Pfizer has dosed the first patient in its Phase Ib clinical trial evaluating the safety and tolerability of its PF-06939926 therapy to treat patients with Duchenne muscular dystrophy (DMD).
The ascending dose trial will enrol around 12 ambulatory boys aged five to 12 years with DMD.
It is designed to analyse a single intravenous infusion of PF-06939926, as well as measurements of dystrophin expression and distribution and assessments of muscle strength, quality and function.
During the screening process, potential candidates for treatment will be tested to confirm a negative result for antibodies against the adeno-associated virus serotype 9 (AAV9) capsid and for a T-cell (immune) response to dystrophin.
Pfizer intends to continue screening and enrolling patients for the trial at up to four clinical research sites in the US.
Duke University Medical Center Pediatrics and Neurology associate professor Edward Smith is the principal investigator of the trial, which aims to provide early data by the first half of next year.
Pfizer Rare Disease Research Unit senior vice-president and chief scientific officer Greg LaRosa said: “Investment in this trial represents the culmination of years of research on behalf of patients by scientists at Pfizer and academic medical centres, along with the support of the DMD patient advocacy community, in the important quest to advance a programme that could potentially change the trajectory of this debilitating disease.
“We’ve listened to the patient community and we know there is a dire need for treatment options; with this in mind, we have built on important scientific advances to design a therapy with the potential to deliver the mini-dystrophin gene to the body and address the underlying cause of DMD, regardless of mutation.
“This trial will assess the safety of this approach to gene therapy and could provide valuable data demonstrating its potential impact to slow down or stop the progression of DMD.”
DMD, a serious genetic disease characterised by progressive muscle degeneration and weakness, usually shows symptoms in early childhood between the ages of three and five.