A Randomized, Double-blind, Placebo-controlled Phase 4 Clinical Trial to Evaluate the Long-term Safety and Efficacy of Avacopan in Subjects With Antineutrophil Cytoplasmic Antibody (ANCA)-associated Vasculitis

Date Added
April 10th, 2024
PRO Number
Pro00136129
Researcher
Kathleen Maksimowicz-McKinnon

List of Studies

Keywords
Autoimmune disease, Drug Studies, Inflammation, Rare Diseases, Stage IV, Vasculitis
Summary

The purpose of this study is to learn more about long-term safety (good or bad effects) of avacopan and its efficacy (how well it is working) in treatment of antineutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis.

Avacopan is currently approved as an adjunctive (another treatment used along with primary treatment) treatment in adult people with severe active ANCA-associated vasculitis in the US and is also approved in the EU, Canada, Japan, and other countries.

In this study, avacopan will be compared with a placebo (a drug that looks likes the study medication but does not contain any medication). This is a randomized study, meaning that you will be assigned by chance (like flipping a coin) into a treatment group. You will have an equal chance of being placed in either of the 3 following groups: treatment with avacopan for 5 years in group A, or treatment with avacopan for 1 year followed by placebo for 4 years in group B, or treatment with placebo for 5 years in group C. The study is also a double-blinded study, meaning you and your study doctor will not know what you are receiving, the avacopan or placebo.

The study is sponsored by Amgen, Inc. Participation in the study will require 27 visits to the MUSC main campus over approximately 63 months, and visits will include the following procedures: blood draw, urine collection, physician-led assessments of your disease (for example physical exam and medical history review), and health questionnaires. You will also be provided with a paper diary to record any missed doses/overdoses of the study drug.

Compensation is available for participation.

Institution
MUSC
Recruitment Contact
Kathleen Maksimowicz-McKinnon
843-792-0066
maksimow@musc.edu

A Phase 2, Double-blind, Randomized, Placebo-controlled Study to Investigate the Efficacy and Safety of NS-229 in the Treatment of Eosinophilic Granulomatosis With Polyangiitis

Date Added
February 23rd, 2024
PRO Number
Pro00133321
Researcher
Kathleen Maksimowicz-McKinnon

List of Studies

Keywords
Autoimmune disease, Drug Studies, Inflammation, Rare Diseases, Vasculitis
Summary

The purpose of this study is to test whether a drug called NS-229 (the study drug) is a potential treatment for patients with Eosinophilic Granulomatosis With Polyangiitis (EGPA).

NS-229 is an investigational drug that is provided in an oral pill form. 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, NS-229 will be compared with a placebo (dummy drug), having no active drug in it. 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. The study is also double-blinded study, meaning you and your study doctor will not know what you are receiving, the NS-229 or placebo.

The study is sponsored by a NS Pharma, Inc. Participation in the study will require 12 visits to the MUSC main campus over approximately 8 months. Visits are much like the your standard of care and include the following procedures: blood draw, urine collection, physician-led assessments of your disease (for example physical exam and medical history review), tests to assess your lung function and health (Pulmonary Function Test (PFT) and spirometry, health questionnaires. You will also be asked to complete a daily diary regarding your medication use and vasculitis symptoms.

Compensation is available for participation

Institution
MUSC
Recruitment Contact
Kathleen Maksimowicz-McKinnon
843-792-0066
maksimow@musc.edu

Mechanisms of New-Onset Autoimmunity/Longitudinal Immune Systems Analysis (MONA-LISA)

Date Added
June 14th, 2023
PRO Number
Pro00127526
Researcher
Diane Kamen

List of Studies


Keywords
Autoimmune disease, Lupus, Non-interventional
Summary

This is an observational non-medication study. The purpose of this study is to understand the immune system in people who are at risk for developing systemic lupus erythematosus ("lupus" or "SLE"). The investigators hope to develop better ways to predict who will get lupus and possibly come up with ideas for new treatments that can prevent or treat the disease.

Participants will be asked to complete 4 annual study visits and monthly telephone contacts with the study team over the course of 3 years. Visits will include a physical exam, collection of blood and urine, and the completion of some surveys/questionnaires about your health and wellbeing. The monthly phone calls will be a brief contact to check on any changes in your health and should take no longer than 15 minutes to complete.

Compensation is available for participation.

