Amblyopia
Conditions
Keywords
amblyopia, levodopa
Brief summary
The purpose of this study is to evaluate the efficacy and safety of oral levodopa and patching versus oral placebo and patching as treatment for residual amblyopia in children 7 to \<13 years old with visual acuity of 20/50 to 20/400 in the amblyopic eye.
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. Despite best efforts with conventional amblyopia treatment, many older children and teenagers with amblyopia fail to achieve normal visual acuity in the amblyopic eye. In a previous PEDIG study where children 7 to 12 years old were treated with atropine and patching, only 36% of the children with moderate amblyopia and only 23% of the children with severe amblyopia achieved 20/40 or better acuity. Many clinicians have recognized that conventional therapies with patching and atropine have not been universally successful and have sought alternatives. PEDIG has discussed for several years the problem of residual amblyopia and how the remaining visual acuity deficit could be reduced. A number of research groups have evaluated the short term use of oral levodopa-carbidopa as an adjunct to patching therapy for older children.
Interventions
Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid
DRUGPlacebo
Oral placebo tid
OTHERPatching
Two hours of daily patching
Sponsors
National Eye Institute (NEI)
Jaeb Center for Health Research
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
7 Years to 12 Years
Healthy volunteers
No
Inclusion criteria
1. Age 7 to 12 2. Amblyopia associated with strabismus, anisometropia, or both * Criteria for strabismus: One of the following criteria must be met: Heterotropia at distance and/or near fixation on examination (with or without spectacles); History of strabismus surgery; Documented history of strabismus which is no longer present (which in the judgment of the investigator could have caused amblyopia) * Criteria for anisometropia: One of the following criteria must be met: ≥0.50 D difference between eyes in spherical equivalent; ≥1.50 D difference between eyes in astigmatism in any meridian 3. Visual acuity, measured in each eye (amblyopic eye without cycloplegia) within 7 days prior to enrollment using the E-ETDRS protocol by a study certified visual acuity tester as follows: * Visual acuity in the amblyopic eye 18 to 67 letters inclusive (20/50 to 20/400) * Visual acuity in the fellow eye ≥78 letters (20/25 or better) 4. Current amblyopia treatment (other than spectacles) * 12 weeks of at least 2 hours of occlusion per day prescribed for the fellow eye during the immediate pre-enrollment period. * While on current treatment, visual acuity has not improved one line (5 letters) or more since a non-study visit at least 6 weeks ago. Both acuity measurements to define no improvement must have been done using the same testing method. * Treatment with atropine at any time during this pre-enrollment period is not allowed. * Any treatment prior to the current patching episode with stable acuity is acceptable. 5. Spectacle correction (if applicable) for measurement of enrollment visual acuity must meet the following criteria and be based on a cycloplegic refraction that is no more than 6 months old: 1. Requirements for spectacle correction: * Spherical equivalent must be within 0.50 D of fully correcting the anisometropia. * Hypermetropia of 3.00D or more must be corrected. * Hypermetropia must not be under corrected by more than 1.50 D spherical equivalent, and reduction in plus sphere must be symmetric in the two eyes. * Cylinder power in both eyes must be within 0.50 D of fully correcting the astigmatism. * Cylinder axis in both eyes is within 6 degrees of the axis in the spectacles when cylinder power is ≥1.00 D. * Myopia of amblyopic eye greater than 0.50 D by spherical equivalent must be corrected, and the glasses must not under correct the myopia by more than 0.25 D or overcorrect it by more than 0.50 D. 2. Spectacles meeting above criteria must be worn :until visual acuity in amblyopic eye is stable (defined as 2 consecutive visual acuity measurements by the same testing method at least 4 weeks apart with no improvement of one line (5 letters) or more. 6. Eye examination within 6 months prior to enrollment 7. Parent available for at least one year of follow-up, has access to phone), and willing to be contacted by clinical site and Jaeb Center staff 8. In the investigator's judgment, the subject is likely to comply with prescribed treatment (e.g., no history of poor compliance with patching treatment) and unlikely to continue to improve by using 2 hours of patching per day alone.
