A Phase 2/3 Adaptive, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of VX-147 in Adult and Pediatric Subjects with APOL1-mediated Proteinuric Kidney Disease

Date Added
May 11th, 2022
PRO Number
Pro00117678
Researcher
Roberto Pisoni

List of Studies


Keywords
Drug Studies, Genetics, Kidney, Minorities, Rare Diseases
Summary

This randomized, double-blind, placebo-controlled Phase 2/3 adaptive study involves an initial investigational blood test to determine if you have a specific variation related to kidney disease. The investigational blood test is to see if you have changes in your DNA of a gene called APOL1. People who have this gene variation may be at risk of losing their kidney function faster than others. If you have the variants (changes in DNA) you may be eligible to continue participation in the study. If you do not have the variants, you will not be eligible, and the study doctor will discuss your other options with you. If you decide to participate, there will be no cost to you and you will be compensated. This study will start by comparing two doses of VX-147 against placebo in subjects with APOL1-mediated kidney disease for 12 weeks. Subjects in Phase 2 will continue to Phase 3 once a dose for Phase 3 is selected. Then the Phase 3 dose of VX-147 will be evaluated for safety and effectiveness. If you meet the requirements and choose to take part in the study, you will be randomly assigned to a treatment group. You will not know which study treatment group you are assigned to and it is possible that you will receive placebo instead of VX-147. The study includes a screening, treatment, and follow-up period. The study will end after the last patient enrolled has completed 2 years in the study. This means some patients enrolling earlier could be in the study for up to 4 years.

Institution
MUSC
Recruitment Contact
Linda Walker
843-792-6109
walkerlp@musc.edu

Hyperhydration to Improve Kidney Outcomes in Children with Shiga Toxin-Producing E. coli Infection: A Multinational Embedded Cluster Crossover Randomized Trial (HIKO STEC Trial)

Date Added
May 11th, 2022
PRO Number
Pro00117570
Researcher
Christopher Pruitt

List of Studies


Keywords
Kidney, Pediatrics
Summary

This research study aims to learn more about children and adolescents who have a shiga toxin-producing E. coli (STEC) infection. E. coli is a type of bacteria found in the intestines. Although most types of E. coli are harmless, some produce toxins that can make children sick. This study will assess what type of treatment is best for this infection.

Institution
MUSC
Recruitment Contact
Christian Conley
843-792-1213
conelyc@musc.edu

A Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy and Safety of Depemokimab in Adults with Hypereosinophilic Syndrome (HES)

Date Added
May 3rd, 2022
PRO Number
Pro00119532
Researcher
Kelli Williams

List of Studies


Keywords
Blood Disorders, Rare Diseases
Summary

The purpose of this study is to see if taking depemokimab is safe and effective in treating Hypereosinophilic syndrome (HES) in adults (≥18 years) with uncontrolled HES receiving standard of care (SoC) therapy. The study will last approximately 52 weeks and is a placebo-controlled, double blind, multicentre study.

Institution
MUSC
Recruitment Contact
Natalie Naylon
843-792-5824
naylon@musc.edu

WATCHMAN FLX versus NOAC for EMbolic ProtectION in the management of patients with Non-Valvular Atrial Fibrillation (CHAMPION-AF)

Date Added
April 26th, 2022
PRO Number
Pro00116671
Researcher
Loren Morgan

List of Studies

Keywords
Cardiovascular, Heart, Vascular
Summary

This study will compare WATCHMAN FLXTM ("DEVICE") to a category of OAC called non-vitamin K antagonist oral anticoagulation, also known as NOACs ("CONTROL"). NOAC medications include Pradaxa® (dabigatran), Eliquis® (apixaban), Xarelto® (rivaroxaban), or Savaysa® (edoxaban).The study will research the safety and effectiveness of the WATCHMAN FLXTM device compared to treatment with NOAC therapy to determine if it works for patients with your type of atrial fibrillation who require treatment for potential blood clot formation (thrombus) in the LAA.

Institution
MUSC Health Columbia Medical Center
Recruitment Contact
Jacqueline Sheriod-Scott
803-255-2927
sheriods@musc.edu

Enhanced Study Recruitment for Stroke Survivors through Longitudinal Data Collection in Multiple Domains

Date Added
April 5th, 2022
PRO Number
Pro00119024
Researcher
Alyssa Chesnutt

List of Studies

Keywords
Military, Rehabilitation Studies, Stroke, Stroke Recovery
Summary

People who experience a stroke often have problems performing many different tasks during daily life. Most studies investigating stroke recovery and rehabilitation focus only on one type of task. The purpose of this research study is to measure individuals' function in many areas that can be affected by a stroke, such as their balance, mood, memory, reaching, and speech. This study will also track changes that can occur with task performance over time.

