Ignite Creation Date:
2024-05-05 @ 12:03 PM
Last Modification Date:
2024-10-26 @ 9:19 AM
Study NCT ID:
NCT00229736
Status:
COMPLETED
Last Update Posted:
2013-12-04
First Post:
2005-09-28
Brief Title:
A Study of AAV-hAADC-2 in Subjects With Parkinsons Disease
Sponsor:
Genzyme a Sanofi Company
Organization:
Sanofi
Study Overview
Official Title:
A Phase1 Open Label Safety Study of Intrastriatal Infusion of Adeno-Associated Virus Encoding Human Aromatic L-Amino Acid Decarboxylase AAV-hAADC-2 in Subjects With Parkinsons Disease AAV-hAADC-2-003
Status:
COMPLETED
Status Verified Date:
2013-12
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:
Safety Study in subjects with Parkinsons Disease
Detailed Description:
In a patient with Parkinsons disease the part of the brain called the substantia nigra progressively loses the ability to send dopamine signals to the striatum
To compensate for the loss of striatal dopamine most patients take L-dopa an approved drug which is converted to dopamine by an essential enzyme - AADC
With time however the brain loses its remaining ability to convert L-dopa to dopamine and thus the drug becomes progressively less effective
In the therapy being studied the gene coding for the enzyme that converts L-dopa to dopamine AADC is inserted into a common non-pathogenic virus AAV to which 90 of humans have been exposed
The AAV will help to transport the AADC into the brain cells
AAV-hAADC-2 the investigational drug being studied is injected into the striatum during a surgical procedure
Patients who undergo the procedure would continue to take L-dopa AAV-hAADC-2 is intended to provide directly to the brain the missing enzyme needed to convert L-dopa to dopamine
To compensate for the loss of striatal dopamine most patients take L-dopa an approved drug which is converted to dopamine by an essential enzyme - AADC
With time however the brain loses its remaining ability to convert L-dopa to dopamine and thus the drug becomes progressively less effective
In the therapy being studied the gene coding for the enzyme that converts L-dopa to dopamine AADC is inserted into a common non-pathogenic virus AAV to which 90 of humans have been exposed
The AAV will help to transport the AADC into the brain cells
AAV-hAADC-2 the investigational drug being studied is injected into the striatum during a surgical procedure
Patients who undergo the procedure would continue to take L-dopa AAV-hAADC-2 is intended to provide directly to the brain the missing enzyme needed to convert L-dopa to dopamine
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