A Phase 2, Randomized, Placebo-controlled, Double-blind, Open-label Extension Multicenter Study to Evaluate the Efficacy and Safety of KD025 in Subjects with Diffuse Cutaneous Systemic Sclerosis Save

Date Added
October 22nd, 2019
PRO Number
Pro00089320
Researcher
Richard Silver

List of Studies


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

KD025 is an investigational medication undergoing testing to determine if it may be effective in the treatment of diffuse systemic sclerosis (skin thickening on more than just the hands). KD025 has previously been tested in graft-versus-host disease, idiopathic pulmonary fibrosis, and psoriasis. It has shown preliminary effectiveness and safety in the treatment of these conditions. This study will randomly assign subjects to one of three treatment groups, 20mg of KD025 twice per day, 20mg of KD025 once per day, and placebo. 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 patient reported outcomes. The study treatment period will last 1 year. The drug may help mitigate symptoms of systemic sclerosis and thus may be helpful with the disease in study. The population to be enrolled in this study will involve patients diagnosed with systemic sclerosis, diffuse subset, 18 years of age or older.

Institution
MUSC
Recruitment Contact
Trevor Faith
843-792-8997
faitht@musc.edu

The Implementation of Decision Aid for Lupus Patients in Practice Settings for Shared Decision-Making (SDM): IDEAL Study Save

Date Added
July 23rd, 2019
PRO Number
Pro00086269
Researcher
Diane Kamen

List of Studies


Profiles_link
Keywords
Autoimmune disease, Lupus
Summary

The purpose of this research study is to evaluate the value of educational information given on an iPad about the risks and benefits of lupus medications. The information is intended to encourage conversations between the patient and doctor about lupus treatments. This research will test the feasibility and effectiveness of using the iPad in lupus clinics nationwide. Participants will be given information about lupus treatments on an iPad during the clinic visit before seeing their doctor and will be interviewed about the feasibility of using the iPad during a regular clinic visit.

Institution
MUSC
Recruitment Contact
Marcie Pregulman
843-792-5290
lupusresearch@musc.edu

A Phase 2, Randomized, Double-blind, Placebo-controlled Study of Cemdisiran in Patients with IGA Nephropathy Save

Date Added
July 23rd, 2019
PRO Number
Pro00085871
Researcher
Anand Achanti

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Profiles_link
Keywords
Autoimmune disease, Inflammation, Kidney
Summary

The study is using cemdisiran compared to placebo injections to determine the safety and efficay of cemdisiran in treating IgA Nephropathy. The study includes a screening period (up to 90 days), 8 month treating period, and 52 week Open Label extension period. Injections occur monthly during the treatment period. Patients will be randomized 2:1 to the cemdisiran or placebo arms.

Institution
MUSC
Recruitment Contact
Kellean Van Cleef
8437928166
vancleef@musc.edu

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Ravulizumab in Complement-Inhibitor-Na Save

Date Added
July 9th, 2019
PRO Number
Pro00086756
Researcher
Katherine Ruzhansky

List of Studies

Silhouette
Keywords
Autoimmune disease
Summary

Generalized Myasthenia gravis (gMG) is a rare neuromuscular inflamation disorder that involve all voluntary muscle groups. This study is to evaluate the safety and efficacy of ravulizumab for the treatment of patients with generalized myasthenia gravis (gMG).

There will be 3 periods in this study; a 4 week Screening Period, a 26-week Randomized-Controlled Period consisting of 12 study visits, and an Open-Label Extension Period consisting of 18 visits that will continue for up to 2 years. Meningococcal vaccination is required for all patients prior to intravenous administration of study drug. The overall study duration for an individual participant is estimated to take up to 2.5 years.

Institution
MUSC
Recruitment Contact
Katrina Madden
843-792-9186
maddenka@musc.edu

An Open-Label, Multicenter Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Eculizumab in Pediatric Patients with Refractory Generalized Myasthenia Gravis Save

Date Added
June 25th, 2019
PRO Number
Pro00086363
Researcher
Katherine Ruzhansky

List of Studies

Silhouette
Keywords
Autoimmune disease
Summary

The purpose of this study is to evaluate how safe and effective intravenous eculizumab is in pediatric patients 6 to less than 18 years old with generalized Myasthenia gravis (gMG). Eculizumab is already approved for use in adult patients with gMG in the US, Europe and Japan, but currently has not been approved for use in pediatric patients.

The study's duration is approximately 4.7 years with 4 treatment periods consisting of a Screening Period (2 to 4 weeks), Primary Evaluation Treatment Period (26 weeks), Extension Period (up to an additional 208 weeks), and Follow-up Period (8 weeks). All patients who complete Week 26 of Study ECU-MG-303 will continue receiving eculizumab in the Extension Period of this study for up to an additional 208 weeks.

