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Gaucher Disease Clinical Trials

A listing of Gaucher Disease medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

RESULTS

Found (3) clinical trials

Study of the Effect of Velaglucerase Alfa (VPRIV ) on Bone-related Pathology in Treatment-na ve Participants With Type 1 Gaucher Disease

The primary purpose of this study is to evaluate the effect of VPRIV therapy (60 units per kilogram [U/kg] every other week [EOW]) in treatment-naive participants with type 1 Gaucher disease on change from baseline in lumbar spine (LS) bone mineral density (BMD) Z-score as measured by DXA after 24 ...

Phase

Pharmacokinetics Pharmacodynamics And Safety Study Of Elelyso(tm) In Pediatric Subjects With Type 1 Gaucher Disease

This study (B3031003) is an open-label study in pediatric subjects with Type 1 Gaucher Disease to characterize PK, PD and safety following an infusion of taliglucerase alfa in at least 5 subjects with body weight less than 15 kg; at least 5 subjects with body weight 15 to less than ...

Phase

Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Treatment-naive Patients With Type 1 Gaucher Disease

Protocol Study Design: This will be a single-center, open-label trial to assess the safety and efficacy of rapid administration of velaglucerase alfa (VPRIV) to treatment-naive patients with type I Gaucher disease. The first six infusions will be administered in the hospital: the first three infusions within 60, 30 and 20 ...

Phase