Institution
MUSC
Recruitment Contact
Emma Hatch
843-792-4296
hatchem@musc.edu

A Phase 3, Multi-center, Randomized, Quadruple-blind, Placebo-controlled Study to Assess the Efficacy and Safety of Batoclimab as Induction and Maintenance Therapy in Adult Participants with Generalized Myasthenia Gravis (gMG)

Date Added
May 23rd, 2023
PRO Number
Pro00128352
Researcher
Katherine Ruzhansky

List of Studies


Keywords
Autoimmune disease
Summary

The FLEX study is a randomized, blinded, and placebo-controlled study assessing the efficacy and safety of an investigational drug for adults diagnosed with Generalized Myasthenia Gravis (gGM). The injectable investigational drug is being evaluated over a 12-week induction period and a 12-week maintenance period. Based on completion of these periods and a participant's response to the investigational injection, participants may qualify to continue in a 52-week extension study of the investigational drug. If you choose to enter this study, you will participate in the study for up to approximately 84 weeks (1 ½ years) and you will be asked to attend up to 34 study visits; visits can last from 1 to 4 hours.

It is unknown at this time whether the study treatment will improve participant's health. The potential risks of this study include the possibility of infections, low albumin levels, and increases in blood cholesterol.

Institution
MUSC
Recruitment Contact
Della MacNicholas
843-792-8385
macnichd@musc.edu

A Phase 3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Deucravacitinib in Participants with Active Systemic Lupus Erythematosus (SLE) (POETYK SLE-2)

Date Added
April 11th, 2023
PRO Number
Pro00124902
Researcher
Kathleen Maksimowicz-McKinnon

List of Studies

Keywords
Autoimmune disease, Drug Studies, Lupus
Summary

The purpose of this research study is to determine how well and safe deucravacitinib, an investigational/non-approved United States Food and Drug Administration (US FDA) medication, is in treating Systemic Lupus Erythematosus (SLE, lupus).

Participation in the study will involve being assigned deucravacitinib or a placebo (a drug that looks likes the study medication but does not contain any medication) and completing a series of in person clinic visits much like those completed as part of standard of care. Blood and urine samples will be collected for both routine health and safety tests as well as research/experimental testing.

The study will require 16 visits over a period of 14 months. There is an optional extension to the study possible, should you wish to continue and the study doctor feels that you remain a good candidate for participation. Study medication is provided and compensation is available for participation.

Institution
MUSC
Recruitment Contact
Emma Hatch
843-792-4296
hatchem@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

Daily Topical Rapamycin Therapy for the Treatment of Vitiligo

Date Added
March 1st, 2022
PRO Number
Pro00115924
Researcher
Lara Wine Lee

List of Studies


Keywords
Adolescents, Autoimmune disease, Pediatrics, Skin
Summary

In current Dermatology practice, options for vitiligo remain limited. The purpose of this study is to determine if once daily dosed topical rapamycin is effective for the treatment of patients with vitiligo. Participants will apply either 0.1% topical rapamcyin or 0.001% topical rapamycin for six months to a lesion on one side of the body, and topical placebo to a corresponding lesion on the opposite side of the body. The study also aims to evaluate patient satisfaction and identify any adverse effects on these dosing regimens.

Institution
MUSC
Recruitment Contact
Chelsea Shope
8437549577
shopec@musc.edu

A Double Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of MK-7240/PRA023 in Subjects with Systemic Sclerosis Associated with Interstitial Lung Disease (SSc-ILD) - The ATHENA SSc-ILD Study

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/MK7240 (the study drug) is a good treatment for patients with Systemic Sclerosis associated with Interstitial Lung Disease (SSc-ILD). The study drug PRA023/7240 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/MK7240 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/MK7240 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., a subsidiary of Merck & Co., 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. If you complete the initial blinded treatment period of 50 weeks, the study doctor will discuss whether you are eligible to enter the open label period of the study, meaning no placebo. If you are eligible and agree, you will receive 500 mg of study drug once every 4 weeks for an additional 52 weeks. Compensation is available for participation.

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

Social factors, epigenomics, and lupus in African American women (SELA)

Date Added
August 17th, 2021
PRO Number
Pro00112945
Researcher
Diane Kamen

List of Studies


Keywords
Autoimmune disease, Environmental Factors, Ethnicity and Disease, Genetics, Healthy Volunteer Studies, Lupus, Minorities, Women's Health
Summary

The goal of this study is to understand how positive and negative social experiences affect epigenetic marks and gene function, and thereby influence lupus in African American women. This study requires one study visit to answer a few questionnaires and donate a blood sample. We are looking for volunteers with and without lupus to participate. Compensation is available.

Institution
MUSC
Recruitment Contact
Lusawasra King
843-792-7645
kinglu@musc.edu

Fibrotic Mechanisms in Systemic Sclerosis

Date Added
August 17th, 2021
PRO Number
Pro00111928
Researcher
Deanna Baker Frost

List of Studies


Keywords
Autoimmune disease, Skin
Summary

We plan to obtain skin biopsies from patients with systemic sclerosis and healthy donors to grow cells from biopsies. With those cells, we will use them in experiments to see why the cells from systemic sclerosis patients have more fibrosis compared to patients without systemic sclerosis.

Institution
MUSC
Recruitment Contact
Deanna Baker Frost
8437928461
bakerde@musc.edu



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