drugset / Trial / NCT03116113
A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using BIIB112
RandomizedSequentialQuadruple-blindTreatment
Summary
The objective of the study is to evaluate the safety, tolerability and efficacy of a single sub-retinal injection of BIIB112 in participants with X-linked retinitis pigmentosa (XLRP).
Timeline
- Start
- 2017-03-08
- Primary completion
- 2020-11-18
- Completion
- 2020-11-18
Outcome
Missed primary endpoint
paper The primary microperimetry end point was not met. PMID 38423215 ↗
registry analysis (superiority test); Part 2: Untreated Group vs Part 2: BIIB112 Low Dose; p =0.3181; Fisher's Exact-Boschloo test NCT03116113 ↗
release “While the Phase 2/3 XIRIUS study did not meet its primary endpoint, data indicated positive trends in several prespecified secondary endpoints” biogen.com ↗
Publications
- Results Cehajic-Kapetanovic J, Xue K, Martinez-Fernandez de la Camara C, Nanda A, Davies A, Wood LJ, Salvetti AP, Fischer MD, Aylward JW, Barnard AR, Jolly JK, Luo E, Lujan BJ, Ong T, Girach A, Black GCM, Gregori NZ, Davis JL, Rosa PR, Lotery AJ, Lam BL, Stanga PE, MacLaren RE. Initial results from a first-in-human gene therapy trial on X-linked retinitis pigmentosa caused by mutations in RPGR. Nat Med. 2020 Mar;26(3):354-359. doi: 10.1038/s41591-020-0763-1. Epub 2020 Feb 24.
- Raji S, Taylor LJ, Josan AS, MacLaren RE, Cehajic-Kapetanovic J. Early-Onset Cone Photoreceptor Degeneration Is Associated With High Myopia in RPGR-Related Retinal Dystrophy. J Ophthalmol. 2025 Jun 11;2025:4244740. doi: 10.1155/joph/4244740. eCollection 2025.
- Lam BL, Pennesi ME, Kay CN, Panda S, Gow JA, Zhao G, MacLaren RE; XIRIUS Study Group. Assessment of Visual Function with Cotoretigene Toliparvovec in X-Linked Retinitis Pigmentosa in the Randomized XIRIUS Phase 2/3 Study. Ophthalmology. 2024 Sep;131(9):1083-1093. doi: 10.1016/j.ophtha.2024.02.023. Epub 2024 Feb 28.
- von Krusenstiern L, Liu J, Liao E, Gow JA, Chen G, Ong T, Lotery AJ, Jalil A, Lam BL, MacLaren RE; XIRIUS Part 1 Study GroupXOLARIS Study Group. Changes in Retinal Sensitivity Associated With Cotoretigene Toliparvovec in X-Linked Retinitis Pigmentosa With RPGR Gene Variations. JAMA Ophthalmol. 2023 Mar 1;141(3):275-283. doi: 10.1001/jamaophthalmol.2022.6254.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | cotoretigene toliparvovec | Gene therapy (AAV / viral vector) | 5e+10 vg | Other |
| Subject | cotoretigene toliparvovec | Gene therapy (AAV / viral vector) | 2.5e+11 vg | Other |