Trials / NCT05085964
An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa
Terminated Phase 2 RNA / antisense
Why it stopped: The sponsor decided to terminate the study early
- Sponsor
- Laboratoires Thea
- Treatment
- RNA antisense oligonucleotide for intravitreal injection
- Gene
- USH2A
- Conditions
- Retinitis Pigmentosa, Usher Syndrome Type 2
- Ages
- 12 Years to no upper limit
- Enrollment
- 21 participants
- Started
- 2021-09-16
- Main results expected
- 2022-10-18
- Results posted
- Yes
- Record last updated
- 2024-08-07
What the study is testing
PQ-421a-002 (Helia) is an open-label, extension study to evaluate the safety, tolerability and efficacy of QR 421a (ultevursen) administered via intravitreal (IVT) injection in one or both eyes, in subjects ≥ 12 years of age with RP due to mutations in exon 13 of the USH2A gene, for an anticipated period of 24 months, or until provision of continued treatment by other means is available, provided the subject's benefit-risk determination remains positive.
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