NCT04855045View on ClinicalTrials.gov

An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene.

PHASE2/PHASE3UnknownINTERVENTIONAL
Enrollment

15

Participants

Timeline

Start Date

March 23, 2021

Primary Completion Date

December 31, 2023

Study Completion Date

December 31, 2023

Conditions
Leber Congenital Amaurosis 10BlindnessLeber Congenital AmaurosisVision DisordersSensation DisordersNeurologic ManifestationsEye DiseasesEye Disorders CongenitalRetinal DiseaseRetinal DegenerationRetinal Dystrophies
Interventions
DRUG

sepofarsen

RNA antisense oligonucleotide for intravitreal injection

Trial Locations (9)

9000

RECRUITING

Universitair Ziekenhuis Gent (UZ), Ghent

35392

RECRUITING

Justus-Liebig Universität - Department of Ophthalmology, Giessen

72076

NOT_YET_RECRUITING

University of Tübingen - Institute for Ophthalmic Research, Tübingen

Unknown

RECRUITING

INRET Clinica e Centro de Pesquisa / Santa Casa BH, Belo Horizonte

RECRUITING

Federal University of Sao Paulo - Hospital Sao Paulo, São Paulo

NOT_YET_RECRUITING

University of Alberta, Edmonton

NOT_YET_RECRUITING

Eye Clinic University of Campania Liugi Vanvitelli, Naples

1105 AZ

RECRUITING

Amsterdam University Medica Center - Locatie AMC, Amsterdam

EC1V 2PD

NOT_YET_RECRUITING

Moorfields Eye Hospital - NHS Foundation Trust, London

Sponsors

Lead Sponsor

ProQR Therapeutics
All Listed Sponsors
lead

ProQR Therapeutics

INDUSTRY

NCT04855045 - An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene. | Biotech Hunter | Biotech Hunter