A Double Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of PRA023 in Subjects with Systemic Sclerosis Associated with Interstitial Lung Disease (SSc-ILD)

Date Added
February 22nd, 2022
PRO Number
Pro00117883
Researcher
Richard Silver

List of Studies


Keywords
Autoimmune disease, Drug Studies, Rare Diseases, Scleroderma, Skin
Summary

The purpose of this study is to test whether a drug called PRA023 (the study drug) is a good treatment for patients with Systemic Sclerosis associated with Interstitial Lung Disease (SSc-ILD). The study drug PRA023 is an investigational drug that is given by infusion every 4 weeks. An investigational drug is not approved by The US Food and Drug Administration. It can only be used in a research study like this one. In this study, PRA023 will be compared with a placebo (dummy drug). The placebo will be a saline solution that does not have any study drug in it. The comparison with the placebo helps to determine whether the effects seen in your body is because of the PRA023 or not. This is a randomized study meaning that you will be assigned by chance (like flipping a coin) to receive either the study drug or placebo. This will be done with the help of a computer-based program and you will have 50% chance of receiving either the study drug or placebo. The study is double-blinded study and 50 weeks long, meaning you and your study doctor will not know what you are receiving, the study drug or placebo.

The study is sponsored by Prometheus Biosciences, Inc. The study is being done at approximately 25 sites across the United States. The main portion of the study will require 15 visits to the MUSC main campus and will have the following procedures completed over the course of your participation: blood draw, physician-led assessments of your disease (for example physical exam and skin thickness testing), tests to assess your pulmonary function and health (Pulmonary Function Test (PFT) and High Resolution Computed Tomography (HRCT)), electrocardiogram, as well as asked to complete surveys. Compensation is available for participation.

Institution
MUSC
Recruitment Contact
Brittany Frasier
843-792-8613
frasibri@musc.edu

A Randomized, Double-blind, Placebo-controlled, Repeat-dose, Multicenter Trial to Evaluate the Efficacy, Safety, Tolerability and Pharmacokinetics of HZN-825 in Patients with Diffuse Cutaneous Systemic Sclerosis

Date Added
September 14th, 2021
PRO Number
Pro00110634
Researcher
Richard Silver

List of Studies


Keywords
Autoimmune disease, Drug Studies, Rare Diseases, Scleroderma, Skin
Summary

The purpose of this study is to obtain information on the safety, efficacy, and tolerability of an oral drug called HZN-825 for the treatment of diffuse cutaneous systemic sclerosis (dcSSc) in participants 18-75. The study drug is taken as a tablet twice a day for 52 weeks. This study has 12 planned visits over a 60 week period. The study will measure the improvement, stabilization or worsening of your symptoms, such as changes in your fatigue and pain levels, lung function, skin thickness and other participant reported outcomes.

Institution
MUSC
Recruitment Contact
Brittany Frasier
843-792-8613
frasibri@musc.edu

National Institute of Allergy and Infectious Diseases (NIAID) "BOOSTER EFFECTS WITH AUTOIMMUNE TREATMENTS IN PATIENTS WITH POOR RESPONSE TO INITIAL COVID-19 VACCINE"

Date Added
September 7th, 2021
PRO Number
Pro00113395
Researcher
Diane Kamen

List of Studies


Keywords
Autoimmune disease, Coronavirus, Lupus, Rheumatoid, Scleroderma, Vaccine
Summary

This research is being done to see if an additional shot of COVID-19 vaccine, called a "booster", will help people with autoimmune disease who are taking medications that weaken their immune system develop a better response to the vaccine. The study is also being done to see if temporarily stopping the medication you take for your autoimmune disease will help your immune system have a positive response to the vaccine booster.

This study will use the COVID-19 vaccines that are authorized under an Emergency Use Authorization by the United States Food and Drug Administration (FDA) for initial vaccination, but they are not currently authorized or approved to be used as booster vaccinations – so their use in this study is experimental. This includes the Pfizer, Moderna, and Johnson & Johnson COVID-19 vaccines.

This study is enrolling people over the age of 18 who have a diagnosis of lupus, scleroderma, or rheumatoid arthritis. You will be asked to come to the clinic for up to 8 research visits over 13 months. There might be no direct medical benefit to you for being in this study. The information learned from this study may someday benefit people with autoimmune disorders. Compensation is available for participation.

Institution
MUSC
Recruitment Contact
Stephanie Bray
843-792-8997
brays@musc.edu

Right Ventricular Reserve Measures with Cardiac MRI

Date Added
October 16th, 2019
PRO Number
Pro00085562
Researcher
Ryan Tedford

List of Studies


Keywords
Cardiovascular, Heart, Sarcoidosis, Scleroderma, Shortness of Breath
Summary

This research study aims to determine a less invasive way to assess heart function by taking measurements of the heart while subjects are performing an exercise cardiac MRI. Subjects will undergo two exercise phases and MRI measurements will be taken after each exercise phase. These measurements will be compared to available clinical data (including demographic, hemodynamic, radiologic, and functional) and future outcome data.

