New hope for rare immune cell diseases: targeted drug trial opens

NCT ID NCT04079179

First seen Nov 01, 2025 · Last updated Apr 28, 2026 · Updated 24 times

Summary

This study tests a drug called cobimetinib in children and adults with Langerhans cell histiocytosis and related disorders that have not responded to standard treatment or have returned. Cobimetinib blocks a faulty growth signal in the disease cells. The goal is to see if the drug can shrink tumors or slow disease progression, while monitoring side effects.

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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

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Arkansas Children's Hospital

    RECRUITING

    Little Rock, Arkansas, 72202, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Children's Hospital of Orange County

    RECRUITING

    Orange, California, 92868, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Children's Medical Center- UTSW

    RECRUITING

    Dallas, Texas, 75235, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Children's National Hospital

    RECRUITING

    Washington D.C., District of Columbia, 20010, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Dana Farber Cancer Institute, Boston Children's

    RECRUITING

    Boston, Massachusetts, 02215, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • John Hopkins University School of Medicine

    RECRUITING

    Baltimore, Maryland, 21287, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Memorial Sloan Kettering Cancer Center

    RECRUITING

    New York, New York, 10065, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • NACHO Consortium

    NOT_YET_RECRUITING

    Memphis, Tennessee, 38105, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Phoenix Children's Hospital

    RECRUITING

    Phoenix, Arizona, 85016, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Texas Children's Hospital

    RECRUITING

    Houston, Texas, 77030, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • UCSF Benioff Children's Hospital

    RECRUITING

    San Francisco, California, 94158, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • University of Wisconsin-American Family Children's Hospital

    TERMINATED

    Madison, Wisconsin, 53792, United States

Conditions

Explore the condition pages connected to this study.