In short
There is currently 1 ongoing clinical trial investigating treatment for Adenomatous Polyposis Coli, also known as Familial Adenomatous Polyposis. This trial is focused on evaluating the safety of Rapamycin (sirolimus) in adolescent patients aged 12 to 17 years and is being conducted in France.
Clinical trial locations
Study on the Safety of Sirolimus for Children with Familial Adenomatous Polyposis
This clinical trial is investigating the safety of Rapamycin, also known as sirolimus, in adolescents with Familial Adenomatous Polyposis. This genetic disorder causes numerous polyps to grow in the colon and rectum, which can develop into cancer without proper treatment.
Who can participate:
- Adolescents between 12 and 17 years old
- Those who have had a colonoscopy showing at least 5 polyps larger than 2 millimeters
- Patients with a confirmed family history of APC mutation or multiple adenomatous polyps
- Those covered by or affiliated with a social security scheme
- Both parents or legal guardians must provide informed consent, and the child must also agree to participate
Who cannot participate:
The trial has specific exclusion criteria to ensure patient safety, though detailed exclusion criteria were not provided in the source data.
What the trial involves:
The study evaluates the safety of Rapamycin, which is taken as coated tablets by mouth. Participants will receive one of two different doses over a 6-month treatment period. Rapamycin works by inhibiting a protein called mTOR, which plays an important role in cell growth and multiplication. The medication is typically used to prevent organ transplant rejection, but researchers are investigating whether it can safely help manage polyp growth in young patients with this genetic condition.
Throughout the study, doctors will closely monitor participants for any unwanted effects. Regular colonoscopies will be performed to examine the colon and observe how the polyps change over time. This monitoring continues for one month after the medication is stopped. The colonoscopy results will be compared to those taken before treatment began to assess the medication's impact on the number and size of polyps in different sections of the colon.
Study goals:
The main focus of this trial is to determine how safe Rapamycin is for adolescents with Familial Adenomatous Polyposis. Researchers aim to understand how the body reacts to the medication and ensure it does not cause harmful side effects. The information gathered could be valuable in developing effective treatments for this condition in young patients and potentially reduce the number of polyps, delaying the need for surgical intervention.
Summary
Currently, there is one clinical trial available for patients with Adenomatous Polyposis Coli, specifically targeting adolescent patients aged 12 to 17 years. The trial is being conducted in France and focuses exclusively on evaluating the safety profile of Rapamycin (sirolimus) in this young patient population. The study represents an important step in exploring alternative treatment options for this genetic condition, which traditionally requires surgical management. The trial's emphasis on safety assessment is particularly important given the young age of participants and the medication's novel application in this context. Families interested in participating should consult with their healthcare providers to determine if they meet the eligibility criteria.
