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  • Research type

    Research Study

  • Full title

    Long-term follow-up of patients with spinal muscular atrophy Treated with OAV101 IT or OAV101 IV in Clinical Trials



  • Contact name

    Lucas Dirson

  • Contact email

  • Sponsor organisation

    Novartis Pharma AG

  • Eudract number


  • Identifier


  • Research summary

    This research study is organised and supported by Novartis. The study purpose is to evaluate long-term follow-up safety and efficacy on patients with Spinal Muscular Atrophy (SMA) who were treated with OAV101 (Zolgensma®) by intravenous (IV) or intrathecal (IT) administration in Novartis-sponsored studies.
    Spinal Muscular Atrophy (SMA) is a genetic (inherited) neuromuscular disease that causes muscles to become weak and waste away that occurs in early childhood with an incidence of approximately 1:10,000 live births. People with SMA lose a specific type of nerve cell in the spinal cord (called motor neurons) that control muscle movement. Without these motor neurons, muscles don’t receive nerve signals that make muscles move. SMA is the leading cause of infant mortality due to genetic diseases.
    Safety and efficacy will be assessed for up to 15 years following the treatment with OAV101 from a previous clinical trial. Approximately 120 to 260 people with spinal muscular atrophy at least 2 years old and up will be invited to join this study.
    Participants will not receive study treatment at any time during this study. Participants will be in the study for up to 15 years and during that time they will need to visit the Study Doctor about 8 times at the study site. The study will recruit approximately 181 number of participants in 81 number of sites across 26 number of countries.

  • REC name

    North East - Newcastle & North Tyneside 1 Research Ethics Committee

  • REC reference


  • Date of REC Opinion

    9 Feb 2023

  • REC opinion

    Further Information Favourable Opinion

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