Amblyopia
Conditions
Keywords
Amblyopia, patching, occlusion
Brief summary
This study is designed to evaluate the effectiveness of increasing prescribed patching treatment from 2 to 6 daily hours after visual acuity has stabilized with initial treatment and amblyopia is still present. Children ages 3 to \<8 years with visual acuity of 20/50 to 20/400 in the amblyopic eye will be enrolled in a run-in phase with 2 hours daily patching until no improvement, followed by randomization of eligible patients to patching 2 hours daily versus an average of 6 hours daily (42 hours per week). The primary objective is to determine if increasing patching dosage will improve visual acuity in patients with amblyopia still present after visual acuity has stabilized with initial treatment.
Detailed description
Amblyopia is the most common cause of monocular visual impairment in both children and young and middle-aged adults. Both patching and atropine are accepted treatment modalities for the management of moderate amblyopia in children.1 Many practitioners prescribe 2 hours daily patching as initial therapy for amblyopia. However, many children fail to achieve normal visual acuity in the amblyopic eye with this regimen. In a randomized trial conducted by PEDIG comparing patching regimens, 71 of 92 patients with moderate amblyopia (77%) had amblyopic eye visual acuity of 20/32 or worse after 4 months of patching 2 hours daily.2 In another PEDIG randomized trial comparing patching to spectacles alone after a period of refractive adaptation, patients were treated with 2 hours daily patching and followed every 5 weeks until there was no improvement in amblyopic eye acuity. Fifty-five of 70 patients with moderate amblyopia (79%) and 14 of 14 patients with severe amblyopia (100%) had best-measured amblyopic eye visual acuity of 20/32 or worse after a median treatment period of 10 weeks.3 When improvement with initial therapy stops and amblyopia is still present, treatment options include increasing the dosage of current treatment, switching to another treatment, maintaining the same treatment and dosage for additional months, or combining treatments. Many clinicians will choose to increase the dosage of the current treatment, in part because families have become comfortable with that particular mode of treatment. However, it is unknown whether increasing occlusion dosage will improve amblyopic eye visual acuity in these patients. We are unaware of any reports of response to intensified treatment of amblyopia.
Interventions
DEVICEEye Patch
42 hours patching per week (averaging 6 hours patching daily)
Sponsors
National Eye Institute (NEI)
Jaeb Center for Health Research
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
3 Years to 7 Years
Healthy volunteers
No
Inclusion criteria
Major Eligibility Criteria for Run-in Phase * Age 3 to \< 8 years * Amblyopia associated with strabismus, anisometropia, or both * Visual acuity in the amblyopic eye between 20/50 and 20/400 inclusive * Visual acuity in the sound eye 20/32 or better and inter-eye acuity difference \>3 logMAR lines * Amblyopia treatment within the past 6 months subject to the following stipulations: * No more than 6 weeks of any amblyopia treatment other than spectacles (except for patients being treated with 2 hours of patching per day who are entering the study on treatment) * No simultaneous treatment with patching and atropine * No use of atropine in combination with the sound eye spectacle lens reduced by more than 1.50 D * Maximum level of treatment within the past 6 months: * Patching: up to 2 hours daily * Atropine: up to once daily * Wearing spectacles with optimal correction (if applicable); if amblyopic eye acuity is 20/80 or better, then VA must be stable in glasses. If amblyopic eye acuity is 20/100 or worse, then spectacles and patching can be initiated simultaneously. Eligibility Criteria for Randomization: * Amblyopic eye acuity of 20/40 to 20/160 with an inter-ocular difference of \>2 lines, or amblyopic eye acuity of 20/32 with 3 lines of IOD. * Reasonable compliance with prescribed treatment, defined as wearing the patch at least 10 hours per week.
Exclusion criteria
* Currently using vision therapy or orthoptics * Ocular cause for reduced visual acuity (nystagmus per se does not exclude the patient if the above visual acuity criteria are met) * Prior intraocular or refractive surgery * Known skin reaction to patch or bandage adhesives
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Distribution of 10-week Amblyopic Eye Visual Acuity | 10 weeks after randomization | The masked 10-week amblyopic eye visual acuity scores were tabulated for both treatment groups, and included data from 10-week visual acuity exams completed between 8 to 15 weeks (inclusive) with no imputation for missing data. The primary outcome analysis followed the intent-to-treat principle. Therefore, data from randomized participants were included in the analysis regardless of whether the assigned treatment was actually received or whether they deviated from treatment against protocol. In addition, randomized participants who were found to be ineligible upon subsequent review of enrollment data were included in the primary outcome analysis. |
| Mean 10-week Amblyopic Eye Visual Acuity | 10 weeks after randomization | The primary outcome analysis was a treatment group comparison of the masked 10-week amblyopic eye visual acuity using an analysis of covariance (ANCOVA) model, adjusting for visual acuity at randomization. The analysis included data from 10-week visual acuity exams completed between 8 to 15 weeks (inclusive) with no imputation for missing data. The primary outcome analysis followed the intent-to-treat principle. Therefore, data from randomized participants were included in the analysis regardless of whether the assigned treatment was actually received or whether they deviated from treatment against protocol. In addition, randomized participants who were found to be ineligible upon subsequent review of enrollment data were included in the primary outcome analysis. |
| Distribution of the Change in Amblyopic Eye Visual Acuity at 10 Weeks From Randomization | Randomization to 10 weeks | The change in 10-week amblyopic eye visual acuity scores since randomization was tabulated for both treatment groups, and included data from 10-week visual acuity exams completed between 8 to 15 weeks (inclusive) with no imputation for missing data. The primary outcome analysis followed the intent-to-treat principle. Therefore, data from randomized participants were included in the analysis regardless of whether the assigned treatment was actually received or whether they deviated from treatment against protocol. In addition, randomized participants who were found to be ineligible upon subsequent review of enrollment data were included in the primary outcome analysis. |
| Mean Change in Amblyopic Eye Visual Acuity at 10 Weeks From Randomization | Randomization to 10 weeks | The change in 10-week amblyopic eye visual acuity was computed for both treatment groups and included data from 10-week visual acuity exams completed between 8 to 15 weeks (inclusive) with no imputation for missing data. The primary outcome analysis followed the intent-to-treat principle. Therefore, data from randomized participants were included in the analysis regardless of whether the assigned treatment was actually received or whether they deviated from treatment against protocol. In addition, randomized participants who were found to be ineligible upon subsequent review of enrollment data were included in the primary outcome analysis. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Compliance With Prescribed Patching by Treatment Group at 10 Weeks | 10 weeks after randomization | The distribution of compliance with prescribed treatment was tabulated for the 10-week outcome and as averaged scores across all study follow-up visits. Compliance was evaluated as excellent (\>75%), good (51%-75%), fair (26%-50%), or poor (\<26%) based on discussions with the parent and by reviewing study calendars maintained by the parent, who recorded the number of hours the child patched each day. |
| Average Compliance With Prescribed Patching by Treatment Group | 10 weeks after randomization or later | The distribution of compliance with prescribed treatment was tabulated for the 10-week outcome and as averaged scores across all study follow-up visits. Compliance was evaluated as excellent (\>75%), good (51%-75%), fair (26%-50%), or poor (\<26%) based on discussions with the parent and by reviewing study calendars maintained by the parent, who recorded the number of hours the child patched each day. |
| Treatment Group Comparison of the Proportion of Participants Who Have Improved by 2 or More logMAR Visual Acuity Lines at 10 Weeks Since Randomization | 10 weeks after randomization | The proportion of participants who improved at least 2 logMAR lines since randomization was computed at the 10-week outcome. The secondary outcome analysis was a treatment group comparison of the proportion of participants whose 10-week masked amblyopic eye visual acuity improved at least 2 logMAR lines since randomization using logistic regression, adjusting for visual acuity at randomization. The analysis included data from 10-week visual acuity exams completed between 8 to 15 weeks (inclusive) according to the principles specified in the primary outcome analysis. |
| Treatment Group Comparison of 10-week Interocular Difference | 10 weeks after randomization | The secondary outcome analysis was a treatment group comparison of the 10-week interocular difference (IOD), computed as the difference between the masked amblyopic and fellow eye visual acuities, using an analysis of covariance (ANCOVA) model, adjusting for IOD at randomization. The analysis included data from 10-week visual acuity exams completed between 8 to 15 weeks (inclusive) according to the principles specified in the primary outcome analysis. |
| Distribution of Baseline Characteristics at the 10-week Outcome | 10 weeks after randomization | The number of participants was tabulated by treatment group within categorical levels of prespecified baseline subgroup factors for participants with 10-week visual acuity exams completed between 8 to 15 weeks (inclusive)according to principles specified in the primary outcome analysis. |
| Mean Amblyopic Eye Visual at Randomization According to Baseline Characteristics for 10-week Outcome | 10 weeks after randomization | Mean amblyopic eye visual acuity at randomization was computed by treatment group within categorical levels of prespecified baseline subgroup factors. The analysis included data from participants with 10-week exams completed between 8 to 15 weeks (inclusive) according to the principles specified in the primary outcome analysis. |
| Treatment Comparison of Mean Amblyopic Eye Visual Acuity Change at 10-weeks According to Baseline Characteristics | 10 weeks after randomization | A treatment comparison of mean amblyopic eye visual acuity change since randomization was performed at the 10-week outcome according to categorical levels of prespecified baseline subgroup factors. The analysis included data from participants with 10-week exams completed between 8 to 15 weeks (inclusive) according to the principles specified in the primary outcome analysis. |
| Distribution of Amblyopic Eye Visual Acuity at Visit of Best Post-randomization Visual Acuity | 10 weeks after randomization or later | Participants in both groups who have improved by one or more lines from randomization to the 10-week outcome exam will continue in the study and visits will occur every 10 weeks until no improvement of one or more lines from the previous visit. The distribution of best post-randomization (10 weeks or later) visual acuity scores in the amblyopic eye was tabulated for both treatment groups using the initial visual acuity score (if a retest was obtained.) |
| Mean Amblyopic Eye Visual Acuity at Visit of Best Post-randomization Visual Acuity | 10 weeks after randomization or later | Participants in both groups who have improved by one or more lines from randomization to the 10-week outcome exam will continue in the study and visits will occur every 10 weeks until no improvement of one or more lines from the previous visit. A treatment comparison of mean amblyopic eye visual acuity at the visit of best post-randomization visual acuity (10 weeks or later) was performed using an analysis of covariance, adjusting for amblyopic eye visual acuity at randomization. |
