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 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

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

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

Lung Cancer Screening, Lung Nodule, and Lung Cancer Biorepository

Date Added
April 24th, 2025
PRO Number
Pro00143404
Researcher
Gerard Silvestri

List of Studies


Keywords
Cancer, Lung
Summary

This study will enroll patients and collect blood samples from those who are either in lung cancer screening, have a lung nodule that is suspicious for malignancy, a newly diagnosed cancer, or healthy control subjects. These samples that are being collected and banked will be used for future development of blood tests to detect lung cancer in future patients at the earliest stages.

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

Lung Cancer Screening, Lung Nodule, and Lung Cancer Biorepository

Date Added
April 24th, 2025
PRO Number
Pro00143404
Researcher
Gerard Silvestri

List of Studies


Keywords
Cancer, Lung
Summary

This study will enroll patients and collect blood samples from those who are either in lung cancer screening, have a lung nodule that is suspicious for malignancy, a newly diagnosed cancer, or healthy control subjects. These samples that are being collected and banked will be used for future development of blood tests to detect lung cancer in future patients at the earliest stages.

Institution
MUSC Health Florence Medical Center
Recruitment Contact
Michael Balassone
843-792-6696
Balassom@musc.edu

Lung Cancer Screening, Lung Nodule, and Lung Cancer Biorepository

Date Added
April 24th, 2025
PRO Number
Pro00143404
Researcher
Gerard Silvestri

List of Studies


Keywords
Cancer, Lung
Summary

This study will enroll patients and collect blood samples from those who are either in lung cancer screening, have a lung nodule that is suspicious for malignancy, a newly diagnosed cancer, or healthy control subjects. These samples that are being collected and banked will be used for future development of blood tests to detect lung cancer in future patients at the earliest stages.

Institution
MUSC Health Lancaster Medical Center
Recruitment Contact
Michael Balassone
843-792-6696
Balassom@musc.edu

Lung Cancer Screening, Lung Nodule, and Lung Cancer Biorepository

Date Added
April 24th, 2025
PRO Number
Pro00143404
Researcher
Gerard Silvestri

List of Studies


Keywords
Cancer, Lung
Summary

This study will enroll patients and collect blood samples from those who are either in lung cancer screening, have a lung nodule that is suspicious for malignancy, a newly diagnosed cancer, or healthy control subjects. These samples that are being collected and banked will be used for future development of blood tests to detect lung cancer in future patients at the earliest stages.

Institution
MUSC Health Columbia Medical Center
Recruitment Contact
Michael Balassone
843-792-6696
Balassom@musc.edu



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