Institution
MUSC
Recruitment Contact
Alyssa Chesnutt
843-792-8171
hydar@musc.edu

Targeted spinal cord plasticity for alleviating SCI-related neuropathic pain

Date Added
April 5th, 2022
PRO Number
Pro00118771
Researcher
Aiko Thompson

List of Studies


Keywords
Central Nervous System, Nerve, Nervous System, Pain, Rehabilitation Studies, Spinal Cord
Summary

The purpose of this study is to examine the relationship between reflexes in the leg and the presence of neuropathic pain. The researchers are recruiting 30 individuals with spinal cord injury (SCI) total, 15 individuals with neuropathic pain due to SCI and 15 individuals without neuropathic pain. For this portion of the study, there are 2 visits. The first visit will examine cutaneous reflexes in the leg. During the second visit, the study team will assess sensation in the leg and administer questionnaires about pain, functioning, and quality of life.

The purpose of the second part of the study is to examine the effect of reflex training in the leg to decrease neuropathic pain. For this, the researchers are recruiting 15 individuals with neuropathic pain due to spinal cord injury to participate in the reflex training procedure. The study involves approximately 50 visits with a total study duration of about 6.5 months (3 months for baseline and training phases followed by 1 month and 3 month follow-up visits).

Institution
MUSC
Recruitment Contact
Blair Dellenbach
843-792-6313
stecb@musc.edu

Community use of wearable wrist stimulation device

Date Added
April 5th, 2022
PRO Number
Pro00118317
Researcher
Na Jin Seo

List of Studies


Keywords
Cerebral Palsey, Stroke, Stroke Recovery
Summary

Sensory stimulation has been shown to enhance rehabilitation outcomes. However, most sensory stimulation devices interfere with natural hand tasks. Thus, a new wearable stimulation device has been developed to deliver imperceptible vibration to wrist skin. This study is to evaluate the community use of the device for patients with neurologic movement disorders. Participation will include wearing the provided device and charging the device every night. The knowledge regarding community use of the device may contribute to improving the device functionality and usability for future users of the device.

Institution
MUSC
Recruitment Contact
Na Jin Seo
8437920084
seon@musc.edu

Randomized Phase II Trial of Postoperative Adjuvant Capecitabine and Temozolomide Versus Observation in High-Risk Pancreatic Neuroendocrine Tumors

Date Added
March 24th, 2022
PRO Number
Pro00119817
Researcher
William Hawkins

List of Studies

Keywords
Cancer/Gastrointestinal, Pancreas
Summary

This study is for patients who have had surgery for pancreatic neuroendocrine tumor (pNET). This study is being done to find out if giving chemotherapy after surgery is better or worse than the usual approach for patients that have had surgery for pNET.

Institution
MUSC
Recruitment Contact
HCC Clinical Trials Office
(843) 792-9321
hcc-clinical-trials@musc.edu

Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of Disease Consortium

Date Added
March 16th, 2022
PRO Number
Pro00117423
Researcher
Charlie Strange

List of Studies


Keywords
Autoimmune disease, Liver, Lung, Non-interventional, Pulmonary, Rare Diseases
Summary

The purpose of this study is to create a de-identified, public use,
repository of data of Chronic Obstructive Pulmonary Disease (COPD)
patients with by Alpha-1 antitrypsin deficiency (AATD), a rare genetic
condition that can cause COPD and emphysema.

Institution
MUSC
Recruitment Contact
Kristin Neff
843-792-1219
neffk@musc.edu

Pediatric Acute Leukemia (PedAL) Screening Trial Developing New Therapies for Relapsed Leukemias

Date Added
March 11th, 2022
PRO Number
Pro00118800
Researcher
Jacqueline Kraveka

List of Studies


Keywords
Adolescents, Cancer, Cancer/Leukemia, Pediatrics
Summary

This study is for patients that have been diagnosed with leukemia. This study is called a screening study and we are doing this study to find better ways to diagnose and treat leukemia in children, adolescents and young adults. Bone marrow, blood, and medical information about participant's cancer and treatment will be collected. Participants can expect to be on this study for 5 years.

Institution
MUSC
Recruitment Contact
HCC Clinical Trials Office
843-792-9321
hcc-clinical-trials@musc.edu



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