| Distribution of the Change in Best Post-randomization Visual Acuity in the Amblyopic Eye | Randomization to 10 weeks or later | Participants in both groups who have improved by one or more lines from randomization to the 10-week outcome exam will continue in the study and visits will occur every 10 weeks until no improvement of one or more lines from the previous visit. The distribution of change in best post-randomization (10 weeks or later) visual acuity in the amblyopic eye since randomization was tabulated for both treatment groups using the initial visual acuity score (if a retest was obtained.) |
| Mean Change in Amblyopic Eye Visual Acuity Since Randomization at Visit of Best Post-randomization Visual Acuity | 10 weeks after randomization or later | Participants in both groups who have improved by one or more lines from randomization to the 10-week outcome exam will continue in the study and visits will occur every 10 weeks until no improvement of one or more lines from the previous visit. The mean change in amblyopic eye visual acuity since randomization was computed for both treatment groups based on the visit of best post-randomization visual acuity (10 weeks or later) using the initial visual acuity score (if a retest was obtained.) |
| Treatment Group Comparison of the Proportion of Participants Who Have Improved by 2 or More logMAR Visual Acuity Lines Based on Visual Acuity at Best Post-randomization Visit | 10 weeks after randomization or later | Participants in both groups who have improved by one or more lines from randomization to the 10-week outcome exam will continue in the study and visits will occur every 10 weeks until no improvement of one or more lines from the previous visit. The proportion of participants who improved at least 2 logMAR lines since randomization was computed based on the best post-randomization visual acuity in the amblyopic eye. The initial visual acuity score was used if a retest was obtained. |
| Distribution of Best Fellow Eye Visual Acuity at 10-week Outcome | 10 weeks after randomization | — |
| Mean Best Fellow Eye Visual Acuity at 10-week Outcome | 10 weeks after randomization | — |
| Distribution of Best Fellow Eye Visual Acuity at Final Visit | 10 weeks after randomization or later | — |
| Mean Best Fellow Eye Visual Acuity at Final Visit | 10 weeks after randomization or later | — |
| Distribution of Change in Best Fellow Eye Visual Acuity Since Randomization at 10 Weeks | 10 weeks after randomization | — |
| Mean Change in Best Fellow Eye Visual Acuity Since Randomization at 10 Weeks | 10 weeks after randomization | — |
| Distribution of Change in Best Fellow Eye Visual Acuity Since Randomization at Final Visit | 10 weeks after randomization or later | — |
| Mean Change in Best Fellow Eye Visual Acuity Since Randomization at Final Visit | 10 weeks after randomization or later | — |
| Distribution of Randot Preschool Stereoacuity Scores at Randomization | Randomization | The Preschool Randot test measures random dot stereoacuity from 800 to 40 arc seconds (800, 400, 200, 100, 60, 40). Lower scores indicate better stereoacuity and subjects who fail the first level (800 seconds of arc) are assigned a score of \>800. We administer a pretest, and those with a failed or uninterpretable score do not proceed with the Randot testing. The Preschool Randot test consists of 3 booklets each with 2 sets of 4 random dot shapes (one is blank, 3 are actual figures), which can be matched to non-stereo shapes on the opposite side of the booklets. There are six levels (seconds of arc) in the test with two levels in each book. Each level has 4 rectangles that contain 3 shapes and one blank. |
| Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks | 10 weeks after randomization | — |
| Change in Randot Preschool Stereoacuity Level at 10-week Outcome Since Randomization | 10 weeks after randomization | — |
| Distribution of Randot Preschool Stereoacuity Scores at Randomization for Participants With Anisometropic Amblyopia | Randomization | — |
| Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks for Participants With Anisometropic Amblyopia | 10 weeks after randomization | — |
| Change in Randot Preschool Stereoacuity Level at 10-week Outcome Since Randomization for Participants With Anisometropic Amblyopia | 10 weeks after randomization | — |
Countries
United States
Participant flow
Recruitment details
Between September 2009 and December 2012, 45 sites randomized 169 participants to either increase patching time to an average of 6 hours per day (n=86) or continue 2 hours daily patching (n=83).
Pre-assignment details
Prior to being considered for the randomized trial, participants who had not already completed at least 12 weeks of 2 hours of daily patching were enrolled into a run-in phase and treated with 2 hours of daily patching (and spectacles if needed) with follow-up every 6 weeks until no improvement.
Participants by arm
| Arm | Count |
|---|---|
| Control 2 hours daily patching | 83 |
| Intensified Treatment 42 hours per week of patching (averaging 6 hours daily) | 86 |
| Total | 169 |
Baseline characteristics
| Characteristic | Control | Intensified Treatment | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 83 Participants | 86 Participants | 169 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Continuous | 5.9 years STANDARD_DEVIATION 1.3 | 5.9 years STANDARD_DEVIATION 1.2 | 5.9 years STANDARD_DEVIATION 1.3 |
| Age, Customized 3 to <4 years | 5 participants | 8 participants | 13 participants |
| Age, Customized 4 to <5 years | 15 participants | 15 participants | 30 participants |
| Age, Customized 5 to <6 years | 27 participants | 19 participants | 46 participants |
| Age, Customized 6 to <7 years | 17 participants | 25 participants | 42 participants |
| Age, Customized 7 to 8 years | 19 participants | 19 participants | 38 participants |
| Amblyopia Cause Anisometropia | 41 participants | 34 participants | 75 participants |
| Amblyopia Cause Strabismus | 16 participants | 16 participants | 32 participants |
| Amblyopia Cause Strabismus and Anisometropia | 26 participants | 36 participants | 62 participants |
| Best distance visual acuity in the amblyopic eye at randomization 20/100 | 2 participants | 7 participants | 9 participants |
| Best distance visual acuity in the amblyopic eye at randomization 20/125 | 3 participants | 1 participants | 4 participants |
| Best distance visual acuity in the amblyopic eye at randomization 20/160 | 2 participants | 3 participants | 5 participants |
| Best distance visual acuity in the amblyopic eye at randomization 20/32 | 6 participants | 6 participants | 12 participants |
| Best distance visual acuity in the amblyopic eye at randomization 20/40 | 21 participants | 18 participants | 39 participants |
| Best distance visual acuity in the amblyopic eye at randomization 20/50 | 26 participants | 24 participants | 50 participants |
| Best distance visual acuity in the amblyopic eye at randomization 20/63 | 17 participants | 21 participants | 38 participants |
| Best distance visual acuity in the amblyopic eye at randomization 20/80 | 6 participants | 6 participants | 12 participants |
| Best distance visual acuity in the amblyopic eye at randomization | 0.43 logMAR STANDARD_DEVIATION 0.15 | 0.45 logMAR STANDARD_DEVIATION 0.16 | 0.44 logMAR STANDARD_DEVIATION 0.16 |
| Best distance visual acuity in the fellow eye at randomization 20/16 | 34 participants | 34 participants | 68 participants |
| Best distance visual acuity in the fellow eye at randomization 20/20 | 27 participants | 26 participants | 53 participants |
| Best distance visual acuity in the fellow eye at randomization 20/25 | 17 participants | 21 participants | 38 participants |
| Best distance visual acuity in the fellow eye at randomization 20/32 | 5 participants | 5 participants | 10 participants |
| Best distance visual acuity in the fellow eye at randomization | -0.01 logMAR STANDARD_DEVIATION 0.09 | 0.00 logMAR STANDARD_DEVIATION 0.09 | -0.01 logMAR STANDARD_DEVIATION 0.09 |
| Best interocular eye visual acuity difference at randomization | 4.4 logMAR lines STANDARD_DEVIATION 1.6 | 4.5 logMAR lines STANDARD_DEVIATION 1.7 | 4.5 logMAR lines STANDARD_DEVIATION 1.7 |
| Duration of patching prior to randomization (weeks) 12 to <16 weeks | 14 participants | 21 participants | 35 participants |
| Duration of patching prior to randomization (weeks) 16 to <25 weeks | 39 participants | 31 participants | 70 participants |
| Duration of patching prior to randomization (weeks) 25 weeks or more | 30 participants | 34 participants | 64 participants |
| Duration of patching prior to randomization (weeks) | 24.5 Weeks STANDARD_DEVIATION 10.7 | 24.4 Weeks STANDARD_DEVIATION 11.9 | 24.4 Weeks STANDARD_DEVIATION 11.3 |
| Enrollment Disposition No current treatment | 48 participants | 49 participants | 97 participants |
| Enrollment Disposition On treatment | 18 participants | 18 participants | 36 participants |
| Enrollment Disposition Ready for randomization | 17 participants | 19 participants | 36 participants |
| Race/Ethnicity, Customized Asian | 2 participants | 3 participants | 5 participants |
| Race/Ethnicity, Customized Black/African American | 8 participants | 2 participants | 10 participants |
| Race/Ethnicity, Customized Hispanic | 14 participants | 14 participants | 28 participants |
| Race/Ethnicity, Customized More than one race | 2 participants | 1 participants | 3 participants |
| Race/Ethnicity, Customized White | 57 participants | 66 participants | 123 participants |
| Refractive error in amblyopic eye at enrollment 0 to <+1.00D | 9 participants | 3 participants | 12 participants |
| Refractive error in amblyopic eye at enrollment +1.00 D to <+2.00 D | 5 participants | 3 participants | 8 participants |
| Refractive error in amblyopic eye at enrollment +2.00 D to <+3.00 D | 2 participants | 4 participants | 6 participants |
| Refractive error in amblyopic eye at enrollment +3.00 D to <+4.00 D | 13 participants | 6 participants | 19 participants |
| Refractive error in amblyopic eye at enrollment +4.00 D or more | 54 participants | 70 participants | 124 participants |
| Refractive error in amblyopic eye at enrollment | 4.58 Diopters STANDARD_DEVIATION 2.26 | 5.27 Diopters STANDARD_DEVIATION 1.94 | 4.93 Diopters STANDARD_DEVIATION 2.12 |
| Refractive error in fellow eye at enrollment <0 D | 1 participants | 1 participants | 2 participants |
| Refractive error in fellow eye at enrollment 0 to <+1.00D | 22 participants | 14 participants | 36 participants |
| Refractive error in fellow eye at enrollment +1.00 D to <+2.00 D | 30 participants | 19 participants | 49 participants |
| Refractive error in fellow eye at enrollment +2.00 D to <+3.00 D | 10 participants | 14 participants | 24 participants |
| Refractive error in fellow eye at enrollment +3.00 D to <+4.00 D | 4 participants | 12 participants | 16 participants |
| Refractive error in fellow eye at enrollment +4.00 D or more | 16 participants | 26 participants | 42 participants |
| Refractive error in fellow eye at enrollment | 2.10 Diopters STANDARD_DEVIATION 1.86 | 2.89 Diopters STANDARD_DEVIATION 2.06 | 2.50 Diopters STANDARD_DEVIATION 2 |
| Region of Enrollment Canada | 0 participants | 1 participants | 1 participants |
| Region of Enrollment United Kingdom | 3 participants | 3 participants | 6 participants |
| Region of Enrollment United States | 80 participants | 82 participants | 162 participants |
| Sex: Female, Male Female | 43 Participants | 43 Participants | 86 Participants |
| Sex: Female, Male Male | 40 Participants | 43 Participants | 83 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 6 / 83 | 4 / 86 |
| serious Total, serious adverse events | 0 / 83 | 0 / 86 |
Outcome results
Primary
Distribution of 10-week Amblyopic Eye Visual Acuity
The masked 10-week amblyopic eye visual acuity scores were tabulated for both treatment groups, and included data from 10-week visual acuity exams completed between 8 to 15 weeks (inclusive) with no imputation for missing data. The primary outcome analysis followed the intent-to-treat principle. Therefore, data from randomized participants were included in the analysis regardless of whether the assigned treatment was actually received or whether they deviated from treatment against protocol. In addition, randomized participants who were found to be ineligible upon subsequent review of enrollment data were included in the primary outcome analysis.
