Phase 3 Trial of Pamrevlumab Enrolling DMD Patients Unable to Walk

The LELANTOS Phase 3 clinical trial, which is evaluating FibroGen‘s pamrevlumab in people with Duchenne muscular dystrophy (DMD) who are no longer able to walk, has enrolled a first patient.

This global study is currently recruiting up to 90 boys and men, ages 12 and older, using systemic corticosteroids. Most sites are yet to be announced; the Rare Disease Research Center in Atlanta is one. Contact information can be found here.

“Initiation of the LELANTOS Phase 3 clinical trial represents a key milestone for FibroGen and for our DMD clinical program, as well as an important development for the patients and families affected by Duchenne,” Enrique Conterno, FibroGen’s CEO, said in a press release. “We are grateful for the collaboration of global regulators, investigators, caregivers, and patients in enabling the conduct of this trial.”

Pamrevlumab is an antibody therapy designed to block the activity of connective tissue growth factor (CTGF), a pro-inflammatory protein that promotes fibrosis (scarring) and is found at unusually high levels in the muscles of people with DMD. Fibrosis is a hallmark of muscular dystrophies, contributing to muscle weakness and injury, including to cardiac muscle.

“In DMD, inhibition of connective tissue growth factor (CTGF) by pamrevlumab could result in decreased fibrosis in muscles leading to increased muscle function,” said Elias Kouchakji, MD, senior vice president of clinical development, drug safety, and pharmacovigilance at FibroGen. “We hope pamrevlumab provides a meaningful treatment for the patients.”

Preliminary results from an ongoing and open-label Phase 2 clinical trial (NCT02606136) indicate that the treatment helps to slow the decline or may even improve lung and heart function in non-ambulatory boys and young men with DMD.

Treatment with pamrevlumab was well tolerated, and showed a potential to preserve patients’ use of their upper limbs.

Patients enrolled in the newly initiated LELANTOS trial (NCT04371666), sponsored by FibroGen, will be randomly assigned to receive pamrevlumab or a placebo, both given with corticosteroids, via intravenous infusion (into the bloodstream) every two weeks for up to 52 weeks (one year). Pamrevlumab will be tested at a dose of 35 mg/kg.

The trial’s primary goal is changes in the total score of the performance of upper limb (PUL) assessment from the study’s start to week 52. PUL 2.0 is a measure of upper limb function specifically developed for DMD. Lung and heart health will also be assessed.

Participants who complete the yearlong study will be eligible to continue or start treatment into an open-label extension study, in which all will given both pamrevlumab and corticosteroids.

FibroGen is also investigating pamrevlumab in three other diseases where lessening fibrosis may be beneficial: locally advanced unresectable pancreatic cancer, idiopathic pulmonary fibrosis, and COVID-19.