Trial Outcomes & Findings for Levodopa for the Treatment of Residual Amblyopia (NCT NCT01190813)
NCT ID: NCT01190813
Last Updated: 2016-06-02
Results Overview
The primary outcome is the amblyopic eye visual acuity letter score measured at the 18-week primary outcome visit following a rapid taper of study medicine beginning at week 16. The primary analytic approach will be a treatment group comparison of the mean amblyopic eye visual acuity adjusted for baseline acuity. Visual acuity was measured in each eye (right eye first) by a study-certified VA tester using the Electronic Early Treatment of Diabetic Retinopathy Study (E-ETDRS©) visual acuity protocol. Five letters is equivalent to one logMAR line.
COMPLETED
PHASE3
139 participants
18 weeks after enrollment
2016-06-02
Participant Flow
Between September 2010 and October 2013, 139 participants from 27 sites were randomly assigned to levodopa (n=90) or placebo (n=49).
Participant milestones
| Measure |
Levodopa/Carbidopa
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Overall Study
STARTED
|
90
|
49
|
|
Overall Study
2-week Phone Call
|
87
|
48
|
|
Overall Study
4-week Visit
|
88
|
47
|
|
Overall Study
7-week Phone Call
|
85
|
46
|
|
Overall Study
10-week Visit
|
88
|
48
|
|
Overall Study
13-week Phone Call
|
82
|
48
|
|
Overall Study
16-week Visit
|
87
|
46
|
|
Overall Study
18-week Visit (Masked Primary)
|
87
|
45
|
|
Overall Study
23-week Phone Call
|
82
|
41
|
|
Overall Study
26-week Visit
|
86
|
45
|
|
Overall Study
COMPLETED
|
86
|
45
|
|
Overall Study
NOT COMPLETED
|
4
|
4
|
Reasons for withdrawal
| Measure |
Levodopa/Carbidopa
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Overall Study
Lost to Follow-up
|
1
|
2
|
|
Overall Study
Withdrawal by Subject
|
2
|
1
|
|
Overall Study
Refused study treatment after randomized
|
1
|
0
|
|
Overall Study
Adverse Event
|
0
|
1
|
Baseline Characteristics
Levodopa for the Treatment of Residual Amblyopia
Baseline characteristics by cohort
| Measure |
Levodopa/Carbidopa
n=90 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=49 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
Total
n=139 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Sex: Female, Male
Male
|
50 Participants
n=99 Participants
|
25 Participants
n=107 Participants
|
75 Participants
n=206 Participants
|
|
Race/Ethnicity, Customized
Hispanic or Latino
|
5 participants
n=99 Participants
|
2 participants
n=107 Participants
|
7 participants
n=206 Participants
|
|
Race/Ethnicity, Customized
White
|
77 participants
n=99 Participants
|
44 participants
n=107 Participants
|
121 participants
n=206 Participants
|
|
Race/Ethnicity, Customized
Unknown/Not Reported
|
5 participants
n=99 Participants
|
1 participants
n=107 Participants
|
6 participants
n=206 Participants
|
|
Region of Enrollment
United States
|
90 participants
n=99 Participants
|
49 participants
n=107 Participants
|
139 participants
n=206 Participants
|
|
Race/Ethnicity, Customized
Asian
|
2 participants
n=99 Participants
|
1 participants
n=107 Participants
|
3 participants
n=206 Participants
|
|
Race/Ethnicity, Customized
Black/African American
|
1 participants
n=99 Participants
|
1 participants
n=107 Participants
|
2 participants
n=206 Participants
|
|
Age, Categorical
<=18 years
|
90 Participants
n=99 Participants
|
49 Participants
n=107 Participants
|
139 Participants
n=206 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Age, Continuous
|
9.4 years
STANDARD_DEVIATION 1.8 • n=99 Participants
|
9.5 years
STANDARD_DEVIATION 1.7 • n=107 Participants
|
9.5 years
STANDARD_DEVIATION 1.7 • n=206 Participants
|
|
Age, Customized
7 to <9 years
|
39 participants
n=99 Participants
|
21 participants
n=107 Participants
|
60 participants
n=206 Participants
|
|
Age, Customized
9 to <11 years
|
33 participants
n=99 Participants
|
14 participants
n=107 Participants
|
47 participants
n=206 Participants
|
|
Age, Customized
11 to <13 years
|
18 participants
n=99 Participants
|
14 participants
n=107 Participants
|
32 participants
n=206 Participants
|
|
Sex: Female, Male
Female
|
40 Participants
n=99 Participants
|
24 Participants
n=107 Participants
|
64 Participants
n=206 Participants
|
|
Cause of Amblyopia
Strabismus
|
22 participants
n=99 Participants
|
5 participants
n=107 Participants
|
27 participants
n=206 Participants
|
|
Cause of Amblyopia
Anisometropia
|
20 participants
n=99 Participants
|
16 participants
n=107 Participants
|
36 participants
n=206 Participants
|
|
Cause of Amblyopia