Institution
MUSC
Recruitment Contact
Melissa Lamicq
843-876-5783
lamicq@musc.edu

Collaborative, National Quality and Efficacy Registry for Tracking Disease Progression in Systemic Sclerosis (Scleroderma) Patients

Date Added
November 20th, 2018
PRO Number
Pro00080285
Researcher
Faye Hant

List of Studies


Keywords
Autoimmune disease, Non-interventional, Rare Diseases, Scleroderma, Skin
Summary

The goal of this study is to develop an early systemic sclerosis (SSc) registry in the United States (US). A registry is a group of patients that are observed over time. This is a non-interventional study, meaning that they are no study specific medications to take or procedures to undergo. The specific aims include ongoing assessment of the natural history of early SSc patients by capturing and analyzing clinical data, patient reported outcomes, and laboratory data. This is a multi-center study with sites spread across the U.S. This study is funded by the Scleroderma Research Foundation.

Institution
MUSC
Recruitment Contact
Brittany Frasier
843-792-8613
frasibri@musc.edu

Human Samples Biorepository

Date Added
August 21st, 2018
PRO Number
Pro00072807
Researcher
Federica del Monte

List of Studies


Keywords
Aging, Cardiovascular, Coronary Artery Disease, Genetics, Heart, Military, Sarcoidosis, Scleroderma, Transplant, Vascular
Summary

The purpose of the study is to generate a bio bank of specimens for research. We will tissue that would otherwise be discarded from clinical or surgical procedure and information from medical records. We will also collect discarded blood, urines and sputum. Collecting samples will help to better understanding the mechanisms of cardiovascular diseases, identify biomarkers for early diagnosis and to predict safety and efficacy of new therapies.

Institution
MUSC
Recruitment Contact
Federica del Monte
843-792-8397
delmonte@musc.edu

Cardiac biopsies in pulmonary hypertension

Date Added
August 21st, 2018
PRO Number
Pro00077070
Researcher
Ryan Tedford

List of Studies


Keywords
Pulmonary Hypertension, Scleroderma
Summary

Patients with systemic sclerosis (SSc) related pulmonary arterial hypertension (SScPAH) have a worse prognosis than those with idiopathic PAH. We have recently discovered that heart cells in SScPAH do not contract or squeeze as well as in other forms of pulmonary hypertension. However, the mechanism leading to this dysfunction is not understood. To better study this and in hopes of developing a future therapy, we plan to collect tissue samples via a heart biopsy at the time of a clinically indicated heart catheterization.

Institution
MUSC
Recruitment Contact
Brandon Sykes
843-792-1105
sykesb@musc.edu

Evaluation of Brentuximab Vedotin for Diffuse Cutaneous Systemic Sclerosis: A Phase 1/2 Multicenter Randomized, Double Blinded, Safety Study

Date Added
October 17th, 2017
PRO Number
Pro00070403
Researcher
Richard Silver

List of Studies


Keywords
Autoimmune disease, Drug Studies, Scleroderma, Skin
Summary

This study is being conducted in order to test the safety of Brentuximab Vedotin in subjects with Diffuse Cutaneous Systemic Sclerosis. The study involves a Screening visit, a Baseline visit and 12 study visits occurring every 3 to 4 weeks, with enrollment lasting approximately 52 weeks, during which enrolled subjects will receive either the study compound or placebo.

Institution
MUSC
Recruitment Contact
Brittany Frasier
843-792-8613
frasibri@musc.edu

Observational Study for Pediatric Rheumatic Diseases: The CARRA Registry

Date Added
November 3rd, 2015
PRO Number
Pro00048606
Researcher
Natasha Ruth

List of Studies


Keywords
Arthritis, Autoimmune disease, Fibromyalgia, Lupus, Pediatrics, Rheumatoid, Sarcoidosis, Scleroderma
Summary

The purpose of this study is to create and maintain a registry, which is a database (a searchable collection of information) about children, adolescents and young adults with pediatric onset of rheumatic diseases. This data may help in the evaluation of the safety and benefit of medications that are prescribed to patients who have rheumatic diseases.

Institution
MUSC
Recruitment Contact
Shameeka Bowman
843-792-8317
bowm@musc.edu

The Core Center for Clinical Research, Improving Minority Health in Rheumatic Diseases

Date Added
January 15th, 2013
PRO Number
Pro00021985
Researcher
Gary Gilkeson

List of Studies


Keywords
Autoimmune disease, Environmental Factors, Ethnicity and Disease, Genetics, Healthy Volunteer Studies, Lupus, Minorities, Rare Diseases, Scleroderma
Summary

Systemic lupus erythematosus (lupus; SLE) and Systemic Sclerosis (scleroderma; SSc) are relatively rare rheumatic diseases that disproportionately impact the African American community, and particularly African American women. The causes of lupus and scleroderma are unknown, but thought to include both genetic and environmental factors. We are enrolling lupus and scleroderma patients, and healthy control subjects. This is not a drug study. The purpose of this study is to better understand the factors that predispose people to develop lupus and scleroderma. Information about medical, social and family history, medications, physical exam findings, and laboratory tests will be collected for analysis. This study will involve approximately 1360 volunteers.

Institution
MUSC
Recruitment Contact
Jonathan Flume
843-792-3926
jof63@musc.edu



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