Clinical trials: Gaucher Disease, Type I
Gaucher Disease, Type 1
A Gene Therapy Study in Patients With Gaucher Disease Type 1
This study is a first-in-human, open-label, safety, tolerability, and efficacy study in adult patients with Gaucher disease Type 1.
- Trial name
- GALILEO-1 (NCT05324943)
- Testing
- FLT201
- Where
- 10 sites in United States, Brazil and 4 more
- Volunteers
- 10 people
- Sponsor
- Spur Therapeutics
Lysosomal Storage Disorders
Molecular and Cellular Mechanisms of Lysosomal Storage Diseases
The lysosome is a specialized part of the cell that functions to degrade metabolic wastes in the cell. Defects in the functioning of the lysosome result in accumulation and subsequent storage of such metabolic wastes.
- Trial name
- NCT02000310
- Where
- United States
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- O & O Alpan LLC
Gaucher's Disease
Effects of Enzyme Replacement in Gaucher's Disease
Gaucher disease is a lysosomal storage disease resulting from glycocerebroside accumulation in macrophages due to a genetic deficiency of the enzyme glucocerebrosidase.
- Trial name
- NCT00001289
- Where
- United States
- Volunteers
- 70 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Gaucher's Disease
Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease
OBJECTIVES: I. Evaluate the efficacy and toxicity of glucocerebrosidase enzyme therapy in patients with Gaucher disease.
- Trial name
- NCT00004293
- Testing
- glucocerebrosidase
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- National Center for Research Resources (NCRR)
Gaucher Disease, Type 1, Gaucher Disease, Type 3
Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3
Multicenter, doubleblinded, randomized placebo-controlled study of arimoclomol in patients with Gaucher Disease Type 1 or 3
- Trial name
- NCT03746587
- Testing
- Arimoclomol
- Where
- 8 sites in India
- Volunteers
- 39 people
- Sponsor
- ZevraDenmark
Gaucher Disease Type 1
A Second-generation AI Based Therapeutic Regimen in Patients With Gaucher Disease Treated With Enzyme Replacement Therapy.
An open-labeled, prospective, single-center proof-of-concept study. Patients with Gaucher Disease aged 18-75 who received intravenous Enzyme Replacement Therapy once every two weeks were enrolled.
- Trial name
- NCT06050967
- Testing
- Enzyme replacement therapy for Gaucher disease in combination with altus care application
- Where
- Israel
- Volunteers
- 5 people
- Sponsor
- Hadassah Medical Organization
Gaucher Disease
Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease
This was a multinational, open-label study to assess the safety and efficacy of AVR-RD-02 in approximately 8 to 16 subjects (male or female) who are ≥18 and ≤50 years of age and post pubertal at Screening with a confirmed…
- Trial name
- NCT04145037
- Testing
- AVR-RD-02
- Where
- 5 sites in United States, Canada
- Volunteers
- 8 people
- Sponsor
- AVROBIO
Type I Gaucher Disease
Clinical Trial of Ambroxol in Patients With Type I Gaucher Disease
Ambroxol is expected to improve the signs and symptoms of patients with Type I Gaucher Disease.
- Trial name
- NCT01463215
- Testing
- Ambroxol
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Exsar Corporation
Gaucher Disease
A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Young Children With Gaucher Disease
The main aim of this study is to learn if velaglucerase alfa (VPRIV) improves growth and symptoms in participants up to 5 years of age with Gaucher disease. Symptoms will be checked with blood tests.
- Trial name
- PEDS (NCT04721366)
- Testing
- Standard of Care
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Takeda
Gaucher Disease
Taste Evaluation of Different Liquid Formulations With Eliglustat
Primary Objective: The purpose of this study is to assess the palatability of eliglustat prototype liquid formulations in healthy subjects.
- Trial name
- NCT02422654
- Testing
- eliglustat
- Where
- United States
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- Genzyme, a Sanofi Company
Gaucher Disease
Validating a New Severity Score System for Adults With Type 1 Gaucher Disease (GD1)
With the participation of an international consortium of investigators, the investigators will evaluate the validity of a new severity score system called DS3 for adult patients with Gaucher disease.
- Trial name
- NCT01136304
- Testing
- Imiglucerase
- Where
- 2 sites in United States
- Volunteers
- 173 people
- Sponsor
- University Research Foundation for Lysosomal Storage Diseases, Inc.
Gaucher Disease
Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase
This is a multi-center, open-label, switchover trial to assess the safety of taliglucerase alfa in 30 patients with Gaucher disease who are currently being treated with imiglucerase (Cerezyme®) enzyme replacement therapy.
- Trial name
- NCT00712348
- Testing
- Taliglucerase alfa
- Where
- 10 sites in United States, Australia and 4 more
- Volunteers
- 31 people
- Sponsor
- Pfizer
Gaucher Disease
Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT)
Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside accumulates within macrophages leading to cellular…
- Trial name
- NCT00391625
- Testing
- GA-GCB
- Where
- 3 sites in Israel, Romania and 1 more
- Volunteers
- 10 people
- Sponsor
- Shire
Gaucher Disease
Genzyme Osteopenia/Osteoporosis Study
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.
- Trial name
- NCT02785744
- Testing
- Gaucher disease DNA mutation analysis
- Where
- United States
- Volunteers
- 76 people
- Sponsor
- NYU Langone Health
Gaucher Disease, Splenomegaly
A National Study in Patients With Unexplained Splenomegaly
Primary Objective: To estimate the prevalence of Gaucher disease and of other etiologies, in patients of 15 years old or more presenting with unexplained splenomegaly after exclusion of first intention-diagnoses (e.g.
- Trial name
- SMS (NCT04430881)
- Where
- France
- Volunteers
- 506 people
- Sponsor
- Sanofi
Gaucher Disease, Type 1
Phase III Study of ISU302 in Patients With Type 1 Gaucher Disease
The purpose of this study is to evaluate the safety and efficacy of ISU302 in patients with Type 1 Gaucher disease.
- Trial name
- NCT02770625
- Testing
- ISU302
- Volunteers
- 8 people
- Sponsor
- ISU Abxis Co., Ltd.
Gaucher Disease
Clinical Audit of Managment of Gausher Disease in Children
clinical audit on managment of gausher disease in children
- Trial name
- NCT03590548
- Testing
- gausher disease
- Volunteers
- 1 people
- Sponsor
- Yasmeen Abd Elbaset Hamzaa
Gaucher Disease
A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)
The primary objective of this study was to evaluate the efficacy and safety of once daily (QD) versus twice daily (BID) dosing of eliglustat tartrate (Genz-112638) in participants with Gaucher disease type 1 who had demonstrated…
- Trial name
- NCT01074944
- Testing
- Eliglustat tartrate
- Where
- 46 sites in United States, Australia and 15 more
- Volunteers
- 170 people
- Sponsor
- Genzyme, a Sanofi Company
Gaucher Disease, Hepatitis C
Examining the Immune Response in Patients With Gaucher Disease and Hepatitis C
Study objectives: Investigate the anti-HCV response in patients with Gaucher disease(GD) Define the potential role of high levels of Glucocerebroside in the immune system Study hypothesis: High levels of Glucocerebroside can be…
- Trial name
- NCT01274208
- Where
- 2 sites in Israel
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Shaare Zedek Medical Center
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
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…
- Trial name
- LY-M001 (NCT06162338)
- Testing
- LY-M001 Injection
- Where
- China
- Volunteers
- 3 people
- Sponsor
- He Huang