Researchers are looking for people with myasthenia gravis (MG) to participate in a phase 2 clinical trial, sponsored by Ra Pharmaceuticals, to test the experimental drug RA101495 in people with generalized MG who are positive for acetylcholine receptor autoantibodies.
Administered subcutaneously (an injection under the skin), RA101495 is designed to prevent the body’s attack on the space across which nerve fibers transmit signals to muscle fibers, called the neuromuscular junction (NMJ).
RA101495 acts by targeting and blocking a part of the immune system called the complement system, which is responsible for helping antibodies clear damaged cells and potentially toxic microbes that could cause infections. In patients with acetylcholine receptor antibody positive generalized MG, the body’s own immune system turns on itself to produce antibodies against the acetylcholine receptor (a receptor located on muscle cells at the NMJ) activating the complement system.
Goals of the trial are to assess the safety of RA101495 and determine whether the treatment is able to reduce muscle weakness in people with generalized MG.
Trial length is approximately three months, during which participants will visit with study investigators seven times in person. At in-person visits, treatment effects will be measured across two MG-specific assessment scales: the Quantitative Myasthenia Gravis (QMG) and the Myasthenia Gravis – Activities of Daily Living profile (MG-ADL), along with other clinical tests.
In order to be eligible to participate, individuals must have a diagnosis of generalized MG, test positive for acetylcholine receptor (AChR) autoantibodies, and meet additional criteria.
The trial is taking place at 21 trial sites across the United States, and support for travel costs may be available.
To learn more about this trial, visit clinicaltrials.gov and enter NCT03315130 into the “Other Terms” search box.