SIROLIMUS for Familial adenomatous polyposis

Inclusion criteria

• {"criterion_text":"- Children aged 12 to 17 included at time of inclusion.\n- Patients with colonoscopy for diagnosis or follow-up of familial adenomatous polyposis. (The diagnosis of PAF is made when there is a family history of APC mutation in the child, or when several adenomatous polyps are found because of rectal discharge.)\n- Visualization of at least 5 polyps (> 2 mm).\n- Free and informed consent, signed by all holders of parental authority/legal representative, and assent of the minor patient.\n- Covered by or affiliated to a social security scheme."}

Exclusion criteria

• {"criterion_text":"- Inability to understand the nature and goals of the study and/or communication difficulties with the investigator.\n- Contraindication to performing a colonoscopy.\n- Advanced disease with high-grade dysplasia adenoma or even adenocarcinoma in situ that should required colectomy.\n- Signs of primary tuberculosis infection or respiratory infection\n- Any other medical or psychological condition deemed incompatible with the proper conduct of the study according to the investigator, in particular: History of cancer, Severe infections, immune deficiency, Family history of tuberculosis, Chronic pathology\n- Contraindications rapamycin use : Known hypersensitivity to rapamycin ; Unadvisable drug combinations (drugs interfering with CYP3A4 by inhibiting it (ketoconazole, voriconazole, itraconazole, telithromycin, clarithromycin) or activating it (rifampicin, rifabutin), live vaccines) ; Fructose intolerance, glucose-galactose malabsorption, sucrose isomaltase, lactase deficiency ; Liver disease with transaminases > 2.5 x normal ; Hematological involvement: anemia with Hb < 9 g/dL, leukopenia with leukocytes < 1000/mm3, platelets < 80,000/mm3 ; Hypercholesterolemia with LDL-cholesterol > 2 g/L ; Quantiferon positive\n- Participation in another clinical trial taking an investigational drug in the 3 months prior to the inclusion visit, or subject in an exclusion period for another clinical trial.\n- Patient with one or both parents under legal protection (guardianship, curatorship, safeguard of justice).\n- Pregnancy, breastfeeding, not agree to use effective contraception for the entire duration of the study and for 3 months after the end of the research."}

Primary endpoints

• {"endpoint_text":"- Monitoring of adverse events (AEs) and serious adverse events (SAEs) for the duration of the experimental drug (rapamycin) and up to one month after discontinuation.","definition_or_measurement_approach":"Safety monitoring of AEs and SAEs throughout rapamycin treatment period and for one month after treatment discontinuation; adverse events will be recorded and followed per standard safety reporting procedures."}

Secondary endpoints

• {"endpoint_text":"- Individual analysis of colonoscopies, blinded to patient identity and the timing of colonoscopy in relation to treatment (V1 or V12), enabling descriptive analysis of the number of polyps over 2 mm per segment (rectum, left colon, transverse colon, right colon) and the size of the largest polyp in each segment.","definition_or_measurement_approach":"Blinded individual review of colonoscopy images/reports to count polyps >2 mm per colon segment and measure the largest polyp size per segment; descriptive analysis planned."}
• {"endpoint_text":"- Analysis of colonoscopies in a matched manner for each patient (pre-treatment (V1) / post-treatment (V12)) in order to be able to carry out a more specific evaluation of the evolution of polyps after 6 months of rapamycin treatment.","definition_or_measurement_approach":"Paired (pre/post) colonoscopy comparison for each patient to assess change in polyp number and size after approximately 6 months of treatment."}

France

Earliest CTIS Part Ii Submission Date

05-09-2024

Latest Decision Or Authorization Date

10-04-2025

Processing Time Days

217

Number Of Sites

4

Number Of Participants

25

Primary sponsor

Full Name

Centre Hospitalier Universitaire De Toulouse

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

France