NF1
Clinical trials for NF1 explained in plain language.
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New drug trial targets painful skin tumors in NF1 patients
Disease control Recruiting nowThis study tests a drug called mirdametinib in adults with NF1 who have at least 24 skin tumors (cutaneous neurofibromas). The goal is to find a safe dose and see if the drug can shrink the tumors. The trial has two phases: first finding the right dose, then testing how well it w…
Matched conditions: NF1
Phase: PHASE1, PHASE2 • Sponsor: Johns Hopkins University • Aim: Disease control
Last updated May 22, 2026 13:52 UTC
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New hope for kids with recurrent tumors: drug combo enters safety trial
Disease control Recruiting nowThis early-stage study is testing a combination of two drugs, avutometinib and defactinib, in children and young adults (ages 3 to 30) with solid tumors that have returned or not responded to treatment. The main goal is to find a safe dose and check for side effects. The study is…
Matched conditions: NF1
Phase: PHASE1 • Sponsor: Memorial Sloan Kettering Cancer Center • Aim: Disease control
Last updated May 22, 2026 13:50 UTC
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Cancer drug repurposed to shrink NF1 skin bumps in early trial
Disease control Recruiting nowThis early-stage study tests if a drug called IMLYGIC, already used for certain skin cancers, can safely treat painful or disfiguring skin bumps (cutaneous neurofibromas) in adults with NF1. Ten participants will receive up to 4 cycles of injections directly into their tumors. Th…
Matched conditions: NF1
Phase: PHASE1 • Sponsor: Johns Hopkins University • Aim: Disease control
Last updated May 20, 2026 11:57 UTC