A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)

PHASE2/PHASE3Active, not recruitingINTERVENTIONAL
Enrollment

36

Participants

Timeline

Start Date

April 4, 2019

Primary Completion Date

January 31, 2022

Study Completion Date

March 31, 2023

Conditions
Leber Congenital Amaurosis 10BlindnessLeber Congenital AmaurosisVision DisordersSensation DisordersNeurologic ManifestationsEye DiseasesEye Diseases, HereditaryEye Disorders CongenitalRetinal Disease
Interventions
DRUG

sepofarsen

RNA antisense oligonucleotide for intravitreal injection

OTHER

Sham

Sham-Procedure (no experimental drug administered)

Trial Locations (14)

35392

Justus-Liebig Universität - Department of Ophthalmology, Giessen

52242

University of Iowa, Iowa City

67091

Hospital Civil de Strasbourg, Strasbourg

72076

University of Tuebingen - Inst. for Ophthalmic Research, Tübingen

75012

Centre de maladies rares CHNO des Quinze Vingt, Paris

80131

Eye Clinic University of Campania Luigi Vanvitelli, Naples

30150270

INRET Clínica/ Santa Casa de Misericórdia de Belo Horizonte, Belo Horizonte

Unknown

Universitair Ziekenhuis Gent (UZ), Ghent

04023-062

Federal University of São Paulo - Hospital São Paulo (UNIFESP-HSP), São Paulo

M5G 2L3

The Hospital for Sick Children - SickKids, Toronto

H4A 3J1

McGill University Health Centre - Centre for Innovative Medicine, Montreal

1105 AZ

Amsterdam University Medica Center - Locatie AMC, Amsterdam

3011 BH

Het Oogziekenhuis Rotterdam, Rotterdam

EC1V 2PD

Moorfields Eye Hospital - NHS Foundation Trust, London

Sponsors

Lead Sponsor

All Listed Sponsors
lead

ProQR Therapeutics

INDUSTRY

NCT03913143 - A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE) | Biotech Hunter | Biotech Hunter