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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 (25) 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

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

Gaucherite - A Study to Stratify Gaucher Disease

Gaucher disease is part of a rare group of genetic metabolic diseases which are caused by an inherited deficiency of the enzyme glucocerebrosidase. The most common form, Type 1 affects 1 in 40,000 to 60,000 individuals in the general population. In Type 1 symptoms may appear at any time from ...

Phase N/A

Investigating Lysosomal Storage Diseases in Minority Groups

Although lysosomal storage disorders, such as Fabry disease, Gaucher disease, and Pompe disease, represent serious challenges in the healthcare system, no study has yet investigated the prevalence of these diseases in the US. Frequently, patients show progressive worsening of symptoms for several years before they get diagnosed. Since many of ...

Phase N/A

Thrombocytopathy in Gaucher Disease Patients

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

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

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: BioGaucher (BioGaucher)

Gaucher disease (GD) is an autosomal recessive hereditary lysosomal storage disorder. Occurrence of the disease is due to a hereditary deficiency of the Glucocerebrosidase, a lysosomal enzyme which divides Glucocerebroside into Glucose and Ceramides. The unmetabolised Glucocerebrosides are stored throughout the whole reticulo-endothelial system. Accumulation of Glycolipid-enriched macrophages establishes a ...

Phase N/A

A Long-term Follow-up Study of Gaucher Disease

The study aims (aims a-c) will be accomplished by the systematic monitoring of health records. Health information will be extracted from medical records for patients followed at Duke. Medical records will be requested from treating physicians outside of Duke. The PI, (Dr. Kishnani) a recognized expert in Gaucher disease, is ...

Phase N/A