Exclusion criteria
1. Myopia more than -6.00 D (spherical equivalent) in either eye. 2. Current vision therapy or orthoptics 3. Ocular cause for reduced visual acuity * nystagmus per se does not exclude the subject if the above visual acuity criteria are met 4. Prior intraocular or refractive surgery 5. History of narrow-angle glaucoma 6. Bronchial asthma or severe pulmonary disease 7. Strabismus surgery planned within 26 weeks 8. Known allergy to levodopa or carbidopa 9. History of dystonic reactions 10. Current use of oral iron supplements including multivitamins containing iron during treatment with levodopa-carbidopa 11. Current use of antihypertensive, anti-depressant medications, phenothiazines, butyrophenones, risperidone and isoniazid, non-specific monoamine oxidase inhibitors, or medication for the treatment of attention deficit hyperactivity disorder 12. Known liver disease 13. History of melanoma 14. Known psychological problems 15. Known skin reactions to patch or bandage adhesives 16. Prior levodopa treatment 17. Treatment with topical ophthalmic atropine within the past 12 weeks 18. A physician-prescribed diet high in protein 19. Females who are pregnant, lactating, or intend to become pregnant within the next 34 weeks. * A negative urine pregnancy test will be required for all females who have experienced menarche. * Requirements regarding pregnancy testing prior to enrollment may be further defined by each individual Institutional Review Board.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Distribution of Amblyopic Eye Visual Acuity Change From Baseline | 18 weeks after enrollment | 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. |
| Mean Amblyopic Eye Visual Acuity Change From Baseline | 18 weeks after enrollment | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Mean Amblyopic Eye Visual Acuity at 18 Weeks | 18 weeks after enrollment | 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. |
| Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 4 Weeks | 4 weeks after enrollment | Treatment 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. |
| Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 10 Weeks | 10 weeks after enrollment | Treatment group comparisons of the proportion of subjects who have improved from baseline by 10 or more letters. |
| Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 16 Weeks | 16 weeks after enrollment | Treatment group comparisons of the proportion of subjects who have improved from baseline by 10 or more letters. |
| Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 26 Weeks | 26 weeks after enrollment | Treatment 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. |
| Amblyopia Resolutionat 4 Weeks | 4 weeks after enrollment | Treatment group comparisons adjusted for baseline acuity scores using logistic regression of the proportion of subjects with 20/25 or better visual acuity. |
| Amblyopia Resolution at 10 Weeks | 10 weeks after enrollment | Treatment group comparisons adjusted for baseline acuity scores using logistic regression of the proportion of subjects with 20/25 or better visual acuity. |
| Amblyopia Resolution at 16 Weeks | 16 weeks after enrollment | Treatment group comparisons adjusted for baseline acuity scores using logistic regression of the proportion of subjects with 20/25 or better visual acuity. |
| Amblyopia Resolution at 18 Weeks | 18 weeks after enrollment | Treatment group comparisons adjusted for baseline acuity scores using logistic regression of the proportion of subjects with 20/25 or better visual acuity. |
| Amblyopia Resolution at 26 Weeks | 26 weeks after enrollment | Treatment group comparisons adjusted for baseline acuity scores using logistic regression of the proportion of subjects with 20/25 or better visual acuity. |
| Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 4 Weeks | 4 weeks after enrollment | 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. |
| Mean Change in Amblyopic Eye Visual Acuity From Baseline at 4 Weeks | 4 weeks after enrollment | A treatment group comparison of the mean amblyopic eye visual acuity at 4 weeks after enrollment, adjusted for baseline acuity. |
| Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 10 Weeks | 10 weeks after enrollment | 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. |
| Mean Change in Amblyopic Eye Visual Acuity From Baseline at 10 Weeks | 10 weeks after enrollment | A 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. |
| Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 16 Weeks | 16 weeks after enrollment | 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. |
| Mean Change in Amblyopic Eye Visual Acuity From Baseline at 16 Weeks | 16 weeks after enrollment | A 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. |
| Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 26 Weeks | 26 weeks after enrollment | 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. |
| Mean Change in Amblyopic Eye Visual Acuity From Baseline at 26 Weeks | 26 weeks after enrollment | A 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. |
| Distribution of Fellow Eye Visual Acuity at 18 Weeks | 18 weeks after enrollment | 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 18 weeks after enrollment, adjusted for baseline acuity. |
| Mean Fellow Eye Visual Acuity Change From Baseline at 26 Weeks | 26 weeks after enrollment | 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. |
