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Venglustat + Cerezyme for Gaucher Disease (LEAP Trial)
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.
LEAP Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowLEAP Trial Timeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.LEAP Trial Design
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Who is running the clinical trial?
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- I experience sudden, brief muscle jerks.I need regular blood transfusions to manage my condition.I do not have any serious health issues other than Gaucher's disease that would stop me from joining.I am willing to use birth control or practice abstinence during and after the study.My kidney function is low, with a filtration rate under 30 mL/min.My Gaucher disease is under control, meeting all treatment goals.My seizures are under control with medication that doesn't strongly affect CYP3A.I haven't taken strong or moderate drugs that affect liver enzymes within the last 15 days or 5 half-lives before screening.I am scheduled to be hospitalized for surgery during the study.I have been diagnosed with Gaucher disease Type 1 or 3.I have been on enzyme replacement therapy for at least 3 years and on a stable dose for 6 months.I have had cancer before, but not basal cell carcinoma.I have a significant cataract, but not in the center of the lens.I can't or won't take Cerezyme due to an allergy or personal choice.I am taking medication that could worsen cataract-related vision problems.I have difficulty moving my eyes side to side.I have had a major organ transplant.I am between 18 and 40 years old.I am a woman able to have children and have a negative pregnancy test.You have met the treatment goals required to enter the next part of the study.I am 18 years old or older.I use a breathing support machine for more than 12 hours a day while awake.My liver function tests are normal, or I have Gilbert Syndrome if they're high.You need to use a machine to help you breathe.I had surgery to remove all or part of my spleen within the last 3 years.I have not had substrate reduction or chaperone therapy for Gaucher's disease in the last 6 months.I am willing to avoid grapefruit and its products before and during the treatment.
- Group 1: Open label (OL) venglustat
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- 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
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."
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."
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."
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."
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."
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