Combined Mechanism
|
48 participants
n=99 Participants
|
28 participants
n=107 Participants
|
76 participants
n=206 Participants
|
|
Patching Duration at Randomization
2 hours per day
|
81 participants
n=99 Participants
|
43 participants
n=107 Participants
|
124 participants
n=206 Participants
|
|
Patching Duration at Randomization
>= 3 hours per day
|
9 participants
n=99 Participants
|
6 participants
n=107 Participants
|
15 participants
n=206 Participants
|
|
Visual Acuity in the Amblyopic Eye at Randomization
20/200 or worse (<=37 letters)
|
7 participants
n=99 Participants
|
9 participants
n=107 Participants
|
16 participants
n=206 Participants
|
|
Visual Acuity in the Amblyopic Eye at Randomization
20/100 to <20/200 (38-52 letters)
|
34 participants
n=99 Participants
|
13 participants
n=107 Participants
|
47 participants
n=206 Participants
|
|
Visual Acuity in the Amblyopic Eye at Randomization
20/80 (53-57 letters)
|
15 participants
n=99 Participants
|
8 participants
n=107 Participants
|
23 participants
n=206 Participants
|
|
Visual Acuity in the Amblyopic Eye at Randomization
20/63 (58-62 letters)
|
20 participants
n=99 Participants
|
6 participants
n=107 Participants
|
26 participants
n=206 Participants
|
|
Visual Acuity in the Amblyopic Eye at Randomization
20/50 (63-67 letters)
|
14 participants
n=99 Participants
|
13 participants
n=107 Participants
|
27 participants
n=206 Participants
|
|
Visual Acuity in the Amblyopic Eye at Randomization
|
52.8 letters
STANDARD_DEVIATION 9.8 • n=99 Participants
|
51.7 letters
STANDARD_DEVIATION 11.9 • n=107 Participants
|
52.4 letters
STANDARD_DEVIATION 10.5 • n=206 Participants
|
|
Visual Acuity in the Fellow Eye at Randomization
20/25 (78-82 letters)
|
15 participants
n=99 Participants
|
4 participants
n=107 Participants
|
19 participants
n=206 Participants
|
|
Visual Acuity in the Fellow Eye at Randomization
20/20 (83-87 letters)
|
35 participants
n=99 Participants
|
20 participants
n=107 Participants
|
55 participants
n=206 Participants
|
|
Visual Acuity in the Fellow Eye at Randomization
20/16 (88-92 letters)
|
34 participants
n=99 Participants
|
23 participants
n=107 Participants
|
57 participants
n=206 Participants
|
|
Visual Acuity in the Fellow Eye at Randomization
20/12 (93-97 letters)
|
6 participants
n=99 Participants
|
2 participants
n=107 Participants
|
8 participants
n=206 Participants
|
|
Visual Acuity in the Fellow Eye at Randomization
|
86.7 letters
STANDARD_DEVIATION 4.1 • n=99 Participants
|
87.3 letters
STANDARD_DEVIATION 3.5 • n=107 Participants
|
86.9 letters
STANDARD_DEVIATION 3.9 • n=206 Participants
|
|
Intraocular Visual Acuity Difference at Randomization
|
33.9 letters
STANDARD_DEVIATION 10.9 • n=99 Participants
|
35.6 letters
STANDARD_DEVIATION 13.1 • n=107 Participants
|
34.5 letters
STANDARD_DEVIATION 11.7 • n=206 Participants
|
|
SE Refractive Error in Amblyopic Eye at Randomization
|
4.0 diopters
STANDARD_DEVIATION 2.6 • n=99 Participants
|
4.5 diopters
STANDARD_DEVIATION 2.3 • n=107 Participants
|
4.2 diopters
STANDARD_DEVIATION 2.5 • n=206 Participants
|
|
SE Refractive Error in Fellow Eye at Randomization
|
1.9 diopters
STANDARD_DEVIATION 2.0 • n=99 Participants
|
1.6 diopters
STANDARD_DEVIATION 1.7 • n=107 Participants
|
1.8 diopters
STANDARD_DEVIATION 1.9 • n=206 Participants
|
|
Anisometropia at Randomization
|
2.5 diopters
STANDARD_DEVIATION 2.1 • n=99 Participants
|
3.1 diopters
STANDARD_DEVIATION 2.0 • n=107 Participants
|
2.7 diopters
STANDARD_DEVIATION 2.1 • n=206 Participants
|
PRIMARY outcome
Timeframe: 18 weeks after enrollmentPopulation: The ITT principle was followed. For subjects with no visit in the +/- 1 wk window for the 18-wk visit, data from a visit 14-27 wks after randomization were used, if available. Multiple imputation by the Monte Carlo Markov Chain method was used for missing 18-wk VA outcomes based on tx group, baseline VA, \& VA scores from completed follow-up visits.
The primary outcome is the amblyopic eye visual acuity letter score measured at the 18-week primary outcome visit following a rapid taper of study medicine beginning at week 16. The primary analytic approach will be a treatment group comparison of the mean amblyopic eye visual acuity adjusted for baseline acuity. Visual acuity was measured in each eye (right eye first) by a study-certified VA tester using the Electronic Early Treatment of Diabetic Retinopathy Study (E-ETDRS©) visual acuity protocol. Five letters is equivalent to one logMAR line.
