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

NCT06308445 | Not Yet Recruiting Phase 2 DMC

• 1 other identifier: RC31/23/0388
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 4

Brief Summary

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.

Conditions

Familial Adenomatous Polyposis

Keywords

Safety use; rapamycin; prophylaxis; teenager

Outcome Measures

Primary Outcomes (1)

• 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).

  For the entire duration of taking the experimental drug (rapamycin) and up to one month after stopping.

Secondary Outcomes (3)

• 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

  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.

  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

  After 6 months of treatment with rapamycin

Study Arms (1)

Rapamycin

EXPERIMENTAL

Drug: Rapamycin

Interventions

Rapamycin DRUG

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.

Rapamycin

Eligibility Criteria

• Age: 12 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• University Hospital, Toulouse: lead

Study Sites (4)

CHU Bordeaux Hôpital Pellegrin

Bordeaux, 33076, France

Location

CHU Montpellier Hôpital Arnaud de Villeneuve

Montpellier, 34295, France

Location

APHP Hôpital Robert Debré

Paris, 75019, France

Location

CHU Toulouse Hôpital des Enfants

Toulouse, 31300, France

Location

MeSH Terms

Conditions

Adenomatous Polyposis Coli

Interventions

Sirolimus

Condition Hierarchy (Ancestors)

Adenomatous Polyps; Adenoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms; Colorectal Neoplasms; Intestinal Neoplasms; Gastrointestinal Neoplasms; Digestive System Neoplasms; Neoplasms by Site; Neoplastic Syndromes, Hereditary; Digestive System Diseases; Gastrointestinal Diseases; Colonic Diseases; Intestinal Diseases; Intestinal Polyposis; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals

Study Officials

• Emmanuel Mas, Pr

  University Hospital, Toulouse

  STUDY CHAIR

Central Study Contacts

Emmanuel Mas, Pr

CONTACT

+33 5 34 55 84 45; mas.e@chu-toulouse.fr

Isabelle KIEFFER, CRA

CONTACT

kieffer.i@chu-toulouse.fr

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 22, 2024
• First Posted: March 13, 2024
• Study Start: August 1, 2025
• Primary Completion (Estimated): August 1, 2030
• Study Completion (Estimated): August 1, 2030
• Last Updated: January 31, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will not share

Locations

FR (4)