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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
Indication Myotubular Myopathy, X-Linked
Compound Name Resamirigene bilparvovec (AT132), rAAV8-des-hMTM1
Sponsor Astellas Gene Therapies
Funder Type Industry
Status
Active not recruiting
Enrollment Count 27

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
Dose 1 1.3 x 10^14 vg/kg
Dose 2 3.5 x 10^14 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 2024-10-29

Participation Criteria


Eligible Age <=5 Years
Standard Ages Child
Eligible Sex MALE

Locations


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

Regulatory Information


Has US IND True
Recent Updates On clinical hold since 2021

Resources/Links