Hackensack Meridian Health Hackensack University Medical Center announced that an international phase 3 study of a novel treatment for Charcot-Marie-Tooth (CMT) hereditary neuropathy, yielded positive results. The United States clinical trial was led by Florian P. Thomas, M.D., Ph.D, chair of the Neuroscience Institute and the Department of Neurology, and director of the Hereditary Neuropathy Foundation Center of Excellence at Hackensack University Medical Center,
CMT disease is a chronic, often severe and debilitating inherited condition affecting some 125,000 people in the United States, of which approximately 50 percent have a particular subtype called Charcot-Marie-Tooth type 1A disease (CMT1A). It causes progressive weakness and impairs sensation resulting, among many other symptoms, in weakness, joint deformities and difficulty walking.
“While CMT is not life-threatening in most cases, it does lead to significant disability and comorbities,” explains Dr. Thomas. “We are excited at the prospect of taking our findings to the Food and Drug Administration and applying for approval of this important new treatment option for our patients at the Hereditary Neuropathy Center.”
For this trial Pharnext, a pharmaceutical company based in France, had developed a novel therapy, PXT3003, designed for patients with mild to moderate CMT1A. The study assessed the efficacy and safety of PXT3003 compared to placebo in over 300 participants in Europe, the USA and Canada.
“While in our institution, child and adult neurologists, geneticists, genetic counselors, rehab specialists and counseling psychologists work together to help patients with safe ambulation, hand function, disease adjustment and family planning,” adds Dr. Thomas. “Until now we had no options to slow disease progression. Now we have reasons for optimism that the FDA will approve the first treatment designed to directly affect the disease process.”
In 2016, Hackensack University Medical Center partnered with the Hereditary Neuropathy Foundation (HNF) to address CMT hereditary neuropathies, serving as a hub for clinical care, community engagement, research, training of health care professionals and education for the CMT community.
“Hackensack University Medical Center is one of HNF’s leading Centers of Excellence supporting patients through their interprofessional approach for patients and families with CMT,” said Allison Moore, founder and CEO of the Hereditary Neuropathy Foundation. “Dr. Thomas’ empathic approach in evaluating and treating patients is unique. His mission is to improve patient outcomes and quality of life by pursuing clinical trials of novel medications that can help people with CMT.”
Allison, who has CMT, often consults with Dr. Thomas on approaches to HNF’s research initiatives, clinical study designs and for her own medical care.
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