A new pilot study led by Justin Leach, PhD, aims to improve clinical trials for myasthenia gravis by incorporating the impact of emergency therapy. The Myasthenia Gravis Rare Disease Network (MGNet), Conquer MG, and Myasthenia Gravis Foundation of America are collaborating to support the project.
Dr. Leach, an assistant professor at the University of Alabama at Birmingham (UAB), will work with UAB colleagues Inmaculada Aban, PhD, and Gary Cutter, PhD, as well as Michael Benatar, MD, PhD, of the University of Miami.
Myasthenia gravis (MG) is a rare neuromuscular disorder caused by an autoimmune response which blocks or damages acetylcholine receptors on muscles. Most patients with MG experience generalized muscle weakness and fatigue with prolonged activity, which can improve with rest.
Clinical trials for MG typically depend on accurate measures of participants’ baseline symptoms. This allows researchers to understand how symptoms change during the trial, as well as ensure participant safety. However, some participants may need to increase medications or receive emergency therapy before the baseline measurement, which could change how they react to treatment. If the impact of emergency therapy is not considered, this can lead to biased results.
The new study, “Novel approaches for incorporating rescue therapy into the statistical analysis of clinical trials in myasthenia gravis,” aims to fill this gap by developing the first mathematical models to account for emergency therapies in clinical trials for MG. These models will help researchers understand how emergency treatments affect the outcomes of clinical trials.
Findings from the study will not only improve the design of clinical trials for MG, but also for other diseases with fluctuating symptoms.
The Myasthenia Gravis Rare Disease Network (MGNet) is part of the Rare Diseases Clinical Research Network (RDCRN), which is funded by the National Institutes of Health (NIH) and led by the National Center for Advancing Translational Sciences (NCATS) through its Division of Rare Diseases Research Innovation (DRDRI). MGNet is funded under grant number U54NS115054 as a collaboration between NCATS and the National Institute of Neurological Disorders and Stroke (NINDS).
