COMPASS Ancillary NeuroDevelopmental Outcomes (CAN-DO)

Date Added
April 15th, 2026
PRO Number
Pro00148202
Researcher
Sinai Zyblewski

List of Studies


Keywords
Cardiovascular, Pediatrics
Summary

The main reason for this research study is for researchers to evaluate the relationship between congenital heart disease and development. Currently, there is not enough long-term information available to researchers to predict a child's development if they have been diagnosed with Ductal Dependent Pulmonary Blood Flow (DDPBF), a type of congenital heart disease.

Institution
MUSC
Recruitment Contact
Susannah Wakefield
843-792-8317
wakefies@musc.edu

The Impact of Toxic Exposures on Vascular Autoantibodies in Lupus Associated Cardiovascular Disease

Date Added
April 10th, 2026
PRO Number
Pro00147299
Researcher
Helen Butler

List of Studies

Keywords
Cardiovascular, Lupus
Summary

The purpose of this study is to understand how exposure to harmful substances during military service may affect the health of Veterans with or without lupus. Lupus is an autoimmune disease that can increase the risk of cardiovascular problems.

We believe that Veterans who were exposed to toxic substances during their military service may develop more harmful antibodies that attack the lining of their blood vessels. These antibodies may contribute to poorer blood vessel and heart health, and could contribute to the development of lupus.

This study aims to improve our understanding of how toxic military exposures may increase the risk of blood vessel complications in Veterans with and without lupus. Ultimately, this research may help identify new ways to better prevent, monitor, or treat cardiovascular disease in this population.

Research procedures for this study will include:

1. The study team will check subject medical records to gather information about medical history and medications being taking. The study team may continue to follow updates in the medical record.
2. Subjects will be given a survey to assess military and occupational toxic inhalant exposures.
3. Subjects will have a brief physical examination during which vitals will be recorded (height, weight, heart rate, respiration, temperature). Women of childbearing ages will be asked for the date of their last menstrual cycle within the past 2 months.
4. Subjects will have blood pressure taken three times three minutes apart.
5. Subjects will then provide a urine sample. Urine collection will occur in a private restroom using a sterile container provided by the study team. For women of childbearing ages, a pregnancy dipstick test will be undertaken on urine to confirm subjects are not pregnant.
6. Subjects will undergo a blood draw where approximately 4 teaspoons of blood will be drawn.

Institution
MUSC
Recruitment Contact
Helen Butler
7046540686
butlehel@musc.edu

A Phase 1 Dose Escalation Trial Evaluating an Intravenously Administered Recombinant Adeno-associated Virus Serotype rh.74 (AAVrh.74) Vector Containing the Human BCL2-associated Athanogene 3 (BAG3) Gene Coding Sequence (RP-A701, AAVrh.74-BAG3) in Subjects with Dilated Cardiomyopathy Arising from Pathogenic BAG3 Variants (BAG3-DCM)

Date Added
February 10th, 2026
PRO Number
Pro00149195
Researcher
Daniel Judge

List of Studies


Keywords
Cardiovascular, Drug Studies, Genetics, Heart
Summary

This study is seeking participants with BAG3-associated dilated cardiomyopathy (DCM). BAG3-DCM is a rare genetic disorder. Dilated cardiomyopathy is a condition that causes the heart to have a harder time pumping blood to the rest of the body which can lead to heart failure. Current treatment for BAG3-DCM is focused on improving heart function and preventing advanced heart failure with medicines, procedures and devices.

This study involves gene therapy. This will be the first time that a BAG3 gene containing study drug will be tested in human volunteers. The purpose of this research is to learn whether the investigational gene therapy RP-A701 is safe and effective for patients with BAG3-DCM. Gene therapy involves the addition of one or more genes to your cells to replace a missing gene or correct malfunctioning genes. Investigational means it is not currently approved by the Food and Drug Administration (FDA). RP-A701 will be given as a one time infusion into a vein in your arm.

