Safety, tolerability and efficacy study of OAV101 administered intrathecally to participants 2 to < 18 years of age with spinal muscular atrophy (SMA) who have discontinued treatment with nusinersen (Spinraza) or risdiplam (Evrysdi)
- Conditions
- Spinal muscular atrophy
- Registration Number
- JPRN-jRCT2073220094
- Lead Sponsor
- Hirano Takamitsu
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 4
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
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
4. At Baseline, participants are excluded if they received:
Nusinersen (Spinraza) or risdiplam (Evrysdi) within a defined timeframe
5. Vaccinations 2 weeks prior to administration of OAV101
6. Hospitalization for a pulmonary event, or for nutritional support within 2 months prior to Screening or inpatient major surgery planned.
7. Presence of an infection or febrile illness
8. Requiring invasive ventilation
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method umber and percentage of participants reporting AEs, related AEs, SAEs, and AESIs
- Secondary Outcome Measures
Name Time Method