NF1
Clinical trials for NF1 explained in plain language.
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Could a cholesterol drug boost reading skills in kids with NF1?
Disease control OngoingThis study investigates whether combining the medication lovastatin with intensive reading tutoring can improve reading abilities in children and young adults (ages 8-20) with Neurofibromatosis Type 1 (NF1) who have reading disabilities. Participants first receive either lovastat…
Matched conditions: NF1
Phase: PHASE2 • Sponsor: Vanderbilt University • Aim: Disease control
Last updated Jul 02, 2026 04:00 UTC
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Promising drug may shrink inoperable tumors in kids with rare genetic condition
Disease control OngoingThis study tests a drug called selumetinib in children and young adults (ages 3-18) with neurofibromatosis type 1 (NF1) who have nerve tumors that cannot be removed by surgery. The goal is to see if the drug can shrink or slow the growth of these tumors. Participants take the dru…
Matched conditions: NF1
Phase: PHASE1, PHASE2 • Sponsor: National Cancer Institute (NCI) • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC
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New hope for kids with resistant tumors: drug combo enters safety trial
Disease control OngoingThis early-stage study tests a combination of two drugs, avutometinib and defactinib, in children and young adults (ages 3 to 30) with advanced or recurrent solid tumors that have specific genetic changes. The main goal is to find the safest dose with the fewest side effects. The…
Matched conditions: NF1
Phase: PHASE1 • Sponsor: Memorial Sloan Kettering Cancer Center • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC
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Blood test could spot hidden tumors in NF1 patients
Knowledge-focused ENROLLING_BY_INVITATIONThis study aims to find blood-based biomarkers that signal the presence and size of plexiform neurofibromas in people with Neurofibromatosis type 1 (NF1). Researchers will collect blood samples and use whole-body MRI to compare biomarker levels in 200 participants with different …
Matched conditions: NF1
Sponsor: Ann & Robert H Lurie Children's Hospital of Chicago • Aim: Knowledge-focused
Last updated Jun 26, 2026 13:08 UTC