Accession Number : AD1047178

Title :   LAM Pilot Study with Imatinib Mesylate (LAMP-1)

Descriptive Note : Technical Report,30 Sep 2016,29 Sep 2017

Corporate Author : Medical University of South Carolina Charleston United States

Personal Author(s) : Strange,Charlie ; Brown,Kimberly

Full Text :

Report Date : 01 Oct 2017

Pagination or Media Count : 16

Abstract : Lymphangioleiomyomatosis (LAM) is a rare disease in which tumor cells (LAM cells) proliferate and destroy healthy lung tissue, leading to respiratory compromise or failure. Vascular endothelial growth factor-D (VEGF-D) is generated by LAM cells and is a robust biomarker for LAM disease activity and therapeutic response. Studies in the laboratory of Dr. D'Armiento suggest that imatinib mesylate (imatinib) could completely block the growth of LAM cells through initiation of targeted cell death. Currently, most LAM patients are treated with Sirolimus (rapamycin). Rapamycin growth inhibits but does not kill LAM cells. This pilot trial employs a dual agent design intended to generate safety and efficacy data sufficient to power and design a phase 3 study of imatinib vs placebo for LAM. The hypothesis is that imatinib will be equivalent to rapamycin in short term efficacy and safety. Importantly, VEGF-D level will be used as a marker for LAM disease activity in this small clinical trial design using 20 participants at two institutions. IRB and HRPO approvals are in place and enrollment of participants is anticipated in the upcoming quarter.

Descriptors :   lung diseases , growth factors , biological markers , drugs , clinical trials

Subject Categories : Medicine and Medical Research

Distribution Statement : APPROVED FOR PUBLIC RELEASE