Trial Outcomes & Findings for An Open-Label, Phase 1 Clinical Study to Evaluate the Safety and Tolerability of Subcutaneous Elamipretide in Subjects With Intermediate Age-Related Macular Degeneration (NCT NCT02848313)
NCT ID: NCT02848313
Last Updated: 2020-10-20
Results Overview
Change from Baseline in Mean Standard Luminance Best-corrected Visual Acuity (BCVA) at 4 meters, letters from Baseline to Day 7, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24, and Week 28.
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
40 participants
Primary outcome timeframe
Day 0 (Baseline to Day 7, and to Weeks 4, 8, 12, 16, 20, 24, and 28.
Results posted on
2020-10-20
Participant Flow
Participant milestones
| Measure |
High-Risk Drusen (HRD)
The HRD cohort was defined as the presence of either at least 1 large (≥125 μm) druse or multiple medium-size (63-124 μm) drusen in one eye. Subjects had to have 1 eye with intermediate AMD (high-risk drusen \[HRD\] without geographic atrophy \[GA\]).
|
NCGA (Noncentral GA)
The NCGA cohort was defined as evidence of GA with cumulative area ≥1.27 mm2 (approximately 0.5 disc area) by fundus autofluorescence (FAF) that spared the fovea (defined as retinal pigment epithelium and outer retina intact by spectral-domain optical coherence tomography \[SD-OCT\]). Subjects had to have 1 eye with intermediate AMD with noncentral GA \[NCGA\]
|
|---|---|---|
|
Overall Study
STARTED
|
21
|
19
|
|
Overall Study
COMPLETED
|
18
|
15
|
|
Overall Study
NOT COMPLETED
|
3
|
4
|
Reasons for withdrawal
| Measure |
High-Risk Drusen (HRD)
The HRD cohort was defined as the presence of either at least 1 large (≥125 μm) druse or multiple medium-size (63-124 μm) drusen in one eye. Subjects had to have 1 eye with intermediate AMD (high-risk drusen \[HRD\] without geographic atrophy \[GA\]).
|
NCGA (Noncentral GA)
The NCGA cohort was defined as evidence of GA with cumulative area ≥1.27 mm2 (approximately 0.5 disc area) by fundus autofluorescence (FAF) that spared the fovea (defined as retinal pigment epithelium and outer retina intact by spectral-domain optical coherence tomography \[SD-OCT\]). Subjects had to have 1 eye with intermediate AMD with noncentral GA \[NCGA\]
|
|---|---|---|
|
Overall Study
Adverse Event
|
1
|
2
|
|
Overall Study
Lack of Efficacy
|
0
|
1
|
|
Overall Study
Withdrawal by Subject
|
1
|
1
|
|
Overall Study
Lost to follow up
|
1
|
0
|
Baseline Characteristics
An Open-Label, Phase 1 Clinical Study to Evaluate the Safety and Tolerability of Subcutaneous Elamipretide in Subjects With Intermediate Age-Related Macular Degeneration
Baseline characteristics by cohort
| Measure |
High-Risk Drusen (HRD)
n=21 Participants
The HRD cohort was defined as the presence of either at least 1 large (≥125 μm) druse or multiple medium-size (63-124 μm) drusen in one eye. Subjects had to have 1 eye with intermediate AMD (high-risk drusen \[HRD\] without geographic atrophy \[GA\]).
|
NCGA (Noncentral GA)
n=19 Participants
The NCGA cohort was defined as evidence of GA with cumulative area ≥1.27 mm2 (approximately 0.5 disc area) by fundus autofluorescence (FAF) that spared the fovea (defined as retinal pigment epithelium and outer retina intact by spectral-domain optical coherence tomography \[SD-OCT\]). Subjects had to have 1 eye with intermediate AMD with noncentral GA \[NCGA\]
|
Total
n=40 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
70.9 years
STANDARD_DEVIATION 8.54 • n=39 Participants
|
76.0 years
STANDARD_DEVIATION 8.22 • n=41 Participants
|
73.3 years
STANDARD_DEVIATION 8.67 • n=35 Participants
|
|
Sex: Female, Male
Female
|
13 Participants
n=39 Participants
|
11 Participants
n=41 Participants
|
24 Participants
n=35 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=39 Participants
|
8 Participants
n=41 Participants
|
16 Participants
n=35 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=39 Participants
|
1 Participants
n=41 Participants
|
2 Participants
n=35 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
20 Participants
n=39 Participants
|
18 Participants
n=41 Participants
|
38 Participants
n=35 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
|
Race (NIH/OMB)
White
|
21 Participants
n=39 Participants
|
19 Participants
n=41 Participants
|
40 Participants
n=35 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
|
Smoking Status
Never smoker
|
13 Participants
n=39 Participants
|
8 Participants
n=41 Participants
|
21 Participants
n=35 Participants
|
|
Smoking Status
Former smoker
|
8 Participants
n=39 Participants
|
11 Participants
n=41 Participants
|
19 Participants
n=35 Participants
|
|
Smoking Status
Current smoker
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
PRIMARY outcome
Timeframe: Day 0 (Baseline to Day 7, and to Weeks 4, 8, 12, 16, 20, 24, and 28.Population: All participants for whom Standard Luminance BCVA was measured.
Change from Baseline in Mean Standard Luminance Best-corrected Visual Acuity (BCVA) at 4 meters, letters from Baseline to Day 7, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24, and Week 28.
Outcome measures
| Measure |
AMD-High-Risk Drusen (HRD)
n=21 Participants
The HRD cohort was defined as the presence of either at least 1 large (≥125 μm) druse or multiple medium-size (63-124 μm) drusen in one eye. Subjects had to have 1 eye with intermediate AMD (high-risk drusen \[HRD\] without geographic atrophy \[GA\]).
|
AMD-NCGA (Noncentral GA)
n=19 Participants
The NCGA cohort was defined as evidence of GA with cumulative area ≥1.27 mm2 (approximately 0.5 disc area) by fundus autofluorescence (FAF) that spared the fovea (defined as retinal pigment epithelium and outer retina intact by spectral-domain optical coherence tomography \[SD-OCT\]). Subjects had to have 1 eye with intermediate AMD with noncentral GA \[NCGA\]
|
|---|---|---|
|
Change From Baseline in Mean Standard Luminance Best-corrected Visual Acuity (BCVA)
Day 7
|
0.810 letters
Standard Deviation 4.8746
|
0.526 letters
Standard Deviation 4.1281
|
|
Change From Baseline in Mean Standard Luminance Best-corrected Visual Acuity (BCVA)
Week 4
|
2.762 letters
Standard Deviation 5.5759
|
1.833 letters
Standard Deviation 5.0904
|
|
Change From Baseline in Mean Standard Luminance Best-corrected Visual Acuity (BCVA)
Week 8
|
1.950 letters
Standard Deviation 4.4066
|
1.556 letters
Standard Deviation 6.2987
|
|
Change From Baseline in Mean Standard Luminance Best-corrected Visual Acuity (BCVA)
Week 12
|
1.150 letters
Standard Deviation 5.5845
|
1.438 letters
Standard Deviation 5.4156
|
|
Change From Baseline in Mean Standard Luminance Best-corrected Visual Acuity (BCVA)
Week 16
|
2.789 letters
Standard Deviation 5.8933
|
2.813 letters
Standard Deviation 5.5883
|
|
Change From Baseline in Mean Standard Luminance Best-corrected Visual Acuity (BCVA)
Week 20
|
3.842 letters
Standard Deviation 4.1401
|
1.438 letters
Standard Deviation 5.7847
|
|
Change From Baseline in Mean Standard Luminance Best-corrected Visual Acuity (BCVA)
Week 24
|
3.579 letters
Standard Deviation 6.3972
|
4.600 letters
Standard Deviation 5.0681
|
|
Change From Baseline in Mean Standard Luminance Best-corrected Visual Acuity (BCVA)
Week 28
|
1.333 letters
Standard Deviation 10.4881
|
3.667 letters
Standard Deviation 5.8023
|
SECONDARY outcome
Timeframe: Baseline to Day 7, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24, and Week 28.Population: All participants for whom Standard Luminance BCVA was measured.
