Ignite Creation Date:
2024-05-05 @ 10:00 AM
Last Modification Date:
2024-10-26 @ 9:02 AM
Study NCT ID:
NCT00001410
Status:
COMPLETED
Last Update Posted:
2008-03-04
First Post:
1999-11-03
Brief Title:
PEG-Glucocerebrosidase for the Treatment of Gaucher Disease
Sponsor:
National Institute of Mental Health NIMH
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
A Phase I and II Study of PEG-Glucocerebrosidase in Patients With Type 1 or Type 3 Gaucher Disease
Status:
COMPLETED
Status Verified Date:
2001-12
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Gaucher disease is a lysosomal storage disease resulting from glucocerebroside accumulation in macrophages due to a genetic deficiency of the enzyme glucocerebrosidase It may occur in patients of all ages The condition is marked by enlargement of the liver and spleen hepatosplenomegaly low blood and platelet counts and bone abnormalities The condition is passed from generation to generation on via autosomal recessive inheritance There are actually three types of Gaucher disease
Type I is the most common form It is a chronic non-neuronopathic form meaning the disease does not affect the nervous system The symptoms of type I can appear at any age
Type 2 Gaucher disease presents prenatally or in infancy and usually results in death for the patient Type 2 is an acute neuronopathic form and can affect the brain stem It is the most severe form of the disease
Type 3 Gaucher disease is also neuronopathic however it is subacute in nature This means the course of the illness lies somewhere between long-term chronic and short-term acute
Currently there is not a cure for Gaucher disease Treatment for the disease has traditionally been supportive In some severely affected patients bone-marrow transplants have corrected the enzyme deficiency but it is considered a high-risk procedure and recovery can be very slow Enzyme replacement therapy is another therapy option and has been approved by the Food and Drug Administration FDA for use in type 1 patients
PEG-glucocerbrosidase is a drug designed to clear out the accumulation of lipid glucocerebroside from the blood stream The drug is actually an enzyme attached to large molecules called polyethylene glycol PEG The large molecules of PEG allow the enzyme to remain in the blood stream for long periods of time By modifying glucocerebrosidase with PEG it is believed that smaller doses will be required meaning a reduction in cost for the patient and more convenient administration of the drug The purpose of this study is to evaluate the effects and safety of enzyme replacement therapy using PEG- glucocerebrosidase for the treatment of Gaucher disease
Type I is the most common form It is a chronic non-neuronopathic form meaning the disease does not affect the nervous system The symptoms of type I can appear at any age
Type 2 Gaucher disease presents prenatally or in infancy and usually results in death for the patient Type 2 is an acute neuronopathic form and can affect the brain stem It is the most severe form of the disease
Type 3 Gaucher disease is also neuronopathic however it is subacute in nature This means the course of the illness lies somewhere between long-term chronic and short-term acute
Currently there is not a cure for Gaucher disease Treatment for the disease has traditionally been supportive In some severely affected patients bone-marrow transplants have corrected the enzyme deficiency but it is considered a high-risk procedure and recovery can be very slow Enzyme replacement therapy is another therapy option and has been approved by the Food and Drug Administration FDA for use in type 1 patients
PEG-glucocerbrosidase is a drug designed to clear out the accumulation of lipid glucocerebroside from the blood stream The drug is actually an enzyme attached to large molecules called polyethylene glycol PEG The large molecules of PEG allow the enzyme to remain in the blood stream for long periods of time By modifying glucocerebrosidase with PEG it is believed that smaller doses will be required meaning a reduction in cost for the patient and more convenient administration of the drug The purpose of this study is to evaluate the effects and safety of enzyme replacement therapy using PEG- glucocerebrosidase for the treatment of Gaucher disease
Detailed Description:
The purpose of this clinical study is to evaluate the biochemical and therapeutic effects and safety of enzyme replacement therapy using polyethyleneglycol PEG modified glucocerebrosidase for the treatment of Gaucher disease and to evaluate the benefit to risk ratio The study is designed to determine the safety and efficacy in Gaucher patients of recombinantly produced human glucocerebrosidase which is PEG modified Parameters to be monitored include hemoglobin platelet counts organ size and extent of bony involvement Pharmacokinetic pharmacodynamic and antibody studies will also be evaluated
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 94-M-0014 | None | None | None |