Trial tests alpelisib for MCAP with PIK3CA mutations
Treatments studied
Part of Brain & nervous system, Genetic & congenital, Mental health clinical trials.
This Phase 2 study tests alpelisib in children and adults with MCAP, a condition caused by a specific PIK3CA gene mutation. The goal is to see if the treatment is effective and safe, while carefully monitoring blood sugar and organ function.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Be age 2 to 40 when you sign consent (and assent if applicable).
- Have a confirmed MCAP diagnosis with a developmental learning/ability challenge.
- Have test-confirmed PIK3CA gene mutation in tumor/affected-tissue or body DNA using a local lab DNA test.
- Have blood and organ lab results that meet safety thresholds (blood counts, kidney, liver, electrolytes, and clotting).
- Be able to take the study medicine by mouth (tablets or suitable liquid form).
- If you can become pregnant (or have partners who can): agree to pregnancy prevention rules during the study and for 1 week after stopping.
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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