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.
Disclaimer
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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.
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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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
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Mother and Child Institute
RECRUITINGWarsaw, Mazovian, 01-211, Poland
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••