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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 (17) clinical trials

International Collaborative Gaucher Group (ICGG) Gaucher Registry

The ICGG Gaucher Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Gaucher disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician. The ...

Phase N/A

Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease

PROTOCOL OUTLINE: Patients are treated with intravenous glucocerebrosidase every 2 weeks. The dose is based on clinical severity of disease and response to therapy.

Phase

GZ/SAR402671 in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3

The total duration for GD1 patients is 45 days (Part 1), while for GD3 patients, the total duration is up to 168 weeks, including 2 part-treatment of 52 weeks and 104 weeks, respectively.

Phase

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

LYSO-PROVE - Determine the Prognostic Value of Lyso-Gb1 for Monitoring the Progress of Gaucher Disease

Gaucher disease is an autosomal recessive inherited lysosomal storage disorder. The disease is caused by the hereditary deficiency of the glucocerebrosidase, a lysosomal enzyme that breaks down glucocerebroside into glucose and ceramide. Gaucher disease is the most common sphingolipidosis and it is among the most frequent inherited diseases among the ...

Phase N/A

Nervous System Degeneration in Glycosphingolipid Storage Disorders

The GM1 and GM2 gangliosidoses are lysosomal storage disorders that primarily affect the brain and are uniformly fatal. No effective therapy for patients with these diseases has yet been demonstrated. Historically, since these disorders are fatal very little natural history information or disease characterization using modern medical techniques has been ...

Phase N/A

Clinical and Pathophysiological Investigations Into Erdheim Chester Disease

Rare and potentially lethal, Erdheim-Chester Disease (ECD) is a histiocytic neoplasm about which little is known, and for which there remains a paucity of effective treatments. Histologically, it is categorized as a non-Langerhans cell histiocytosis, and most commonly occurs in men between the ages of 50 and 70, although cases ...

Phase N/A