Gangliosidoses, GM2 clinical trials
The registry lists 26 studies for Gangliosidoses, GM2 across more than 10 countries. 6 are recruiting or about to start. 10 of them are mainly about Gangliosidoses, GM2 (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-12. Counting its 2 narrower conditions too, such as Tay-Sachs Disease: 37 studies, 9 open.
Get an email when these change. Save the Gangliosidoses, GM2 studies recruiting or about to start — once a week, free: studies that open, close, add sites or post results.
See an example email
Example · made-up studies
Breast cancer · Recruiting: 2 came in, 3 changed
What changed in your saved searches, Oct 31 – Nov 6:
A study of drug A after surgery for early breast cancer
Came in · now Recruiting
Drug B with hormone therapy in advanced breast cancer
Changed
· Sites: 4 added, including Seoul and Busan
· Enrollment: 300 → 450 participants
Exercise during chemotherapy
Now Active, not recruiting
One email a week for all your saved searches. Stop any time.
Who runs them
Azafaros B.V. (6) · University of Minnesota (3) · Children's Hospital of Philadelphia (2) · The Hospital for Sick Children (2) · Terence Flotte (2) · Exsar Corporation (1) · Dr. Anupam Sehgal (1) · Children's National Research Institute (1)
What is being tested here
Drugs under test in Gangliosidoses, GM2 studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 3
- Mar 2027A Natural History Study of the Gangliosidoses · University of Minnesota
- Mar 2027First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis · Dr. Anupam Sehgal
- Jun 2027A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease · Azafaros B.V.
Who these studies take
Of the 5 Gangliosidoses, GM2 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 26 studies, recruiting or not: 31% stop at 75 or younger and 31% at 65; 88% take children; 4% are for women only and 0% for men only; 0% 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 Oct 14, 2026.
Newly registered1
Studies
Recruiting first, then most recently updated. Showing 26 of 26. search them.
- Natural History of Glycosphingolipid Storage Disorders and Glycoprotein DisordersRecruitingNCT000299651 country
- The Myelin Disorders Biorepository ProjectRecruitingNCT030473691 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
- Long-Term Safety and Tolerability of Nizubaglustat in Participants With Rare Lysosomal DisordersNot yet recruitingNCT07885917Phase 2/3
- A Natural History Study of the GangliosidosesRecruitingNCT006681871 country
- Registry GangliosidosesRecruitingNCT046247891 country
- A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C DiseaseActive, not recruitingNCT07399704Phase 21 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
- Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C DiseaseCompletedNCT05758922Phase 21 country
- A Natural History of Late Onset Tay-Sachs DiseaseCompletedNCT028518621 country
- LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for LeukodystrophiesCompletedNCT026991901 country
- Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff DiseaseActive, not recruitingNCT066145691 country
- GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)CompletedNCT051097936 countries
- Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD)TerminatedNCT03822013Phase 31 country
- A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff DiseaseTerminatedNCT04669535Phase 11 country
- N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)CompletedNCT03759665Phase 24 countries
- Impact of Cardiopulmonary Bypass Flow on Renal Oxygenation, Blood Flow and Tubular InjuryCompletedNCT04084301Not applicable1 country
- First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 GangliosidosisActive, not recruitingNCT04798235Phase 1/21 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
- Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage DiseasesWithdrawnNCT01003912Phase 11 country
- Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 GangliosidosisCompletedNCT00418847Phase 21 country
- Gene Therapy for Tay-Sachs DiseaseCompletedNCT018692701 country
- A Phase I Study of Pyrimethamine in Patients With GM2 GangliosidosisWithdrawnNCT00679744Phase 12 countries
- Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)CompletedNCT01102686Phase 1/21 country
- Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral DosesCompletedNCT00672022Phase 31 country