sepofarsen

DRUG3 trials

Sponsors

ProQR Therapeutics, Laboratoires Thea

Conditions

BlindnessEye DiseasesEye Diseases, HereditaryEye Disorders CongenitalLeber Congenital AmaurosisLeber Congenital Amaurosis 10Neurologic ManifestationsNeurological Manifestations

Phase 2

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

Active, not recruitingNCT03913143

ProQR TherapeuticsBlindness, Eye Diseases, Eye Diseases, Hereditary +7

Start: 2019-04-04End: 2023-03-31Updated: 2022-03-17

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.

ProQR TherapeuticsBlindness, Eye Diseases, Eye Disorders Congenital +8

Start: 2021-03-23End: 2023-12-31Target: 15Updated: 2022-03-25

Phase 3

Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)

RecruitingNCT06891443

Laboratoires TheaBlindness, Eye Diseases, Eye Diseases, Hereditary +7

Start: 2025-06-04End: 2028-10-01Target: 32Updated: 2026-06-25

Related Papers

Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b/2 trial

Nature Medicine2022-04-04121 citations