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

NCT04281485 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 114

Last updated 2026-03-02

Study results available
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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

Interventions

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

Sponsors & Collaborators

Principal Investigators

  • Pfizer CT.gov Call Center · Pfizer

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
4 Years
Max Age
7 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-11-05
Primary Completion
2024-05-15
Completion
2039-04-15
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Belgium
  • Canada
  • France
  • Germany
  • Israel
  • Italy
  • Japan
  • Russia
  • South Korea
  • Spain
  • Switzerland
  • Taiwan
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04281485 on ClinicalTrials.gov