LAM Pilot Study with Imatinib Mesylate (LAMP-1)
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. VEGF-D level will define LAM disease activity in this small clinical trial design using 20 participants at two institutions. At the time of this report 13 participants have been enrolled, 12 completed the protocol and 1 withdrew due to inability to perform spirometry.
Document Details
- Document Type
- Technical Report
- Publication Date
- Oct 01, 2018
- Accession Number
- AD1094194
Entities
People
- Charlie Strange
- Kimberly Foil
Organizations
- Medical University of South Carolina