RecruitingPhase 3NCT07082543
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
Studying Gangliosidosis
Last synced from ClinicalTrials.gov
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Clinical trial records are synced from ClinicalTrials.gov through automated extraction.
Report missing dataKey facts
- Sponsor
- Azafaros A.G.
- Intervention
- AZ-3102(drug)
- Enrollment
- 75 enrolled
- Eligibility
- 4 years · All sexes
- Timeline
- 2025 – 2027
Study locations (30)
- 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
- Royal Children's Hospital Melbourne - PIN, Parkville, Victoria, 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
- Hospital Pequeno Principe, Curitiba, Brazil
- M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary, Calgary, Alberta, Canada
- University of Alberta Medical Genetics Clinic, Edmonton, Alberta, Canada
- Centre Hospitalier de l'Universite de Montreal-1000 rue Saint-Denis, Montreal, Quebec, Canada
- +15 more locations on ClinicalTrials.gov
Primary source
Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.
Open NCT07082543 on ClinicalTrials.govOther trials for Gangliosidosis
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- RECRUITINGNCT00668187A Natural History Study of the GangliosidosesUniversity of Minnesota