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

PHASE2/PHASE3Active, not recruitingINTERVENTIONAL
Enrollment

27

Participants

Timeline

Start Date

August 2, 2017

Primary Completion Date

September 9, 2021

Study Completion Date

March 31, 2030

Conditions
X-Linked Myotubular Myopathy
Interventions
GENETIC

Resamirigene bilparvovec

Resamirigene bilparvovec is an AAV8 vector containing a functional copy of the human MTM1 (hMTM1) gene.

Trial Locations (6)

60611

Ann & Robert H Lurie Children's Hospital of Chicago, Chicago

75012

Hopital Armad Trousseau, Paris

80337

Kinderklinik und Kinderpoliklinik im Dr. Von Haunerschen Kinderspital Klinikum der Universitat Munchen, München

90095

UCLA Medical Center, Los Angeles

208892

National Institute of Neurological Disorders and Stroke/NIH Porter, Bethesda

M5G0A4

Hospital for Sick Children, Toronto

Sponsors
All Listed Sponsors
lead

Astellas Gene Therapies

INDUSTRY

NCT03199469 - A Study of AT132 in Young Children With X-Linked Myotubular Myopathy (XLMTM) | Biotech Hunter | Biotech Hunter