Institution
MUSC
Recruitment Contact
Katrina Madden
843-792-9186
maddenka@musc.edu

Identifying functional regulatory marks underlying monocyte dysfunction in scleroderma Save

Date Added
February 19th, 2019
PRO Number
Pro00085936
Researcher
Paula Ramos

List of Studies


Profiles_link
Keywords
Autoimmune disease, Ethnicity and Disease, Genetics, Healthy Volunteer Studies, Immune System, Inflammation, Minorities, Scleroderma, Women's Health
Summary

Scleroderma (systemic sclerosis) is a chronic autoimmune disease, characterized by dysregulation of immune cells in the blood and subsequent fibrosis and vascular dysfunction, associated with significant mortality and morbidity, disproportionately affecting women and African Americans, and without satisfactory treatments. Monocytes, a type of blood immune cells, are critically involved, but the mechanisms responsible for their deregulation in scleroderma remain largely unknown. The goal of this project is to understand how the regulation of monocytes differs between scleroderma and healthy individuals. Volunteers will be asked to provide a blood sample, for which modest compensation will be provided. This is not a drug study.

Institution
MUSC
Recruitment Contact
Elizabeth Szwast
843-792-4457
hinsone@musc.edu

A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 3 Trial to Evaluate Efficacy and Safety of Lenabasum in Dermatomyositis Save

Date Added
February 12th, 2019
PRO Number
Pro00085567
Researcher
Katherine Ruzhansky

List of Studies

Silhouette
Keywords
Autoimmune disease, Inflammation, Rare Diseases
Summary

The primary objective of this study is to evaluate the efficacy of lenabasum compared to placebo in participants with dermatomyositis (DM), and to evaluate the safety and tolerability of lenabasum in participants with Dermatomyositis (DM).

Autoimmune diseases such as DN result from the immune system becoming over-active and attacking parts of the body. This over-active immune response also causes chronic inflammation. The growth of scar tissue in muscle, skin and internal organs with chronic inflammation from DM makes them not work as well as they should. Lenabasum may help the body stop the chronic inflammation and stop scarring fro getting worse without preventing the normal response of the immune system.

Lenabasum is an investigational drug that will be taken orally twice a day. It will take about one year to complete this research study. During this time, participants will make a total of 12 study visits.

Institution
MUSC
Recruitment Contact
Katrina Madden
843-792-9186
Maddenka@musc.edu

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

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

List of Studies


Profiles_link
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
Trevor Faith
843-792-8997
faitht@musc.edu

A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy and Safety of BMS-986165 in Subjects with Systemic Lupus Erythematosus Save

Date Added
October 24th, 2018
PRO Number
Pro00077684
Researcher
Eric Zollars

List of Studies


Profiles_link
Keywords
Autoimmune disease, Lupus, Skin
Summary

The purpose of this research study is to measure how well and how safe BMS-986165 is in treating patients with Systemic Lupus Erythematosus (SLE) and to determine the optimal dose level.

Lupus is an autoimmune disease, which means that your immune system not only attacks bacteria and viruses but also attacks your healthy cells and organs, affecting many parts of the body. Lupus can cause fever, joint pain, rash (redness of the skin), sensitivity of the skin to sunlight, as well as other symptoms, and may lead to inflammation and organ damage.

Current treatments for Lupus are mainly drugs that suppress the immune system such as cortisone-like drugs (such as prednisone) and cyclophosphamide (a potent drug sometimes used in treating certain types of cancer), and drugs commonly used to treat or prevent malaria (called antimalarials) such as hydroxychloroquine. Many of these treatments may have serious side effects if used for a long time.

Therefore, there is a need for new and effective treatments for Lupus.

Institution
MUSC
Recruitment Contact
Angela Robinson
843-792-6043
robia@musc.edu

Odor Disturbances: Clinical Care Registry Save

Date Added
September 26th, 2018
PRO Number
Pro00080333
Researcher
Thomas Uhde

List of Studies


Profiles_link
Keywords
ADD/ADHD, Adolescents, Aging, Allergy, Alzheimers, Anxiety, Asthma, Autism, Autoimmune disease, Central Nervous System, Chronic Fatigue, Depression, Environmental Factors, Fibromyalgia, Inflammation, Memory Loss, Nervous System, Parkinsons, Psychiatry
Summary

Candidates for this study may or may not report disturbances in odor perception as their primary reason for seeking treatment at MUSC. This study is designed to collect long term, observational data from patients who are being treated with routine clinical care in health clinics at MUSC. Data from clinical questionnaires will be de-identified and stored in a database.

Institution
MUSC
Recruitment Contact
Richard Simmons
843-792-7439
simmr@musc.edu

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