New hope for kids with rare cell disease: targeted pill tested

NCT ID NCT06582745

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

Summary

This study tests a targeted drug called trametinib in 75 children with Langerhans cell histiocytosis (LCH), a rare disease where immune cells grow out of control. The goal is to see if the drug can safely stop the disease from getting worse or coming back. Children with new, relapsed, or hard-to-treat LCH may join.

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Conditions

The condition(s) this trial relates to.

Histiocytosis, Langerhans-Cell Langerhans cell histiocytosis

As listed by the trial registrant

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

Contacts and locations

Study contacts

  • Contact

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

  • Contact

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

Locations

  • Cook Children's Health Care System

    RECRUITING

    Fort Worth, Texas, 76104, United States

    Contact

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

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