Contact SCGE




Clinical Trial Report

Gene Therapy Trial Report

Summary

A Study of AT132 in Young Children With X-Linked Myotubular Myopathy (XLMTM)


NCTID NCT03199469 (View at clinicaltrials.gov)
Description
Development Status Active
Indication X-Linked Myotubular Myopathy
Disease Ontology Term DOID:0111225
Compound Name AT132
Compound Alias Resamirigene bilparvovec
Compound Description rAAV8-des-hMTM1
Sponsor Astellas Gene Therapies
Funder Type Industry
Recruitment Status
Active not recruiting
Enrollment Count 27
Results Posted View Results

Therapy Information


Target Gene/Variant MTM1
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV8
Editor Type none
Dose 1 1.3E14 vg/kg
Dose 2 3.5E14 vg/kg
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase2, Phase3
Submit Date 2017-06-21
Completion Date 2030-03-31
Last Update 2025-03-17

Participation Criteria


Eligible Age <=5 Years
Standard Ages Child
Sexes Eligible for Study MALE

Locations


No.of Trial Sites 6
Locations Canada,United States,France,Germany

Regulatory Information


Has US IND True
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
Recent Updates On clinical hold since 2021

Resources/Links