Study to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene

Phase 2 Active not recruiting 81 enrolled Laboratoires Thea Sepul Bio · collab

RandomizedParallel-groupDouble-blindTreatment

Summary

The purpose of this Phase 2b study is to evaluate the safety and tolerability of ultevursen administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene. This is a multicenter Double-masked, Randomized, Sham-controlled study which will enroll 81 subjects.

Timeline

Start: 2024-12-11
Primary completion: 2028-07
Completion: 2028-07

Publications

Stephenson KAJ, Dhanji SR, Kolawole OU, Gregory-Evans CY, Gregory-Evans K. Ethnic disparities in inherited retinal degenerations. Can J Ophthalmol. 2025 Dec;60(6):e869-e878. doi: 10.1016/j.jcjo.2025.04.007. Epub 2025 Jun 6.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Ultevursen	Antisense oligonucleotide	60 ug	Intravitreal

Study to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene

Summary

Timeline

Publications

Drugs

Indications