Managed Access Program of Venglustat in Pediatric and Adult Patients With Type 3 Gaucher Disease (GD3).
ConditionType 3 Gaucher Disease
Trial statusAvailable
Trial phaseNot listed
Trial typeExpanded Access
Biological sexAll
Age12+
SponsorSanofi
About this trial
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.
Eligibility criteria
Qualifiers
≥ 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
Disqualifiers
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
Trial design
Treatments tested in this trial
- GZ402671
Treatment groups
No treatment groups listed
Locations
Sponsors and collaborators
Source ClinicalTrials.gov