Ignite Creation Date:
2024-05-06 @ 5:27 PM
Last Modification Date:
2024-10-26 @ 2:29 PM
Study NCT ID:
NCT05317780
Status:
NO_LONGER_AVAILABLE
Last Update Posted:
2023-08-23
First Post:
2022-03-24
Brief Title:
Canavan-Single Patient IND
Sponsor:
University of Florida
Organization:
University of Florida
Study Overview
Official Title:
Expanded Access Trial of Systemic Delivery of Aspartoacylase ASPA rAAV9-CB6-AspA Gene Vector in a Single Patient With Canavan Disease
Status:
NO_LONGER_AVAILABLE
Status Verified Date:
2023-08
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:
A recombinant virus vector constructed from adeno-associated virus AAV has been engineered to carry the human aspartoacylase ASPA gene expressed from a modified CMV-enhancer chicken β-actin CB6 promoter The construct has been shown to produce ASPA in animal models of Canavan disease which closely match the proposed human study The proposed clinical trial is an open label expanded access study administering rAAV9-CB6-AspA gene vector by simultaneous systemic and intracerebroventricular routes to a single human subject 18-24 months of age with Canavan disease The subject will also receive immune modulation to transiently ablate B-cells Rituximab and modulate T-cell response Sirolimus prior to the initial exposure to AAV9 Given the null AspA mutations of the subject and current AAV seronegative status this regimen will allow for later exposure to the therapeutic vector if needed and block any immuno-toxicity in the CNS The goal of this study is to measure the safety and efficacy of AAV-mediated gene therapy as a treatment approach for neuronal pathology in Canavan disease The subject will act as their own control and change from baseline will be assessed in regards to levels of brain NAA brain water content and morphology improved clinical status and peripheral levels of NAA Safety parameters measured in this study will include serum chemistries and hematology urinalysis physical assessments whole blood assay for vector genomes immunologic response to ASPA and AAV as well as reported subject symptom history
Detailed Description:
None
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