Time frame: 10 weeks after randomization
Population: The primary outcome analysis followed the intent-to-treat principle and included data from 10-week visual acuity exams completed between 8 and 15 weeks (inclusive) with no imputation for missing data.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Control | Distribution of 10-week Amblyopic Eye Visual Acuity | 20/125 | 3 participants |
| Control | Distribution of 10-week Amblyopic Eye Visual Acuity | 20/50 | 19 participants |
| Control | Distribution of 10-week Amblyopic Eye Visual Acuity | 20/160 | 0 participants |
| Control | Distribution of 10-week Amblyopic Eye Visual Acuity | 20/40 | 23 participants |
| Control | Distribution of 10-week Amblyopic Eye Visual Acuity | 20/100 | 3 participants |
| Control | Distribution of 10-week Amblyopic Eye Visual Acuity | 20/80 | 8 participants |
| Control | Distribution of 10-week Amblyopic Eye Visual Acuity | 20/25 | 3 participants |
| Control | Distribution of 10-week Amblyopic Eye Visual Acuity | 20/200 | 1 participants |
| Control | Distribution of 10-week Amblyopic Eye Visual Acuity | 20/20 | 2 participants |
| Control | Distribution of 10-week Amblyopic Eye Visual Acuity | 20/63 | 6 participants |
| Control | Distribution of 10-week Amblyopic Eye Visual Acuity | 20/16 | 0 participants |
| Control | Distribution of 10-week Amblyopic Eye Visual Acuity | 20/32 | 14 participants |
| Intensified Treatment | Distribution of 10-week Amblyopic Eye Visual Acuity | 20/16 | 0 participants |
| Intensified Treatment | Distribution of 10-week Amblyopic Eye Visual Acuity | 20/200 | 0 participants |
| Intensified Treatment | Distribution of 10-week Amblyopic Eye Visual Acuity | 20/100 | 2 participants |
| Intensified Treatment | Distribution of 10-week Amblyopic Eye Visual Acuity | 20/160 | 0 participants |
| Intensified Treatment | Distribution of 10-week Amblyopic Eye Visual Acuity | 20/125 | 3 participants |
| Intensified Treatment | Distribution of 10-week Amblyopic Eye Visual Acuity | 20/80 | 4 participants |
| Intensified Treatment | Distribution of 10-week Amblyopic Eye Visual Acuity | 20/63 | 9 participants |
| Intensified Treatment | Distribution of 10-week Amblyopic Eye Visual Acuity | 20/50 | 15 participants |
| Intensified Treatment | Distribution of 10-week Amblyopic Eye Visual Acuity | 20/40 | 20 participants |
| Intensified Treatment | Distribution of 10-week Amblyopic Eye Visual Acuity | 20/32 | 15 participants |
| Intensified Treatment | Distribution of 10-week Amblyopic Eye Visual Acuity | 20/25 | 12 participants |
| Intensified Treatment | Distribution of 10-week Amblyopic Eye Visual Acuity | 20/20 | 2 participants |
Primary
Distribution of the Change in Amblyopic Eye Visual Acuity at 10 Weeks From Randomization
The change in 10-week amblyopic eye visual acuity scores since randomization was tabulated for both treatment groups, and included data from 10-week visual acuity exams completed between 8 to 15 weeks (inclusive) with no imputation for missing data. The primary outcome analysis followed the intent-to-treat principle. Therefore, data from randomized participants were included in the analysis regardless of whether the assigned treatment was actually received or whether they deviated from treatment against protocol. In addition, randomized participants who were found to be ineligible upon subsequent review of enrollment data were included in the primary outcome analysis.
Time frame: Randomization to 10 weeks
Population: The primary outcome analysis followed the intent-to-treat principle and included data from 10-week visual acuity exams completed between 8 and 15 weeks (inclusive) with no imputation for missing data.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Control | Distribution of the Change in Amblyopic Eye Visual Acuity at 10 Weeks From Randomization | No change (0 logMAR line) | 19 participants |
| Control | Distribution of the Change in Amblyopic Eye Visual Acuity at 10 Weeks From Randomization | 3 or more logMAR lines worse | 1 participants |
| Control | Distribution of the Change in Amblyopic Eye Visual Acuity at 10 Weeks From Randomization | 2 logMAR lines worse | 3 participants |
| Control | Distribution of the Change in Amblyopic Eye Visual Acuity at 10 Weeks From Randomization | 2 logMAR lines improved | 13 participants |
| Control | Distribution of the Change in Amblyopic Eye Visual Acuity at 10 Weeks From Randomization | 1 logMAR line improved | 31 participants |
| Control | Distribution of the Change in Amblyopic Eye Visual Acuity at 10 Weeks From Randomization | 3 or more logMAR lines improved | 2 participants |
| Control | Distribution of the Change in Amblyopic Eye Visual Acuity at 10 Weeks From Randomization | 1 logMAR line worse | 13 participants |
| Intensified Treatment | Distribution of the Change in Amblyopic Eye Visual Acuity at 10 Weeks From Randomization | 3 or more logMAR lines improved | 14 participants |
| Intensified Treatment | Distribution of the Change in Amblyopic Eye Visual Acuity at 10 Weeks From Randomization | 1 logMAR line worse | 7 participants |
| Intensified Treatment | Distribution of the Change in Amblyopic Eye Visual Acuity at 10 Weeks From Randomization | 2 logMAR lines worse | 2 participants |
| Intensified Treatment | Distribution of the Change in Amblyopic Eye Visual Acuity at 10 Weeks From Randomization | No change (0 logMAR line) | 17 participants |
| Intensified Treatment | Distribution of the Change in Amblyopic Eye Visual Acuity at 10 Weeks From Randomization | 1 logMAR line improved | 23 participants |
| Intensified Treatment | Distribution of the Change in Amblyopic Eye Visual Acuity at 10 Weeks From Randomization | 2 logMAR lines improved | 19 participants |
| Intensified Treatment | Distribution of the Change in Amblyopic Eye Visual Acuity at 10 Weeks From Randomization | 3 or more logMAR lines worse | 0 participants |
Primary
Mean 10-week Amblyopic Eye Visual Acuity
The primary outcome analysis was a treatment group comparison of the masked 10-week amblyopic eye visual acuity using an analysis of covariance (ANCOVA) model, adjusting for visual acuity at randomization. The analysis included data from 10-week visual acuity exams completed between 8 to 15 weeks (inclusive) with no imputation for missing data. The primary outcome analysis followed the intent-to-treat principle. Therefore, data from randomized participants were included in the analysis regardless of whether the assigned treatment was actually received or whether they deviated from treatment against protocol. In addition, randomized participants who were found to be ineligible upon subsequent review of enrollment data were included in the primary outcome analysis.
Time frame: 10 weeks after randomization
Population: The primary outcome analysis followed the intent-to-treat principle and included data from 10-week visual acuity exams completed between 8 and 15 weeks (inclusive) with no imputation for missing data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Control | Mean 10-week Amblyopic Eye Visual Acuity | 0.38 logMAR | Standard Deviation 0.19 |
| Intensified Treatment | Mean 10-week Amblyopic Eye Visual Acuity | 0.33 logMAR | Standard Deviation 0.18 |
Comparison: The primary analysis was a treatment group comparison of the masked 10-week amblyopic eye visual acuity using an analysis of covariance (ANCOVA) model, adjusting for visual acuity at randomization. The analysis was a 2-sided test for efficacy to test the null hypothesis of no treatment difference, assuming 90% power and a type I error rate of 5%.p-value: 0.00295% CI: [0.3, 1]ANCOVA
Primary
Mean Change in Amblyopic Eye Visual Acuity at 10 Weeks From Randomization
The change in 10-week amblyopic eye visual acuity was computed for both treatment groups and included data from 10-week visual acuity exams completed between 8 to 15 weeks (inclusive) with no imputation for missing data. The primary outcome analysis followed the intent-to-treat principle. Therefore, data from randomized participants were included in the analysis regardless of whether the assigned treatment was actually received or whether they deviated from treatment against protocol. In addition, randomized participants who were found to be ineligible upon subsequent review of enrollment data were included in the primary outcome analysis.
Time frame: Randomization to 10 weeks
Population: The primary outcome analysis followed the intent-to-treat principle and included data from 10-week visual acuity exams completed between 8 and 15 weeks (inclusive) with no imputation for missing data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Control | Mean Change in Amblyopic Eye Visual Acuity at 10 Weeks From Randomization | 0.5 logMAR lines | Standard Deviation 1.2 |
| Intensified Treatment | Mean Change in Amblyopic Eye Visual Acuity at 10 Weeks From Randomization | 1.2 logMAR lines | Standard Deviation 1.4 |
Secondary
Average Compliance With Prescribed Patching by Treatment Group
The distribution of compliance with prescribed treatment was tabulated for the 10-week outcome and as averaged scores across all study follow-up visits. Compliance was evaluated as excellent (\>75%), good (51%-75%), fair (26%-50%), or poor (\<26%) based on discussions with the parent and by reviewing study calendars maintained by the parent, who recorded the number of hours the child patched each day.