Change from Baseline in Mean Low Luminance Best-Corrected Visual Acuity (LLBCVA) from Baseline (Day 0) to Day 7, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24, and Week 28.
Outcome measures
| Measure |
AMD-High-Risk Drusen (HRD)
n=21 Participants
The HRD cohort was defined as the presence of either at least 1 large (≥125 μm) druse or multiple medium-size (63-124 μm) drusen in one eye. Subjects had to have 1 eye with intermediate AMD (high-risk drusen \[HRD\] without geographic atrophy \[GA\]).
|
AMD-NCGA (Noncentral GA)
n=19 Participants
The NCGA cohort was defined as evidence of GA with cumulative area ≥1.27 mm2 (approximately 0.5 disc area) by fundus autofluorescence (FAF) that spared the fovea (defined as retinal pigment epithelium and outer retina intact by spectral-domain optical coherence tomography \[SD-OCT\]). Subjects had to have 1 eye with intermediate AMD with noncentral GA \[NCGA\]
|
|---|---|---|
|
Change From Baseline in Mean Low Luminance Best-Corrected Visual Acuity (LLBCVA)
Day 7
|
2.714 letters
Standard Deviation 5.3399
|
1.105 letters
Standard Deviation 8.4518
|
|
Change From Baseline in Mean Low Luminance Best-Corrected Visual Acuity (LLBCVA)
Week 4
|
2.762 letters
Standard Deviation 4.8673
|
5.000 letters
Standard Deviation 5.5413
|
|
Change From Baseline in Mean Low Luminance Best-Corrected Visual Acuity (LLBCVA)
Week 8
|
2.800 letters
Standard Deviation 6.2205
|
4.167 letters
Standard Deviation 4.2183
|
|
Change From Baseline in Mean Low Luminance Best-Corrected Visual Acuity (LLBCVA)
Week 12
|
2.150 letters
Standard Deviation 6.6986
|
4.688 letters
Standard Deviation 4.5858
|
|
Change From Baseline in Mean Low Luminance Best-Corrected Visual Acuity (LLBCVA)
Week 16
|
5.158 letters
Standard Deviation 7.3126
|
4.875 letters
Standard Deviation 5.9203
|
|
Change From Baseline in Mean Low Luminance Best-Corrected Visual Acuity (LLBCVA)
Week 20
|
5.632 letters
Standard Deviation 7.4923
|
5.438 letters
Standard Deviation 5.8875
|
|
Change From Baseline in Mean Low Luminance Best-Corrected Visual Acuity (LLBCVA)
Week 24
|
5.632 letters
Standard Deviation 7.7832
|
5.400 letters
Standard Deviation 7.8540
|
|
Change From Baseline in Mean Low Luminance Best-Corrected Visual Acuity (LLBCVA)
Week 28
|
2.556 letters
Standard Deviation 14.2095
|
3.733 letters
Standard Deviation 8.1545
|
SECONDARY outcome
Timeframe: Baseline (Day 0) and Week 24Population: All participants for whom Dark Adaptometry was measured at Baseline and Week 24 at 0% 25%, 50%, 75% Bleach Level.
Change from Baseline in Mean Dark Adaptometry from Baseline to Week 24- at 0% 25%, 50%, 75% Bleach Level. Dark adaptometry, used to evaluate night blindness by measuring the absolute thresholds of rod sensitivity, was performed within 14 days before Day 0 and at all in-person visits except for Day 7. Dark adaptation is the delayed recovery of light sensitivity in darkness following prior light exposure (photobleaching). The observed and change from baseline recovery scores (in minutes) for each bleach level (0%, 25%, 50%, and 75%) were summarized descriptively for each eye at each visit and were presented in a listing. Shorter recovery times are better than longer recovery times.
Outcome measures
| Measure |
AMD-High-Risk Drusen (HRD)
n=21 Participants
The HRD cohort was defined as the presence of either at least 1 large (≥125 μm) druse or multiple medium-size (63-124 μm) drusen in one eye. Subjects had to have 1 eye with intermediate AMD (high-risk drusen \[HRD\] without geographic atrophy \[GA\]).
|
AMD-NCGA (Noncentral GA)
n=19 Participants
The NCGA cohort was defined as evidence of GA with cumulative area ≥1.27 mm2 (approximately 0.5 disc area) by fundus autofluorescence (FAF) that spared the fovea (defined as retinal pigment epithelium and outer retina intact by spectral-domain optical coherence tomography \[SD-OCT\]). Subjects had to have 1 eye with intermediate AMD with noncentral GA \[NCGA\]
|
|---|---|---|
|
Change From Baseline in Mean Dark Adaptometry
0% Bleach Level
|
-11.553 minutes
Standard Deviation 10.3229
|
3.543 minutes
Standard Deviation 5.0140
|
|
Change From Baseline in Mean Dark Adaptometry
25% Bleach Level
|
0.409 minutes
Standard Deviation 4.2798
|
8.3302 minutes
Standard Deviation 0.000
|
|
Change From Baseline in Mean Dark Adaptometry
50% Bleach Level
|
0.000 minutes
Standard Deviation 0.0000
|
-3.263 minutes
Standard Deviation 7.9935
|
|
Change From Baseline in Mean Dark Adaptometry
75% Bleach Level
|
1.562 minutes
Standard Deviation 5.3936
|
-3.048 minutes
Standard Deviation 9.1675
|
SECONDARY outcome
Timeframe: Baseline through Week 28Population: All participants for whom compliance was measured.
Mean percentage of treatment compliance of administration of subcutaneous elamipretide. The number of injections (diary) and vials used was used to compute % compliance over the duration of the subject's participation in the trial. Percentage can range from 0-100%, where 0% means the participant followed the correct dosing 0% of the time, and 100% compliance means the participant followed the correct dosing 100% of the time, with a higher % meaning a better outcome.
Outcome measures
| Measure |
AMD-High-Risk Drusen (HRD)
n=21 Participants
The HRD cohort was defined as the presence of either at least 1 large (≥125 μm) druse or multiple medium-size (63-124 μm) drusen in one eye. Subjects had to have 1 eye with intermediate AMD (high-risk drusen \[HRD\] without geographic atrophy \[GA\]).
|
AMD-NCGA (Noncentral GA)
n=19 Participants
The NCGA cohort was defined as evidence of GA with cumulative area ≥1.27 mm2 (approximately 0.5 disc area) by fundus autofluorescence (FAF) that spared the fovea (defined as retinal pigment epithelium and outer retina intact by spectral-domain optical coherence tomography \[SD-OCT\]). Subjects had to have 1 eye with intermediate AMD with noncentral GA \[NCGA\]
|
|---|---|---|
|
Mean Treatment Compliance (%) of Administration of Subcutaneous Elamipretide
|
98.4 % of compliance in study dosing
Standard Deviation 4.04
|
97.3 % of compliance in study dosing
Standard Deviation 6.70
|
SECONDARY outcome
Timeframe: Baseline (Day 0) through Week 24Population: All participants and caregivers whom administered elamipretide injections.
