Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy
NCT ID: NCT04281485
Last Updated: 2026-01-02
Study Results
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View full resultsBasic Information
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ACTIVE_NOT_RECRUITING
PHASE3
114 participants
INTERVENTIONAL
2020-11-05
2039-04-15
Brief Summary
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Detailed Description
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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 15 years after treatment with gene therapy. Participants who received fordadistrogene movaparvovec in Pfizer studies C3391001 and C3391008 or are currently enrolled in Pfizer study C3391011 will be allowed to roll over into the long-term safety follow-up period of this study and will be considered Cohort 3.
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.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Cohort 1
Approximately two thirds of participants will be randomized to Cohort 1.
PF-06939926
PF-06939926 will be administered as a single IV infusion at Year 1 for Cohort 1.
Placebo
Placebo will be administered as a single IV infusion at Year 2 for Cohort 1.
Cohort 2
Approximately one third of participants will be randomized to Cohort 2.
Placebo
Placebo will be administered as a single IV infusion at Year 1 for Cohort 2.
PF-06939926
PF-06939926 will be administered as a single IV infusion at Year 2 for Cohort 2
Interventions
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PF-06939926
PF-06939926 will be administered as a single IV infusion at Year 1 for Cohort 1.
Placebo
Placebo will be administered as a single IV infusion at Year 1 for Cohort 2.
Placebo
Placebo will be administered as a single IV infusion at Year 2 for Cohort 1.
PF-06939926
PF-06939926 will be administered as a single IV infusion at Year 2 for Cohort 2
Eligibility Criteria
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Inclusion Criteria
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
Exclusion Criteria
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
6. Any of the following genetic abnormalities in the dystrophin gene:
1. Any mutation (exon deletion, exon duplication, insertion, or point mutation) affecting any exon between exon 9 and exon 13, inclusive; OR
2. A deletion that affects both exon 29 and exon 30;OR
3. A deletion that affects any exons between 56-71, inclusive.
4 Years
7 Years
MALE
No
Sponsors
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Pfizer
INDUSTRY
Responsible Party
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Principal Investigators
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Pfizer CT.gov Call Center
Role: STUDY_DIRECTOR
Pfizer
Locations
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Arkansas Children's Hospital
Little Rock, Arkansas, United States
Arkansas Children's
Little Rock, Arkansas, United States
UCLA Medical Center
Los Angeles, California, United States
University of Florida
Gainesville, Florida, United States
University of Iowa Hospitals and Clinics
Iowa City, Iowa, United States
KU Clinical Research Center - Clinical and Translational Science Unit (CTSU) - Fairway
Fairway, Kansas, United States
KU Clinical Research Center - Clinical and Translational Science Unit (CTSU) - Rainbow
Kansas City, Kansas, United States
University of Kansas Hospital - Investigational Pharmacy
Kansas City, Kansas, United States
University of Kansas Hospital - Pediatric and Pediatric ICU - Operating Room
Kansas City, Kansas, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
Pediatric Cardiology
Prairie Village, Kansas, United States
Lenox Baker Children's Hospital
Durham, North Carolina, United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Primary Childrens Hospital
Salt Lake City, Utah, United States
University of Utah Clinical Neurosciences Center
Salt Lake City, Utah, United States
University of Utah Hospital
Salt Lake City, Utah, United States
Seattle Children's
Seattle, Washington, United States
The Children's Hospital at Westmead
Westmead, New South Wales, Australia
The Royal Children's Hospital Melbourne
Parkville, Victoria, Australia
Perth Children's Hospital
Nedlands, Western Australia, Australia
UZ Gent
Ghent, , Belgium
UZ leuven
Leuven, , Belgium
Alberta Children's Hospital
Calgary, Alberta, Canada
Children's Hospital - London Health Sciences Centre
London, Ontario, Canada
Childrens Hospital of Eastern Ontario
Ottawa, Ontario, Canada
The Hospital For Sick Children
Toronto, Ontario, Canada
CHU de Nantes- Hotel Dieu
Nantes, , France
Hopital Necker
Paris, , France
Charité - Universitätsmedizin Berlin
Berlin, , Germany
Universitatsklinikum Essen
Essen, , Germany
Hadassah University Medical Center, Ein Kerem
Jerusalem, , Israel
Schneider Children's Medical Center of Israel
Petach Tikvah, , Israel
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Roma, , Italy
IRCCS Ospedale Pediatrico Bambino Gesù
Rome, , Italy
Nagoya City University Hospital
Nagoya, Aichi-ken, Japan
Hyogo College of Medicine College Hospital
Nishinomiya, Hyōgo, Japan
National Center of Neurology and Psychiatry
Tokyo, , Japan
Saint Petersburg State Paediatric Medical University
Saint Petersburg, , Russia
State Autonomous Healthcare Institution of Sverdlovsk Region Children's City Clinical Hospital No 9
Yekaterinburg, , Russia
Pusan National University Yangsan Hospital
Yangsan, Gyeongsangnam-do, South Korea
Seoul National University Hospital
Seoul, , South Korea
Samsung Medical Center
Seoul, , South Korea
Hospital Sant Joan de Déu
Esplugues de Llobregat, Barcelona, Spain
Hospital Universitario Vall d'Hebron
Barcelona, , Spain
Hospital Universitari i Politecnic La Fe de Valencia
Valencia, , Spain
Inselspital, University Children's Hospital Berne
Bern, , Switzerland
Universitaets-Kinderspital Zuerich
Zurich, , Switzerland
Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, , Taiwan
National Taiwan University Hospital
Taipei, , Taiwan
Taipei Veterans General Hospital
Taipei, , Taiwan
The Newcastle upon Tyne Hospitals NHS Foundation Trust, Royal Victoria Infirmary
Newcastle upon Tyne, England, United Kingdom
Alder Hey Children's NHS Foundation Trust
Liverpool, Merseyside, United Kingdom
Great Ormond Street Institute of Child Health
London, , United Kingdom
Countries
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Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Related Links
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To obtain contact information for a study center near you, click here.
Other Identifiers
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2023-508510-42-00
Identifier Type: REGISTRY
Identifier Source: secondary_id
C3391003
Identifier Type: -
Identifier Source: org_study_id
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