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Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension
NCT02843035 · View on ClinicalTrials.gov ↗
Study Summary
Part 1: Biomarker evaluation/screening phase Primary Objectives: * Evaluate cerebrospinal fluid (CSF) biomarkers in adult Gaucher disease Type 3 (GD3) participants that distinguish GD3 from adult Gaucher disease Type 1 (GD1) participants * Screen adult GD3 participants who qualify for treatment with venglustat in Parts 2, Part 3, and Part 4 Parts 2 and 3: Combination treatment phases Primary objectives: * Evaluate short-term (Part 2) and long-term (Part 3) safety and tolerability of venglustat in combination with Cerezyme in adult GD3 participants * Evaluate the change in CSF central nervous system (CNS) biomarkers (glucosylceramide \[GL-1\] and lyso-glucosylceramide \[lyso-GL-1\]) from adult GD3 participants receiving venglustat in combination with Cerezyme (Part 2 only) Part 4: Extended treatment phase with monotherapy Primary objectives: • Evaluate safety and tolerability of venglustat monotherapy in adult GD3 participants who have remained systemically stable on venglustat in combination with Cerezyme Parts 2 and 3: Combination treatment phases Secondary Objectives: * Evaluate the pharmacokinetics (PK) of venglustat in adult GD3 participants * Evaluate the efficacy of venglustat in combination with Cerezyme in systemic disease in adult GD3 participants by assessing spleen volume, liver volume, hemoglobin level and platelet count * Evaluate the efficacy of venglustat in combination with Cerezyme on neurological function in adult GD3 participants by assessing Ataxia using the Scale for the Assessment and Rating of Ataxia (SARA) * Evaluate plasma biomarkers (lyso-GL-1 and GL-1) in adult GD3 participants Part 4: Extended treatment phase with monotherapy Secondary objectives: * Evaluate the efficacy of venglustat in systemic disease in adult GD3 participants by assessing spleen volume, liver volume, hemoglobin level and platelet count * Evaluate the efficacy of venglustat on neurological function in adult GD3 participants by assessing Ataxia using the Scale
Conditions Studied
Interventions
- DRUG imiglucerase
- DRUG venglustat (GZ402671)
Study Locations (7)
Connecticut
- Yale University School of Medicine Site Number : 840002 — New Haven
Texas
- Baylor Institute of Metabolic Diseases Site Number : 840001 — Dallas
Virginia
- Lysosomal and Rare Disorders Research and Treatment Center, Inc Site Number : 840003 — Fairfax
Other
- Investigational Site Number : 276001 — Mainz
Tokyo
- Investigational Site Number : 392001 — Minato-ku
Cambridgeshire
- Investigational Site Number : 826003 — Cambridge
Manchester
- Investigational Site Number : 826002 — Salford
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 12 participants |
| Start Date | 2017-01-04 |
| Est. Completion | 2026-10-30 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT02843035
The ClinicalTrials.gov registry entry for NCT02843035 describes a study currently listed as active not recruiting. It is categorized as Phase 2, 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 12 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 52 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 2 conditions, with Gaucher Disease Type 1 appearing as the primary indexed condition, and to 2 interventions — of which imiglucerase 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: NCT02843035 reports 7 study locations spanning 7 distinct geographic areas — top geographies include Connecticut, Texas, Virginia. 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 NCT02843035 about?
NCT02843035 is a clinical study titled "Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension". Part 1: Biomarker evaluation/screening phase Primary Objectives: * Evaluate cerebrospinal fluid (CSF) biomarkers in adult Gaucher disease Type 3 (GD3) participants that distinguish GD3 from adult Gaucher disease Type 1 (GD1) participants * Screen adult GD3 participants who qualify for treatment wi...
What is the current status of trial NCT02843035?
This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 12 participants. The study started on 2017-01-04. Estimated completion is 2026-10-30.
What conditions does trial NCT02843035 study?
This clinical trial studies the following conditions: Gaucher Disease Type 1, Gaucher Disease Type 3. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT02843035?
The interventions under investigation include: imiglucerase (DRUG), venglustat (GZ402671) (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT02843035?
This trial is sponsored by Genzyme, a Sanofi Company, which has 52 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT02843035 being conducted?
This trial has 7 study locations across Connecticut, Texas, Virginia, Tokyo, Cambridgeshire. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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