Phase 3
Completed N=27
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
Source: ClinicalTrials.gov NCT05386680 ↗Enrolled (actual)
27
Serious AEs
14.8%
Results posted
Jun 2025
Primary outcomePrimary: Overview of Treatment-emergent Adverse Events by Age Subgroup — 27; 13; 4; 0 Participants
◆ Published Evidence
Emerging
10citations · ~10 / year
Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial.
Summary
This was a Phase IIIb open-label, single arm, multi-center study to evaluate the safety, tolerability and efficacy of OAV101B in participants with SMA aged 2 to <18 years after the discontinuation of treatment with nusinersen or risdiplam. The study aimed to enroll approximately 28 participants across each of 2 age brackets (2 to <6 years, and 6 to <18 years).
Linked Publications
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Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Overview of Treatment-emergent Adverse Events by Age Subgroup |
27; 13; 4; 0; 1; 0 | — |
| PRIMARY Treatment-emergent Adverse Events Related to Treatment by System Organ Class, Preferred Term, Age Subgroup (>= 10%) |
13; 7; 6; 5; 3; 6 | — |
| PRIMARY Adverse Events of Special Interest by System Organ Class, Preferred Term, Age Subgroup |
4; 3; 1; 8; 3; 2 | — |
| SECONDARY Change From Baseline at Week 52 Visit in the HFMSE Total Score - Mean (SD) |
0.17 | — |
| SECONDARY Change From Baseline at Week 52 Visit in the HFMSE Total Score - LS Means |
1.05 | — |
| SECONDARY Change From Baseline at Week 52 Visit in the RULM Total Score - Mean (SD) |
0.29 | — |
| SECONDARY Change From Baseline at Week 52 Visit in the RULM Total Score - LS Means |
0.59 | — |
| SECONDARY Change From Baseline at Week 52 Visit in Assessment of Caregiver Experience in ACEND Instrument Score - Mean (SD) |
1.43 | — |
| SECONDARY Change From Baseline at Week 52 Visit in Assessment of Caregiver Experience in ACEND Instrument Score - LS Means |
1.06 | — |
Eligibility 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
- 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
Data sourced from ClinicalTrials.gov (NCT05386680) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.