Phase 2
N=18
A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using BIIB112
X-Linked Retinitis Pigmentosa
Bottom Line
View on ClinicalTrials.gov: NCT03116113 ↗Enrolled (actual)
18
Serious AEs
28.0%
Results posted
Jan 2024
Primary outcome: Primary: Part 1: Number of Participants With Dose-Limiting Toxicities (DLTs) — 0; 0; 0; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- BIIB112 (Biological)
- Age
- Pediatric, Adult, Older Adult · 10+ yrs
- Sex
- Male
- Sponsor
- Biogen
- Primary completion
- Nov 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Part 1: Number of Participants With Dose-Limiting Toxicities (DLTs) |
0; 0; 0; 0; 0; 0 | — |
| PRIMARY Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) |
3; 3; 3; 3; 3; 3 | — |
| PRIMARY Part 2: Percentage of Study Eyes With ≥7 Decibels (dB) Improvement From Baseline at ≥5 Points Out of the 16 Central Loci Points of the 10-2 Grid Assessed by Macular Integrity Assessment (MAIA) Microperimetry |
22.2; 37.5; 25.0 | =0.3181 |
| PRIMARY Part 2: Number of Participants With TEAEs |
5; 11; 12 | — |
| SECONDARY Part 1: Percentage of Study Eyes With ≥7 dB Improvement From Baseline at ≥5 Points Out of the 16 Central Loci Points of the 10-2 Grid Assessed by MAIA Microperimetry |
0.0; 0.0; 100.0; 33.3; 33.3; 33.3 | — |
| SECONDARY Part 1: Percentage of Study Eyes With ≥7 dB Improvement From Baseline at ≥5 Points Out of the 68 Loci Points of the 10-2 Grid Assessed by MAIA Microperimetry |
0.0; 0.0; 100.0; 33.3; 33.3; 33.3 | — |
| SECONDARY Part 1: Change From Baseline in Mean Sensitivity of the 16 Central Loci Points Assessed by MAIA Microperimetry |
0.33; 1.78; 3.35; 6.52; 13.46; 7.75 | — |
| SECONDARY Part 1: Change From Baseline in Mean Sensitivity of the 68 Central Loci Points Assessed by MAIA Microperimetry |
-0.19; 0.20; 0.21; 2.22; 8.87; 4.26 | — |
| SECONDARY Part 1: Change From Baseline in Mean Best Corrected Visual Acuity (BCVA) Reported as Letters |
27.67; 49.67; 64.33; 63.67; 70.67; 67.33 | — |
| SECONDARY Part 1: Change From Baseline in Mean Low Luminance Visual Acuity (LLVA) Reported as Letters |
41.00; 34.33; 58.00; 41.67; 37.50; 37.67 | — |
| SECONDARY Part 1: Percentage of Eyes With a ≥15 Letters Increase From Baseline for BCVA |
0.0; 0.0; 0.0; 0.0; 0.0; 0.0 | — |
| SECONDARY Part 1: Percentage of Eyes With a ≥15 Letters Increase From Baseline for LLVA |
50.0; 0.0; 0.0; 33.3; 50.0; 0.0 | — |
| SECONDARY Part 1: Percentage of Eyes With a ≥10 Letters Increase From Baseline for BCVA |
0.0; 0.0; 0.0; 0.0; 0.0; 0.0 | — |
| SECONDARY Part 1: Percentage of Eyes With a ≥10 Letters Increase From Baseline for LLVA |
50.0; 0.0; 0.0; 66.7; 50.0; 0.0 | — |
| SECONDARY Part 1: Percentage of Eyes With a ≥5 Letters Increase From Baseline for BCVA |
33.3; 0.0; 0.0; 33.3; 0.0; 66.7 | — |
| SECONDARY Part 1: Percentage of Eyes With a ≥5 Letters Increase From Baseline for LLVA |