Mean Number of Home Health Visits Necessary for Participant or Caregiver to Learn How to Administer Elamipretide
Outcome measures
| Measure |
AMD-High-Risk Drusen (HRD)
n=21 Participants
The HRD cohort was defined as the presence of either at least 1 large (≥125 μm) druse or multiple medium-size (63-124 μm) drusen in one eye. Subjects had to have 1 eye with intermediate AMD (high-risk drusen \[HRD\] without geographic atrophy \[GA\]).
|
AMD-NCGA (Noncentral GA)
n=19 Participants
The NCGA cohort was defined as evidence of GA with cumulative area ≥1.27 mm2 (approximately 0.5 disc area) by fundus autofluorescence (FAF) that spared the fovea (defined as retinal pigment epithelium and outer retina intact by spectral-domain optical coherence tomography \[SD-OCT\]). Subjects had to have 1 eye with intermediate AMD with noncentral GA \[NCGA\]
|
|---|---|---|
|
Mean Number of Home Health Visits to Administer Elamipretide
|
2.2 visits
Standard Deviation 0.54
|
2.5 visits
Standard Deviation 1.02
|
SECONDARY outcome
Timeframe: Baseline (Day 0) to Week 24Population: All participants for whom Geographic Atrophy by Fundus Autofluorescence was measured at Week 24.
Change from Baseline in Mean Area of Geographic Atrophy (GA) by Fundus Autofluorescence (FAF) at Week 24. Fluorescein angiography (FA) was used to examine the circulation of the retina and choroid using fluorescein dye and a specialized camera to trace the dye. Fundus autofluorescence imaging of the retinal pigment epithelium and neurosensory retina was performed within 14 days of Day 0 and at every visit with the exception of Day 0 and Day 7. Atrophy is characterized by loss of the retinal pigment epithelium (RPE), overlying photoreceptors and underlying choriocapillaris. Greater area affected means a worse outcome than smaller area affected.
Outcome measures
| Measure |
AMD-High-Risk Drusen (HRD)
The HRD cohort was defined as the presence of either at least 1 large (≥125 μm) druse or multiple medium-size (63-124 μm) drusen in one eye. Subjects had to have 1 eye with intermediate AMD (high-risk drusen \[HRD\] without geographic atrophy \[GA\]).
|
AMD-NCGA (Noncentral GA)
n=12 Participants
The NCGA cohort was defined as evidence of GA with cumulative area ≥1.27 mm2 (approximately 0.5 disc area) by fundus autofluorescence (FAF) that spared the fovea (defined as retinal pigment epithelium and outer retina intact by spectral-domain optical coherence tomography \[SD-OCT\]). Subjects had to have 1 eye with intermediate AMD with noncentral GA \[NCGA\]
|
|---|---|---|
|
Change From Baseline in Mean Area of Geographic Atrophy by Fundus Autofluorescence
Week 12
|
—
|
0.264 mm^2
Standard Deviation 0.1641
|
|
Change From Baseline in Mean Area of Geographic Atrophy by Fundus Autofluorescence
Week 24
|
—
|
0.503 mm^2
Standard Deviation 0.4914
|
SECONDARY outcome
Timeframe: Baseline to Week 24Population: All participants for whom geographic atrophy by SD-OCT was measured.
Change from Baseline in Mean Area of Geographic Atrophy by Sector as measured by Spectral-Domain Optical Coherence Tomography (SD-OCT) at Week 24
Outcome measures
| Measure |
AMD-High-Risk Drusen (HRD)
The HRD cohort was defined as the presence of either at least 1 large (≥125 μm) druse or multiple medium-size (63-124 μm) drusen in one eye. Subjects had to have 1 eye with intermediate AMD (high-risk drusen \[HRD\] without geographic atrophy \[GA\]).
|
AMD-NCGA (Noncentral GA)
n=15 Participants
The NCGA cohort was defined as evidence of GA with cumulative area ≥1.27 mm2 (approximately 0.5 disc area) by fundus autofluorescence (FAF) that spared the fovea (defined as retinal pigment epithelium and outer retina intact by spectral-domain optical coherence tomography \[SD-OCT\]). Subjects had to have 1 eye with intermediate AMD with noncentral GA \[NCGA\]
|
|---|---|---|
|
Change From Baseline in Mean Area of Geographic Atrophy as Measured by Spectral-Domain Optical Coherence Tomography (SD-OCT)
Center Sector
|
—
|
0.02 mm^2
Standard Deviation 0.048
|
|
Change From Baseline in Mean Area of Geographic Atrophy as Measured by Spectral-Domain Optical Coherence Tomography (SD-OCT)
Inner Superior
|
—
|
0.05 mm^2
Standard Deviation 0.096
|
|
Change From Baseline in Mean Area of Geographic Atrophy as Measured by Spectral-Domain Optical Coherence Tomography (SD-OCT)
Inner Nasal
|
—
|
0.04 mm^2
Standard Deviation 0.059
|
|
Change From Baseline in Mean Area of Geographic Atrophy as Measured by Spectral-Domain Optical Coherence Tomography (SD-OCT)
Inner Inferior
|
—
|
0.02 mm^2
Standard Deviation 0.073
|
|
Change From Baseline in Mean Area of Geographic Atrophy as Measured by Spectral-Domain Optical Coherence Tomography (SD-OCT)
Inner Temporal
|
—
|
0.05 mm^2
Standard Deviation 0.097
|
|
Change From Baseline in Mean Area of Geographic Atrophy as Measured by Spectral-Domain Optical Coherence Tomography (SD-OCT)
Outer Superior
|
—
|
0.08 mm^2
Standard Deviation 0.116
|
|
Change From Baseline in Mean Area of Geographic Atrophy as Measured by Spectral-Domain Optical Coherence Tomography (SD-OCT)
Outer Nasal
|
—
|
0.06 mm^2
Standard Deviation 0.114
|
|
Change From Baseline in Mean Area of Geographic Atrophy as Measured by Spectral-Domain Optical Coherence Tomography (SD-OCT)
Outer Inferior
|
—
|
0.05 mm^2
Standard Deviation 0.146
|
|
Change From Baseline in Mean Area of Geographic Atrophy as Measured by Spectral-Domain Optical Coherence Tomography (SD-OCT)
Outer Temporal
|
—
|
0.08 mm^2
Standard Deviation 0.129
|
|
Change From Baseline in Mean Area of Geographic Atrophy as Measured by Spectral-Domain Optical Coherence Tomography (SD-OCT)
Total ETDRS grid
|
—
|
0.45 mm^2
Standard Deviation 0.607
|
SECONDARY outcome
Timeframe: Baseline and Weeks 4, 8, 12, 16 20, 24 and 28Population: All participants for whom Mean Critical Print Size (logMAR) with Standard Luminance was measured
Change from Baseline in Mean Reading Acuity Test: Mean Critical Print Size with Standard Luminance in Weeks 4, 8, 12, 16, 20, 24 and 28 as measured by the Logarithm of the Minimum Angle of Resolution LogMAR chart. Scores range from -0.3 to 1.0, where higher number means worse acuity/worse outcome, a lower number means better acuity/better outcome.
