Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT04281485 · ClinicalTrials.gov registry record · Phase 3
Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy
A Phase 3 study of Duchenne Muscular Dystrophy, sponsored by Pfizer.
- Active
- Registry status
- Phase 3
- Development phase
- 114
- Enrollment target
- 20
- Study locations
NCT04281485 is a Phase 3 study of Duchenne Muscular Dystrophy that is active but no longer recruiting, run by Pfizer. The registered enrollment target is 114 participants, below the 410-participant average among 48 other Duchenne Muscular Dystrophy trials with a reported enrollment target (72% lower). The trial reports 20 study locations across 12 states.
The verdict
NCT04281485, a Phase 3 study of Duchenne Muscular Dystrophy, is active but no longer recruiting, sponsored by Pfizer.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 114 participants
- Enrollment target
- 20
- Study locations
Study 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.
Conditions Studied
Interventions
- OTHER Placebo
- GENETIC PF-06939926
Study Locations (20)
Kansas
- KU Clinical Research Center - Clinical and Translational Science Unit (CTSU) - Fairway - Fairway
- KU Clinical Research Center - Clinical and Translational Science Unit (CTSU) - Rainbow - Kansas City
- University of Kansas Hospital - Investigational Pharmacy - Kansas City
- University of Kansas Hospital - Pediatric and Pediatric ICU - Operating Room - Kansas City
- University of Kansas Medical Center - Kansas City
- Pediatric Cardiology - Prairie Village
Utah
- Primary Childrens Hospital - Salt Lake City
- University of Utah Clinical Neurosciences Center - Salt Lake City
- University of Utah Hospital - Salt Lake City
Arkansas
- Arkansas Children's Hospital - Little Rock
- Arkansas Children's - Little Rock
California
- UCLA Medical Center - Los Angeles
Florida
- University of Florida - Gainesville
Iowa
- University of Iowa Hospitals and Clinics - Iowa City
North Carolina
- Lenox Baker Children's Hospital - Durham
Pennsylvania
- The Children's Hospital of Philadelphia - Philadelphia
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 114 participants |
| Start Date | 2020-11-05 |
| Est. Completion | 2039-04-15 |
| Phase | Phase 3 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04281485
The ClinicalTrials.gov registry entry for NCT04281485 describes a study currently listed as active not recruiting, categorized as Phase 3. The registered enrollment target is 114 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 410-participant average among 48 other Duchenne Muscular Dystrophy trials with a reported enrollment target (72% lower). The listed sponsor is Pfizer, which has 1,845 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Duchenne Muscular Dystrophy appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT04281485 reports 20 study locations spanning 12 distinct geographic areas - top geographies include Kansas, Utah, Arkansas.
Frequently Asked Questions
What is clinical trial NCT04281485 about?
NCT04281485 is a clinical study titled "Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy". 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 trea...
What is the current status of trial NCT04281485?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 114 participants. The study started on 2020-11-05. Estimated completion is 2039-04-15.
What conditions does trial NCT04281485 study?
This clinical trial studies the following conditions: Duchenne Muscular Dystrophy.
What interventions are being tested in trial NCT04281485?
The interventions under investigation include: Placebo (OTHER), PF-06939926 (GENETIC).
Who is sponsoring clinical trial NCT04281485?
This trial is sponsored by Pfizer, which has 1,845 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04281485 being conducted?
This trial has 20 study locations across Arkansas, California, Florida, Iowa, Kansas. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Duchenne Muscular Dystrophy
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
-
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)
ACTIVE NOT RECRUITING · Phase 3
-
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
COMPLETED · Phase 3
-
Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular Dystrophy
COMPLETED · Phase 3
-
Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)
RECRUITING · Phase 1
-
AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)
RECRUITING · Phase 2
-
A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD)
RECRUITING · Phase 2
Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.