50.0; 0.0; 66.7; 66.7; 50.0; 33.3 | — |
| SECONDARY Part 1: Percentage of Eyes With a ≥15 Letters Loss From Baseline for BCVA |
0.0; 0.0; 0.0; 0.0; 0.0; 0.0 | — |
| SECONDARY Part 1: Percentage of Eyes With a ≥15 Letters Loss From Baseline for LLVA |
0.0; 0.0; 0.0; 33.3; 0.0; 0.0 | — |
| SECONDARY Part 1: Percentage of Eyes With a ≥10 Letters Loss From Baseline for BCVA |
0.0; 0.0; 0.0; 0.0; 0.0; 33.3 | — |
| SECONDARY Part 1: Percentage of Eyes With a ≥10 Letters Loss From Baseline for LLVA |
0.0; 0.0; 0.0; 33.3; 0.0; 0.0 | — |
| SECONDARY Part 1: Percentage of Eyes With a ≥5 Letters Loss From Baseline for BCVA |
0.0; 0.0; 0.0; 33.3; 33.3; 33.3 | — |
| SECONDARY Part 1: Percentage of Eyes With a ≥5 Letters Loss From Baseline for LLVA |
0.0; 0.0; 0.0; 33.3; 0.0; 33.3 | — |
| SECONDARY Part 1: Percentage of Eyes With Change From Baseline > -5 Letters for BCVA |
100.0; 100.0; 100.0; 66.7; 66.7; 66.7 | — |
| SECONDARY Part 1: Percentage of Eyes With Change From Baseline > -5 Letters for LLVA |
100.0; 100.0; 100.0; 66.7; 100.0; 66.7 | — |
| SECONDARY Part 1: Change From Baseline in Central Ellipsoid Area |
0.140; 2.750; 0.137; 0.193; 8.327; 3.107 | — |
| SECONDARY Part 1: Change From Baseline in Central Horizontal Ellipsoid Width |
162.67; 1375.33; 248.00; 462.33; 2411.33; 745.67 | — |
| SECONDARY Part 1: Change From Baseline in Fundus Autofluoroscence- Mean Total Area of Preserved Autofluoroscence |
27.050; 22.910; 50.640; -1.820; -2.420; -8.620 | — |
| SECONDARY Part 1: Change From Baseline in Fundus Autofluoroscence- Mean Distance From Foveal Center (FC) to Nearest Border of Preserved Autofluoroscence |
2394.50; -39.00; 3018.00; 361.00; 2853.00; 2609.00 | — |
| SECONDARY Part 1: Change From Baseline in Mean Volume of 30-Degree Hill of Vision |
2.73; 5.07; 5.17; -2.14; -1.10; 0.33 | — |
| SECONDARY Part 1: Change From Baseline in Mean Volume of Full Field Hill of Vision |
15.77; 18.31; 23.41; -0.86; -4.91; 1.29 | — |
| SECONDARY Part 1: Change From Baseline in Contrast Sensitivity (CS) Score |
0.300; 0.533; 0.900; 0.883; 1.267; 1.067 | — |
| SECONDARY Part 2: Percentage of Study Eyes With ≥7 dB Improvement From Baseline at ≥5 Points Out of the 16 Central Loci Points of the 10-2 Grid Assessed by MAIA Microperimetry |
22.2; 30.0; 40.0; 22.2; 30.0; 50.0 | — |
| SECONDARY Part 2: Percentage of Study Eyes With ≥7 dB Improvement From Baseline at ≥5 Points Out of the 68 Loci Points of the 10-2 Grid Assessed by MAIA Microperimetry |
33.3; 60.0; 40.0; 22.2; 80.0; 80.0 | — |
| SECONDARY Part 2: Change From Baseline in Mean Sensitivity of the 16 Central Loci Points of the 10-2 Grid Assessed by MAIA Microperimetry |
6.78; 7.39; 7.38; 9.56; 9.09; 8.98 | — |
| SECONDARY Part 2: Change From Baseline in Mean Sensitivity of the 68 Central Loci Points of the 10-2 Grid Assessed by MAIA Microperimetry |
