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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: Clinical Trial Phase 3 study, sponsored by Genzyme, a Sanofi Company.
NCT04221451 is a Phase 3 study that was terminated before completion, run by Genzyme, a Sanofi Company. The registered enrollment target is 75 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (90% lower). According to ClinicalTrials.gov, the official US trial registry.
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
Primary Outcome
Lumbar puncture was performed for obtaining CSF samples at specified timepoints to assess the presence of GM2 biomarker in PP in PAP. The baseline value was defined as the last available value before or equal to the first dose of study drug date in the PAP.
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 |
Why NCT04221451 stopped before completion
NCT04221451 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 75 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (90% lower).
The record links to 0 conditions, and to 2 interventions - of which placebo is the first listed.
NCT04221451 does not publish any study locations in the registry export this page uses.
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.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT04221451's enrollment target sits among peer trials
75 1773rd of 2000 higher than 221 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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