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.
Children aged 12 to 17 at time of inclusion.
Patients with colonoscopy for diagnosis or follow-up of FAP.
≥ 5 polyps (> 2 mm) at initial colonoscopy (V1).
Free and informed consent, signed by both holders of parental authority/legal representative, with the accord of the minor patient.
Inability to understand the nature and goals of the study and/or communication difficulties observed by the investigator.
Contraindication to performing a colonoscopy.
< 5 polyps (>2 mm) registered during initial colonoscopy (V1).
Advanced disease with high-grade dysplasia adenoma or even adenocarcinoma in situ that should required colectomy
Single group
Rapamycin
DrugThis 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.
Evaluation of the safety profile of two doses of rapamycin in adolescents with Familial Adenomatous Polyposis.
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).
Evaluation of the effect of rapamycin on the number of polyps, on the entire colon and by segments (rectum, left colon, transverse colon and right colon) in adolescents suffering from Familial Adenomatous Polyposis
Analysis of the colonoscopies individually, blinded to the identity of the patient and the temporality of the colonoscopy in relation to the treatment, allowing a descriptive analysis of the number of polyps per segment (rectum, left colon, transverse colon, right colon).
Evaluation of the effect of rapamycin on the size of polyps, on the entire colon and by segments (rectum, left colon, transverse colon and right colon) in adolescents suffering from Familial Adenomatous Polyposis.
Analysis of the colonoscopies individually, blinded to the identity of the patient and the temporality of the colonoscopy in relation to the treatment, allowing a descriptive analysis of the size of polyps per segment (rectum, left colon, transverse colon, right colon).
Evaluation of the effect of rapamycin on the size of the largest polyp in each segment (rectum, left colon, transverse colon and right colon) in adolescents with Familial Adenomatous Polyposis
Colonoscopies will be analyzed in a paired manner for each patient (before treatment / after treatment) in order to be able to make a more specific judgment of the evolution of the polyps.
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