Outcome measures
| Measure |
AMD-High-Risk Drusen (HRD)
n=21 Participants
The HRD cohort was defined as the presence of either at least 1 large (≥125 μm) druse or multiple medium-size (63-124 μm) drusen in one eye. Subjects had to have 1 eye with intermediate AMD (high-risk drusen \[HRD\] without geographic atrophy \[GA\]).
|
AMD-NCGA (Noncentral GA)
n=18 Participants
The NCGA cohort was defined as evidence of GA with cumulative area ≥1.27 mm2 (approximately 0.5 disc area) by fundus autofluorescence (FAF) that spared the fovea (defined as retinal pigment epithelium and outer retina intact by spectral-domain optical coherence tomography \[SD-OCT\]). Subjects had to have 1 eye with intermediate AMD with noncentral GA \[NCGA\]
|
|---|---|---|
|
Change From Baseline in Mean Reading Acuity Test: With Standard Luminance
Week 4
|
-0.04 units on a scale
Standard Deviation 0.121
|
-0.03 units on a scale
Standard Deviation 0.091
|
|
Change From Baseline in Mean Reading Acuity Test: With Standard Luminance
Week 8
|
-0.08 units on a scale
Standard Deviation 0.111
|
-0.02 units on a scale
Standard Deviation 0.115
|
|
Change From Baseline in Mean Reading Acuity Test: With Standard Luminance
Week 12
|
-0.10 units on a scale
Standard Deviation 0.156
|
-0.02 units on a scale
Standard Deviation 0.098
|
|
Change From Baseline in Mean Reading Acuity Test: With Standard Luminance
Week 16
|
-0.11 units on a scale
Standard Deviation 0.151
|
-0.06 units on a scale
Standard Deviation 0.115
|
|
Change From Baseline in Mean Reading Acuity Test: With Standard Luminance
Week 20
|
-0.12 units on a scale
Standard Deviation 0.126
|
-0.01 units on a scale
Standard Deviation 0.109
|
|
Change From Baseline in Mean Reading Acuity Test: With Standard Luminance
Week 24
|
-0.11 units on a scale
Standard Deviation 0.152
|
-0.02 units on a scale
Standard Deviation 0.126
|
|
Change From Baseline in Mean Reading Acuity Test: With Standard Luminance
Week 28
|
-0.04 units on a scale
Standard Deviation 0.201
|
0.01 units on a scale
Standard Deviation 0.173
|
SECONDARY outcome
Timeframe: Assessed at screening, baseline, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24, and Week 28Population: All participants for whom Mean Critical Print Size (logMAR) with Low Luminance was measured at baseline and Week 4 through Week 28.
Change from Baseline in Mean Reading Acuity Test: Mean Critical Print Size with Low Luminance in Weeks 4, 8, 12, 16, 20, 24 and 28 as measured by the Logarithm of the Minimum Angle of Resolution LogMAR chart. Scores range from -0.3 to 1.0, where higher number means worse acuity/worse outcome, a lower number means better acuity/better outcome.
Outcome measures
| Measure |
AMD-High-Risk Drusen (HRD)
n=21 Participants
The HRD cohort was defined as the presence of either at least 1 large (≥125 μm) druse or multiple medium-size (63-124 μm) drusen in one eye. Subjects had to have 1 eye with intermediate AMD (high-risk drusen \[HRD\] without geographic atrophy \[GA\]).
|
AMD-NCGA (Noncentral GA)
n=11 Participants
The NCGA cohort was defined as evidence of GA with cumulative area ≥1.27 mm2 (approximately 0.5 disc area) by fundus autofluorescence (FAF) that spared the fovea (defined as retinal pigment epithelium and outer retina intact by spectral-domain optical coherence tomography \[SD-OCT\]). Subjects had to have 1 eye with intermediate AMD with noncentral GA \[NCGA\]
|
|---|---|---|
|
Change From Baseline in Mean Reading Acuity Test: Low Luminance
Week 24
|
-0.28 units on a scale
Standard Deviation 0.174
|
-0.11 units on a scale
Standard Deviation 0.209
|
|
Change From Baseline in Mean Reading Acuity Test: Low Luminance
Week 4
|
-0.11 units on a scale
Standard Deviation 0.170
|
-0.09 units on a scale
Standard Deviation 0.088
|
|
Change From Baseline in Mean Reading Acuity Test: Low Luminance
Week 8
|
-0.16 units on a scale
Standard Deviation 0.179
|
-0.10 units on a scale
Standard Deviation 0.253
|
|
Change From Baseline in Mean Reading Acuity Test: Low Luminance
Week 12
|
-0.21 units on a scale
Standard Deviation 0.177
|
-0.20 units on a scale
Standard Deviation 0.236
|
|
Change From Baseline in Mean Reading Acuity Test: Low Luminance
Week 16
|
-0.25 units on a scale
Standard Deviation 0.209
|
-0.16 units on a scale
Standard Deviation 0.232
|
|
Change From Baseline in Mean Reading Acuity Test: Low Luminance
Week 20
|
-0.29 units on a scale
Standard Deviation 0.184
|
-0.14 units on a scale
Standard Deviation 0.171
|
|
Change From Baseline in Mean Reading Acuity Test: Low Luminance
Week 28
|
-0.24 units on a scale
Standard Deviation 0.238
|
-0.10 units on a scale
Standard Deviation 0.194
|
SECONDARY outcome
Timeframe: Assessed at baseline (Day 0), Week 12, Week 24 and Week 28Population: All participants for whom the VFQ-39 was measured at Week 12 and Week 24.
Change from baseline (Day 0) at Week 12, 24 and 28. National Eye Institute Visual Function Questionnaire-39 (VFQ-39) score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated.
Outcome measures
| Measure |
AMD-High-Risk Drusen (HRD)
n=20 Participants
The HRD cohort was defined as the presence of either at least 1 large (≥125 μm) druse or multiple medium-size (63-124 μm) drusen in one eye. Subjects had to have 1 eye with intermediate AMD (high-risk drusen \[HRD\] without geographic atrophy \[GA\]).
