Targeted therapy for slow-flow or fast-flow vascular malformations
Treatments studied
Part of Blood & lymphatic, Cancer, Genetic & congenital, Heart & circulation, Skin clinical trials.
This trial tests two targeted medicines for people with certain types of vascular malformations (abnormal blood vessels). If you have a slow-flow malformation, you may receive alpelisib; if you have a fast-flow malformation, you may receive mirdametinib. Genetic testing is needed to see if your malformation has a specific change that these drugs target.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be 2 years or older.
- You must have a vascular malformation that is either slow-flow or fast-flow (your doctor can tell you which type).
- You must have tried standard treatments or your doctor says they aren't appropriate for you.
- You need a genetic test showing a specific change in either the PI3K pathway (for slow-flow) or the RAS-MEK-ERK pathway (for fast-flow).
- You must be able to swallow pills or take a tablet that dissolves in water.
- You must agree to use effective contraception if you or your partner could become pregnant.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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