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About
This was a multinational, open-label, ascending-dose, delayed-treatment concurrent control clinical study to evaluate the safety and efficacy of resamirigene bilparvovec in participants with X-Linked Myotubular Myopathy aged less than 5 years old. Participants received a single dose of resamirigene bilparvovec and followed for safety and efficacy for 10 years
Full description
This study evaluated safety and efficacy of gene transfer in X-Linked Myotubular Myopathy. Participants received a single dose of resamirigene bilparvovec delivered intravenously.
ASPIRO was being conducted in two parts. Part 1 was a dose escalation phase that was evaluating the preliminary safety and efficacy of Resamirigene bilparvovec at doses of 1.3x10^14 vg/kg and 3.5x10^14 vg/kg. Part 2 of ASPIRO was a pivotal expansion cohort designed to confirm the safety and efficacy of resamirigene bilparvovec at a dose of 3.5x10^14 vg/kg. The pivotal expansion cohort enrolled eight participants, consisting of four age-matched pairs (within +/- 6 months of age). One participant from each pair was randomized to receive a single dose of resamirigene bilparvovec at 3.5x10^14 vg/kg, and the other served as a delayed treatment control. Eligible delayed treatment control participants administered resamirigene bilparvovec after that individual participant completed the Week 24 visit as a delayed treatment control.
The primary efficacy endpoint measures assessed at Week 24. Participants were followed for a total of 10 years after administration of resamirigene bilparvovec. Only Part 1 of the study was reported.
This study utilized an independent Data Monitoring Committee (DMC) that monitored participant safety and provided recommendations to Astellas regarding dose escalation, dose expansion, and safety matters.
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Inclusion criteria
Part 1: Subject requires some mechanical ventilatory support (e.g., ranging from 24 hours per day full time mechanical ventilation, to noninvasive support such as continuous positive airway pressure (CPAP) or bilevel positive airway pressure (BiPAP) during sleeping hours).
Part 2: Subject requires invasive mechanical ventilatory support ranging from 20 - 24 hours per day at screening (confirmed by daytime polysomnographic study).
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27 participants in 2 patient groups
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Data sourced from clinicaltrials.gov
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