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About this data Sourced primarily from ClinicalTrials.gov — a listing isn't proof of safety or FDA approval.

Pipeline and clinical trial information here comes primarily from ClinicalTrials.gov. A listing on ClinicalTrials.gov means a study has been registered — not that a treatment is proven safe, effective, or FDA-approved. Investigational treatments may never be approved. Who can join a trial is decided only by that trial's own study team.

Not yet reported

Venglustat

Company
Sanofi
Mechanism
Mechanism not yet reported
Route
Route not yet reported
Recruiting trials
None currently listed

Efficacy results: Not yet reported

No recruiting studies currently listed

See all studies for this drug

Data source: ClinicalTrials.gov · trial data as of Oct 14, 2026

Trial details
  • Completed NCT NCT01674036

    Safety, Tolerability and Pharmacokinetics of Genz-682452 in Healthy Men

    Not yet reported Genzyme, a Sanofi Company 63 participants Est. completion 2013-01 1 site
    Other conditions: Healthy Volunteer
    View official trial listing →
  • Completed NCT NCT01710826

    A Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Genz-682452 in Healthy Volunteers

    Not yet reported Genzyme, a Sanofi Company 36 participants Est. completion 2013-02 1 site
    Other conditions: Healthy
    View official trial listing →
  • Completed NCT NCT02228460

    Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-naïve Adult Male Patients With Fabry Disease

    Not yet reported Genzyme, a Sanofi Company 11 participants Est. completion 2016-09 8 sites
    Other conditions: Fabry Disease
    View official trial listing →
  • Completed NCT NCT02489344

    Evaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Naïve Adult Male Patients With Fabry Disease

    Not yet reported Genzyme, a Sanofi Company 8 participants Est. completion 2018-11-20 7 sites
    Other conditions: Fabry Disease
    View official trial listing →
  • Active, not recruiting NCT NCT02843035

    Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension

    Not yet reported Genzyme, a Sanofi Company 12 participants Est. completion 2026-10-30 7 sites Stand-alone
    Other conditions: Gaucher Disease Type 1, Gaucher Disease Type 3
    View official trial listing →
  • Terminated NCT NCT02906020

    A Global Study to Assess the Drug Dynamics, Efficacy, and Safety of Venglustat (GZ/SAR402671) in Parkinson's Disease Patients Carrying a Glucocerebrosidase (GBA) Gene Mutation

    Not yet reported Genzyme, a Sanofi Company 273 participants Est. completion 2021-05-27 52 sites Stand-alone
    Other conditions: Parkinson's Disease
    View official trial listing →
  • Terminated NCT NCT03523728

    A Medical Research Study Designed to Determine if Venglustat Can be a Future Treatment for ADPKD Patients

    Not yet reported, Not yet reported Genzyme, a Sanofi Company 478 participants Est. completion 2021-08-03 95 sites Stand-alone
    Other conditions: Polycystic Kidney, Autosomal Dominant
    View official trial listing →
  • Completed NCT NCT03687554

    Effect of Venglustat in Patients With Renal Impairment

    Not yet reported Genzyme, a Sanofi Company 24 participants Est. completion 2019-02-27 1 site Stand-alone
    Other conditions: Healthy Volunteers, Polycystic Kidney, Autosomal Dominant
    View official trial listing →
  • Terminated NCT NCT04221451

    A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2

    Not yet reported Genzyme, a Sanofi Company 75 participants Est. completion 2024-12-26 23 sites Stand-alone
    Other conditions: Tay-Sachs Disease, Sandhoff Disease
    View official trial listing →
  • Terminated NCT NCT04705051

    Long-term Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD) With Venglustat

    Not yet reported Sanofi 24 participants Est. completion 2021-07-13 11 sites Stand-alone
    Other conditions: Congenital Cystic Kidney Disease
    View official trial listing →
  • Active, not recruiting NCT NCT05206773

    A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease

    Not yet reported Sanofi 122 participants Est. completion 2027-01-04 50 sites Stand-alone
    Other conditions: Fabry Disease
    View official trial listing →
  • Active, not recruiting NCT NCT05222906

    Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3

    Not yet reported Sanofi 43 participants Est. completion 2026-10-30 22 sites Stand-alone
    Other conditions: Gaucher's Disease Type III
    View official trial listing →
  • Completed NCT NCT05238714

    Excretion Balance, Pharmacokinetics, and Metabolism Following of [14C]-Venglustat Administration in Healthy Male Subjects

    Not yet reported Genzyme, a Sanofi Company 6 participants Est. completion 2020-06-26 1 site Stand-alone
    Other conditions: Disorders of Sphingolipid Metabolism, Healthy Volunteers
    View official trial listing →
  • Active, not recruiting NCT NCT05280548

    A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease

    Not yet reported Sanofi 104 participants Est. completion 2027-12-06 54 sites Stand-alone
    Other conditions: Fabry Disease
    View official trial listing →
  • Completed NCT NCT05718258

    A Study in Adults to Investigate the Impact of Mild, Moderate, and Severe Hepatic Impairment on Pharmacokinetics of Venglustat Compared to Participants With Normal Hepatic Function

    Not yet reported Sanofi 26 participants Est. completion 2024-01-29 6 sites Stand-alone
    Other conditions: Hepatic Function Abnormal
    View official trial listing →
  • Completed NCT NCT06418607

    A Study of the Safety, Tolerability, and Bioequivalence of Orally Administered Venglustat in Healthy Adult Participants

    Not yet reported Sanofi 65 participants Est. completion 2020-07-13 2 sites Stand-alone
    Other conditions: Healthy Volunteers
    View official trial listing →
  • Completed NCT NCT06418620

    A Study of the Safety, Tolerability, and Bioavailability of Orally Administered Venglustat in Healthy Adult Participants

    Not yet reported Sanofi 9 participants Est. completion 2020-08-07 1 site Stand-alone
    Other conditions: Healthy Volunteers
    View official trial listing →
  • Completed NCT NCT06421714

    A Study of the Safety, Tolerability, and Pharmacokinetics of Orally Administered Venglustat and Itraconazole in Healthy Adult Male Participants

    Not yet reported Genzyme, a Sanofi Company 8 participants Est. completion 2018-10-14 1 site Stand-alone
    Other conditions: Healthy Volunteers
    View official trial listing →
  • Available NCT NCT07675031

    Managed Access Program of Venglustat in Pediatric and Adult Patients With Type 3 Gaucher Disease (GD3).

    Sanofi
    Other conditions: Type 3 Gaucher Disease
    View official trial listing →
Efficacy history

Not yet reported.

Safety

Not yet reported.

Sources

Also known as: Venglustat, Venglustat (GZ402671)

Related On The Pen coverage

No related coverage yet.

This drug may be investigational and not yet approved for any use. Whether someone can join a clinical trial is determined only by that trial's own study team — On The Pen does not determine eligibility. Information here can change; always confirm current details on the official ClinicalTrials.gov listing or with the sponsor.