motor neuron disease spinal muscular atrophy Werdnig-Hoffmann disease
Conditions
Interventions
None listed
Sponsors
Novartis
Eligibility
Age
2 Years to 17 Years
Inclusion criteria
Inclusion criteria: STRENGTH Inclusion Criteria * Written informed consent * SMA diagnosis based on gene mutation analysis with bi-allelic SMN1 mutations and any copy of SMN2 gene * Aged 2
Exclusion criteria
Exclusion criteria: STRENGTH Exclusion Criteria * Excluding SMA, any medical condition considered clinically significant * Positive for human immunodeficiency virus (HIV), hepatitis B or hepatitis * Anti Adeno Associated Virus Serotype 9 (AAV9) antibody titer using an immunoassay is reported as elevated at Screening (reference to >1:50 or a validated result consistent with being elevated) * Clinically significant abnormalities in test results during screening period and/or at Baseline * Platelet count less than the lower limit of normal (LLN), or platelet transfusion within 1 month at Screening Visit 1 * Clinically significant abnormal coagulation panel results at Screening * Hepatic dysfunction (i.e. alanine aminotransferase (ALT), total bilirubin (TBL), gamma-glutamyl transferase (GGT) or glutamate dehydrogenase (GLDH) > upper limit of normal (ULN) at Screening (with the exception of isolated AST elevation: in the absence of other liver laboratory abnormalities, isolated elevated AST is not considered exclusionary) * Contraindications for lumbar puncture procedure * At Baseline (Day-1), participants are excluded if they received: * nusinersen (Spinraza®) within 4 months at Baseline * risdiplam (Evrysdi®) within 15 days at Baseline * 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 the following: * An active infectious process requiring systemic antiviral or antimicrobial therapy up to 30 days prior to OAV101 administration, or * An active but untreated viral or bacterial infectious process up to 30 days prior to administration of OAV101, or * Any febrile illness up to 30 days prior to administration of OAV101 * Requiring invasive ventilation, awake noninvasive ventilation for > 6 hours during a 24-hour period, noninvasive ventilation for >12 hours during a 24-hour period or requiring tracheostomy, at Screening and up to OAV101 administration * Concomitant use of any of the following medication categories within 90 days prior to administration of OAV101 * Ongoing systemic immunosuppressive therapy (e.g., corticosteroids, cyclosporine, tacrolimus, methotrexate, cyclophosphamide, intravenous immunoglobulin, rituximab), plasmapheresis, immunomodulators (e.g., adalimumab) * History of hypersensitivity to any of the study treatments or its excipients or drugs of similar chemical classes
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary objective of this study is to evaluate the performance of the Navigate Anti-AAV9 Antibody Assay using serum specimens in subjects with SMA in Novartis clinical studies COAV101B12301 *STEER* and COAV101B12302 *STRENGTH*. The primary measures of performance for the Navigate Assay are linked to the primary and secondary endpoints of Novartis clinical studies COAV101B12301 *STEER* and COAV101B12302 *STRENGTH*: STEER Primary Objective: EFFICACY: To compare the efficacy of OAV101 IT vs. sham control as measured by the change from baseline in HFMSE total score STRENGTH Primary Objective: To characterize the safety and tolerability of OAV101 IT over a 52-week period in patients with SMA aged 2 to 12 years who have discontinued treatment with nusinersen (Spinraza®) or risdiplam (Evrysdi®). | — |
Countries
Netherlands
Outcome results
None listed