An Open-Label, Phase 2 Study Evaluating the Safety of Inhaled Mosliciguat with Inhaled Treprostinil in Participants with Pulmonary Hypertension Associated with Interstitial Lung Disease

Date Added
March 31st, 2026
PRO Number
Pro00150037
Researcher
Rahul Argula

List of Studies


Keywords
Lung, Pulmonary
Summary

The purpose of the study is to find out if mosliciguat, the investigational drug that is being studied, with treprostinil, is safe and effective in treating adults who live with PH-ILD. Approximately 20 participants diagnosed with PH-ILD who are currently on a stable dose of inhaled treprostinil who meet all inclusion and no exclusion criteria will be enrolled. Part 1 of the study will take about 5 ½ months. Part 2 of the study is optional if you choose to participate in the extension period. Part 2 does not have a set end date and your participation in Part 2 will end if you or your study doctor decide to stop your participation or the Sponsor ends the study.

Institution
MUSC
Recruitment Contact
Zerlinna Teague
(843) 792-0965
recruitment@musc.edu

An Open-label Long-term Follow-up Study to Evaluate the Effects of Sotatercept when Added to Background Pulmonary Arterial Hypertension (PAH) Therapy for the Treatment of PAH (MK-7962-038)

Date Added
March 26th, 2026
PRO Number
Pro00150204
Researcher
Rahul Argula

List of Studies


Keywords
Pulmonary, Pulmonary Hypertension
Summary

This is a Phase 3, open-label, follow-up study to evaluate the long-term safety,
tolerability, and efficacy of sotatercept when added to background PAH therapy for the treatment of PAH. Participants enrolled in this study are from the following parent studies: PULSAR, SPECTRA, STELLAR, HYPERION, and ZENITH. Participants eligible to enroll in this study will have participated in and completed the requirements of the MK-7962-004 study (Amendment 07 or later). In this study, participants will receive open-label sotatercept . The duration of participation for each participant will vary based on their enrollment date, but it is anticipated to be up to 3 years.

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

Prospective Observational Study on the Natural History of Alpha-1 Antitrypsin Deficiency and Associated Liver Disease (ALPHATUDE)

Date Added
March 20th, 2026
PRO Number
Pro00147215
Researcher
Charlie Strange

List of Studies


Keywords
Liver, Lung, Pulmonary, Rare Diseases
Summary

This an observational study collecting data for up to 8 years on patients who have been diagnosed with PiZZ or PiSZ Alpha-1 Antitrypsin Deficiency with or without liver disease. Patients' clinical, medical, and laboratory data will be collected prospectively per routine care and questionnaires will be collected during the clinic visits with the hopes of getting a better understanding of the natural progress of lung and liver disease associated with Alpha-1 Antitrypsin Deficiency.

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

Phase 2 Study of the Efficacy and Safety of ORC-13661 for the Prevention of Drug-Induced Hearing Loss in Patients Receiving Intravenous Amikacin Therapy for Pulmonary Non-Tuberculous Mycobacterium Disease

Date Added
January 27th, 2026
PRO Number
Pro00147995
Researcher
Patrick Flume

List of Studies


Keywords
Lung, Nontuberculous mycobacteria (NTM), Pulmonary
Summary

The purpose of the study is to learn more about an experimental drug called ORC-13661 that may be helpful in treating hearing loss due to antibiotic use. The study is researching whether the drug is safe and tolerable, and trying to find out whether the drug may be able to mitigate or prevent hearing loss in patients being treated with intravenous (IV) amikacin. Participation in the study will consist of up to 10 visits over up to 129 days. The Day 1 visit (following the screening visit) will last up to 8 hours, while other visits will last up to 5 hours Participants will be randomly assigned to receive either ORC-13661at a higher dose or a lower dose or a placebo (a pill that looks the same as the study drug but has no real medicine in it), for up to 90 days to be taken along with IV amikacin. Participants will be asked to have a number of tests and procedures, which include questionnaires about family, medical, and hearing history, physical and hearing examination, and assessments of hearing.

