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

Trial statusNot yet recruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age12-17
SponsorUniversity Hospital, Toulouse

About this trial

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 criteria

Qualifiers

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.

Disqualifiers

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

Trial design

Treatments tested in this trial

  • Rapamycin

Treatment groups

25 Participants
are divided into 1 treatment group

Locations

4
CHU Bordeaux Hôpital Pellegrin33076, Bordeaux France
CHU Montpellier Hôpital Arnaud de Villeneuve34295, Montpellier France
APHP Hôpital Robert Debré75019, Paris France
CHU Toulouse Hôpital des Enfants31300, Toulouse France

Sponsors and collaborators

University Hospital, Toulouse

Lead sponsor