Gene Therapy for Duchenne Muscular Dystrophy
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a gene therapy treatment for boys aged 4 to 7 with muscle problems. The treatment aims to fix the genetic issues causing their muscle problems. Participants will receive the treatment and be monitored for safety and effectiveness. The treatment involves using a specific gene therapy, which has been researched for its potential to correct muscle issues in children with muscle problems.
Will I have to stop taking my current medications?
The trial requires participants to be on a stable dose of certain medications like prednisone, prednisolone, or deflazacort for at least 3 months before joining. It doesn't specify if you need to stop other medications, so it's best to ask the trial team for more details.
Is gene therapy for Duchenne Muscular Dystrophy safe for humans?
The research on gene therapy for Duchenne Muscular Dystrophy, including treatments like NS-065/NCNP-01, shows a favorable safety profile with no severe adverse reactions observed in trials. However, adenovirus vectors used in some gene therapies can have dose-dependent toxic effects, indicating a narrow safety margin.12345
How is the treatment PF-06939926 for Duchenne Muscular Dystrophy different from other treatments?
PF-06939926 is unique because it uses a gene therapy approach, delivering a micro-dystrophin gene via a specially designed virus (rAAVrh74) to target and improve muscle function in Duchenne Muscular Dystrophy patients, unlike traditional treatments that do not address the genetic cause of the disease.678910
What data supports the effectiveness of the treatment PF-06939926 for Duchenne Muscular Dystrophy?
Research shows that gene therapy, including micro-dystrophin gene transfer using adeno-associated virus vectors, is promising for treating Duchenne Muscular Dystrophy. This approach has shown potential in animal models and early trials, suggesting it could help improve muscle function in patients.69101112
Who Is on the Research Team?
Pfizer CT.gov Call Center
Principal Investigator
Pfizer
Are You a Good Fit for This Trial?
This trial is for boys with Duchenne muscular dystrophy (DMD) confirmed by genetic testing, who can walk and have been on a stable dose of steroids for at least 3 months. Boys with antibodies to AAV9, recent treatments increasing dystrophin expression, prior gene therapy, non-healed injuries affecting tests or certain genetic abnormalities in the dystrophin gene cannot participate.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive PF-06939926 gene therapy or placebo via intravenous infusion
Follow-up
Participants are monitored for safety and effectiveness after treatment
Open-label extension
Participants in the placebo group receive gene therapy after one year if safe
What Are the Treatments Tested in This Trial?
Interventions
- PF-06939926
- Placebo
Find a Clinic Near You
Who Is Running the Clinical Trial?
Pfizer
Lead Sponsor
Albert Bourla
Pfizer
Chief Executive Officer since 2019
PhD in Biotechnology of Reproduction, Aristotle University of Thessaloniki
Patrizia Cavazzoni
Pfizer
Chief Medical Officer
MD from McGill University