Study to evaluate the efficacy, safety, tolerability and the extent to which the study drug is distributed in the body of multiple doses of QR-110 in subjects with LCA10 compared to a sham procedure. The study will be double-masked, randomized and controlled which means that both patients and study staff will not know and cannot influence who receives which treatment (study drug of sham). : 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

Medienart:

Klinische Studie

Erscheinungsjahr:

2023

Erschienen:

2023

Enthalten in:

WHO International Clinical Trials Registry Platform - (2023) vom: 21. Juni Zur Gesamtaufnahme - year:2023

Sprache:

Englisch

Links:

Volltext [kostenfrei]

Themen:

610
Medical Condition: Leber’s Congenital Amaurosis (LCA) due to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene MedDRA version: 20.0Level: PTClassification code 10070667Term: Leber's congenital amaurosisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11]
Phase: Phase 2
Recruitment Status: Authorised-recruitment may be ongoing or finished
Study Type: Interventional

Anmerkungen:

WHO International Clinical Trials Registry Platform (https://www.who.int/ictrp/en/), First posted: 24-01-2019, Last updated: 2023-06-29

ICTRP ID:

EUCTR2018-003501-25-GB
PQ-110-003
2018-003501-25-DE

Förderinstitution / Projekttitel:

PPN (Katalog-ID):

CTG004739345