A Registry of Cases of Spinal Muscular Atrophy

Study Identifier:
PRESMAREG
ClinicalTrials.gov Identifier:
NCT07805278
EudraCT Identifier:
N/A
EU CT Number:
N/A
Sponsor:
St. Jude Children's Research Hospital
Will Be Recruiting

Study Details

Medical Condition
  • All Spinal Muscular Atrophies
Study Drug
    Date
    Sep 2026 - Aug 2029
    Patient Requirements
    Sex: Female & Male
    Age: N/A
    Requirements Information
    Inclusion and Exclusion Criteria
    Inclusion Criteria
    • Current or past pregnancy during which the fetus received a genetic diagnosis of SMA.
    • Current or past treatment with SMA-directed therapy intended to treat the fetus during gestation (such as risdiplam, nusinersen, onasemnogene abeparvovec, or another product intended to treat SMA).
    Exclusion Criteria
    • Inability or unwillingness of research participant or legal guardian/representative to give written informed consent.

    Protocol Summary

    Because no long-term record of prenatal Spinal Muscular Atrophy (SMA) treatment exists, this study will address an important gap in the understanding of the safety and benefit of treatment while in the womb. The registry will help guide best practices for monitoring pregnant women and their fetus with SMA, if treated with a medication while in the womb, and to follow the baby after birth for signs of safety and added benefit birth and support future research and study development.

    Trial Locations

    Location
    Status
    Location
    St. Jude Children's Research Hospital
    Memphis, Tennessee, United States, 38105
    Status
    N/A

    Contact Cure SMA

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