Gaucher's Disease Clinical Trial
OBJECTIVES:
I. Evaluate the efficacy and toxicity of glucocerebrosidase enzyme therapy in patients with
Gaucher disease.
PROTOCOL OUTLINE:
Patients are treated with intravenous glucocerebrosidase every 2 weeks. The dose is based on
clinical severity of disease and response to therapy.
;
Primary Purpose: Treatment
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT00001289 -
Effects of Enzyme Replacement in Gaucher's Disease
|
N/A | |
| Completed |
NCT00004488 -
Phase II Randomized Study of Alendronate Sodium for Osteopenia in Patients With Gaucher's Disease
|
Phase 2 | |
| Completed |
NCT00001410 -
PEG-Glucocerebrosidase for the Treatment of Gaucher Disease
|
Phase 1 | |
| Completed |
NCT00004294 -
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
|
Phase 1 | |
| Completed |
NCT04656600 -
Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type Ⅲ
|
Phase 4 | |
| Completed |
NCT01427517 -
Intravenous N-acetylcysteine for the Treatment of Gaucher's Disease and Parkinson's Disease
|
Phase 1 | |
| Completed |
NCT00176904 -
Stem Cell Transplant for Inborn Errors of Metabolism
|
Phase 2/Phase 3 | |
| Completed |
NCT00001234 -
Gene Therapy for Gaucher's and Fabry Disease Using Viruses and Blood-Forming Cells
|
Phase 1 | |
| Completed |
NCT06188325 -
A Study to Evaluate Pharmacokinetic Parameters of Eliglustat in Healthy Volunteers Who Are CYP2D6 Extensive or Poor Metabolizers
|
Phase 1 | |
| Completed |
NCT00001416 -
Bone Response to Enzyme Replacement in Gaucher's Disease
|
Phase 2 | |
| Completed |
NCT06193304 -
A Study to Evaluate Pharmacokinetic Parameters and Safety of Eliglustat Absorption Through the Mouth
|
Phase 1 | |
| Active, not recruiting |
NCT00005900 -
Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
|
N/A | |
| Completed |
NCT01586455 -
Human Placental-Derived Stem Cell Transplantation
|
Phase 1 |