Sirolimus Effect on Hypertrophic Syndromes Related Gene PIK3CA
Status:
Completed
Trial end date:
2016-11-01
Target enrollment:
Participant gender:
Summary
It was found that people who have an excessive growth in one or more parts of their body and
normal growth in other parts, may have a change in genes responsible for controlling cell
growth. This genetic discovery identified a possible treatment for this overgrowth, called
Sirolimus.
This is a pilot study, which help the investigators prepare a larger international study,
which will aim to determine whether a treatment based Sirolimus is able to stop the excessive
growth of one or more parties of their body in people who carry a genetic change in the
PIK3CA gene (phosphatidylinosilol-3-kinase encoded by the gene PIK3CA).