Outcome measures
| Measure |
Levodopa/Carbidopa
n=86 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=45 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Distribution of Amblyopic Eye Visual Acuity Change From Baseline
10-14 letters worse
|
0 participants
|
0 participants
|
|
Distribution of Amblyopic Eye Visual Acuity Change From Baseline
5-9 letters worse
|
2 participants
|
1 participants
|
|
Distribution of Amblyopic Eye Visual Acuity Change From Baseline
within 4 letters
|
35 participants
|
23 participants
|
|
Distribution of Amblyopic Eye Visual Acuity Change From Baseline
5-9 letters better
|
36 participants
|
19 participants
|
|
Distribution of Amblyopic Eye Visual Acuity Change From Baseline
10-14 letters better
|
10 participants
|
1 participants
|
|
Distribution of Amblyopic Eye Visual Acuity Change From Baseline
>= 15 letters better
|
3 participants
|
1 participants
|
PRIMARY outcome
Timeframe: 18 weeks after enrollmentPopulation: The ITT principle was followed. For subjects with no visit in the +/- 1 wk window for the 18-wk visit, data from a visit 14-27 wks after randomization were used, if available. Multiple imputation by the Monte Carlo Markov Chain method was used for missing 18-wk VA outcomes based on tx group, baseline VA, \& VA scores from completed follow-up visits.
The primary outcome is the amblyopic eye visual acuity letter score measured at the 18-week primary outcome visit following a rapid taper of study medicine beginning at week 16. The primary analytic approach will be a treatment group comparison of the mean amblyopic eye visual acuity adjusted for baseline acuity. Visual acuity was measured in each eye (right eye first) by a study-certified VA tester using the Electronic Early Treatment of Diabetic Retinopathy Study (E-ETDRS©) visual acuity protocol. Five letters is equivalent to one logMAR line.
Outcome measures
| Measure |
Levodopa/Carbidopa
n=86 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=45 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Mean Amblyopic Eye Visual Acuity Change From Baseline
|
5.2 letters
Standard Deviation 5.3
|
3.8 letters
Standard Deviation 5.0
|
SECONDARY outcome
Timeframe: 18 weeks after enrollmentPopulation: The analysis followed the intent-to-treat principle. For missing primary outcome visits (±1 wk), data from a visit 14-27 wks after randomization were used, if available.Multiple imputation(Monte Carlo Markov Chain method) was used for missing 18-wk VA outcomes based on treatment group, baseline VA, and VA scores from completed follow-up visits
Treatment group comparisons of the proportion of subjects who have improved from baseline by 10 or more letters.
Outcome measures
| Measure |
Levodopa/Carbidopa
n=86 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=45 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 18 Weeks
|
13 participants
|
2 participants
|
SECONDARY outcome
Timeframe: 18 weeks after enrollmentOutcome measures
| Measure |
Levodopa/Carbidopa
n=86 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=45 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Distribution of Amblyopic Eye Visual Acuity at 18 Weeks
20/100 (48-52 letters)
|
17 participants
|
9 participants
|
|
Distribution of Amblyopic Eye Visual Acuity at 18 Weeks
20/25 (78-82 letters)
|
0 participants
|
0 participants
|
|
Distribution of Amblyopic Eye Visual Acuity at 18 Weeks
20/32 (73-77 letters)
|
5 participants
|
1 participants
|
|
Distribution of Amblyopic Eye Visual Acuity at 18 Weeks
20/40 (68-72 letters)
|
7 participants
|
6 participants
|
|
Distribution of Amblyopic Eye Visual Acuity at 18 Weeks
20/50 (63-67 letters)
|
20 participants
|
5 participants
|
|
Distribution of Amblyopic Eye Visual Acuity at 18 Weeks
20/63 (58-62 letters)
|
18 participants
|
9 participants
|
|
Distribution of Amblyopic Eye Visual Acuity at 18 Weeks
20/80 (53-57 letters)
|
12 participants
|
5 participants
|
|
Distribution of Amblyopic Eye Visual Acuity at 18 Weeks
20/125 (43-47 letters)
|
4 participants
|
3 participants
|
|
Distribution of Amblyopic Eye Visual Acuity at 18 Weeks
20/160 or worse (<=42 letters)
|
3 participants
|
7 participants
|
SECONDARY outcome
Timeframe: 18 weeks after enrollmentVisual acuity was measured in each eye (right eye first) by a study-certified VA tester using the Electronic Early Treatment of Diabetic Retinopathy Study (E-ETDRS©) visual acuity protocol. Five letters is equivalent to one logMAR line.
Outcome measures
| Measure |
Levodopa/Carbidopa
n=86 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=45 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Mean Amblyopic Eye Visual Acuity at 18 Weeks
|
58.7 letters
Standard Deviation 8.9
|
54.8 letters
Standard Deviation 12.3
|
SECONDARY outcome
Timeframe: 4 weeks after enrollmentTreatment group comparisons adjusted for baseline acuity scores using logistic regression of the proportion of subjects who have improved from baseline by 10 or more letters.
Outcome measures
| Measure |
Levodopa/Carbidopa
n=88 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=47 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 4 Weeks
Improved 10 or more letters
|
4 participants
|
2 participants
|
|
Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 4 Weeks
Did not improve 10 or more letters
|
84 participants
|
45 participants
|
SECONDARY outcome
Timeframe: 10 weeks after enrollmentTreatment group comparisons of the proportion of subjects who have improved from baseline by 10 or more letters.
Outcome measures
| Measure |
Levodopa/Carbidopa
n=88 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=48 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 10 Weeks
Improved 10 or more letters
|
10 participants
|
9 participants
|
|
Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 10 Weeks
Not improved 10 or more letters
|
78 participants
|
39 participants
|
SECONDARY outcome
Timeframe: 16 weeks after enrollmentTreatment group comparisons of the proportion of subjects who have improved from baseline by 10 or more letters.
