A Phase 3 Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy


Last updated date
Study Location
Children's Hospital - London Health Sciences Centre
London, Ontario, N6A 4G5, Canada


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Eligibility Criteria
The disease, disorder, syndrome, illness, or injury that is being studied.
Duchenne Muscular Dystrophy
Pediatric Trials: 0-17 Years
Adult Trials: 18+ Years
4-7 years


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Advanced Information
Descriptive Information
Brief Title  ICMJE A Phase 3 Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy
Brief Summary The study will evaluate the safety and efficacy of gene therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study with two thirds of participants assigned to gene therapy. The one third of participants who are randomized to the placebo arm will have an opportunity for treatment with gene therapy at the beginning of the second year.
Detailed Description

The study will assess the efficacy of PF-06939926 gene therapy on ambulatory function while also monitoring its safety. Approximately 99 boys with DMD will be enrolled and randomly assigned to one of two groups: approximately two thirds will be in Cohort 1 and receive gene therapy at the start of the study; approximately one third will be in Cohort 2 and receive placebo at the start of the study and receive gene therapy after one year, as long as it remains safe to do so. The treatment (PF-06939926 gene therapy or placebo) will be given as an intravenous infusion lasting up to 2 hours.

The study includes boys who are at least 4 years old and less than 8 years old (including 7 year olds up until their 8th birthday). All boys will need to be on a daily dose of glucocorticoids (prednisone, prednisolone, or deflazacort) for at least 3 months prior to enrolling and to stay on daily glucocorticoids for the first 2 years of the study. All boys will need to be negative for neutralizing antibodies against AAV9, as measured by the test done for the study as part of screening.

The primary outcome of the study will be assessed at 52 weeks. All participants will be followed in the study for 5 years after treatment with gene therapy.

The study medication, all medical tests associated with the study, and the visits to the study sites are free of charge. Participants will also be supported for travel costs associated with study visits.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:
Parallel up to the measurement of the primary outcome at Week 52. At the beginning of study Year 2 participants who were originally assigned to placebo will have the opportunity to receive PF-06939926. All participants will be followed for 5 years following treatment with PF-06939926.
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description:
The study will be quadruple blind.
Primary Purpose: Treatment
Condition  ICMJE Duchenne Muscular Dystrophy
Intervention  ICMJE
  • Genetic: PF-06939926
    PF-06939926 will be administered as a single IV infusion at Year 1 for Cohort 1.
  • Other: Placebo
    Placebo will be administered as a single IV infusion at Year 1 for Cohort 2.
  • Other: Placebo
    Placebo will be administered as a single IV infusion at Year 2 for Cohort 1.
  • Genetic: PF-06939926
    PF-06939926 will be administered as a single IV infusion at Year 2 for Cohort 2
Study Arms  ICMJE
  • Cohort 1
    Approximately two thirds of participants will be randomized to Cohort 1.
    • Genetic: PF-06939926
    • Other: Placebo
  • Cohort 2
    Approximately one third of participants will be randomized to Cohort 2.
    • Other: Placebo
    • Genetic: PF-06939926
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: February 20, 2020)
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE September 29, 2027
Estimated Primary Completion Date September 29, 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Key inclusion criteria:

  1. Confirmed diagnosis of Duchenne muscular dystrophy by prior genetic testing
  2. Receiving a stable daily dose (at least 0.5 mg/kg/day prednisone or prednisolone, or at least 0.75 mg/kg/day deflazacort) for at least 3 months prior to Screening
  3. Ambulatory, as assessed by protocol-specified criteria

Key exclusion criteria:

  1. Positive test performed by Pfizer for neutralizing antibodies to AAV9
  2. Any treatment designed to increase dystrophin expression within 6 months prior to screening (e.g., Translarna?, EXONDYS 51?, VYONDYS 53?)
  3. Any prior treatment with gene therapy
  4. Any non-healed injury that may impact functional testing (eg NSAA)
  5. Abnormality in specified laboratory tests, including blood counts, liver and kidney function
Sex/Gender  ICMJE
Sexes Eligible for Study:Male
Gender Based Eligibility:Yes
Gender Eligibility Description:Male
Ages  ICMJE 4 Years to 7 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Pfizer CT.gov Call Center1-800-718-1021[email protected]
Listed Location Countries  ICMJE Canada,   Israel,   Italy,   Japan,   Korea, Republic of,   Russian Federation,   Spain,   Taiwan,   United Kingdom
Removed Location Countries  
Administrative Information
NCT Number  ICMJE NCT04281485
Other Study ID Numbers  ICMJE C3391003
2019-002921-31 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD:Yes
Plan Description:Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/d….
Responsible Party Pfizer
Study Sponsor  ICMJE Pfizer
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director:Pfizer CT.gov Call CenterPfizer
PRS Account Pfizer
Verification Date April 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP