New hope for kids with rare blood cancer: targeted drug trial launches
NCT ID NCT04943198
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests the drug vemurafenib in children with a rare blood disorder called histiocytosis that has a specific gene mutation (BRAF) and hasn't improved with standard treatments. The goal is to find the best dose and how long to give the drug to stop the disease from getting worse. About 25 children will take part in this phase 2 trial.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Vemurafenib (Zelboraf)
- What this could lead to
- If successful, this could provide an effective treatment option for children with this rare blood disorder that hasn't responded to standard therapies.
- What could go wrong
- This is a small, early-phase trial with only 25 participants, so results may not apply to all patients. The drug may cause side effects or not work as hoped.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for HISTIOCYTOSIS are added.
By submitting, you agree to our Terms of use
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
-
Mother and Child Institute
RECRUITINGWarsaw, Mazovian, 01-211, Poland
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Massive study tracks hidden toll of rare immune diseases
- Rare disease study probes hidden brain effects
- New hope for kids with rare blood disorder: trametinib trial launches
- New imaging test could spot rare childhood disease sooner
- Promising combo aims to tame relapsed childhood cancers
- New pill targets Hard-to-Treat cancers: early trial recruits 554 patients