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)

Phase IIIb, open-label, single-arm, multi-center study to evaluate the safety, tolerability and efficacy of OAV101 administered intrathecally (1.2 x 10^14 vector genomes) to participants 2 to < 18 years of age with spinal muscular atrophy (SMA) who have discontinued treatment with nusinersen (Spinraza) or risdiplam (Evrysdi)

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCT2073220094

Enrollment

Registered

2023-01-22

Start date

2023-01-30

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Spinal muscular atrophy

Interventions

OAV101 administered as a single IT dose of 1.2 x 10^14 vector genomes

Eligibility

Sex/Gender

All

Inclusion 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

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 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

Design outcomes

Primary

Measure	Time frame
Number and percentage of participants reporting AEs, related AEs, SAEs, and AESIs	—

Countries

Australia, Belgium, Canada, France, Japan

Contacts

Public ContactHideki Maruyama

Novartis Pharma. K.K.

rinshoshiken.toroku@novartis.com+81-120-003-293

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026