The trial, LELANTOS-2 (NCT04632940), is enrolling approximately 70 participants ages 6 to 12 at three U.S. sites — Arkansas Children’s Hospital in Little Rock, Rare Disease Research Center in Atlanta, Georgia, and University of Massachusetts Memorial Medical Center in Worcester.
“The initiation of LELANTOS-2, the second Phase 3 clinical trial in the pamrevlumab clinical development program, underscores our commitment to delivering a meaningful treatment for DMD, an area with high unmet medical need,” Elias Kouchakji, MD, senior vice president at FibroGen, said in a press release.
LELANTOS-2 will evaluate how pamrevlumab affects muscle function, compared to a placebo.
Participants will be assigned randomly to receive either treatment, in both cases with systemic corticosteroids given orally every two weeks for up to one year.
The main goal is to assess changes in functional motor abilities from baseline (the study’s start) to 52 weeks, using the North Star Ambulatory Assessment (NSAA), a 17-item scale of motor function used to monitor disease progression and treatment effects.
Secondary goals consist of various tests of muscle function, measuring the ability to stand, climb stairs, and run or walk 10 meters (about 11 yards). Time to loss of ambulation also will be assessed.
Participants who complete the full study will be eligible to enter an open-label extension study in which all will be treated with pamrevlumab and systemic corticosteroids.
Pamrevlumab is an antibody against connective tissue growth factor (CTGF), an inflammatory protein that causes muscle fibrosis, or scarring, and is found at abnormally high levels in skeletal muscles of Duchenne patients. Fibrosis contributes to muscle weakness and to organ dysfunction, with cardiac scarring representing a major complication of DMD.
The first Phase 3 trial of pamrevlumab is evaluating the antibody’s safety and efficacy among males with DMD, ages 12 and older, who no longer can walk. It began last year, is ongoing, and continues to recruit volunteers in the U.S., France, and Spain.
That trial’s primary goal is to measure changes in upper limb function, using the performance of upper limb 2.0 assessment — specifically developed for DMD — from baseline to week 52.
“The initiation of our second Phase 3 study of pamrevlumab for DMD furthers our research on the clinical benefits of inhibiting connective tissue growth factor, an important biological mediator in fibrotic and proliferative disorders,” said Mark Eisner, MD, FibroGen’s chief medical officer.
Pamrevlumab also is being investigated as a treatment for idiopathic pulmonary fibrosis and locally advanced pancreatic cancer.
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