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

X-linked Myotubular Myopathy (XLMTM)

Primary Efficacy Endpoint: • Change from baseline in hours of ventilation support at Week 24

Key Secondary Efficacy Endpoint: Percentage of subjects achieving functionally independent sitting for at least 30 seconds at Week 24, Other Secondary Efficacy Endpoints: • Time to reduction in required ventilator support to ≤ 16 hours a day (only in subjects who require invasive ventilation) at Week 24, • Change from baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) at Week 24, • Change from baseline in maximal inspiratory pressure (MIP) at Week 24, • Change from baseline in quantitative analysis of myotubularin expression in the muscle biopsy at Week 24, • Change from baseline in quality of life assessments at Week 24 (ie, the Assessment of Caregiver Experience with Neuromuscular Disease [ACEND] and Pediatric Quality of Life Inventory [PedsQL]), • Number (%) of age-appropriate clinically relevant gross motor function milestones attained through Week 24, • Percentage of subjects achieving full ventilator independence at Week 24, •Survival, 10 Safety Endpoints: • Adverse events (AEs), serious AEs (SAEs), and findings from safety laboratory tests, 12-lead ECG, echocardiograms (ECHOs), vital signs, growth parameters, physical examinations, liver ultrasounds, antibody formation (anti AAV8, anti MTM1), viral shedding , annualized hospitalization rate , annualized respiratory and non-respiratory SAE rate, and length of stay per hospitalization

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