Participation in this study will last about 2 years and include at least 18 visits including an inpatient hospitalization stay of at least 5 days. Study related procedures include review of your medical records, study drug infusion, immunosuppressant and antibiotic medications, echocardiogram (ultrasound test of your heart) exercise testing, electrocardiogram (recording of your heart's electrical activity), heart biopsy (collecting a piece of heart tissue), cardiac MRI, questionnaires, heart rhythm monitoring and ICD interrogations, and collection of blood, saliva, urine and stool collection. Study related risks related to gene therapy and those related to study procedures including risks of the heart catheterization, radiation, and biopsy, exercise testing, blood draw risks, genetic testing risks, the risk of loss of confidentiality and unknown risks.

Institution
MUSC
Recruitment Contact
Clara Andrews
843-876-5011
andrewcl@musc.edu

A randomized, double-blind, placebo-controlled, multicenter study to evaluate the efficacy, safety, and tolerability of pelacarsen (TQJ230) with background inclisiran in participants with atherosclerotic cardiovascular disease, and elevated LDL-C and Lp(a)

Date Added
January 13th, 2026
PRO Number
Pro00148513
Researcher
Ashley Waring

List of Studies

Keywords
Cardiovascular, Cholesterol
Summary

This study is being done to learn more about the study drug pelacarsen (TQJ230) in people with atherosclerotic cardiovascular disease (ASCVD) and elevated Lp(a) and LDL-C levels who are already taking a medication called inclisiran for the treatment of elevated LDL-C. ASCVD refers to the build up of plaques in the blood vessels that can block blood flow and increase the risk of events like heart attacks, strokes or other blood vessel blockages. LDL-C stands for low density lipoprotein cholesterol which is often referred to as bad cholesterol. Pelacarsen is considered investigational meaning it has not yet been approved by the Food and Drug Administration (FDA). This is a randomized study meaning you will be assigned by chance, like the flip of a coin, to receive the study drug or placebo. You have a 1 in 2 chance (50%) of receiving the study drug. Inclisiran is an FDA approved medication to lower LDL-C. The study drug is given as a monthly injection under the skin. This study will last about 21 months and include about 14 visits.

Institution
MUSC
Recruitment Contact
Melissa Amitrano
843-876-5009
amitrano@musc.edu

Enhancing Heart Allograft Function with the OCS Heart System (ENHANCE) Trial

Date Added
December 16th, 2025
PRO Number
Pro00148033
Researcher
Arman Kilic

List of Studies

Keywords
Cardiovascular, Surgery, Transplant
Summary

This trial is designed to evaluate the safety and effectiveness of the novel OCS Solution and OCS Functional Enhancer (OFE) to support FDA approval in both DBD and DCD heart transplantation. In addition, this trial will evaluate the performance of the novel OCS Solution and OFE compared to Static Cold Storage (SCS) in DBD heart transplantation to potentially demonstrate superiority.

Institution
MUSC
Recruitment Contact
Morgan Overstreet
843-792-8896
overstrm@musc.edu

A Phase 3, Open-Label, Multicenter, Extension Study of Acoramidis in Patients with Newly Diagnosed Variant Transthyretin Amyloid Cardiomyopathy (ACT-EARLY OLE)

Date Added
December 9th, 2025
PRO Number
Pro00148422
Researcher
Jan Griffin

List of Studies

Keywords
Cardiovascular, Drug Studies, Heart
Summary

This study is an open label extension of the ACT-EARLY study. which included those with no evidence of ATTR but are known carriers of disease causing TTR gene. ATTR stands for transthyretin amyloidosis. It is a condition in which a protein called transthyretin (TTR) accumulates in various organs, including the heart (known as ATTR-CM), kidneys, and nerves (known as ATTR-PN). This accumulation can lead to damage and dysfunction in these organs.

This study will continue using the study drug acoramidis (AG-10) to determine if it can help people with the genetic TTR variant slow the progression of ATTR. AG-10 is an investigational drug. Investigational means that AG-10 is not yet approved for use in any settings outside of clinical research studies like this one. Reducing the amount of TTR in your blood may reduce the amount of amyloid deposits in your body and may keep your cardiomyopathy from getting worse over time.

Participation in this study will last up to 60 month and will consist of about 13 clinic visits and about 11 telephone follow up visits. Some tests required include physical exams, medical and surgical history, bloodwork, questionnaire, electrocardiogram (test that records your heart's electrical activity), echocardiogram (ultrasound test of your heart) and study drug administration.

