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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: Active Phase 2 study of Gaucher Disease, Type 1 and Gaucher Disease, Type 3, sponsored by Genzyme, a Sanofi Company.

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. According to ClinicalTrials.gov, the official US trial registry.

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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

Gaucher Disease, Type 1 Gaucher Disease, Type 3

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

FieldValue
Enrollment Target 12 participants
Start Date 2017-01-04
Est. Completion 2026-10-30
Phase Phase 2
Genzyme, a Sanofi Company

196 total trials

What the registry record for NCT02843035 still lists

NCT02843035 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 12 participants, a relatively small participant target, below the 58-participant average among 8 other Gaucher Disease, Type 1 trials with a reported enrollment target (79% lower).

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 lists 7 locations in 7 states (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.

Learn More About Clinical Trials

Similar trials for Gaucher Disease, Type 1

Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.

Where NCT02843035's enrollment target sits among peer trials

12 9th of 8 the lowest of 8 other Gaucher Disease, Type 1 trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Gaucher Disease, Type 1 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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Related registry views

Source: ClinicalTrials.gov NCT02843035, the US trial registry maintained by the National Library of Medicine. NCT02843035 (small enrollment · multi site footprint · active not recruiting) retrieved and formatted by PlainTrial, see methodology.