Status:
UNKNOWN
Efficacy of Rapamycin (Sirolimus) in the Treatment of Peutz-Jeghers Syndrome
Lead Sponsor:
Peking Union Medical College Hospital
Collaborating Sponsors:
Air Force General Hospital of the PLA
Cancer Institute and Hospital, Chinese Academy of Medical Sciences
Conditions:
Peutz-Jeghers Syndrome
Eligibility:
All Genders
Phase:
PHASE4
Brief 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.
Detailed Description
PJS (Peutz-Jeghers Syndrome) is mostly caused by mutations in Lkb1 gene, which leads to an increased activity of mTOR pathway, thus making mTOR inhibitor (sirolimus) a promising candidate in treatment...
Eligibility Criteria
Inclusion
- 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
- 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 of the results. This type of diseases includes:
- Known severe blood coagulation disorders
- Known anemia that is not caused by intestinal polyps
- Known hemoglobinopathy
- Other gastrointestinal infectious diseases
- Serious heart, liver, kidney and other concomitant diseases that may endanger lives
- Patients are in pregnancy and lactation.
- Alcohol or drug (such as aperient) abuse
- Patients took part in another clinical trial that may influence this study.
- The researchers believe that there are other unfavorable reasons for the patient to become a subject.
Key Trial Info
Start Date :
September 16 2018
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
July 1 2022
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT03781050
Start Date
September 16 2018
End Date
July 1 2022
Last Update
January 23 2019
Active Locations (1)
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1
Peking Union Medical College Hospital, Chinese Academy of Medicine Sciences
Beijing, China/Beijing, China, 100000