A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis

Официальное название

18-month Double-blind, Randomized, Placebo-controlled, Multicenter, Phase 3 Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease and in Late-infantile and Juvenile-onset Forms of GM1 Gangliosidosis or GM2 Gangliosidosis

Обзор

An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick type C disease and in late-infantile and juvenile-onset forms of GM1 gangliosidosis or GM2 gangliosidosis

Подробное описание

This is a Phase 3 randomized, double-blinded, placebo-controlled study that will evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in multiple disease areas using a Master Protocol Research Program. Participants are randomized to different subprotocols based on disease type: Niemann-Pick type C (NPC), GM1 gangliosidosis or GM2 gangliosidosis.

Treatment specific procedures will be described in the disease-specific subprotocol. Individual subprotocols may have additional eligibility requirements, safety and efficacy procedures, or endpoints, which will be described in the corresponding subprotocols.

For information specific to each individual subprotocol included in this trial, please refer to the corresponding, separate, clinicaltrials.gov records: Niemann-Pick type C disease NCT07082725 (Recruiting) and GM1 gangliosidosis or GM2 gangliosidosis NCT07082543 (Active, not recruiting - No longer looking for participants) .

Вмешательства

• Препарат AZ-3102
  Oral dispersible tablets
• Препарат Placebo
  A matching placebo will be administered in the same regimen as the intervention

Первичные конечные точки

• Number of participants allocated to subprotocol AZA-001-301-NPC [Срок оценки: Baseline to month 18]
• Number of participants allocated to subprotocol AZA-001-301-GMx [Срок оценки: Baseline to month 18]

Критерии участия

Критерии включения

• Male and female participants, aged 4 years and older with a diagnosis of the late-infantile or juvenile form of NPC disease. Detailed inclusion criteria are presented in the NPC disease-specific subprotocol AZA-001-301-NPC (NCT07082725).
• Male and female participants, aged 4 years and older with a diagnosis of GM1 or GM2 (Tay-Sachs, Sandhoff, or GM2AB variant disease) gangliosidosis of late-infantile/ juvenile onset. Detailed inclusion criteria are presented in the GM1/GM2 gangliosidosis-specific subprotocol AZA-001-301-GMx (NCT07082543).

Критерии исключения

• Detailed exclusion criteria are presented in the NPC disease-specific subprotocol AZA-001-301-NPC
• Detailed exclusion criteria are presented in the GM1/GM2 gangliosidosis-specific subprotocol AZA-001-301-GMx

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

Четверное слепое

Основная цель

Лечение

Центры проведения

США · 5 центров

• UCSF Children's Hospital and Research Center at Oakland — Oakland
• University of Minnesota Medical School — Minneapolis
• Mayo Clinic Childrens Center - PIN — Rochester
• Children's Medical Center Dallas — Dallas
• Lysosomal Rare Disorders Research and Treatment Center — Fairfax

Индия · 4 центра

• Amrita Institute of Medical Sciences and Research Centre — Ernākulam
• All India Institute of Medical Sciences (AIIMS) - New Delhi — New Delhi
• JK Lone Hospital — Jaipur
• Christian Medical College and Hospital — Vellore

Австралия · 3 центра

• Women's and Children's Hospital — North Adelaide
• Royal Melbourne Hospital — Parkville
• Royal Children's Hospital Melbourne - PIN — Parkville

Бразилия · 3 центра

• Instituto Fernandes Figueira — Rio de Janeiro
• Hospital de Clinicas de Porto Alegre (HCPA) - PPDS — Porto Alegre
• Hospital Pequeno Príncipe — Curitiba

Канада · 3 центра

• M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary — Calgary
• University of Alberta Medical Genetics Clinic — Edmonton
• Centre Hospitalier de l'Universite de Montreal-1000 rue Saint-Denis — Montreal

Turkey (Türkiye) · 3 центра

• Balcali Hastanesi Saglik Uygulama ve Arastirma Merkezi — Adana
• Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi — Çankaya
• Ege Universitesi Tip Fakultesi — Bornova

Великобритания · 3 центра

• Great Ormond Street Hospital — London
• University College London Hospitals (UCLH) — London
• Royal Manchester Children's Hospital — Manchester

Аргентина · 2 центра

• Hospital Universitario Austral — Ciudad Autónoma Buenos Aires
• Hospital de Niños de La Santisima Trinidad — Córdoba

Мексика · 2 центра

• Hospital Universitario Dr. Jose Eleuterio González — Monterrey
• Centenario Hospital Miguel Hidalgo — Aguascalientes

Португалия · 2 центра

• ULS de Santo António, EPE - Centro Materno Infantil Norte — Porto
• ULS de Santa Maria,EPE - Hospital de Santa Maria - PPDS — Lisbon

Испания · 2 центра

• Hospital Universitario Vall d'Hebron - PPDS — Barcelona
• Hospital Infantil Universitario Niño Jesus - PIN — Madrid

Франция · 1 центр

• AP-HP - Hôpital Armand Trousseau — Paris

Германия · 1 центр

• SphinCS GmbH — Höchheim

Италия · 1 центр

• Fondazione IRCCS Istituto Neurologico Carlo Besta — Milan

Пакистан · 1 центр

• The Children's Hospital & the University of Child Health Lahore — Lahore

Швеция · 1 центр

• Sahlgrenska universitetssjukhuset Östra — Gothenburg

Швейцария · 1 центр

• Inselspital - Universitätsspital Bern — Bern

Идентификаторы

NCT: NCT07054515 · AZA-001-301 · 2024-515778-28-00

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