|
AMD-NCGA (Noncentral GA)
n=16 Participants
The NCGA cohort was defined as evidence of GA with cumulative area ≥1.27 mm2 (approximately 0.5 disc area) by fundus autofluorescence (FAF) that spared the fovea (defined as retinal pigment epithelium and outer retina intact by spectral-domain optical coherence tomography \[SD-OCT\]). Subjects had to have 1 eye with intermediate AMD with noncentral GA \[NCGA\]
|
|---|---|---|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 12 Color Vision
|
8.8 score on a scale
Standard Deviation 18.63
|
10.9 score on a scale
Standard Deviation 20.35
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 12 Composite
|
7.6 score on a scale
Standard Deviation 7.47
|
5.5 score on a scale
Standard Deviation 8.90
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 12 Dependency
|
1.9 score on a scale
Standard Deviation 10.94
|
2.0 score on a scale
Standard Deviation 14.02
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 12 Distance Activities
|
7.0 score on a scale
Standard Deviation 8.13
|
4.6 score on a scale
Standard Deviation 12.19
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 12 Driving
|
10.0 score on a scale
Standard Deviation 12.85
|
0.6 score on a scale
Standard Deviation 13.38
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 12 General Health
|
1.4 score on a scale
Standard Deviation 8.98
|
1.9 score on a scale
Standard Deviation 9.33
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 12 General Vision
|
5.3 score on a scale
Standard Deviation 11.18
|
7.2 score on a scale
Standard Deviation 14.02
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 12 Mental Health
|
9.3 score on a scale
Standard Deviation 13.79
|
9.1 score on a scale
Standard Deviation 14.63
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 12 Near Activities
|
8.2 score on a scale
Standard Deviation 10.43
|
2.4 score on a scale
Standard Deviation 11.51
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 12 Ocular Pain
|
7.5 score on a scale
Standard Deviation 20.84
|
3.1 score on a scale
Standard Deviation 9.68
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 12 Peripheral Vision
|
12.5 score on a scale
Standard Deviation 19.02
|
7.8 score on a scale
Standard Deviation 19.83
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 12 Role Difficulties
|
5.9 score on a scale
Standard Deviation 12.25
|
5.9 score on a scale
Standard Deviation 26.37
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 12 Social Functioning
|
7.5 score on a scale
Standard Deviation 8.51
|
5.2 score on a scale
Standard Deviation 18.48
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 24 Color Vision
|
11.8 score on a scale
Standard Deviation 22.62
|
11.7 score on a scale
Standard Deviation 26.50
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 24 Composite
|
9.2 score on a scale
Standard Deviation 9.19
|
7.0 score on a scale
Standard Deviation 9.38
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 24 Dependency
|
2.3 score on a scale
Standard Deviation 14.61
|
6.7 score on a scale
Standard Deviation 10.94
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 24 Distance Activities
|
8.4 score on a scale
Standard Deviation 11.11
|
4.7 score on a scale
Standard Deviation 16.13
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 24 Driving
|
14.5 score on a scale
Standard Deviation 14.66
|
0.6 score on a scale
Standard Deviation 10.46
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 24 General Health
|
-0.3 score on a scale
Standard Deviation 9.01
|
3.3 score on a scale
Standard Deviation 10.21
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 24 General Vision
|
8.7 score on a scale
Standard Deviation 12.78
|
6.3 score on a scale
Standard Deviation 13.69
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 24 Mental Health
|
9.5 score on a scale
Standard Deviation 17.55
|
7.7 score on a scale
Standard Deviation 9.80
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 24 Near Activities
|
9.8 score on a scale
Standard Deviation 11.00
|
5.6 score on a scale
Standard Deviation 13.84
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 24 Ocular Pain
|
5.9 score on a scale
Standard Deviation 19.26
|
5.0 score on a scale
Standard Deviation 14.02
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 24 Peripheral Vision
|
17.1 score on a scale
Standard Deviation 16.78
|
15.0 score on a scale
Standard Deviation 18.42
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 24 Role Difficulties
|
6.9 score on a scale
Standard Deviation 11.58
|
5.8 score on a scale
Standard Deviation 17.27
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 24 Social Functioning
|
6.6 score on a scale
Standard Deviation 10.96
|
5.6 score on a scale
Standard Deviation 15.64
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 28 Color Vision
|
12.5 score on a scale
Standard Deviation 17.68
|
5.0 score on a scale
Standard Deviation 28.66
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 28 Composite
|
7.9 score on a scale
Standard Deviation 14.04
|
4.3 score on a scale
Standard Deviation 10.46
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 28 Dependency
|
1.7 score on a scale
Standard Deviation 17.91
|
-1.3 score on a scale
Standard Deviation 20.07
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 28 Distance Activities
|
5.7 score on a scale
Standard Deviation 16.92
|
7.1 score on a scale
Standard Deviation 13.95
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 28 Driving
|
12.3 score on a scale
Standard Deviation 12.54
|
0.8 score on a scale
Standard Deviation 9.47
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 28 General Health
|
4.2 score on a scale
Standard Deviation 10.18
|
4.0 score on a scale
Standard Deviation 9.20
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 28 General Vision
|
8.6 score on a scale
Standard Deviation 16.25
|
3.7 score on a scale
Standard Deviation 12.60
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 28 Mental Health
|
9.4 score on a scale
Standard Deviation 19.84
|
5.7 score on a scale
Standard Deviation 10.50
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 28 Near Activities
|
6.0 score on a scale
Standard Deviation 20.17
|
1.7 score on a scale
Standard Deviation 12.63
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 28 Ocular Pain
|
7.6 score on a scale
Standard Deviation 20.17
|
0.0 score on a scale
Standard Deviation 14.94
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 28 Peripheral Vision
|
15.3 score on a scale
Standard Deviation 21.25
|
13.3 score on a scale
Standard Deviation 20.85
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 28 Role Difficulties
|
4.2 score on a scale
Standard Deviation 21.00
|
5.4 score on a scale
Standard Deviation 20.03
|
|
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Week 28 Social Functioning
|
5.6 score on a scale
Standard Deviation 17.85
|
3.3 score on a scale
Standard Deviation 10.82
|
SECONDARY outcome
Timeframe: Baseline (Day 0), Week 12, and Week 24 and Week 28Population: All participants for whom Low-Luminance visual function by the Visual Function Questionnaire was measured.
Change from baseline (Day 0) at Week 12, Week 24, and Week 28 in LLQ score. The LLQ is a 32-item questionnaire with six subscales related to low luminance settings: driving, mobility, extreme lighting, general dim lighting, and peripheral vision. Each question is scored on a scale ranging from 0, or maximal difficulty, to 100, or no difficulty in low luminance settings. Higher scores mean better functioning. The questions are assigned to different subscales and are averaged to generate one score per subscale. After weighting each subscale for the number of questions, the weighted subscales are averaged to generate a composite LLQ score.
Outcome measures
| Measure |
AMD-High-Risk Drusen (HRD)
n=21 Participants
The HRD cohort was defined as the presence of either at least 1 large (≥125 μm) druse or multiple medium-size (63-124 μm) drusen in one eye. Subjects had to have 1 eye with intermediate AMD (high-risk drusen \[HRD\] without geographic atrophy \[GA\]).
|
AMD-NCGA (Noncentral GA)
n=16 Participants
The NCGA cohort was defined as evidence of GA with cumulative area ≥1.27 mm2 (approximately 0.5 disc area) by fundus autofluorescence (FAF) that spared the fovea (defined as retinal pigment epithelium and outer retina intact by spectral-domain optical coherence tomography \[SD-OCT\]). Subjects had to have 1 eye with intermediate AMD with noncentral GA \[NCGA\]
