Gangliosidosis, GM1 clinical trials
The registry lists 20 studies for Gangliosidosis, GM1 across more than 10 countries. 8 are recruiting or about to start. Per ClinicalTrials.gov, last updated 2026-10-21.
Keep track of what changes. Save the Gangliosidosis, GM1 studies recruiting or about to start, and get an email each week — studies that open, close, add sites or post results.
Watch these studiesStatus
Who runs them
Azafaros B.V. (4) · Masonic Cancer Center, University of Minnesota (2) · LYSOGENE (2) · University of Minnesota (2) · xCures (1) · Idorsia Pharmaceuticals Ltd. (1) · Sun Yat-sen University (1) · Gemma Biotherapeutics (1)
Who these studies take
Of the 5 Gangliosidosis, GM1 studies recruiting now, 4 (80%) set no upper age limit and 0 (0%) stop at 75 or younger. 5 (100%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 0 (0%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 5 (100%) | 5 (100%) |
| 18 | 5 (100%) | 5 (100%) |
| 30 | 5 (100%) | 5 (100%) |
| 40 | 5 (100%) | 5 (100%) |
| 50 | 5 (100%) | 5 (100%) |
| 65 | 5 (100%) | 5 (100%) |
| 75 | 5 (100%) | 5 (100%) |
| 85 | 5 (100%) | 5 (100%) |
Across all 20 studies, recruiting or not: 35% stop at 75 or younger and 25% at 65; 95% take children; 0% are for women only and 0% for men only; 5% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
What changed recently
From the registry's own updates since Sep 26, 2026.
Newly registered1
Studies
Recruiting first, then most recently updated. Showing 20 of 20. search them.
- Long-Term Safety and Tolerability of Nizubaglustat in Participants With Rare Lysosomal DisordersNot yet recruitingNCT07885917Phase 2/3
- Natural History of Glycosphingolipid Storage Disorders and Glycoprotein DisordersRecruitingNCT000299651 country
- 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 GangliosidosisRecruitingNCT07054515Phase 317 countries
- Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis Clinical TrialNot yet recruitingNCT07479953Phase 1
- ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening ProgramEnrolling by invitationNCT053680381 country
- A Natural History Study of the GangliosidosesRecruitingNCT006681871 country
- FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare DiseasesRecruitingNCT065391691 country
- Registry GangliosidosesRecruitingNCT046247891 country
- A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 GangliosidosisActive, not recruitingNCT07082543Phase 314 countries
- Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 GangliosidosisActive, not recruitingNCT04713475Phase 1/24 countries
- Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) PatientsCompletedNCT040411025 countries
- GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)CompletedNCT051097936 countries
- GM1 Prophylaxis for WBRT Related Cognitive DysfunctionTerminatedNCT04395339Phase 31 country
- A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 GangliosidosisTerminatedNCT04273269Phase 1/23 countries
- Interviews and Video Capture in Patients With GM1 GangliosidosisCompletedNCT043101631 country
- Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2CompletedNCT0447071310 countries
- Synergistic Enteral Regimen for Treatment of the GangliosidosesTerminatedNCT02030015Phase 41 country
- Natural History of Morquio B and Late-Onset of GM1 GangliosidosisStatus unknownNCT043203291 country
- HSCT for High Risk Inherited Inborn ErrorsCompletedNCT00383448Phase 21 country
- Stem Cell Transplant for Inborn Errors of MetabolismCompletedNCT00176904Phase 2/31 country