TReatment for ImmUne Mediated PathopHysiology (TRIUMPH) is a multi-center, three-arm clinical trial of immunosuppressive therapy for children with acute liver failure. This is a double-blind study, so the study doctors and participants will not know to which arm the participants are assigned. The study will determine if corticosteroids or equine anti-…
The purpose of this study is to examine the causes and pathology of alopecia areata and other hair and skin disorders. Skin biopsies and blood and/or clinical information collected in the study will be used for research aiming at developing new options for the treatment of alopecia areata and other skin and hair disorders.
Genetic studies help us to understand why some people develop certain diseases while others don't and also provide important clues about how best to treat the disease.
We are conducting a research study on how psoriasis might be caused or worsened by high levels of the blood sugar-lowering hormone insulin. We are seeking people with plaque psoriasis, ages 18-65, who are overweight or obese with a body mass index of 25-35. Participants will have blood drawn and small skin biopsies taken from psoriasis plaques and nearby…
The purpose of this research study is to determine if an investigational drug, VS-01, will help in the treatment of acute-on-chronic liver failure (ACLF) compared to supportive care (standard of therapy). ACLF is a potentially life-threatening condition characterized by a group of signs that occur together (syndrome) in patients with a late stage of…
The purpose of this study is to evaluate the efficacy and safety of ION-682884 given for 24 months in patients with hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN) also known as Familial Amyloid Polyneuropathy (FAP), and determine if it can help people with mild or moderate polyneuropathy. To be in this study you must have FAP and be…
HELIOS-A is a global Phase 3 randomized, open-label study designed to evaluate the safety and efficacy of ALN-TTRSC02 in adult patients with hATTR amyloidosis experiencing neurologic symptoms brought on by the disease. The study will also evaluate any changes in quality of life experienced by study participants. ALN-TTRSC02 utilizes the mechanism of RNA…