The First Duchenne Muscular Dystrophy Patient Receives Gene Therapy Treatment in Pfizer’s Phase 3 Trial

The very first patient has just been dosed in Pfizer’s Phase 3 clinical trial for Duchenne muscular dystrophy (DMD). The trial is called CIFFREO. This patient received the therapy at a trial site in Spain on the 29th of December, 2020.

Since DMD is a progressive disease, the earlier a treatment is provided, the better outcomes a patient will have. Unfortunately, there are few treatments available to patients. Pfizer hopes to change this.


The CIFFREO trial will include 99 DMD patients across 15 different countries. All patients in this trial will be males who are between the ages of 4 and 7. There are a total of 55 trial locations. The trial is randomized, placebo-controlled, and double blind. However, all patients “treated” with placebo will be able to receive the therapy one year after the completion of the trial.

This trial aims to uncover both the safety and the efficacy of a gene therapy for DMD called PF-06939926. It’s primary endpoint is change of NSAA, or the North Star Ambulatory Assessment. NSAA will be analyzed  at one year following the dose of the therapy. This assessment uses 17 different items to measure the motor function of patients.

All patients in the trial will be monitored for 5 years following the study.

Looking Forward

This gene therapy has already received FDA Fast Track Designation, Orphan Drug Designation, as well as Rare Pediatric Disease Designation. These designations not only represent the potential value of this therapy, but the need for a treatment for this group of patients.

If this Phase 3 trial proves successful, this therapy could change the lives of DMD patients.

As such, Pfizer is already working with regulatory agencies across the world to attempt to make this therapy as widely available as possible, as soon as they possibly can.

You can read more about this clinical trial and gene therapy treatment here.

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