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Real-World data sought on DMD Exon-Skipping drugs

NCT ID NCT06606340

Knowledge-focused Sponsor: Sarepta Therapeutics, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only This study
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 24, 2026 · Last updated Jun 26, 2026 · Updated 1 time

Summary

This observational study will follow 300 people with Duchenne muscular dystrophy who are already taking exon-skipping therapies (eteplirsen, golodirsen, or casimersen) as part of their routine care. Researchers will collect data on movement, lung function, and heart function over time to see how these treatments work in everyday practice. No new drugs are being tested; the goal is to gather long-term safety and effectiveness information.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
eteplirsen, golodirsen, casimersen
What this could lead to
If successful, this study could provide real-world evidence on how well these exon-skipping therapies work over the long term for Duchenne muscular dystrophy.
What could go wrong
This is an observational study, not a controlled trial, so results may be influenced by factors like differences in patient care. It does not test new treatments, only monitors existing ones.

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.

Participants

About 300 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jan 2019

Expected to finish

Dec 2033

An estimate. End dates often move.

Lead sponsor

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.

Who is studied

Participants within each prescriber's practice who are receiving eteplirsen, golodirsen, or casimersen, in routine clinical practice, and who meet the study eligibility criteria and provide informed consent (either by the participant or through authorization by a legal guardian), will be invited to enroll into the study and will be followed according to the protocol.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Male participants only

Healthy volunteers

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

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: * Is willing to provide informed assent or consent (if applicable) and has a parent(s) or legal guardian(s) or is a participant ≥18 years of age who is (are) willing to provide informed consent for the participant to participate in the study and comply with study data collection procedures. * Has an established clinical diagnosis of DMD, as documented prior to screening by a genetic report. * Receiving, or initiating treatment with, eteplirsen, golodirsen, or casimersen at the time of observational study enrollment. Note: Participants with a prescription for eteplirsen, golodirsen, or casimersen at enrollment must initiate the exon-skipping therapy within 6 months of the date of enrollment or will no longer be eligible for this study. Note: Enrollment of eteplirsen participants has been completed, no additional participants will be enrolled. Key Exclusion Criteria: * Is currently participating in any DMD interventional study at the time of this study enrollment. * Has declined to provide consent for collection of their genetic data. * Has a medical condition or confounding circumstances that, in the opinion of the Investigator, might compromise: 1. The participant's ability to comply with the protocol-required procedures 2. The participant's wellbeing or safety, and/or 3. The clinical interpretability of the data collected from the participant. Other inclusion/exclusion criteria may apply.

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Conditions

The condition(s) this trial relates to.

Duchenne muscular dystrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Duchenne muscular dystrophy

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    20 sites. The list below names each one and where it is.

  2. 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.

    Open the record ↗

  3. 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

  • Arkansas Children's Hospital Research Institute

    Little Rock, Arkansas, 72202, United States

  • Atrium Health Wake Forest University Health Sciences

    Winston-Salem, North Carolina, 27157, United States

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027, United States

  • Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Children's National Hospital

    Washington D.C., District of Columbia, 20010, United States

  • Columbia University Medical Center - PIN

    New York, New York, 10032, United States

  • Cook Children's Hospital

    Fort Worth, Texas, 76104, United States

  • Duke Lenox Baker Children's Hospital

    Durham, North Carolina, 27705, United States

  • Kennedy Krieger Institute

    Baltimore, Maryland, 21287, United States

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Penn State Health Milton S. Hershey Medical Center

    Hershey, Pennsylvania, 17033-0850, United States

  • Seattle Children's Hospital

    Seattle, Washington, 98105, United States

  • Texas Children's Hospital

    Houston, Texas, 77030, United States

  • UPMC Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15213, United States

  • UVA Children's Hospital

    Charlottesville, Virginia, 22908, United States

  • University Of Iowa Hospitals And Clinics

    Iowa City, Iowa, 52242, United States

  • University of California Davis Medical Center

    Sacramento, California, 95817, United States

  • University of Florida

    Gainesville, Florida, 32610, United States

  • Washington University

    St Louis, Missouri, 63110, United States

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