Study Details

Gene Transfer Clinical Study in X-Linked Myotubular Myopathy

Recruitment complete
The study is ongoing, and participants are receiving an intervention or being examined, but potential participants are not currently being recruited or enrolled. ID

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Astellas Study ID

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X-Linked Myotubular Myopathy


These clinical trials are usually the first time an experimental treatment is studied in a small group of people.

Phase 1


N/A - 5 Years






A type of clinical study in which participants are assigned to groups that receive one or more intervention/treatment (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


Trial Dates

Aug 2017 - Oct 2030


None (Open Label)

Enrollment number


ASPIRO: A Phase 1/2/3, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Clinical Study to Evaluate the Safety and Efficacy of AT132, an AAV8-Delivered Gene Therapy in X-Linked Myotubular Myopathy (XLMTM) Patients

Study summary

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Ann & Robert H Lurie Children's Hospital of Chicago

Chicago, United States, 60611

UCLA Medical Center

Los Angeles, United States, 90095

Hopital Armad Trousseau

Paris, France, 75012

Hospital for Sick Children

Toronto, Canada, M5G0A4

National Institute of Neurological Disorders and Stroke/NIH Porter

Bethesda, United States, 208892

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

München, Germany, 80337