Yesterday the information for Pfizer’s Phase 3, randomized, double-blind, placebo-controlled study of PF-06939926, exploring the safety and efficacy of gene therapy in patients with Duchenne was posted to clinicaltrials.gov. This will be the largest cohort of patients on gene therapy to date.
When the trial begins recruiting later this spring, the study will include boys who are at least 4 years old and less than 8 years old (including 7 year olds up until their 8th birthday). All boys will need to be on a daily dose of glucocorticoids (prednisone, prednisolone, or deflazacort) for at least 3 months prior to screening and to stay on daily glucocorticoids for the first 2 years of the study. All boys will need to be negative for neutralizing antibodies against AAV9, as measured by the test done for the study as part of screening, be ambulatory, and have a confirmed genetic diagnosis.
PPMD looks forward to continuing our close work with Pfizer and as soon as there is more information on this trial, including recruitment information, we will share it with you. To make sure you and your child stay up-to-date with the latest Duchenne trials, make sure you download The Duchenne Registry’s new app and keep your information updated.
Since launching our Gene Therapy Initiative in 2017, PPMD has been cautiously optimistic that gene therapy may be a potential treatment for Duchenne and we will continue to ensure that our community’s questions and concerns are shared.