According to BioSpace, the first patient has been enrolled in a trial of pamrevlumab, a Duchenne muscular dystrophy treatment. The third phase of LELANTOS plans to evaluate the drug’s effects on muscle function.
About Duchenne Muscular Dystrophy (DMD)
DMD is one of nine forms of muscular dystrophy and is characterized by progressive muscle weakness. It occurs when the body is unable to make dystrophin, which is due to an X-linked recessive mutation. Symptoms will manifest within the first six years of life, and they include frequent falling, fatigue, difficulty with motor skills, learning disabilities, issues with walking and moving positions, muscle weakness that starts in the legs and pelvis that progresses throughout the body, and eventually heart disease and respiratory failure. Doctors will use a variety of tests to diagnose this condition, including EMGs, muscle biopsies, CPKs, and genetic testing. After a diagnosis is obtained, treatment is aimed at slowing progression and managing symptoms. This includes steroids, heart medicine, assisted breathing, amino acid treatment, asthma medication, supplements like creatine, and physical and occupational therapy.
About the Trial
LELANTOS has enrolled the first patient for its third phase. The trial will be double-blind, placebo-controlled, and randomized in order to best understand pamrevlumab’s effects on muscle function in those with non-ambulatory DMD.
90 patients are expected to enroll, and they will be randomized 1:1. One group will take pamrevlumab while the other will receive a placebo in combination with systemic corticosteroids. They will remain in these groups for 52 weeks. Afterwards, researchers will assess the performance of the upper limbs, heart, and lungs. If participants complete all 52 weeks of the third phase, they will be invited to join an extension of the study.
If this study is successful, pamrevlumab may become a viable treatment option for those with Duchenne muscular dystrophy.