FindTrial
Sign In

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

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

Status
AVAILABLE
Phases
Unknown
Study type
Expanded Access
Source
ClinicalTrials.gov
Registry ID
NCT07675031
Enrollment
Unknown
Registered
2026-06-30
Start date
Unknown
Completion date
Unknown
Last updated
2026-06-30

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Type 3 Gaucher Disease

Brief summary

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.

Detailed description

The program provides access to venglustat, an investigational therapy.

Interventions

DRUGGZ402671

Patients will receive venglustat tablets orally.

Sponsors

Sanofi
Lead SponsorINDUSTRY

Eligibility

Sex/Gender
ALL
Age
12 Years to No maximum

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.

Contacts

CONTACTTrial Transparency email recommended (Toll free for US & Canada)
Contact-US@sanofi.com800-633-1610

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Jul 1, 2026