A Study to Learn How Safe ACT-777991 is and How Well it Works in Adults With Non-segmental Vitiligo

Официальное название

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

Обзор

The purpose of this clinical trial is to learn how well ACT-777991 works, how safe it is and how well it is tolerated by adults with non-segmental vitiligo. The main question this clinical trial aims to answer is: • Can ACT-777991 help return color to the skin of the face of adults with non-segmental vitiligo? Researchers will compare ACT-777991 to placebo (a look-alike inactive treatment that contains no medicine) to see if ACT-777991 works to treat non-segmental vitiligo. Trial participants will: * Take the trial intervention (either ACT-777991 or placebo) daily for 24 weeks. * Visit the clinic 7 times for check-up and tests.

Подробное описание

The trial includes three trial periods:

Following a Screening period, during which it will be checked if participants are eligible to take part, eligible participants will be randomized in a 2:1 ratio to receive either ACT-777991 or placebo for 24 weeks (Trial intervention period). On completion of treatment, participants will be followed for 30 (+7) days (Follow-up period).

Trial participation will end with a Follow-up visit (Participant Last Visit) at the end of the Follow-up period.

The maximum trial duration for an individual participant is approximately 33 weeks including a screening period of up to 28 days, a treatment period of 24 weeks, and a follow-up period of up to 37 days.

Вмешательства

• Препарат ACT-777991
  ACT-777991 tablets
• Препарат Placebo
  ACT-777991-matching placebo tablets

Первичные конечные точки

• Main primary outcome measure: Percentage change from baseline in Facial Vitiligo Area Scoring Index (F-VASI) based on Blinded Independent Central Reading (BICR) at Week 24 [Срок оценки: Baseline; Week 24]
• Supplementary primary outcome measure: Percentage change from baseline in F-VASI based on investigator assessment at Week 24 [Срок оценки: Baseline; Week 24]
• Supplementary primary outcome measure: Percentage change from baseline in F-VASI at Week 4, 8 and 16 [Срок оценки: Baseline; Week 4, Week 8; Week 16]
• Supplementary primary outcome measure: Achievement of F-VASI50 at Week 4, 8, 16 and 24 [Срок оценки: Baseline; Week 4; Week 8; Week 16; Week 24]
• Supplementary primary outcome measure: Achievement of F-VASI75 at Week 4, 8, 16 and 24 [Срок оценки: Baseline; Week 4; Week 8; Week 16; Week 24]
• Supplementary primary outcome measure: Achievement of F-VASI90 at Week 4, 8, 16 and 24 [Срок оценки: Baseline; Week 4; Week 8; Week 16; Week 24]

Вторичные конечные точки (12)

• Percentage change from baseline in Total Body Vitiligo Area Scoring Index (T-VASI) at Week 4, 8, 16 and 24 [Срок оценки: Baseline; Week 24]
• Achievement of T-VASI50 at Week 4, 8, 16 and 24 [Срок оценки: Baseline; Week 4; Week 8; Week 16; Week 24]
• Adverse events (AEs) leading to premature discontinuation of trial intervention [Срок оценки: From start of trial intervention to last dose of trial intervention, assessed up to Week 24]
• Treatment-emergent AEs and serious AEs (SAEs) [Срок оценки: From start of trial intervention up to 37 days after last dose of trial intervention (Follow-up visit)]
• Treatment-emergent AEs of special interest (AESI) [Срок оценки: From start of trial intervention up to 37 days after last dose of trial intervention (Follow-up visit)]
• Change from baseline in vital signs: systolic and diastolic blood pressure [Срок оценки: Baseline to all pre-defined time points, up to 37 days after last dose of trial intervention (Follow-up visit)]
• Change from baseline in vital signs: pulse rate [Срок оценки: Baseline to all pre-defined time points, up to 37 days after last dose of trial intervention (Follow-up visit)]
• Change from baseline in hematology variables [Срок оценки: Baseline to all pre-defined time points, up to 37 days after last dose of trial intervention (Follow-up visit)]
• Change from baseline in blood chemistry variables [Срок оценки: Baseline to all pre-defined time points, up to 37 days after last dose of trial intervention (Follow-up visit)]
• Change from baseline in ECG parameters: PR interval, QRS duration, QTcF Value [Срок оценки: Baseline to all pre-defined time points, up to 37 days after last dose of trial intervention (Follow-up visit)]
• Change from baseline in ECG parameters: Heart rate [Срок оценки: Baseline to all pre-defined time points, up to 37 days after last dose of trial intervention (Follow-up visit)]
• Number of Participants with treatment-emergent marked abnormalities in vital signs: systolic and diastolic blood pressure, pulse rate [Срок оценки: From start of trial intervention up to 37 days after last dose of trial intervention (Follow-up visit)]

Критерии участия

Критерии включения

• Clinical diagnosis of either active or stable non segmental vitiligo for at least 3 months prior to Screening and meet all the following criteria:

• F-VASI score ≥ 0.3 based on BICR at Screening.
• 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 agree not to use therapeutic agents and procedures to treat vitiligo from Screening until Participant Last Visit.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Тройное слепое

Основная цель

Лечение

Центры проведения

Список центров уточняется — проверьте первичный протокол.

Идентификаторы

NCT: NCT07667569 · ID-089B201 · 2025-524865-25-00

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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