Phase 3
N=169
Efficacy and Safety of BIIB111 for the Treatment of Choroideremia
Choroideremia
Bottom Line
View on ClinicalTrials.gov: NCT03496012 ↗Enrolled (actual)
169
Serious AEs
18.3%
Results posted
Dec 2023
Primary outcome: Primary: 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 — 0; 2.9; 4.6; 0 percentage of participants — p==0.354
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- BIIB111 (Genetic)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- Male
- Sponsor
- Biogen
- Primary completion
- Dec 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY 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 |
0; 2.9; 4.6; 0; 0; 3.1 | =0.354 |
| SECONDARY Change From Baseline in BCVA at Month 12 Reported as Letters Measured by the ETDRS Chart in Study Eye |
-2.3; -1.5; -0.3 | — |
| SECONDARY Percentage of Participants With a ≥10 -Letter Improvement From Baseline in BCVA at Month 12 Measured by the ETDRS Chart |
1.6; 17.6; 13.8; 0; 8.8; 6.2 | — |
| SECONDARY 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 |
67.7; 70.6; 83.1; 75.8; 85.3; 90.8 | — |
| SECONDARY Change From Baseline in BCVA at Months 4 and 8 Reported as Letters Measured by the ETDRS Chart |
0.2; -0.4; 0.6; -1.1; 0.9; 2.0 | — |
| SECONDARY Change From Baseline in Total Area of Preserved Autofluorescence (AF) at Month 12 in Study Eye |
-0.3872; -0.5413; -0.5136 | — |
| SECONDARY Change From Baseline in Distance From Foveal Center to Nearest Border of Preserved AF at Month 12 in Study Eye |
21.7796; 23.6171; 21.1660 | — |
| SECONDARY Change From Baseline in the Foveal Subfield Thickness Using Spectral Domain Optical Coherence Tomography (SD-OCT) at Month 12 in Study Eye |
-6.3152; -19.8654; -15.7993 | — |
| SECONDARY Change From Baseline in the Total Macular Volume Using SD-OCT at Month 12 in Study Eye |
-0.0895; -0.2876; -0.2088 | — |
| SECONDARY Change From Baseline in the Central Horizontal Ellipsoid Width Using SD-OCT at Month 12 in Study Eye |
-81.9178; -116.3811; -170.7214 | — |
| SECONDARY Change From Baseline in the Central Ellipsoid Area Using SD-OCT at Month 12 in Study Eye |
-0.2382; -0.4229; -0.3440 | — |
| SECONDARY Change From Baseline in the Choroidal Thickness Using SD-OCT at Month 12 in Study Eye |
-5.4897; -7.9579; -4.6371 | — |
| SECONDARY Change From Baseline in the Mean Retinal Sensitivity Using Microperimetry at Month 12 in Study Eye |
-0.3443; -0.2965; -0.5015 | — |
| SECONDARY Change From Baseline in the Bivariate Contour Ellipse Area 63% Using Microperimetry at Month 12 in Study Eye |
-3.2161; -3.3771; -2.5555 | — |
| SECONDARY Change From Baseline in the Bivariate Contour Ellipse Area 95% Using Microperimetry at Month 12 in Study Eye |
-10.1986; -14.6926; -11.6863 | — |
| SECONDARY Change From Baseline in Contrast Sensitivity Score at Month 12 in Study Eye |
-0.0595; -0.0130; -0.0386 | — |
| SECONDARY Change From Baseline in Colour Vision Total Error Score at Month 12 in Study Eye |
35.0003; 43.5915; 54.3777 | — |
| SECONDARY Change From Baseline in Reading Speed Test at Month 12 in Study Eye |
-149.5590; -122.1333; -127.4706 | — |
| SECONDARY Change From Baseline in the 25-Item Visual Function Questionnaire (VFQ-25) Composite Scores at Month 12 in Study Eye |
-2.6971; 1.7236; 0.7964 | — |
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).
Eligibility Criteria
Key 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.
Key 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 1.
- 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-REP1.
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Data sourced from ClinicalTrials.gov (NCT03496012). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.