This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi. Participants will roll over from their respective previous (parent) study into this long-term study for continuous monitoring of safety as well as monitoring of continued efficacy and durability of response to onasemnogene abeparvovec-xioi treatment.
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Number of Participants Who Reach Developmental Milestones
Timeframe: Up to 5 years
Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score
Timeframe: Up to 5 years
Number of Participants Who Experience a Clinically Significant Change From Baseline in Pulmonary Assessment Results and Require Ventilatory Support
Timeframe: Up to 15 years
Number of Participants Who Experience Swallowing Dysfunction and Require Nutritional Support
Timeframe: Up to 5 years
Number of Participants Who Experience a Clinically Significant Change from Baseline in Physical Examination Findings
Timeframe: Up to 5 years
Number of Participants Who Experience a Clinically Significant Change From Baseline in Vital Signs Measurements
Timeframe: Up to 5 years
Change From Baseline in Height Measurements
Timeframe: Up to 5 years
Change From Baseline in Weight Measurements
Timeframe: Up to 5 years
Number of Participants Who Experience a Clinically Significant Change From Baseline in Clinical Laboratory Assessments
Timeframe: Up to 5 years
Number of Participants Who Experience a Clinically Significant Change From Baseline in Cardiac Assessments
Timeframe: Up to 5 years
Number of Participants Who Experience a Clinically Significant Change From Baseline in Observational Phase Questionnaire Results
Timeframe: Year 6 to Year 15
Number of Participants Who Experience at Least One Serious Adverse Event (SAE)
Timeframe: Up to 15 years
Number of Participants Who Experience at Least One Adverse Event of Special Interest (AESI)
Timeframe: Up to 15 years
Change From Baseline in Bayley Scales of Infant and Toddler Development
Timeframe: Up to 42 months, 15 days of age
Change From Baseline in Revised Upper Limb Module (RULM) Score
Timeframe: Up to 5 years
Change From Baseline in Cogstate Computerized Cognitive Battery Performed in Age 48 Months and Older
Timeframe: Up to 5 years
Change From Baseline in Clinical Evaluation of Language Fundamentals Fifth Edition (CELF-5) Performed in All Participants 5 to 21 Years of Age
Timeframe: Up to 5 years
Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND)
Timeframe: Up to 5 years
Number of Participants With Concomitant Medications Overall and by Type of Medications
Timeframe: Up to 5 years
Number of Participants With Other SMA Therapies Overall and by Type of Medications
Timeframe: Year 6 to Year 15