Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy
Public ClinicalTrials.gov record NCT04281485. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO CONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PF 06939926 FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT04281485
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 114 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 4 Years to 7 Years
- Sex
- Male
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Nov 5, 2020 (Actual)
- Primary completion
- May 15, 2024 (Actual)
- Completion
- Apr 15, 2039 (Estimated)
- Last update posted
- Jul 22, 2026
2020 – 2039
United States locations
- U.S. sites
- 17
- U.S. states
- 9
- U.S. cities
- 11
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Arkansas Children's Hospital | Little Rock | Arkansas | 72202 | — |
| Arkansas Children's | Little Rock | Arkansas | 72202 | — |
| UCLA Medical Center | Los Angeles | California | 90095 | — |
| University of Florida | Gainesville | Florida | 32610 | — |
| University of Iowa Hospitals and Clinics | Iowa City | Iowa | 52242 | — |
| KU Clinical Research Center - Clinical and Translational Science Unit (CTSU) - Fairway | Fairway | Kansas | 66205 | — |
| KU Clinical Research Center - Clinical and Translational Science Unit (CTSU) - Rainbow | Kansas City | Kansas | 66160 | — |
| University of Kansas Hospital - Investigational Pharmacy | Kansas City | Kansas | 66160 | — |
| University of Kansas Hospital - Pediatric and Pediatric ICU - Operating Room | Kansas City | Kansas | 66160 | — |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | — |
| Pediatric Cardiology | Prairie Village | Kansas | 66208 | — |
| Lenox Baker Children's Hospital | Durham | North Carolina | 27705 | — |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| Primary Childrens Hospital | Salt Lake City | Utah | 84113 | — |
| University of Utah Clinical Neurosciences Center | Salt Lake City | Utah | 84132 | — |
| University of Utah Hospital | Salt Lake City | Utah | 84132 | — |
| Seattle Children's | Seattle | Washington | 98105 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 36 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04281485, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jul 22, 2026 · Synced Oct 23, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT04281485 live on ClinicalTrials.gov.