Familial adenomatous polyposis
Conditions
Interventions
Sponsors
Peking Union Medical College Hospital
Eligibility
Sex/Gender
All
Age
12 Years to No maximum
Inclusion criteria
Inclusion criteria: 1. A confirmed FAP patient with APC gene mutation confirmed by genetic testing; 2. Aged >= 12 years, no gender limit; 3. Physical status ECOG 0-3; 4. There are FAP-related lesions (such as gastrointestinal adenomas, desmoid tumors, etc.) in the colon and rectum with observable curative effects; 5. The organ function is good, and the biochemical examination meets the following conditions: AST<=2.5 x upper limit of normal (ULN), ALT<=2.5 x upper limit of normal (ULN), serum total bilirubin <=1.5 x upper limit of normal (ULN), Creatinine<=1.5 x upper limit of normal (ULN); 6. Volunteer to participate in the trial, and signed an informed consent form by the person or legal guardian.
Exclusion criteria
Exclusion criteria: 1. Patients with intestinal obstruction, intussusception, gastrointestinal hemorrhage, etc. who need emergency surgery; 2. A history of surgery or endoscopic treatment within 1 month; 3. Allergic to rapamycin; 4. Patients with malignant tumors that can be radically removed; 5. There are any diseases that require immediate blood transfusion; 6. Any disease or condition that may affect the implementation of the research or the interpretation of the results, including: (1) Suffer from gastrointestinal infection at the same time; (2) Those who have serious heart, liver, kidney and other serious concomitant diseases that may be life-threatening; (3) Patients with contraindications for digestive endoscopy; (4) Long-term use of other drugs (including non-steroidal anti-inflammatory drugs) that have an impact on the interpretation of research results; 7. Pregnant or lactating women; 8. Abusers of alcohol, drugs or drugs (for example, laxatives); 9. Patients who participate in another clinical trial that may affect the purpose of this research at the same time or within one month; 10. Researchers believe that other reasons are not suitable for clinical trials.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The number, size, and load of colorectal adenomas; | — |
Secondary
| Measure | Time frame |
|---|---|
| Nutritional status;Pains and quality of life;Frequency and severity of adverse events at therapeutic dose;Anemia related indicators;Pathological changes of adenomas;Changes of extra-intestinal manifestations; | — |
Countries
China
Contacts
Public ContactZhou Jiaolin
Peking Union Medical College Hospital
Outcome results
None listed