|
|---|---|---|
|
Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score
Week 12 Control questions
|
10.5 score on a scale
Standard Deviation 15.47
|
-2.3 score on a scale
Standard Deviation 9.86
|
|
Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score
Week 12 Dim light reading
|
9.7 score on a scale
Standard Deviation 12.58
|
4.3 score on a scale
Standard Deviation 13.05
|
|
Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score
Week 12 Driving or riding in car
|
12.2 score on a scale
Standard Deviation 16.41
|
3.9 score on a scale
Standard Deviation 10.92
|
|
Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score
Week 12 General dim light vision
|
11.9 score on a scale
Standard Deviation 13.26
|
6.9 score on a scale
Standard Deviation 12.62
|
|
Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score
Week 12 Light transitions and glare
|
9.3 score on a scale
Standard Deviation 15.07
|
6.3 score on a scale
Standard Deviation 13.35
|
|
Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score
Week 12 Mobility
|
6.7 score on a scale
Standard Deviation 11.66
|
6.3 score on a scale
Standard Deviation 10.32
|
|
Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score
Week 12 Other Activities of Daily Living (ADLs)
|
11.5 score on a scale
Standard Deviation 18.36
|
10.2 score on a scale
Standard Deviation 15.29
|
|
Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score
Week 12 Peripheral vision
|
16.3 score on a scale
Standard Deviation 19.49
|
3.9 score on a scale
Standard Deviation 17.51
|
|
Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score
Week 24 Control questions
|
7.2 score on a scale
Standard Deviation 12.56
|
0.7 score on a scale
Standard Deviation 12.47
|
|
Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score
Week 24 Dim light reading
|
15.8 score on a scale
Standard Deviation 14.34
|
7.1 score on a scale
Standard Deviation 11.30
|
|
Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score
Week 24 Driving or riding in car
|
16.4 score on a scale
Standard Deviation 18.77
|
4.2 score on a scale
Standard Deviation 14.11
|
|
Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score
Week 24 General dim light vision
|
15.6 score on a scale
Standard Deviation 15.17
|
7.7 score on a scale
Standard Deviation 12.10
|
|
Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score
Week 24 Light transitions and glare
|
17.4 score on a scale
Standard Deviation 13.98
|
6.7 score on a scale
Standard Deviation 13.71
|
|
Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score
Week 24 Mobility
|
9.6 score on a scale
Standard Deviation 20.27
|
2.8 score on a scale
Standard Deviation 16.57
|
|
Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score
Week 24 Other Activities of Daily Living (ADLs)
|
17.8 score on a scale
Standard Deviation 17.95
|
10.4 score on a scale
Standard Deviation 20.82
|
|
Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score
Week 24 Peripheral vision
|
17.8 score on a scale
Standard Deviation 20.12
|
5.8 score on a scale
Standard Deviation 24.03
|
|
Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score
Week 28 Control Questions
|
3.2 score on a scale
Standard Deviation 18.44
|
0.2 score on a scale
Standard Deviation 14.25
|
|
Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score
Week 28 Dim light reading
|
12.2 score on a scale
Standard Deviation 19.70
|
7.1 score on a scale
Standard Deviation 12.47
|
|
Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score
Week 28 Driving or riding in car
|
10.8 score on a scale
Standard Deviation 23.85
|
4.3 score on a scale
Standard Deviation 14.34
|
|
Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score
Week 28 General dim light vision
|
11.7 score on a scale
Standard Deviation 19.43
|
3.3 score on a scale
Standard Deviation 10.73
|
|
Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score
Week 28 Light transitions and glare
|
15.0 score on a scale
Standard Deviation 14.75
|
5.3 score on a scale
Standard Deviation 14.07
|
|
Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score
Week 28 Mobility
|
7.4 score on a scale
Standard Deviation 23.02
|
-1.1 score on a scale
Standard Deviation 14.39
|
|
Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score
Week 28 Other Activities of Daily Living
|
12.2 score on a scale
Standard Deviation 23.82
|
3.8 score on a scale
Standard Deviation 13.73
|
|
Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score
Week 28 Peripheral Vision
|
13.9 score on a scale
Standard Deviation 20.06
|
5.0 score on a scale
Standard Deviation 15.53
|
SECONDARY outcome
Timeframe: Baseline (Day 0) and Weeks 4,8,12,16,20,and 24Population: All participants for whom mean mesopic light sensitivity was measured.
Change from baseline in mean mesopic light sensitivity for Weeks, 4, 8, 12, 16, 20, and 24 as assessed by microperimetry for number of loci \<25dB and \<14dB. Mesopic microperimetry, used to measure retinal sensitivity, was performed within 14 days before Day 0 and at all in-person visits except for Day 7.
Outcome measures
| Measure |
AMD-High-Risk Drusen (HRD)
n=20 Participants
The HRD cohort was defined as the presence of either at least 1 large (≥125 μm) druse or multiple medium-size (63-124 μm) drusen in one eye. Subjects had to have 1 eye with intermediate AMD (high-risk drusen \[HRD\] without geographic atrophy \[GA\]).
|
AMD-NCGA (Noncentral GA)
n=17 Participants
The NCGA cohort was defined as evidence of GA with cumulative area ≥1.27 mm2 (approximately 0.5 disc area) by fundus autofluorescence (FAF) that spared the fovea (defined as retinal pigment epithelium and outer retina intact by spectral-domain optical coherence tomography \[SD-OCT\]). Subjects had to have 1 eye with intermediate AMD with noncentral GA \[NCGA\]
|
|---|---|---|
|
Change From Baseline in Mean Mesopic Light Sensitivity
Number of loci <25dB Week 4
|
0.6 loci
Standard Deviation 6.08
|
0.6 loci
Standard Deviation 1.79
|
|
Change From Baseline in Mean Mesopic Light Sensitivity
Number of loci <25dB Week 8
|
1.3 loci
Standard Deviation 8.21
|
0.7 loci
Standard Deviation 1.94
|
|
Change From Baseline in Mean Mesopic Light Sensitivity
Number of loci <25dB Week 12
|
0.5 loci
Standard Deviation 7.87
|
0.1 loci
Standard Deviation 3.09
|
|
Change From Baseline in Mean Mesopic Light Sensitivity
Number of loci <25dB Week 16
|
0.9 loci
Standard Deviation 8.56
|
0.1 loci
Standard Deviation 2.35
|
|
Change From Baseline in Mean Mesopic Light Sensitivity
Number of loci <25dB Week 20
|
-1.2 loci
Standard Deviation 5.88
|
-0.1 loci
Standard Deviation 1.44
|
|
Change From Baseline in Mean Mesopic Light Sensitivity
Number of loci <25dB Week 24
|
-0.5 loci
Standard Deviation 8.27
|
-0.1 loci
Standard Deviation 3.45
|
|
Change From Baseline in Mean Mesopic Light Sensitivity
Number of loci <14dB Week 4
|
0.3 loci
Standard Deviation 0.3
|
1.7 loci
Standard Deviation 1.7
|
|
Change From Baseline in Mean Mesopic Light Sensitivity
Number of loci <14dB Week 8
|
-1.4 loci
Standard Deviation 3.65
|
1.7 loci
Standard Deviation 4.89
|
|
Change From Baseline in Mean Mesopic Light Sensitivity
Number of loci <14dB Week 12
|
1.3 loci
Standard Deviation 10.05
|
3.2 loci
Standard Deviation 5.90
|
|
Change From Baseline in Mean Mesopic Light Sensitivity
Number of loci <14dB Week 16
|
-2.5 loci
Standard Deviation 2.38
|
1.5 loci
Standard Deviation 5.01
|
|
Change From Baseline in Mean Mesopic Light Sensitivity
Number of loci <14dB Week 20
|
-0.2 loci
Standard Deviation 6.14
|
1.5 loci
Standard Deviation 4.88
|
|
Change From Baseline in Mean Mesopic Light Sensitivity
Number of loci <14dB Week 24
|
-0.3 loci
Standard Deviation 8.30
|
3.3 loci
Standard Deviation 5.82
|
SECONDARY outcome
Timeframe: Baseline (Day 0) and Week 24Population: All participants for whom RPEDC) thickness as Measured by SD-OCT by sector at Baseline and Week 24
Change from baseline in mean retinal pigment epithelium - drusen complex (RPEDC) thickness as Measured by SD-OCT by sector at Week 24
Outcome measures
| Measure |
AMD-High-Risk Drusen (HRD)
n=19 Participants
The HRD cohort was defined as the presence of either at least 1 large (≥125 μm) druse or multiple medium-size (63-124 μm) drusen in one eye. Subjects had to have 1 eye with intermediate AMD (high-risk drusen \[HRD\] without geographic atrophy \[GA\]).
