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NCT04221451 · ClinicalTrials.gov registry record · Phase 3
A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2
A Phase 3 study, sponsored by Genzyme, a Sanofi Company.
- Terminated
- Registry status
- Phase 3
- Development phase
- 75
- Enrollment target
NCT04221451 is a Phase 3 study that was terminated before completion, run by Genzyme, a Sanofi Company. The registered enrollment target is 75 participants.
The verdict
NCT04221451, a Phase 3 study, was terminated before completion, sponsored by Genzyme, a Sanofi Company.
- TERMINATED
- Registry status
- Phase 3
- Development phase
- 75 participants
- Enrollment target
Study Summary
Primary Objectives: Primary population (adult participants with late-onset GM2 gangliosidosis): To assess the efficacy and pharmacodynamics (PD) of daily oral dosing of venglustat when administered over a 104-week period Secondary population (participants with juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1 or juvenile/adult galactosialidosis): To assess PD response (plasma and CSF GL-1 biomarker and disease specific biomarkers) of venglustat when administered once daily over a 104-week period Secondary Objectives: Primary population: * To assess the PD of daily oral dosing of venglustat and the effect of venglustat on selected performance test and scale over a 104-week period * To determine the safety and tolerability of venglustat when administered orally once daily over a 104-week period * To assess the pharmacokinetics (PK) of venglustat in plasma and cerebrospinal fluid (CSF) Secondary population: * To assess the effect of venglustat on selected performance tests and scale over a 104-week period * To determine the safety and tolerability of venglustat when administered once daily over a 104-week period * To assess the PK of venglustat in plasma and CSF * To assess the acceptability and palatability of the venglustat tablet
Interventions
- DRUG placebo
- DRUG venglustat GZ402671
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 75 participants |
| Start Date | 2020-06-29 |
| Est. Completion | 2024-12-26 |
| Phase | Phase 3 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04221451
The ClinicalTrials.gov registry entry for NCT04221451 describes a study currently listed as terminated, categorized as Phase 3. The registered enrollment target is 75 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Genzyme, a Sanofi Company, which has 196 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 2 interventions - of which placebo is the first listed.
NCT04221451 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT04221451 about?
NCT04221451 is a clinical study titled "A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2". Primary Objectives: Primary population (adult participants with late-onset GM2 gangliosidosis): To assess the efficacy and pharmacodynamics (PD) of daily oral dosing of venglustat when administered over a 104-week period Secondary population (participants with juvenile/adolescent late-onset GM2 ga...
What is the current status of trial NCT04221451?
This trial is currently terminated. It is a Phase 3 study. The enrollment target is 75 participants. The study started on 2020-06-29. Estimated completion is 2024-12-26.
What interventions are being tested in trial NCT04221451?
The interventions under investigation include: placebo (DRUG), venglustat GZ402671 (DRUG).
Who is sponsoring clinical trial NCT04221451?
This trial is sponsored by Genzyme, a Sanofi Company, which has 196 total clinical trials registered on ClinicalTrials.gov.
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