A Randomised, Open Label, Outcomes-Assessor Masked, Prospective, Parallel Controlled Group, Phase 3 Clinical Trial of Retinal Gene Therapy for Choroideremia Using an Adeno-Associated Viral Vector (AAV2) Encoding Rab Escort Protein 1 (REP1)

Biogen1 site in 1 country169 target enrollmentDecember 11, 2017

ConditionsChoroideremia

Overview

Phase: Phase 3
Intervention: Not specified
Conditions: Choroideremia
Sponsor: Biogen
Enrollment: 169
Locations: 1
Primary Endpoint: Percentage of Participants With a ≥15 -Letter Improvement From Baseline in Best Corrected Visual Acuity (BCVA) at Month 12 as Measured by the Early Treatment of Diabetic Retinopathy Study (ETDRS) Chart
Status: Completed
Last Updated: 2 years ago

Overview Investigators Eligibility Criteria Outcomes Sites Similar Trials

Overview

Brief Summary

The objective of the study is to evaluate the efficacy and safety of a single sub-retinal injection of BIIB111 in participants with choroideremia (CHM).

Detailed Description

This study was previously posted by NightstaRx Ltd. In October 2020, sponsorship of the trial was transferred to Biogen.

Registry

clinicaltrials.gov

Start Date

December 11, 2017

End Date

December 1, 2020

Last Updated

2 years ago

Study Type

Interventional

Study Design

Parallel

Sex

Male

Investigators

Sponsor

Biogen

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Are willing and able to give informed consent for participation in the study.
•Have a documented genetically-confirmed diagnosis of CHM.
•Have active disease clinically visible within the macular region in the study eye.
•Have a BCVA of 34-73 ETDRS letters (equivalent to worse than or equal to 6/12 or 20/40 Snellen acuity, but better than or equal to 6/60 or 20/200 Snellen acuity) in the study eye.

Exclusion Criteria

•Have a history of amblyopia in the eligible eye.
•Have had previous intraocular surgery performed in the study eye within 3 months of Visit
•Have any other significant ocular or non-ocular disease/disorder which, in the opinion of the investigator, may either put the participants at risk because of participation in the study, or may influence the results of the study, or the participant's ability to participate in the study.
•Have participated in another research study involving an investigational product in the past 12 weeks or received a gene/cell-based therapy at any time previously.
•Are unwilling to use barrier contraception methods, or abstain from sexual intercourse, for a period of 3 months, if treated with AAV2-REP
•NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Outcomes

Primary Outcomes

Percentage of Participants With a ≥15 -Letter Improvement From Baseline in Best Corrected Visual Acuity (BCVA) at Month 12 as Measured by the Early Treatment of Diabetic Retinopathy Study (ETDRS) Chart

Time Frame: Month 12

BCVA was assessed for both eyes using the ETDRS visual acuity (VA) chart. BCVA test should be performed prior to pupil dilation, and distance refraction should be carried out before BCVA is measured. Initially, letters are read at a distance of 4 meters from the chart. If \<20 letters are read at 4 meters, testing at 1 meter should be performed. The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). Percentage of participants with a ≥15 -letter improvement from baseline in BCVA at Month 12 was reported for both eyes.

Secondary Outcomes

Change From Baseline in the Choroidal Thickness Using SD-OCT at Month 12 in Study Eye(Baseline, Month 12)
Change From Baseline in BCVA at Month 12 Reported as Letters Measured by the ETDRS Chart in Study Eye(Baseline, Month 12)
Percentage of Participants With a ≥10 -Letter Improvement From Baseline in BCVA at Month 12 Measured by the ETDRS Chart(Month 12)
Percentage of Participants With No Decrease From Baseline in BCVA or a Decrease From Baseline in BCVA of <5 ETDRS Letters at Month 12 Measured by the EDRS Chart(Month 12)
Change From Baseline in BCVA at Months 4 and 8 Reported as Letters Measured by the ETDRS Chart(Baseline, Months 4 and 8)
Change From Baseline in the Foveal Subfield Thickness Using Spectral Domain Optical Coherence Tomography (SD-OCT) at Month 12 in Study Eye(Baseline, Month 12)
Change From Baseline in Total Area of Preserved Autofluorescence (AF) at Month 12 in Study Eye(Baseline, Month 12)
Change From Baseline in Distance From Foveal Center to Nearest Border of Preserved AF at Month 12 in Study Eye(Baseline, Month 12)
Change From Baseline in the Total Macular Volume Using SD-OCT at Month 12 in Study Eye(Baseline, Month 12)
Change From Baseline in the Central Horizontal Ellipsoid Width Using SD-OCT at Month 12 in Study Eye(Baseline, Month 12)
Change From Baseline in the Central Ellipsoid Area Using SD-OCT at Month 12 in Study Eye(Baseline, Month 12)
Change From Baseline in the Mean Retinal Sensitivity Using Microperimetry at Month 12 in Study Eye(Baseline, Month 12)
Change From Baseline in the Bivariate Contour Ellipse Area 63% Using Microperimetry at Month 12 in Study Eye(Baseline, Month 12)
Change From Baseline in the Bivariate Contour Ellipse Area 95% Using Microperimetry at Month 12 in Study Eye(Baseline, Month 12)
Change From Baseline in Reading Speed Test at Month 12 in Study Eye(Baseline, Month 12)
Change From Baseline in the 25-Item Visual Function Questionnaire (VFQ-25) Composite Scores at Month 12 in Study Eye(Baseline, Month 12)
Change From Baseline in Contrast Sensitivity Score at Month 12 in Study Eye(Baseline, Month 12)
Change From Baseline in Colour Vision Total Error Score at Month 12 in Study Eye(Baseline, Month 12)