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Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA)
Bu çalışma, spinal musküler atrofi (spinal muscular atrophy, SMA) olan çocuklarda damar içi (intravenöz) OAV101 gen terapisinin güvenliğini ve etkinliğini incelemiştir. Çalışmaya, iki kopyalı SMN1 mutasyonu ile SMA tanısı doğrulanmış, tedavi anında 24 aya kadar olan ve 17 kg altında olan çocuklar alınmıştır. Faz 4 aşamasında yürütülmüş ve tamamlanmıştır.
Yukarıdaki özet, resmî kaydın sade dile aktarılmış halidir. Orijinal İngilizce başlık referans için gösterilir.
Kriterler ClinicalTrials.gov kaydından orijinal İngilizce haliyle alınmıştır. Uygunluk kararını yalnızca deneyi yürüten ekip verebilir.
Inclusion Criteria: 1. Written informed consent/assent obtained prior to any assessment performed 2. Symptomatic SMA diagnosis based on gene mutation analysis with bi-allelic SMN1 mutations (deletion or point mutations) and any copy of SMN2 gene. 3. Age ≤ 24 months of age at time of treatment 3\. Weight ≤17 kg at the time of Screening Period 4. Naïve to treatment or have discontinued an approved drug/therapy 5. Up-to date on recommended childhood vaccinations and RSV prophylaxis with palivizumab (also known as Synagis), per local standard of care Key Exclusion Criteria: 1. Previous use of OAV101 or any AAV9 gene therapy 2. Participant with history of aspiration pneumonia or signs of aspiration (eg, coughing or sputtering of food) within 4 weeks prior to Screening 3. Participant dependent on gastrostomy feeding tube for 100% of nutritional intake. 4. Anti-AAV9 antibody titer \> 1:50 as determined by ligand binding immunoassay at the time of screening 5. Inability to take corticosteroids 6. Concomitant use of immunosuppressive therapy, plasmapheresis, immunomodulators such as adalimumab, or immunosuppressive therapy within 3 months prior to gene replacement therapy (eg, cyclosporine, tacrolimus, methotrexate, rituximab cyclophosphamide, IV immunoglobulin) 7. Hepatic dysfunction (i.e. AST, ALT, bilirubin, GGT or GLDH, ≥ ULN; CTCAE ≥ 1) at Screening (with the exception of isolated AST elevation: in the absence of other liver laboratory abnormalities, isolated AST elevation is not considered exclusionary) 8. Previously treated with nusinersen (Spinraza®) within 4 months prior to Screening 9. Previously treated with risdiplam (EvrysdiTM) within 15 days prior to Screening (washout period of at least 5 half-lives before Screening)