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NCT05222906 · ClinicalTrials.gov registry record · Phase 3
Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3
A Phase 3 study of Gaucher's Disease Type III, sponsored by Sanofi.
- Active
- Registry status
- Phase 3
- Development phase
- 43
- Enrollment target
- 20
- Study locations
NCT05222906: Active Phase 3 study of Gaucher's Disease Type III, sponsored by Sanofi.
NCT05222906 is a Phase 3 study of Gaucher's Disease Type III that is active but no longer recruiting, run by Sanofi. The registered enrollment target is 43 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (94% lower). The trial reports 20 study locations across 9 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05222906, a Phase 3 study of Gaucher's Disease Type III, is active but no longer recruiting, sponsored by Sanofi.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 43 participants
- Enrollment target
- 20
- Study locations
Study Summary
This is a parallel arm, Phase 3, double-blind, double-dummy, active-comparator, 2 arm study to evaluate the efficacy and safety of daily oral venglustat versus intravenous Cerezyme infusions every two weeks for improvement or stabilization of the neurological manifestations and maintenance of systemic disease stability in participants aged ≥12 and \<18 years and adult patients with Gaucher disease Type 3 (GD3) who have been treated with Enzyme Replacement Therapy (ERT) for at least 3 years.
Conditions Studied
Interventions
- DRUG Venglustat
- DRUG imiglucerase
Study Locations (20)
Other
- Hospital de Ninos - Investigational Site Number: 320001 - Buenos Aires
- Peking Union Medical College Hospital - Investigational Site Number: 1560001 - Beijing
- The First Affiliated Hospital - Investigational Site Number: 1560002 - Guangzhou
- Xinhua Hospital - Investigational Site Number: 1560004 - Shanghai
- 47-87, boulevard de l'hôpital - Investigational Site Number: 2500003 - Paris
- Hopital Necker - Investigational Site Number: 2500001 - Paris
- SphinCS GmbH - Investigational Site Number: 2760001 - Hochheim am Main
- Debreceni Egyetem, Klinikai Központ, Reumatológiai Klinika - Investigational Site Number: 3480001 - Debrecen
- Azienda Ospedaliera Universitaria (AOU) "Federico II" - Investigational Site Number: 3800002 - Naples
- Tohoku University School of Medicine - Investigational Site Number: 3920001 - Sendai
- Cukurova University Medical School Hospital-Investigational Site Number : 7920001 - Adana
- Gazi University Medical Hospital-Investigational Site Number : 7920002 - Ankara
Connecticut
- Yale University School of Medicine - Investigational Site Number: 8400003 - New Haven
Iowa
- University of Iowa - Investigational Site Number: 8400002 - Iowa City
Texas
- Texas Oncology - Medical City Dallas Site Number : 8400008 - Dallas
Virginia
- Lysosomal & Rare Disorders Research & Treatment Center, Inc - Investigational Site Number: 8400001 - Fairfax
Manitoba
- Children's Hospital Research Institute of Manitoba - Investigational Site Number: 1240001 - Winnipeg
Taiwan
- National Taiwan University Hospital-Investigational Site Number: 1580001 - Taipei
Lombardy
- Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico - Investigational Site Number : 3800003 - Milan
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 43 participants |
| Start Date | 2022-04-18 |
| Est. Completion | 2026-10-30 |
| Phase | Phase 3 |
What the registry record for NCT05222906 still lists
NCT05222906 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 43 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (94% lower).
The record links to 1 condition, with Gaucher's Disease Type III appearing as the primary indexed condition, and to 2 interventions - of which Venglustat is the first listed.
NCT05222906 names 20 study sites across 9 states, led by Other, Connecticut, Iowa.
Frequently Asked Questions
What is clinical trial NCT05222906 about?
NCT05222906 is a clinical study titled "Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3". This is a parallel arm, Phase 3, double-blind, double-dummy, active-comparator, 2 arm study to evaluate the efficacy and safety of daily oral venglustat versus intravenous Cerezyme infusions every two weeks for improvement or stabilization of the neurological manifestations and maintenance of system...
What is the current status of trial NCT05222906?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 43 participants. The study started on 2022-04-18. Estimated completion is 2026-10-30.
What conditions does trial NCT05222906 study?
This clinical trial studies the following conditions: Gaucher's Disease Type III.
What interventions are being tested in trial NCT05222906?
The interventions under investigation include: Venglustat (DRUG), imiglucerase (DRUG).
Who is sponsoring clinical trial NCT05222906?
This trial is sponsored by Sanofi, which has 713 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05222906 being conducted?
This trial has 20 study locations across Connecticut, Iowa, Texas, Virginia, Manitoba. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT05222906's enrollment target sits among peer trials
43 1876th of 2000 higher than 124 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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