Pfizer has announced that it has enrolled the first UK participant in a global Phase III study aiming to evaluate its investigational gene therapy candidate in boys with Duchenne muscular dystrophy (DMD).
The UK participant was enrolled at the Newcastle upon Tyne NHS Foundation Trust, which is one of three sites in the UK involved in the clinical trial.
The CIFFREO study is being conducted at 55 sites globally across 15 countries and is expected to enrol 99 ambulatory male patients aged four to seven years old.
The Phase III CIFFREO study’s primary endpoint is the change from baseline in the North Star Ambulatory Assessment (NSAA) at one year – the NSAA is a test that aims to measure gross motor function in people living with DMD.
Pfizer added that eligible participants will be scheduled to receive the investigational gene therapy – known as PF-06939926 – either at the beginning of the study or after one year following placebo treatment, regardless of cohort.
“The enrolment of the first UK patient in this Phase III gene therapy programme is a great achievement for the UK Duchenne community and we are very pleased to contribute to innovative research in the pursuit of future therapies,” said Michela Guglieri, consultant neurologist at Newcastle Hospitals NHS Foundation Trust and UK chief investigator of the CIFFREO trial.
“There are currently no approved disease-modifying treatment options available for all genetic forms of the disease in the UK. I’m very proud to be leading the UK arm of this global study,” she added.
DMD is caused by mutations in the gene encoding dystrophin which is needed for muscle membrane stability. The condition mainly affects boys as DMD is an X-linked disease, with symptoms including progressive muscle degeneration that worsens with age.
In the US, PF-06939926 has received a fast track designation from the Food and Drug Administration (FDA) as well as orphan drug and rare paediatric disease designations.