Subjects ages 18–75 with generalized myasthenia gravis (MG) will be screened up to 42 days prior to enrollment into the clinical study. Note: If subjects are currently on a standard of care therapy, they will remain on their current standard of care therapy during the course of the clinical study at the discretion of the Investigator.
Subjects who meet all inclusion and no exclusion criteria after completing the Screening Visit will be enrolled in the clinical study.
Subjects will be randomized on Day 1 into the current cohort in a 2:1 ratio to receive two separate administrations of intravenous CNP-106 or Placebo (0.9% Sodium Chloride Injection USP) on Day 1 and Day 8. Investigational Product (IP) will be administered by IV infusion over approximately 3-4 hours using a graduated rate of infusion. Subjects will undergo medical observation in the clinic for acute AEs for 4 hours following infusion on Day 1 and Day 8.

The goal of this study is to determine if an investigational product (a substance not approved by the US Food and Drug Administration) made from mesenchymal stromal cells (MSC) and taken as a single subcutaneous injection, is safe and well tolerated in individuals with active lupus while also receiving standard of care treatment for their lupus.

The MSCs used in this trial are cells that were obtained from the umbilical cords of healthy donors having an elective Caesarean section and who have been screened to be sure that they are free of any infectious diseases.

Participation in the study will take about 9 visits and 2 telephone calls over a period of 7 months. With participation, compensation is available.

At study visits you will have blood and urine labs collected, physical exam/lupus specific assessments, and be asked to complete a questionnaire. These procedures are much like those completed at your routine standard of care visits with your rheumatologist.

The risks to study participation are that only a few hundred people worldwide have been treated with MSCs. So far, there have been no serious side effects when MSCs have been used. Potential risks could however include an allergic reaction to the cells, development of infection, or MSCs changing into cancer cells.

Prior to any use of the MSC injection, participants will have a thorough review of the study and given an opportunity to consider their options and have all questions/concerns answered. A screening visit will be completed to ensure that participation is appropriate - participant safety is a priority of the study.

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.

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

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.

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.

The purpose of this study is to determine if the investigational (not FDA approved) drug VIB4920 can be an effective treatment for patients with active Lupus Nephritis (LN).

This study will compare treatment with MMF (mycophenolate mofetil), Prednisone, plus the study medication VIB4920 to treatment with MMF and Prednisone alone.

Participants will have up to 15 scheduled visits that will involve routine monitoring of disease activity, up to two kidney biopsies, and blood and urine specimen collections. Participation in this study will last approximately 1 year and 2 months. Compensation is available for participation.

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.

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.

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.