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NCT02843035 · ClinicalTrials.gov registry record · Phase 2
Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension
A Phase 2 study of Gaucher Disease, Type 1 and Gaucher Disease, Type 3, sponsored by Genzyme, a Sanofi Company.
- Active
- Registry status
- Phase 2
- Development phase
- 12
- Enrollment target
- 7
- Study locations
NCT02843035 is a Phase 2 study of Gaucher Disease, Type 1 and Gaucher Disease, Type 3 that is active but no longer recruiting, run by Genzyme, a Sanofi Company. The registered enrollment target is 12 participants, below the 58-participant average among 8 other Gaucher Disease, Type 1 trials with a reported enrollment target (79% lower). The trial reports 7 study locations across 7 states.
The verdict
NCT02843035, a Phase 2 study of Gaucher Disease, Type 1 and Gaucher Disease, Type 3, is active but no longer recruiting, sponsored by Genzyme, a Sanofi Company.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 2
- Development phase
- 12 participants
- Enrollment target
- 7
- Study locations
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 |
Interested in This Trial?
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, categorized as Phase 2. The registered enrollment target is 12 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 58-participant average among 8 other Gaucher Disease, Type 1 trials with a reported enrollment target (79% lower). The listed sponsor is Genzyme, a Sanofi Company, which has 196 total studies on file at ClinicalTrials.gov.
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.
NCT02843035 reports 7 study locations spanning 7 distinct geographic areas - top geographies include Connecticut, Texas, Virginia.
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.
What interventions are being tested in trial NCT02843035?
The interventions under investigation include: imiglucerase (DRUG), venglustat (GZ402671) (DRUG).
Who is sponsoring clinical trial NCT02843035?
This trial is sponsored by Genzyme, a Sanofi Company, which has 196 total clinical trials registered on ClinicalTrials.gov.
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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