Phase 2 study of Vamorolone (Formerly VBP15) in people with Duchenne

A Potential Steroid Alternative Customized for Duchenne

Questions and Answers

What stage is this research?

There are multiple trials going on in parallel:

  • A Phase 2a clinical trial (VBP15-002) of 48 Duchenne boys (4 to <7 years, steroid naïve) is completed. This is a 2 week treatment, with a focus on safety and biomarkers. Data is currently under interpretation.
  • A Phase 2a extension clinical trial (VBP15-003) of the same 48 boys is fully enrolled and ongoing. This is 24 weeks of treatment. Last patient last visit is Q2 2018.
  • A Long Term Extension study (VBP15-LTE) is open for the same 48 boys, enabling dose escalation. This is a 2 year treatment period, and is ongoing.
  • A Phase 2b clinical trial (VBP15-004) for boys with Duchenne muscular dystrophy is currently recruiting. This is a 48 week study of about 120 boys with Duchenne (4 to <7 years, steroid naïve).
  • This study plans to recruit at about 30 sites in the following countries (US, CANADA, UNITED KINGDOM, ISRAEL, AUSTRALIA, SWEDEN, GERMANY, ITALY, NETHERLANDS, CZECH REPUBLIC, BELGIUM). The first 24 weeks (~6 months) will include four arms (low dose vamorolone, high dose vamorolone, prednisone, and placebo; 1:1:1:1), and for the second 24 weeks all Duchenne boys will transition to vamorolone (high dose or low dose).

What is the goal or purpose of this research study?

The goal of this research is to see if an investigational drug called vamorolone is effective (improves or stabilizes muscle strength and function) and has fewer side effects than steroids in boys with Duchenne.

Vamorolone is hoped to retain the beneficial anti-inflammatory and muscle strengthening aspects of corticosteroids (prednisone, deflazacort), while decreasing some of the undesirable side effects (bone fragility, stunted growth, insulin resistance, mood changes, delay of puberty and others).

Vamorolone has additional activities such as a mineralocorticoid receptor antagonism and membrane stabilization that may increase benefit to boys with Duchenne.

Who is funding this research study?

Many non-profit foundations and governments have funded the vamorolone program. A complete description of funders can be found at
The National Institute of Neurological Disorders and Stroke (1R44NS095423-01) and the National Institute of Arthritis and Musculoskeletal and Skin Diseases (1U34AR068616-01) have provided funding for VBP15-002 and VBP15-003 trials.

The European Union’s Horizon 2020 research and innovation program under grant agreement No 667078 has provided funding for VBP15-004 (via Newcastle University, UK).

To date, ReveraGen has worked through public-private partnerships to develop other aspects of the vamorolone development program:

  • PPMD ($750,000) and Foundation to Eradicate Duchenne ($250,000) are co-funding the chronic toxicology studies
  • The $2.1M Phase 1 trial was funded by MDA (50%), and three UK foundations (Joining Jack, Duchenne Research Fund, Duchenne Children’s Trust)
  • Additional funding has provided by the Save Our Sons, NIH TRND, CDMRP Department of Defense, CureDuchenne, and the Duchenne Alliance Research Foundation (Save Our Sons, Michael’s Cause, Pietro’s Fight, Alex’s Wish, and Ryan’s Quest).

Who is eligible to participate in this research study?

Boys with Duchenne who have never taken steroids and who are ages 4, 5, or 6 at study entry.

Additional eligibility criteria may apply and can be discussed with a study team member.

How long will this research study last, and will I have access to the investigational drug once the study has ended?

The newly recruiting VBP15-004 study will last approximately one year.

After completion of the study, there may be an extension study to allow continued access to vamorolone (long term extension).

Where does this research study take place?

The newly recruiting VBP15-004 study will take place at sites located in the USA, Canada, Europe, Israel, and Australia.

Detailed site information can be found on (NCT03439670).

How many visits to the study site are necessary?

Approximately 15 visits over about 12 months.

Can any visits be done locally?

Visits must be done at one of the participating sites.

Will I get paid for participating in this research study?

No, but travel, stay, and meal expenses are paid for by the study.

Why should I consider participating in this research study?

Participation will help determine whether vamorolone is an effective therapy for Duchenne and how its effectiveness and side effects compare to prednisone.

Showing effectiveness for Duchenne could have implications for many other disorders where steroids are used.

The vamorolone program includes many innovations in clinical trial design that, if successful, will speed other drug development programs, including blood biomarkers and mobile health outcomes.

Where can I learn more about this research study?

Information will be posted on the ReveraGen website, as well as

Contact Andrea Smith at

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