New hope for kids with rare blood disorder: trametinib trial launches

NCT ID NCT04943224

Recruiting now Disease control Sponsor: Anna Raciborska Source: ClinicalTrials.gov ↗

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

Summary

This study tests the drug trametinib in children with histiocytosis, a rare blood disorder, that hasn't responded to standard treatments or has a specific genetic profile. The trial aims to find the best dose and timing to improve how long children live without their disease getting worse. Only 12 participants will be enrolled in this single-center study.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: Trametinib (Mekinist), a targeted cancer drug
What this could lead to: If successful, this could offer a new treatment option for children with histiocytosis that hasn't responded to standard therapies.
What could go wrong: This is a small, early-phase trial with only 12 participants, so results may not apply broadly. The drug may cause side effects or not improve outcomes.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

histiocytosis Histiocytosis, Langerhans-Cell

As listed by the trial registrant

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

HISTIOCYTOSIS

Contacts and locations

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Study contacts

Contact

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

Locations

Mother and Child Institute
RECRUITING

Warsaw, Mazovian, 01-211, Poland

Contact

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

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