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.
The goal of the study is to characterize the features of Infantile Hemangiomas before and after treatment. Certain characteristics of the hemangioma can be seen more clearly with a closer and more resolute image of the lesion (abnormal vessels etc.). Developing a greater understanding of these characteristics? may help clinicians better predict the course of infantile hemangiomas in children.
Specific aim 1: to correlate images seen on dermoscopy with regression of the hemangioma.
Specific aim 2: to provide features that may help to predict a better response to treatment.
Alpha-1 antitrypsin (Alpha-1, AAT) deficiency is an inherited disease which results from a defect in the alpha-1 gene. Severe AAT deficiency causes emphysema predominant chronic obstructive pulmonary disease (COPD). This study is designed to test the effectiveness of an drug (Alvelestat) on lung damage caused by Alpha-1 Antitrypsin Deficiency. This is blinded study and there is a 50% chance of receiving a placebo.
Alpha-1 Antitrypsin (AAT) is a naturally occurring protein involved in the protection of lungs from inflammation. A mutation in the AAT gene (a change in the body's genetic instructions on how to make AAT) causes it to be made incorrectly and very little of it gets into the bloodstream.This results
in the lung damage known as emphysema. ARO-AAT is an investigational drug, which means that it is not approved by the Food and Drug Administration. ARO-AAT works by interrupting a step in the production of AAT. In a patient with AATD, this would stop the mutated protein from being made. This study is being carried out to see how safe and well tolerated ARO-AAT is, and to see if low, medium and high doses of the study treatment will decrease Alpha-1 Antitrypsin in the blood and in the liver compared to a placebo, or dummy injection. The Study medication is given via injection on Day 1, 29 and 133 and then every 84 days. The study includes approximately 17 visits over a period of 24 month. Compensation will be provided for study site visits. .
This is a global phase 3 open-label study designed to evaluate the efficacy and safety of ALN-TTRSC02 in adult patients (18 - 85 years of age) with hATTR amyloidosis. The estimated time on the study is approximately 3 years, including 42 days of Screening, an 18 month Treatment Period and an 18 month Treatment Extension Period.
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.
This study is designed to follow individuals who participated in "Phase 1/2 Study of Intravenous or Intrapleural Administration of a Serotype rh.10 Replication Deficient Adeno-associated Virus Gene Transfer Vector Expressing the Human Alpha-1 Antitrypsin cDNA to Individuals with Alpha-1 Antitrypsin Deficiency" for 5 years after receiving treatment.
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.
This research studies the effects of brain stimulation (transcranial magnetic stimulation, or "TMS") on balance in progressive supranuclear palsy (PSP). The purpose of this research is to look for improvements in balance when subjects are on a tilting platform after stimulating the brain with a magnetic wand held over the scalp over an area at the back of the brain called the cerebellum. Participants will receive both active and inactive stimulation during the course of the study. There is no surgery involved. There are also optional portions of the study that include functional magnetic resonance imaging (fMRIs) and speaking samples.
This is a first-in-humans gene therapy study for subjects diagnosed with severe hemophilia A. A one time infusion of the gene therapy product (BAX888) will be given with the hope of eliminating or reducing the need for prophylactic and/or on-demand use of FVIII concentrate therapy. Up to 10 subjects will be enrolled study-wide with up to two subjects enrolled at MUSC. This study will require weekly visits to the study clinic after initial infusion for the first 15 weeks followed by monthly visits for the first year. Compensation will be provided.