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
Recruiting now · Phase 3 · Has a placebo group
Conditions studied: Niemann-Pick Type C Disease, GM1 Gangliosidosis, GM2 Gangliosidosis
In brief
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
Key facts
- Study ID
- NCT07054515
- Run by
- Azafaros B.V.
- People needed
- 147
- Starts
- 2025-06-30
- Expected to finish
- 2028-06-14
- Last updated by the study team
- 2026-08-04
Who can join
Age: 4 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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).
You may not qualify if…
- 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
Where it is running
- UCSF Children's Hospital and Research Center at Oakland — Oakland, California, United States (enrolling)
- Amrita Institute of Medical Sciences and Research Centre — Ernākulam, Kerala, India (enrolling)
- All India Institute of Medical Sciences (AIIMS) - New Delhi — New Delhi, National Capital Territory of Delhi, India (enrolling)
- JK Lone Hospital — Jaipur, Rajasthan, India (enrolling)
- Fondazione IRCCS Istituto Neurologico Carlo Besta — Milan, Italy (enrolling)
- ULS de Santo António, EPE - Centro Materno Infantil Norte — Porto, Porto District, Portugal (enrolling)
- ULS de Santa Maria,EPE - Hospital de Santa Maria - PPDS — Lisbon, Portugal (enrolling)
- Hospital Universitario Vall d'Hebron - PPDS — Barcelona, Barcelona, Spain (enrolling)
- Hospital Infantil Universitario Niño Jesus - PIN — Madrid, Madrid, Spain (enrolling)
- Inselspital - Universitätsspital Bern — Bern, Canton of Bern, Switzerland (enrolling)
- Balcali Hastanesi Saglik Uygulama ve Arastirma Merkezi — Adana, Adana, Turkey (Türkiye) (enrolling)
- Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi — Çankaya, Ankara, Turkey (Türkiye) (enrolling)
- Ege Universitesi Tip Fakultesi — Bornova, İzmir, Turkey (Türkiye) (enrolling)
- Great Ormond Street Hospital — London, London, United Kingdom (enrolling)
- Mayo Clinic Childrens Center - PIN — Rochester, Minnesota, United States (enrolling)
- Children's Medical Center Dallas — Dallas, Texas, United States (enrolling)
- Lysosomal Rare Disorders Research and Treatment Center — Fairfax, Virginia, United States (enrolling)
- Hospital de Niños de La Santisima Trinidad — Córdoba, Córdoba Province, Argentina (enrolling)
- Women's and Children's Hospital — North Adelaide, South Australia, Australia (enrolling)
- Royal Melbourne Hospital — Parkville, Victoria, Australia (enrolling)
- Instituto Fernandes Figueira — Rio de Janeiro, Rio de Janeiro, Brazil (enrolling)
- Hospital de Clinicas de Porto Alegre (HCPA) - PPDS — Porto Alegre, Rio Grande do Sul, Brazil (enrolling)
- M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary — Calgary, Alberta, Canada (enrolling)
- AP-HP - Hôpital Armand Trousseau — Paris, France (enrolling)
- SphinCS GmbH — Höchheim, Germany (enrolling)
Full record on ClinicalTrials.gov
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