A Phase 2, Randomized, Double-blind, Placebo-controlled, Multiple Dose Ranging Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Solrikitug in Participants with Chronic Obstructive Pulmonary Disease (ZION)

Date Added
September 9th, 2025
PRO Number
Pro00145511
Researcher
Charlie Strange

List of Studies


Keywords
Breathing, Inflammation, Pulmonary, Shortness of Breath
Summary

Solrikitug is an investigational drug, a drug that is still in development and testing, that can potentially benefit individuals with chronic obstructive pulmonary disease (COPD) by the pathway that causes type 2 inflammation. Solrikitug is given as an injection in the arm. Eligible participants will be randomized to receive a single dose of study medication (Solrikitug 375mg), three doses (day 1, week 4 and week 8) or placebo (inactive drug). The study is double blinded so neither the treatment team nor the participant will know what drug treatment they received. Enrollment in the study is up to 32 weeks, 4 weeks for screening and 28 weeks for treatment and follow up. Assessments conducted at the study visits include, but are not limited to, medical history and medication review, physical exams, blood tests, EKGs to look at the heart, breathing tests and questionnaires.

Institution
MUSC
Recruitment Contact
Gwen Hayden
843-792-8438
blantonm@musc.edu

BTX-302-001: A Phase 1/2 dose-exploration and dose-expansion study to evaluate the safety and efficacy of BEAM-302 in adult patients with Alpha-1 Antitrypsin Deficiency (AATD)-associated lung disease and/or liver disease.

Date Added
September 9th, 2025
PRO Number
Pro00146485
Researcher
Charlie Strange

List of Studies


Keywords
Genetics, Liver, Lung, Pulmonary, Rare Diseases
Summary

BTX-302-001 is a research study investigating the safety (how many side effects participants may have) and tolerability (how tolerable the side effects are) of BEAM-302 for individuals with Alpha-1 Antitrypsin Deficiency (AATD)-associated lung and/or liver disease. This study also aims to gather additional information regarding how BEAM-302 moves through the participant's body, how long it stays, and how long it takes to eliminate it - which is defined as the study drug's pharmacokinetics or "PK". Researchers would like to determine through this research study how BEAM-302 impacts the disease course (progression) of AATD in terms of AATD blood biomarkers, which are substances in blood that the body normally makes and will help show if an individual's AATD is improving, staying the same, or getting worse, along with lung and liver function testing results and the quality of life of participants.

This research study will be split into two main parts, Part A (which is for individuals with AATD-associated lung disease with no clear evidence of AATD-associated liver disease) and Part B (which is for individuals with AATD-associated liver disease). Additionally, each Part will be split into two separate cohorts, where one cohort will receive a single intravenous (IV) infusion of BEAM-302 (single-dose cohort) and the other will receive two IV infusions of BEAM-302 approximately 8 weeks apart (multi-dose cohort). Within these cohorts (single-dose and multi-dose), there are also separate smaller cohorts that will vary by the dose of BEAM-302 administered to participants, so a participant in this study could receive any of the following dosages - 15mg, 30mg, 60mg, 75mg, or 90mg. Overall, the research study will last up to around 29 months for each participant, depending on which cohort they are in, and their participation will be split into three main study periods - Screening, Dose and Dose-limiting toxicity (DLT), and Follow-up. It is also important to note that when a participant is receives their infusion(s) of BEAM-302 during the Dose and DLT period, the administration of the study drug will be done as a part of an in-patient hospital stay that will last up to 48 hours so that they can be closely monitored by the study team.

The key eligibility criteria for this study are that individuals (male or female) must be 18 to 70 years old, possess the PiZZ type of AATD, and have either AATD-associated lung disease with no clear evidence of AATD-associated liver disease or AATD-associated liver disease. There are additional eligibility criteria that must be met in order to be able to participate in the study, which will be assessed across up to 2 study visits that will occur during the Screening period.

Institution
MUSC
Recruitment Contact
Mary Hayden
843-792-8432
blantonm@musc.edu

A randomized controlled trial of transcutaneous electrical nerve stimulation for pain mitigation during intrauterine device placement

Date Added
September 16th, 2025
PRO Number
Pro00143776
Researcher
Michelle Meglin

List of Studies


Keywords
Obstetrics and Gynecology
Summary

We are conducting this study to determine whether using a TENS (nerve stimulation) unit is effective at reducing pain in women having an IUD inserted.

