Yükleniyor... / Loading...
This study examines the safety and effectiveness of an intravenously delivered gene therapy called OAV101 (AVXS-101) in children with spinal muscular atrophy (SMA). It enrolled children with confirmed bi-allelic SMN1 mutations on gene testing who were within a defined weight range. It was conducted in Phase 3 and has been completed.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion * Symptomatic SMA diagnosis based on gene mutation analysis with bi-allelic survival motor neuron 1 (SMN1) mutations (deletion or point mutations) and any copy of the survival motor neuron 2 (SMN2) gene. * Weight ≥ 8.5 kg and ≤ 21 kg at the time of Screening Visit 2 * Naive to treatment or have discontinued an approved drug/therapy Exclusion: * Previous OAV101 use or previous use of any adeno-associated virus serotype 9 (AAV9) gene therapy * BMI \< 3rd percentile * Participant with history of aspiration pneumonia or signs of aspiration * Elevated anti-AAV9 antibody * History of gene therapy, hematopoietic transplantation, or solid organ transplantation * Inability to take corticosteroids * Concomitant use of immunosuppressive therapy * Requiring invasive ventilation, tracheostomy or awake non-invasive ventilation 9. Administration of vaccines 2 weeks prior to infusion of OAV101 * Awake hypoxemia or awake oxygen saturation level decrease * Hepatic dysfunction * Presence of a confirmed or suspected infection * If previously treated with disease modifying therapy, specified washout times apply * Documented any parental consanguinity.