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 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

An open-label, multi-centre, rollover study to characterise long-term safety and efficacy of etavopivat in adults, adolescents and children who have sickle cell disease or thalassaemia and have completed a treatment period in an etavopivat study

Date Added
November 21st, 2025
PRO Number
Pro00139301
Researcher
Shayla Bergmann

List of Studies


Keywords
Blood Disorders
Summary

The purpose of this rollover study is to investigate the long-term safety of etavopivat in participants11 months of age and older with SCD or thalassaemia who have completed a treatment period in previous etavopivat studies (parent studies, see Section 4.1). Long-term clinical efficacy measures of etavopivat treatment will also be assessed. This study will also ensure that participants who are benefiting from etavopivat treatment have prolonged access to the drug in the time before it is commercially available in their country.

Institution
MUSC
Recruitment Contact
Lexi Schorg
8437920603
schorg@musc.edu

EndoForce Post Approval Study

Date Added
December 11th, 2025
PRO Number
Pro00147536
Researcher
MARK LONDON

List of Studies

Keywords
Kidney
Summary

The goal of this observational study is to collect long-term safety and performance data for the use of the EndoForce System for connecting a hemodialysis graft to a vein in patients with End Stage Renal Disease. This is not an experimental procedure or an experimental therapy. This means that the study device has been approved by the FDA.

Institution
MUSC Health Orangeburg
Recruitment Contact
Kristen Covington
803-395-2021
covingkr@musc.edu

A Randomized, Double-Blind, Vehicle-Controlled (Period 1) and Open-Label (Period 2)Phase 3 Study to Investigate the Safety and Efficacy of Tapinarof Cream, 1% in Pediatric Participants Ages 3 Months to < 24 Months with Atopic Dermatitis

Date Added
January 6th, 2026
PRO Number
Pro00148113
Researcher
Lara Wine Lee

List of Studies


Keywords
Skin
Summary

This Phase 3 study evaluates the safety and efficacy of tapinarof cream, 1%, for treating atopic dermatitis (AD) in pediatric participants aged 3 to under 24 months. Previous research, including two Phase 3 pivotal studies and a long-term study, assessed the cream in adults and children as young as 2 years. These studies showed minimal to no systemic exposure in children aged 2 to 17 with extensive AD. The findings support investigating the cream's safety and efficacy in younger participants, specifically infants and toddlers.

Institution
MUSC
Recruitment Contact
Andie Hoskins
843-792-6882
hoskinsa@musc.edu

MB-TBS: Mindfulness-Based Theta-Burst Stimulation: A Pilot Study Integrating Mindfulness Training Into Accelerated Neuromodulation Therapy for Depression

Date Added
January 8th, 2026
PRO Number
Pro00148491
Researcher
Clayton Olash

List of Studies

Keywords
Depression
Summary

This study examines whether adding brief guided mindfulness exercises to accelerated Transcranial Magnetic Stimulation (TMS) treatment can help people with depression feel better and maintain those improvements longer. Participants will already be receiving accelerated intermittent theta-burst stimulation (aiTBS) as part of their clinical care. During the breaks between TMS sessions, they will have the option to listen to short, app-based mindfulness practices using the commercially available and free Healthy Minds App. We will track whether this combined approach is practical, how easy participants find the meditations, and whether practicing mindfulness during treatment improves mood, well-being, or long-term recovery. The information gathered will help us design a larger study focused on improving the durability of TMS outcomes.

Institution
MUSC
Recruitment Contact
Clayton Olash
5023778275
olash@musc.edu

Ehlers Danlos Syndrome Research Biorepository and Database

Date Added
January 9th, 2026
PRO Number
Pro00147160
Researcher
Russell Norris

List of Studies


Keywords
Allergy, Chronic Fatigue, Genetics, Nervous System, Pain, Rare Diseases, Sleep Disorders, Surgery
Summary

This study aims to create a long-term Ehlers-Danlos syndrome (EDS) biorepository and clinical research database to support gene and biomolecular discovery. The repository will serve as a sustainable resource for advancing EDS-related research by collecting both clinical data and biological samples. Participants who consent will be included in the EDS registry, which stores demographic and operative information, contact details, and biological specimens for current and future studies. Enrolled patients may also choose to be recontacted for future research opportunities. The database will link participants across specialties using identifiers such as name, date of birth, and medical record number. Data collected will include information from electronic health records, such as clinical notes, diagnoses, medications, labs, imaging, anthropometric measures, and procedure reports.

Institution
MUSC
Recruitment Contact
Tayler Goectau
8437921544
goectau@musc.edu



-- OR --