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ILLUMINATE

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Clinical Trial

NCT03913143 Active, Not recruiting
ILLUMINATE

A double-masked, randomized, controlled, multiple-dose study to evaluate the efficacy, safety, tolerability and systemic exposure of QR-110 in Subjects with Leber’s Congenital Amaurosis (LCA) due to c.2991+1655A>G mutation (p.Cys998X) in the CEP290 gene (Illuminate).

Phase 2/3
Interventional

Disease

Disease type

Paediatric clinical trials

Orphan drug recognition

No

Patient type

Adult

Children

Inclusion / Exclusion

Opening date

04/04/2019

Closing date

04/12/2021

Inclusion criteria :

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Exclusion criteria :

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Funding

industry

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 Elfride DE BAERE

Substitute

Belgium

Genetic Diagnostics (TWG6), Retinal Rare Eye Diseases (WG1)
Working Group Leader
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Pr Carel HOYNG

Representative

Netherlands

Genetic Diagnostics (TWG6), Registries & Epidemiology (TWG7), Research (TWG8), Retinal Rare Eye Diseases (WG1)
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PD Dr Markus Preising

Representative

Germany

Pediatric Ophthalmology Rare Diseases (WG3), Research (TWG8), Retinal Rare Eye Diseases (WG1)
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Dr 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 Isabelle Audo

Representative

France

Genetic Diagnostics (TWG6), Low Vision Daily Life and Patients Groups (TWG5), National Integration (TWG9), Neuro-Ophthalmology Rare Diseases (WG2), Pediatric Ophthalmology Rare Diseases (WG3), Registries & Epidemiology (TWG7), Research (TWG8), Retinal Rare Eye Diseases (WG1)
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Pr Hélène Dollfus

Representative

France

Anterior Segment Rare Eye Diseases (WG4), CPMS & Digital Medecine (TWG10), Epag, ERN-EYE Coordinating committee, Genetic Diagnostics (TWG6), Low Vision Daily Life and Patients Groups (TWG5), National Integration (TWG9), Neuro-Ophthalmology Rare Diseases (WG2), Pediatric Ophthalmology Rare Diseases (WG3), Registries & Epidemiology (TWG7), Research (TWG8), Retinal Rare Eye Diseases (WG1)
Working Group Leader
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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

CARGO, Hôpitaux Universitaires de Strasbourg, France

Adress

Hôpital Civil -Bâtiment du Centre de Recherche en Biomédecine de Strasbourg (CRBS),1 Rue Eugène Boeckel

67000 STRASBOURG

France