Test clinico
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).
Malattia
Tipo di malattia
Studi clinici pediatrici
Riconoscimento dei farmaci orfani
Non
Tipo di paziente
Corsi
Bambini
Inclusione/Esclusione
Finanziamento
industria
Membri coinvolti
Altri investigatori

Pr Bart LEROY
Belgio

Pr Elfride DE BAERE
Belgio

Pr Carel HOYNG
Olanda

Pr Dr Markus Preising
Germania

Pr Katarina Stingl
Germania

Pr Isabelle Audo
Francia

Pr Hélène Dollfus
Francia

Dr Ingeborgh VAN DEN BORN
Olanda
Sito di indagine da parte di un membro dell'ERN EYE
HCP: investigatori principali

Ghent University Hospital, Ghent, Belgium
Adress
Corneel Heymanslaan 10
9000 Ghent
Belgium

Radboud university medical center, Nijmegen, Netherlands
Adress
Geert Grooteplein Zuid 10
6525 GA Nijmegen
Netherlands

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

RÉFÉRET National Hospital of Ophthalmology of Quinze-Vingts, Paris, France
Adress
28 Rue de Charenton
75012 Paris
France

The Rotterdam Eye Hospital, Rotterdam, Netherlands
Adress
Schiedamse Vest 180
3011 BH Rotterdam
Netherlands

Erasmus MC: University Medical Center Rotterdam, Netherlands
Adress
Doctor Molewaterplein 40
3015 GD Rotterdam
Netherlands