AGH chosen as test site for Innovative Myasthenia Gravis medication


From AGH – The Myasthenia Gravis Association of Western Pennsylvania at Allegheny General Hospital has been selected to participate in a clinical trial of a new investigational medication believed to have the potential to treat myasthenia gravis, a chronic, autoimmune neuromuscular disease that can be controlled but not cured.

The trial investigates the safety and effectiveness of CK-2017357, an investigational new drug which earlier clinical trials suggest may increase muscle strength by a different mechanism than traditional medications used to treat myasthenia gravis, said neurologist George A. Small, MD, Medical Director of the AGH Myasthenia Gravis Center.

“This is a novel substance that increases muscle strength, but not by affecting the patient’s immune system as other drugs do,” Dr. Small said. “Most therapies for myasthenia gravis  depress the immune system’s functioning, and one of the most common therapies, prednisone, has numerous side effects.

The AGH Myasthenia Gravis Center is one of about 15 centers nationwide selected to participate in this Phase II clinical trial. The trial is funded by the National Institute of Neurological Disorders and Stroke and Cytokinetics, Inc., the California company that discovered and is developing CK-2017357.

As many as 36 patients may enroll in the trial, and will receive single doses of a placebo, 250 mg and 500 mg of CK-2017357 in random order (given 1 week apart) during the course of the trial.

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