A Phase 2 interventional study of Rapamycin in Familial Adenomatous Polyposis, sponsored by University Hospital, Toulouse. Not yet recruiting at 4 sites in France. Open to participants aged 12 Years to 17 Years. Per ClinicalTrials.gov, last updated 2026-05-08.
Sponsored by University Hospital, Toulouse · Phase 2, Interventional, and Treatment
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.
FAP is a rare genetic disease linked to mutations of APC gene. Adenomatous polyps appear around the age of 10 and will evolve into colic adenocarcinoma. To date, there is no effective treatment; 100% of patients will develop colorectal cancer before 40 years. This risk is addressed by regular colonoscopy monitoring, in order to propose a timely prophylactic colectomy.
Rapamycin (sirolimus) is a drug that targets the mTOR (mammalian target of rapamycin) protein involved in the PI3K-Akt signalling pathway downstream of PI3K. There are interactions between the PI3K-Akt pathway and the Wnt/APC/-catenin pathway that is hyperactivated in FAP. Rapamycin was used out of indication with efficacy and good tolerance in 2 adolescents whose parents had refused colectomy. Researchers recently demonstrated its effectiveness in a child with very severe juvenile polyposis. Data are also available in animals, but no proof-of-concept studies have been conducted in humans.
In France, Rapamycin use is allowed in adults with kidney transplantation and pulmonary lymphangioleiomyomatosis. However, it is used on children over the market. According to the literature and the field experience, the hypothesize is that a through level of 3-8 ng/ml should be effective in children with FAP, with a lower rate of adverse events.
90 studies on the registry are indexed under Adenomatous Polyposis Coli; 28 are open to participants now.
This study's planned enrollment of 25 is below the median of 43 across 60 interventional studies indexed under Adenomatous Polyposis Coli.
Browse Adenomatous Polyposis Coli studies →University Hospital, Toulouse is the lead sponsor of 794 studies on the registry; 214 are open to participants now.
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Exclusion Criteria:
Drug: 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.
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).
Time frame: For the entire duration of taking the experimental drug (rapamycin) and up to one month after stopping.
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).
Time frame: Visit 1 inclusion and 6 month after
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).
Time frame: Visit 1 inclusion and 6 month after
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.
Time frame: After 6 months of treatment with rapamycin
Plan to share: No
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University Hospital, Toulouse