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

Feasibility and Acceptability of A Mobile Application "SUPPORT-T" to increase access to Palliative Care in patients with Progressive Pulmonary Fibrosis (PPF) and their Caregivers.

Date Added
September 10th, 2025
PRO Number
Pro00146761
Researcher
Kathleen Lindell

List of Studies

Keywords
Education, Interstitial Lung Disease (ILD)
Summary

The goal of this research study is to explore and evaluate the acceptability and feasibility of a 8-week, nurse-led Palliative Care program (SUPPORT-T) delivered over the internet to help educate and support individuals with Progressive Pulmonary Fibrosis (PFF) and their caregivers. In this study, we will enroll 80 PFF patients and 80 careparnters (total N=160 participants) who will then either receive the online SUPPORT-T program or enhanced usual care (educational handouts). At the end of the study, 15 PFF patients and caregivers who received SUPPORT-T and 5 community partners will take part in one-on-one interviews with the researchers to provide their feedback on the program; so as to improve it for a larger study. This study is being conducted by the Medical University of South Carolina in South Carolina and Mount Sinai Hospital in New York.

Institution
MUSC
Recruitment Contact
Mary Chris Pittman
843-792-3512
pittmama@musc.edu

A Prospective, Multi-Center, Open Label, Randomized Control Clinical Trial Evaluating the Safety and Efficacy of the Cordella Pulmonary Artery Sensor System in New York Heart Association (NYHA) Class II - III Heart Failure Patients

Date Added
November 12th, 2025
PRO Number
Pro00147296
Researcher
Vishal Rao

List of Studies

Keywords
Heart, Pulmonary Hypertension
Summary

This study is enrolling patients diagnosed with New York Heart Association (NYHA) Class II and III Heart Failure (HF). The main purpose of this study is to investigate the safety and effectiveness of the study device in helping to reduce Heart Failure Hospitalizations (HFH). The study is divided into 3 time periods: a screening period, implant/treatment period, and a follow-up period.

The study involves implanting a Cordella™ Pulmonary Artery Sensor during a right-heart catheterization, after which, you will be randomized into the treatment group (your study doctor will use the data from your daily weight, blood pressure, heart rate measurements, and your daily sensor readings to treat your heart failure) or the active control group (your study doctor will only use the data from your daily weight, blood pressure, and heart rate measurements to treat your heart failure). You will have a 66% (2 in 3) chance of being in the treatment group and a 33% (1 in 3) chance of being in the active control group. Randomization is much like a like drawing straws where you have a 66/33 chance of being assigned to the treatment group or the active control group.

After you have been implanted for 6 months, you and your study doctor will be "unblinded" (able to see) the daily measurements from the Cordella™ Pulmonary Artery Sensor System. Follow-up visits will occur at 1, 3, 6, 12, 18, and 24 months, then annually up to 5 years.

Risks include complications from the implant procedure (bleeding, infection, damage to blood vessels, allergic reactions), risks from contrast dye, anesthesia, or medications (e.g., bleeding from blood thinners), and potential unknown device-related risks.

Institution
MUSC
Recruitment Contact
Shaquanda Goodwine
843-876-5783
shr37@musc.edu



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