Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 IT or OAV101 IV in Clinical Trials

What this Trial Is About

This research investigates the long-term safety and effectiveness of OAV101 in patients with Spinal Muscular Atrophy (SMA) who previously took part in OAV101 clinical trials. It is a global, multi-center study designed to follow these patients for five years after enrolling to monitor ongoing outcomes related to the treatment. The study is sponsored by Novartis Pharmaceuticals and focuses on continued safety assessments and developmental milestones in this patient group. Participants in this study received OAV101 either as an intravenous (IV) infusion or an intrathecal (IT) injection during earlier trials. The study includes a baseline visit followed by two follow-up periods: the first two years involve visits every six months, while years three to five have annual visits. All assessments take place at the investigational sites. During the study, participants will undergo various evaluations including safety monitoring for serious adverse events and specific adverse events of interest. Researchers will also track developmental milestones and changes in motor function using standardized scales such as the Hammersmith Functional Motor Scale – Expanded and the Revised Upper Limb Module. Vital signs including blood pressure, respiratory rate, pulse, temperature, and oxygen saturation will be measured regularly. The total participation duration is five years from baseline enrollment.

Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials