Ifetroban for Treating DMD-Associated Cardiomyopathy, continued from page 6 lar Dystrophy” appeared in the December 2019 issue of the Journal of American Heart Association. Although the Phase II trial hasn’t yet begun recruiting, Macias-Perez said the first site at Children’s National in Washington, D.C., will begin this month. The plan, she continued, is to have six to nine sites across the country participate to quickly enroll 48 DMD patients. The boys in the study must be at least seven years old and have stable heart disease. Although this is the first ifetroban trial for DMD patients, Macias-Perez noted the safety profile of ifetroban is well established with more than two dozen clinical trials, including Cumberland Pharmaceutical’s ongoing trials in scleroderma and aspirin-exacerbated respiratory disease (AERD). “We worked with families, patient
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advocacy groups and caregivers to help us design a study that focuses most importantly on the patient and their families,” Macias-Perez said. For this reason, she continued, study participants only have to visit the clinician at least three times within a 12-month period to minimize the burden of travel. Additionally, a travel stipend will be provided for those traveling more than 50 miles to the clinical sites in an effort to keep participation from becoming a financial burden. “Cumberland also designed a new method of measuring ifetroban pharmacokinetics for the DMD trial using a finger-stick method that can be done at home. We were informed during the study design review process that traditional methods using multiple blood draws over 24 hours is a challenge for
DMD patients and their families,” said Macias-Perez. “The patient families also told us that having the option of continuing on ifetroban after completing the trial is important to them when considering participation on a clinical trial. We changed our protocol to include an optional, open-label extension for all patients that complete the initial 12 months of treatment. “Incorporating patient feedback into our DMD trial design has added greater value and meaning. We are so grateful to our DMD community for their support of our clinical trial,” she added. “This new program is an excellent strategic fit for our company given our mission to develop new medicines that address unmet medical needs,” said Cumberland Pharmaceuticals CEO A.J.
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Easing the Suffering of Serious Illnesses, continued from page 11
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Kazimi. “As ifetroban may uniquely address the heart failure associated with this deadly disease, we very much appreciate the FDA grant support of our novel treatment for these A.J. Kazimi critically ill patients.” He added, “The FDA’s involvement through the Orphan Drug Grant is the first time they have gotten involved with DMD from a financial support standpoint.” Kazimi said it is exciting the FDA believed this was the right program, the right product and the right clinical sites to move forward.
has been in the news. The state recently scored a D on an access report. “Part of this was the math and how the scoring was calculated,” Tucker said. “Alabama has quite a few small hospitals, under 50 beds, that aren’t large enough to offer a palliative care program. But it is true that there is a real problem in access to palliative care, especially in rural counties. In light of this, we’re working with county nurses in health departments using telemedicine to bring palliative care to patients.” Tucker and the center also advocate for legislation to expand education, training and research to make palliative care more widely available. “A bill for creating centers of excellence for training and research in palliative care has passed the House of Representatives and is now in a Senate committee with bipartisan support,” Tucker said. “We hope to see passage of that bill soon.” The center is also gearing up to begin a new training program for physicians to assist them in communicating with patients who are experiencing serious health challenges. “In these conversations, there are three essential questions doctors need to ask,” Tucker said. “What matters most, what gives you strength, and who speaks for you?”
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