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
Status: Recruiting
Location: See all (32) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
SUMMARY
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
Eligibility
Participation Requirements
Sex: All
Minimum Age: 4
Healthy Volunteers: f
View:
• 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).
Locations
United States
California
Children's Hospital and Research Center at Oakland
NOT_YET_RECRUITING
Oakland
Minnesota
Mayo Clinic Childrens Center - PIN
NOT_YET_RECRUITING
Rochester
Virginia
Lysosomal Rare Disorders Research and Treatment Center
NOT_YET_RECRUITING
Fairfax
Other Locations
Argentina
Hospital Universitario Austral
NOT_YET_RECRUITING
Ciudad Autónoma Buenos Aires
Hospital de Niños de La Santisima Trinidad
NOT_YET_RECRUITING
Córdoba
Australia
Women's and Children's Hospital
NOT_YET_RECRUITING
North Adelaide
Royal Children's Hospital Melbourne - PIN
NOT_YET_RECRUITING
Parkville
Royal Melbourne Hospital
NOT_YET_RECRUITING
Parkville
Brazil
Hospital Pequeno Príncipe
NOT_YET_RECRUITING
Curitiba
Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
NOT_YET_RECRUITING
Porto Alegre
Instituto Fernandes Figueira
NOT_YET_RECRUITING
Rio De Janeiro
Canada
M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary
NOT_YET_RECRUITING
Calgary
University of Alberta Medical Genetics Clinic
NOT_YET_RECRUITING
Edmonton
Centre Hospitalier de l'Universite de Montreal-1000 rue Saint-Denis
NOT_YET_RECRUITING
Montreal
France
AP-HM- Hôpital de La Timone
NOT_YET_RECRUITING
Marseille
AP-HP - Hôpital Armand Trousseau
NOT_YET_RECRUITING
Paris
India
Amrita Institute of Medical Sciences and Research Centre
NOT_YET_RECRUITING
Ernākulam
JK Lone Hospital
NOT_YET_RECRUITING
Jaipur
Seth G S Medical College and K E M Hospital
NOT_YET_RECRUITING
Mumbai
All India Institute of Medical Sciences (AIIMS) - New Delhi
NOT_YET_RECRUITING
New Delhi
Italy
Fondazione IRCCS Istituto Neurologico Carlo Besta
RECRUITING
Milan
Portugal
ULS de Santa Maria,EPE - Hospital de Santa Maria - PPDS
RECRUITING
Lisbon
ULS de Santo António, EPE - Centro Materno Infantil Norte
RECRUITING
Porto
Spain
Hospital Universitario Vall d'Hebron - PPDS
RECRUITING
Barcelona
Hospital Infantil Universitario Niño Jesus - PIN
RECRUITING
Madrid
Sweden
Sahlgrenska universitetssjukhuset Östra
RECRUITING
Gothenburg
Switzerland
Inselspital - Universitätsspital Bern
NOT_YET_RECRUITING
Bern
Turkey
Balcali Hastanesi Saglik Uygulama ve Arastirma Merkezi
NOT_YET_RECRUITING
Adana
Ege Universitesi Tip Fakultesi
NOT_YET_RECRUITING
Bornova
Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi
NOT_YET_RECRUITING
Çankaya
United Kingdom
Great Ormond Street Hospital
NOT_YET_RECRUITING
London
University College London Hospitals (UCLH)
NOT_YET_RECRUITING
London
Contact Information
Primary
Patient Advocacy Representative
info@azafaros.com
Please reach out by email
Backup
Contact for Healthcare Professionals
medinfo@azafaros.com
Please reach out by email
Time Frame
Start Date: 2025-06-30
Estimated Completion Date: 2027-11-04
Participants
Target number of participants: 147
Treatments
Experimental: Subprotocol NCT07082725: Intervention Group NPC
Participants will be randomized to receive either nizubaglustat or placebo at a 2:1 ratio (nizubaglustat:placebo)
Experimental: Subprotocol NCT07082543: Intervention Group GM1/GM2
Participants will be randomized to receive either nizubaglustat or placebo at a 2:1 ratio (nizubaglustat:placebo)
Related Therapeutic Areas
Sponsors
Leads: Azafaros A.G.