Ignite Creation Date:
2024-05-05 @ 11:21 AM
Last Modification Date:
2024-10-26 @ 9:05 AM
Study NCT ID:
NCT00005903
Status:
COMPLETED
Last Update Posted:
2008-03-04
First Post:
2000-06-07
Brief Title:
Continuously Infused Recombinant-Methionyl Human Glial Cell Line-Derived Neurotrophic Factor GDNF to Treat Progressive Supranuclear Palsy
Sponsor:
National Institute of Neurological Disorders and Stroke NINDS
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
Dose-Escalation Trial of Continuously Infused Recombinant-Methionyl Human Glial Cell Line-Derived Neurotrophic Factor for the Treatment of PSP
Status:
COMPLETED
Status Verified Date:
2005-05
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:
This study will examine the safety and effectiveness of an experimental drug called GDNF delivered through an investigational device to treat progressive supranuclear palsy PSP The drug will be administered directly into the brain through catheters attached to an infusion pump implanted in the abdomen The study will evaluate 1 if the drug is safe and well tolerated when given by this method 2 the performance of the catheters and pump system and 3 the effects of GDNF on PSP symptoms
PSP is a rare neurological disease that causes eye movement dysfunction muscle rigidity slowness of movement swallowing speech emotional cognitive and personality problems Patients 35 to 75 years old with PSP may be eligible for this study Candidates will be screened at the National Institutes of Health outpatient clinic in Bethesda MD with a medical history physical examination neurological and neuropsychiatric evaluations blood tests electrocardiogram CT scan of the brain and baseline studies including a special eye examination evaluation of symptoms lumbar puncture spinal tap and psychiatric interview
Patients enrolled in the study will undergo surgery to place two catheters into the brain and two infusion pumps under the skin in the upper abdomen The surgery will be performed at Vanderbilt University Medical Center in Nashville TN It will be done under general anesthesia and will require a 3 day hospitalization Within 24 hours after the surgery a CT scan of the brain will be done to ensure the catheters are properly placed Patients return to NIH two weeks after surgery for post-surgery examination and treatment initiation
All patients will receive continuous infusions of GNDF through one catheter and placebo an inactive salt solution through the other for 6 months Half of the patients will receive placebo in the right side of the brain and GNDF in the left and half will receive GNDF in the right side of the brain and placebo in the left All patients will also undergo the following procedures
Brief physical examination and evaluation of symptoms and adverse side effects - every 2 weeks
Blood and urine tests - every 2 weeks for the first 2 months and then every 8 weeks until the end of the study
CT scan to check catheter placement - weeks 9 and 27
Thorough evaluation of symptoms - before beginning treatment and weeks 1 5 9 17 and 27
Neuropsychiatric evaluation - week 27
Special eye examination - weeks 1 and 27
Lumbar puncture - week 27
Additional blood tests to measure drug concentration and antibodies - 6 times during the study
In addition some patients may be asked to have positron emission tomography PET scans or a single photon emission tomography SPECT scan or both
The potential benefit of GDNF is unknown In studies with rats and monkeys GNDF increased the number and size of brain cells containing the chemical messenger dopamine and some movement and balance problems were lessened Earlier studies of GDNF infused into the ventricles of patients with Parkinsons disease showed no benefit and no serious harm
PSP is a rare neurological disease that causes eye movement dysfunction muscle rigidity slowness of movement swallowing speech emotional cognitive and personality problems Patients 35 to 75 years old with PSP may be eligible for this study Candidates will be screened at the National Institutes of Health outpatient clinic in Bethesda MD with a medical history physical examination neurological and neuropsychiatric evaluations blood tests electrocardiogram CT scan of the brain and baseline studies including a special eye examination evaluation of symptoms lumbar puncture spinal tap and psychiatric interview
Patients enrolled in the study will undergo surgery to place two catheters into the brain and two infusion pumps under the skin in the upper abdomen The surgery will be performed at Vanderbilt University Medical Center in Nashville TN It will be done under general anesthesia and will require a 3 day hospitalization Within 24 hours after the surgery a CT scan of the brain will be done to ensure the catheters are properly placed Patients return to NIH two weeks after surgery for post-surgery examination and treatment initiation
All patients will receive continuous infusions of GNDF through one catheter and placebo an inactive salt solution through the other for 6 months Half of the patients will receive placebo in the right side of the brain and GNDF in the left and half will receive GNDF in the right side of the brain and placebo in the left All patients will also undergo the following procedures
Brief physical examination and evaluation of symptoms and adverse side effects - every 2 weeks
Blood and urine tests - every 2 weeks for the first 2 months and then every 8 weeks until the end of the study
CT scan to check catheter placement - weeks 9 and 27
Thorough evaluation of symptoms - before beginning treatment and weeks 1 5 9 17 and 27
Neuropsychiatric evaluation - week 27
Special eye examination - weeks 1 and 27
Lumbar puncture - week 27
Additional blood tests to measure drug concentration and antibodies - 6 times during the study
In addition some patients may be asked to have positron emission tomography PET scans or a single photon emission tomography SPECT scan or both
The potential benefit of GDNF is unknown In studies with rats and monkeys GNDF increased the number and size of brain cells containing the chemical messenger dopamine and some movement and balance problems were lessened Earlier studies of GDNF infused into the ventricles of patients with Parkinsons disease showed no benefit and no serious harm
Detailed Description:
The safety and initial efficacy of the unilateral intralenticular infusion of recombinant-methionyl human glial cell line-derived neurotrophic factor r-metHuGDNF will be compared with the contralateral intralenticular infusion of a placebo solution both continuously administered using chronically implanted catheters and pumps in up to 10 patients with progressive supranuclear palsy Safety will be evaluated by analyzing implant- post-implant- and treatment-emergent adverse events clinical laboratory test results and disease status Efficacy will be studied by comparing left and right sided neurologic function using validated clinical scales as well as by putative surrogate biochemical and radiographic measures
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 |
|---|---|---|---|
| 00-N-0145 | None | None | None |