A Phase IIIb, open-label, single-arm, single-dose, multicenter study to evaluate the safety, tolerability and efficacy of gene replacement therapy with intravenous OAV101 (AVXS-101) in pediatric patients with spinal muscular atrophy (SMA) - SMART

OVARTIS PHARMA AG0 sites30 target enrollmentAugust 17, 2021

Overview

No summary available.

Registry

who.int

Start Date

August 17, 2021

End Date

TBD

Last Updated

4 years ago

Study Type

Interventional clinical trial of medicinal product

Sex

All

Sponsor

•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\. Weight \= 8\.5 kg and \= 21 kg at the time of Screening Visit 2
•4\. Naive 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
•Are the trial subjects under 18? yes
•Number of subjects for this age range:
•F.1\.2 Adults (18\-64 years) no
•F.1\.2\.1 Number of subjects for this age range
•F.1\.3 Elderly (\>\=65 years) no

•Participants meeting any of the following criteria are not eligible for inclusion in this study.
•1\. Previous OAV101 use or previous use of any AAV9 gene therapy
•2\. BMI \< 3rd percentile based on WHO Child Growth Standard
•3\. Participant with history of aspiration pneumonia or signs of aspiration (eg, coughing or sputtering of food) within 4 weeks prior to screening
•4\. Anti\-AAV9 antibody titer \> 1:50 as determined by ligand binding immunoassay at the time of screening
•5\. History of gene therapy, hematopoietic transplantation, or solid organ transplantation
•6\. Inability to take corticosteroids
•7\. 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)
•8\. Requiring invasive ventilation, tracheostomy or awake non\-invasive ventilation (standard of care nocturnal BiPAP is not considered exclusionary)
•9\. Administration of vaccines 2 weeks prior to infusion of OAV101