Study summary

This is a multinational, open-label, ascending-dose, delayed-treatment concurrent control clinical study to evaluate the safety and efficacy of AT132 in subjects with X-Linked Myotubular Myopathy aged less than 5 years old. Subjects will receive a single dose of AT132 and will be followed for safety and efficacy for 10 years

Additional Study Details

Phase
Phase 2/3
Product
Type
Interventional
Masking
None (Open Label)
Enrollment number
26
Show Additional Study Details

Get more information

Would you like more information about clinical trial sites that are recruiting participants for Gene Transfer Clinical Study in X-Linked Myotubular Myopathy? Contact us by filling out your information to the right and we’ll respond to you.

  • A patient
  • A caregiver
  • A parent
  • A healthcare provider
  • A patient advocate
  • Algeria
  • Argentina
  • Armenia
  • Australia
  • Austria
  • Belarus
  • Belgium
  • Bosnia And Herzegovina
  • Brazil
  • Bulgaria
  • Canada
  • Chile
  • China
  • Colombia
  • Croatia
  • Czech Republic
  • Denmark
  • Dominican Republic
  • Egypt
  • Estonia
  • Finland
  • Former Serbia and Montenegro
  • France
  • Georgia
  • Germany
  • Greece
  • Guatemala
  • Hong Kong
  • Hungary
  • Iceland
  • India
  • Indonesia
  • Ireland
  • Israel
  • Italy
  • Japan
  • Jordan
  • Kazakhstan
  • Latvia
  • Lebanon
  • Lithuania
  • Macedonia
  • Malaysia
  • Mexico
  • Montenegro
  • Morocco
  • Netherlands
  • New Zealand
  • Norway
  • Panama
  • Peru
  • Philippines
  • Poland
  • Portugal
  • Puerto Rico
  • Republic of Korea
  • Republic of Moldova
  • Romania
  • Russian Federation
  • Saudi Arabia
  • Serbia
  • Singapore
  • Slovakia
  • Slovenia
  • South Africa
  • Spain
  • Sweden
  • Switzerland
  • Taiwan, Province of China
  • Thailand
  • Turkey
  • Ukraine
  • United Kingdom
  • United States
  • Vietnam

By clicking ["Continue/Submit"], you agree that Astellas may contact you by email with information to help you connect with clinical trial sites. While we can connect you to clinical trial sites, we cannot answer questions about any investigational therapy through email. Your consent to receiving emails is not a requirement to participate in a clinical trial or study. For more information, including how to unsubscribe at any time, see our Privacy Notice & Cookies Policy.

    Locations

    Contact Us
    Contact Us
    Active, not recruiting
    Ann & Robert H Lurie Children's Hospital of Chicago
    Chicago, Illinois, United States, 60611
    Active, not recruiting
    UCLA Medical Center
    Los Angeles, California, United States, 90095
    Active, not recruiting
    Hopital Armad Trousseau
    Paris, France, 75012
    Active, not recruiting
    Hospital for Sick Children
    Toronto, Ontario, Canada, M5G0A4
    Active, not recruiting
    National Institute of Neurological Disorders and Stroke/NIH Porter
    Bethesda, Maryland, United States, 208892
    Active, not recruiting
    Kinderklinik und Kinderpoliklinik im Dr. Von Haunerschen Kinderspital Klinikum der Universitat Munchen
    München, Germany, 80337

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