Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)

Study Identifier:
COAV101A12306
ClinicalTrials.gov Identifier:
NCT04851873
EudraCT Identifier:
2020-005995-37
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
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Study Details

Medical Condition
  • All Spinal Muscular Atrophies
Study Drug
    Date
    Sep 2021 - Jun 2023
    Phase 1
    Phase 2
    Phase 3
    Phase 4
    Patient Requirements
    Sex: Female & Male
    Age: N/A - 17 Years
    Requirements Information
    Inclusion and Exclusion Criteria
    Inclusion Criteria
    • Inclusion
    • Symptomatic SMA diagnosis based on gene mutation analysis with bi-allelic survival motor neuron 1 (SMN1) mutations (deletion or point mutations) and any copy of the survival motor neuron 2 (SMN2) gene.
    • Weight ≥ 8.5 kg and ≤ 21 kg at the time of Screening Visit 2
    • Naive to treatment or have discontinued an approved drug/therapy
    • Exclusion:
    • Previous OAV101 use or previous use of any adeno-associated virus serotype 9 (AAV9) gene therapy
    • BMI \< 3rd percentile
    • Participant with history of aspiration pneumonia or signs of aspiration
    • Elevated anti-AAV9 antibody
    • History of gene therapy, hematopoietic transplantation, or solid organ transplantation
    • Inability to take corticosteroids
    • Concomitant use of immunosuppressive therapy
    • Requiring invasive ventilation, tracheostomy or awake non-invasive ventilation 9. Administration of vaccines 2 weeks prior to infusion of OAV101
    • Awake hypoxemia or awake oxygen saturation level decrease
    • Hepatic dysfunction
    • Presence of a confirmed or suspected infection
    • If previously treated with disease modifying therapy, specified washout times apply
    • Documented any parental consanguinity.

    Protocol Summary

    To evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene weighing ≥ 8.5 kg and ≤ 21 kg, over a 12 month period.

    Trial Locations

    Location
    Status
    Location
    Novartis Investigative Site
    Boston, Massachusetts, United States, 02115
    Status
    N/A
    Location
    Novartis Investigative Site
    St Louis, Missouri, United States, 63110
    Status
    N/A
    Location
    Novartis Investigative Site
    Randwick, New South Wales, Australia, 2031
    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
    Location
    Novartis Investigative Site
    Garches, France, 92380
    Status
    N/A
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