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BRIGHTEN

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Clinical Trial

NCT04855045 Unknown
BRIGHTEN

An Open-Label, Dose Escalation and Double-Masked, Randomized, Controlled Study to Evaluate the Safety and Tolerability of Sepofarsen in Pediatric Subjects G (p.Cys998X) Mutation.

Phase 2/3
Interventional

Disease

Disease type

Other

Paediatric clinical trials

Patient type

Adult

Children

Inclusion / Exclusion

Opening date

23/03/2023

Closing date

31/12/2023

Inclusion criteria :

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Exclusion criteria :

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Funding

private

Members involved

Others investigators

Pr Bart LEROY

Representative

Belgium

Genetic Diagnostics (TWG6), Low Vision Daily Life and Patients Groups (TWG5), National Integration (TWG9), Research (TWG8), Retinal Rare Eye Diseases (WG1)
Working Group Leader
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Pr Dr Markus Preising

Representative

Germany

Pediatric Ophthalmology Rare Diseases (WG3), Research (TWG8), Retinal Rare Eye Diseases (WG1)
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Pr Katarina Stingl

Representative

Germany

Low Vision Daily Life and Patients Groups (TWG5), Registries & Epidemiology (TWG7), Research (TWG8), Retinal Rare Eye Diseases (WG1)
Working Group Leader
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Pr Francesca Simonelli

Representative

Italy

Genetic Diagnostics (TWG6), National Integration (TWG9), Registries & Epidemiology (TWG7), Retinal Rare Eye Diseases (WG1)
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ERN EYE member investigating site

HCP : Principal investigators

Universitätsklinikum Gießen und Marburg GmbH, Gießen, Germany

Adress

Klinik und Poliklinik für Augenheilkunde Friedrichstr. 18

35392 Gießen

Germany

Centre for Ophthalmology, University Eye Hospital, Tübingen, Germany

Adress

Department für Augenheilkunde Tübingen Forschungsinstitut für Augenheilkunde Elfriede-Aulhorn-Str. 7

72076 Tübingen

Germany