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Be Part of Research - Trial Details - Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

Not Recruiting

Open to: MALE

Age: 4.0 - 16.0

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 primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) doses of DYNE-251 in participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.

The study consists of 3 periods: a multiple-ascending dose (MAD) / placebo-controlled period (24 weeks), an open-label period (24 weeks) and a long-term extension (LTE) period (192 weeks).

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:

Aug 2022 Nov 2029

INTERVENTIONAL

Intervention Type : DRUG
Intervention Description : Administered by IV infusion

Intervention Arm Group : Open-Label and Long-Term Extension Period - DYNE-251;Placebo-Controlled MAD Period - DYNE-251;

Intervention Type : DRUG
Intervention Description : Administered by IV infusion

Intervention Arm Group : Placebo-Controlled MAD Period - Placebo;



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.

  • Great Ormond Street Hospital
    London
    WC1N 3JH
  • Alder Hey Children's Hospital
    Liverpool
    Merseyside
    L12 2AP
  • Royal Victoria Infirmary
    Newcastle Upon Tyne
    Northumberland
    NE1 4LP
  • Leeds Teaching Hospitals NHS Trust
    Leeds
    West Yorkshire
    LS1 3EX
  • Bristol Childrens Hospital
    Bristol
    BS2 8BJ


The study is sponsored by Dyne Therapeutics




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Read full details for Trial ID: NCT05524883
Last updated 10 June 2025

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