Type 3 Gaucher DiseaseNCT07675031
Managed Access Program of Venglustat in Pediatric and Adult Patients With Type 3 Gaucher Disease (GD3).
A study testing GZ402671 in people with Type 3 Gaucher Disease, run by Sanofi.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Type 3 Gaucher Disease
- What is being tested?
- GZ402671
- Type of treatment
- Pill or oral medicine
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- ≥ 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
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- 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.
Who cannot join
- 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)
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- 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.
Trial locations and contacts
No sites are listed yet.
Trial Transparency email recommended (Toll free for US & Canada) contact
About this study
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.
Read the detailed description
The program provides access to venglustat, an investigational therapy.
- Conditions
- Type 3 Gaucher Disease
- Being tested
- GZ402671
- Medical subject headings
- Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GZ402671Drug
Patients will receive venglustat tablets orally.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jun 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Type 3 Gaucher DiseaseStudies of the same condition, open and under way.
- More trials run by SanofiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Sanofi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07675031
- Sponsor's protocol ID
- SCP25092
- Status last verified
- Jun 2026
- First submitted
- 7 Apr 2026
- Last updated
- 30 Jun 2026
- Results posted
- No