Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-25 @ 2:42 AM
Ignite Modification Date:
2025-12-25 @ 2:42 AM
NCT ID:
NCT05073133
Eligibility Criteria:
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)
Healthy Volunteers:
False
Sex:
ALL
Maximum Age:
24 Months
Study:
NCT05073133
Study Brief:
Protocol Section:
NCT05073133