Leber Congenital Amaurosis 10 research hub
4 indexed studies · 2 currently recruiting
- active not recruitingPHASE2, PHASE3
A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)
This study looks at a new treatment called sepofarsen for a rare eye condition called Leber Congenital Amaurosis 10 (LCA10), which causes blindness. We want to see if it helps improve sight, how safe it is, and how well people tolerate it.
United States · Belgium · Brazil - active not recruiting
Rate of Progression of PCDH15-Related Retinal Degeneration in Usher Syndrome 1F
This study looks at how a specific eye condition, linked to Usher Syndrome 1F and a gene called PCDH15, changes over time. Its goal is to help scientists better measure these changes, which will speed up the development of new treatments for this type of sight loss.
United States · Canada · France - recruitingPHASE2
Study to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene
This study is looking at a new treatment called Ultevursen for a type of eye problem called Retinitis Pigmentosa (RP) caused by specific gene changes. It's in the second stage of testing, focusing on how safe and well people tolerate the treatment when it's given as an injection into the eye.
United States · Belgium · Brazil - recruitingPHASE3
Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)
This study is testing a new medicine called sepofarsen for a rare eye condition called Leber Congenital Amaurosis Type 10 (LCA10). It aims to see if sepofarsen can improve vision safely in people with this condition.
United States · Belgium · Brazil