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Designing a clinical trial to evaluate the safety and efficacy of oral soraprazan in Stargardt Disease

Designing a clinical trial to evaluate the safety and efficacy of oral soraprazan in Stargardt Disease
Designing a clinical trial to evaluate the safety and efficacy of oral soraprazan in Stargardt Disease
Purpose: To date there are no treatments available for Stargardt disease (STGD). The investigational drug soraprazan has demonstrated (in pre-clinical studies) the ability to remove lipofuscin, one of the hallmarks in STGD pathogenesis, from Retinal Pigment Epithelium (RPE) cells. Soraprazan was originally designed to treat gastro-esophageal reflux disease and successfully completed phase I and phase II clinical trials, where safety and tolerability was already demonstrated. The aim is to start a phase II trial to evaluate the efficacy of soraprazan in reducing the amount of lipofuscin in RPE cells of subjects with STGD.
Methods: An international consortium was formed consisting of 5 investigator sites (2 in Netherlands and 1 in each of Germany, UK, and Italy), a Contract Research Organisation, and a start-up company as a sponsor (Katairo). A draft protocol and a business plan were fully costed and timelines
0146-0404
5704
Hoyng, Carel
112a6c5f-c5da-464f-b3e3-48a40a09a93a
Lotery, Andrew
5ecc2d2d-d0b4-468f-ad2c-df7156f8e514
Stingl, Katarina
52653c1c-ce76-4108-b5c9-f205f83080f0
Boon, Camiel J.F.
0656f60f-744b-4548-908c-2b9e0aac098f
Parodi, Maurizio
bb7cc543-bd24-4256-a068-02ff2fc06782
Dhooge, Patty
9affbfd0-0443-4f73-8c90-ecb4f2e18b72
Peters, Tobias
95522fed-4a2d-4b97-9135-b47d5b4f876e
Klein, Wolfgang
7fcbe274-0cfb-4b07-af26-ba25bae95dd3
Fsadni, Mario G.
e18cdbe3-e7c2-4d81-9ecb-a0d93a1ba48e
Müller, Hans
3cd86913-0147-49db-bc73-ecc416df5e3d
Jungmann, Oliver
a753d873-4f97-443a-9b3e-832bc5775c25
Hoyng, Carel
112a6c5f-c5da-464f-b3e3-48a40a09a93a
Lotery, Andrew
5ecc2d2d-d0b4-468f-ad2c-df7156f8e514
Stingl, Katarina
52653c1c-ce76-4108-b5c9-f205f83080f0
Boon, Camiel J.F.
0656f60f-744b-4548-908c-2b9e0aac098f
Parodi, Maurizio
bb7cc543-bd24-4256-a068-02ff2fc06782
Dhooge, Patty
9affbfd0-0443-4f73-8c90-ecb4f2e18b72
Peters, Tobias
95522fed-4a2d-4b97-9135-b47d5b4f876e
Klein, Wolfgang
7fcbe274-0cfb-4b07-af26-ba25bae95dd3
Fsadni, Mario G.
e18cdbe3-e7c2-4d81-9ecb-a0d93a1ba48e
Müller, Hans
3cd86913-0147-49db-bc73-ecc416df5e3d
Jungmann, Oliver
a753d873-4f97-443a-9b3e-832bc5775c25

Hoyng, Carel, Lotery, Andrew, Stingl, Katarina, Boon, Camiel J.F., Parodi, Maurizio, Dhooge, Patty, Peters, Tobias, Klein, Wolfgang, Fsadni, Mario G., Müller, Hans and Jungmann, Oliver (2019) Designing a clinical trial to evaluate the safety and efficacy of oral soraprazan in Stargardt Disease. Investigative Ophthalmology & Visual Science, 60 (9), 5704.

Record type: Meeting abstract

Abstract

Purpose: To date there are no treatments available for Stargardt disease (STGD). The investigational drug soraprazan has demonstrated (in pre-clinical studies) the ability to remove lipofuscin, one of the hallmarks in STGD pathogenesis, from Retinal Pigment Epithelium (RPE) cells. Soraprazan was originally designed to treat gastro-esophageal reflux disease and successfully completed phase I and phase II clinical trials, where safety and tolerability was already demonstrated. The aim is to start a phase II trial to evaluate the efficacy of soraprazan in reducing the amount of lipofuscin in RPE cells of subjects with STGD.
Methods: An international consortium was formed consisting of 5 investigator sites (2 in Netherlands and 1 in each of Germany, UK, and Italy), a Contract Research Organisation, and a start-up company as a sponsor (Katairo). A draft protocol and a business plan were fully costed and timelines

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More information

Published date: 22 July 2019

Identifiers

Local EPrints ID: 433976
URI: http://eprints.soton.ac.uk/id/eprint/433976
ISSN: 0146-0404
PURE UUID: a04fcf69-498d-476d-aca8-b6099358e6a7
ORCID for Andrew Lotery: ORCID iD orcid.org/0000-0001-5541-4305

Catalogue record

Date deposited: 09 Sep 2019 16:30
Last modified: 10 Nov 2021 03:08

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Contributors

Author: Carel Hoyng
Author: Andrew Lotery ORCID iD
Author: Katarina Stingl
Author: Camiel J.F. Boon
Author: Maurizio Parodi
Author: Patty Dhooge
Author: Tobias Peters
Author: Wolfgang Klein
Author: Mario G. Fsadni
Author: Hans Müller
Author: Oliver Jungmann

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