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

Study Identifier:
COAV101B12302
ClinicalTrials.gov Identifier:
NCT05386680
EudraCT Identifier:
N/A
EU CT Number:
N/A
Sponsor:
Novartis Pharmaceuticals
Study Complete

Trial Results

Study Protocol
Available Languages: English
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Statistical Analysis Plan
Available Languages: English
View PDF

Study Details

Medical Condition
  • All Spinal Muscular Atrophies
Study Drug
    Date
    Jan 2023 - Nov 2024
    Phase 1
    Phase 2
    Phase 3
    Phase 4
    Patient Requirements
    Sex: Female & Male
    Age: 2 - 17 Years
    Requirements Information
    Inclusion and Exclusion Criteria
    Inclusion 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

    Protocol 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).

    Trial Locations

    Location
    Status
    Location
    Boston Childrens Hospital
    Boston, Massachusetts, United States, 02215
    Status
    N/A
    Location
    Child Hosp Of The Kings Daughters
    Norfolk, Virginia, United States, 23507
    Status
    N/A
    Location
    University of Wisconsin Madison Medical School
    Madison, Wisconsin, United States, 53792-7375
    Status
    N/A
    Location
    Novartis Investigative Site
    Parkville, Victoria, Australia, 3052
    Status
    N/A
    Location
    Novartis Investigative Site
    Leuven, Belgium, 3000
    Status
    N/A
    Location
    Novartis Investigative Site
    Montreal, Quebec, Canada, H4A 3J1
    Status
    N/A
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