Time frame: 10 weeks after randomization or later
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Control | Average Compliance With Prescribed Patching by Treatment Group | Fair | 1 participants |
| Control | Average Compliance With Prescribed Patching by Treatment Group | Excellent | 65 participants |
| Control | Average Compliance With Prescribed Patching by Treatment Group | Good | 11 participants |
| Control | Average Compliance With Prescribed Patching by Treatment Group | Poor | 2 participants |
| Control | Average Compliance With Prescribed Patching by Treatment Group | Missing (Not applicable) | 3 participants |
| Control | Average Compliance With Prescribed Patching by Treatment Group | Treatment not per protocol | 0 participants |
| Intensified Treatment | Average Compliance With Prescribed Patching by Treatment Group | Missing (Not applicable) | 0 participants |
| Intensified Treatment | Average Compliance With Prescribed Patching by Treatment Group | Fair | 4 participants |
| Intensified Treatment | Average Compliance With Prescribed Patching by Treatment Group | Poor | 4 participants |
| Intensified Treatment | Average Compliance With Prescribed Patching by Treatment Group | Excellent | 60 participants |
| Intensified Treatment | Average Compliance With Prescribed Patching by Treatment Group | Treatment not per protocol | 1 participants |
| Intensified Treatment | Average Compliance With Prescribed Patching by Treatment Group | Good | 15 participants |
Secondary
Change in Randot Preschool Stereoacuity Level at 10-week Outcome Since Randomization
Time frame: 10 weeks after randomization
Population: Change in stereoacuity level at 10 weeks was computed for participants with measureable stereoacuity at randomization and at the 10-week primary outcome exam.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Control | Change in Randot Preschool Stereoacuity Level at 10-week Outcome Since Randomization | 2 or more levels worse | 2 participants |
| Control | Change in Randot Preschool Stereoacuity Level at 10-week Outcome Since Randomization | Within 1 level | 62 participants |
| Control | Change in Randot Preschool Stereoacuity Level at 10-week Outcome Since Randomization | 2 or more levels improved | 5 participants |
| Intensified Treatment | Change in Randot Preschool Stereoacuity Level at 10-week Outcome Since Randomization | 2 or more levels improved | 7 participants |
| Intensified Treatment | Change in Randot Preschool Stereoacuity Level at 10-week Outcome Since Randomization | 2 or more levels worse | 2 participants |
| Intensified Treatment | Change in Randot Preschool Stereoacuity Level at 10-week Outcome Since Randomization | Within 1 level | 59 participants |
Comparison: An Exact Wilcoxon rank sum test was used for the difference between treatment groups in the distribution of levels of change in Randot Preschool Stereoacuity from randomization to 10 weeks.p-value: 0.28Wilcoxon (Mann-Whitney)
Secondary
Change in Randot Preschool Stereoacuity Level at 10-week Outcome Since Randomization for Participants With Anisometropic Amblyopia
Time frame: 10 weeks after randomization
Population: Change in stereoacuity level at 10 weeks was computed for participants with anisometropic amblyopia (no strabismus) who had measureable stereoacuity at randomization and at the 10-week primary outcome exam.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Control | Change in Randot Preschool Stereoacuity Level at 10-week Outcome Since Randomization for Participants With Anisometropic Amblyopia | 2 or more levels worse | 0 participants |
| Control | Change in Randot Preschool Stereoacuity Level at 10-week Outcome Since Randomization for Participants With Anisometropic Amblyopia | Within 1 level | 32 participants |
| Control | Change in Randot Preschool Stereoacuity Level at 10-week Outcome Since Randomization for Participants With Anisometropic Amblyopia | 2 or more levels improved | 3 participants |
| Intensified Treatment | Change in Randot Preschool Stereoacuity Level at 10-week Outcome Since Randomization for Participants With Anisometropic Amblyopia | 2 or more levels worse | 2 participants |
| Intensified Treatment | Change in Randot Preschool Stereoacuity Level at 10-week Outcome Since Randomization for Participants With Anisometropic Amblyopia | Within 1 level | 22 participants |
| Intensified Treatment | Change in Randot Preschool Stereoacuity Level at 10-week Outcome Since Randomization for Participants With Anisometropic Amblyopia | 2 or more levels improved | 4 participants |
Comparison: An Exact Wilcoxon rank sum test was used for the difference between treatment groups in the distribution of levels of change in Randot Preschool Stereoacuity from randomization to 10 weeks.p-value: 0.45Wilcoxon (Mann-Whitney)
Secondary
Compliance With Prescribed Patching by Treatment Group at 10 Weeks
The distribution of compliance with prescribed treatment was tabulated for the 10-week outcome and as averaged scores across all study follow-up visits. Compliance was evaluated as excellent (\>75%), good (51%-75%), fair (26%-50%), or poor (\<26%) based on discussions with the parent and by reviewing study calendars maintained by the parent, who recorded the number of hours the child patched each day.
Time frame: 10 weeks after randomization
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Control | Compliance With Prescribed Patching by Treatment Group at 10 Weeks | Good | 10 participants |
| Control | Compliance With Prescribed Patching by Treatment Group at 10 Weeks | Poor | 2 participants |
| Control | Compliance With Prescribed Patching by Treatment Group at 10 Weeks | Fair | 0 participants |
| Control | Compliance With Prescribed Patching by Treatment Group at 10 Weeks | Missing (Not applicable) | 4 participants |
| Control | Compliance With Prescribed Patching by Treatment Group at 10 Weeks | Excellent | 66 participants |
| Intensified Treatment | Compliance With Prescribed Patching by Treatment Group at 10 Weeks | Missing (Not applicable) | 1 participants |
| Intensified Treatment | Compliance With Prescribed Patching by Treatment Group at 10 Weeks | Excellent | 59 participants |
| Intensified Treatment | Compliance With Prescribed Patching by Treatment Group at 10 Weeks | Good | 14 participants |
| Intensified Treatment | Compliance With Prescribed Patching by Treatment Group at 10 Weeks | Fair | 5 participants |
| Intensified Treatment | Compliance With Prescribed Patching by Treatment Group at 10 Weeks | Poor | 4 participants |
Secondary
Distribution of Amblyopic Eye Visual Acuity at Visit of Best Post-randomization Visual Acuity
Participants in both groups who have improved by one or more lines from randomization to the 10-week outcome exam will continue in the study and visits will occur every 10 weeks until no improvement of one or more lines from the previous visit. The distribution of best post-randomization (10 weeks or later) visual acuity scores in the amblyopic eye was tabulated for both treatment groups using the initial visual acuity score (if a retest was obtained.)
Time frame: 10 weeks after randomization or later
Population: The analysis includes data from participants who completed the 10-week exam and/or a later visit.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Control | Distribution of Amblyopic Eye Visual Acuity at Visit of Best Post-randomization Visual Acuity | 20/20 | 3 participants |
| Control | Distribution of Amblyopic Eye Visual Acuity at Visit of Best Post-randomization Visual Acuity | 20/200 | 1 participants |
| Control | Distribution of Amblyopic Eye Visual Acuity at Visit of Best Post-randomization Visual Acuity | 20/160 | 0 participants |
| Control | Distribution of Amblyopic Eye Visual Acuity at Visit of Best Post-randomization Visual Acuity | 20/125 | 2 participants |
| Control | Distribution of Amblyopic Eye Visual Acuity at Visit of Best Post-randomization Visual Acuity | 20/100 | 3 participants |
| Control | Distribution of Amblyopic Eye Visual Acuity at Visit of Best Post-randomization Visual Acuity | 20/80 | 7 participants |
| Control | Distribution of Amblyopic Eye Visual Acuity at Visit of Best Post-randomization Visual Acuity | 20/63 | 7 participants |
| Control | Distribution of Amblyopic Eye Visual Acuity at Visit of Best Post-randomization Visual Acuity | 20/50 | 15 participants |
| Control | Distribution of Amblyopic Eye Visual Acuity at Visit of Best Post-randomization Visual Acuity | 20/40 | 16 participants |
| Control | Distribution of Amblyopic Eye Visual Acuity at Visit of Best Post-randomization Visual Acuity | 20/32 | 15 participants |
| Control | Distribution of Amblyopic Eye Visual Acuity at Visit of Best Post-randomization Visual Acuity | 20/25 | 12 participants |
| Control | Distribution of Amblyopic Eye Visual Acuity at Visit of Best Post-randomization Visual Acuity | 20/16 | 1 participants |
| Intensified Treatment | Distribution of Amblyopic Eye Visual Acuity at Visit of Best Post-randomization Visual Acuity | 20/80 | 4 participants |
| Intensified Treatment | Distribution of Amblyopic Eye Visual Acuity at Visit of Best Post-randomization Visual Acuity | 20/20 | 5 participants |
| Intensified Treatment | Distribution of Amblyopic Eye Visual Acuity at Visit of Best Post-randomization Visual Acuity | 20/40 | 21 participants |
| Intensified Treatment | Distribution of Amblyopic Eye Visual Acuity at Visit of Best Post-randomization Visual Acuity | 20/200 | 0 participants |
| Intensified Treatment | Distribution of Amblyopic Eye Visual Acuity at Visit of Best Post-randomization Visual Acuity | 20/63 | 7 participants |
| Intensified Treatment | Distribution of Amblyopic Eye Visual Acuity at Visit of Best Post-randomization Visual Acuity | 20/160 | 0 participants |
| Intensified Treatment | Distribution of Amblyopic Eye Visual Acuity at Visit of Best Post-randomization Visual Acuity | 20/25 | 15 participants |
| Intensified Treatment | Distribution of Amblyopic Eye Visual Acuity at Visit of Best Post-randomization Visual Acuity | 20/125 | 2 participants |
| Intensified Treatment | Distribution of Amblyopic Eye Visual Acuity at Visit of Best Post-randomization Visual Acuity | 20/50 | 11 participants |
| Intensified Treatment | Distribution of Amblyopic Eye Visual Acuity at Visit of Best Post-randomization Visual Acuity | 20/16 | 1 participants |
| Intensified Treatment | Distribution of Amblyopic Eye Visual Acuity at Visit of Best Post-randomization Visual Acuity | 20/100 | 3 participants |
| Intensified Treatment | Distribution of Amblyopic Eye Visual Acuity at Visit of Best Post-randomization Visual Acuity | 20/32 | 14 participants |
Secondary
Distribution of Baseline Characteristics at the 10-week Outcome
The number of participants was tabulated by treatment group within categorical levels of prespecified baseline subgroup factors for participants with 10-week visual acuity exams completed between 8 to 15 weeks (inclusive)according to principles specified in the primary outcome analysis.
