Clinical Trial
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).
Disease
Disease type
Paediatric clinical trials
Orphan drug recognition
No
Patient type
Adult
Children
Inclusion / Exclusion
Funding
industry
Members involved
Others investigators
Pr Bart LEROY
Belgium
Pr Elfride DE BAERE
Belgium
Pr Carel HOYNG
Netherlands
Pr Dr Markus Preising
Germany
Pr Katarina Stingl
Germany
Pr Isabelle Audo
France
Pr Hélène Dollfus
France
ERN EYE member investigating site
HCP : Principal investigators
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