|
AMD-NCGA (Noncentral GA)
n=15 Participants
The NCGA cohort was defined as evidence of GA with cumulative area ≥1.27 mm2 (approximately 0.5 disc area) by fundus autofluorescence (FAF) that spared the fovea (defined as retinal pigment epithelium and outer retina intact by spectral-domain optical coherence tomography \[SD-OCT\]). Subjects had to have 1 eye with intermediate AMD with noncentral GA \[NCGA\]
|
|---|---|---|
|
Change From Baseline in Mean Retinal Pigment Epithelium - Drusen Complex (RPEDC) Thickness as Measured by SD-OCT
Center
|
0.57 um
Standard Deviation 5.147
|
0.38 um
Standard Deviation 4.137
|
|
Change From Baseline in Mean Retinal Pigment Epithelium - Drusen Complex (RPEDC) Thickness as Measured by SD-OCT
Inner Superior
|
1.29 um
Standard Deviation 3.699
|
-0.85 um
Standard Deviation 2.389
|
|
Change From Baseline in Mean Retinal Pigment Epithelium - Drusen Complex (RPEDC) Thickness as Measured by SD-OCT
Inner Nasal
|
1.43 um
Standard Deviation 3.143
|
-0.83 um
Standard Deviation 2.801
|
|
Change From Baseline in Mean Retinal Pigment Epithelium - Drusen Complex (RPEDC) Thickness as Measured by SD-OCT
Inner Inferior
|
0.72 um
Standard Deviation 4.393
|
-0.82 um
Standard Deviation 3.798
|
|
Change From Baseline in Mean Retinal Pigment Epithelium - Drusen Complex (RPEDC) Thickness as Measured by SD-OCT
Inner temporal
|
1.00 um
Standard Deviation 3.680
|
-0.34 um
Standard Deviation 2.897
|
|
Change From Baseline in Mean Retinal Pigment Epithelium - Drusen Complex (RPEDC) Thickness as Measured by SD-OCT
Outer Superior
|
0.34 um
Standard Deviation 2.279
|
-0.10 um
Standard Deviation 1.580
|
|
Change From Baseline in Mean Retinal Pigment Epithelium - Drusen Complex (RPEDC) Thickness as Measured by SD-OCT
Outer Nasal
|
0.34 um
Standard Deviation 2.301
|
-0.09 um
Standard Deviation 1.919
|
|
Change From Baseline in Mean Retinal Pigment Epithelium - Drusen Complex (RPEDC) Thickness as Measured by SD-OCT
Outer Inferior
|
-0.19 um
Standard Deviation 2.356
|
-0.04 um
Standard Deviation 2.023
|
|
Change From Baseline in Mean Retinal Pigment Epithelium - Drusen Complex (RPEDC) Thickness as Measured by SD-OCT
Outer Temporal
|
0.25 um
Standard Deviation 2.672
|
-0.04 um
Standard Deviation 1.594
|
|
Change From Baseline in Mean Retinal Pigment Epithelium - Drusen Complex (RPEDC) Thickness as Measured by SD-OCT
Total ETDRS
|
0.39 um
Standard Deviation 1.885
|
-0.25 um
Standard Deviation 1.590
|
SECONDARY outcome
Timeframe: Baseline (Day 0) to Week 24Population: All participants for whom the retinal pigment epithelium-drusen complex volume was measured
Change from baseline in mean Retinal Pigment Epithelium-Drusen Complex volume at Week 24 by sector
Outcome measures
| Measure |
AMD-High-Risk Drusen (HRD)
n=19 Participants
The HRD cohort was defined as the presence of either at least 1 large (≥125 μm) druse or multiple medium-size (63-124 μm) drusen in one eye. Subjects had to have 1 eye with intermediate AMD (high-risk drusen \[HRD\] without geographic atrophy \[GA\]).
|
AMD-NCGA (Noncentral GA)
n=15 Participants
The NCGA cohort was defined as evidence of GA with cumulative area ≥1.27 mm2 (approximately 0.5 disc area) by fundus autofluorescence (FAF) that spared the fovea (defined as retinal pigment epithelium and outer retina intact by spectral-domain optical coherence tomography \[SD-OCT\]). Subjects had to have 1 eye with intermediate AMD with noncentral GA \[NCGA\]
|
|---|---|---|
|
Change From Baseline in Mean Retinal Pigment Epithelium-Drusen Complex Volume
Center
|
0.00 mm^3
Standard Deviation 0.004
|
0.00 mm^3
Standard Deviation 0.003
|
|
Change From Baseline in Mean Retinal Pigment Epithelium-Drusen Complex Volume
Inner superior
|
0.00 mm^3
Standard Deviation 0.006
|
-0.00 mm^3
Standard Deviation 0.004
|
|
Change From Baseline in Mean Retinal Pigment Epithelium-Drusen Complex Volume
Inner nasal
|
0.00 mm^3
Standard Deviation 0.005
|
-0.00 mm^3
Standard Deviation 0.004
|
|
Change From Baseline in Mean Retinal Pigment Epithelium-Drusen Complex Volume
Inner inferior
|
0.00 mm^3
Standard Deviation 0.007
|
-0.00 mm^3
Standard Deviation 0.006
|
|
Change From Baseline in Mean Retinal Pigment Epithelium-Drusen Complex Volume
Inner temporal
|
0.00 mm^3
Standard Deviation 0.006
|
-0.00 mm^3
Standard Deviation 0.005
|
|
Change From Baseline in Mean Retinal Pigment Epithelium-Drusen Complex Volume
Outer superior
|
0.00 mm^3
Standard Deviation 0.012
|
-0.00 mm^3
Standard Deviation 0.008
|
|
Change From Baseline in Mean Retinal Pigment Epithelium-Drusen Complex Volume
Outer nasal
|
0.00 mm^3
Standard Deviation 0.012
|
-0.00 mm^3
Standard Deviation 0.010
|
|
Change From Baseline in Mean Retinal Pigment Epithelium-Drusen Complex Volume
Outer inferior
|
-0.00 mm^3
Standard Deviation 0.012
|
-0.00 mm^3
Standard Deviation 0.011
|
|
Change From Baseline in Mean Retinal Pigment Epithelium-Drusen Complex Volume
Outer temporal
|
0.00 mm^3
Standard Deviation 0.014
|
-0.00 mm^3
Standard Deviation 0.009
|
|
Change From Baseline in Mean Retinal Pigment Epithelium-Drusen Complex Volume
Total ETDRS
|
0.01 mm^3
Standard Deviation 0.054
|
-0.01 mm^3
Standard Deviation 0.046
|
Adverse Events
High-Risk Drusen (HRD)
Serious events: 1 serious events
Other events: 21 other events
Deaths: 0 deaths
NCGA (Noncentral GA)
Serious events: 1 serious events
Other events: 19 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
High-Risk Drusen (HRD)
n=21 participants at risk
The HRD cohort was defined as the presence of either at least 1 large (≥125 μm) druse or multiple medium-size (63-124 μm) drusen in one eye. Subjects had to have 1 eye with intermediate AMD (high-risk drusen \[HRD\] without geographic atrophy \[GA\]).
|
NCGA (Noncentral GA)
n=19 participants at risk
The NCGA cohort was defined as evidence of GA with cumulative area ≥1.27 mm2 (approximately 0.5 disc area) by fundus autofluorescence (FAF) that spared the fovea (defined as retinal pigment epithelium and outer retina intact by spectral-domain optical coherence tomography \[SD-OCT\]). Subjects had to have 1 eye with intermediate AMD with noncentral GA \[NCGA\]
|
|---|---|---|
|
Renal and urinary disorders
Calculus urinary
|
4.8%
1/21 • 28 Weeks
|
0.00%
0/19 • 28 Weeks
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/21 • 28 Weeks
|
5.3%
1/19 • 28 Weeks
|
|
Infections and infestations
Sepsis
|
0.00%
0/21 • 28 Weeks
|
5.3%
1/19 • 28 Weeks
|
Other adverse events
| Measure |
High-Risk Drusen (HRD)
n=21 participants at risk
The HRD cohort was defined as the presence of either at least 1 large (≥125 μm) druse or multiple medium-size (63-124 μm) drusen in one eye. Subjects had to have 1 eye with intermediate AMD (high-risk drusen \[HRD\] without geographic atrophy \[GA\]).
