A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy
A Phase 1b Multicenter, Open-label, Single Ascending Dose Study To Evaluate The Safety And Tolerability Of Pf-06939926 In Ambulatory Subjects With Duchenne Muscular Dystrophy
Doctors at 20 research centers in the U.S. seek patients with Duchenne Muscular Dystrophy (DMD) to trial an investigatory gene therapy, a virus engineered to transfer a functional micro-dystrophin gene to the patient, so that their bodies can produce normal dystrophin and normal muscle fibers.
Participants will undergo a single IV infusion of the investigatory gene therapy at one of 2 dose levels,, then be assessed at treatment time, baseline, 2 months and 1 year post-treatment, with a total follow-up period of 5 years.
- Be male, 5 to 12 years of age
- Have a clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype
- Have body weight between 15 and 50 kg
- Not have received live attenuated vaccination within 3 months prior or exposure to a systemic antiviral and/or interferon therapy within 30 days prior to receipt of PF-06939926
THE STUDY INVOLVES:
- Screening to confirm eligibility.
- Participants receive a single infusion of the investigatory gene therapy.
- Lab and biopsy assessments will continue for 12 months, with long-term follow up for 5 years.
LOCATIONS AND CONTACTS:
There are 20 study locations in the United States. Find the closest to you here.
Pfizer CT.gov Call Center | 1-800-718-1021 | [email protected]
Or go online: