Clinical trials: Gaucher's Disease Type I
Gaucher Disease
A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease
The main purpose of this study is to observe the side effects of VPRIV in participants with type 1 Gaucher disease who are either treatment-naïve (newly diagnosed) or who are currently being treated with enzyme replacement…
- Trial name
- NCT05529992
- Testing
- Velaglucerase Alfa
- Where
- 10 sites in China
- Volunteers
- 20 people
- Sponsor
- Takeda
Type 1 Gaucher Disease
A Long-Term Follow-up Study of Subjects With Gaucher Disease Who Previously Received AVR-RD-02
This is a multicenter, multinational long-term follow-up study to assess the long-term safety and durability of effect of AVR-RD-02 treatment in subjects who previously received AVR-RD-02 treatment (single dose administration).
- Trial name
- NCT04836377
- Testing
- Safety and efficacy assessments
- Where
- 3 sites in United States, Canada
- Volunteers
- 4 people
- Sponsor
- AVROBIO
Gaucher Disease
A Clinical Study for the Treatment of Pediatric and Adolescent Patients With Type 1 Gaucher Disease
The purpose of this study was to evaluate the safety, tolerability, efficacy, immunogenicity, PD and PK characteristics of LY-M001 injection in children with GD1 aged 6 years ≤ age < 18 years.
- Trial name
- NCT06528080
- Testing
- LY-M001
- Where
- China
- Volunteers
- 3 people
- Sponsor
- Shanghai Jiao Tong University School of Medicine
Gaucher's Disease
Phase I Study of Retrovirally Mediated Transfer of the Human Glucocerebrosidase Gene Into Peripheral Blood Stem Cells for Autologous Transplantation in Patients With Type I Gaucher Disease
OBJECTIVES: I. Transfer the human glucocerebrosidase (GC) gene into peripheral blood stem cells (PBSC) obtained from patients with type I Gaucher disease using a retroviral vector. II.
- Trial name
- NCT00004294
- Testing
- human glucocerebrosidase gene into autologous peripheral blood stem cells
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- National Center for Research Resources (NCRR)
Lysosomal Storage Diseases, Gaucher Disease
Lyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher Disease
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…
- Conditions
- Lysosomal Storage Diseases, Gaucher Disease, Sphingolipidoses
- Trial name
- LYSO-PROOF (NCT02416661)
- Where
- 8 sites in Albania, Greece and 5 more
- Volunteers
- 299 people
- Sponsor
- CENTOGENE GmbH Rostock
Gaucher Disease, Type 1
Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV)
Background: In order to allow our satisfied patients, who have successfully completed 24 months of rapid intravenous infusion of Velaglucerase alfa (VPRIV), to continue with the 10 minutes IV therapy, the clinical trial framework…
- Trial name
- NCT04120506
- Testing
- VPRIV
- Volunteers
- 15 people
- Sponsor
- Shaare Zedek Medical Center
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
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
Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease
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…
- Trial name
- NCT02574286
- Testing
- Velaglucerase alfa, Vitamin D
- Where
- 15 sites in United States, Israel and 2 more
- Volunteers
- 21 people
- Sponsor
- Shire
Gaucher Disease Type 1
Comparison of BMD Measurement by DEXA to BeamMed Speed-of-Sound Measurement at Forearm in Patients With Gaucher Disease
The purpose of this study is to compare the accuracy and comparability and secondarily to assess the values achieved by measurement of the forearm BMD by DXA and SOS by BeamMed, relative to standard DXA evaluations at the FN and…
- Trial name
- NCT02067247
- Testing
- BeamMed Speed-of-Sound bone strength test at forearm
- Where
- Israel
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Shaare Zedek Medical Center
Gaucher Disease Type I, Cerebroside Lipidosis Syndrome
A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.
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.
- Conditions
- Gaucher Disease Type I, Cerebroside Lipidosis Syndrome, Clucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease
- Trial name
- NCT00365131
- Testing
- Cerezyme (imiglucerase for injection)
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Genzyme, a Sanofi Company
Gaucher Disease, Type 1
Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease
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
- NCT00553631
- Testing
- velaglucerase alfa, imiglucerase
- Where
- 11 sites in United States, Argentina and 7 more
- Volunteers
- 34 people
- Sponsor
- Shire
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