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Gaucher Disease, Type 3 clinical trials

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NCT ID: NCT05586243 Recruiting - Clinical trials for Gaucher Disease, Type 3

MAGNETIC RESONANCE SPECTROSCOPY BIOMARKERS IN TYPE 3 GAUCHER DISEASE (GD3)

Start date: January 1, 2023
Phase:
Study type: Observational

Recent studies have has shown that magnetic resonance spectroscopy (MRS) can provide validated neuronal markers in patients with Type 1 GD (GD1) who are on stable therapy. However, alterations in neurometabolites in adult patients with GD3, who have established neurological involvement, are not well understood. The goal of this study is to characterize neurometabolite profiles in adult patients with GD3 using MRS to identify novel biomarkers that can demonstrate treatment response. Additionally, a secondary aim is to evaluate relationships between neurometabolites and disease parameters, such as genotype, enzyme levels and Gaucher disease (GD) biomarkers.

NCT ID: NCT05447494 Recruiting - Clinical trials for Gaucher Disease, Type 1

Phase 1/2 Study of CAN103 in Subjects With Gaucher Disease

Start date: July 11, 2022
Phase: Phase 1/Phase 2
Study type: Interventional

Gaucher disease is a rare lysosomal storage disorder caused by deficient activity of the enzyme acid β-glucosidase, causing glucosylceramide to accumulate within macrophages and leading to hepatosplenomegaly, anemia, thrombocytopenia, and bone disease. In the non-neuronpathic form (type 1), disease manifestations are mostly systemic, whereas in the neuronopathic forms, glucosylceramide also accumulates in the central nervous sysem and leads to acute (type 2) or chronic (type 3) neurodegeneration. The purpose of this Phase 1/2 first-in-human study is to initially evaluate the safety and tolerability of two doses of CAN103, and then barring any safety concerns, to evaluate the efficacy and safety of the two doses administered intravenously every other week in treatment-naive subjects with Gaucher disease type 1 or type 3.

NCT ID: NCT04532047 Recruiting - Wolman Disease Clinical Trials

In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases

IUERT
Start date: July 1, 2021
Phase: Phase 1
Study type: Interventional

The investigators aims to determine the the maternal and fetal safety and feasibility of in utero fetal enzyme replacement therapy in fetuses with Lysosomal Storage Diseases.