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.
Patients who have been diagnosted with Myasthenia Gravis having generalized muscle weakness may qualify to participate in this study.
Subjects that volunteer to participate will undergo a screening visit to assess their health. The purpose of the screening visit is to determine if the requirements to take part in this study are met. If you meet all of the requirements, you will be randomly assigned (like the flip of a coin) to either the study drug that is being researched called ARGX-113 or a placebo treatment group. You will have a one in two chance of being placed in either group. Neither you nor your study doctor will know what group you will be in. If you are in the placebo group, you will receive a substance that looks like ARGX-113 but contains no active medication. This is called a placebo. During the study, both treatment groups will be given the ARGX-113 or placebo through a vein in their arm, called an infusion. Participants will continue to take their current medicines for gMG.
The trial will include a Screening period of a maximum of 2 weeks, a first Treatment Cycle and a maximum of 2 of subsequent Treatment Cycles given on an "as needed basis". Each Treatment Cycle has 9 visits over 8 weeks consisting of a Treatment period of 4 weekly infusions and a Follow-up period of 5 weeks. There are also Inter-treatment Cycle Visits every 2 weeks between Treatment Cycles. The study will last for approximately 28 weeks, and qualified participants will need to come to MUSC at least 19 times over this period.
This is an observational study to develop a research registry to collect information from subjects with Myasthenia Gravis (MG) to evaluate the effects of the treatments they receive and to understand how their medical condition and treatment affects their daily life.