Ignite Creation Date:
2024-05-05 @ 11:58 AM
Last Modification Date:
2024-10-26 @ 9:17 AM
Study NCT ID:
NCT00195143
Status:
COMPLETED
Last Update Posted:
2008-03-27
First Post:
2005-09-15
Brief Title:
Safety Study of Subthalamic Nucleus Gene Therapy for Parkinsons Disease
Sponsor:
Neurologix Inc
Organization:
Neurologix Inc
Study Overview
Official Title:
Phase I Study of Subthalamic GAD Gene Transfer in Medically Refractory Parkinsons Disease Patients
Status:
COMPLETED
Status Verified Date:
2005-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The purpose of this study is to determine the safety of using a modified virus to transfer a gene called GAD into a region of the brain called the subthalamic nucleus in patients with advanced Parkinsons disease The overall goal of this approach is to ultimately normalize the flow of information in several brain regions responsible for movement to ultimately improve function in patients with this disorder The current study is primarily designed to evaluate the safety of this approach but patients are also being monitored for possible signs of effectiveness as well
Detailed Description:
This study involves treatment of patients with medically refractory Parkinsons disease PD with gene therapy The patients are chosen from a population of patients who would normally be candidates for standard deep brain stimulation DBS surgery for PD These patients respond to medical therapy but develop substantially reduced responses over time often with severe fluctuations in their condition between a functional and severely non-functional state Some patients also develop dose-limiting side effects from medication including involuntary movements called dyskinesias and nightmares When there are no medical contraindications DBS is often performed in these patients to try to quiet hyperactive brain regions such as the subthalamic nucleus STN In PD the STN is overactive due to a loss of GABA inputs to this region which normally reduces neuronal firing In turn the STN drives other brain regions including the globus pallidus GPi and substantia nigra SNr which are also hyperactive and which also have reduced GABA inputs The goal of this gene therapy trial is to introduce the gene for glutamic acid decarboxylase GAD into the STN using an adeno-associated virus AAV vector in order to permit the STN to produce its own GABA as well as release GABA into the GPi and SNr targets which also have reduced GABA inputs This is anticipated to restore a more normal pattern of information flow from this basal ganglia circuit to the thalamus and higher cortical structures in order to reduce the motor symptoms of PD while eliminating complications arising from inserting DBS electrodes and batteries into the human body
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