Clinical Study Alert: Phase 2 Study of Vamorolone in Adults with BMD

Researchers at the University of Pittsburgh are seeking adults living with Becker muscular dystrophy (BMD) to participate in a phase 2 double-blind, placebo-controlled study to evaluate the safety and efficacy of vamorolone to treat BMD. Studies conducted on boys with Duchenne muscular dystrophy (DMD) have shown that vamorolone has beneficial effects with lower side effects than traditional glucocorticoid treatment. The goal of this study is to determine the potential of vamorolone as treatment for people with BMD.

The study involves an optional virtual pre-screening visit and 7 in-person visits over 33 weeks. It consists of 3 parts:

• First, participants will have a pre-screening and/or a screening visit. If they are eligible to participate in the study based on the results of their screening visit, they will be randomly assigned to one of the study groups. Group #1 will receive vamorolone, and group #2 will receive an inactive drug called ‘placebo.’
• Second, participants will receive study medication for 24 weeks. During this treatment period, they will have 4 study visits.
• When the participants finish the treatment period, they will safely stop taking study products by progressively reducing their dose. Then, they will complete one last visit.

The drug/placebo will be administered orally (by mouth). The effects of vamorolone will be evaluated using multiple tests, including physical exams, laboratory testing, motor function tests, and electrocardiograms.

To be eligible, individuals must meet the following inclusion criteria:

• 18+ years of age (up to 65 years)
• Confirmed genetic diagnosis of BMD
• Able to walk about 11 yards in less than 30 seconds
• No cardiac symptoms
• Not received any corticosteroid treatment for the past 3 months

Individuals may not be eligible to participate if they are affected by another illness or receiving another treatment that might interfere with the ability to undergo safe testing.

Please visit this link for the full listing of inclusion and exclusion criteria.

This study is run at the University of Pittsburgh in Pittsburgh, PA. The sponsor will reimburse participants for any reasonable travel expenses (travel, lodging, food, and other direct costs) related to study visits.

To learn more about the study or inquire about participation, please contact the study coordinator Gabi Niizawa at email: niizawaga@upmc.edu or telephone: 412-383-9775.

Si desea participar en este estudio o desea recibir información en español, por favor, comuníquese con la coordinadora del estudio Gabi Niizawa. Correo electrónico: niizawaga@upmc.edu. Tel.: 412-383 9775.