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

Peutz-Jeghers Syndrome Clinical Trial

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

Clinical Trial 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.

Clinical Trial 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. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT03781050
Study type Interventional
Source Peking Union Medical College Hospital
Contact Jiaolin Zhou, MD
Phone 13910136704
Email conniezhjl@yahoo.com
Status Recruiting
Phase Phase 4
Start date September 16, 2018
Completion date July 1, 2022
View Details
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