VAMOROLONE - A Potential Steroid Alternative Customized for Duchenne

Frequently Asked Questions (FAQs) about this research:

 

What stage is this research?

  • This study is a Phase IIa clinical trial (VBP15-002/VBP15-003) and is active but no longer recruiting.

 

What is the goal or purpose of this study?

  • The goal of this research is to see if vamorolone can be safely and effectively used for the treatment of 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). 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 study?

  • The study sponsor ReveraGen has received funding from the National Institutes of Health - National Institute of Neurological Disorders and Glossary Link Stroke to carry out the Phase IIa and Phase IIa extension study (1R44NS095423-01). The National Institutes of Health - National Institute of Arthritis and Musculoskeletal and Skin Diseases funded the planning stages of the Phase IIa and extension studies (1U34AR068616-01.
  • To date, ReveraGen has worked through public-private partnerships to develop other aspects of the vamorolone development program: 
    • The European Comission is funding the Phase IIb study through Europe (667078 - VISION DMD - RIA)
    • PPMD ($750,000) and Foundation to Eradicate Duchenne ($250,000) are co-funding the Glossary Link 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 study?

  • The Phase IIa clinical trial has begun, and will enroll approximately 48 boys with Duchenne who have never taken steroids for Duchenne and who are ages 4, 5, or 6 at study entry.

 

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

  • The Phase IIa study will include an extension study. Those who opt to enter the Phase IIa and Phase IIa extension study will have a 2 week drug treatment, 2+ week drug tapering period, and then 6 month extension study on drug. An additional 2-year extension study is also available.

 

Where does this study take place?

  • The Phase IIa clinical trials in boys with Duchenne will take place at sites of the Cooperative International Neuromuscular Research Group (CINRG) network in the USA, Canada, Australia, Sweden, United Kingdom, and Sweden.

 

How many study visits are necessary?

  • Phase IIa = approximately 6 visits over 1-2 months; Phase IIa extension = approximately 9 visits.

 

Can visits be done locally?

  • Visits must be done at one of the participating CINRG sites.

 

Will I get paid for participating in this study?

  • Travel 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. 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 (Microsoft Band).

 

Where can I learn more about this study?

  • Information will be posted on the ReveraGen website, as well as www.clinicaltrials.gov (IIa study: NCT02760264 and IIa extension study: NCT02760277).
  • Please check www.DuchenneConnect.org for updates to this FAQ sheet.
  • Contact Andrea Smith at This email address is being protected from spambots. You need JavaScript enabled to view it.

 

(Updated 12/18/2017)