Plain-English translation of NCT05577754 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Phase 2 — Testing in a bigger group (up to a few hundred people) to see if the treatment actually works and is still safe.
This trial is testing a medication called alpelisib to see if it can help people with MCAP, a rare genetic condition that causes an enlarged brain and problems with learning and development. In the first 6 months, some participants will receive the medication while others receive a placebo (dummy pill) to compare how well the treatment works. After that, everyone will have the option to receive the medication for another 18 months.
MCAP is caused by changes in a specific gene, and there are currently no proven treatments for it. This trial is testing whether this medication, which targets the genetic change that causes the condition, might slow or improve symptoms in affected patients.
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You will start with a screening visit lasting up to 90 days to make sure you are eligible. Then you will be randomly assigned to either receive the medication or a placebo for 6 months, with monthly visits to your local clinic for check-ups. At 6 months, you will have detailed testing including brain scans, blood work, and developmental assessments. After that, all participants will receive the medication for another 18 months, with similar monthly visits and testing every 6 months. The entire study lasts about 2 years.
AI-generated summary from trial data · Jun 1, 2026 · Not medical advice
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