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Clinical Trial Summary

A prospective, nonrandomized, open-label, single-arm clinical trial to study efficacy of rapamycin (sirolimus) in the treatment of Blue Rubber Bleb Nevus Syndrome, hereditary or sporadic venous malformation


Clinical Trial Description

Blue rubber bleb nevus syndrome (BRBNS) and venous malformation are mainly caused by somatic mutation of TEK and PIK3CA, which activates the PI3K/AKT signaling pathway. As an important protein kinase downstream of the PI3K/AKT pathway, mTOR can serve as a potential therapeutic target for BRBNS. Experiments of mice have shown that rapamycin inhibited the progression of venous malformation lesions. There are a few human cases reported using rapamycin treatment. The investigator's study is designed to be a prospective, nonrandomized, open-label, single-arm clinical trial to investigate its efficacy and safety. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT03767660
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 July 31, 2018
Completion date July 1, 2022

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