AZ-3102: Oral dispersible tablets
Placebo: A matching placebo will be administered in the same regimen as the intervention
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
| Facility | City | Region | Status |
|---|---|---|---|
| UCSF Children's Hospital and Research Center at Oakland | Oakland | California | Recruiting |
| University of Minnesota Medical School | Minneapolis | Minnesota | Not Yet Recruiting |
| Mayo Clinic Childrens Center - PIN | Rochester | Minnesota | Recruiting |
| Children's Medical Center Dallas | Dallas | Texas | Recruiting |
| Lysosomal Rare Disorders Research and Treatment Center | Fairfax | Virginia | Recruiting |
| Hospital Universitario Austral | Ciudad Autónoma Buenos Aires | Buenos Aires | Active Not Recruiting |
| Hospital de Niños de La Santisima Trinidad | Córdoba | Córdoba Province | Recruiting |
| Women's and Children's Hospital | North Adelaide | South Australia | Recruiting |
| Royal Melbourne Hospital | Parkville | Victoria | Recruiting |
| Royal Children's Hospital Melbourne - PIN | Parkville | Victoria | Recruiting |
| Instituto Fernandes Figueira | Rio de Janeiro | Rio de Janeiro | Recruiting |
| Hospital de Clinicas de Porto Alegre (HCPA) - PPDS | Porto Alegre | Rio Grande do Sul | Recruiting |
| Hospital Pequeno Príncipe | Curitiba | Brazil | Not Yet Recruiting |
| M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary | Calgary | Alberta | Recruiting |
| University of Alberta Medical Genetics Clinic | Edmonton | Alberta | Not Yet Recruiting |
| Centre Hospitalier de l'Universite de Montreal-1000 rue Saint-Denis | Montreal | Quebec | Not Yet Recruiting |
| AP-HP - Hôpital Armand Trousseau | Paris | France | Recruiting |
| SphinCS GmbH | Höchheim | Germany | Recruiting |
| Amrita Institute of Medical Sciences and Research Centre | Ernākulam | Kerala | Recruiting |
| All India Institute of Medical Sciences (AIIMS) - New Delhi | New Delhi | National Capital Territory of Delhi | Recruiting |
| JK Lone Hospital | Jaipur | Rajasthan | Recruiting |
| Christian Medical College and Hospital | Vellore | Tamil Nadu | Recruiting |
| Fondazione IRCCS Istituto Neurologico Carlo Besta | Milan | Italy | Recruiting |
| Hospital Universitario Dr. Jose Eleuterio González | Monterrey | Nuevo León | Not Yet Recruiting |
| Centenario Hospital Miguel Hidalgo | Aguascalientes | Mexico | Not Yet Recruiting |
| The Children's Hospital & the University of Child Health Lahore | Lahore | Punjab Province | Not Yet Recruiting |
| ULS de Santo António, EPE - Centro Materno Infantil Norte | Porto | Porto District | Recruiting |
| ULS de Santa Maria,EPE - Hospital de Santa Maria - PPDS | Lisbon | Portugal | Recruiting |
| Hospital Universitario Vall d'Hebron - PPDS | Barcelona | Barcelona | Recruiting |
| Hospital Infantil Universitario Niño Jesus - PIN | Madrid | Madrid | Recruiting |
| Sahlgrenska universitetssjukhuset Östra | Gothenburg | Västra Götaland County | Active Not Recruiting |
| Inselspital - Universitätsspital Bern | Bern | Canton of Bern | Recruiting |
| Cukurova Universitesi Balcali Hastanesi Saglik Uygulama ve Arastirma Merkezi | Adana | Adana | Recruiting |
| Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi | Çankaya | Ankara | Recruiting |
| Ege Universitesi Tip Fakultesi | Bornova | İzmir | Recruiting |
| Great Ormond Street Hospital | London | London | Recruiting |
| University College London Hospitals (UCLH) | London | Middlesex | Not Yet Recruiting |
| Royal Manchester Children's Hospital | Manchester | United Kingdom | Not Yet Recruiting |
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT07054515 on ClinicalTrials.gov ↗ ← All trials in the UK