Venglustat vs Imiglucerase for Gaucher Disease
(LEAP2MONO Trial)

Recruiting in Palo Alto (17 mi)

+48 other locations

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Genzyme, a Sanofi Company

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?This trial compares a pill to injections in patients aged 12-18 and adults with Gaucher disease Type 3. The pill aims to help with brain symptoms, while the injections maintain overall health. The pill has the potential to improve Gaucher disease by rebalancing certain substances in the body.

Eligibility Criteria

This trial is for adults and children aged ≥12 with Gaucher Disease Type 3 (GD3) who have been on Enzyme Replacement Therapy for at least 3 years. They must not be pregnant, breastfeeding, or planning to donate sperm; should weigh over 30 kg; and have controlled seizures if present. Exclusions include those needing ventilatory support, with allergies to study drugs, recent use of certain CYP3A affecting substances including grapefruit products, history of cancer (except basal cell carcinoma), planned hospitalizations, significant other diseases or conditions that could interfere with the study.

Inclusion Criteria

Signed written informed assent/consent

My balance and coordination are affected.

I am 18 years old or older.

+7 more

Exclusion Criteria

Participant not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures

My liver function tests are not more than twice the normal limit, unless I have Gilbert Syndrome.

My kidney function is low, with a filtration rate under 30 mL/min.

+16 more

Participant Groups

The trial compares the effectiveness and safety of an oral drug called Venglustat against intravenous infusions of Imiglucerase (Cerezyme). It's designed as a Phase 3 study where participants are randomly assigned to one of two groups: one taking Venglustat daily by mouth and the other receiving Cerezyme injections every two weeks. The goal is to see which treatment better improves or stabilizes neurological symptoms while maintaining overall health in GD3 patients.

2Treatment groups

Experimental Treatment

Active Control

Group I: VenglustatExperimental Treatment1 Intervention

Venglustat

Group II: CerezymeActive Control1 Intervention

Cerezyme