Update on phase III study of fordadistrogene movaparvovec, for ambulatory boys with Duchenne Muscular Dystrophy
Pfizer Inc. announced that CIFFREO, a Phase III global, multicenter, randomized, double-blind, placebo-controlled study evaluating the investigational mini-dystrophin gene therapy, fordadistrogene movaparvovec, in ambulatory patients with Duchenne muscular dystrophy (DMD) did not meet its primary endpoint of improvement in motor function among boys 4 to 7 years of age treated with the gene therapy compared to placebo
The primary endpoint in the final analysis was assessed by change in the North Star Ambulatory Assessment (NSAA) at one year after treatment. Key secondary endpoints, including 10-meter run/walk velocity and time to rise from floor velocity, also did not show a significant difference between participants treated with fordadistrogene movaparvovec and placebo.
The overall safety profile of fordadistrogene movaparvovec in the CIFFREO trial was manageable, with mostly mild to moderate adverse events, and treatment-related serious adverse events generally responding to clinical management.
"We are extremely disappointed that these results did not demonstrate the relative improvement in motor function that we had hoped. We plan to share more detailed results from the study at upcoming medical and patient advocacy meetings, with the goal of ensuring that learnings from this trial can help improve future clinical research and development of treatment options that can improve care for boys living with Duchenne muscular dystrophy,” said Dan Levy, MD, PhD, Development Head for Duchenne muscular dystrophy, Pfizer. “We are grateful for the boys, their families, advocates, and the investigators who have participated in this research and the continuing effort to advance treatment options for this debilitating disease.”
About the Fordadistrogene Movaparvovec Clinical Program CIFFREO is a Phase III global, multi-center, randomized, double-blind, placebo-controlled study to assess the safety and efficacy of fordadistrogene movaparvovec investigational gene therapy in ambulatory male participants, aged 4 to 7 years, with a genetic diagnosis of DMD who are on a stable daily regimen of glucocorticoids. The primary endpoint of the study is a change from baseline to one year in the North Star Ambulatory Assessment (NSAA) total score. For more information, visit ciffreoduchennetrial.com or clinicaltrials.gov.
The CIFFREO study is currently on a dosing pause due to a fatal serious adverse event in the Phase II DAYLIGHT trial (NCT05429372). DAYLIGHT is a study that is evaluating the safety and tolerability of fordadistrogene movaparvovec in participants 2 years to 3 years of age with DMD. Pfizer is actively working to gather additional information on the event to understand the potential cause.
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