Neurofibromatosis
MONDO:0021061A hereditary neoplastic syndrome in which tumors grow in the nervous system. There are typically 3 main types recognized, but other forms with uncertain etiology exist.
Also known as: Recklinghausen's neurofibromatosis, acoustic neurofibromatosis, central Neurofibromatosis, neurofibromatosis, neurofibromatosis syndrome, peripheral Neurofibromatosis, type IV neurofibromatosis of riccardi, von Reklinghausen disease
125 clinical trials for this condition and its sub-types, 19 tagged with Neurofibromatosis itself.
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Sub-types of Neurofibromatosis
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Neurofibromatosis type 1 73 trials
3 sub-types
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NF2-related schwannomatosis 21 trials
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Schwannomatosis 3 trials
4 sub-types
- 22q-related schwannomatosis 0 trials
- LZTR1-related schwannomatosis 0 trials
- SMARCB1-related schwannomatosis 0 trials
- Neurofibromatosis, type III, mixed central and peripheral 0 trials
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Neurofibromatosis-Noonan syndrome 2 trials
1 sub-type
- Watson syndrome 0 trials
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Immune cells take on nerve tumors: new trial launches
Disease control Stopped earlyThis early-stage trial is testing whether specially engineered immune cells (called CAR-T and CTL cells) and a dendritic cell vaccine can safely treat people with neurofibromatosis or schwannomatosis, conditions that cause nerve tumors. The study will enroll 100 participants aged…
Phase 1/2 • Sponsor: Shenzhen Geno-Immune Medical Institute • Aim: Disease control
Last updated Aug 26, 2026 00:00 UTC
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Experimental drug REC-2282 targets NF2 brain tumors in early trial
Disease control Stopped earlyThis study tested a drug called REC-2282 in people aged 12 and older with progressive meningiomas (brain tumors) linked to NF2 gene mutations. Participants took the drug or a placebo three times a week in cycles. The goal was to see if the drug could slow tumor growth. The trial …
Phase 2/3 • Sponsor: Recursion Pharmaceuticals Inc. • Aim: Disease control
Last updated Aug 19, 2026 00:00 UTC
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Light-Activated cream could slow growth of NF1 skin tumors
Disease control Stopped earlyThis trial tests whether a light-activated cream (Levulan Kerastick) followed by red light therapy can slow the growth of benign skin tumors called neurofibromas in people with neurofibromatosis type 1 (NF1). About 30 participants aged 14 and older with superficial tumors on the …
Phase 2 • Sponsor: Donald Basel • Aim: Disease control
Last updated Jul 12, 2026 00:00 UTC
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Real-World study tracks Selumetinib's impact on NF1 tumors in children
Disease control Stopped earlyThis study observes children aged 3 to 18 with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas and are starting treatment with selumetinib. Researchers will track how well the drug shrinks or stabilizes tumors, how long patients stay on tre…
Sponsor: AstraZeneca • Aim: Disease control
Last updated Jul 10, 2026 00:00 UTC
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Experimental cancer drug study halted early
Disease control Stopped earlyThis early-stage study tested a new drug called PF-07284892, alone or with other medicines, in people with advanced solid tumors that had specific genetic changes. The goal was to find the safest dose and check for side effects. The study was stopped early, so results are limited…
Phase 1 • Sponsor: Pfizer • Aim: Disease control
Last updated Jun 27, 2026 13:02 UTC
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New cocktail of cancer drugs tested in desperate cases
Disease control Stopped earlyThis early-phase trial tested combinations of the drugs bevacizumab and temsirolimus, sometimes with valproic acid or cetuximab, in 154 people with advanced cancers or certain non-cancerous but progressive diseases. The main goal was to find safe doses and look for any signs that…
Phase 1 • Sponsor: M.D. Anderson Cancer Center • Aim: Disease control
Last updated Jun 27, 2026 12:32 UTC
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Experimental drug AR-42 tested on brain tumors – early results uncertain
Disease control Stopped earlyThis early-phase trial tested the drug AR-42 in 7 adults with vestibular schwannoma or meningioma brain tumors who were already scheduled for surgery. Participants took AR-42 pills for about 3 weeks before their operation. The main goal was to see if the drug could lower a protei…
Early phase 1 • Sponsor: Massachusetts Eye and Ear Infirmary • Aim: Disease control
Last updated Jun 27, 2026 08:01 UTC