Managed Access Program of Venglustat in Pediatric and Adult Patients With Type 3 Gaucher Disease … (NCT07675031) | Clinical Trial Compass
AVAILABLENot Applicable
Managed Access Program of Venglustat in Pediatric and Adult Patients With Type 3 Gaucher Disease (GD3).
Plain-language 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.
Who can participate
Age range
12 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Trial details
NCT IDNCT07675031
SponsorSanofi
Sponsor typeINDUSTRY
Study typeEXPANDED_ACCESS
Contact for this trial
Trial Transparency email recommended (Toll free for US & Canada)