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

Launched by UNIVERSITY HOSPITAL, TOULOUSE · Mar 8, 2024

Keywords

ClinConnect Summary

This clinical trial is studying the safety and effectiveness of a medication called rapamycin for teenagers aged 12 to 17 who have a condition known as Familial Adenomatous Polyposis (FAP). FAP causes many polyps, which are small growths, to form in the colon, and this study aims to see if rapamycin can help reduce the number and size of these polyps. The researchers will be giving participants two low doses of rapamycin and will closely monitor how well the teens tolerate the medication and whether it helps with their condition.

To participate in this study, teens must have at least five polyps larger than 2 millimeters found during an initial colonoscopy (a procedure to examine the colon). They also need consent from their parents or legal guardians and must agree to use contraception if they are capable of becoming pregnant. This trial is not yet recruiting participants, so it’s important for families to know that they will need to wait until the study starts to apply. Participants will receive careful medical supervision throughout the study and will help researchers learn more about managing FAP in young people.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 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.
• • Affiliated with a social security scheme.
• • Patients of childbearing potential must agree to the use of a method of contraception during the study.
• Exclusion Criteria:
• • 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
• • Signs of primary tuberculosis infection or respiratory infection
• • Any other medical or psychological condition deemed incompatible with the proper conduct of the study according to the investigator.
• • Contraindications to rapamycin use
• • Participation in other biomedical research
• • Deprivation of liberty of the legal guardians by judicial or administrative decision.
• • Pregnancy, breastfeeding