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Safety Study for the Use of Rapamycin in Children With Familial Adenomatous Polyposis
Sponsor: University Hospital, Toulouse
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.
Official title: Safety Study for the Use of Rapamycin in Children With Familial Adenomatous Polyposis.
Key Details
Gender
All
Age Range
12 Years - 17 Years
Study Type
INTERVENTIONAL
Enrollment
25
Start Date
2025-08-01
Completion Date
2030-08
Last Updated
2025-01-31
Healthy Volunteers
No
Conditions
Interventions
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.
Locations (4)
CHU Bordeaux Hôpital Pellegrin
Bordeaux, France
CHU Montpellier Hôpital Arnaud de Villeneuve
Montpellier, France
APHP Hôpital Robert Debré
Paris, France
CHU Toulouse Hôpital des Enfants
Toulouse, France