New hope for duchenne: Long-Term drug safety trial underway
NCT ID NCT07209332
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study looks at the long-term safety of an experimental drug called WVE-N531 in people with Duchenne muscular dystrophy (DMD) who have already taken it in a previous study. About 175 participants will receive the drug and be monitored for side effects, heart and lung function, and muscle strength. The goal is to see if the drug remains safe and effective over time.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 175 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Dec 2025
- Expected to finish
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Mar 2029
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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Children (under 18), adults (18 to 64) and older adults (65 and over)
- Sex
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Male participants only
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Participated in a prior study of WVE-N531 and has not experienced any significant toxicities due to WVE-N531 or significant clinical deterioration of general health since the last dose or early discontinuation visit. 2. Of note: if there will be a greater than 31-day gap between the last dose on the prior study and the FD on this study, the case should be discussed between the Investigator and Medical Monitor. Exclusion Criteria: 1. Any clinically significant medical finding or change during or following participation in the prior WVE-N531 study, other than DMD that, in the judgment of the Investigator, would affect the potential safety of the patient to receive WVE-N531 or interfere with participation in the study. 2. Any recreational substance use (including prescribed cannabinoids), with the exception of alcohol and nicotine, irrespective of legality, within 2 months prior to FD and/or unwilling to refrain from such use for the duration of the study.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
3 sites in 2 countries. The list below names each one and where it is.
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The official record
The full official record for this study. This one lists no contact details, but it is the first place any would appear.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Istiklal Hospital/ Clinical Research Unit
Amman, Jordan
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Oxford Children's Hospital, Oxford University Hospitals NHS Foundation Trust
Headington, Oxford, OX3 9DU, United Kingdom
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The Specialty Hospital (TSH)/ Advanced Clinical Center
Amman, Jordan
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a new dosing schedule tame steroid side effects in duchenne?
- Can a daily supplement ease the toll of duchenne muscular dystrophy?
- Can a lower steroid dose preserve strength in young boys with DMD?
- Can a targeted infusion slow muscle decline in duchenne? a new trial aims to find out.
- Can a massive patient database unlock new treatments for muscular dystrophy?
- Umbilical cord stem cells aim to slow muscle loss in duchenne boys