Time frame: 10 weeks after randomization
Population: The analysis included participants who completed 10-week exams between 8 and 15 weeks (inclusive) according to the principles specified in the primary outcome analysis.
| Arm | Measure | Group | Value (NUMBER) | Dispersion |
|---|---|---|---|---|
| Control | Distribution of Baseline Characteristics at the 10-week Outcome | Cause of amblyopia: Combined mechanism | 26 participants | — |
| Control | Distribution of Baseline Characteristics at the 10-week Outcome | Age at randomization: 3 to <5 years | 20 participants | 0.17 |
| Control | Distribution of Baseline Characteristics at the 10-week Outcome | Gender: Female | 43 participants | 0.17 |
| Control | Distribution of Baseline Characteristics at the 10-week Outcome | Gender: Male | 39 participants | 0.13 |
| Control | Distribution of Baseline Characteristics at the 10-week Outcome | Race/Ethnicity: White (non-Hispanic) | 56 participants | 0.16 |
| Control | Distribution of Baseline Characteristics at the 10-week Outcome | Race/Ethnicity: Non-White/Hispanic | 26 participants | 0.14 |
| Control | Distribution of Baseline Characteristics at the 10-week Outcome | Age at randomization: 5 years or older | 62 participants | 0.11 |
| Control | Distribution of Baseline Characteristics at the 10-week Outcome | Amblyopic eye VA at randomization: 20/80 or worse | 12 participants | — |
| Control | Distribution of Baseline Characteristics at the 10-week Outcome | Amblyopic eye VA at randomization: 20/63 | 17 participants | — |
| Control | Distribution of Baseline Characteristics at the 10-week Outcome | Amblyopic eye VA at randomization: 20/50 | 26 participants | — |
| Control | Distribution of Baseline Characteristics at the 10-week Outcome | Amblyopic eye VA at randomization: 20/40 or better | 27 participants | — |
| Control | Distribution of Baseline Characteristics at the 10-week Outcome | Cause of amblyopia: Strabismus | 16 participants | — |
| Control | Distribution of Baseline Characteristics at the 10-week Outcome | Cause of amblyopia: Anisometropia | 40 participants | — |
| Intensified Treatment | Distribution of Baseline Characteristics at the 10-week Outcome | Amblyopic eye VA at randomization: 20/40 or better | 22 participants | — |
| Intensified Treatment | Distribution of Baseline Characteristics at the 10-week Outcome | Amblyopic eye VA at randomization: 20/80 or worse | 15 participants | — |
| Intensified Treatment | Distribution of Baseline Characteristics at the 10-week Outcome | Cause of amblyopia: Combined mechanism | 36 participants | — |
| Intensified Treatment | Distribution of Baseline Characteristics at the 10-week Outcome | Cause of amblyopia: Anisometropia | 31 participants | — |
| Intensified Treatment | Distribution of Baseline Characteristics at the 10-week Outcome | Gender: Female | 41 participants | 0.16 |
| Intensified Treatment | Distribution of Baseline Characteristics at the 10-week Outcome | Amblyopic eye VA at randomization: 20/63 | 21 participants | — |
| Intensified Treatment | Distribution of Baseline Characteristics at the 10-week Outcome | Gender: Male | 41 participants | 0.14 |
| Intensified Treatment | Distribution of Baseline Characteristics at the 10-week Outcome | Cause of amblyopia: Strabismus | 15 participants | — |
| Intensified Treatment | Distribution of Baseline Characteristics at the 10-week Outcome | Race/Ethnicity: White (non-Hispanic) | 63 participants | 0.14 |
| Intensified Treatment | Distribution of Baseline Characteristics at the 10-week Outcome | Amblyopic eye VA at randomization: 20/50 | 24 participants | — |
| Intensified Treatment | Distribution of Baseline Characteristics at the 10-week Outcome | Age at randomization: 3 to <5 years | 23 participants | 0.15 |
| Intensified Treatment | Distribution of Baseline Characteristics at the 10-week Outcome | Race/Ethnicity: Non-White/Hispanic | 19 participants | 0.17 |
| Intensified Treatment | Distribution of Baseline Characteristics at the 10-week Outcome | Age at randomization: 5 years or older | 59 participants | — |
Secondary
Distribution of Best Fellow Eye Visual Acuity at 10-week Outcome
Time frame: 10 weeks after randomization
Population: The analysis includes data from participants who completed a 10-week exam.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Control | Distribution of Best Fellow Eye Visual Acuity at 10-week Outcome | 20/40 | 1 participants |
| Control | Distribution of Best Fellow Eye Visual Acuity at 10-week Outcome | 20/25 | 19 participants |
| Control | Distribution of Best Fellow Eye Visual Acuity at 10-week Outcome | 20/50 | 1 participants |
| Control | Distribution of Best Fellow Eye Visual Acuity at 10-week Outcome | 20/20 | 20 participants |
| Control | Distribution of Best Fellow Eye Visual Acuity at 10-week Outcome | 20/32 | 5 participants |
| Control | Distribution of Best Fellow Eye Visual Acuity at 10-week Outcome | 20/16 | 36 participants |
| Control | Distribution of Best Fellow Eye Visual Acuity at 10-week Outcome | 20/63 | 0 participants |
| Intensified Treatment | Distribution of Best Fellow Eye Visual Acuity at 10-week Outcome | 20/16 | 30 participants |
| Intensified Treatment | Distribution of Best Fellow Eye Visual Acuity at 10-week Outcome | 20/63 | 0 participants |
| Intensified Treatment | Distribution of Best Fellow Eye Visual Acuity at 10-week Outcome | 20/50 | 0 participants |
| Intensified Treatment | Distribution of Best Fellow Eye Visual Acuity at 10-week Outcome | 20/40 | 0 participants |
| Intensified Treatment | Distribution of Best Fellow Eye Visual Acuity at 10-week Outcome | 20/32 | 8 participants |
| Intensified Treatment | Distribution of Best Fellow Eye Visual Acuity at 10-week Outcome | 20/25 | 14 participants |
| Intensified Treatment | Distribution of Best Fellow Eye Visual Acuity at 10-week Outcome | 20/20 | 31 participants |
Secondary
Distribution of Best Fellow Eye Visual Acuity at Final Visit
Time frame: 10 weeks after randomization or later
Population: The analysis includes data from participants who completed the 10-week exam and/or a later visit.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Control | Distribution of Best Fellow Eye Visual Acuity at Final Visit | 20/40 | 1 participants |
| Control | Distribution of Best Fellow Eye Visual Acuity at Final Visit | 20/25 | 15 participants |
| Control | Distribution of Best Fellow Eye Visual Acuity at Final Visit | 20/50 | 1 participants |
| Control | Distribution of Best Fellow Eye Visual Acuity at Final Visit | 20/20 | 20 participants |
| Control | Distribution of Best Fellow Eye Visual Acuity at Final Visit | 20/32 | 4 participants |
| Control | Distribution of Best Fellow Eye Visual Acuity at Final Visit | 20/16 | 40 participants |
| Control | Distribution of Best Fellow Eye Visual Acuity at Final Visit | 20/63 | 1 participants |
| Intensified Treatment | Distribution of Best Fellow Eye Visual Acuity at Final Visit | 20/16 | 32 participants |
| Intensified Treatment | Distribution of Best Fellow Eye Visual Acuity at Final Visit | 20/63 | 0 participants |
| Intensified Treatment | Distribution of Best Fellow Eye Visual Acuity at Final Visit | 20/50 | 0 participants |
| Intensified Treatment | Distribution of Best Fellow Eye Visual Acuity at Final Visit | 20/40 | 0 participants |
| Intensified Treatment | Distribution of Best Fellow Eye Visual Acuity at Final Visit | 20/32 | 5 participants |
| Intensified Treatment | Distribution of Best Fellow Eye Visual Acuity at Final Visit | 20/25 | 18 participants |
| Intensified Treatment | Distribution of Best Fellow Eye Visual Acuity at Final Visit | 20/20 | 28 participants |
Secondary
Distribution of Change in Best Fellow Eye Visual Acuity Since Randomization at 10 Weeks
Time frame: 10 weeks after randomization
Population: The analysis includes data from participants who completed a 10-week exam.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Control | Distribution of Change in Best Fellow Eye Visual Acuity Since Randomization at 10 Weeks | 1 logMAR line worse | 14 participants |
| Control | Distribution of Change in Best Fellow Eye Visual Acuity Since Randomization at 10 Weeks | 1 logMAR line improved | 8 participants |
| Control | Distribution of Change in Best Fellow Eye Visual Acuity Since Randomization at 10 Weeks | 2 logMAR lines worse | 0 participants |
| Control | Distribution of Change in Best Fellow Eye Visual Acuity Since Randomization at 10 Weeks | 2 logMAR lines improved | 1 participants |
| Control | Distribution of Change in Best Fellow Eye Visual Acuity Since Randomization at 10 Weeks | No change (0 logMAR line) | 58 participants |
| Control | Distribution of Change in Best Fellow Eye Visual Acuity Since Randomization at 10 Weeks | 3 or more logMAR lines improved | 0 participants |
| Control | Distribution of Change in Best Fellow Eye Visual Acuity Since Randomization at 10 Weeks | 3 or more logMAR lines worse | 1 participants |
| Intensified Treatment | Distribution of Change in Best Fellow Eye Visual Acuity Since Randomization at 10 Weeks | 3 or more logMAR lines improved | 0 participants |
| Intensified Treatment | Distribution of Change in Best Fellow Eye Visual Acuity Since Randomization at 10 Weeks | 3 or more logMAR lines worse | 1 participants |
| Intensified Treatment | Distribution of Change in Best Fellow Eye Visual Acuity Since Randomization at 10 Weeks | 2 logMAR lines worse | 0 participants |
| Intensified Treatment | Distribution of Change in Best Fellow Eye Visual Acuity Since Randomization at 10 Weeks | 1 logMAR line worse | 16 participants |
| Intensified Treatment | Distribution of Change in Best Fellow Eye Visual Acuity Since Randomization at 10 Weeks | No change (0 logMAR line) | 49 participants |
| Intensified Treatment | Distribution of Change in Best Fellow Eye Visual Acuity Since Randomization at 10 Weeks | 1 logMAR line improved | 17 participants |
| Intensified Treatment | Distribution of Change in Best Fellow Eye Visual Acuity Since Randomization at 10 Weeks | 2 logMAR lines improved | 0 participants |
Comparison: Fisher's exact test was used to compare the proportion of participants in each treatment group with a loss of 2 or more lines in the better of the initial test and retest (if indicated) fellow eye visual acuities at the 10-week exam.p-value: >0.99Fisher Exact
Secondary
Distribution of Change in Best Fellow Eye Visual Acuity Since Randomization at Final Visit
Time frame: 10 weeks after randomization or later
Population: The analysis includes data from participants who completed the 10-week exam and/or a later visit.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Control | Distribution of Change in Best Fellow Eye Visual Acuity Since Randomization at Final Visit | 3 or more logMAR lines improved | 0 participants |
| Control | Distribution of Change in Best Fellow Eye Visual Acuity Since Randomization at Final Visit | 2 logMAR lines worse | 1 participants |
| Control | Distribution of Change in Best Fellow Eye Visual Acuity Since Randomization at Final Visit | 2 logMAR lines improved | 2 participants |
| Control | Distribution of Change in Best Fellow Eye Visual Acuity Since Randomization at Final Visit | 1 logMAR line worse | 11 participants |
| Control | Distribution of Change in Best Fellow Eye Visual Acuity Since Randomization at Final Visit | 3 or more logMAR lines worse | 2 participants |
| Control | Distribution of Change in Best Fellow Eye Visual Acuity Since Randomization at Final Visit | No change (0 logMAR line) | 53 participants |
| Control | Distribution of Change in Best Fellow Eye Visual Acuity Since Randomization at Final Visit | 1 logMAR line improved | 13 participants |
| Intensified Treatment | Distribution of Change in Best Fellow Eye Visual Acuity Since Randomization at Final Visit | No change (0 logMAR line) | 50 participants |
| Intensified Treatment | Distribution of Change in Best Fellow Eye Visual Acuity Since Randomization at Final Visit | 1 logMAR line improved | 16 participants |
| Intensified Treatment | Distribution of Change in Best Fellow Eye Visual Acuity Since Randomization at Final Visit | 2 logMAR lines improved | 1 participants |
