Clinical Trials for Gaucher Disease
Clinical trials are research studies conducted in an effort to improve overall patient health and care.
Each trial involves running supervised tests to determine the effectiveness and safety of new drugs, procedures and/or devices with the aim of answering scientific questions about a disease or condition.
Risks and side-effects are also evaluated during these trials with results ranging from being unpleasant, with side effects such as headaches, to more serious or even life-threatening risks.
A clinical trial may be separated into phases, or steps, with each step designed to answer a separate research question. This maybe reflected in the trial name.
A brief description of each phase:
- Phase I: Usually designed to evaluate safety, determine a safe dosage range, and identify side effects on a small group of patients.
- Phase II: If Phase I is successful, the trial is then repeated with a larger group to further evaluate its effect and safety.
- Phase III: Trials are conducted on an ever larger group and are compared with the best current treatment while gathering more information on effect and safety.
- Phase IV: These studies monitor long-term side effects after the treatment has been marketed.
Clinical Trials data for Gaucher Disease is grouped into the following categories:-
The format for each of the following is:
- The name of the trial,
- followed by whether the trial is recruiting patients,
- The trial may not have started and not yet recruiting, or the trial may have started and do not need any more recruitements, or the trial may be completed.
- Note**: Please follow the link to determine the current trial status.
- followed by a list of drug/s, if any, used in the trial
ClinicalTrials.gov has listed the following trials for Gaucher Disease:
- A Study of the Efficacy and Safety of Genz-112638 in Type 1 Gaucher Patients - This study is currently recruiting patients (Current: 23 Nov 2006) - Genz-112638
- Safety and Efficacy of Cerezyme Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease - This study is no longer recruiting patients (Current: 23 Nov 2006) - Cerezyme
- A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease. - This study has been completed (Current: 23 Nov 2006) - Cerezyme (imiglucerase for injection)
- International Collaborative Gaucher Registry (ICGG) - This study is currently recruiting patients (Current: 23 Nov 2006)
- Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease - This study is currently recruiting patients (Current: 23 Nov 2006)
- Convection-Enhanced Delivery of Glucocerebrosidase to Treat Type 2 Gaucher Disease - This study is currently recruiting patients (Current: 23 Nov 2006) - Glucocerebrosidase
- Positron Emission Tomography (PET) Imaging in People With Gaucher Mutations - This study is currently recruiting patients (Current: 23 Nov 2006) - 15-0 H20
- OGT 918-006: A Phase I/II Randomized, Controlled Study of OGT 918 in Patients With Neuronopathic Gaucher Disease - This study is no longer recruiting patients (Current: 23 Nov 2006) - OGT 918
- Effects of Enzyme Replacement in Gaucher's Disease - This study is currently recruiting patients (Current: 23 Nov 2006) - Macrophage-targeted glucocerebrosidase
- Oral Miglustat in Adult Patients With Stable Type 1 Gaucher Disease - This study is currently recruiting patients (Current: 23 Nov 2006) - miglustat
- Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT) - This study is no longer recruiting patients (Current: 23 Nov 2006) - Gene-Activated® human glucocerebrosidase (GA-GCB)
- PEG-Glucocerebrosidase for the Treatment of Gaucher Disease - This study has been completed (Current: 23 Nov 2006) - Lysodase
- Bone Response to Enzyme Replacement in Gaucher's Disease - This study has been completed (Current: 23 Nov 2006)
- Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease - This study is currently recruiting patients (Current: 23 Nov 2006) - glucocerebrosidase
- 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 - This study has been completed (Current: 23 Nov 2006)
- Phase II Randomized Study of Alendronate Sodium for Osteopenia in Patients With Gaucher's Disease - This study is currently recruiting patients (Current: 23 Nov 2006) - alendronate sodium,calcium carbonate,cholecalciferol
- Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation - This study is no longer recruiting patients (Current: 23 Nov 2006)
- Nervous System Degeneration in Glycosphingolipid Storage Disorders - This study is currently recruiting patients (Current: 23 Nov 2006)
- Phase I Single Dose-Escalation Safety Study of prGCD - This study is currently recruiting patients (Current: 23 Nov 2006) - Human Glucocerebrosidase (prGCD)
- A Phase III, Multicenter, Randomized, Double-Blind Trial to Assess the Safety and Efficacy of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients With Gaucher Disease - This study is not yet open for patient recruitment (Current: 23 Nov 2006) - Human Glucocerebrosidase (prGCD)
- Genetic Studies of Lysosomal Storage Disorders - This study is currently recruiting patients (Current: 23 Nov 2006)
- Gene Therapy for Gaucher's and Fabry Disease Using Viruses and Blood-Forming Cells - This study has been completed (Current: 23 Nov 2006)
- Stem Cell Transplant for Inborn Errors of Metabolism - This study is currently recruiting patients (Current: 23 Nov 2006) - Busulfan,Cyclophosphamide,ATG
- Phase II Study of Allogeneic Bone Marrow or Umbilical Cord Blood Transplantation in Patients With Lysosomal or Peroxisomal Inborn Errors of Metabolism - This study is currently recruiting patients (Current: 23 Nov 2006) - anti-thymocyte globulin,cyclophosphamide,cyclosporine,filgrastim,methylprednisolone
The format for each of the following is:
- The drug,
- followed by the name of the trial,
- followed by whether the trial is recruiting patients,
- The trial may not have started and not yet recruiting, or the trial may have started and do not need any more recruitements, or the trial may be completed.
- Note**: Please follow the link to determine the current trial status.
ClinicalTrials.gov has listed the following drugs in trials for Gaucher Disease:
The trial data on this page has been sourced from
