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

June 22, 2026 updated by: Sanofi
The objective of this program is to provide early access to venglustat for certain patients with Gaucher disease type 3 in response to unsolicited requests from healthcare providers. No approved therapies for the CNS manifestations of the disease are currently available. The program will provide access to venglustat prior to registration and the availability of commercial product (including reimbursement where applicable) in the country of the patient.

Study Overview

Status

Available

Conditions

Intervention / Treatment

Detailed Description

The program provides access to venglustat, an investigational therapy.

Study Type

Expanded Access

Expanded Access Type

  • Treatment IND/Protocol

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

N/A

Description

Inclusion criteria:

  • ≥ 12 years of age
  • Clinical diagnosis of GD3 with at least 1 CNS manifestation
  • Body weight ≥15 kg
  • On a stable ERT dose for at least 6 months
  • Clinically stable with respect to hematologic manifestations
  • Clinically stable with respect to spleen and liver volume
  • If the patient has a history of seizures, they must be well controlled without use of medications that are strong/moderate inducers or strong/moderate inhibitors of CYP3A
  • Documented negative pregnancy test
  • Contraceptive use consistent with local regulations.

Exclusion criteria:

  • Inability to safely swallow (or chew and swallow) a venglustat tablet
  • Use of any investigational drugs within the last 30 days or 5 half-lives or gene therapy at any time
  • Current use of any unapproved therapy for GD
  • Pregnant or breastfeeding females
  • Drug contraindications
  • History of major organ transplant (eg, bone marrow or liver)
  • History of drug and/or alcohol abuse within the last year
  • Patients with severe depression and/or a history of a major affective disorder in the past year
  • Patients with suicidal ideation or behavior prior to study.

The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sanofi

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

April 7, 2026

First Submitted That Met QC Criteria

June 22, 2026

First Posted (Actual)

June 30, 2026

Study Record Updates

Last Update Posted (Actual)

June 30, 2026

Last Update Submitted That Met QC Criteria

June 22, 2026

Last Verified

June 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SCP25092

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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