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VISION-DMD - Phase 2 Clinical Trials of VBP15: An Innovative Steroid-like Intervention on Duchenne Muscular Dystrophy

Objective

VISION-DMD aims to advance clinical development of the orphan drug VBP15 as a new therapy to revolutionise care for all patients with Duchenne muscular dystrophy (DMD) by 2020, in line with IRDiRC goals. DMD is an incurable, rare muscle wasting disease; boys progressively weaken, lose ambulation and death occurs by early adulthood. Corticosteroids (CS) are widely recognised to increase muscle strength and delay disease progression but global acceptance as standard of care is very variable due to severe side effects. VBP15 is an innovative steroid-like drug designed to retain or better CS efficacy and improve membrane stabilization with reduced or no side effects. VBP15 will increase the therapeutic window to slow disease progression and improve quality of life and lifespan for all DMD patients.
Building on positive preclinical and Phase 1 results funded by government grants and international patient groups and based on FDA and EMA advice, VISION-DMD proposes a Phase 2 registration directed clinical programme aimed at an affordable therapy: Phase 2a will study the safety and tolerability of ascending doses of VBP15 in ambulant DMD boys; Phase 2b will demonstrate the efficacy and safety of two doses of VBP15 in young ambulant DMD boys. Both studies will be followed by extension studies for long term safety and efficacy data collection leading to cumulative exposure of up to 2100 drug months. The project proposes the Time to Stand Test as a highly relevant and reliable primary endpoint. Innovative exploratory serum biomarkers and novel wide scale MRI techniques will be used to investigate the VBP15 pharmacodynamics and the effect on muscle cellular pathology. VBP15 will meet the unmet need for better treatment for DMD with widespread acceptance and potentially be used in combination with stratified therapies as they are developed. The Consortium links the leading networks TREAT-NMD and CINRG with ECRIN-ERIC, for trial delivery and regulatory undertakings in Europe/US

Call for proposal

H2020-PHC-2015-two-stage
See other projects for this call

Coordinator

UNIVERSITY OF NEWCASTLE UPON TYNE
Address
Kings Gate
NE1 7RU Newcastle Upon Tyne
United Kingdom
Activity type
Higher or Secondary Education Establishments
EU contribution
€ 2 918 331,29

Participants (7)

Reveragen Biopharma Limited
United Kingdom
EU contribution
€ 0
Address
The Oaks, 3 Village Road
CH48 3JN West Kirby
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
STICHTING UNITED PARENT PROJECTS MUSCULAR DYSTROPHY
Netherlands
EU contribution
€ 339 500
Address
Koninginnelaan 69
3905 GG Veenendaal
Activity type
Other
ECRIN EUROPEAN CLINICAL RESEARCH INFRASTRUCTURE NETWORK

Participation ended

France
EU contribution
€ 336 365,87
Address
5 Rue Watt
75013 Paris
Activity type
Research Organisations
CERATIUM LIMITED
United Kingdom
EU contribution
€ 467 343,75
Address
The Haven 20 Burlingham Avenue
CH48 8AP West Kirby
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
FAKULTNI NEMOCNICE V MOTOLE
Czechia
EU contribution
€ 168 625
Address
V Uvalu 84
150 06 Praha 5
Activity type
Research Organisations
Children's Research Institute (CRI)

Participation ended

United States
EU contribution
€ 0
Address
Michigan Avenue 111
20010 Washington, Dc
Activity type
Research Organisations
REVERAGEN BIOPHARMA INC.
United States
EU contribution
€ 1 769 834,09
Address
155 Gibbs Street Suite 433
20850 Rockville Md
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)