Outcome measures
| Measure |
Levodopa/Carbidopa
n=87 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=46 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 16 Weeks
Improved 10 or more letters
|
15 participants
|
10 participants
|
|
Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 16 Weeks
Not improved 10 or more letters
|
72 participants
|
36 participants
|
SECONDARY outcome
Timeframe: 26 weeks after enrollmentTreatment group comparisons adjusted for baseline acuity scores using logistic regression of the proportion of subjects who have improved from baseline by 10 or more letters.
Outcome measures
| Measure |
Levodopa/Carbidopa
n=86 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=45 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 26 Weeks
Improved 10 or more letters
|
17 participants
|
5 participants
|
|
Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 26 Weeks
Not improved 10 or more letters
|
69 participants
|
40 participants
|
SECONDARY outcome
Timeframe: 4 weeks after enrollmentTreatment group comparisons adjusted for baseline acuity scores using logistic regression of the proportion of subjects with 20/25 or better visual acuity.
Outcome measures
| Measure |
Levodopa/Carbidopa
n=88 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=47 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Amblyopia Resolutionat 4 Weeks
20/25 or better
|
0 participants
|
0 participants
|
|
Amblyopia Resolutionat 4 Weeks
20/32 or worse
|
88 participants
|
47 participants
|
SECONDARY outcome
Timeframe: 10 weeks after enrollmentTreatment group comparisons adjusted for baseline acuity scores using logistic regression of the proportion of subjects with 20/25 or better visual acuity.
Outcome measures
| Measure |
Levodopa/Carbidopa
n=88 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=48 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Amblyopia Resolution at 10 Weeks
20/25 or better
|
0 participants
|
0 participants
|
|
Amblyopia Resolution at 10 Weeks
20/32 or worse
|
88 participants
|
48 participants
|
SECONDARY outcome
Timeframe: 16 weeks after enrollmentTreatment group comparisons adjusted for baseline acuity scores using logistic regression of the proportion of subjects with 20/25 or better visual acuity.
Outcome measures
| Measure |
Levodopa/Carbidopa
n=87 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=46 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Amblyopia Resolution at 16 Weeks
20/25 or better
|
1 participants
|
0 participants
|
|
Amblyopia Resolution at 16 Weeks
20/32 or worse
|
86 participants
|
46 participants
|
SECONDARY outcome
Timeframe: 18 weeks after enrollmentTreatment group comparisons adjusted for baseline acuity scores using logistic regression of the proportion of subjects with 20/25 or better visual acuity.
Outcome measures
| Measure |
Levodopa/Carbidopa
n=86 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=45 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Amblyopia Resolution at 18 Weeks
20/25 or better
|
0 participants
|
0 participants
|
|
Amblyopia Resolution at 18 Weeks
20/32 or worse
|
86 participants
|
45 participants
|
SECONDARY outcome
Timeframe: 26 weeks after enrollmentTreatment group comparisons adjusted for baseline acuity scores using logistic regression of the proportion of subjects with 20/25 or better visual acuity.
Outcome measures
| Measure |
Levodopa/Carbidopa
n=86 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=45 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Amblyopia Resolution at 26 Weeks
20/25 or better
|
0 participants
|
0 participants
|
|
Amblyopia Resolution at 26 Weeks
20/32 or worse
|
86 participants
|
45 participants
|
SECONDARY outcome
Timeframe: 4 weeks after enrollmentVisual acuity was measured in each eye (right eye first) by a study-certified VA tester using the Electronic Early Treatment of Diabetic Retinopathy Study (E-ETDRS©) visual acuity protocol. Five letters is equivalent to one logMAR line.
Outcome measures
| Measure |
Levodopa/Carbidopa
n=88 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=47 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 4 Weeks
10-14 letters worse
|
0 participants
|
0 participants
|
|
Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 4 Weeks
5-9 letters worse
|
6 participants
|
2 participants
|
|
Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 4 Weeks
within 4 letters
|
55 participants
|
28 participants
|
|
Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 4 Weeks
5-9 letters better
|
23 participants
|
15 participants
|
|
Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 4 Weeks
10-14 letters better
|
4 participants
|
1 participants
|
|
Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 4 Weeks
>= 15 letters better
|
0 participants
|
1 participants
|
SECONDARY outcome
Timeframe: 4 weeks after enrollmentA treatment group comparison of the mean amblyopic eye visual acuity at 4 weeks after enrollment, adjusted for baseline acuity.
Outcome measures
| Measure |
Levodopa/Carbidopa
n=88 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=47 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Mean Change in Amblyopic Eye Visual Acuity From Baseline at 4 Weeks
|
2.2 letters
Standard Deviation 4.1
|
2.5 letters
Standard Deviation 4.7
|
SECONDARY outcome
Timeframe: 10 weeks after enrollmentVisual acuity was measured in each eye (right eye first) by a study-certified VA tester using the Electronic Early Treatment of Diabetic Retinopathy Study (E-ETDRS©) visual acuity protocol. Five letters is equivalent to one logMAR line.
Outcome measures
| Measure |
Levodopa/Carbidopa
n=88 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=48 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 10 Weeks
10-14 letters worse
|
1 participants
|
0 participants
|
|
Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 10 Weeks
5-9 letters worse
|
4 participants
|
1 participants
|
|
Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 10 Weeks
within 4 letters
|
43 participants
|
30 participants
|
|
Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 10 Weeks
5-9 letters better
|
30 participants
|
8 participants
|
|
Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 10 Weeks
10-14 letters better
|
7 participants
|
9 participants
|
|
Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 10 Weeks
>= 15 letters better
|
3 participants
|
0 participants
|
SECONDARY outcome
Timeframe: 10 weeks after enrollmentA treatment group comparison of the mean amblyopic eye visual acuity at 10 weeks after enrollment, adjusted for baseline acuity. Visual acuity was measured in each eye (right eye first) by a study-certified VA tester using the Electronic Early Treatment of Diabetic Retinopathy Study (E-ETDRS©) visual acuity protocol. Five letters is equivalent to one logMAR line.
