NF1
Clinical trials for NF1 explained in plain language.
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Can a cancer drug shrink painful NF1 skin tumors? early trial launches
Disease control Recruiting nowThis early study tests if a drug called IMLYGIC, already used for some skin cancers, can safely treat bothersome skin tumors in adults with NF1. Ten participants will receive injections directly into their tumors over four cycles. The goal is to see if the treatment is feasible a…
Matched conditions: NF1
Phase: PHASE1 • Sponsor: Johns Hopkins University • Aim: Disease control
Last updated May 15, 2026 11:53 UTC
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New drug shows promise for NF1 skin tumors in early trial
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 the safest dose and see if it can shrink the tumors. The trial involves 24 participants and is run at Johns Hopkins University.
Matched conditions: NF1
Phase: PHASE1, PHASE2 • Sponsor: Johns Hopkins University • Aim: Disease control
Last updated May 15, 2026 11:53 UTC
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New hope for kids with resistant tumors: drug combo enters safety trial
Disease control Recruiting nowThis early-phase study tests 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 with manageable side effects. The drugs targe…
Matched conditions: NF1
Phase: PHASE1 • Sponsor: Memorial Sloan Kettering Cancer Center • Aim: Disease control
Last updated May 06, 2026 16:15 UTC