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This Phase IIIb open-label, single-arm, multi-center study evaluated the safety, tolerability, and efficacy of OAV101 (AVXS-101, Zolgensma) given intrathecally to people aged 2 to under 18 with spinal muscular atrophy (SMA) after they discontinued treatment with nusinersen or risdiplam. It aimed to enroll roughly 28 participants in each of two age brackets: 2 to under 6 years and 6 to under 18 years. Eligibility required an SMA diagnosis, symptoms as defined in the protocol, and at least four loading doses of nusinersen (Spinraza®) or at least 3 months of risdiplam (Evrysdi®) at screening.
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 Criteria * SMA diagnosis * Aged 2 to \< 18 years * Have had at least four loading doses of nusinersen (Spinraza®) or at least 3 months of treatment with risdiplam (Evrysdi®) at Screening * Must have symptoms of SMA as defined in the protocol Exclusion Criteria: * Anti Adeno Associated Virus Serotype 9 (AAV9) antibody titer using an immunoassay is reported as elevated * Clinically significant abnormalities in test results during screening * Contraindications for lumbar puncture procedure * At Baseline, participants are excluded if they received: * nusinersen (Spinraza®) or * risdiplam (Evrysdi®) within a defined timeframe * Vaccinations 2 weeks prior to administration of OAV101 * Hospitalization for a pulmonary event, or for nutritional support within 2 months prior to Screening or inpatient major surgery planned. * Presence of an infection or febrile illness up to 30 days prior to administration of OAV101 * Requiring invasive ventilation