Clinical Trials Logo

Gaucher Disease clinical trials

View clinical trials related to Gaucher Disease.

Filter by:

NCT ID: NCT03519646 Completed - Clinical trials for Gaucher Disease, Type III

Eliglustat on Gaucher Disease Type IIIB

Start date: April 23, 2018
Phase: N/A
Study type: Interventional

Evaluation of the safety in the combination usage of Cerdelga and Cerezyme in type III Gaucher disease patients and the efficacy on soft tissue diseases.

NCT ID: NCT03333447 Completed - MRI Clinical Trials

Retrospective and Prospective Observational Study of MRI Changes in Bone and Visceral Lesions of Patients With Type 1 Gaucher Disease Treated With VPRIV® (Velaglucerase Alfa)

EIROS
Start date: January 17, 2017
Phase:
Study type: Observational

This study with standardized reading MRIs, will provide for the first time objective and quantified data on organomegaly (liver and spleen volumes) as well as bone alteration (Bone Marrow Burden11) of French patients treated with VPRIV®. These data will help to better assess the impact of this treatment on these parameters. The result of this study will also answer in part to the request of the French Transparency Commission (CT: Commission de Transparence) of the French National Health Authority to provide them with data of French patients treated with VPRIV®.

NCT ID: NCT02785744 Completed - Gaucher Disease Clinical Trials

Genzyme Osteopenia/Osteoporosis Study

Start date: April 1, 2016
Phase:
Study type: Observational

Gaucher disease is a most common genetic metabolic disease characterized by low platelet number, liver and spleen enlargement and various forms of bone diseases including low bone mineral density leading to brittle bones. Various treatment options are now available for this disease. The purpose of this research study is to determine the prevalence of Gaucher disease in patients with low bone mineral density as observed by DEXA scan, which is a form of X-Ray of the bone.

NCT ID: NCT02650219 Completed - Gaucher Disease Clinical Trials

Auto-antibodies Prevalence and CD1 Role in Gaucher Disease

Start date: January 2010
Phase: N/A
Study type: Observational

Hypergammaglobulinaemia is frequently observed in type 1 Gaucher disease (GD1), being either polyclonal or monoclonal gammopathies. Polyclonal hypergammaglobulinemia may be related to the presence of autoantibodies. The clinical significance of such antibodies is questioned in Gaucher disease (GD), as some cases of immunologic thrombocytopenia and autoimmune hemolytic anemia have also been reported. Objectives: To evaluate the prevalence of autoantibodies and autoimmune diseases in GD1 patients, we conducted a multicenter national study. The investigators investigated whether there was a link between splenectomy, genotype, therapeutic options and the presence of these autoantibodies.They also investigated whether there was a correlation with some clinical manifestations of GD1

NCT ID: NCT02574286 Completed - Gaucher Disease Clinical Trials

Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease

Start date: June 29, 2016
Phase: Phase 4
Study type: Interventional

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 dual energy x-ray absorptiometry (DXA) after 24 months of treatment.

NCT ID: NCT02536937 Completed - Gaucher Disease Clinical Trials

A Study of the Effects of Renal Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate

Start date: September 2015
Phase: Phase 1
Study type: Interventional

Primary Objective: To study the effect of mild, moderate, and severe renal impairment on the pharmacokinetics (PK) of eliglustat. Secondary Objective: To assess the tolerability of eliglustat tartrate given as a single dose in subjects with mild, moderate, and severe renal impairment in comparison with matched subjects with normal renal function.

NCT ID: NCT02536911 Completed - Gaucher Disease Clinical Trials

A Study of the Effects of Hepatic Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate

Start date: September 2015
Phase: Phase 1
Study type: Interventional

Primary Objective: To study the effect of mild and moderate hepatic impairment on the pharmacokinetics (PK) of eliglustat. Secondary Objective: To assess the tolerability of eliglustat tartrate given as a single dose in subjects with mild and moderate hepatic impairment in comparison with matched subjects with normal hepatic function.

NCT ID: NCT02536755 Completed - Gaucher Disease Clinical Trials

Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies

EXOSKEL
Start date: October 27, 2015
Phase: Phase 3
Study type: Interventional

Primary Objective: Evaluate long term skeletal response to eliglustat in adult participants who successfully completed one of the Phase 2 or Phase 3 eliglustat studies. Secondary Objective: Evaluate the safety of eliglustat (by serious adverse event continuous monitoring), the quality of life (Short Form-36 Health Survey [SF-36]) and biomarkers of Gaucher disease type 1 (GD1) (chitotriosidase, plasma glucosylceramide [GL-1] and lyso glucosylceramide [lyso-GL-1]) in adult participants who successfully completed one of the Phase 2 or Phase 3 studies.

NCT ID: NCT02422654 Completed - Gaucher Disease Clinical Trials

Taste Evaluation of Different Liquid Formulations With Eliglustat

Start date: April 2015
Phase: Phase 1
Study type: Interventional

Primary Objective: The purpose of this study is to assess the palatability of eliglustat prototype liquid formulations in healthy subjects.

NCT ID: NCT02416661 Completed - Gaucher Disease Clinical Trials

Lyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher Disease

LYSO-PROOF
Start date: August 27, 2018
Phase:
Study type: Observational

International, multicenter, epidemiological study to demonstrate the correlation and predictive value of lyso-Gb1 concentration with the clinical severity of naïve, initially non-ERT/SRT Gaucher disease type 1 and during the study ERT/SRT-newly started Gaucher type 1 patients and to correlate lyso-Gb1 concentration with the clinical improvement of ERT or SRT treated Gaucher type 1 and the clinical course of non-treated patients based on GD-DS3