VISION-DMD

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

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  • The Project
    • Summary
    • Project Objectives
    • Project Consortium
    • Contact us
  • About DMD
    • What is DMD?
    • Living with Duchenne
    • Current treatment options
    • Duchenne boy’s stories
    • DMD Community
    • 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
    • Patient Group Funders
    • What is Venture Philanthropy?
  • Resources
    • Relevant publications
      • VISION-DMD INFORMATION APP OVERVIEW
      • VISION-DMD Thought paper
    • VISION-DMD Workshops
      • Workshop series: Standards of Care
      • Workshop series: Biomarkers and Imaging
    • Webinars and presentations
    • Press Releases
    • 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
    • VISION-DMD Project Newsletters
      • VISION-DMD newsletter sign up
    • VISION-DMD Poster presentations
    • Our privacy policy
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Phase 2b Preliminary Results

During period 1 (first 24 weeks) of the study recruited boys received either vamorolone, prednisolone or placebo. The last patient last visit has now been completed in the first treatment period of the Phase 2b study. The aim of this pivotal placebo and active controlled 24-week study is to demonstrate the efficacy and safety of vamorolone administered orally at doses of 2.0 mg/kg/day and 6.0 mg/kg/day versus prednisone 0.75 mg/kg/day and placebo. The efficacy outcome  measures are motor function and strength outcomes with Time to Stand test (TTSTAND) as the primary study  endpoint. Additional analyses compare safety  and  tolerability  between  the  vamorolone  dose  groups,  placebo  and  prednisone.

On March 3rd 2021 Santhera and ReveraGen announced the completion of the first 6-month period of this pivotal VISION-DMD trial (see Press Release). Topline 6-month data are expected in Q2-2021, paving the way for a US NDA submission in Q1-2022. Vamorolone has been granted Orphan Drug status in the US and in Europe, and has received Fast Track and Rare Pediatric Disease designations by the US FDA and Promising Innovative Medicine (PIM) status from the UK MHRA.

During treatment period 2 (week 28 to week 48) another 24 weeks of continued study will be conducted.   All boys will receive vamorolone at either 2.0 or 6.0 mg/kg/day. The patients who previously received prednisone or placebo have been randomized and switched to one of the two doses of vamorolone. This second treatment period is designed to evaluate the persistence of the drug effect in the longer term. In addition to efficacy, the study aims to confirm the differentiated safety and favourable tolerability profile of vamorolone. The results will demonstrate the potential of vamorolone to offer an alternative to current standard of care.

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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 [email protected]

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