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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 (11) 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

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

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

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

Gaucher Disease Outcome Survey (GOS)

The Gaucher Outcomes Survey (GOS) is an ongoing observational, international, multi-center, long-term Registry of Patients with Gaucher Disease irrespective of their treatment status or type of treatment received. No experimental intervention is involved. Patients undergo clinical assessments and receive care as determined by the patients' treating physician. The objectives of ...

Phase N/A

Biomarker for Gaucher Disease

The unmetabolised Glucocerebrosides are stored throughout the whole reticulo-endothelial system. Accumulation of Glycolipid-enriched Macrophages establishes a pathoanatomical phenomenon, the so-called Gaucher cells, which can be verified by light microscopy. Activation markers of the Macrophages, like the enzyme Chitotriosidase or CCL18, are parameters which follow the course of Gaucher dis-ease. Gaucher ...

Phase N/A

Thrombocytopathy in Gaucher Disease Patients

Objectives: Delineating the cause of the thrombocytopathy in Gaucher disease patients: 1. Identifying thrombocytopathy among a cohort of 70 Gaucher patients managed (treated and untreated) in our clinic using a panel of platelets function tests. 2. Understanding the etiology for platelets dysfunction in Gaucher disease. 3. Evaluating the impact of ...

Phase N/A