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
100 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 · 28 incl. sub-types
6 sub-types
- Noonan syndrome 21 trials · 24 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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New drug cocktail targets Hard-to-Treat endometrial cancer
Disease control Recruiting nowThis early-phase study tests a combination of three drugs (avutometinib, defactinib, and everolimus) in people with recurrent endometrial cancer that has specific genetic changes in the RAS pathway. The main goal is to find a safe dose and understand side effects. About 31 partic…
Phase 1 • Sponsor: M.D. Anderson Cancer Center • Aim: Disease control
Last updated Jul 01, 2026 00:00 UTC
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New study tests online therapy to ease stress for parents of kids with RASopathies
Symptom relief Recruiting nowThis study tests whether Acceptance and Commitment Therapy (ACT), delivered through a smartphone app, can help caregivers of children with RASopathies (like Neurofibromatosis type 1 and Noonan syndrome) cope with parenting stress. The trial is fully remote and involves 70 adult c…
Sponsor: National Cancer Institute (NCI) • Aim: Symptom relief
Last updated Aug 26, 2026 00:00 UTC
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Scientists launch major study to unravel rare genetic conditions
Knowledge-focused Recruiting nowThis study aims to learn more about RASopathies, a group of genetic conditions that can cause developmental issues, birth defects, and increased cancer risk. Researchers will follow up to 500 people of any age who have or may have a RASopathy, along with their family members, for…
Sponsor: National Cancer Institute (NCI) • Aim: Knowledge-focused
Last updated Aug 22, 2026 00:00 UTC
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Hunt for hidden cancer genes: families needed to unlock hereditary secrets
Knowledge-focused Recruiting nowThis study aims to discover new genes that may cause certain cancers to run in families. Researchers will collect blood samples and health information from 1,500 people in families where multiple members have had cancer, especially childhood cancers. The goal is to build a regist…
Sponsor: St. Jude Children's Research Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:00 UTC
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Noonan syndrome study aims to uncover hidden heart risks
Knowledge-focused Recruiting nowThis study looks at cholesterol and blood sugar levels in 200 people with Noonan syndrome and related conditions, aged 2 to 35. Researchers want to see if these levels differ by age, gender, or genetic type. The goal is to improve long-term care by identifying who might be at hig…
Sponsor: IRCCS Azienda Ospedaliero-Universitaria di Bologna • Aim: Knowledge-focused
Last updated Jun 27, 2026 11:01 UTC
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Heart data hunt: no treatment, but paving way for future cures
Knowledge-focused Recruiting nowThis study looks back at medical records of 100 children with a genetic condition called RASopathy that causes severe heart thickening and heart failure. Researchers want to collect detailed information on how the disease progressed in the past. This will help design future studi…
Sponsor: Deutsches Herzzentrum Muenchen • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:06 UTC
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Massive study aims to unlock secrets of rare genetic disorders
Knowledge-focused Recruiting nowThis study is collecting blood, tissue, and medical information from up to 1,000 people with RASopathies—a group of genetic conditions that affect development and raise cancer risk. Researchers will store these samples and data in a database for future studies. The goal is to lea…
Sponsor: Children's Hospital Medical Center, Cincinnati • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:05 UTC