A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Participants with Immunoglobulin A Nephropathy (IgAN)

Date Added
February 12th, 2025
PRO Number
Pro00137540
Researcher
Anand Achanti

List of Studies


Keywords
Kidney
Summary

IgAN is a progressive condition that causes chronic kidney disease. Over time, some patients develop end-stage kidney disease (ESKD) requiring dialysis or kidney transplant.

The purpose of this study is to see if an investigational medication is safe and effective in the treatment of IgA nephropathy.

If you are eligible for the study and decide to participate, you will come to MUSC for study visits for about 3 years. These visits will involve blood and urine samples, questions about your health, EKGs, and physical exams. Participants will be randomized in a 1:1 ratio to receive either a weight-based IV infusion of ravulizumab or placebo for 106 weeks. Depending on your response to initial treatment, there is a possibility of additional treatment. An exploratory, open-label cohort of approximately 20 participants with eGFR 20-29 mL/min/1.73m2 may also be enrolled to expand the evidence of ravulizumab in participants with advanced kidney impairment at high risk for ESKD progression.

There is no guarantee that being in this study will help you. There is no cost to participate and compensation is available for the study visits.

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

A Phase 3, Randomized, Double-blind, Add-on Study Evaluating the Safety and Efficacy of Navtemadlin Plus Ruxolitinib vs Placebo Plus Ruxolitinib in JAK Inhibitor-Naïve Patients with Myelofibrosis Who Have a Suboptimal Response to Ruxolitinib

Date Added
March 5th, 2025
PRO Number
Pro00138827
Researcher
Alexander Coltoff

List of Studies

Keywords
Cancer, Drug Studies, Men's Health, Women's Health
Summary

This phase 3 study is recruiting patients who have myelofibrosis who have never had a JAK inhibitor. This study will measure the safety and effectiveness of a tumor protein inhibitor treatment called navtemadlin combined with another tumor protein inhibitor called ruxolitinib. Navtemadlin is an "investigational" (not yet FDA approved) treatment, Ruxolitinib is FDA approved. The main purpose of the study is to see if navtemadlin combined with ruxolitinib is an effective treatment for myelofibrosis. The study will enroll approximately 180 patients with each patient initially receiving ruxolitinib. The study includes a screening period, run-in period, and a randomized (like flipping a coin) add-on period. The first two periods will be over the course of 18-24 weeks while the randomized add-on period is for those whose treatment with ruxolitinib is not effective enough and will last for a different amount of time for each patient. In the run-in period after screening, patients will take ruxolitinib at the dose determined by their study doctor for 18-24 weeks. If treatment with ruxolitinib alone is not effective, the participate will be randomized into one of two groups. In the randomized add-on period, participants will either receive ruxolitinib with navtemadlin 240 mg or a matching placebo (a pill that contains no medicine) daily for one week out of the 28-day cycle in combination with ruxolitinib at a dose determined by their study doctor. Patients in this group will continue treatment until disease progression, unacceptable toxicity, study closure, death, or withdrawal of consent. The main risk is that medical treatments often cause side effects. Patients may have none, some, or all of the side effects listed or not listed in the protocol, and they may be mild, moderate, or severe. There is no direct benefit for them in participating in this study.

Institution
MUSC
Recruitment Contact
HCC Clinical Trials Office
8437929321
hcc-clinical-trials@musc.edu

Drowning Risks of Patients Presenting to the Pediatric Emergency Department at MUSC's Shawn Jenkins Children's Hospital

Date Added
March 11th, 2025
PRO Number
Pro00142153
Researcher
M. Titus

List of Studies


Keywords
Pediatrics
Summary

This study is seeking to assess two known risk factors of drowning, swimming ability and water safety knowledge, in parents of children and adolescents presenting to the pediatric emergency department using questionnaires.

By collecting this data, we hope to highlight factors that contribute to disparities in drowning rates in minority racial and ethnic groups and to aid local and federal governments in developing programs that effectively combat the number one cause of unintentional injury-associated death in children ages 1-4 years. This will also provide data that may help guide pediatricians in effective anticipatory guidance for families regarding water safety. All in the effort to minimize disparities in medicine and provide more equitable care to the patients that we see.

Institution
MUSC
Recruitment Contact
M. Titus
8434753656
titusda@musc.edu

MIrikizumab in UC a Real-woRld prOspective multicenteR registry

Date Added
March 18th, 2025
PRO Number
Pro00142550
Researcher
Erin Forster

List of Studies


Keywords
Inflammatory Bowel Disease
Summary

The goal is to see how well mirikizumab works for people with ulcerative colitis. This involves looking at its effects in their everyday lives. Understanding its real-world benefits helps determine how effective the medicine is outside of controlled clinical trials.