Outcome measures
| Measure |
Levodopa/Carbidopa
n=88 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=48 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Mean Change in Amblyopic Eye Visual Acuity From Baseline at 10 Weeks
|
3.8 letters
Standard Deviation 4.9
|
3.7 letters
Standard Deviation 4.9
|
SECONDARY outcome
Timeframe: 16 weeks after enrollmentVisual acuity was measured in each eye (right eye first) by a study-certified VA tester using the Electronic Early Treatment of Diabetic Retinopathy Study (E-ETDRS©) visual acuity protocol. Five letters is equivalent to one logMAR line.
Outcome measures
| Measure |
Levodopa/Carbidopa
n=87 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=46 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 16 Weeks
10-14 letters worse
|
0 participants
|
0 participants
|
|
Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 16 Weeks
5-9 letters worse
|
1 participants
|
0 participants
|
|
Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 16 Weeks
within 4 letters
|
41 participants
|
26 participants
|
|
Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 16 Weeks
5-9 letters better
|
30 participants
|
10 participants
|
|
Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 16 Weeks
10-14 letters better
|
9 participants
|
10 participants
|
|
Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 16 Weeks
>= 15 letters better
|
6 participants
|
0 participants
|
SECONDARY outcome
Timeframe: 16 weeks after enrollmentA treatment group comparison of the mean amblyopic eye visual acuity at 16 weeks after enrollment, adjusted for baseline acuity. Visual acuity was measured in each eye (right eye first) by a study-certified VA tester using the Electronic Early Treatment of Diabetic Retinopathy Study (E-ETDRS©) visual acuity protocol. Five letters is equivalent to one logMAR line.
Outcome measures
| Measure |
Levodopa/Carbidopa
n=87 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=46 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Mean Change in Amblyopic Eye Visual Acuity From Baseline at 16 Weeks
|
5.1 letters
Standard Deviation 6.3
|
4.2 letters
Standard Deviation 4.9
|
SECONDARY outcome
Timeframe: 26 weeks after enrollmentVisual acuity was measured in each eye (right eye first) by a study-certified VA tester using the Electronic Early Treatment of Diabetic Retinopathy Study (E-ETDRS©) visual acuity protocol. Five letters is equivalent to one logMAR line.
Outcome measures
| Measure |
Levodopa/Carbidopa
n=86 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=45 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 26 Weeks
5-9 letters worse
|
3 participants
|
1 participants
|
|
Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 26 Weeks
within 4 letters
|
36 participants
|
23 participants
|
|
Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 26 Weeks
5-9 letters better
|
30 participants
|
16 participants
|
|
Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 26 Weeks
10-14 letters better
|
14 participants
|
5 participants
|
|
Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 26 Weeks
>= 15 letters better
|
3 participants
|
0 participants
|
SECONDARY outcome
Timeframe: 26 weeks after enrollmentA treatment group comparison of the mean amblyopic eye visual acuity at 26 weeks after enrollment, adjusted for baseline acuity. Visual acuity was measured in each eye (right eye first) by a study-certified VA tester using the Electronic Early Treatment of Diabetic Retinopathy Study (E-ETDRS©) visual acuity protocol. Five letters is equivalent to one logMAR line.
Outcome measures
| Measure |
Levodopa/Carbidopa
n=86 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=45 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Mean Change in Amblyopic Eye Visual Acuity From Baseline at 26 Weeks
|
5.0 letters
Standard Deviation 5.7
|
4.2 letters
Standard Deviation 4.7
|
SECONDARY outcome
Timeframe: 18 weeks after enrollmentSimilar to the analysis for the amblyopic eye, the fellow eye visual acuity will be evaluated to determine if study treatment had an adverse effect on the occluded eye. The analysis will be a treatment group comparison of the mean fellow eye visual acuity at 18 weeks after enrollment, adjusted for baseline acuity.
Outcome measures
| Measure |
Levodopa/Carbidopa
n=87 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=45 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Distribution of Fellow Eye Visual Acuity at 18 Weeks
20/12 (93-97 letters)
|
12 participants
|
4 participants
|
|
Distribution of Fellow Eye Visual Acuity at 18 Weeks
20/16 (88-92 letters)
|
44 participants
|
22 participants
|
|
Distribution of Fellow Eye Visual Acuity at 18 Weeks
20/20 (83-87 letters)
|
22 participants
|
16 participants
|
|
Distribution of Fellow Eye Visual Acuity at 18 Weeks
20/25 (78-82 letters)
|
8 participants
|
3 participants
|
|
Distribution of Fellow Eye Visual Acuity at 18 Weeks
20/32 (73-77 letters)
|
1 participants
|
0 participants
|
SECONDARY outcome
Timeframe: 18 weeks after enrollmentVisual acuity was measured in each eye (right eye first) by a study-certified VA tester using the Electronic Early Treatment of Diabetic Retinopathy Study (E-ETDRS©) visual acuity protocol. Five letters is equivalent to one logMAR line.
