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Safety study for the use of Rapamycin in children with familial adenomatous polyposis - RAPA-4-FAP
EU CTIS ID: 2024-512079-11-00
What this study is testing
To evaluate the safety profile of two doses of rapamycin in adolescents with Familial Adenomatous Polyposis.
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Children aged 12 to 17 included at time of inclusion.
- 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.)
- Visualization of at least 5 polyps (> 2 mm).
- Free and informed consent, signed by all holders of parental authority/legal representative, and assent of the minor patient.
- Covered by or affiliated to a social security scheme.
You likely can't join if
- Inability to understand the nature and goals of the study and/or communication difficulties with the investigator.
- Contraindication to performing a colonoscopy.
- 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, in particular: History of cancer, Severe infections, immune deficiency, Family history of tuberculosis, Chronic pathology
- 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
See the full eligibility criteria
Who can join
- Children aged 12 to 17 included at time of inclusion.
- 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.)
- Visualization of at least 5 polyps (> 2 mm).
- Free and informed consent, signed by all holders of parental authority/legal representative, and assent of the minor patient.
- Covered by or affiliated to a social security scheme.
What rules you out
- Inability to understand the nature and goals of the study and/or communication difficulties with the investigator.
- Contraindication to performing a colonoscopy.
- 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, in particular: History of cancer, Severe infections, immune deficiency, Family history of tuberculosis, Chronic pathology
- 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
- 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.
- Patient with one or both parents under legal protection (guardianship, curatorship, safeguard of justice).
- 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.
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.