| Intensified Treatment | Distribution of Change in Best Fellow Eye Visual Acuity Since Randomization at Final Visit | 3 or more logMAR lines improved | 0 participants |
| Intensified Treatment | Distribution of Change in Best Fellow Eye Visual Acuity Since Randomization at Final Visit | 3 or more logMAR lines worse | 0 participants |
| Intensified Treatment | Distribution of Change in Best Fellow Eye Visual Acuity Since Randomization at Final Visit | 2 logMAR lines worse | 0 participants |
| Intensified Treatment | Distribution of Change in Best Fellow Eye Visual Acuity Since Randomization at Final Visit | 1 logMAR line worse | 16 participants |
Comparison: Fisher's exact test was used to compare the proportion of participants in each treatment group with a loss of 2 or more lines in the better of the initial test and retest (if indicated) fellow eye visual acuities at the final exam.p-value: 0.12Fisher Exact
Secondary
Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks
Time frame: 10 weeks after randomization
Population: The analysis includes all participants who completed the 10-week exam.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Control | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks | Not done | 0 participants |
| Control | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks | 200 seconds of arc | 5 participants |
| Control | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks | 800 seconds of arc | 13 participants |
| Control | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks | 100 seconds of arc | 11 participants |
| Control | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks | > 800 seconds of arc | 33 participants |
| Control | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks | 60 seconds of arc | 0 participants |
| Control | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks | 400 seconds of arc | 10 participants |
| Control | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks | 40 seconds of arc | 2 participants |
| Control | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks | Failed pretest | 8 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks | 40 seconds of arc | 1 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks | Failed pretest | 7 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks | Not done | 1 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks | > 800 seconds of arc | 44 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks | 800 seconds of arc | 10 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks | 400 seconds of arc | 12 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks | 200 seconds of arc | 6 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks | 100 seconds of arc | 0 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks | 60 seconds of arc | 2 participants |
Secondary
Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks for Participants With Anisometropic Amblyopia
Time frame: 10 weeks after randomization
Population: The analysis includes participants with anisometropic amblyopia (no strabismus) who completed the 10-week exam.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Control | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks for Participants With Anisometropic Amblyopia | 800 seconds of arc | 7 participants |
| Control | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks for Participants With Anisometropic Amblyopia | Failed pretest | 3 participants |
| Control | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks for Participants With Anisometropic Amblyopia | 400 seconds of arc | 6 participants |
| Control | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks for Participants With Anisometropic Amblyopia | 100 seconds of arc | 7 participants |
| Control | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks for Participants With Anisometropic Amblyopia | 200 seconds of arc | 4 participants |
| Control | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks for Participants With Anisometropic Amblyopia | 40 seconds of arc | 2 participants |
| Control | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks for Participants With Anisometropic Amblyopia | > 800 seconds of arc | 11 participants |
| Control | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks for Participants With Anisometropic Amblyopia | Not done | 0 participants |
| Control | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks for Participants With Anisometropic Amblyopia | 60 seconds of arc | 0 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks for Participants With Anisometropic Amblyopia | Not done | 0 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks for Participants With Anisometropic Amblyopia | > 800 seconds of arc | 17 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks for Participants With Anisometropic Amblyopia | 800 seconds of arc | 4 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks for Participants With Anisometropic Amblyopia | 200 seconds of arc | 3 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks for Participants With Anisometropic Amblyopia | 100 seconds of arc | 0 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks for Participants With Anisometropic Amblyopia | 60 seconds of arc | 1 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks for Participants With Anisometropic Amblyopia | 400 seconds of arc | 4 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks for Participants With Anisometropic Amblyopia | 40 seconds of arc | 1 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at 10 Weeks for Participants With Anisometropic Amblyopia | Failed pretest | 2 participants |
Secondary
Distribution of Randot Preschool Stereoacuity Scores at Randomization
The Preschool Randot test measures random dot stereoacuity from 800 to 40 arc seconds (800, 400, 200, 100, 60, 40). Lower scores indicate better stereoacuity and subjects who fail the first level (800 seconds of arc) are assigned a score of \>800. We administer a pretest, and those with a failed or uninterpretable score do not proceed with the Randot testing. The Preschool Randot test consists of 3 booklets each with 2 sets of 4 random dot shapes (one is blank, 3 are actual figures), which can be matched to non-stereo shapes on the opposite side of the booklets. There are six levels (seconds of arc) in the test with two levels in each book. Each level has 4 rectangles that contain 3 shapes and one blank.
Time frame: Randomization
Population: The analysis includes all participants who completed the 10-week exam.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Control | Distribution of Randot Preschool Stereoacuity Scores at Randomization | 40 seconds of arc | 2 participants |
| Control | Distribution of Randot Preschool Stereoacuity Scores at Randomization | Failed pretest | 10 participants |
| Control | Distribution of Randot Preschool Stereoacuity Scores at Randomization | > 800 seconds of arc | 39 participants |
| Control | Distribution of Randot Preschool Stereoacuity Scores at Randomization | 800 seconds of arc | 10 participants |
| Control | Distribution of Randot Preschool Stereoacuity Scores at Randomization | 400 seconds of arc | 6 participants |
| Control | Distribution of Randot Preschool Stereoacuity Scores at Randomization | 200 seconds of arc | 4 participants |
| Control | Distribution of Randot Preschool Stereoacuity Scores at Randomization | 100 seconds of arc | 7 participants |
| Control | Distribution of Randot Preschool Stereoacuity Scores at Randomization | 60 seconds of arc | 2 participants |
| Control | Distribution of Randot Preschool Stereoacuity Scores at Randomization | Not done | 2 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at Randomization | 60 seconds of arc | 0 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at Randomization | 200 seconds of arc | 7 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at Randomization | Failed pretest | 7 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at Randomization | 40 seconds of arc | 2 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at Randomization | > 800 seconds of arc | 47 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at Randomization | 100 seconds of arc | 1 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at Randomization | 800 seconds of arc | 8 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at Randomization | Not done | 6 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at Randomization | 400 seconds of arc | 5 participants |
Secondary
Distribution of Randot Preschool Stereoacuity Scores at Randomization for Participants With Anisometropic Amblyopia
Time frame: Randomization
Population: The analysis includes participants with anisometropic amblyopia (no strabismus) who completed the 10-week exam.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Control | Distribution of Randot Preschool Stereoacuity Scores at Randomization for Participants With Anisometropic Amblyopia | > 800 seconds of arc | 15 participants |
| Control | Distribution of Randot Preschool Stereoacuity Scores at Randomization for Participants With Anisometropic Amblyopia | 100 seconds of arc | 3 participants |
| Control | Distribution of Randot Preschool Stereoacuity Scores at Randomization for Participants With Anisometropic Amblyopia | 400 seconds of arc | 5 participants |
| Control | Distribution of Randot Preschool Stereoacuity Scores at Randomization for Participants With Anisometropic Amblyopia | 60 seconds of arc | 2 participants |
| Control | Distribution of Randot Preschool Stereoacuity Scores at Randomization for Participants With Anisometropic Amblyopia | 800 seconds of arc | 7 participants |
| Control | Distribution of Randot Preschool Stereoacuity Scores at Randomization for Participants With Anisometropic Amblyopia | 40 seconds of arc | 1 participants |
| Control | Distribution of Randot Preschool Stereoacuity Scores at Randomization for Participants With Anisometropic Amblyopia | 200 seconds of arc | 3 participants |
| Control | Distribution of Randot Preschool Stereoacuity Scores at Randomization for Participants With Anisometropic Amblyopia | Not done | 1 participants |
| Control | Distribution of Randot Preschool Stereoacuity Scores at Randomization for Participants With Anisometropic Amblyopia | Failed pretest | 3 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at Randomization for Participants With Anisometropic Amblyopia | Not done | 1 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at Randomization for Participants With Anisometropic Amblyopia | Failed pretest | 3 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at Randomization for Participants With Anisometropic Amblyopia | > 800 seconds of arc | 17 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at Randomization for Participants With Anisometropic Amblyopia | 800 seconds of arc | 3 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at Randomization for Participants With Anisometropic Amblyopia | 400 seconds of arc | 2 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at Randomization for Participants With Anisometropic Amblyopia | 200 seconds of arc | 3 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at Randomization for Participants With Anisometropic Amblyopia | 100 seconds of arc | 1 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at Randomization for Participants With Anisometropic Amblyopia | 60 seconds of arc | 0 participants |
| Intensified Treatment | Distribution of Randot Preschool Stereoacuity Scores at Randomization for Participants With Anisometropic Amblyopia | 40 seconds of arc | 2 participants |
Secondary
Distribution of the Change in Best Post-randomization Visual Acuity in the Amblyopic Eye
Participants in both groups who have improved by one or more lines from randomization to the 10-week outcome exam will continue in the study and visits will occur every 10 weeks until no improvement of one or more lines from the previous visit. The distribution of change in best post-randomization (10 weeks or later) visual acuity in the amblyopic eye since randomization was tabulated for both treatment groups using the initial visual acuity score (if a retest was obtained.)