Outcome measures
| Measure |
Levodopa/Carbidopa
n=87 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=45 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Mean Fellow Eye Visual Acuity at 18 Weeks
|
88.1 letters
Standard Deviation 4.4
|
88.0 letters
Standard Deviation 3.2
|
SECONDARY outcome
Timeframe: 18 weeks after enrollmentVisual acuity was measured in each eye (right eye first) by a study-certified VA tester using the Electronic Early Treatment of Diabetic Retinopathy Study (E-ETDRS©) visual acuity protocol. Five letters is equivalent to one logMAR line.
Outcome measures
| Measure |
Levodopa/Carbidopa
n=87 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=45 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Distribution of Fellow Eye Visual Acuity Change From Baseline at 18 Weeks
>=15 letters worse
|
0 participants
|
0 participants
|
|
Distribution of Fellow Eye Visual Acuity Change From Baseline at 18 Weeks
10-14 letters worse
|
0 participants
|
0 participants
|
|
Distribution of Fellow Eye Visual Acuity Change From Baseline at 18 Weeks
5-9 letters worse
|
4 participants
|
3 participants
|
|
Distribution of Fellow Eye Visual Acuity Change From Baseline at 18 Weeks
within 4 letters
|
68 participants
|
39 participants
|
|
Distribution of Fellow Eye Visual Acuity Change From Baseline at 18 Weeks
5-9 letters better
|
15 participants
|
3 participants
|
|
Distribution of Fellow Eye Visual Acuity Change From Baseline at 18 Weeks
10-14 letters better
|
0 participants
|
0 participants
|
SECONDARY outcome
Timeframe: 18 weeks after enrollmentVisual acuity was measured in each eye (right eye first) by a study-certified VA tester using the Electronic Early Treatment of Diabetic Retinopathy Study (E-ETDRS©) visual acuity protocol. Five letters is equivalent to one logMAR line.
Outcome measures
| Measure |
Levodopa/Carbidopa
n=87 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=45 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Mean Fellow Eye Visual Acuity Change From Baseline at 18 Weeks
|
1.5 letters
Standard Deviation 3.0
|
0.5 letters
Standard Deviation 3.1
|
SECONDARY outcome
Timeframe: At enrollmentA treatment group comparison of symptom survey scores at enrollment. The average of the overall item responses will be calculated and compared by treatment group with a t-test for difference in means. A higher number reflects a more negative response (5=always, 4=often, 3=sometimes, 2=rarely, 1=never).
Outcome measures
| Measure |
Levodopa/Carbidopa
n=90 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=49 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Mean Child Symptom Survey Score at Enrollment
|
1.60 units on a scale
Standard Deviation 0.37
|
1.73 units on a scale
Standard Deviation 0.45
|
SECONDARY outcome
Timeframe: 4 weeks after enrollmentA treatment group comparison of symptom survey scores at the 4 week visit. The average of the overall item responses will be calculated and compared by treatment group with a t-test for difference in means. A higher number reflects a more negative response (5=always, 4=often, 3=sometimes, 2=rarely, 1=never).
Outcome measures
| Measure |
Levodopa/Carbidopa
n=88 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=47 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Mean Child Symptom Survey Score at 4 Weeks
|
1.42 units on a scale
Standard Deviation 0.37
|
1.51 units on a scale
Standard Deviation 0.42
|
SECONDARY outcome
Timeframe: 10 weeks after enrollmentA treatment group comparison of symptom survey scores at the 10 week visit. The average of the overall item responses will be calculated and compared by treatment group with a t-test for difference in means. A higher number reflects a more negative response (5=always, 4=often, 3=sometimes, 2=rarely, 1=never).
Outcome measures
| Measure |
Levodopa/Carbidopa
n=88 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=47 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Mean Child Symptom Survey Score at 10 Weeks
|
1.33 units on a scale
Standard Deviation 0.31
|
1.42 units on a scale
Standard Deviation 0.37
|
SECONDARY outcome
Timeframe: 16 weeks after enrollmentA treatment group comparison of symptom survey scores at the 16 week visit. The average of the overall item responses will be calculated and compared by treatment group with a t-test for difference in means. A higher number reflects a more negative response (5=always, 4=often, 3=sometimes, 2=rarely, 1=never).
Outcome measures
| Measure |
Levodopa/Carbidopa
n=87 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=46 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Mean Child Symptom Survey Score at 16 Weeks
|
1.25 units on a scale
Standard Deviation 0.27
|
1.38 units on a scale
Standard Deviation 0.36
|
SECONDARY outcome
Timeframe: 18 weeks after enrollmentA treatment group comparison of symptom survey scores at the primary outcome (18 week) visit. The average of the overall item responses will be calculated and compared by treatment group with a t-test for difference in means. A higher number reflects a more negative response (5=always, 4=often, 3=sometimes, 2=rarely, 1=never).
Outcome measures
| Measure |
Levodopa/Carbidopa
n=87 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=45 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Mean Child Symptom Survey Score at 18 Weeks
|
1.17 units on a scale
Standard Deviation 0.22
|
1.28 units on a scale
Standard Deviation 0.32
|
SECONDARY outcome
Timeframe: 26 weeks after enrollmentA treatment group comparison of symptom survey scores at the 26 week visit. The average of the overall item responses will be calculated and compared by treatment group with a t-test for difference in means. A higher number reflects a more negative response (5=always, 4=often, 3=sometimes, 2=rarely, 1=never).
