Safety Study for the Use of Rapamycin in Children With Familial Adenomatous Polyposis

Condition(s) studied

Investigational drug(s) / intervention(s)

Rapamycin: This is a 2-dose rapamycin safety study, with a target through level of 3 to \<5 ng/ml for the first 3 months and 5-8 ng/ml for the next 3 months for each of the included patients. To avoid the possible cumulative effect, the two treatment phases will be separated by a 3-weeks wash-out period.

Study summary

The hypothesize of this research is that rapamycin is effective and well-tolerated in teenagers with familial adenomatous polyposis (FAP). Rapamycin could be effective in blocking the formation of adenomas and/or their evolution by decreasing their size and number. Researchers aim to assess the safety profile of rapamycin in FAP adolescents using a 2 low dose regimen.

Eligibility

Primary outcome measure(s)

• Evaluation of the safety profile of two doses of rapamycin in adolescents with Familial Adenomatous Polyposis. — For the entire duration of taking the experimental drug (rapamycin) and up to one month after stopping.
  Monitoring of adverse events (EvI) and serious adverse events (SvIG). Particular attention will be paid to known adverse events attributable to rapamycin according to the summary of product characteristics (SmPC).

Trial sites (4)

More University Hospital, Toulouse trials in France

Other trials for the same condition