New granule drug hopes to shrink tumors in toddlers with rare genetic condition
NCT ID NCT05309668
First seen Jan 05, 2026 · Last updated May 07, 2026 · Updated 21 times
Summary
This study tests a new granule (sprinkle) form of the drug selumetinib in children aged 1 to 6 years who have neurofibromatosis type 1 (NF1) and painful, inoperable tumors called plexiform neurofibromas. The goal is to find the right dose, check safety, and see if the medicine can shrink these tumors. About 36 children will take part, and the medicine is given by mouth.
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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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Locations
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Research Site
Akron, Ohio, 44308, United States
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Research Site
Philadelphia, Pennsylvania, 19104, United States
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Research Site
Houston, Texas, 77030, United States
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Research Site
Richmond, Virginia, 23219, United States
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Research Site
Hamburg, 20246, Germany
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Research Site
München, 80337, Germany
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Research Site
Tübingen, 72076, Germany
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Research Site
Milan, 20133, Italy
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Research Site
Rome, 00165, Italy
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Research Site
Nagoya, 466-8560, Japan
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Research Site
Setagaya City, 157-8535, Japan
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Research Site
Rotterdam, 3015 GD, Netherlands
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Research Site
Moscow, 119620, Russia
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Research Site
Moscow, 125412, Russia
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Research Site
Barcelona, 08950, Spain
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Research Site
Madrid, Spain
Conditions
Explore the condition pages connected to this study.