Time frame: Randomization to 10 weeks or later
Population: The analysis includes data from participants who completed the 10-week exam and/or a later visit.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Control | Distribution of the Change in Best Post-randomization Visual Acuity in the Amblyopic Eye | 1 logMAR line worse | 12 participants |
| Control | Distribution of the Change in Best Post-randomization Visual Acuity in the Amblyopic Eye | 1 logMAR line improved | 21 participants |
| Control | Distribution of the Change in Best Post-randomization Visual Acuity in the Amblyopic Eye | 2 logMAR lines worse | 3 participants |
| Control | Distribution of the Change in Best Post-randomization Visual Acuity in the Amblyopic Eye | 2 logMAR lines improved | 22 participants |
| Control | Distribution of the Change in Best Post-randomization Visual Acuity in the Amblyopic Eye | No change (0 logMAR line) | 13 participants |
| Control | Distribution of the Change in Best Post-randomization Visual Acuity in the Amblyopic Eye | 3 or more logMAR lines improved | 10 participants |
| Control | Distribution of the Change in Best Post-randomization Visual Acuity in the Amblyopic Eye | 3 or more logMAR lines worse | 1 participants |
| Intensified Treatment | Distribution of the Change in Best Post-randomization Visual Acuity in the Amblyopic Eye | 3 or more logMAR lines improved | 21 participants |
| Intensified Treatment | Distribution of the Change in Best Post-randomization Visual Acuity in the Amblyopic Eye | 3 or more logMAR lines worse | 0 participants |
| Intensified Treatment | Distribution of the Change in Best Post-randomization Visual Acuity in the Amblyopic Eye | 2 logMAR lines worse | 2 participants |
| Intensified Treatment | Distribution of the Change in Best Post-randomization Visual Acuity in the Amblyopic Eye | 1 logMAR line worse | 6 participants |
| Intensified Treatment | Distribution of the Change in Best Post-randomization Visual Acuity in the Amblyopic Eye | No change (0 logMAR line) | 17 participants |
| Intensified Treatment | Distribution of the Change in Best Post-randomization Visual Acuity in the Amblyopic Eye | 1 logMAR line improved | 16 participants |
| Intensified Treatment | Distribution of the Change in Best Post-randomization Visual Acuity in the Amblyopic Eye | 2 logMAR lines improved | 21 participants |
Secondary
Mean Amblyopic Eye Visual Acuity at Visit of Best Post-randomization Visual Acuity
Participants in both groups who have improved by one or more lines from randomization to the 10-week outcome exam will continue in the study and visits will occur every 10 weeks until no improvement of one or more lines from the previous visit. A treatment comparison of mean amblyopic eye visual acuity at the visit of best post-randomization visual acuity (10 weeks or later) was performed using an analysis of covariance, adjusting for amblyopic eye visual acuity at randomization.
Time frame: 10 weeks after randomization or later
Population: The primary outcome analysis followed the intent-to-treat principle and included data from 10-week visual acuity exams completed between 8 and 15 weeks (inclusive) with no imputation for missing data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Control | Mean Amblyopic Eye Visual Acuity at Visit of Best Post-randomization Visual Acuity | 0.33 logMAR | Standard Deviation 0.21 |
| Intensified Treatment | Mean Amblyopic Eye Visual Acuity at Visit of Best Post-randomization Visual Acuity | 0.30 logMAR | Standard Deviation 0.19 |
95% CI: [0.04, 1]ANCOVA
Secondary
Mean Amblyopic Eye Visual at Randomization According to Baseline Characteristics for 10-week Outcome
Mean amblyopic eye visual acuity at randomization was computed by treatment group within categorical levels of prespecified baseline subgroup factors. The analysis included data from participants with 10-week exams completed between 8 to 15 weeks (inclusive) according to the principles specified in the primary outcome analysis.
Time frame: 10 weeks after randomization
Population: The analysis included participants who completed 10-week exams between 8 and 15 weeks (inclusive) according to the principles of the primary outcome analysis.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Control | Mean Amblyopic Eye Visual at Randomization According to Baseline Characteristics for 10-week Outcome | Gender: Male | 0.41 logMAR | Standard Deviation 0.13 |
| Control | Mean Amblyopic Eye Visual at Randomization According to Baseline Characteristics for 10-week Outcome | Race/Ethnicity: White (non-Hispanic) | 0.42 logMAR | Standard Deviation 0.16 |
| Control | Mean Amblyopic Eye Visual at Randomization According to Baseline Characteristics for 10-week Outcome | Cause of amblyopia: Anisometropia | 0.39 logMAR | Standard Deviation 0.11 |
| Control | Mean Amblyopic Eye Visual at Randomization According to Baseline Characteristics for 10-week Outcome | Gender: Female | 0.44 logMAR | Standard Deviation 0.17 |
| Control | Mean Amblyopic Eye Visual at Randomization According to Baseline Characteristics for 10-week Outcome | Race/Ethnicity: Non-White/Hispanic | 0.43 logMAR | Standard Deviation 0.14 |
| Control | Mean Amblyopic Eye Visual at Randomization According to Baseline Characteristics for 10-week Outcome | Age at randomization: 3 to <5 years | 0.45 logMAR | Standard Deviation 0.14 |
| Control | Mean Amblyopic Eye Visual at Randomization According to Baseline Characteristics for 10-week Outcome | Age at randomization: 5 years or older | 0.42 logMAR | Standard Deviation 0.16 |
| Control | Mean Amblyopic Eye Visual at Randomization According to Baseline Characteristics for 10-week Outcome | Cause of amblyopia: Strabismus | 0.46 logMAR | Standard Deviation 0.17 |
| Control | Mean Amblyopic Eye Visual at Randomization According to Baseline Characteristics for 10-week Outcome | Cause of amblyopia: Combined mechanism | 0.47 logMAR | Standard Deviation 0.19 |
| Intensified Treatment | Mean Amblyopic Eye Visual at Randomization According to Baseline Characteristics for 10-week Outcome | Cause of amblyopia: Strabismus | 0.43 logMAR | Standard Deviation 0.15 |
| Intensified Treatment | Mean Amblyopic Eye Visual at Randomization According to Baseline Characteristics for 10-week Outcome | Gender: Male | 0.44 logMAR | Standard Deviation 0.14 |
| Intensified Treatment | Mean Amblyopic Eye Visual at Randomization According to Baseline Characteristics for 10-week Outcome | Age at randomization: 3 to <5 years | 0.44 logMAR | Standard Deviation 0.12 |
| Intensified Treatment | Mean Amblyopic Eye Visual at Randomization According to Baseline Characteristics for 10-week Outcome | Race/Ethnicity: White (non-Hispanic) | 0.45 logMAR | Standard Deviation 0.14 |
| Intensified Treatment | Mean Amblyopic Eye Visual at Randomization According to Baseline Characteristics for 10-week Outcome | Cause of amblyopia: Anisometropia | 0.43 logMAR | Standard Deviation 0.13 |
| Intensified Treatment | Mean Amblyopic Eye Visual at Randomization According to Baseline Characteristics for 10-week Outcome | Age at randomization: 5 years or older | 0.45 logMAR | Standard Deviation 0.16 |
| Intensified Treatment | Mean Amblyopic Eye Visual at Randomization According to Baseline Characteristics for 10-week Outcome | Gender: Female | 0.45 logMAR | Standard Deviation 0.16 |
| Intensified Treatment | Mean Amblyopic Eye Visual at Randomization According to Baseline Characteristics for 10-week Outcome | Cause of amblyopia: Combined mechanism | 0.46 logMAR | Standard Deviation 0.16 |
| Intensified Treatment | Mean Amblyopic Eye Visual at Randomization According to Baseline Characteristics for 10-week Outcome | Race/Ethnicity: Non-White/Hispanic | 0.43 logMAR | Standard Deviation 0.17 |
Secondary
Mean Best Fellow Eye Visual Acuity at 10-week Outcome
Time frame: 10 weeks after randomization
Population: The analysis includes data from participants who completed a 10-week exam.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Control | Mean Best Fellow Eye Visual Acuity at 10-week Outcome | 0.00 logMAR | Standard Deviation 0.11 |
| Intensified Treatment | Mean Best Fellow Eye Visual Acuity at 10-week Outcome | 0.00 logMAR | Standard Deviation 0.1 |
Secondary
Mean Best Fellow Eye Visual Acuity at Final Visit
Time frame: 10 weeks after randomization or later
Population: The analysis includes data from participants who completed the 10-week exam and/or a later visit.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Control | Mean Best Fellow Eye Visual Acuity at Final Visit | -0.01 logMAR | Standard Deviation 0.12 |
| Intensified Treatment | Mean Best Fellow Eye Visual Acuity at Final Visit | 0.00 logMAR | Standard Deviation 0.09 |
Secondary
Mean Change in Amblyopic Eye Visual Acuity Since Randomization at Visit of Best Post-randomization Visual Acuity
Participants in both groups who have improved by one or more lines from randomization to the 10-week outcome exam will continue in the study and visits will occur every 10 weeks until no improvement of one or more lines from the previous visit. The mean change in amblyopic eye visual acuity since randomization was computed for both treatment groups based on the visit of best post-randomization visual acuity (10 weeks or later) using the initial visual acuity score (if a retest was obtained.)
Time frame: 10 weeks after randomization or later
Population: The analysis includes data from participants who completed the 10-week exam and/or a later visit.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Control | Mean Change in Amblyopic Eye Visual Acuity Since Randomization at Visit of Best Post-randomization Visual Acuity | 0.9 logMAR lines | Standard Deviation 1.5 |
| Intensified Treatment | Mean Change in Amblyopic Eye Visual Acuity Since Randomization at Visit of Best Post-randomization Visual Acuity | 1.5 logMAR lines | Standard Deviation 1.6 |
Secondary
Mean Change in Best Fellow Eye Visual Acuity Since Randomization at 10 Weeks
Time frame: 10 weeks after randomization
Population: The analysis includes data from participants who completed a 10-week exam.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Control | Mean Change in Best Fellow Eye Visual Acuity Since Randomization at 10 Weeks | -0.10 logMAR lines | Standard Deviation 0.7 |
| Intensified Treatment | Mean Change in Best Fellow Eye Visual Acuity Since Randomization at 10 Weeks | -0.02 logMAR lines | Standard Deviation 0.7 |
Secondary
Mean Change in Best Fellow Eye Visual Acuity Since Randomization at Final Visit
Time frame: 10 weeks after randomization or later
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Control | Mean Change in Best Fellow Eye Visual Acuity Since Randomization at Final Visit | -0.04 logMAR lines | Standard Deviation 0.9 |
| Intensified Treatment | Mean Change in Best Fellow Eye Visual Acuity Since Randomization at Final Visit | 0.02 logMAR lines | Standard Deviation 0.7 |
Secondary
Treatment Comparison of Mean Amblyopic Eye Visual Acuity Change at 10-weeks According to Baseline Characteristics
A treatment comparison of mean amblyopic eye visual acuity change since randomization was performed at the 10-week outcome according to categorical levels of prespecified baseline subgroup factors. The analysis included data from participants with 10-week exams completed between 8 to 15 weeks (inclusive) according to the principles specified in the primary outcome analysis.
