- 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 - recruiting
Universal Rare Gene Study: A Registry and Natural History Study of Retinal Dystrophies Associated With Rare Disease-Causing Genetic Variants
This study looks at rare genetic eye conditions called retinal dystrophies. It aims to understand how these conditions develop and affect people over time, using genetic information and regular check-ups to improve knowledge and future treatments.
United States · Australia · Belgium - ongoing, recruitingPhase I and Phase II (Integrated)- First administration to humans
Safety and Efficacy of a Unilateral Subretinal Administration of HORA PDE6B in Patients with Retinitis Pigmentosa Harbouring Mutations in the PDE6B Gene Leading to a Defect in PDE6ß Expression
This study is testing a new gene therapy for people with retinitis pigmentosa caused by a specific faulty gene. It's looking at how safe the treatment is and if it helps improve vision. Researchers are giving the treatment into one eye.
France - active not recruitingPHASE3
Oral N-acetylcysteine for Retinitis Pigmentosa
This study is testing if a medicine called N-acetylcysteine (NAC) can help slow down vision loss in people with an eye condition called Retinitis Pigmentosa (RP). RP causes changes in your genes that lead to gradual vision loss, especially night vision and side vision.
United States · Austria · Canada