sepofarsen: Clinical Trials

1 active not recruiting, 1 completed, 1 recruiting, 1 unknown. 3 Phase 2, 3 Phase 3, 1 Phase 1.

By phase and status

Phase	Trials	Status
Phase 2	3	Active not recruiting 1, Completed 1, Unknown 1
Phase 3	3	Active not recruiting 1, Recruiting 1, Unknown 1
Phase 1	1	Completed 1

Follow sepofarsen: new trials by email Literature Review of sepofarsen trials Research Report on sepofarsen

Trials newest first · largest first

Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.

Trial	Phase	Status	Sponsor	Enrollment	Start	Updated
Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION) sepofarsen, Placebo IVT - Leber Congenital Amaurosis 10, Blindness	Phase 3	Recruiting	Laboratoires Thea	32	2025-06-04	2026-10-21
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. sepofarsen - Leber Congenital Amaurosis 10, Blindness	Phase 2 / Phase 3	Unknown	ProQR Therapeutics	15	2021-03-23	2022-03-25
A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE) sepofarsen, Sham - Leber Congenital Amaurosis 10, Blindness	Phase 2 / Phase 3	Active not recruiting	ProQR Therapeutics	36	2019-04-04	2022-03-17
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 QR-110 - Leber's Congenital Amaurosis	Phase 1 / Phase 2	Completed	Laboratoires Thea	11	2017-10-16	2024-10-15

Conditions studied

Meckel Syndrome, Type 4 (4)  ·  Blindness (3)  ·  Eye Abnormalities (3)  ·  Eye Diseases (3)  ·  Leber Congenital Amaurosis (3)  ·  Leber Congenital Amaurosis 10 (3)  ·  Neurologic Manifestations (3)  ·  Optic Atrophy, Hereditary, Leber (3)  ·  Retinal Diseases (3)  ·  Sensation Disorders (3)  ·  Vision Disorders (3)  ·  Eye Diseases, Hereditary (2)  ·  Retinal Degeneration (1)  ·  Retinal Dystrophies (1)

Sponsors

Laboratoires Thea (2)  ·  ProQR Therapeutics (2)

Countries

Belgium (4)  ·  Brazil (3)  ·  Canada (3)  ·  Germany (3)  ·  Netherlands (3)  ·  United Kingdom (3)  ·  United States (3)  ·  France (2)  ·  Italy (2)  ·  Australia (1)  ·  Spain (1)

Results publications

PMID 30559420, PMID 31215818, PMID 33795869, PMID 35379979

NIH funding

2017 (1), 2018 (1)