Cancer drug shows promise for rare blood vessel disorders

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Signed informed consent and assent (when applicable) 2. Males or females age \> 2 months to \< 30 years at the time of informed consent. 3. Have a documented Ras/MAPK-pathway variant. 4. Have a symptomatic vascular anomaly in need of medical therapy. 5. Have a disease-related lesion or lesions which can be measured 6. Have a Lansky or Karnofsky performance status score of ≥ 50. 7. Have acceptable organ function 8. (For persons who can get pregnant) Have a negative serum or urine pregnancy test within 7 days prior to starting study medication. 9. Must agree to the use an acceptable method of contraception 10. Subjects must be able to swallow tablets or liquid or use a nasogastric or gastric tube Exclusion Criteria: 1. Subjects seeking treatment for hypertrophic cardiomyopathy without a vascular anomaly 2. Prior medications that are not allowable per the study protocol 3. Confirmed or possible infections 4. History of hepatic sinusoidal obstructive syndrome (veno-occlusive disease of the liver) in the last 3 months. 5. Active gastrointestinal (GI) disease 6. Subjects with history of or current risk of retinal vein occlusion (RVO) or central serous retinopathy (CSR) or uncontrolled glaucoma or ocular hypertension. 7. Allergic reactions: Subjects with a history of allergic reaction attributed to compounds of similar chemical or biological composition to trametinib are not eligible. 8. History of prior and or ongoing cancer or ongoing investigations or treatment for cancer at time of informed consent. 9. Subjects unable to comply with safety monitoring requirements. 10. Subject may not be pregnant or breast feeding. 11. Parents/guardian/LAR or subjects who, in the opinion of the Investigator, may be non-compliant with study schedules or procedures. 12. Debulking or other major surgery performed within 30 days, at time of informed consent; 13. Clinically meaningful bleeding related to VA: Grade 2 within 14 days or Grade 3 and more within 28 days before study treatment start as per CTCAE v. 5.0. Sclerotherapy/embolization for vascular complications performed within 14 days before informed consent. 14. Subjects not able to understand and to comply with study instructions and requirements (in patients, legally authorized representatives, or guardians as applicable) at time of informed consent.