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Be Part of Research - Trial Details - Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy

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

Not Recruiting

Open to: MALE

Age: 4.0 - 7.0

Medical Conditions

Muscular Dystrophies
Muscular Dystrophy, Duchenne


This information is provided directly by researchers, and we recognise that it isn't always easy to understand. We are working with researchers to improve the accessibility of this information. In some summaries, you may come across links to external websites. These websites will have more information to help you better understand the study.


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.

Start dates may differ between countries and research sites. The research team are responsible for keeping the information up-to-date.  

The recruitment start and end dates are as follows:

Nov 2020 May 2024

INTERVENTIONAL

Intervention Type : GENETIC
Intervention Description : PF-06939926 will be administered as a single IV infusion at Year 1 for Cohort 1.

Intervention Arm Group : Cohort 1;

Intervention Type : OTHER
Intervention Description : Placebo will be administered as a single IV infusion at Year 1 for Cohort 2.

Intervention Arm Group : Cohort 2;

Intervention Type : OTHER
Intervention Description : Placebo will be administered as a single IV infusion at Year 2 for Cohort 1.

Intervention Arm Group : Cohort 1;

Intervention Type : GENETIC
Intervention Description : PF-06939926 will be administered as a single IV infusion at Year 2 for Cohort 2

Intervention Arm Group : Cohort 2;



You can take part if:



You may not be able to take part if:


This is in the inclusion criteria above


Below are the locations for where you can take part in the trial. Please note that not all sites may be open.

  • Alder Hey Children's NHS Foundation Trust
    Liverpool
    Merseyside
    L12 2AP
  • The Newcastle upon Tyne Hospitals NHS Foundation Trust, Royal Victoria Infirmary
    Newcastle upon Tyne
    England
    NE1 4LP
  • Great Ormond Street Institute of Child Health
    London
    WCIN 1EH


The study is sponsored by Pfizer




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Read full details for Trial ID: NCT04281485
Last updated 05 June 2025

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