VISION-DMD

Designed to ensure a timely & cost-effective drug development for Duchenne muscular dystrophy

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  • The Project
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  • About DMD
    • What is DMD?
    • Living with Duchenne
    • Current treatment options
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    • Standards of Care for Duchenne muscular dystrophy
  • Study Information
    • What is a clinical study?
    • Phase 2a clinical trial information
    • Phase 2a preliminary study results
    • Phase 2b clinical trial information
    • Phase 2b Preliminary Results
    • MRI – Magnetic Resonance Imaging
  • Drug Development
    • Development of Vamorolone
    • Vamorolone development programme
  • Innovations
    • Return of patient data
    • Bone Health Outcomes
    • Biomarkers
    • Vamorolone study APP for boys
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      • VISION-DMD INFORMATION APP OVERVIEW
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      • Workshop series: Standards of Care
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    • VISION-DMD project Interviews
      • Collaborating for progress on rare diseases: an interview with Dr. Daria Julkowska, Scientific Coordinator of the European Joint Program on Rare Diseases.
      • Returning vamorolone study data to patient caregivers. What, when and how clinical trial data is returned to study participants. An interview with Dr. Laurie Conklin
      • An Interview with Andrea D’Alessandro – Clinical research project manager at TRiNDS
      • Interview with Jana Haberlová – Paediatric Neurologist at University Hospital Motol
      • Interview with Alex Johnson: a parent and patient foundation funder
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Phase 2a clinical trial information

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      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

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This project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement number 667078.

To find out more about the project contact us at vision-dmd_team@ceratium.eu

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