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Gaucher Disease, Type 3 clinical trials

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NCT ID: NCT05815004 Withdrawn - Clinical trials for Gaucher Disease, Type 3

An Efficacy and Safety Study of AVR-RD-02 Compared to Enzyme Replacement Therapy for Treatment of Gaucher Disease Type 3

Start date: October 2023
Phase: Phase 2/Phase 3
Study type: Interventional

The purpose of this study is to evaluate the efficacy and safety of autologous hemotopoietic stem cell (HSC) gene therapy, AVR-RD-02, compared to enzyme replacement therapy, for the treatment of Gaucher disease Type 3 in male and female participants aged 2 to 25 years. The study will consist of 2 parts - Core (Part 1) followed by the ERT-crossover (Part 2)

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.

NCT ID: NCT04002830 Completed - Clinical trials for Gaucher Disease, Type 3

A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease

Start date: November 20, 2020
Phase: Phase 4
Study type: Interventional

This is a multicenter study to assess the safety and efficacy of taliglucerase alfa (60 units/kg) in previously untreated subjects of any age with Type 3 GD. Subjects will receive an infusion of taliglucerase alfa every 2 weeks for 12 months. Subjects who tolerate the infusions well, and who are treated in centers where home therapy is the SOC will be allowed to switch from site to home treatment at the discretion of the PI but after no less than 3 uneventful infusions at the site.

NCT ID: NCT03746587 Terminated - Clinical trials for Gaucher Disease, Type 1

Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3

Start date: June 6, 2018
Phase: Phase 2
Study type: Interventional

Multicenter, doubleblinded, randomized placebo-controlled study of arimoclomol in patients with Gaucher Disease Type 1 or 3

NCT ID: NCT02843035 Active, not recruiting - Clinical trials for Gaucher Disease Type 1

Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension

LEAP
Start date: January 4, 2017
Phase: Phase 2
Study type: Interventional

Part 1: Biomarker evaluation/screening phase Primary Objectives: - Evaluate cerebrospinal fluid (CSF) biomarkers in adult Gaucher disease Type 3 (GD3) patients that distinguish GD3 from adult Gaucher disease Type 1 (GD1) patients - Screen adult GD3 patients who qualify for treatment with venglustat in Parts 2, Part 3, and Part 4 Parts 2 and 3: Combination treatment phases Primary objectives: - Evaluate short-term (Part 2) and long-term (Part 3) safety and tolerability of venglustat in combination with Cerezyme in adult GD3 patients - Evaluate the change in CSF central nervous system (CNS) biomarkers (glucosylceramide [GL-1] and lyso-glucosylceramide [lyso-GL-1]) from adult GD3 patients receiving venglustat in combination with Cerezyme (Part 2 only) Part 4: Extended treatment phase with monotherapy Primary objectives: • Evaluate safety and tolerability of venglustat monotherapy in adult GD3 patients who have remained systemically stable on venglustat in combination with Cerezyme Parts 2 and 3: Combination treatment phases Secondary Objectives: - Evaluate the pharmacokinetics (PK) of venglustat in adult GD3 patients - Explore the efficacy of venglustat in combination with Cerezyme in infiltrative lung disease (ILD) in adult GD3 patients (Part 2 only) - Explore the efficacy of venglustat in combination with Cerezyme in systemic disease in adult GD3 patients - Explore the efficacy of venglustat in combination with Cerezyme on neurological function in adult GD3 patients - Explore plasma biomarkers (lyso-GL-1 and GL-1) in adult GD3 patients - Explore CSF biomarkers other than lyso-GL-1 and GL-1 in adult GD3 patients (Part 2 only) Part 4: Extended treatment phase with monotherapy Secondary objectives: - Explore the efficacy of venglustat in systemic disease in adult GD3 patients - Explore the efficacy of venglustat on neurological function in adult GD3 patients - Explore plasma biomarkers (lyso-GL-1 and GL-1) in adult GD3 patients

NCT ID: NCT02528617 Withdrawn - Clinical trials for Gaucher Disease Type 1

The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease

Start date: July 2015
Phase: Phase 4
Study type: Interventional

The purpose of this trial is to study the effect of Velaglucerase Alfa on skeletal bone development of children with Type 1 or Type 3 Gaucher Disease. In addition, the natural history and neurological status of children with Type 3 Gaucher Disease will be studied.

NCT ID: NCT01685216 Completed - Clinical trials for Gaucher Disease, Type 3

Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease

Start date: September 14, 2012
Phase: Phase 1/Phase 2
Study type: Interventional

Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Gaucher disease has been classified into 3 clinical subtypes based on the presence or absence of neurological symptoms and the severity of these neurological symptoms. Patients with type 2 Gaucher disease present with acute neurological deterioration, and those with type 3 disease typically display a more sub acute neurological course. Type 1 Gaucher disease, the most common form accounting for more than 90% of all Gaucher disease cases, does not involve the central nervous system. The purpose of this clinical research study is to investigate the safety and effectiveness of velaglucerase alfa in patients with type 3 Gaucher disease.