Study to Evaluate QR-110 in Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene

PHASE1/PHASE2CompletedINTERVENTIONAL

Enrollment

Participants

Timeline

Start Date

October 16, 2017

Primary Completion Date

October 2, 2019

Study Completion Date

October 2, 2019

Conditions

Leber's Congenital Amaurosis

Interventions

DRUG

QR-110

RNA antisense oligonucleotide for intravitreal injection

Trial Locations (3)

Scheie Eye Institute, University of Pennsylvania, Philadelphia

University of Iowa, Iowa City

Ghent University Hospital and Ghent University, Ghent