Efficacy of Rapamycin (Sirolimus) in the Treatment of Peutz-Jeghers Syndrome

Last updated: January 18, 2019
Sponsor: Peking Union Medical College Hospital
Overall Status: Active - Recruiting

Phase

4

Condition

Precancerous Condition

Colon Polyps

Treatment

N/A

Clinical Study ID

NCT03781050
HS-1607
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Patients are diagnosed with PJS.

  • Patients have gastrointestinal polyps related syndromes, including abdominal pain,abdominal distension, gastrointestinal bleeding, etc, with imageological examinationsuggesting intestinal obstruction or intussusception; or whose symptoms recur afterprevious digestive endoscopic treatment and surgery; or who are inappropriate orunwilling 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 tothe clinical trial.

  • Patients participate in the trial voluntarily and have signed the informed consent bythe participant or his/her legal guardian.

Exclusion

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 thestudy 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 endangerlives

  • 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 tobecome a subject.

Study Design

Total Participants: 10
Study Start date:
September 16, 2018
Estimated Completion Date:
July 01, 2022

Study 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 and prevention of PJS. The efficacy of sirolimus (rapamycin) on PJS has been demonstrated in mouse model, but no clinical trials have been reported. Our study was designed as a prospective non-randomized open label single arm clinical trial to examine its efficacy and safety.

Connect with a study center

  • Peking Union Medical College Hospital, Chinese Academy of Medicine Sciences

    Beijing, China/Beijing 100000
    China

    Active - Recruiting

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