ABOUT THIS STUDY
1. Male or female of any age; however, if female:
- must be using contraception if of childbearing potential or must be surgically sterile
- must not be lactating
2. Diagnosis of Type 3 GD by enzyme and sequence analysis; and confirmed by the Medical Monitor.
3. Splenomegaly at least 5 x multiples of normal (MN).
Eligible subjects may not have any of the following exclusion criteria:
1. Type 2 GD.
2. Presence of myoclonic seizures.
3. At least one allele of:
- N370S (N409S in recent nomenclature)
- R496H (R535H in recent nomenclature)
4. Presence of calcification in heart valves or arteries in echocardiography.
5. Presence of untreated iron, folic acid, vitamin B12 deficiency and/or hypothyroidism.
(Resolved anemia is not an exclusion criterion.)
6. Presence of human immunodeficiency virus (HIV), hepatitis B surface antigen (HBsAg),
and/or hepatitis C infections.
7. Splenectomy and bone marrow transplantation.
8. Presence of any medical, emotional, behavioural, or psychological condition that in
the judgment of the Investigator would interfere with the subject's compliance with
the requirements of the study.
9. Any other disorder that may interfere with the results of the efficacy endpoints.
10. Pregnancy or breastfeeding.
11. Currently taking another investigational drug for any condition or any therapeutic
drug for Gaucher disease.
12. The subject and/or subject's parent(s) or legal guardian(s) are unable to understand
the nature, scope, and possible consequences of the study.
13. Medical history of any food/drugs allergy.
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