A Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, Safety, and Tolerability of Venglustat in Late-onset GM2 Gangliosidosis (Tay-Sachs Disease and Sandhoff Disease) Together With a Separate Basket for Juvenile/Adolescent Late-onset GM2 Gangliosidosis and Ultra-rare Diseases Within the Same and Similar Glucosylceramide-based Sphingolipid Pathway Conditions - Join Clinical Trial NCTNCT04221451
How to Join This Clinical Trial - NCTNCT04221451
Learn how to participate in this PHASE3 trial studying venglustat (GZ402671) for Late-onset GM2 gangliosidosis, Tay-Sachs disease, Sandhoff disease, GM2 gangliosidosis. Current status: TERMINATED.
Am I Eligible for This Clinical Trial?
This clinical research study is looking for participants with Late-onset GM2 gangliosidosis, Tay-Sachs disease, Sandhoff disease, GM2 gangliosidosis. Eligibility requirements include specific age ranges, health status, and medical history. Contact the study team to learn if you qualify.
- Condition Being Studied
- Late-onset GM2 gangliosidosis, Tay-Sachs disease, Sandhoff disease, GM2 gangliosidosis
- Treatment Being Tested
- venglustat (GZ402671)
- Study Phase
- PHASE3 - Large-scale efficacy study
- Enrollment Status
- TERMINATED
- Study Identifier
- NCTNCT04221451 - ClinicalTrials.gov Identifier
- Sponsored By
- Study sponsor
What to Expect as a Participant
Participating in this clinical trial involves:
- Initial screening to determine eligibility
- Regular study visits and health assessments
- Receiving the study treatment or placebo
- Medical monitoring and follow-up care
- Contributing to medical research that may help others
- Potential access to new treatments before they're widely available
Clinical Trial Benefits and Compensation
Participants in this clinical research study may receive:
- Close medical monitoring by healthcare professionals
- Access to potential new treatments
- Compensation for time and travel (varies by study)
- No-cost study-related medical care
- The opportunity to help advance medical knowledge
Where Is This Clinical Trial Located?
This study is enrolling participants at multiple locations. Find a study site near you:
How to Enroll in This Study
To learn more about participating in this PHASE3 clinical trial for Late-onset GM2 gangliosidosis, Tay-Sachs disease, Sandhoff disease, GM2 gangliosidosis:
- Review the eligibility criteria with your healthcare provider
- Contact the study team for a pre-screening interview
- Schedule an in-person screening visit if eligible
- Review and sign the informed consent form
- Begin participation in the clinical trial
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Quri.ai is a free clinical trial matching service that helps patients connect with research sites. We make it easier to:
- Find clinical trials that match your condition
- Get matched to the right study location near you
- Connect directly with study coordinators
- Understand if you qualify with simplified eligibility screening
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- Access new treatments through clinical research participation