New study tracks Long-Term safety of rare disease treatment

NCT ID NCT04632953

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study follows 150 people with long-chain fatty acid oxidation disorders (LC-FAOD) to check the long-term safety of their treatment, including for pregnant women and their babies. Researchers track serious side effects and disease complications. The goal is to better understand how the medication triheptanoin works over time.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

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

More trials for these conditions

Other studies related to the condition(s) this trial covers.