RecruitingPhase 3NCT07082725
Rare condition
GM1 gangliosidosis type 3
2
studies recruiting now
as of 7 Sept 2026
4
studies registered in total
as of 7 Sept 2026
17
countries with a recruiting site
as of 7 Sept 2026
24 Jul 2025
most recent study posted
among recruiting studies
Recruiting trials
RecruitingPhase 3NCT07054515
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis
Showing the 2 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.
Search all GM1 gangliosidosis type 3 studiesWhere recruiting studies are running
Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.
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Patient organisations
We do not yet list a dedicated organisation for this condition. The directories below are the best route.
About GM1 gangliosidosis type 3
RareTrial does not yet hold a plain-language description of this condition. The most reliable starting point is Orphanet’s expert page, which lists specialist centres, registries and patient organisations, and the gene involved (GLB1).
Treatments being studied
5 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.
Phase 2/3Cyclophosphamide (Cyclophosphamide)
Phase 2/3Busulfan (Busilvex)
Phase 1/2Lixmabegagene Relduparvovec
Phase 1/2Bomtabegagene Bavoparvovec
PRECLINICALDeoxygalactonojirimycin
Related conditions
Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).