Trials / NCT03780257
Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene
Completed Phase 1 / Phase 2 RNA / antisense
- Sponsor
- ProQR Therapeutics
- Treatment
- QR-421a; Sham-procedure (dose cohort 1&2 only)
- Gene
- USH2A
- Conditions
- Retinitis Pigmentosa, Usher Syndrome Type 2, Deaf Blind, Retinal Disease, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Vision Disorders
- Ages
- 18 Years to no upper limit
- Enrollment
- 20 participants
- Started
- 2019-03-06
- Main results expected
- 2021-10-14
- Results posted
- Not yet
- Record last updated
- 2022-04-20
What the study is testing
The purpose of this study is to evaluate the safety and tolerability of QR-421a administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene.
Where it runs
Canada (1)
- Centre for Innovative Medicine, Department of Paediatric Surgery, Montreal Children's Hospital at the McGill University Health Centre, Montreal
France (2)
- Hôpital Gui de Chauliac - CHRU de Montpellier - Maladies Sensorielles Génétique, Montpellier
- Centre de maladies rares CHNO des Quinze Vingts, Paris
United States (4)
- Center for Clinical Research Operations, Massachusetts Eye and Ear, Boston, Massachusetts
- University of Michigan, Kellogg Eye Center, Ann Arbor, Michigan
- Casey Eye Institute, Oregon Health & Science University, Portland, Oregon
- Retina Foundation of the Southwest, Dallas, Texas