RELIANCE: A phase 1, randomized, double-blinded, placebo-controlled, trial evaluating the long-term safety and tolerability of Imatinib for the treatment of Lymphangioleiomyomatosis (LAMP-2 Trial)

Date Added
July 30th, 2024
PRO Number
Pro00136539
Researcher
Charlie Strange

List of Studies


Keywords
Kidney, Lung, Pulmonary, Rare Diseases, Women's Health
Summary

The purpose of this medical research study is to evaluate the safety and effectiveness of a new medication called imatinib mesylate in the treatment of Lymphangioleiomyomatosis (LAM). LAM is a rare disease in which abnormal cells (called LAM cells) grow out of control. Over time, LAM cells destroy healthy lung tissue and cause respiratory disease or failure.

Many patients with LAM are currently treated with a medication called sirolimus (rapamycin). Sirolimus slows the growth of LAM cells.

Imatinib mesylate (hereafter called imatinib) is approved by the Food and Drug Administration (FDA) for the treatment of some cancers that share common pathways with LAM cells. Laboratory studies suggest that imatinib could completely block the growth of LAM cells through initiation of targeted cell death.

An important purpose of this research is to determine the safety of imatinib in people with LAM. This study will also evaluate the short-term effectiveness of imatinib. Participants will be randomized to receiving imatinib (study medication) or placebo (no treatment) for the 180 day duration of participation. The study is being conducted at the Medical University of South Carolina and at Columbia University in New York (CUMC). Each site will enroll 10 participants.

Institution
MUSC
Recruitment Contact
Mary Hayden
(843) 792-8438
blantonm@musc.edu

Patient reported outcomes in patients with nontuberculous mycobacterial pulmonary disease

Date Added
July 29th, 2024
PRO Number
Pro00133747
Researcher
Patrick Flume

List of Studies


Keywords
Lung, Pulmonary
Summary

Nontuberculous mycobacteria (NTM) cause a chronic pulmonary infection associated with cough, fatigue, and shortness of breath. Our primary objectives are to better understand the trajectory of patient-reported outcomes (PROs), e.g.
respiratory symptoms and health-related quality of life (HRQoL), across the entire disease course and measure toxicity and tolerability using patient-reported symptomatic adverse events in treated patients with nontuberculous mycobacterial pulmonary disease (NTM-PD).

Institution
MUSC
Recruitment Contact
Zerlinna Teague
8437920965
recruitment@musc.edu

Rapid Onsite Cytopathologic Evaluation for Improved Molecular Marker Testing via Endobronchial Ultrasound Bronchoscopy - A Randomized Controlled Trial

Date Added
July 3rd, 2024
PRO Number
Pro00128969
Researcher
Christopher Gilbert

List of Studies

Keywords
Cancer, Cancer/Lung, Lung
Summary

In this study, all eligible study participants will have a clinically indicated reason to undergo a bronchoscopy procedure, using a EBUS-TBNA scope, before enrolling in the study. EBUS-TBNA stands for "endobronchial ultrasound-guided transbronchial needle aspiration" which is a procedure to collect lymph node tissue from within the lungs by inserting a flexible tube-like camera through the mouth, then down into the lungs.

After providing consent to participate in this study, each subject will be randomly assigned (by chance, like the flip of a coin) to either have or not have a cytopathology technologist (person who looks at samples under a microscope) in the room during your bronchoscopy, and then randomly assigned again (by chance, like the flip of a coin) to either have your tissue specimen prepared with clot or liquid preparation. Clot preparation is placing the tissue biopsy in a gel prior to being viewed under a microscope, whereas liquid preparation is placing the tissue biopsy in a liquid prior to being viewed under a microscope.

It is important to note that, by participating in this study, subjects will still receive the standard of care with a proven track record for obtaining lymph node tissue.

The reason for this research study is to determine whether rapid on-site cytopathologic evaluation, or the clot based method, can increase the chances of gathering adequate tissue for Next Generation Sequencing. Next generation sequencing is a test which detects molecular markers from tissue, or blood, samples and can provide possible treatment options for specific forms of lung cancer.

Institution
MUSC
Recruitment Contact
Michael Balassone
843-792-6696
balassom@musc.edu

A Phase II Study of Fingolimod in Patients with Non-Small Cell and Small Cell Lung Cancer

Date Added
June 18th, 2024
PRO Number
Pro00137404
Researcher
Mariam Alexander

List of Studies

Keywords
Cancer, Cancer/Lung, Cancer/Other, Drug Studies, Lung, Men's Health, Women's Health
Summary

This study is for subjects that have been diagnosed with lung cancer and the disease has progressed on prior therapy. The purpose of the study is to determine the safety and efficacy of Fingolimod and whether it can aid in treatment against lung cancer tumors. Fingolimod is not FDA approved and is considered an investigational drug. Subjects can expect to be in this study for about 8 months, with routine visits occurring at MUSC.

Institution
MUSC
Recruitment Contact
HCC Clinical Trials Office
843-792-9321
hcc-clinical-trials@musc.edu