Institution
MUSC
Recruitment Contact
Natalie Forrester
843-876-5037
drain@musc.edu

Saline Enhanced Radiofrequency (SERF) Needle Ablation for Refractory VT

Date Added
November 18th, 2025
PRO Number
Pro00147339
Researcher
Jeffrey Winterfield

List of Studies


Keywords
Cardiovascular, Heart
Summary

This study is testing a new treatment for people with a dangerous heart rhythm problem called ventricular tachycardia (VT). VT can cause the heart to beat too fast, leading to fainting, heart failure, or even sudden death. Some people continue to have VT even after taking medicines and undergoing standard ablation procedures. For these patients, current treatment options are very limited.

The investigational treatment uses the Thermedical Ablation System with the Durablateā„¢ catheter. This device delivers both heat and saline (salt water) deep into the heart muscle to target the areas causing abnormal rhythms. The goal is to safely and effectively reduce or eliminate VT episodes in patients who have not responded to other therapies.

About 130 patients will be enrolled at up to 25 hospitals in the U.S. and Canada. Participants will have the procedure and then be followed for six months with regular checkups to see if the treatment reduces their VT episodes and improves their quality of life. This study will help determine if the new system should be approved for wider clinical use.

Institution
MUSC
Recruitment Contact
Shaquanda Goodwine
843-876-5783
shr37@musc.edu

The Single Ventricle Outcomes Network (SV-ONE)

Date Added
November 14th, 2025
PRO Number
Pro00147843
Researcher
Frances Woodard

List of Studies


Keywords
Cardiovascular, Heart, Infant
Summary

SV-ONE represents the integration of NPC-QIC within the existing FON framework. As such, SV-ONE will engage in research and improvement efforts through the entire lifespan of patients with SVHD, including but not limited to those with a Fontan circulation. The larger objective of this study is to increase longevity and enhance the QoL by improving physical health and functioning, mental health and resilience, and neurodevelopment for individuals with SVHD and their families. A longer-term goal of SV-ONE will be to serve as a platform for research and improvement that will
accelerate advances, with the potential to nest clinical trials and to link to registries and programs,
nationally and internationally.

Institution
MUSC
Recruitment Contact
Frances Woodard
843-792-3292
klinefl@musc.edu

A Phase 1/2 Long-term Follow-up Study of the Safety and Efficacy of LX2020 Gene Therapy in Patients with Arrhythmogenic Cardiomyopathy due to a Plakophilin-2 Pathogenic Variant (GRIT-PKP2)

Date Added
November 12th, 2025
PRO Number
Pro00147805
Researcher
Daniel Judge

List of Studies


Keywords
Cardiovascular, Heart
Summary

The GRIT-PKP2 study will evaluate the long-term safety and tolerability of LX2020 gene therapy for subjects with PKP2-ACM who have previously received an investigational study medication called LX2020. PKP2-ACM is an inherited heart condition which can cause heart muscle and electrical damage. Investigational means it is not approved for use by the Food and Drug Administration (FDA). There is no investigational treatment being administered in this study. The overall study duration is 4 years. The study consists of 7 visits: a rollover visit, an outpatient monitoring period, (5 visits, 18 months to 48 months after LX2020 administration in Study LX2020-01), and an end-of-study visit. Procedures and activities that subjects will undergo are: quality of life questionnaires, ECG, MRI, ultrasound, ECHO, collection of samples, remote cardiac monitoring, and collection of vital signs.

Institution
MUSC
Recruitment Contact
Tania Pegues
843-876-6996
peguest@musc.edu

Evaluation of the GORE Ascending Stent Graft in the Treatment of De Novo Type A Aortic Dissections (ARISE III)

Date Added
October 23rd, 2025
PRO Number
Pro00147128
Researcher
Sanford Zeigler

List of Studies


Keywords
Cardiovascular, Surgery
Summary

The purpose of this research is to assess the safety and effectiveness of the ASG device in the treatment of de novo Type A aortic dissections.

Institution
MUSC
Recruitment Contact
Morgan Overstreet
843-792-8896
overstrm@musc.edu



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