Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)

Study Identifier:
COAV101B12301
ClinicalTrials.gov Identifier:
NCT05089656
EudraCT Identifier:
2021-003474-31
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
  • Spinal Muscular Atrophy Type II
Study Drug
    Date
    Feb 2022 - 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
    • Diagnostic confirmation during screening period of 5q SMA
    • The patient must be treatment naive (historical or current use) for all SMN-targeting therapies (e.g., risdiplam (Evrysdi) and nusinersen (Spinraza)).
    • Onset of clinical signs and symptoms at ≥ 6 months of age
    • A complete Hammersmith Functional Motor Scale - Expanded (HFMSE) assessment during the screening period for trial eligibility
    • Able to sit independently at screening, but has never had the ability to walk independently.
    Exclusion Criteria
    • Anti-adeno-associated virus serotype 9 (AAV9) antibody titer reported as elevated (reference to \> 1:50 or validated result consistent with being elevated) at screening as determined by sponsor designated lab.
    • Infectious process (e.g., viral, bacterial) or febrile illness within 30 days prior to OAV101 treatment or sham procedure
    • Hepatic dysfunction (i.e. alanine aminotransferase (ALT), total bilirubin, gamma-glutamyl transferase (GGT) or glutamate dehydrogenase (GLDH), \> upper limit of normal (ULN).
    • Requiring invasive ventilation, awake noninvasive ventilation for \> 6 hours during a 24-hour period, noninvasive ventilation for \> 12 hours during a 24-hour period or requiring tracheostomy
    • Complications at screening that would interfere with motor efficacy assessments including but not limited to, severe contractures or Cobb angle \> 40 in a sitting position
    • Surgery for scoliosis or hip fixation in the 12 months prior to Screening or planned within the next 64 weeks
    • Clinically significant sensory abnormalities in the neurological examination at Screening

    Protocol Summary

    This was a Phase III multi-center, single dose (1.2 x 10\^14 vector genomes), randomized, sham controlled, double-blind study that investigates the efficacy, safety and tolerability of OAV101B in treatment naive, sitting and never ambulatory SMA patients 2 to \<18 years of age.

    Trial Locations

    Location
    Status
    Location
    Connecticut Children's Medical Center
    Farmington, Connecticut, United States, 06032
    Status
    N/A
    Location
    Ann & Robert H. Lurie Children's Hospital of Chicago
    Chicago, Illinois, United States, 60611
    Status
    N/A
    Location
    Clinic for Special Children
    Strasburg, Pennsylvania, United States, 17579
    Status
    N/A
    Location
    St Jude Children's Research Hospital
    Memphis, Tennessee, United States, 38105
    Status
    N/A
    Location
    Child Hosp Of The Kings Daughters
    Norfolk, Virginia, United States, 23507
    Status
    N/A
    Location
    Children's Specialty Group/CHKD
    Norfolk, Virginia, United States, 23507
    Status
    N/A
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