RASopathy
MONDO:0021060Developmental syndromes caused by germline mutations (or in rare cases by somatic mosaicism) in genes that alter the Ras subfamily and mitogen-activated protein kinases that control signal transduction.
Also known as: RASopathy, Ras protein signal transduction disease, disorder of Ras protein signal transduction
101 clinical trials for this condition and its sub-types, 9 tagged with RASopathy itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of RASopathy
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Neurofibromatosis type 1 73 trials
3 sub-types
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Noonan syndrome and Noonan-related syndrome 1 trial · 29 incl. sub-types
6 sub-types
- Noonan syndrome 22 trials · 25 incl. sub-types Sub-types →
- Costello syndrome 7 trials
- Cardiofaciocutaneous syndrome 7 trials Sub-types →
- Legius syndrome 5 trials
- Noonan syndrome with multiple lentigines 3 trials Sub-types →
- Noonan syndrome-like disorder with loose anagen hair 0 trials Sub-types →
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Neurofibromatosis-Noonan syndrome 2 trials
1 sub-type
- Watson syndrome 0 trials
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CBL-related disorder 1 trial
Most studied deeper sub-types
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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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New cancer pill shows early promise, but trial halted
Disease control Stopped earlyThis early-stage trial tested an oral drug called TNO155, alone or with another drug (nazartinib), in 227 adults with advanced solid tumors like lung cancer, melanoma, and head/neck cancer. The main goal was to check safety and find the right dose. The study was terminated early,…
Phase 1 • Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Jul 11, 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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Scientists dive into cells to unravel costello Syndrome's secrets
Knowledge-focused Stopped earlyThis study collects small skin samples from children aged 2 to 17 with Costello syndrome or a related condition. Researchers will analyze the cells to understand how a mutation in the HRAS gene affects energy use and mitochondria. The goal is to learn more about the disease's und…
Sponsor: University Hospital, Bordeaux • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:05 UTC