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Enzyme Replacement Therapy

Venglustat + Cerezyme for Gaucher Disease (LEAP Trial)

Phase 2
Waitlist Available
Research Sponsored by Genzyme, a Sanofi Company
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Female participants of childbearing potential and male patients must be willing to practice true abstinence in line with their preferred and usual lifestyle, or use 2 acceptable effective methods of contraception for the duration of the study and for at least 6 weeks for females and 90 days for males following their last dose of venglustat
Participant has a clinical diagnosis of Gaucher disease Type 1 (GD1) or Gaucher disease Type 3 (GD3) and documented deficiency of acid beta-glucosidase activity confirming this diagnosis
Timeline
Screening 3 weeks
Treatment Varies
Follow Up from screening up to end of study, up to approximately 8.7 years
Awards & highlights

LEAP Trial Summary

This trial is evaluating a new drug, venglustat, for the treatment of Gaucher Disease Type 3. The trial is divided into four parts. The first part is to screen patients and evaluate their biomarkers. The second and third parts are to study the safety and efficacy of venglustat in combination with another drug, Cerezyme. The fourth part is to study the safety and efficacy of venglustat as a monotherapy.

Who is the study for?
Adults aged 18+ with Gaucher Disease Type 3 who have been on enzyme replacement therapy for at least 3 years and meet specific health goals can join. They must not be pregnant, agree to use contraception, and avoid grapefruit products. Exclusions include myoclonic seizures, severe kidney issues, hypersensitivity to Cerezyme, or other significant health problems.Check my eligibility
What is being tested?
The trial tests the safety and effects of venglustat in combination with Cerezyme on brain biomarkers in adults with Gaucher Disease Type 3. It includes a screening phase followed by short-term and long-term treatment phases with both drugs, then an extension phase where only venglustat is given.See study design
What are the potential side effects?
Potential side effects are not detailed but may include reactions related to drug infusion or organ-specific inflammation due to immune response changes. Side effects from long-term monotherapy will also be assessed.

LEAP Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am willing to use birth control or practice abstinence during and after the study.
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I have been diagnosed with Gaucher disease Type 1 or 3.
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I have been on enzyme replacement therapy for at least 3 years and on a stable dose for 6 months.
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My Gaucher disease is under control, meeting all treatment goals.
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My seizures are under control with medication that doesn't strongly affect CYP3A.
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I have difficulty moving my eyes side to side.
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I am between 18 and 40 years old.
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I am a woman able to have children and have a negative pregnancy test.
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I am 18 years old or older.
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I am willing to avoid grapefruit and its products before and during the treatment.

LEAP Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~from screening up to end of study, up to approximately 8.7 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and from screening up to end of study, up to approximately 8.7 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Assessment of pharmacodynamic (PD) parameter: Lyso-glucosylceramide (lyso-GL1) and glucosylceramide (GL-1) in cerebrospinal fluid (CSF)
Number of patients with Treatment Emergent Adverse Events (TEAEs)
Secondary outcome measures
Assessment of CSF pharmacokinetic parameter: Cmax
Assessment of pharmacodynamic (PD) parameter: Lyso-glucosylceramide (lyso-GL1) and glucosylceramide (GL-1) in plasma
Assessment of pharmacokinetic parameter: CSF area under the curve (AUC)
+5 more

LEAP Trial Design

1Treatment groups
Experimental Treatment
Group I: Open label (OL) venglustatExperimental Treatment2 Interventions
Administered once a day orally for up to approximately 8.7 years. Patients will continue their usual dose of Cerezyme during Part 1, Part 2 and Part 3. There is no administration of Cerezyme in Part 4 unless administrated as rescue treatment.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
imiglucerase
2008
Completed Phase 3
~40

Find a Location

Who is running the clinical trial?

Genzyme, a Sanofi CompanyLead Sponsor
524 Previous Clinical Trials
85,630 Total Patients Enrolled
16 Trials studying Gaucher Disease
12,960 Patients Enrolled for Gaucher Disease
Clinical Sciences & OperationsStudy DirectorSanofi
857 Previous Clinical Trials
2,018,943 Total Patients Enrolled
10 Trials studying Gaucher Disease
965 Patients Enrolled for Gaucher Disease

Media Library

Imiglucerase (Enzyme Replacement Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT02843035 — Phase 2
Gaucher Disease Research Study Groups: Open label (OL) venglustat
Gaucher Disease Clinical Trial 2023: Imiglucerase Highlights & Side Effects. Trial Name: NCT02843035 — Phase 2
Imiglucerase (Enzyme Replacement Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02843035 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What is the total number of people taking part in this clinical trial?

"Presently, this trial is not enrolling patients. The clinical study was first posted on January 4th, 2017 and was most recently updated on August 9th, 2022. However, if you are seeking other studies related to gaucher disease type 1 or imiglucerase, there are 964 and 4 trials respectively that are looking for participants."

Answered by AI

Are we currently looking for new participants in this research?

"This study has already closed recruitment. The listing was created on January 4th, 2017 and last updated on August 9th, 2022. If you are seeking other trials, there are 964 clinical studies actively recruiting patients with gaucher disease type 1 and 4 trials for imiglucerase that still need participants."

Answered by AI

Does imiglucerase have the green light from the FDA?

"Imiglucerase has some clinical data supporting its safety, but not yet any efficacy data. Therefore, it received a 2."

Answered by AI

Are there any other examples of research teams testing imiglucerase?

"imiglucerase was first trialed in 2017 at location 826001. As of now, there have been 10 completed studies with 4 more ongoing. A large portion of these are taking place in Fairfax, Virginia."

Answered by AI

Has this type of trial been conducted before?

"Imiglucerase, a medication used to treat Gaucher disease, is being trialed in 4 live experiments conducted across the globe. The first study began in 2017 and 13 patients completed the Phase 2 drug approval stage. Genzyme, a subsidiary of Sanofi Company, sponsored the trial. In total, 10 trials have been completed as of now."

Answered by AI
~2 spots leftby Sep 2025