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Medical Condition
gm1 gangliosidosis
Total Trials
2
Recruiting Now
2
Trial Phases
Phase 3
ClinicalMetric tracks all active clinical trials for gm1 gangliosidosis sourced from ClinicalTrials.gov. Each listing includes the NCT identifier, current recruitment status, phase, primary endpoints, sponsor, estimated enrollment, and participating countries — updated daily as new studies open and status changes.
- Recruiting trials currently enrolling participants, with eligibility criteria and age requirements
- Phase 1–4 studies from early safety trials to post-marketing surveillance
- NIH, pharmaceutical sponsor, and academic medical center studies worldwide
- AI-powered plain-language summary available for every trial listing
Frequently Asked Questions — gm1 gangliosidosis Clinical Trials
How many clinical trials are currently recruiting for gm1 gangliosidosis?
ClinicalMetric currently tracks 2 actively recruiting clinical trials for gm1 gangliosidosis, sourced in real time from ClinicalTrials.gov. The total number of registered studies—including those not yet enrolling or in active follow-up—is 2. Trial availability changes daily as new studies open enrollment and existing ones reach capacity.
What trial phases are available for gm1 gangliosidosis?
gm1 gangliosidosis research spans Phase 3 (1 trial). Phase 1 studies evaluate safety and dosing in small groups, Phase 2 studies assess preliminary efficacy in 100–300 participants, and Phase 3 trials compare the new treatment against the standard of care in 300–3,000+ patients. Phase 4 post-approval studies monitor long-term outcomes in real-world populations.
How do I find out if I qualify for a gm1 gangliosidosis clinical trial?
Eligibility criteria for gm1 gangliosidosis trials vary by study and typically specify age range, disease stage or severity, prior treatment history, and specific diagnostic or laboratory parameters. Each listing on ClinicalMetric links to the full protocol on ClinicalTrials.gov, where inclusion and exclusion criteria are documented. Contact the sponsoring site's research coordinator directly to confirm your eligibility—your treating physician or specialist can also help identify the most appropriate trial based on your medical history and current treatment status.
Trial Phases
Phase 3 1
Top Sponsors
1 trial
1 trial
Recruiting Clinical Trials
NCT06539169
Recruiting
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
Enrollment
1,000 pts
Location
United States
Sponsor
xCures
NCT07054515 Phase 3
Recruiting
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
Enrollment
147 pts
Location
United States, Argen...
Sponsor
Azafaros A.G.
ClinicalMetric — Independent clinical trial intelligence platform. Not affiliated with NIH, ClinicalTrials.gov, the U.S. FDA, or any pharmaceutical company, hospital, or clinical research organization. Trial data is sourced from ClinicalTrials.gov for informational purposes only and does not constitute medical advice. Do not make any treatment, enrollment, or health decisions based solely on information found here — always consult a qualified healthcare professional. Full Disclaimer · Last Reviewed: April 2026 · Data Methodology