New hope for kids with rare blood disease: drug timing trial launched
NCT ID NCT04943198
First seen Apr 10, 2026 · Last updated May 19, 2026 · Updated 7 times
Summary
This study looks at the best way to give the drug vemurafenib to children with a rare disease called histiocytosis that has not responded to standard treatments. The disease is linked to a specific gene change called BRAF. The goal is to find the right timing and dose to control the disease and prevent it from coming back. About 25 children will take part in this phase 2 trial.
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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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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: •••••@•••••
Conditions
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