A Phase 2a, proof-of-concept, multicenter, double-blind, randomized, placebo-controlled, parallel-group trial to assess the efficacy and safety of ACT-777991 in adults with non-segmental vitiligo
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 24
- 试验地点
- 6
- 主要终点
- Main primary endpoint: Percentage change from baseline in F-VASI based on BICR at Week 24 VASI is a validated clinician-reported outcome measure that scores both the extent (surface area) and degree (level of depigmentation) of vitiligo lesions over time. The F-VASI describes involvement of the face, with higher scores indicating more severe disease. Negative changes from baseline indicate improvement.
研究概览
简要总结
To assess the effect of ACT-777991, compared with placebo, on facial repigmentation in participants with non- segmental vitiligo.
研究设计
- 分配方式
- Na
- 主要目的
- Follow-up period
- 盲法
- Double (Investigator, Analyst, Subject, Monitor)
入排标准
- 年龄范围
- 18 years 至 65+ years（18-64 Years, 65+ Years）
- 接受健康志愿者
- 否
入选标准
- •Clinical diagnosis of either active or stable non-segmental vitiligo for at least 3 months prior to Screening and meet all the following criteria: – Facial Vitiligo Area Scoring Index (F-VASI) score ≥ 0.3 based on Blinded Independent Central Reading (BICR) at Screening. – Total Body Vitiligo Area Scoring Index (T-VASI) score ≥ 5 based on investigator assessment at Screening and Randomization. – Total body surface area (BSA) involvement, including the face, ≤ 50% based on investigator assessment at Screening and Randomization.
- •Participants must not have discontinued, reduced, or modified a vitiligo treatment/procedure for the purpose of meeting trial eligibility criteria or a wash-out requirement. Participants must also agree not to use therapeutic agents and procedures to treat vitiligo from Screening until Patient Last Visit (PLV).
排除标准
- •Clinical diagnosis of other forms of vitiligo (e.g., segmental) or other hypo- or depigmentation disorders (e.g., piebaldism, leukoderma, Vogt-Koyanagi-Harada disease, malignancy-induced hypopigmentation).
- •Any autoimmune disease, except adequately treated thyroid disease.
- •History of systemic immunotherapy treatment, including JAK inhibitors, for any inflammatory disease in the 12 months prior to Randomization.
- •History of topical JAK inhibitors for any inflammatory disease in the 6 weeks prior to Screening.
- •Use of laser or light-based treatment (phototherapy), including tanning beds, in the 8 weeks prior to Screening.
- •eGFR < 90 mL/min/1.73 m2, defined by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) creatinine equation, at Screening.
研究组 & 干预措施
ACT-777991 matching placebo
干预措施: ACT-777991 matching placebo (Drug)
结局指标
主要结局
Main primary endpoint: Percentage change from baseline in F-VASI based on BICR at Week 24 VASI is a validated clinician-reported outcome measure that scores both the extent (surface area) and degree (level of depigmentation) of vitiligo lesions over time. The F-VASI describes involvement of the face, with higher scores indicating more severe disease. Negative changes from baseline indicate improvement.
Main primary endpoint: Percentage change from baseline in F-VASI based on BICR at Week 24 VASI is a validated clinician-reported outcome measure that scores both the extent (surface area) and degree (level of depigmentation) of vitiligo lesions over time. The F-VASI describes involvement of the face, with higher scores indicating more severe disease. Negative changes from baseline indicate improvement.
Supplementary primary endpoint: Percentage change from baseline in F-VASI based on investigator assessment at Week 24
Supplementary primary endpoint: Percentage change from baseline in F-VASI based on investigator assessment at Week 24
Supplementary primary endpoint: Percentage change from baseline in F-VASI at Week 4, 8, and 16 F-VASI will be assessed by the investigator and by BICR.
Supplementary primary endpoint: Percentage change from baseline in F-VASI at Week 4, 8, and 16 F-VASI will be assessed by the investigator and by BICR.
Supplementary primary endpoint: Achievement of F-VASI50 at Week 4, 8, 16 and 24 Proportion of patients achieving at least a 50% improvement from baseline in F-VASI.
Supplementary primary endpoint: Achievement of F-VASI50 at Week 4, 8, 16 and 24 Proportion of patients achieving at least a 50% improvement from baseline in F-VASI.
Supplementary primary endpoint: Achievement of F-VASI75 at Week 4, 8, 16 and 24 Proportion of patients achieving at least a 75% improvement from baseline in F-VASI.
Supplementary primary endpoint: Achievement of F-VASI75 at Week 4, 8, 16 and 24 Proportion of patients achieving at least a 75% improvement from baseline in F-VASI.
Supplementary primary endpoint: Achievement of F-VASI90 at Week 4, 8, 16 and 24 Proportion of patients achieving at least a 90% improvement from baseline in F-VASI.
Supplementary primary endpoint: Achievement of F-VASI90 at Week 4, 8, 16 and 24 Proportion of patients achieving at least a 90% improvement from baseline in F-VASI.
次要结局
- Treatment-emergent marked abnormalities for vital signs, clinical laboratory variables, and ECG measurements
- Percentage change from baseline in T-VASI at Week 4, 8, 16 and 24 T-VASI will be assessed by the investigator. The T-VASI is calculated using a formula that includes contributions from all body regions, with higher scores indicating more severe disease. Negative changes from baseline indicate improvement.
- Achievement of T-VASI50 based on investigator assessment at Week 4, 8, 16 and 24 Proportion of patients achieving at least a 50% improvement from baseline in T-VASI.
- Adverse events (AEs) leading to premature discontinuation of trial intervention
- Treatment-emergent AEs and serious AEs (SAEs)
- Treatment-emergent AEs of special interest (AESIs)
- Change from baseline to all assessed time points in vital signs, clinical laboratory variables, and ECG measurements
研究者
Idorsia Clinical Trials Information
Scientific
Idorsia Pharmaceuticals Ltd.