Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam
About the study
This is an open-label, single arm, multi-center study. Approximately 28 participants aged 2 to <18 years will be enrolled stratified as 2 to 5 years and 6 to < 18 years. The study is comprised of 3 periods, Screening (up to 45 days), Treatment (1 day), and Follow-up (52 weeks).
Who can take part
You may be eligible to participate in the study if you meet the following criteria:
INCLUSION CRITERIA
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
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
Study Locations
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How to Apply
Contact the study center to learn if this study is a good match for you.
Study’s details
Contition
Spinal Muscular Atrophy
Age (in years)
2 - 17
Phase
Phase 3
Participants needed
28
Est. Completion Date
Dec 6, 2024
Treatment type
Interventional
Sponsor
Novartis
ClinicalTrials.gov identifier
NCT05386680
Study number
COAV101B12302
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