Outcome measures
| Measure |
Levodopa/Carbidopa
n=86 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=45 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Mean Child Symptom Survey Score at 26 Weeks
|
1.23 units on a scale
Standard Deviation 0.26
|
1.33 units on a scale
Standard Deviation 0.32
|
SECONDARY outcome
Timeframe: At enrollmentA treatment group comparison of symptom survey scores at enrollment. The average of the overall item responses will be calculated and compared by treatment group with a t-test for difference in means. A higher number reflects a more negative response (5=always, 4=often, 3=sometimes, 2=rarely, 1=never).
Outcome measures
| Measure |
Levodopa/Carbidopa
n=90 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=49 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Mean Parent Symptom Survey Score at Enrollment
|
1.42 units on a scale
Standard Deviation 0.34
|
1.44 units on a scale
Standard Deviation 0.29
|
SECONDARY outcome
Timeframe: 4 weeks after enrollmentA treatment group comparison of symptom survey scores at the 4 week visit. The average of the overall item responses will be calculated and compared by treatment group with a t-test for difference in means. A higher number reflects a more negative response (5=always, 4=often, 3=sometimes, 2=rarely, 1=never).
Outcome measures
| Measure |
Levodopa/Carbidopa
n=88 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=47 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Mean Parent Symptom Survey Score at 4 Weeks
|
1.29 units on a scale
Standard Deviation 0.27
|
1.26 units on a scale
Standard Deviation 0.27
|
SECONDARY outcome
Timeframe: 10 weeks after enrollmentA treatment group comparison of symptom survey scores at the 10 week visit. The average of the overall item responses will be calculated and compared by treatment group with a t-test for difference in means. A higher number reflects a more negative response (5=always, 4=often, 3=sometimes, 2=rarely, 1=never).
Outcome measures
| Measure |
Levodopa/Carbidopa
n=88 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=47 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Mean Parent Symptom Survey Score at 10 Weeks
|
1.22 units on a scale
Standard Deviation 0.22
|
1.30 units on a scale
Standard Deviation 0.25
|
SECONDARY outcome
Timeframe: 16 weeks after enrollmentA treatment group comparison of symptom survey scores at the 16 week visit. The average of the overall item responses will be calculated and compared by treatment group with a t-test for difference in means. A higher number reflects a more negative response (5=always, 4=often, 3=sometimes, 2=rarely, 1=never).
Outcome measures
| Measure |
Levodopa/Carbidopa
n=87 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=46 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Mean Parent Symptom Survey Score at 16 Weeks
|
1.24 units on a scale
Standard Deviation 0.27
|
1.25 units on a scale
Standard Deviation 0.22
|
SECONDARY outcome
Timeframe: 18 weeks after enrollmentA treatment group comparison of symptom survey scores at the primary outcome (18 week) visit. The average of the overall item responses will be calculated and compared by treatment group with a t-test for difference in means. A higher number reflects a more negative response (5=always, 4=often, 3=sometimes, 2=rarely, 1=never).
Outcome measures
| Measure |
Levodopa/Carbidopa
n=87 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=45 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Mean Parent Symptom Survey Score at 18 Weeks
|
1.17 units on a scale
Standard Deviation 0.21
|
1.17 units on a scale
Standard Deviation 0.20
|
SECONDARY outcome
Timeframe: 26 weeks after enrollmentA treatment group comparison of symptom survey scores at the 26 week visit. The average of the overall item responses will be calculated and compared by treatment group with a t-test for difference in means. A higher number reflects a more negative response (5=always, 4=often, 3=sometimes, 2=rarely, 1=never).
Outcome measures
| Measure |
Levodopa/Carbidopa
n=86 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=45 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Mean Parent Symptom Survey Score at 26 Weeks
|
1.19 units on a scale
Standard Deviation 0.24
|
1.22 units on a scale
Standard Deviation 0.29
|
SECONDARY outcome
Timeframe: Enrollment through 26 weeksOutcome measures
| Measure |
Levodopa/Carbidopa
n=90 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=49 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Mean Systemic Adverse Events
|
1.28 events
Standard Deviation 1.39
|
1.55 events
Standard Deviation 1.90
|
SECONDARY outcome
Timeframe: 26 weeks after enrollmentPopulation: All participants continued in the study regardless of whether they continued with study medication after the 18-week visit; 49 participants in the levodopa group and 24 participants in the placebo group continued study medication at the 18-week visit.
Similar to the analysis for the amblyopic eye, the fellow eye visual acuity will be evaluated to determine if study treatment had an adverse effect on the occluded eye. The analysis will be a treatment group comparison of the mean fellow eye visual acuity at 26 weeks after enrollment, adjusted for baseline acuity.
Outcome measures
| Measure |
Levodopa/Carbidopa
n=86 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=45 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Distribution of Fellow Eye Visual Acuity at 26 Weeks
20/12 (93-97 letters)
|
15 participants
|
7 participants
|
|
Distribution of Fellow Eye Visual Acuity at 26 Weeks
20/16 (88-92 letters)
|
34 participants
|
22 participants
|
|
Distribution of Fellow Eye Visual Acuity at 26 Weeks
20/20 (83-87 letters)
|
28 participants
|
13 participants
|
|
Distribution of Fellow Eye Visual Acuity at 26 Weeks
20/25 (78-82 letters)
|
9 participants
|
2 participants
|
|
Distribution of Fellow Eye Visual Acuity at 26 Weeks
20/32 (73-77 letters)
|
0 participants
|
1 participants
|
SECONDARY outcome
Timeframe: 26 weeks after enrollmentPopulation: All participants continued in the study regardless of whether they continued with study medication after the 18-week visit; 49 participants in the levodopa group and 24 participants in the placebo group continued study medication at the 18-week visit.
