2024-512079-11-00招募中2 期
Safety study for the use of Rapamycin in children with familial adenomatous polyposis - RAPA-4-FAP
适应症
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 25
- 试验地点
- 4
- 主要终点
- Monitoring of adverse events (AEs) and serious adverse events (SAEs) for the duration of the experimental drug (rapamycin) and up to one month after discontinuation.
研究概览
简要总结
To evaluate the safety profile of two doses of rapamycin in adolescents with Familial Adenomatous Polyposis.
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •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.
排除标准
- •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.
结局指标
主要结局
Monitoring of adverse events (AEs) and serious adverse events (SAEs) for the duration of the experimental drug (rapamycin) and up to one month after discontinuation.
Monitoring of adverse events (AEs) and serious adverse events (SAEs) for the duration of the experimental drug (rapamycin) and up to one month after discontinuation.
次要结局
- Individual analysis of colonoscopies, blinded to patient identity and the timing of colonoscopy in relation to treatment (V1 or V12), enabling descriptive analysis of the number of polyps over 2 mm per segment (rectum, left colon, transverse colon, right colon) and the size of the largest polyp in each segment.
- Analysis of colonoscopies in a matched manner for each patient (pre-treatment (V1) / post-treatment (V12)) in order to be able to carry out a more specific evaluation of the evolution of polyps after 6 months of rapamycin treatment.
研究者
Pr MAS Emmanuel
Scientific
Centre Hospitalier Universitaire De Toulouse
研究点 (4)
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