Researchers at the State University of New York at Binghamton’s School of Pharmacy and Pharmaceutical Sciences are seeking participants living with Becker muscular dystrophy (BMD) to participate in a biomarker and patient-reported outcomes study. The purpose of the Becker Muscular Dystrophy Biomarker and Patient-Reported Outcomes Study is to learn more about disease progression in patients living with BMD, with the hope that information gathered will enable future biomarker-focused clinical trials of new therapeutic agents in BMD. The study seeks to enroll participants who are 6 years of age and older, who weigh more than 30 lbs., and who are diagnosed with BMD.
This is a remote study, meaning that participants will not be asked to visit a clinical site for medical evaluation. For the biomarker portion of the study, a biological sample (blood and urine) will be collected through the use of a mobile phlebotomist via Phlebotek, a company that hires highly trained phlebotomists to perform blood and urine collection at the convenience of patients and research participants in their home or other appropriately designed locations. The phlebotomist will collect a small sample of blood and a urine sample to be used for this biomarker analysis.
Additionally, participants will be sent questionnaires, either electronically or via postal mail, that will ask about one’s physical, emotional and social well-being as it relates to their BMD diagnosis. This type of patient-reported survey has the potential to help provide meaningful insights that can better inform patient care and, potentially, clinical trial design. The questionnaires should take less than 30 minutes to complete.
Research analysis of the study will take place at SUNY Binghamton’s School of Pharmacy and Pharmaceutical Sciences in Binghamton, N.Y.
For additional information, or if you are interested in participating, please contact clinical coordinator Marissa Barbieri at barbieri@binghamton.edu or 607-777-5970.