The senior vice president of clinical development, drug safety, and pharmacovigilance at FibroGen provides details of the phase 3 LELANTOS trial of pamrevlumab in Duchenne muscular dystrophy.
“Although our primary end point is to assess muscle function by the PUL, we are looking at these other parameters that we think collectively as combined data have additional benefits. We are hoping that this combined data can indicate that the drug can slow the progression of the disease.”
Pamrevlumab, a first-in-class antibody developed by FibroGen, will be evaluated in a phase 3 study that will explore the effects of the drug on muscle function in nonambulatory patients with Duchenne muscular dystrophy (DMD). Dubbed LELANTOS (NCT04371666), the recently initiated study will use change in the total score of performance of upper limb (PUL) assessment as the primary end point over a 52-week treatment period.
Approximately 90 patients will be randomized 1:1 to receive pamrevlumab plus systemic corticosteroids or placebo plus corticosteroids during the double-blind treatment period. Elias Kouchakji, MD, senior vice president of clinical development, drug safety, and pharmacovigilance, FibroGen, noted that the design of the study was inspired by previous results from a phase 2 study, which ultimately showed pamrevlumab’s ability to decrease fibrosis in muscles, leading to increased muscle function.
Kouchakji sat down with NeurologyLive to provide insight on the parameters of the study, including how they formulated the end points and other important secondary objectives.