Recruitment is now complete for this trial
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 k.cornett@duke.edu
- Ann & Robert H. Lurie Children’s Hospital, Chicago, Illinois.
- Contact Thereas Oswald 312-227-3019 TOswald@luriechildrens.org
- University of Florida, Gainesville, Florida.
- Contact Katie Dickinson 352-273-7573 k.dickinson@ufl.edu
- Nemours Children’s Hospital, Orlando, Florida.
- Contact Kristin McCrary 407-650-7175 Kristin.mccrary@nemours.org
- University of California Davis, California.
- Contact Colleen Anthonisen 916-734-4307 canthonisen@ucdavis.edu
- University of Texas Southwestern Medical Center, Dallas.
- Contact Maria Martinez 214-648-5606 MariaR.Martinez@UTSouthwestern.edu
Canada
- Alberta Children’s Hospital, Calgary, Alberta, Canada.
- Contact Tiffany Haig 403-955-3192 tiffany.haig@albertahealthservices.ca
Israel
- Schneider Children’s Medical Center, Petah Tikvah.
- Contact Ori Raz +972-3-9253559 orira@clalit.org.il
United Kingdom
- Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle.
- Contact Becky Davis +44 (0) 191-241-8649 beckydavis@newcastle.ac.uk
Sweden
- Queen Silvia Children’s Hospital, Gothenburg.
- Contact Mar Tulinus +46 (0)31-3434780 mar.tulinus@gu.se
Australia
- Royal Children’s Hospital, Melbourne.
- Contact Jemima Mitchell 03 9936 6157 Jemima.mitchell@rch.org.au
- Sydney Children’s Hospital Westmead.
- Contact Eshwini Tadiyal (02) 02 9845 3048 eshwini.tadiyal@health.nsw.gov.au
Click here for more information including eligibility and inclusion/exclusion criteria
Trial contact: Andrea Smith, smithal7@upmc.edu; 1-412-383-7207
