Efficacy of Rapamycin (Sirolimus) in the Treatment of Peutz-Jeghers Syndrome (NCT03781050) | Clinical Trial Compass
UnknownPhase 4
Efficacy of Rapamycin (Sirolimus) in the Treatment of Peutz-Jeghers Syndrome
China10 participantsStarted 2018-09-16
Plain-language summary
A prospective non-randomized open label single arm clinical trial to examine the efficacy and safety of sirolimus in patients with Peutz-Jeghers Syndrome.
Who can participate
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Patients are diagnosed with PJS.
* Patients have gastrointestinal polyps related syndromes, including abdominal pain, abdominal distension, gastrointestinal bleeding, etc, with imageological examination suggesting intestinal obstruction or intussusception; or whose symptoms recur after previous digestive endoscopic treatment and surgery; or who are inappropriate or unwilling to accept the above treatment again and wish to receive pharmacotherapy.
* Conventional treatment didn't work well in patients combined with PJS-related tumors.
* Physical condition (ECGO): 0\~3
* Organ function is good and biochemical indices meet the following conditions:
* AST≤2.5×upper limit of normal value (ULN),
* ALT≤2.5×upper limit of normal value (ULN),
* Serum total bilirubin (TSB)≤1.5×upper limit of normal value (ULN),
* Creatinine≤1.5×upper limit of normal value (ULN).
* No other medications have been received for intestinal polyps within 3 months prior to the clinical trial.
* Patients participate in the trial voluntarily and have signed the informed consent by the participant or his/her legal guardian.
Exclusion Criteria:
* Patients underwent a surgery within 2 weeks.
* Patients may need emergency surgery in the near future.
* Patients are allergic to any ingredient of rapamycin.
* Patients suffer from a disease requiring immediate blood transfusion.
* Patients suffer from any disease or condition that may impact implementation of the study or interpretation…
What they're measuring
1
Total load of PJS-related intestinal polyps Total load of PJS-related intestinal polyps Total load of PJS-related intestinal polyps
Timeframe: The time from start of therapy to 1 year