Contact SCGE




Clinical Trial Report

Gene Therapy Trial Report

Summary

A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease


NCTID NCT04669535 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Tay-Sachs Disease, Sandhoff Disease
Disease Ontology Term DOID:3320; DOID:3323
Compound Name AXO-AAV-GM2
Compound Description AAVrh8-CB-ci-HEXA + AAVrh8-CB-ci-HEXB
Sponsor Terence Flotte
Funder Type Other
Recruitment Status
Completed
Enrollment Count 9
Results Posted Not Available

Therapy Information


Target Gene/Variant HEXA/HEXB
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intracisternal/intrathecal
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type dual AAVrh.8
Editor Type
Dose 1 1E14 vg
Dose 2 Undisclosed 3-part dose escalation
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1
Submit Date 2020-11-23
Completion Date 2024-12-16
Last Update 2025-01-09

Participation Criteria


Eligible Age 6 Months - 12 Years
Standard Ages Child
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 2
Locations United States

Regulatory Information


Has US IND True
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates Sio Gene Therapies licensed this program from the University of Massachusetts Medical School in 2018, Sio terminated this agreement in 2022, Sio dissolved in 2023

Resources/Links