Phase 3 N=27 Treatment

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

Spinal Muscular Atrophy

Bottom Line

View on ClinicalTrials.gov: NCT05386680 ↗

Enrolled (actual)

Serious AEs

14.8%

Results posted

Jun 2025

Primary outcome: Primary: Overview of Treatment-emergent Adverse Events by Age Subgroup — 27; 13; 4; 0 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: OAV101 (Genetic)
Age: Pediatric · 2+ yrs
Sex: All
Sponsor: Novartis Pharmaceuticals
Primary completion: Nov 2024

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	—

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).

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT05386680). 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.