2.15; 2.46; 3.84; 3.32; 3.34; 4.47 | — |
| SECONDARY Part 2: Change From Baseline in BCVA Reported as Letters |
68.78; 65.90; 68.20; 72.89; 68.50; 68.80 | — |
| SECONDARY Part 2: Change From Baseline in Mean LLVA Reported as Letters |
50.22; 39.30; 47.90; 54.78; 49.50; 51.60 | — |
| SECONDARY Part 2: Percentage of Eyes With a ≥15 Letter Increase From Baseline for BCVA |
0.0; 0.0; 10.0; 0.0; 0.0; 0.0 | — |
| SECONDARY Part 2: Percentage of Eyes With a ≥15 Letter Increase From Baseline for LLVA |
0.0; 40.0; 20.0; 0.0; 0.0; 10.0 | — |
| SECONDARY Part 2: Percentage of Eyes With a ≥10 Letter Increase From Baseline for BCVA |
0.0; 0.0; 10.0; 0.0; 0.0; 10.0 | — |
| SECONDARY Part 2: Percentage of Eyes With a ≥10 Letter Increase From Baseline for LLVA |
11.1; 40.0; 30.0; 0.0; 0.0; 0.0 | — |
| SECONDARY Part 2: Percentage of Eyes With a ≥5 Letter Increase From Baseline for BCVA |
0.0; 60.0; 20.0; 0.0; 10.0; 40.0 | — |
| SECONDARY Part 2: Percentage of Eyes With a ≥5 Letter Increase From Baseline for LLVA |
22.2; 60.0; 40.0; 22.2; 30.0; 30.0 | — |
| SECONDARY Part 2: Percentage of Eyes With a ≥15 Letters Loss From Baseline for BCVA |
0.0; 0.0; 8.3; 0.0; 0.0; 0.0 | — |
| SECONDARY Part 2: Percentage of Eyes With a ≥15 Letters Loss From Baseline for LLVA |
0.0; 0.0; 33.3; 0.0; 18.2; 0.0 | — |
| SECONDARY Part 2: Percentage of Eyes With a ≥10 Letters Loss From Baseline for BCVA |
0.0; 9.1; 8.3; 0.0; 0.0; 0.0 | — |
| SECONDARY Part 2: Percentage of Eyes With a ≥10 Letters Loss From Baseline for LLVA |
11.1; 0.0; 33.3; 0.0; 18.2; 0.0 | — |
| SECONDARY Part 2: Percentage of Eyes With a ≥5 Letters Loss From Baseline for BCVA |
11.1; 9.1; 33.3; 11.1; 9.1; 0.0 | — |
| SECONDARY Part 2: Percentage of Eyes With a ≥5 Letters Loss From Baseline for LLVA |
33.3; 18.2; 41.7; 33.3; 36.4; 0.0 | — |
| SECONDARY Part 2: Percentage of Eyes With Change From Baseline >-5 Letters for BCVA |
88.9; 90.0; 70.0; 88.9; 90.0; 100.0 | — |
| SECONDARY Part 2: Percentage of Eyes With Change From Baseline >-5 Letters for LLVA |
66.7; 80.0; 70.0; 66.7; 60.0; 100.0 | — |
| SECONDARY Part 2: Change From Baseline in Volume of 30-Degree Hill of Vision Assessed by Octopus 900 |
3.61; 3.58; 5.70; 3.05; 3.71; 6.23 | — |
| SECONDARY Part 2: Change From Baseline in Volume of Full Field Hill of Vision Assessed by Octopus 900 |
7.26; 17.51; 19.48; 7.05; 16.33; 21.08 | — |
Summary
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).
Eligibility Criteria
Key Inclusion Criteria
Part 1:
- 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.
Key exclusion Criteria
Parts 1 and 2:
- 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.
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Data sourced from ClinicalTrials.gov (NCT03116113). 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.