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What is the purpose of this clinical study?
The purpose of this Phase III study, called LEAP2MONO, is to assess for improvement or stabilization of neurological signs and symptoms, maintenance of systemic disease stability (organ effects – spleen and liver size and blood levels) and safety of a daily, oral investigational medication (venglustat) compared to another medication (Cerezyme) given as an intravenous infusion (injecting the medication directly into a vein in a drip) every 2 weeks for neuronopathic GD3.
Breakdown of study design periods
The study will consist of the following periods:
Depending on the time of enrollment, participants will take part in the trial a minimum of about 2 years and 2 months and a maximum of about 3 years and 3 months.
How many people are in this study?
The study is being conducted in multiple countries.
What is a clinical research study?
What is a Phase III (Phase 3) trial?
Why should I join a clinical research study?
Thousands of people volunteer to participate in clinical trials every year. The reasons for participation vary, but all of them help to advance medicine. Some reasons include:
What is an investigational medication?
An investigational medication is what is being studied to see if your symptoms improve while taking it. The drug is not yet approved for general use by regulatory authorities. During the open label treatment period, all participants will receive the investigational medication.
What is a placebo?
A placebo is an inactive substance or treatment that looks the same as, and is given in the same way as, an active drug or intervention/treatment being studied. This study has a 50/50 chance of receiving a placebo for up to 52 weeks during the treatment period. Neither you nor the study doctor will know if you are receiving the placebo or investigational medication.
What is a double-blind study?
Neither the study participant nor the study doctor will know which treatment the patient is receiving.
What is an open label study?
Both the study participant and study doctor will know the treatment assignment.
What type of study-related medical care is provided during a study?
Complete this brief questionnaire (approximately 5 to 10 minutes) to determine if you or your child are eligible to participate in this clinical study and be referred to a recruiting site.
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