Condition: Niemann-Pick Type C Disease · GM1 Gangliosidosis · GM2 Gangliosidosis · Sponsor: Azafaros B.V.
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
This description comes directly from the study's public registry record.
Patient Advocacy Representative · Please reach out by email · info@azafaros.com
Contact for Healthcare Professionals · Please reach out by email · medinfo@azafaros.com
Always discuss trial participation with your own doctor first.
| UCSF Children's Hospital and Research Center at Oakland | Oakland, California, United States | Recruiting |
| University of Minnesota Medical School | Minneapolis, Minnesota, United States | Not Yet Recruiting |
| Mayo Clinic Childrens Center - PIN | Rochester, Minnesota, United States | Recruiting |
| Children's Medical Center Dallas | Dallas, Texas, United States | Recruiting |
| Lysosomal Rare Disorders Research and Treatment Center | Fairfax, Virginia, United States | Recruiting |
| Hospital Universitario Austral | Ciudad Autónoma Buenos Aires, Buenos Aires, Argentina | Recruiting |
| Hospital de Niños de La Santisima Trinidad | Córdoba, Córdoba Province, Argentina | Recruiting |
| Women's and Children's Hospital | North Adelaide, South Australia, Australia | Recruiting |
| Royal Melbourne Hospital | Parkville, Victoria, Australia | Recruiting |
| Royal Children's Hospital Melbourne - PIN | Parkville, Victoria, Australia | Not Yet Recruiting |
| Instituto Fernandes Figueira | Rio de Janeiro, Rio de Janeiro, Brazil | Recruiting |
| Hospital de Clinicas de Porto Alegre (HCPA) - PPDS | Porto Alegre, Rio Grande do Sul, Brazil | Recruiting |
+ 26 more locations — full list on the registry record.
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Source record: clinicaltrials.gov/study/NCT07054515