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 (36) 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 GM1 gangliosidosis or GM2 gangliosidosis
Eligibility
Participation Requirements
Sex: All
Minimum Age: 4
Healthy Volunteers: f
View:
• Confirmed GM1 gangliosidosis or Tay-Sachs, Sandhoff, or GM2AB variant
• Male and female participants aged 4 years and older at the time of informed consent
• Onset of neurological symptoms from 1 to 10 years
• Disability level at Baseline: Ataxic disturbances with a total SARA score of ≥3 and ≤30 at Baseline
• Females of childbearing potential who are sexually active willing to follow the contraceptive guidance
• Male participants with a female partner of childbearing potential willing to follow the contraceptive guidance
Locations
United States
California
UCSF Children's Hospital and Research Center at Oakland
RECRUITING
Oakland
Minnesota
University of Minnesota Medical School
NOT_YET_RECRUITING
Minneapolis
Mayo Clinic Children's Center - PIN
RECRUITING
Rochester
Texas
Children's Medical Center Dallas
NOT_YET_RECRUITING
Dallas
Virginia
Lysosomal Rare Disorders Research and Treatment Center
RECRUITING
Fairfax
Other Locations
Argentina
Hospital Universitario Austral
NOT_YET_RECRUITING
Ciudad Autónoma Buenos Aires
Hospital de Niños de La Santisima Trinidad
RECRUITING
Córdoba
Australia
Women's and Children's Hospital
RECRUITING
North Adelaide
Royal Children's Hospital Melbourne - PIN
NOT_YET_RECRUITING
Parkville
Brazil
Hospital Pequeno Principe
NOT_YET_RECRUITING
Curitiba
Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
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-HP - Hôpital Armand Trousseau
RECRUITING
Paris
Germany
SphinCS GmbH
NOT_YET_RECRUITING
Höchheim
India
Amrita Institute of Medical Sciences and Research Centre
RECRUITING
Ernākulam
JK Lone Hospital
RECRUITING
Jaipur
All India Institute of Medical Sciences (AIIMS) - New Delhi
RECRUITING
New Delhi
Christian Medical College and Hospital
NOT_YET_RECRUITING
Vellore
Italy
Fondazione IRCCS Istituto Neurologico Carlo Besta
RECRUITING
Milan
Mexico
Centenario Hospital Miguel Hidalgo
NOT_YET_RECRUITING
Aguascalientes
Hospital Universitario Dr. Jose Eleuterio González
NOT_YET_RECRUITING
Monterrey
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 NHSFT
NOT_YET_RECRUITING
London
University College London Hospitals (UCLH)
NOT_YET_RECRUITING
London
Royal Manchester Children's Hospital
NOT_YET_RECRUITING
Manchester
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: 75
Treatments
Experimental: Nizubaglustat
Once daily oral dispersible tablets
Placebo_comparator: Placebo
Related Therapeutic Areas
Sponsors
Leads: Azafaros A.G.