Institution
MUSC
Recruitment Contact
Isabella Brookshire
8437920965
recruitment@musc.edu

An Open-label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of EDG-7500 in Adults with Hypertrophic Cardiomyopathy

Date Added
March 25th, 2025
PRO Number
Pro00142239
Researcher
Jessica Atkins

List of Studies

Keywords
Drug Studies, Heart
Summary

This study is studying the investigational medication EDG-7500 for the treatment of obstructive and non obstructive hypertrophic cardiomyoapthy. Investigational means it is not approved for sale by the Food and Drug Administration (FDA) The study will test to see if the medication can help the heart expand and fill to help its workload. The study has 4 Parts, each of varying durations. The study will last a minimum of 6 weeks to a maximum of 18 months. Study related procedures include physical exam, electrocardiogram (ECG) - a tracing of the heart's electrical activity, echocardiogram (echo) - ultrasound test of the heart, blood and urine collection, possible genetic testing, and exercise testing. Risks related to the study medication include atrial fibrillation (fast irregular heartbeat), constipation, diarrhea, dizziness/lightheadedness and upper respiratory infection. Other study related risks include loss of confidentiality, unknown risks and risks related to procedures.

Institution
MUSC
Recruitment Contact
Cara Breunig
843-792-7519
breunig@musc.edu

cfDNA Assay Multicenter Prospective Observational Validation for Early Cancer Detection, Minimal Residual Disease, and Relapse (CAMPERR)

Date Added
April 9th, 2025
PRO Number
Pro00143358
Researcher
Gerard Silvestri

List of Studies


Keywords
Cancer
Summary

This study aims to develop a new blood test to detect and identify many different types of cancer, using a special technique that looks at tiny changes in your DNA. Some participants will be followed over time to see if this method can also find leftover cancer cells (after treatment), and if it could warn if the cancer comes back. However, this test still under development, so there are no results reported back to participants. The goal is to create a reliable tool that one day could help doctors diagnose and monitor cancer(s) more effectively.

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

cfDNA Assay Multicenter Prospective Observational Validation for Early Cancer Detection, Minimal Residual Disease, and Relapse (CAMPERR)

Date Added
April 9th, 2025
PRO Number
Pro00143358
Researcher
Gerard Silvestri

List of Studies


Keywords
Cancer
Summary

This study aims to develop a new blood test to detect and identify many different types of cancer, using a special technique that looks at tiny changes in your DNA. Some participants will be followed over time to see if this method can also find leftover cancer cells (after treatment), and if it could warn if the cancer comes back. However, this test still under development, so there are no results reported back to participants. The goal is to create a reliable tool that one day could help doctors diagnose and monitor cancer(s) more effectively.

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

An Open-label Extension Study Evaluating the Long-term Safety and Efficacy of Seralutinib Orally Inhaled for the Treatment of Pulmonary Arterial Hypertension (PAH)

Date Added
April 24th, 2025
PRO Number
Pro00137101
Researcher
Rahul Argula

List of Studies


Keywords
Pulmonary Hypertension
Summary

This is a multicenter, open-label extension (OLE) study to see how safe and effective an inhaled medication called seralutinib is for people with Pulmonary Arterial Hypertension (PAH). The primary objective of this study was to assess improvement in cardiopulmonary hemodynamics as measured by change in pulmonary vascular resistance (PVR) from baseline to Week 24. The secondary objective of this study was to assess improvement in exercise capacity as measured by change in six-minute walk distance (6MWD) from baseline to Week 24.

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

Circuitry-guided repetitive transcranial magnetic stimulation (rTMS) for tobacco use in Veterans: A comparison of insula-rTMS and prefrontal-rTMS

Date Added
May 6th, 2025
PRO Number
Pro00141919
Researcher
Xingbao Li

List of Studies


Keywords
Brain, Healthy Volunteer Studies, Smoking, Substance Use
Summary

Quitting smoking is hard, and many Veterans struggle even with current treatments. This study is testing a safe, non-invasive brain stimulation called repetitive transcranial magnetic stimulation (rTMS), which is already FDA-approved to help people stop smoking. We're comparing two types—standard and personalized—to see which works better. We aim to find the best option to help Veterans quit for good.

Institution
MUSC
Recruitment Contact
Cyra Valente
843 792-0136
VA email in process (valentec@musc.edu)

Neuron23, Inc. / "A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of NEU-411 in Companion Diagnostic-Positive Participants with Early Parkinson's Disease (NEULARK)"

Date Added
May 13th, 2025
PRO Number
Pro00142584
Researcher
Christine Cooper

List of Studies


Keywords
Parkinsons
Summary

The goal of the research study is to see if NEU-411 (1) will help prevent or slow the worsening of LRRK2 driven Parkinson's Disease (PD) and, (2) is safe. This study will also determine how your body processes NEU 411 and what NEU-411 does to your body. The study doctor will discuss with you whether you are eligible to be part of this study. As part of this study, you will receive either NEU-411 or placebo (inactive substance). The study drug is in tablet form and is taken by mouth. The study will last about 67 weeks. You will come in for 19 visits. In addition to in-person visits with the study team, you will be required to complete daily tests using a smartphone and occasionally be asked to wear a smartwatch continuously for 1-week periods. These technologies will be provided to you for use during the duration of the study and you will be trained on how to perform these activities. The NEU-411 is investigational and has not been approved by the US Food and Drug Administration as well as the investigational Companion Diagnostic (CDx).

Institution
MUSC
Recruitment Contact
Robin Bulgarino
8437921115
bulgarino@musc.edu



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