Visual acuity was measured in each eye (right eye first) by a study-certified VA tester using the Electronic Early Treatment of Diabetic Retinopathy Study (E-ETDRS©) visual acuity protocol. Five letters is equivalent to one logMAR line.
Outcome measures
| Measure |
Levodopa/Carbidopa
n=86 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=45 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Mean Fellow Eye Visual Acuity at 26 Weeks
|
88.0 letters
Standard Deviation 4.5
|
88.2 letters
Standard Deviation 4.1
|
SECONDARY outcome
Timeframe: 26 weeks after enrollmentPopulation: All participants continued in the study regardless of whether they continued with study medication after the 18-week visit; 49 participants in the levodopa group and 24 participants in the placebo group continued study medication at the 18-week visit.
Visual acuity was measured in each eye (right eye first) by a study-certified VA tester using the Electronic Early Treatment of Diabetic Retinopathy Study (E-ETDRS©) visual acuity protocol. Five letters is equivalent to one logMAR line.
Outcome measures
| Measure |
Levodopa/Carbidopa
n=86 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=45 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Distribution of Fellow Eye Visual Acuity Change From Baseline at 26 Weeks
>=15 letters worse
|
1 participants
|
0 participants
|
|
Distribution of Fellow Eye Visual Acuity Change From Baseline at 26 Weeks
10-14 letters worse
|
0 participants
|
0 participants
|
|
Distribution of Fellow Eye Visual Acuity Change From Baseline at 26 Weeks
5-9 letters worse
|
2 participants
|
4 participants
|
|
Distribution of Fellow Eye Visual Acuity Change From Baseline at 26 Weeks
within 4 letters
|
74 participants
|
35 participants
|
|
Distribution of Fellow Eye Visual Acuity Change From Baseline at 26 Weeks
5-9 letters better
|
7 participants
|
6 participants
|
|
Distribution of Fellow Eye Visual Acuity Change From Baseline at 26 Weeks
10-14 letters better
|
2 participants
|
0 participants
|
SECONDARY outcome
Timeframe: 26 weeks after enrollmentVisual acuity was measured in each eye (right eye first) by a study-certified VA tester using the Electronic Early Treatment of Diabetic Retinopathy Study (E-ETDRS©) visual acuity protocol. Five letters is equivalent to one logMAR line.
Outcome measures
| Measure |
Levodopa/Carbidopa
n=86 Participants
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=45 Participants
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
Mean Fellow Eye Visual Acuity Change From Baseline at 26 Weeks
|
1.4 letters
Standard Deviation 3.6
|
0.8 letters
Standard Deviation 3.3
|
Adverse Events
Levodopa/Carbidopa
Placebo
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Levodopa/Carbidopa
n=90 participants at risk
Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid
Levodopa/Carbidopa: Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
Patching: Two hours of daily patching
|
Placebo
n=49 participants at risk
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
|
|---|---|---|
|
General disorders
Headache
|
20.0%
18/90 • Number of events 23 • From enrollment to last follow-up visit (26 weeks)
|
8.2%
4/49 • Number of events 6 • From enrollment to last follow-up visit (26 weeks)
|
|
General disorders
Nausea
|
6.7%
6/90 • Number of events 7 • From enrollment to last follow-up visit (26 weeks)
|
12.2%
6/49 • Number of events 6 • From enrollment to last follow-up visit (26 weeks)
|
|
General disorders
Cough
|
4.4%
4/90 • Number of events 4 • From enrollment to last follow-up visit (26 weeks)
|
6.1%
3/49 • Number of events 4 • From enrollment to last follow-up visit (26 weeks)
|
|
Gastrointestinal disorders
Gastroenteritis viral
|
4.4%
4/90 • Number of events 5 • From enrollment to last follow-up visit (26 weeks)
|
6.1%
3/49 • Number of events 3 • From enrollment to last follow-up visit (26 weeks)
|
|
General disorders
Nasopharyngitis
|
4.4%
4/90 • Number of events 4 • From enrollment to last follow-up visit (26 weeks)
|
8.2%
4/49 • Number of events 4 • From enrollment to last follow-up visit (26 weeks)
|
|
General disorders
Pharyngitis streptococcal
|
3.3%
3/90 • Number of events 3 • From enrollment to last follow-up visit (26 weeks)
|
6.1%
3/49 • Number of events 3 • From enrollment to last follow-up visit (26 weeks)
|
|
Skin and subcutaneous tissue disorders
Rash
|
5.6%
5/90 • Number of events 6 • From enrollment to last follow-up visit (26 weeks)
|
6.1%
3/49 • Number of events 3 • From enrollment to last follow-up visit (26 weeks)
|
|
Eye disorders
Dry eye
|
0.00%
0/90 • From enrollment to last follow-up visit (26 weeks)
|
8.2%
4/49 • Number of events 4 • From enrollment to last follow-up visit (26 weeks)
|
|
Eye disorders
Eye irritation
|
0.00%
0/90 • From enrollment to last follow-up visit (26 weeks)
|
6.1%
3/49 • Number of events 3 • From enrollment to last follow-up visit (26 weeks)
|
Additional Information
Raymond Kraker, M.S.P.H., Director of PEDIG Coordinating Center
Jaeb Center for Health Research
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place