Ignite Creation Date:
2024-05-06 @ 3:33 PM
Last Modification Date:
2024-10-26 @ 1:51 PM
Study NCT ID:
NCT04669535
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2023-12-14
First Post:
2020-11-23
Brief Title:
A Dose-escalation and Safety Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease
Sponsor:
Terence Flotte
Organization:
University of Massachusetts Worcester
Study Overview
Official Title:
A Two-Stage Dose-Escalation and Safety Efficacy Study of Bilateral Intraparenchymal Thalamic and IntracisternalIntrathecal Administration of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2023-12
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:
The AXO-GM2-001 study is an open-label two-stage clinical trial designed to evaluate safety and dose-escalation Stage 1 and safety and efficacy Stage 2 of a bilateral thalamic and intracisternalintrathecal infusion of AXO-AAV-GM2 in pediatric participants with GM2 Gangliosidosis also known as Tay-Sachs or Sandhoff Diseases a set of rare and fatal pediatric neurodegenerative genetic disorders caused by defects in the HEXA leading to Tay-Sachs disease or HEXB leading to Sandhoff disease genes that encode the two subunits of the β-hexosaminidase A HexA enzyme AXO-AAV-GM2 is an investigational gene therapy that aims to restore HexA function by introducing a functional copy of the HEXA and HEXB genes via co-administration of two vectors utilizing the neurotropic adeno-associated virus recombinant human 8 serotype AAVrh8 capsid carrying the human HEXA or HEXB cDNA
The trial is expected to enroll pediatric participants with Tay-Sachs or Sandhoff Diseases where infantile-onset participants will range from 6 months to 20 months old and juvenile-onset participants will range from 2 years to 12 years old
The trial is expected to enroll pediatric participants with Tay-Sachs or Sandhoff Diseases where infantile-onset participants will range from 6 months to 20 months old and juvenile-onset participants will range from 2 years to 12 years old
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None