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)

JRCT ID: jRCT2073220094

Registered date:22/01/2023

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: Spinal muscular atrophy
• Date of first enrollment: 30/01/2023
• Target sample size: 4
• Countries of recruitment: Australia,Japan,Belgium,Japan,Canada,Japan,France,Japan
• Study type: Interventional
• Intervention(s): OAV101 administered as a single IT dose of 1.2 x 10^14 vector genomes

Outcome(s)

• Primary Outcome: Number and percentage of participants reporting AEs, related AEs, SAEs, and AESIs

Key inclusion & exclusion criteria

• Age minimum: >= 2age old
• Age maximum: < 12age old
• Gender: Both

Include 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

Exclude 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

Related Information

• Primary Sponsor: Hirano Takamitsu
• Secondary ID(s): NCT05386680