This Phase 3 Study Will Evaluate Pamrevlumab as a Treatment for DMD

Pamrevlumab is a first-in-class antibody that treats Duchenne muscular dystrophy (DMD) by inhibiting the effects and activity of connective tissue disorder growth factor (CTGF). It is being developed by FibroGen, and the biopharmaceutical company has recently initiated a Phase 3 trial titled LELANTOS-2. The study will investigate pamrevlumab’s effect on muscle function.

About the Trial

LELANTOS-2 will be double-blind, randomized, and placebo-controlled with the intention of evaluating pamrevlumab’s effect on muscle function. About 70 participants, all between the ages of six and twelve, will receive either pamrevlumab or placebo in combination with systemic corticosteroids. They will be randomized 1:1 and given treatment every two weeks.

Researchers will use the North Star Ambulatory Assessment (NSAA) to judge the effects of pamrevlumab on muscle function. Secondary endpoints of the trial have not yet been released.

If participants complete this study, which will run for 52 weeks, they will be invited to join an open-label extension study and treated with systemic corticosteroids in combination with pamrevlumab.

About Pamrevlumab

This first-in-class antibody is able to treat DMD by stopping CTGF’s activity. CTGF often plays a role in proliferative and fibrotic disorders, especially those that see severe scarring that results in the dysfunction and eventual failure of the organs. By preventing its expression, pamrevlumab is able to treat DMD.

About DMD

Duchenne muscular dystrophy is one of the nine forms of muscular dystrophy. Affected individuals experience progressive muscle weakness and wasting due to a lack of dystrophin. Symptoms include issues with motor skills, muscle weakness, falling, fatigue, issues with changing positions, problems walking, learning disabilities, and eventually heart disease and respiratory failure. All of these effects are caused by a mutation passed down on the X chromosome in a recessive pattern. This is why males are more likely to be affected than females. There is currently no cure for this condition, but treatments can help to manage symptoms.

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