drug)
Double-Blind Period: Vehicle cream BID
Participants applied matching vehicle cream BID for 24 weeks.
干预措施: Vehicle (Drug)
Treatment-Extension Period: Ruxolitinib cream 1.5% BID
Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied ruxolitinib cream 1.5% BID during the Double-Blind Period continued to apply ruxolitinib cream 1.5% BID for an additional 28 weeks in the Treatment-Extension Period.
干预措施: Ruxolitinib cream (Drug)
Treatment-Extension Period: Vehicle cream to Ruxolitinib cream 1.5% BID
Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5%m BID for 28 weeks in the Treatment-Extension Period.
干预措施: Ruxolitinib cream (Drug)
Treatment-Extension Period: Vehicle cream to Ruxolitinib cream 1.5% BID
Participants who completed the Week 24 assessments with no safety concerns could continue into the 28-week Treatment-Extension Period. Participants who applied vehicle cream BID during the Double-Blind Period applied ruxolitinib cream 1.5%m BID for 28 weeks in the Treatment-Extension Period.
干预措施: Vehicle (Drug)
结局指标
主要结局
Percentage of Participants Achieving a ≥ 75% Improvement From Baseline in the Face Vitiligo Area Scoring Index (F-VASI75) Score at Week 24
时间窗: Baseline; Week 24
An F-VASI75 responder achieved at least 75% improvement from Baseline in F-VASI, measured by the percentage of vitiligo involvement (percentage of body surface area \[BSA\]) and the degree of depigmentation: 0% (no depigmentation), 10% (only specks of depigmentation), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment), or 100% (no pigment). The percentage of BSA (hand unit) vitiligo involvement was estimated to the nearest 0.1% by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate the percentage of BSA vitiligo involvement. F-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site on the face and summing the values of all sites (possible range: 0-3; lower scores indicate increased improvement).
次要结局
- Percentage of Participants Achieving a ≥ 50% Improvement From Baseline in the Face Vitiligo Area Scoring Index (F-VASI50) Score at Week 24(Baseline; Week 24)
- Percentage of Participants Achieving a ≥ 50% Improvement From Baseline in the Total Body Vitiligo Area Scoring Index (T-VASI50) Score at Week 24(Baseline; Week 24)
- Percentage Change From Baseline in Facial Body Surface Area (F-BSA) at Week 24(Baseline; Week 24)
- Percentage of Participants Achieving a ≥ 25% Improvement in the Face Vitiligo Area Scoring Index (F-VASI25) Score at Week 24(Baseline; Week 24)
- Percentage of Participants Achieving a ≥ 90% Improvement From Baseline in the Face Vitiligo Area Scoring Index (F-VASI90) Score at Week 24(Baseline; Week 24)
- Percentage of Participants Achieving a Vitiligo Noticeability Scale (VNS) of 4 or 5 at Week 24(Baseline; Week 24)
- Percentage Change From Baseline in F-VASI at Week 52(Baseline; Week 52)
- Percentage Change From Baseline in T-VASI at Week 52(Baseline; Week 52)
- Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Double-Blind Period(from the time of Informed Consent Form signing until at least 30 days after the last application of study drug (up to Week 24))
- Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Treatment-Extension Period(from the completion of the Week 24 assessments until at least 30 days after the last application of study drug (up to Week 52 + 30 days))
- Percentage of Participants Achieving a ≥ %25, ≥ %50, ≥ 75%, and ≥ 90% Improvement in the Face Vitiligo Area Scoring Index (F-VASI25/50/75/90) Score at Week 52(Baseline; Week 52)
- Percentage Change From Baseline in T-BSA at Week 24(Baseline; Week 24)
- Percentage Change From Baseline in F-VASI at Week 24(Baseline; Week 24)
- Percentage Change From Baseline in F-BSA at Week 52(Baseline; Week 52)
- Percentage Change From Baseline in T-VASI at Week 24(Baseline; Week 24)
- Percentage Change From Baseline in T-BSA at Week 52(Baseline; Week 52)
- Percentage of Participants Achieving a ≥ 25%, ≥ 75%, and ≥ 90% Improvement in the Total Body Vitiligo Area Scoring Index (T-VASI25/75/90) Score at Week 24(Baseline; Week 24)
- Percentage of Participants Achieving a ≥ 25%, ≥ 50%, ≥ 75%, and ≥ 90% Improvement in the Total Body Vitiligo Area Scoring Index (T-VASI25/50/75/90) Score at Week 52(Baseline; Week 52)
- Percentage of Participants in Each Category of VNS During the Treatment Period (Double-Blind and Treatment-Extension Periods)(Baseline; Week 24 and Week 52)
- Change From Baseline in DLQI at Week 52(Baseline; Week 52)
- Change From Baseline in Children's Dermatology Life Quality Index (CDLQI) During the Treatment Period (Double-Blind and Treatment-Extension Periods)(Baseline; Week 24 and Week 52)
- Change From Baseline in Dermatology Life Quality Index (DLQI) at Week 24(Baseline; Week 24)
- Trough Plasma Concentrations of Ruxolitinib at Weeks 4, 24, and 40(pre-dose at Weeks 4, 24, and 40)