Study on the Safety of Sirolimus for Children with Familial Adenomatous Polyposis

2 1 1 1

What is this study about?

This clinical trial is focused on studying the safety of a medication called Rapamycin in children who have a condition known as Familial Adenomatous Polyposis. Familial Adenomatous Polyposis is a genetic disorder that causes the growth of numerous polyps in the colon and rectum, which can lead to cancer if not treated. The medication being tested, Rapamycin, is taken in the form of coated tablets and is intended to help manage this condition.

The purpose of the study is to evaluate how safe Rapamycin is for adolescents aged 12 to 17 who have been diagnosed with Familial Adenomatous Polyposis. Participants in the study will receive one of two doses of Rapamycin. Throughout the study, the safety of the medication will be closely monitored by checking for any adverse events, which are unwanted effects that might occur during or after taking the medication. The study will also involve regular colonoscopies, which are procedures that allow doctors to look inside the colon to see how the polyps are changing over time.

The study will last for several months, during which participants will take Rapamycin and undergo various assessments to ensure their safety and to observe any changes in the polyps. The results from these assessments will help researchers understand the safety profile of Rapamycin in treating Familial Adenomatous Polyposis in young patients. This information could be valuable in developing effective treatments for this condition in the future.

1 initial assessment

Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes a review of medical history and a colonoscopy to verify the presence of familial adenomatous polyposis, characterized by at least 5 polyps larger than 2 mm.

Consent forms must be signed by the legal guardians, and the minor patient must provide assent.

2 medication administration

The study involves the administration of Rapamune (sirolimus) 2 mg coated tablets, taken orally. The dosage and frequency are determined by the study protocol and are aimed at evaluating the safety of two different doses.

The treatment period lasts for 6 months, during which the patient will take the medication as prescribed.

3 monitoring and follow-up

Throughout the treatment period, regular monitoring is conducted to observe any adverse events (AEs) or serious adverse events (SAEs). This monitoring continues for one month after the medication is discontinued.

Follow-up colonoscopies are performed to analyze the number and size of polyps in different segments of the colon. These are compared to pre-treatment colonoscopies to assess changes over the course of the study.

4 final evaluation

At the end of the treatment period, a final evaluation is conducted. This includes a comprehensive review of the patient’s response to the medication and any side effects experienced.

The results of the colonoscopies are analyzed to determine the impact of the treatment on the polyps.

Who Can Join the Study?

  • Children must be between 12 and 17 years old at the time they join the study.
  • Patients need to have had a colonoscopy, which is a test to look inside the colon, for diagnosing or checking on familial adenomatous polyposis. This condition is identified if there is a family history of an APC mutation in the child, or if several adenomatous polyps are found due to rectal discharge.
  • Doctors must see at least 5 polyps (small growths) that are larger than 2 millimeters.
  • All parents or legal guardians must sign a form giving their free and informed consent, and the child must also agree to participate.
  • The child must be covered by or affiliated with a social security scheme.

Who Cannot Join the Study?

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Centre Hospitalier Universitaire De Bordeaux Bordeaux France
Oncopole Claudius Regaud Toulouse France

Other Sites

Site Name City Country Status
Robert Debre University Hospital Paris France
Centre Hospitalier Universitaire De Montpellier Montpellier France

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
France France
Not yet recruiting
01.10.2024

Trial locations

Investigated drugs:

Rapamycin is being studied for its safety in children with familial adenomatous polyposis. This medication is being tested to see if it can be used safely in adolescents who have this genetic condition, which causes the growth of numerous polyps in the colon and rectum. The trial aims to understand how the body reacts to the medication and to ensure that it does not cause harmful side effects.

Familial Adenomatous Polyposis – This is a genetic disorder characterized by the development of numerous polyps in the lining of the colon and rectum. These polyps typically begin to form during adolescence and can increase in number over time. If left untreated, the polyps have a high likelihood of becoming cancerous. The condition is inherited in an autosomal dominant pattern, meaning a single copy of the altered gene in each cell is sufficient to cause the disorder. Individuals with this condition may also develop polyps in other parts of the gastrointestinal tract. Regular monitoring and management are essential to address the progression of the disease.

Trial ID:
2024-512079-11-00
Protocol code:
RC31/23/0388
NCT ID:
NCT06308445
Trial Phase:
Therapeutic exploratory (Phase II)

Other Trials to Consider

  • A study testing sirolimus to slow disease progression in patients with familial adenomatous polyposis

    Recruiting

    3 1
    Investigated drugs:
    Denmark Germany Italy The Netherlands Spain