Clinical trial
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
NCT07054515 is registered in ClinicalTrials.gov with Azafaros B.V. as lead sponsor.
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Registry record
As filed in ClinicalTrials.gov.
- Title
- 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
- Lead sponsor
- Azafaros B.V.
- Phase
- Phase 3
- Overall status
- Recruiting
- Start date
- June 30, 2025
- Completion date
- June 14, 2028
- Enrollment
- 147 (estimated)
- Sites
- 39
Conditions
As the registry lists them; a condition with its own page is linked.
gm1 gangliosidosis, gm2 gangliosidosis, niemann-pick type c disease