Status:
COMPLETED
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
Lead Sponsor:
Novartis Pharmaceuticals
Conditions:
Spinal Muscular Atrophy
Eligibility:
All Genders
2-17 years
Phase:
PHASE3
Brief 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...
Detailed Description
Eligible participants received a single OAV101B administration of 1.2x1014 vector genomes on Day 1 (Treatment period) and were followed for a period of 52 weeks. Participants were admitted to the hos...
Eligibility Criteria
Inclusion
- 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
Exclusion
Key Trial Info
Start Date :
January 12 2023
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 29 2024
Estimated Enrollment :
27 Patients enrolled
Trial Details
Trial ID
NCT05386680
Start Date
January 12 2023
End Date
November 29 2024
Last Update
October 16 2025
Active Locations (13)
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1
Boston Childrens Hospital
Boston, Massachusetts, United States, 02215
2
Child Hosp Of The Kings Daughters
Norfolk, Virginia, United States, 23507
3
University of Wisconsin Madison Medical School
Madison, Wisconsin, United States, 53792-7375
4
Novartis Investigative Site
Parkville, Victoria, Australia, 3052