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A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis
Condition: Gangliosidoses, GM2 · Gangliosidosis, GM1 · Sponsor: Azafaros B.V.
About this study
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
This description comes directly from the study's public registry record.
Talk to the study team
No contact information is available for this study as per the public registry record.
Always discuss trial participation with your own doctor first.
Locations (26)
| UCSF Children's Hospital and Research Center at Oakland | Oakland, California, United States | — |
| University of Minnesota Medical School | Minneapolis, Minnesota, United States | — |
| Mayo Clinic Children's Center - PIN | Rochester, Minnesota, United States | — |
| Children's Medical Center Dallas | Dallas, Texas, United States | — |
| Lysosomal Rare Disorders Research and Treatment Center | Fairfax, Virginia, United States | — |
| Hospital Universitario Austral | Ciudad Autónoma Buenos Aires, Buenos Aires, Argentina | — |
| Hospital de Niños de La Santisima Trinidad | Córdoba, Córdoba Province, Argentina | — |
| Women's and Children's Hospital | North Adelaide, South Australia, Australia | — |
| Instituto Fernandes Figueira | Rio de Janeiro, Rio de Janeiro, Brazil | — |
| Hospital de Clinicas de Porto Alegre (HCPA) - PPDS | Porto Alegre, Rio Grande do Sul, Brazil | — |
| M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary | Calgary, Alberta, Canada | — |
| AP-HP - Hôpital Armand Trousseau | Paris, France | — |
+ 14 more locations — full list on the registry record.
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Source record: clinicaltrials.gov/study/NCT07082543