Institution
MUSC
Recruitment Contact
Jesslyn Payne
843-792-7525
paynej@musc.edu

Accelerated High-Dose tDCS for Depression: An Open-Label Outpatient Pilot Study

Date Added
October 7th, 2025
PRO Number
Pro00146983
Researcher
Mark George

List of Studies


Keywords
Depression, Nervous System
Summary

In this study, we are testing whether a higher dose of a non-invasive brain stimulation technique, called transcranial direct current stimulation (tDCS), can be safely used in people with depression. Participants will come to the Brain Stimulation Lab and receive mild electrical stimulation through electrodes placed on their scalp.

The study begins with a safety run-in, where the first few participants will receive stimulation at gradually increasing levels (2, 4, and 6 milliamps) while being closely monitored. If no serious side effects are found, later participants will receive repeated 6 milliamp sessions for 5 days total. We will check skin comfort, mood, and overall tolerability after each session.

Institution
MUSC
Recruitment Contact
Clayton Olash
843-243-7305
olash@musc.edu

A Pilot Study to Assess the Effects of Simeox 200 Airway Clearance Device in Bronchiectasis Patients with Chronic Mucus Hypersecretion

Date Added
October 14th, 2025
PRO Number
Pro00147345
Researcher
Patrick Flume

List of Studies


Keywords
Bronchiectasis, Lung
Summary

This will be a study in the home care setting to further validate the concept of Simeox 200 in a cohort of patients with bronchiectasis and overproduction of mucous as an alternative to other methods of ACT(airway clearance techniques). Bronchiectasis is a chronic lung disease where airways become permanently damaged, widened, and lose their ability to clear mucus, leading to a buildup of bacteria, recurrent infections, and symptoms like a daily cough, thick, discolored phlegm, shortness of breath, and fatigue. The device is a 510(k) FDA cleared device that works using by air and vibration to help clear mucous from the lungs. There are 4 visits and an at home usage period. The study will be running approximately a year or until the cohort is filled. The data from this study will be used to power a future randomized controlled pivotal clinical study comparing Simeox 200 against other ACTs such as High Frequency Chest Wall Oscillation (HFCWO).

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

TRITON-CM: A Phase 3 Global, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Nucresiran in Patients with Transthyretin-Mediated Amyloidosis with Cardiomyopathy (ATTR amyloidosis with cardiomyopathy)

Date Added
October 14th, 2025
PRO Number
Pro00146373
Researcher
Daniel Judge

List of Studies


Keywords
Drug Studies, Heart
Summary

This study is enrolling participants with symptomatic ATTR-CM (transthyretin amyloidosis cardiomyopathy). ATTR-CM is a rare and serious disease that occurs when a protein in the blood called transthyretin (TTR) builds up throughout the body, including in the heart and nerves. When the abnormal protein, known as amyloid, deposits in the heart, the heart muscle thickens and stiffens, causing the heart to fail. This research study is designed to test whether the medication nucresiran is safe and helps people with ATTR-CM, in comparison to the effects of placebo.

Nucresiran is considered investigational, meaning it is not currently approved by the U.S. Food and Drug Administration (FDA) for the treatment of ATTR-CM. Nucresiran is a TTR silencer. It is like a "quiet button" that turns down the amount of the disease-causing protein that is made. Because there is less TTR, there may be less buildup in the heart and other organs over time.

This is a randomized study meaning once eligibility is confirmed, participants will be assigned by chance, like drawing straws, to either receive nucresiran or placebo. You will have a 2 out of 3 chance of being assigned to nucresiran and a 1 out of 3 chance of being assigned placebo. Placebo is a substance that looks like the actual medication and is given the same way but contains no active substance. The study drug, which can be either nucresiran or placebo, will be given as an injection under the skin in the abdomen (avoiding the area around the navel), thigh, or the side or back of the upper arms. Neither the participants nor the study doctor will know who is assigned to nucresiran or placebo but this information can be made available if need be.

