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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.


Found (27) clinical trials

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. The glycoproteinoses sialidosis and galactosialidosis are ultra-rare disorders involving predominantly the skeletal and central nervous systems that are likewise fatal or severely debilitating. No effective therapy for patients with these diseases has ...

Phase N/A

Chronic Hepatitis C Treatment in Egyptian Children With Gaucher Disease.

This is a prospective open label study, all Gaucher disease pediatric patients (6-18 years), diagnosed at or referred to the HCV Egyptian treatment site, Mansoura University Children's Hospital Gastroenterology and hepatology unit, after positive HCV screen results by Anti HCV antibodies and confirmatory positive quantitative HCV polymerase chain reaction (PCR) ...


Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therapy

Introduction Description of the Problem: Enzyme replacement therapy (ERT) makes a significant impact on the clinical manifestations and quality of life of patients with Gaucher disease (GD) . The goals of therapy focus mainly on platelets, hemoglobin, spleen, liver, bones and growth parameters for children. ERT is mostly effective in ...


Liver and the Spleen Stifness in Adult Patients With Gaucher's Disease Using Ultrasound Shear Wave Elastography

To assess liver and spleen stiffness measurement using Shear Wave Elastography-SWE and evaluate liver and spleen fibrosis in patients with Gaucher Disease. To compare the elastography with the biomarkers that taken at the GD haematology clinic and to check the correlation with severity of liver and spleen fibrosis. SWE is ...

Phase N/A

Immune Biomarkers Related to Bone Pathology in Patients With Type 1 Gaucher Disease

This clinical observational study is designed to identify specific biomarkers for bone involvement in patients with GD1 with decreased bone density and/or bone structural abnormalities Aims Identify novel immune cell surface and biochemical markers in peripheral blood correlating with bone involvement in GD. Assess the correlation between cytokine levels in ...

Phase N/A

Canadian VPRIV Study in Participants Previously Treated With Other Enzyme Replacement Therapies (ERTs)/Substrate Reduction Therapies (SRTs)

The purpose of the study is to assess safety of velaglucerase alfa (VPRIV) in participants affected with Gaucher disease type 1 (GD1) transitioning from other enzyme replacement therapies (ERTs)/ substrate reduction therapies (SRTs) to VPRIV in a Canadian real-world setting across all age groups.

Phase N/A

Phase 1/2 Lentiviral Vector Gene Therapy AVR-RD-02 for Subjects With Type 1 Gaucher Disease

The duration of each subject's participation in this study will be approximately 68 weeks (or 1 year, 16 weeks), comprised of a five study periods (Screening, Baseline, Pre-transplant, Transplant, and Post-transplant Follow-up). During the Screening Period (up to 60 days), written informed consent (and assent, if applicable) will be obtained ...