|
NCGA (Noncentral GA)
n=19 participants at risk
The NCGA cohort was defined as evidence of GA with cumulative area ≥1.27 mm2 (approximately 0.5 disc area) by fundus autofluorescence (FAF) that spared the fovea (defined as retinal pigment epithelium and outer retina intact by spectral-domain optical coherence tomography \[SD-OCT\]). Subjects had to have 1 eye with intermediate AMD with noncentral GA \[NCGA\]
|
|---|---|---|
|
Ear and labyrinth disorders
Motion sickness
|
0.00%
0/21 • 28 Weeks
|
5.3%
1/19 • 28 Weeks
|
|
Eye disorders
Punctate keratitis
|
14.3%
3/21 • 28 Weeks
|
0.00%
0/19 • 28 Weeks
|
|
Eye disorders
Retinal haemorrhage
|
9.5%
2/21 • 28 Weeks
|
0.00%
0/19 • 28 Weeks
|
|
Eye disorders
Neovascular age-related macular degeneration
|
4.8%
1/21 • 28 Weeks
|
5.3%
1/19 • 28 Weeks
|
|
Eye disorders
Visual acuity reduced
|
4.8%
1/21 • 28 Weeks
|
5.3%
1/19 • 28 Weeks
|
|
Eye disorders
Vitreous floaters
|
0.00%
0/21 • 28 Weeks
|
5.3%
1/19 • 28 Weeks
|
|
Gastrointestinal disorders
Nausea
|
4.8%
1/21 • 28 Weeks
|
10.5%
2/19 • 28 Weeks
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/21 • 28 Weeks
|
5.3%
1/19 • 28 Weeks
|
|
General disorders
Injection site pruritis
|
100.0%
21/21 • 28 Weeks
|
89.5%
17/19 • 28 Weeks
|
|
General disorders
Injection site bruising
|
76.2%
16/21 • 28 Weeks
|
68.4%
13/19 • 28 Weeks
|
|
General disorders
Injection site erythema
|
76.2%
16/21 • 28 Weeks
|
73.7%
14/19 • 28 Weeks
|
|
General disorders
Injection site induration
|
76.2%
16/21 • 28 Weeks
|
73.7%
14/19 • 28 Weeks
|
|
General disorders
Injection site pain
|
42.9%
9/21 • 28 Weeks
|
31.6%
6/19 • 28 Weeks
|
|
General disorders
Injection site haemorrhage
|
28.6%
6/21 • 28 Weeks
|
36.8%
7/19 • 28 Weeks
|
|
General disorders
Injection site urticaria
|
23.8%
5/21 • 28 Weeks
|
21.1%
4/19 • 28 Weeks
|
|
General disorders
Injection site swelling
|
4.8%
1/21 • 28 Weeks
|
5.3%
1/19 • 28 Weeks
|
|
General disorders
Injection site extravasation
|
0.00%
0/21 • 28 Weeks
|
10.5%
2/19 • 28 Weeks
|
|
General disorders
Injury associated with device
|
0.00%
0/21 • 28 Weeks
|
5.3%
1/19 • 28 Weeks
|
|
General disorders
Pyrexia
|
0.00%
0/21 • 28 Weeks
|
10.5%
2/19 • 28 Weeks
|
|
Immune system disorders
Seasonal allergy
|
9.5%
2/21 • 28 Weeks
|
0.00%
0/19 • 28 Weeks
|
|
Infections and infestations
Upper respiratory tract infection
|
33.3%
7/21 • 28 Weeks
|
15.8%
3/19 • 28 Weeks
|
|
Infections and infestations
Gastroenteritis
|
4.8%
1/21 • 28 Weeks
|
5.3%
1/19 • 28 Weeks
|
|
Infections and infestations
Influenza
|
4.8%
1/21 • 28 Weeks
|
5.3%
1/19 • 28 Weeks
|
|
Infections and infestations
Sinusitis
|
4.8%
1/21 • 28 Weeks
|
5.3%
1/19 • 28 Weeks
|
|
Infections and infestations
Urinary Tract Infection
|
4.8%
1/21 • 28 Weeks
|
5.3%
1/19 • 28 Weeks
|
|
Infections and infestations
Gastroenteritis viral
|
0.00%
0/21 • 28 Weeks
|
10.5%
2/19 • 28 Weeks
|
|
Injury, poisoning and procedural complications
Procedural nausea
|
9.5%
2/21 • 28 Weeks
|
0.00%
0/19 • 28 Weeks
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/21 • 28 Weeks
|
5.3%
1/19 • 28 Weeks
|
|
Investigations
Intraocular pressure increased
|
9.5%
2/21 • 28 Weeks
|
0.00%
0/19 • 28 Weeks
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
9.5%
2/21 • 28 Weeks
|
0.00%
0/19 • 28 Weeks
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.00%
0/21 • 28 Weeks
|
5.3%
1/19 • 28 Weeks
|
|
Musculoskeletal and connective tissue disorders
Rotator cuff syndrome
|
0.00%
0/21 • 28 Weeks
|
5.3%
1/19 • 28 Weeks
|
|
Musculoskeletal and connective tissue disorders
Tendonitis
|
0.00%
0/21 • 28 Weeks
|
5.3%
1/19 • 28 Weeks
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Squamous cell carcinoma
|
0.00%
0/21 • 28 Weeks
|
5.3%
1/19 • 28 Weeks
|
|
Nervous system disorders
Headache
|
9.5%
2/21 • 28 Weeks
|
10.5%
2/19 • 28 Weeks
|
|
Nervous system disorders
Dizziness
|
4.8%
1/21 • 28 Weeks
|
10.5%
2/19 • 28 Weeks
|
|
Psychiatric disorders
Anxiety
|
0.00%
0/21 • 28 Weeks
|
5.3%
1/19 • 28 Weeks
|
|
Nervous system disorders
Insomnia
|
0.00%
0/21 • 28 Weeks
|
5.3%
1/19 • 28 Weeks
|
|
Reproductive system and breast disorders
Gynaecomastia
|
0.00%
0/21 • 28 Weeks
|
5.3%
1/19 • 28 Weeks
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
4.8%
1/21 • 28 Weeks
|
5.3%
1/19 • 28 Weeks
|
|
Skin and subcutaneous tissue disorders
Dermatitis contact
|
4.8%
1/21 • 28 Weeks
|
5.3%
1/19 • 28 Weeks
|
|
Skin and subcutaneous tissue disorders
Rash
|
4.8%
1/21 • 28 Weeks
|
5.3%
1/19 • 28 Weeks
|
|
Skin and subcutaneous tissue disorders
Dermal cyst
|
0.00%
0/21 • 28 Weeks
|
5.3%
1/19 • 28 Weeks
|
|
Skin and subcutaneous tissue disorders
Erythema
|
0.00%
0/21 • 28 Weeks
|
5.3%
1/19 • 28 Weeks
|
|
Skin and subcutaneous tissue disorders
Skin cryotherapy
|
0.00%
0/21 • 28 Weeks
|
5.3%
1/19 • 28 Weeks
|
|
Vascular disorders
Haematoma
|
0.00%
0/21 • 28 Weeks
|
5.3%
1/19 • 28 Weeks
|
|
Vascular disorders
Hypertension
|
0.00%
0/21 • 28 Weeks
|
5.3%
1/19 • 28 Weeks
|
|
Vascular disorders
Labile hypertension
|
0.00%
0/21 • 28 Weeks
|
5.3%
1/19 • 28 Weeks
|
Additional Information
Jim Carr, Pharm.D. Chief Clinical Development Officer
Stealth BioTherapeutics, Inc
Phone: 1-617-600-6888
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: GT60