Recruitment is now complete for this trial. See preliminary results here
The Phase IIa clinical study of vamorolone (VBP15) in the treatment of boys with Duchenne muscular dystrophy (DMD), includes a clinical study followed by a IIa extension study. These studies will be conducted at sites of the Cooperative International Neuromuscular Research Group (CINRG) network within the United States, Canada, United Kingdom, Sweden, Israel, and Australia and started recruitment in July 2016.
The IIa clinical trial is an open label, multiple ascending dose study of vamorolone (VBP15) on 48 steroid naïve boys aged from 4 years old to less than 7 years old. Boys will be enrolled in four dose cohorts sequentially – 0.25 mg/kg, 0.75 mg/kg, 2.0 mg/kg and 6.0 mg/kg vamorolone, dosing once a day for 2 weeks followed by 2 weeks of follow up off treatment.
The primary outcome of this trial is
- acute safety
- tolerability
- pharmacokinetics of vamorolone administration.
The Phase IIa extension study will be conducted with the participants from the IIa study, at the same dose level and will continue for 24 weeks.
The primary outcome of the IIa extension trial is
- long term safety
- tolerability
- efficacy as measured by the Time to Stand test
- Safety as measured by body mass index
Study sites:
United States
- Duke University, Durham, North Carolina.
- Contact Karen Cornett 9119-684-1143 [email protected]
- Ann & Robert H. Lurie Children’s Hospital, Chicago, Illinois.
- Contact Thereas Oswald 312-227-3019 [email protected]
- University of Florida, Gainesville, Florida.
- Contact Katie Dickinson 352-273-7573 [email protected]
- Nemours Children’s Hospital, Orlando, Florida.
- Contact Kristin McCrary 407-650-7175 [email protected]
- University of California Davis, California.
- Contact Colleen Anthonisen 916-734-4307 [email protected]
- University of Texas Southwestern Medical Center, Dallas.
- Contact Maria Martinez 214-648-5606 [email protected]
Canada
- Alberta Children’s Hospital, Calgary, Alberta, Canada.
- Contact Tiffany Haig 403-955-3192 [email protected]
Israel
- Schneider Children’s Medical Center, Petah Tikvah.
- Contact Ori Raz +972-3-9253559 [email protected]
United Kingdom
- Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle.
- Contact Becky Davis +44 (0) 191-241-8649 [email protected]
Sweden
- Queen Silvia Children’s Hospital, Gothenburg.
- Contact Mar Tulinus +46 (0)31-3434780 [email protected]
Australia
- Royal Children’s Hospital, Melbourne.
- Contact Jemima Mitchell 03 9936 6157 [email protected]
- Sydney Children’s Hospital Westmead.
- Contact Eshwini Tadiyal (02) 02 9845 3048 [email protected]
Click here for more information including eligibility and inclusion/exclusion criteria
