To Assess the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva

Официальное название

A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva

Обзор

This Phase 2, Randomized, Double-Blind, Placebo-Controlled Study is intended to evaluate the Efficacy, Safety, and Tolerability and PK of INCB000928 administered to participants with a clinical diagnosis of fibrodysplasia ossificans progressiva (FOP).

Вмешательства

• Препарат INCB000928
  INCBG000928 will be administered QD orally.
• Препарат Placebo
  Placebo will be administered QD orally.

Первичные конечные точки

• Double Blind Period: Occurrence of new heterotopic ossification (HO) lesions from baseline [Срок оценки: Week 24]

Вторичные конечные точки (12)

• Double Blind Period: Number of new HO lesions from baseline [Срок оценки: Week 24]
• Double Blind Period: Total volume of new HO lesions from baseline [Срок оценки: Week 24]
• Double Blind Period: Change in the total volume of all HO lesions from baseline [Срок оценки: Week 24]
• Double Blind Period: Number of new flares from baseline [Срок оценки: Week 24]
• Number of Participants with Treatment Emergent Adverse Events (TEAE) [Срок оценки: Up to 316 weeks]
• Open-Label Extension: Occurrence of new HO lesions from Week 24 [Срок оценки: Week 48]
• Open-Label Extension: Number of new HO lesions from Week 24 [Срок оценки: Week 48]
• Open-Label Extension: Total volume of new HO lesions from Week 24 [Срок оценки: Week 48]
• Open-Label Extension: Change in the total volume of all HO lesions from Week 24 [Срок оценки: Week 48]
• Open-Label Extension: Number of new flares from Week 24 [Срок оценки: Week 48]
• Pharmacokinetics Parameter: Cmax of INCB000928 [Срок оценки: Baseline, Weeks 12, 24, 48 and 76]
• Pharmacokinetics Parameter: Tmax of INCB000928 [Срок оценки: Baseline, Weeks 12, 24, 48 and 76]

Критерии участия

Критерии включения

• Female and male participants:

• Cohort 1: ≥ 12 years of age.
• Cohort 2: 6 to < 12 years of age.
• Cohort 3: 2 to < 12 years of age (after eDMC review of safety data from Cohort 2).
• Clinical diagnosis of FOP.
• Willingness to avoid pregnancy or fathering children based on the criteria below.
• Willing and able to undergo low-dose WBCT (excluding the head) imaging without requiring intubation.
• Further inclusion criteria apply.

Критерии исключения

• Pregnant or breast-feeding.
• CAJIS score ≥ 24.
• FOP disease severity that in the investigator's opinion precludes participation.
• Any clinically significant medical condition other than FOP that would, in the investigator's judgment, interfere with full participation in the study, pose a significant risk to the participant, or interfere with interpretation of study data.
• Chronic or current active infectious disease requiring systemic antibiotic, antifungal, or antiviral treatment.
• HIV, HBV, or HCV infection. Note:
• Further exclusion criteria apply.

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

Двойное слепое

Основная цель

Лечение

Центры проведения

Китай · 4 центра

• Beijing Childrens Hospital Capital Medical University — Пекин
• Tongji Hospital of Tongji University — Шанхай
• Shanghai Childrens Medical Center — Шанхай
• Childrens Hospital of Fudan University — Шанхай

США · 3 центра

• Mayo Clinic Rochester — Rochester
• Children'S Hospital of Philadelphia — Philadelphia
• Penn Medicine - Perelman Center For Advanced Medicine — Philadelphia

Австралия · 2 центра

• Royal North Shore Hospital — St Leonards
• Murdoch Children'S Research Institute — Parkville

Франция · 2 центра

• Ap-Hp Hopital Lariboisiere — Paris
• Hopital Necker-Enfants Malades — Paris

Аргентина · 1 центр

• Hospital Italiano de Buenos Aires — Buenos Aires

Бразилия · 1 центр

• Albert Einstein Israelite Hospital — São Paulo

Канада · 1 центр

• University Health Network Toronto General Hospital — Toronto

Чили · 1 центр

• Centro de Estudios Reumatologicos — Santiago

Германия · 1 центр

• Uniklinik Koln — Cologne

Италия · 1 центр

• Policlinico Universitario Agostino Gemelli Universita Cattolica Del Sacro Cuore — Rome

Мексика · 1 центр

• Instituto Nacional de Rehabilitacion Luis Guillermo Ibarra — Tlalpan

Нидерланды · 1 центр

• Amsterdam Umc - Vu Medisch Centrum (Vumc) — Amsterdam

Новая Зеландия · 1 центр

• Starship Childrens Hospital — Auckland

ЮАР · 1 центр

• Groote Schuur Hospital Radiation Oncology — Cape Town

South Korea · 1 центр

• Seoul National University Hospital — Seoul

Испания · 1 центр

• Hospital Universitario Ramon Y Cajal — Madrid

Великобритания · 1 центр

• Royal National Orthopaedic Hospital — Stanmore

Публикации

• Yang YO, Fang Y, Wang P, Liu X, Getsy J, Rockich K. Effects of Renal and Hepatic Impairment on the Pharmacokinetics of Zilurgisertib. Br J Clin Pharmacol. 2026 May;92(5):1339-1351. doi: 10.1002/bcp.70372. Epub 2025 Dec 8. PMID 41360500

Идентификаторы

NCT: NCT05090891 · INCB 00928-201 · 2023-504129-38-00 · 2021-002286-17

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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