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Gaucher Disease Type I clinical trials

View clinical trials related to Gaucher Disease Type I.

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NCT ID: NCT06162338 Recruiting - Clinical trials for Gaucher Disease Type I

A Study of the Safety and Preliminary Efficacy of LY-M001 Injection in the Treatment of Adult Patients With Gaucher Disease Type I

LY-M001
Start date: October 30, 2023
Phase: N/A
Study type: Interventional

This is a prospective single-center, open, single-arm, single-dose intravenous infusion study to evaluate the safety and initial efficacy, pharmacodynamic characteristics, immunogenicity, biodistribution, and viral shedding of LY-M001 injection.This study mainly includes the main study stage and the long-term follow-up study stage.

NCT ID: NCT03240653 Recruiting - Clinical trials for Gaucher Disease, Type III

Gaucherite - A Study to Stratify Gaucher Disease

Gaucherite
Start date: January 1, 2014
Phase:
Study type: Observational

The purpose of this research is to review data already collected and to collect new data from adults and children in England with Gaucher Disease to determine clinical factors which predict severity and response to therapy of Gaucher disease especially in the areas of bone, cancer and brain conditions.

NCT ID: NCT02437396 Recruiting - Inflammation Clinical Trials

Oxidative Stress and Inflammatory Biomarkers in Gaucher Disease

Start date: October 2015
Phase:
Study type: Observational

The objective of this study is to evaluate oxidative stress and/or inflammation in patients with Gaucher disease type I using a series of biomarkers and correlate with measurements of currently used diagnostic biomarkers.

NCT ID: NCT00365131 Completed - Clinical trials for Cerebroside Lipidosis Syndrome

A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.

Start date: December 1997
Phase: Phase 4
Study type: Interventional

This is a multicenter, open-label, prospective study of the efficacy of Cerezyme in treating patients with skeletal manifestations secondary to Type I Gaucher disease. The study objective is to evaluate and quantify skeletal responses as compared to baseline in Type I gaucher disease patients receiving Cerezyme therapy for 48 months. Additional objectives were to assess the usefulness of various skeletal parameters, such as bone pain, bone crises, bone mineral density, and serum and urine bone markers, as indicative of treatment response and may be useful in dose management.