Institution
MUSC
Recruitment Contact
Rohini Rao
8437926109
recuitment@musc.edu

A Phase 2a randomized, double-blind, placebo-controlled proof-of-concept study to evaluate the safety, tolerability, pharmacodynamics, and pharmacokinetics of SION-719 when added to physician-prescribed Trikafta™ in people with cystic fibrosis who are homozygous for the F508del mutation

Date Added
November 20th, 2025
PRO Number
Pro00147883
Researcher
Christina Mingora

List of Studies

Keywords
Cystic Fibrosis, Lung, Pulmonary
Summary

The purpose of this research study is to assess if the study drug SION-719 is safe and well-tolerated in people with cystic fibrosis who are already taking Trikafta, understand the effect of SION-719 on the body by measuring chloride levels, and to learn how the body absorbs, distributes, and gets rid of the study drug in people with cystic fibrosis. This study is a placebo controlled cross-over study with two study treatment periods, and participants will take part in both study treatment periods. Participants will be assigned either study drug or placebo. Participants will remain on standard dose of physician-prescribed Trikafta throughout the study. This study will be approximately 16 weeks, or 112 days long.

Institution
MUSC
Recruitment Contact
Rohini Rao
8437926109
recuitment@musc.edu

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 Phase 2, Randomized, Double-Blind, Placebo-Controlled Study of the Safety, Tolerability and Efficacy of Caveolin-1-Scaffolding-Protein-Derived Peptide (LTI-03) in Patients with Idiopathic Pulmonary Fibrosis

Date Added
June 17th, 2025
PRO Number
Pro00145146
Researcher
Timothy Whelan

List of Studies


Keywords
Interstitial Lung Disease (ILD), Lung, Pulmonary
Summary

This study will compare the safety, tolerability, and efficacy of inhaled study drug LTI-03 to placebo in participants diagnosed with IPF within 5 years of Screening who may be receiving standard of care (SoC) antifibrotic therapy. Taking part in this research is expected to last up to 32 weeks, which includes a 4-week screening period, a 24-week treatment period, and 4-week post-treatment follow-up period. The general procedures include review of eligibility; review of medical and medication history and changes in health; completing questionnaires; undergoing physical exams and having vital signs measured; collecting blood samples; examinations such as ECG, spirometry, HRCT, and DLCO and using an inhaler to take the study drug.

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

Sleep and Activity Habits of Inpatients with Chest Tubes: A Pilot Observational Trial

Date Added
April 9th, 2025
PRO Number
Pro00140546
Researcher
Christopher Gilbert

List of Studies

Keywords
Lung, Pulmonary
Summary

This is a non-invasive, observational study aiming to collect data on the impact chest tubes have on inpatients during their hospitalizations. Sleep and activity patterns, as well as select vital signs will be tracked using an MUSC-owned Apple Watch, which subjects will wear, and an MUSC-owned iPhone, for up to seven days during admission to the hospital. Each day, a study coordinator will visit subjects to collect a brief sleep survey. Participation may last up to 7 days while subjects are admitted and have a chest tube in place. Upon completion of the study, a study coordinator will collect the Apple Watch and iPhone for data analysis.

This pilot data will provide initial information on the feasibility of collecting and performing a larger study on the inpatient mobility, activity, and sleep.

Institution
MUSC
Recruitment Contact
Randal Neff
8437924557
Neffr@musc.edu

A Multicenter Randomized Trial of EBUS-TBNA versus Transbronchial Mediastinal Cryobiopsy for Adequacy of Next Generation Sequencing: FROSTBITE-3

Date Added
March 18th, 2025
PRO Number
Pro00140380
Researcher
Christopher Gilbert

List of Studies

Keywords
Cancer, Lung, Pulmonary
Summary

This study is for patients scheduled for a bronchoscopy, a procedure that allows doctors to examine lungs and airways for evaluation of suspicious lung nodules. Those who give consent to participate in this study will be randomized into one of two lung biopsy sampling method groups based on the Rapid On Site Evaluation of the initial sample obtained by the doctor performing the bronchoscopy as part of the standard of care procedure for the patient. the potential groups the subject will be randomized into are: Cryoprobe or transbronchial biopsy needle. These devices are used with a bronchoscope to obtain lung tissue biopsy samples and are being evaluated to determine which is better for confirming a diagnosis. All procedures will be done via standard of care and screening will be accomplished via medical chart review. 7 days following the procedure, a member of the study team will check the subjects medical chart to assess whether any adverse events have occurred.

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



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