Evaluate the safety and pharmacokinetics of eliglustat in pediatric patients (≥2 to <18 years
Evaluate the efficacy of eliglustat and quality of life in pediatric patients (≥2 to <18
The study will include a screening period of up to 60 days (Day -60 to -1), a primary
analysis treatment period (Day 1 to Week 52), a long-term treatment period (Week 53 to Week
104), and an extension period continuing up to Week 364 (for patients who continue to
demonstrate the clinical benefit from eliglustat monotherapy at Week 104). After study
completion, patients will be encouraged to enroll in the International Collaborative Gaucher
Group (ICGG) Gaucher Registry.
Gaucher's Disease Type I, Gaucher's Disease Type III
If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.
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