New hope for Tough-to-Treat rare diseases: experimental drug targets faulty cell signals

NCT ID NCT04079179

Summary

This study is testing whether the drug cobimetinib can help control several rare disorders caused by overactive immune cells, particularly when standard treatments have failed. The trial will enroll 90 children and adults with conditions like Langerhans cell histiocytosis (LCH) and related disorders. Cobimetinib works by blocking a specific protein (MEK) that sends incorrect growth signals in these diseases.

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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.