Last updated on July 2019

Gene Transfer Clinical Study in X-Linked Myotubular Myopathy


Brief description of study

This is a Phase 1/2, multinational, open-label, ascending-dose, delayed-treatment concurrent control clinical study to evaluate the safety and preliminary efficacy of AT132 in subjects with X-Linked Myotubular Myopathy aged less than 5 years old. Subjects will receive a single dose of AT132 and will be followed for safety and efficacy for 5 years

Detailed Study Description

This study will evaluate safety and preliminary efficacy of gene transfer in X-Linked Myotubular Myopathy. Subjects will receive a single dose of AT132 delivered intravenously. A maximum of 3 dose levels of AT132 are planned for evaluation in this study. Four subjects will be enrolled at each dose level, including 1 subject at each dose level randomized to control with delayed administration of the investigational medicinal product. Dose escalation to the next dose level will be considered after evaluation of at least 4 weeks of data from all subjects dosed at the current dose level. One of the dose levels will be chosen for dose expansion, and the chosen dose will be administered to all delayed-treatment control subjects.

The primary efficacy endpoint measures will be assessed at Week 48. Subjects will be followed for a total of 5 years after administration of AT132.

This study will utilize an independent Data Monitoring Committee (DMC) that will monitor subject safety and provide recommendations to Audentes regarding dose escalation, dose expansion, and safety matters.

Clinical Study Identifier: NCT03199469

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