Decade-Long watch: tracking a rare Disease's impact on families

NCT ID NCT04632953

Summary

This study aims to collect long-term safety and health information from up to 150 children and adults with a rare metabolic disorder called LC-FAOD. It will follow participants for up to 10 years to understand the natural course of the disease and monitor the safety of a specific treatment, triheptanoin, especially during pregnancy and for newborns. The program observes patients under their doctor's care but does not provide the treatment itself.

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes NO responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Locations

  • Ann & Robert H. Lurie Children's Hospital

    Chicago, Illinois, 60611, United States

  • Boston Children's Hospital

    Boston, Massachusetts, 02215, United States

  • CHEO (Children's Hospital Eastern Ontario)

    Ottawa, Ontario, K1H 8L1, Canada

  • Children's Hospital of Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital of Pittsburgh of UPMC

    Pittsburgh, Pennsylvania, 15224, United States

  • Columbia University

    New York, New York, 10032, United States

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Phoenix Children's Hospital

    Phoenix, Arizona, 85016, United States

  • Seattle Children's Hospital

    Seattle, Washington, 98020, United States

  • SickKids (The Hospital for Sick Children)

    Toronto, Ontario, M5G 1X8, Canada

  • The Emory Clinic

    Atlanta, Georgia, 30322, United States

  • University of Alberta

    Edmonton, Alberta, T6G 1C9, Canada

  • University of California San Francisco

    San Francisco, California, 94158, United States

  • University of Minnesota

    Minneapolis, Minnesota, 55454, United States

  • University of South Florida

    Tampa, Florida, 33606, United States

  • University of Utah

    Salt Lake City, Utah, 84108, United States

Conditions

Explore the condition pages connected to this study.