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Substrate Reduction Therapy
Venglustat vs Imiglucerase for Gaucher Disease (LEAP2MONO Trial)
Phase 3
Recruiting
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
The participant has a modified SARA score of 1 or above
Adult participant is ≥18 years of age
Timeline
Screening 3 weeks
Treatment Varies
Follow Up from baseline to max of 3.5 years
Awards & highlights
LEAP2MONO Trial Summary
This trial will compare the effects of two different treatments for Gaucher disease Type 3. One treatment is a daily pill, and the other is an infusion every two weeks. The trial will measure how well each treatment works in improving or stabilizing the neurological symptoms and overall disease stability.
Who is the study for?
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.Check my eligibility
What is being tested?
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.See study design
What are the potential side effects?
While specific side effects aren't listed here, common ones associated with medications like Venglustat may include stomach issues, fatigue, headache, dizziness or allergic reactions. For Imiglucerase infusions such as Cerezyme side effects can range from discomfort at injection site to more serious immune responses.
LEAP2MONO Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
My balance and coordination are affected.
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I am 18 years old or older.
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I weigh at least 30 kg.
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I have been diagnosed with Gaucher's disease type 3.
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I have difficulty moving my eyes side to side quickly.
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I am between 12 and 17 years old.
LEAP2MONO Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ from baseline to max of 3.5 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~from baseline to max of 3.5 years
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Change in Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) total scale index score
Change in Scale for Assessment and Rating of Ataxia (SARA) modified total score
Secondary outcome measures
Change in Patient Health Questionnaire 9 (PHQ-9) during the treatment-emergent period (for participants 12 years of age at baseline)
Change in hemoglobin level
Change in score of Beck Depression Inventory II (BDI-II) during the treatment-emergent period (for participants 13 years of age and above at baseline)
+6 moreLEAP2MONO Trial Design
2Treatment groups
Experimental Treatment
Active Control
Group I: VenglustatExperimental Treatment1 Intervention
Venglustat
Group II: CerezymeActive Control1 Intervention
Cerezyme
Find a Location
Who is running the clinical trial?
Genzyme, a Sanofi CompanyLead Sponsor
524 Previous Clinical Trials
85,589 Total Patients Enrolled
16 Trials studying Gaucher Disease
12,932 Patients Enrolled for Gaucher Disease
SanofiLead Sponsor
2,164 Previous Clinical Trials
3,514,940 Total Patients Enrolled
13 Trials studying Gaucher Disease
6,950 Patients Enrolled for Gaucher Disease
Clinical Sciences & OperationsStudy DirectorSanofi
862 Previous Clinical Trials
2,019,798 Total Patients Enrolled
10 Trials studying Gaucher Disease
937 Patients Enrolled for Gaucher Disease
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- 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.My balance and coordination are affected.I have had a major organ transplant.I am 18 years old or older.I weigh at least 30 kg.I need a breathing machine for more than 12 hours a day while awake.I have been diagnosed with Gaucher's disease type 3.I haven't taken any experimental drugs recently.I've been on a stable enzyme replacement therapy for 3 years, stable for the last 6 months, and am clinically stable.I have not used certain strong medications or consumed grapefruit products recently and am willing to avoid them during treatment.I had my spleen completely removed or partially removed within the last 3 years.My seizures are under control with medication that doesn't strongly affect CYP3A.I haven't had chaperone or substrate reduction therapy, except venglustat, in the last 6 months.I have had cancer before, but not skin cancer (basal cell carcinoma).I will be hospitalized for surgery during the study.I am using birth control, not pregnant, not breastfeeding, and not donating sperm.I have difficulty moving my eyes side to side quickly.You have had problems with drugs or alcohol in the past year.I am between 12 and 17 years old.I need a machine to help me breathe.I do not have any serious health issues besides Gaucher's disease that would stop me from joining the study.I need regular blood transfusions to manage my condition.I have been diagnosed with progressive myoclonic epilepsy.
Research Study Groups:
This trial has the following groups:- Group 1: Venglustat
- Group 2: Cerezyme
Awards:
This trial has 2 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Can patients currently sign up for this experiment?
"That is accurate. The clinical trial is currently looking for 40 participants from 8 different sites, as stated on clinicaltrials.gov. This information was initially posted on 4/18/2022 and was last updated on 10/25/2022."
Answered by AI
Does Venglustat put patients at risk for any adverse health effects?
"Venglustat is considered safe based on its Phase 3 status and prior clinical data supporting its efficacy and safety."
Answered by AI
Are there different research teams in charge of this project in different areas?
"Presently, this experiment is being conducted in 8 different locations, which include Philadelphia, Fairfax, and Winnipeg. If you are considering participating in this trial, it would be most convenient to select a location near you to reduce travel."
Answered by AI
How many people are being included this experiment?
"The sponsor, Genzyme, a Sanofi Company, needs a total of 40 eligible patients in order to run the study. They will be recruiting from various sites including Children's Hospital of Philadelphia - Investigational Site Number: 8400004 in Philadelphia, Pennsylvania and Lysosomal and Rare Disorders Research and Treatment Center, Inc - Investigational Site Number: 8400001 in Fairfax, Virginia."
Answered by AI
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