For Healthcare Professionals

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

  1. SMA diagnosis
  2. Aged 2 to < 18 years
  3. Have had at least four loading doses of nusinersen (Spinraza®) or at least 3 months of treatment with risdiplam (Evrysdi®) at Screening
  4. Must have symptoms of SMA as defined in the protocol


Exclusion Criteria:

  1. Anti Adeno Associated Virus Serotype 9 (AAV9) antibody titer using an immunoassay is reported as elevated
  2. Clinically significant abnormalities in test results during screening
  3. Contraindications for lumbar puncture procedure

At Baseline, participants are excluded if they received:

  1. nusinersen (Spinraza®) or
  2. risdiplam (Evrysdi®) within a defined timeframe
  3. Vaccinations 2 weeks prior to administration of OAV101
  4. Hospitalization for a pulmonary event, or for nutritional support within 2 months prior to Screening or inpatient major surgery planned.
  5. Presence of an infection or febrile illness up to 30 days prior to administration of OAV101
  6. Requiring invasive ventilation
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Study Locations

Enter your ZIP code/Postal code/PIN code to locate study sites near you:

How to Apply

Contact the study center to learn if this study is a good match for you.

Study’s details


Spinal Muscular Atrophy

Age (in years)

2 - 17


Phase 3

Participants needed


Est. Completion Date

Dec 6, 2024

Treatment type



Novartis identifier


Study number


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