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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-2014-2015

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Sub call

H2020-PHC-2015-two-stage

Coordinator

UNIVERSITY OF NEWCASTLE UPON TYNE
Net EU contribution
€ 2 918 331,29
Address
Kings Gate
NE1 7RU Newcastle Upon Tyne
United Kingdom

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Region
North East (England) Northumberland and Tyne and Wear Tyneside
Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 0,00

Participants (9)

Reveragen Biopharma Limited
United Kingdom
Net EU contribution
€ 0,00
Address
The Oaks, 3 Village Road
CH48 3JN West Kirby

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SME

The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.

Yes
Region
North West (England) Merseyside Wirral
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Non-EU contribution
€ 0,00
STICHTING UNITED PARENT PROJECTS MUSCULAR DYSTROPHY
Netherlands
Net EU contribution
€ 339 500,00
Address
Koninginnelaan 69
3905 GG Veenendaal

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Region
West-Nederland Utrecht Utrecht
Activity type
Other
Non-EU contribution
€ 0,00
ECRIN EUROPEAN CLINICAL RESEARCH INFRASTRUCTURE NETWORK

Participation ended

France
Net EU contribution
€ 336 365,87
Address
5 Rue Watt
75013 Paris

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Region
Ile-de-France Ile-de-France Paris
Activity type
Research Organisations
Non-EU contribution
€ 0,00
Third-party

Legal entity other than a subcontractor which is affiliated or legally linked to a participant. The entity carries out work under the conditions laid down in the Grant Agreement, supplies goods or provides services for the action, but did not sign the Grant Agreement. A third party abides by the rules applicable to its related participant under the Grant Agreement with regard to eligibility of costs and control of expenditure.

LUXEMBOURG INSTITUTE OF HEALTH

Participation ended

Luxembourg
Net EU contribution
€ 0,00
Address
1a Rue Rue Thomas Edison
1445 Strassen

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Region
Luxembourg Luxembourg Luxembourg
Activity type
Research Organisations
Non-EU contribution
€ 0,00
Third-party

Legal entity other than a subcontractor which is affiliated or legally linked to a participant. The entity carries out work under the conditions laid down in the Grant Agreement, supplies goods or provides services for the action, but did not sign the Grant Agreement. A third party abides by the rules applicable to its related participant under the Grant Agreement with regard to eligibility of costs and control of expenditure.

Masarykova univerzita

Participation ended

Czechia
Net EU contribution
€ 0,00
Address
Zerotinovo Namesti 9
601 77 Brno

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Region
Jihovýchod Jihomoravský kraj
Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 0,00
CERATIUM LIMITED
United Kingdom
Net EU contribution
€ 467 343,75
Address
The Haven 20 Burlingham Avenue
CH48 8AP West Kirby

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SME

The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.

Yes
Region
North West (England) Merseyside Wirral
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Non-EU contribution
€ 0,00
FAKULTNI NEMOCNICE V MOTOLE
Czechia
Net EU contribution
€ 168 625,00
Address
V Uvalu 84
150 06 Praha 5

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Region
Praha Hlavní město Praha
Activity type
Research Organisations
Non-EU contribution
€ 0,00
Children's Research Institute (CRI)

Participation ended

United States
Net EU contribution
€ 0,00
Address
Michigan Avenue 111
20010 Washington, Dc

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Activity type
Research Organisations
Non-EU contribution
€ 0,00
REVERAGEN BIOPHARMA INC.
United States
Net EU contribution
€ 1 769 834,09
Address
155 Gibbs Street Suite 433
20850 Rockville Md

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SME

The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.

Yes
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Non-EU contribution
€ 10 858 748,72