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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
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