BMD natural history study may help EDG-5506, research into other therapies
A new natural history study that seeks to gather information about the progression of Becker muscular dystrophy (BMD) in the absence of treatment is being recruited.
The study is led by the GRASP consortium (General Resolution and Assessments Solving Phenotypes) and Virginia Commonwealth University, its main sponsor. Also collaborating on the study ImagingDMD University of Florida and Edgewise Therapeutics, which is developing an experimental oral therapy for BMD called EDG-5506.
The trial (NCT05257473) aims to enroll approximately 150 male patients with BMD, ages 8 and older, at sites in the United States and Europe. Contacts and locations can be found here. As a natural history study, the objective is to assess the evolution of BMD in the absence of treatment.
BMD is caused by a mutation in the gene coding for dystrophin, a protein that functions as a “shock absorber” in muscle cells, helping to protect them from damage during muscle contractions. EDG-5506 is designed to protect muscle fibers from damage by selectively targeting a protein called myosin, which is involved in muscle contraction.
Early data from clinical trials suggested that the experimental therapy is well tolerated and reduces markers of muscle damage in patients with BMD.
Over the course of the two-year study, researchers will assess changes in several measures of muscle function, such as the time it takes to climb four stairs or walk 100 meters (about 330 feet). The North Star rating for limb-girdle muscular dystrophy (LGMD) is also used. LGMD and BMD tend to affect some of the same muscle groups near the shoulders and hips.
The study also tracks changes in measures of heart and lung function, as well as muscle imaging and testing of blood biomarker levels.
The results should provide insight into how BMD tends to evolve without treatment.
The disease has some variation between people, which has proven a challenge for clinical trial design because it can be difficult to disentangle the effects of a treatment amid all the “noise” of data. By providing a better understanding of natural disease progression in a large group of patients, the results may help assess the effectiveness of potential future treatments such as EDG-5506.
“The severity and progression of common BMD symptoms, such as progressive muscle weakness, varies from person to person and has posed a challenge in the design of clinical trials,” said Joanne Donovan, PhD, MD, chief medical officer at Edgewise, in a press release. . “With this study, we hope to advance assessments of disease progression as we advance our lead clinical candidate, EDG-5506, into Phase 2 clinical trials for adolescents and adults with BMD.”