Summary
Soraprazan – a new regenerative therapy for Stargardt’s disease
Abnormal accumulation of lipofuscin in the RPE cells is a hallmark in Stargardt's disease. As lipofuscin cannot be removed from the RPE cells in the eyes neither spontaneously nor with existing therapies, the accumulation of lipofuscin in the RPE cells of the eyes results in the degradation of the RPE cells and consequently in worsening of visual acuity and could lead to blindness. Soraprazan was able to show removal of lipofuscin in RPE cells. Aim of the phase II trial in this project is to evaluate savety and efficacy or orally taken Soraprazan treatment compared to placebo.
Abnormal accumulation of lipofuscin in the RPE cells is a hallmark in Stargardt's disease. As lipofuscin cannot be removed from the RPE cells in the eyes neither spontaneously nor with existing therapies, the accumulation of lipofuscin in the RPE cells of the eyes results in the degradation of the RPE cells and consequently in worsening of visual acuity and could lead to blindness. Soraprazan was able to show removal of lipofuscin in RPE cells. Aim of the phase II trial in this project is to evaluate savety and efficacy or orally taken Soraprazan treatment compared to placebo.
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| Web resources: | https://cordis.europa.eu/project/id/779317 |
| Start date: | 01-12-2017 |
| End date: | 30-11-2021 |
| Total budget - Public funding: | 5 792 425,00 Euro - 5 792 425,00 Euro |
Cordis data
Original description
Soraprazan – a new regenerative therapy for Stargardt’s diseaseAbnormal accumulation of lipofuscin in the RPE cells is a hallmark in Stargardt's disease. As lipofuscin cannot be removed from the RPE cells in the eyes neither spontaneously nor with existing therapies, the accumulation of lipofuscin in the RPE cells of the eyes results in the degradation of the RPE cells and consequently in worsening of visual acuity and could lead to blindness. Soraprazan was able to show removal of lipofuscin in RPE cells. Aim of the phase II trial in this project is to evaluate savety and efficacy or orally taken Soraprazan treatment compared to placebo.
Status
CLOSEDCall topic
SC1-PM-11-2016-2017Update Date
26-10-2022
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Organisations
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| EBERHARD KARLS UNIVERSITAET TUEBINGEN (EKUT) (Coördinator)
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| ACADEMISCH ZIEKENHUIS LEIDEN (LUMC)
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| KATAIRO GMBH
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| OSPEDALE SAN RAFFAELE SRL
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| SMERUD MEDICAL RESEARCH GERMANY GMBH
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| SMERUD MEDICAL RESEARCH INTERNATIONAL AS
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| SMERUD MEDICAL RESEARCH UK LIMITED
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| STICHTING KATHOLIEKE UNIVERSITEIT (SKU/RU)
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| STICHTING RADBOUD UNIVERSITAIR MEDISCH CENTRUM
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| UNIVERSITATSKLINIKUM BONN
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| UNIVERSITY HOSPITALS SOUTHAMPTON NHS FOUNDATION TRUST
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| UNIVERSITY OF SOUTHAMPTON