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Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3
NCT05222906 · View on ClinicalTrials.gov ↗
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05222906
The ClinicalTrials.gov registry entry for NCT05222906 describes a study currently listed as active not recruiting. It is categorized as Phase 3, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 43 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Sanofi, which has 275 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT05222906 reports 20 study locations spanning 9 distinct geographic areas — top geographies include Other, Connecticut, Iowa. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT05222906?
The interventions under investigation include: Venglustat (DRUG), imiglucerase (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT05222906?
This trial is sponsored by Sanofi, which has 275 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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.
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