PLEXIFORM NEUROFIBROMA
Clinical trials for PLEXIFORM NEUROFIBROMA explained in plain language.
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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: PLEXIFORM NEUROFIBROMA
Phase: PHASE1 • Sponsor: Memorial Sloan Kettering Cancer Center • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC
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New drug shows promise for shrinking painful NF1 tumors
Disease control OngoingThis study tests a drug called mirdametinib in 114 adults and children with a genetic condition called NF1 that causes nerve tumors (plexiform neurofibromas) that cannot be removed by surgery and cause serious problems. The drug works by blocking a protein that helps tumors grow.…
Matched conditions: PLEXIFORM NEUROFIBROMA
Phase: PHASE2 • Sponsor: SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany • Aim: Disease control
Last updated Jun 27, 2026 12:29 UTC
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Could a cancer drug shrink Children's brain tumors?
Disease control OngoingThis phase 2 trial tests the drug trametinib in children and young adults (ages 1 month to 25 years) with low-grade glioma or plexiform neurofibroma that has not responded to prior treatment. Participants take a daily oral dose for up to 18 cycles. The study aims to see if the dr…
Matched conditions: PLEXIFORM NEUROFIBROMA
Phase: PHASE2 • Sponsor: St. Justine's Hospital • Aim: Disease control
Last updated Jun 27, 2026 12:23 UTC
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10-Year NF1 study aims to unlock secrets of rare genetic disorder
Knowledge-focused OngoingThis natural history study follows 259 children, adolescents, and adults with Neurofibromatosis Type 1 (NF1) for up to 10 years. Researchers will track tumor growth, monitor quality of life, and perform genetic testing to better understand how the disease changes over time. The g…
Matched conditions: PLEXIFORM NEUROFIBROMA
Sponsor: National Cancer Institute (NCI) • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:00 UTC
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New study tracks Long-Term safety of NF1 drug in kids
Knowledge-focused OngoingThis study monitors the safety of selumetinib in children aged 3 to 18 with neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas. Researchers will track potential side effects on the heart, bones, muscles, liver, eyes, and puberty. The goal is to better un…
Matched conditions: PLEXIFORM NEUROFIBROMA
Sponsor: AstraZeneca • Aim: Knowledge-focused
Last updated Jun 27, 2026 11:00 UTC