A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using BIIB112
NCT ID: NCT03116113
Last Updated: 2024-01-18
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE1/PHASE2
50 participants
INTERVENTIONAL
2017-03-08
2020-11-18
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
SEQUENTIAL
TREATMENT
QUADRUPLE
Study Groups
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Part 1: BIIB112 Dose 1
Participants will receive a single Dose 1 of BIIB112 by sub-retinal injection on Day 0.
BIIB112
Administered as specified in the treatment arm.
Part 1: BIIB112 Dose 2
Participants will receive a single Dose 2 of BIIB112 by sub-retinal injection on Day 0.
BIIB112
Administered as specified in the treatment arm.
Part 1: BIIB112 Dose 3
Participants will receive a single Dose 3 of BIIB112 by sub-retinal injection on Day 0.
BIIB112
Administered as specified in the treatment arm.
Part 1: BIIB112 Dose 4
Participants will receive a single Dose 4 of BIIB112 by sub-retinal injection on Day 0.
BIIB112
Administered as specified in the treatment arm.
Part 1: BIIB112 Dose 5
Participants will receive a single Dose 5 of BIIB112 by sub-retinal injection on Day 0.
BIIB112
Administered as specified in the treatment arm.
Part 1: BIIB112 Dose 6
Participants will receive a single Dose 6 of BIIB112 by sub-retinal injection on Day 0.
BIIB112
Administered as specified in the treatment arm.
Part 2: BIIB112 High Dose
Participants will receive a single high dose of BIIB112 by sub-retinal injection.
BIIB112
Administered as specified in the treatment arm.
Part 2: BIIB112 Low Dose
Participants will receive a single low dose of BIIB112 by sub-retinal injection.
BIIB112
Administered as specified in the treatment arm.
Part 2: Untreated Group
Participants will receive no intervention to allow for a controlled comparison.
No interventions assigned to this group
Interventions
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BIIB112
Administered as specified in the treatment arm.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Participants with genetically confirmed diagnosis of XLRP (with RPGR mutation).
* Participant with active disease clinically visible within the macular region in both eyes.
Part 2:
\- Participant with mean total retinal sensitivity in the study eye as assessed by microperimetry ≥ 0.1 dB and ≤8 dB.
Exclusion Criteria
* Participant with history of amblyopia in either eye.
* Participated in a gene therapy trial previously or a clinical trial with an investigational drug in the past 12 weeks or received a gene/cell-based therapy at any time previously.
10 Years
MALE
No
Sponsors
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Biogen
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director
Role: STUDY_DIRECTOR
Biogen
Locations
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Research Site
Gainesville, Florida, United States
Research Site
Miami, Florida, United States
Research Site
Portland, Oregon, United States
Research Site
Philadelphia, Pennsylvania, United States
Research Site
Dallas, Texas, United States
Research Site
Manchester, , United Kingdom
Research Site
Oxford, , United Kingdom
Research Site
Southampton, , United Kingdom
Countries
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References
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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.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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2016-003852-60
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
274RP101
Identifier Type: -
Identifier Source: org_study_id
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