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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 (15) clinical trials

The Rare Disease Registry Program (including but not limited to, Gaucher, Fabry, MPS I, and Pompe diseases) is a multi-center, international, longitudinal, observational program that tracks the natural history and outcomes of patients with these rare diseases.

Phase N/A

ABOUT GAUCHER DISEASE AND THE SHP-GCB-402 STUDY If you have type I Gaucher disease, glucocerebroside (a fatty substance) builds up in your body because you lack the enzyme glucocerebrosidase (GBC), which is needed to break it down. When this substance accumulates in your bone marrow, it leads to loss of ...

Phase N/A

The SHP-GCB-402 study for Gaucher disease is evaluating the effect velaglucerase alfa (VPRIV®) has on bone pain and bone density in people with type I Gaucher disease.

Phase

Miglustat on Gaucher Disease Type IIIB

understand if Miglustat (glucosylceramide synthase inhibitor) could improve neuropathy in patients with Gaucher disease

Phase N/A

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

Genzyme Osteopenia/Osteoporosis Study

Gaucher disease is a potential secondary cause of low bone mineral density and it is prevalent among patients with low BMD. This cross sectional design study will measure point prevalence of Gaucher disease in patients with low bone mineral density (BMD).

Phase N/A

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

The total duration for GD1 patients is 45 days while for GD3 patients, the total duration is up to 61 weeks.

Phase

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

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