Participation in this study is expected to last for 5-8 years, Study related procedures include physical exams, vital signs, echocardiograms (ultrasound test of the heart), electrocardiograms, (ECG, a tracing of the heart's electrical activity), blood work, urine samples and questionnaires. Participants will also take Vitamin A daily. Study related risks include risks related to the study drug including injection site reactions, abnormal liver function or an allergic reaction. There may be risks related to study procedures including loss of confidentiality. There may not be any direct benefit, but the information learned may benefit others with ATTR-CM in the future.

Institution
MUSC
Recruitment Contact
Deborah Everidge
843-792-2944
adamsde@musc.edu

A Phase 2 Study to Evaluate the Safety, Tolerability, and Efficacy of BLU-808, a Wild Type KIT Inhibitor, in Chronic Inducible Urticaria and Chronic Spontaneous Urticaria

Date Added
October 23rd, 2025
PRO Number
Pro00145633
Researcher
Kelli Williams

List of Studies


Keywords
Allergy, Autoimmune disease, Skin
Summary

The purpose of this study is evaluate safety and effectiveness of an investigational drug in adults diagnosed with chronic urticaria (hives). The investigational drug is known as BLU-808 and will be taken daily orally. Participation in the study is expected to be approximately 18 weeks.

Institution
MUSC
Recruitment Contact
Emma McLeod
843-792-8008
mcleodem@musc.edu

Developing a communication support intervention for cancer caregivers in emerging and young adulthood

Date Added
October 29th, 2025
PRO Number
Pro00146111
Researcher
Amanda Kastrinos

List of Studies

Keywords
Cancer, Cancer/Brain, Cancer/Breast, Cancer/Gastrointestinal, Cancer/Genitourinary, Cancer/Leukemia, Cancer/Lung, Cancer/Lymphoma, Cancer/Myeloma, Cancer/Other, Cancer/Sarcoma, Cancer/Skin, Healthy Volunteer Studies
Summary

Cancer caregivers in emerging and young adulthood (ages 18-35) are an underresearched and unsupported group of caregivers, and yet they are not uncommon. To address this critical support gap, the goal of this study is to develop and pilot test a caregiving support intervention specifically tailored for emerging and young adults caring for a parent with cancer. This phase of the study is intended to capture feedback on the intervention via focus groups with emerging and young adult caregivers of a parent with cancer.

Institution
MUSC
Recruitment Contact
Amanda Kastrinos
843-876-9076
kastrinos@musc.edu

A Multinational, Long-Term, Open-Label Extension Study of Subjects Who Have Participated in Avalyn Pharma Studies of Inhaled Antifibrotic Agents (AP-LTE-008 SAIL)

Date Added
November 5th, 2025
PRO Number
Pro00146978
Researcher
Timothy Whelan

List of Studies


Keywords
Lung
Summary

This study gives people who were part of an earlier Avalyn research protocol with inhaled pirfenidone versus placebo an opportunity to convert to open label or active investigational drug treatment. The goal is to continue learning additional safety and efficacy information in patients over a longer period of treatment time. Pulmonary fibrosis is a disorder with a poor prognosis that is often far worse than many cancers with limited treatment options. While this medication has yet to be proven effective, the safety profile currently is acceptable and the risks versus benefits favor offering this opportunity to patients with informed consent.

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

Functional Outcomes of a Pickleball Community-Based Exercise Program for Individuals with Parkinson's Disease

Date Added
November 10th, 2025
PRO Number
Pro00148039
Researcher
Vanessa Hinson

List of Studies


Keywords
Exercise, Parkinsons
Summary

The purpose of this research is to explore the effects of participation in a pickleball community-based exercise program on function in people with Parkinson's disease (PwPD). Individuals will engage in an 8-week instructional pickleball exercise program designed specifically by Movement Disorder Specialists at the Medical University of South Carolina for PwPD. The pickleball training program involves warm-up exercises, stroke mechanics, fall safety and recovery technique training, effective movement pattern drills, game play strategies, and match play. We aim to quantify the motor function, balance, mobility, and quality of life outcomes through clinical assessments and questionnaires before and after participation in the Pick it Up Pickleball training program. This research will establish an evidence base for pickleball as a therapeutic intervention, opening the door for additional accessible and engaging exercise options for the PD community.

Institution
MUSC
Recruitment Contact
Isabelle Museck
317-478-7198
museck@musc.edu



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