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Boys with DMD get continued access to promising steroid alternative

NCT ID NCT03863119

Disease control Sponsor: Santhera Pharmaceuticals 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
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 Results may not be published yet.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 This study
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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This program offers ongoing access to vamorolone, a steroid-like drug, for boys with Duchenne muscular dystrophy who finished earlier studies. It aims to keep them on treatment while the drug is being reviewed for approval. Participants take vamorolone daily by mouth at doses chosen by their doctor.

What this could mean

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

Active substance
Vamorolone
What this could lead to
If successful, this expanded access program provides continuous treatment for boys with Duchenne muscular dystrophy while the drug is being reviewed for approval.
What could go wrong
This is an expanded access program, not a controlled trial, so results are observational. Vamorolone may not be approved, and long-term risks are still being studied.

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.

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.

Ages

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

Sex

Male participants only

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.

Inclusion Criteria: * Subject's parent or legal guardian has provided written informed consent/HIPAA authorization * Subject has previously completed at a participating US or Canada study site VBP15-LTE up to and including the Month 24 assessments, OR VBP15-004 up to and including the Week 48 assessments, VBP15-006 up to and including the Week 12 assessment * Subject and parent/guardian are willing and able to comply with recommended study drug administration plan, and standard of care follow-up and monitoring as recommended by their Treating Physician Exclusion Criteria: * Subject had a serious or severe adverse event in study VBP15-LTE or VBP15-004 or VBP15-006 that, in the opinion of the Treating Physician and Sponsor, was probably or definitely related to vamorolone use and precludes safe use of vamorolone for the subject in this expanded access program * Subject and/or parent/guardian are unable and/or unwilling to comply with regular medical care and follow-up as recommended by their Treating Physician throughout participation in the VBP15-EAP

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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

    12 sites in 3 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    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

  • Alberta's Children Hospital

    AVAILABLE

    Calgary, Alberta, T3B 6A8, Canada

  • British Columbia Children's Hospital

    AVAILABLE

    Vancouver, British Columbia, V6H 3N1, Canada

  • Children's Hospital of Eastern Ontario

    AVAILABLE

    Ottawa, Ontario, K1H 8L1, Canada

  • Duke University

    AVAILABLE

    Durham, North Carolina, 27710, United States

  • Montreal Childrens Hospital

    AVAILABLE

    Montreal, Quebec, H4A 3J1, Canada

  • Nemours Children's Hospital

    AVAILABLE

    Orlando, Florida, 32827, United States

  • Schneider Chidlren's Medical Center

    AVAILABLE

    Petah Tikva, 4920435, Israel

  • Seattle Children's

    AVAILABLE

    Seattle, Washington, 98105, United States

  • The Hospital for Sick Children

    AVAILABLE

    Toronto, Ontario, M5G 1X8, Canada

  • University of California Davis

    AVAILABLE

    Davis, California, 95616, United States

  • University of Texas Southwestern Medical Center

    AVAILABLE

    Dallas, Texas, 75207, United States

  • urie Children's Hospital of Chicago

    AVAILABLE

    Chicago, Illinois, 60611, United States

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Other studies related to the condition(s) this trial covers.