CORDIS
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CORDIS

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Clinical development of Enzyme Replacement Therapy in alpha-Mannosidosis patients using recombinant human enzyme

Project information

Grant agreement ID: 261331

Status

Closed project

  • Start date

    1 October 2010

  • End date

    31 March 2014

Funded under:

FP7-HEALTH

  • Overall budget:

    € 8 028 469,20

  • EU contribution

    € 5 887 150

Coordinated by:

CHRISTIAN-ALBRECHTS-UNIVERSITAET ZU KIEL

Germany

Objective

Alpha-Mannosidosis is a rare Lysosomal Storage Disorder with a worldwide incidence of about 1:500 000. This orphan and devastating disease is caused by the deficiency of the lysosomal alpha-mannosidase (LAMAN) which is responsible for the intralysosomal degradation of mannosyl linked oligosaccharides. To date no causative treatment for alpha-Mannosidosis is available and since most of the children are born healthy, an early initiated therapy could contribute to a normal development. To develop an efficient therapy for this disease the collaborative research project EURAMAN and HUE-MAN were initiated within FP5 and FP6, respectively. Within these collaborative networks, European scientists, clinicians and the industry successfully i) developed an efficient pre-clinical Enzyme Replacement Therapy (ERT) protocol using recombinant human (rh) LAMAN in a mouse model for alpha-Mannosidosis, ii) built up a database collecting patient data and iii) defined clinical endpoints for the future clinical trials in alpha-Mannosidosis patients by an European wide natural history study. Furthermore, the HUE-MAN network developed the conditions for a large-scale production of the recombinant enzyme and the way is now paved for the first clinical trials in man, which we aim to realize within FP7. The main objectives of the ALPHA-MAN project are i) the performance of efficient clinical trials (phase I-III) in alpha-Mannosidosis patients, ii) a better understanding of the pathophysiology and the mechanism of how the recombinant enzyme enters the cells of the central nervous system by performing ERT in a newly generated immuntolerant alpha-Mannosidosis mouse model, which allows chronic treatment and iii) the determination of the minimal effective dose with chronic treatment in the immuntolerant mice.

Coordinator

CHRISTIAN-ALBRECHTS-UNIVERSITAET ZU KIEL

Address

Olshausenstrasse 40
24118 Kiel

Germany

Activity type

Higher or Secondary Education Establishments

EU Contribution

€ 593 128

Administrative Contact

Paul Saftig (Prof.)

Participants (11)

Zymenex AS

Denmark

EU Contribution

€ 329 732

REGION HOVEDSTADEN

Denmark

EU Contribution

€ 2 826 623

KATHOLIEKE UNIVERSITEIT LEUVEN

Belgium

EU Contribution

€ 299 360

THE UNIVERSITY OF MANCHESTER

United Kingdom

EU Contribution

€ 37 282

UNIVERSITAETSMEDIZIN DER JOHANNES GUTENBERG-UNIVERSITAET MAINZ

Germany

EU Contribution

€ 589 434

HOSPICES CIVILS DE LYON

France

EU Contribution

€ 255 561

UNIVERSITETET I TROMSOE - NORGES ARKTISKE UNIVERSITET

Norway

EU Contribution

€ 97 400

TEKNOLOGISK INSTITUT

Denmark

EU Contribution

€ 187 636

INSTYTUT POMNIK CENTRUM ZDROWIA DZIECKA

Poland

EU Contribution

€ 11 172

LARIX APS

Denmark

EU Contribution

€ 543 958

UNILABAS AS

Denmark

EU Contribution

€ 115 864

Project information

Grant agreement ID: 261331

Status

Closed project

  • Start date

    1 October 2010

  • End date

    31 March 2014

Funded under:

FP7-HEALTH

  • Overall budget:

    € 8 028 469,20

  • EU contribution

    € 5 887 150

Coordinated by:

CHRISTIAN-ALBRECHTS-UNIVERSITAET ZU KIEL

Germany

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