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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using BIIB112
NCTID
NCT03116113
(View at clinicaltrials.gov)
Description
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).
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Development Status
Inactive
Indication
X-Linked Retinitis Pigmentosa
Disease Ontology Term
DOID:0110414
Compound Name
BIIB112
Compound Alias
Cotoretigene toliparvovec
Compound Description
AAV8.coRPGR
Sponsor
Biogen
Funder Type
Industry
Recruitment Status
Completed
Enrollment Count
50
Results Posted
View Results
Therapy Information
Target Gene/Variant
RPGR
Therapy Type
Gene transfer
Therapy Route
In-vivo
Mechanism of Action
Functional gene replacement
Route of Administration
Subretinal
Drug Product Type
Viral vector
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
AAV8
Editor Type
Dose 1
Dose escalation: 5E10 gp/ml - 5E12 gp/ml (6 levels)
Dose 2
Expansion doses: 5E10 vg/eye, 2.5E11 vg/eye
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2017-03-29
Completion Date
2020-11-18
Last Update
2024-01-18
Participation Criteria
Eligible Age
>=10 Years
Standard Ages
Child, Adult, Older adult
Sexes Eligible for Study
MALE
Locations
No.of Trial Sites
8
Locations
United States,United Kingdom
Regulatory Information
Has US IND
True
FDA Designations
Recent Updates
Phase 2/3 study did not meet its primary endpoint, Company suspended development in 2021
Resources/Links
Clinical Publications
Assessment of Visual Function with Cotoretigene Toliparvovec in X-Linked Retinitis Pigmentosa in the Randomized XIRIUS Phase 2/3 Study
Initial results from a first-in-human gene therapy trial on X-linked retinitis pigmentosa caused by mutations in RPGR
News and Press Releases
SEC Form 10-K: BIOGEN INC FY 2021
Preclinical Publications
Codon-Optimized RPGR Improves Stability and Efficacy of AAV8 Gene Therapy in Two Mouse Models of X-Linked Retinitis Pigmentosa
Protocol
Statistical Analysis Plan
Clinical Trial Protocol