Time frame: 10 weeks after randomization
Population: The analysis included participants who completed 10-week exams between 8 and 15 weeks (inclusive) according to the principles of the primary outcome analysis.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Control | Treatment Comparison of Mean Amblyopic Eye Visual Acuity Change at 10-weeks According to Baseline Characteristics | Amblyopic eye VA at randomization: 20/80 or worse | 0.7 units on a scale | Standard Deviation 1.4 |
| Control | Treatment Comparison of Mean Amblyopic Eye Visual Acuity Change at 10-weeks According to Baseline Characteristics | Age at randomization: 5 years or older | 0.4 units on a scale | Standard Deviation 1.2 |
| Control | Treatment Comparison of Mean Amblyopic Eye Visual Acuity Change at 10-weeks According to Baseline Characteristics | Amblyopic eye VA at randomization: 20/63 | 0.7 units on a scale | Standard Deviation 1.3 |
| Control | Treatment Comparison of Mean Amblyopic Eye Visual Acuity Change at 10-weeks According to Baseline Characteristics | Race/Ethnicity: White (non-Hispanic) | 0.5 units on a scale | Standard Deviation 1 |
| Control | Treatment Comparison of Mean Amblyopic Eye Visual Acuity Change at 10-weeks According to Baseline Characteristics | Amblyopic eye VA at randomization: 20/50 | 0.4 units on a scale | Standard Deviation 1.2 |
| Control | Treatment Comparison of Mean Amblyopic Eye Visual Acuity Change at 10-weeks According to Baseline Characteristics | Gender: Male | 0.7 units on a scale | Standard Deviation 1.1 |
| Control | Treatment Comparison of Mean Amblyopic Eye Visual Acuity Change at 10-weeks According to Baseline Characteristics | Race/Ethnicity: Non-White/Hispanic | 0.5 units on a scale | Standard Deviation 1.6 |
| Control | Treatment Comparison of Mean Amblyopic Eye Visual Acuity Change at 10-weeks According to Baseline Characteristics | Cause of amblyopia: Strabismus | 1.1 units on a scale | Standard Deviation 1.2 |
| Control | Treatment Comparison of Mean Amblyopic Eye Visual Acuity Change at 10-weeks According to Baseline Characteristics | Amblyopic eye VA at randomization: 20/40 or better | 0.4 units on a scale | Standard Deviation 1.1 |
| Control | Treatment Comparison of Mean Amblyopic Eye Visual Acuity Change at 10-weeks According to Baseline Characteristics | Cause of amblyopia: Anisometropia | 0.3 units on a scale | Standard Deviation 1.2 |
| Control | Treatment Comparison of Mean Amblyopic Eye Visual Acuity Change at 10-weeks According to Baseline Characteristics | Age at randomization: 3 to <5 years | 0.9 units on a scale | Standard Deviation 1 |
| Control | Treatment Comparison of Mean Amblyopic Eye Visual Acuity Change at 10-weeks According to Baseline Characteristics | Cause of amblyopia: Combined mechanism | 0.5 units on a scale | Standard Deviation 1.1 |
| Control | Treatment Comparison of Mean Amblyopic Eye Visual Acuity Change at 10-weeks According to Baseline Characteristics | Gender: Female | 0.4 units on a scale | Standard Deviation 1.3 |
| Intensified Treatment | Treatment Comparison of Mean Amblyopic Eye Visual Acuity Change at 10-weeks According to Baseline Characteristics | Cause of amblyopia: Combined mechanism | 1.0 units on a scale | Standard Deviation 1.4 |
| Intensified Treatment | Treatment Comparison of Mean Amblyopic Eye Visual Acuity Change at 10-weeks According to Baseline Characteristics | Age at randomization: 5 years or older | 1.0 units on a scale | Standard Deviation 1.4 |
| Intensified Treatment | Treatment Comparison of Mean Amblyopic Eye Visual Acuity Change at 10-weeks According to Baseline Characteristics | Gender: Male | 0.9 units on a scale | Standard Deviation 1.5 |
| Intensified Treatment | Treatment Comparison of Mean Amblyopic Eye Visual Acuity Change at 10-weeks According to Baseline Characteristics | Race/Ethnicity: White (non-Hispanic) | 1.2 units on a scale | Standard Deviation 1.4 |
| Intensified Treatment | Treatment Comparison of Mean Amblyopic Eye Visual Acuity Change at 10-weeks According to Baseline Characteristics | Race/Ethnicity: Non-White/Hispanic | 1.1 units on a scale | Standard Deviation 1.3 |
| Intensified Treatment | Treatment Comparison of Mean Amblyopic Eye Visual Acuity Change at 10-weeks According to Baseline Characteristics | Age at randomization: 3 to <5 years | 1.5 units on a scale | Standard Deviation 1.4 |
| Intensified Treatment | Treatment Comparison of Mean Amblyopic Eye Visual Acuity Change at 10-weeks According to Baseline Characteristics | Amblyopic eye VA at randomization: 20/80 or worse | 1.3 units on a scale | Standard Deviation 1.6 |
| Intensified Treatment | Treatment Comparison of Mean Amblyopic Eye Visual Acuity Change at 10-weeks According to Baseline Characteristics | Amblyopic eye VA at randomization: 20/63 | 1.6 units on a scale | Standard Deviation 1.4 |
| Intensified Treatment | Treatment Comparison of Mean Amblyopic Eye Visual Acuity Change at 10-weeks According to Baseline Characteristics | Amblyopic eye VA at randomization: 20/50 | 1.4 units on a scale | Standard Deviation 1.1 |
| Intensified Treatment | Treatment Comparison of Mean Amblyopic Eye Visual Acuity Change at 10-weeks According to Baseline Characteristics | Amblyopic eye VA at randomization: 20/40 or better | 0.5 units on a scale | Standard Deviation 1.3 |
| Intensified Treatment | Treatment Comparison of Mean Amblyopic Eye Visual Acuity Change at 10-weeks According to Baseline Characteristics | Cause of amblyopia: Strabismus | 1.4 units on a scale | Standard Deviation 1.3 |
| Intensified Treatment | Treatment Comparison of Mean Amblyopic Eye Visual Acuity Change at 10-weeks According to Baseline Characteristics | Cause of amblyopia: Anisometropia | 1.3 units on a scale | Standard Deviation 1.5 |
| Intensified Treatment | Treatment Comparison of Mean Amblyopic Eye Visual Acuity Change at 10-weeks According to Baseline Characteristics | Gender: Female | 1.4 units on a scale | Standard Deviation 1.3 |
Comparison: The treatment effect within gender was assessed by including an interaction term between treatment group and gender in the ANCOVA model, adjusting for amblyopic eye visual acuity at randomization and main effects corresponding to the interaction term.p-value: 0.04ANCOVA
Comparison: The treatment effect within race/ethnicity was assessed by including an interaction term between treatment group and race/ethnicity in the ANCOVA model, adjusting for amblyopic eye visual acuity at randomization and main effects corresponding to the interaction term.p-value: 0.89ANCOVA
Comparison: The treatment effect within age at randomization was assessed by including an interaction term between treatment group and age in the ANCOVA model, adjusting for amblyopic eye visual acuity at randomization and main effects corresponding to the interaction term.p-value: 0.49ANCOVA
Comparison: The treatment effect within amblyopic eye visual acuity at randomization was assessed by including an interaction term between treatment group and visual acuity in the ANCOVA model, adjusting for the main effects corresponding to the interaction term.p-value: 0.37ANCOVA
Comparison: The treatment effect within cause of amblyopia was assessed by including an interaction term between treatment group and amblyopia cause in the ANCOVA model, adjusting for amblyopic eye visual acuity at randomization and main effects corresponding to the interaction term.p-value: 0.5ANCOVA
Secondary
Treatment Group Comparison of 10-week Interocular Difference
The secondary outcome analysis was a treatment group comparison of the 10-week interocular difference (IOD), computed as the difference between the masked amblyopic and fellow eye visual acuities, using an analysis of covariance (ANCOVA) model, adjusting for IOD at randomization. The analysis included data from 10-week visual acuity exams completed between 8 to 15 weeks (inclusive) according to the principles specified in the primary outcome analysis.
Time frame: 10 weeks after randomization
Population: The analysis included data from 10-week visual acuity exams completed between 8 to 15 weeks (inclusive) according to the principles specified in the primary outcome analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Control | Treatment Group Comparison of 10-week Interocular Difference | 3.7 logMAR lines | Standard Deviation 2.2 |
| Intensified Treatment | Treatment Group Comparison of 10-week Interocular Difference | 3.2 logMAR lines | Standard Deviation 2.1 |
p-value: 0.0195% CI: [0.1, 1]ANCOVA
Secondary
Treatment Group Comparison of the Proportion of Participants Who Have Improved by 2 or More logMAR Visual Acuity Lines at 10 Weeks Since Randomization
The proportion of participants who improved at least 2 logMAR lines since randomization was computed at the 10-week outcome. The secondary outcome analysis was a treatment group comparison of the proportion of participants whose 10-week masked amblyopic eye visual acuity improved at least 2 logMAR lines since randomization using logistic regression, adjusting for visual acuity at randomization. The analysis included data from 10-week visual acuity exams completed between 8 to 15 weeks (inclusive) according to the principles specified in the primary outcome analysis.
Time frame: 10 weeks after randomization
Population: The analysis included data from 10-week visual acuity exams completed between 8 to 15 weeks (inclusive) according to the principles specified in the primary outcome analysis.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Control | Treatment Group Comparison of the Proportion of Participants Who Have Improved by 2 or More logMAR Visual Acuity Lines at 10 Weeks Since Randomization | 15 participants |
| Intensified Treatment | Treatment Group Comparison of the Proportion of Participants Who Have Improved by 2 or More logMAR Visual Acuity Lines at 10 Weeks Since Randomization | 33 participants |
p-value: 0.00395% CI: [8, 35]Regression, Logistic
Secondary
Treatment Group Comparison of the Proportion of Participants Who Have Improved by 2 or More logMAR Visual Acuity Lines Based on Visual Acuity at Best Post-randomization Visit
Participants in both groups who have improved by one or more lines from randomization to the 10-week outcome exam will continue in the study and visits will occur every 10 weeks until no improvement of one or more lines from the previous visit. The proportion of participants who improved at least 2 logMAR lines since randomization was computed based on the best post-randomization visual acuity in the amblyopic eye. The initial visual acuity score was used if a retest was obtained.
Time frame: 10 weeks after randomization or later
Population: The analysis includes data from participants who completed the 10-week exam and/or a later visit.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Control | Treatment Group Comparison of the Proportion of Participants Who Have Improved by 2 or More logMAR Visual Acuity Lines Based on Visual Acuity at Best Post-randomization Visit | 32 participants |
| Intensified Treatment | Treatment Group Comparison of the Proportion of Participants Who Have Improved by 2 or More logMAR Visual Acuity Lines Based on Visual Acuity at Best Post-randomization Visit | 42 participants |