| Mean Fellow Eye Visual Acuity at 18 Weeks | 18 weeks after enrollment | 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. |
| Distribution of Fellow Eye Visual Acuity Change From Baseline at 18 Weeks | 18 weeks after enrollment | 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. |
| Mean Fellow Eye Visual Acuity Change From Baseline at 18 Weeks | 18 weeks after enrollment | 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. |
| Mean Child Symptom Survey Score at Enrollment | At enrollment | A 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). |
| Mean Child Symptom Survey Score at 4 Weeks | 4 weeks after enrollment | A 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). |
| Mean Child Symptom Survey Score at 10 Weeks | 10 weeks after enrollment | A 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). |
| Mean Child Symptom Survey Score at 16 Weeks | 16 weeks after enrollment | A 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). |
| Mean Child Symptom Survey Score at 18 Weeks | 18 weeks after enrollment | A 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). |
| Mean Child Symptom Survey Score at 26 Weeks | 26 weeks after enrollment | A 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). |
| Mean Parent Symptom Survey Score at Enrollment | At enrollment | A 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). |
| Mean Parent Symptom Survey Score at 4 Weeks | 4 weeks after enrollment | A 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). |
| Mean Parent Symptom Survey Score at 10 Weeks | 10 weeks after enrollment | A 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). |
| Mean Parent Symptom Survey Score at 16 Weeks | 16 weeks after enrollment | A 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). |
| Mean Parent Symptom Survey Score at 18 Weeks | 18 weeks after enrollment | A 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). |
| Mean Parent Symptom Survey Score at 26 Weeks | 26 weeks after enrollment | A 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). |
| Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 18 Weeks | 18 weeks after enrollment | Treatment group comparisons of the proportion of subjects who have improved from baseline by 10 or more letters. |
| Distribution of Fellow Eye Visual Acuity at 26 Weeks | 26 weeks after enrollment | 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. |
| Mean Fellow Eye Visual Acuity at 26 Weeks | 26 weeks after enrollment | 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. |
| Distribution of Fellow Eye Visual Acuity Change From Baseline at 26 Weeks | 26 weeks after enrollment | 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. |
| Mean Systemic Adverse Events | Enrollment through 26 weeks | — |
| Distribution of Amblyopic Eye Visual Acuity at 18 Weeks | 18 weeks after enrollment | — |
Countries
United States
Participant flow
Recruitment details
Between September 2010 and October 2013, 139 participants from 27 sites were randomly assigned to levodopa (n=90) or placebo (n=49).
Participants by arm
| Arm | Count |
|---|---|
| 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 | 90 |
| Placebo Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching | 49 |
| Total | 139 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Adverse Event | 0 | 1 |
| Overall Study | Lost to Follow-up | 1 | 2 |
| Overall Study | Refused study treatment after randomized | 1 | 0 |
| Overall Study | Withdrawal by Subject | 2 | 1 |
Baseline characteristics
| Characteristic | Placebo | Levodopa/Carbidopa | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 49 Participants | 90 Participants | 139 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 | 9.5 years STANDARD_DEVIATION 1.7 | 9.4 years STANDARD_DEVIATION 1.8 | 9.5 years STANDARD_DEVIATION 1.7 |
| Age, Customized 11 to <13 years | 14 participants | 18 participants | 32 participants |
| Age, Customized 7 to <9 years | 21 participants | 39 participants | 60 participants |
| Age, Customized 9 to <11 years | 14 participants | 33 participants | 47 participants |
| Anisometropia at Randomization | 3.1 diopters STANDARD_DEVIATION 2 | 2.5 diopters STANDARD_DEVIATION 2.1 | 2.7 diopters STANDARD_DEVIATION 2.1 |
| Cause of Amblyopia Anisometropia | 16 participants | 20 participants | 36 participants |
| Cause of Amblyopia Combined Mechanism | 28 participants | 48 participants | 76 participants |
| Cause of Amblyopia Strabismus | 5 participants | 22 participants | 27 participants |
| Intraocular Visual Acuity Difference at Randomization | 35.6 letters STANDARD_DEVIATION 13.1 | 33.9 letters STANDARD_DEVIATION 10.9 | 34.5 letters STANDARD_DEVIATION 11.7 |
| Patching Duration at Randomization 2 hours per day | 43 participants | 81 participants | 124 participants |
| Patching Duration at Randomization >= 3 hours per day | 6 participants | 9 participants | 15 participants |
| Race/Ethnicity, Customized Asian | 1 participants | 2 participants | 3 participants |
| Race/Ethnicity, Customized Black/African American | 1 participants | 1 participants | 2 participants |
| Race/Ethnicity, Customized Hispanic or Latino | 2 participants | 5 participants | 7 participants |
| Race/Ethnicity, Customized Unknown/Not Reported | 1 participants | 5 participants | 6 participants |
| Race/Ethnicity, Customized White | 44 participants | 77 participants | 121 participants |
| Region of Enrollment United States | 49 participants | 90 participants | 139 participants |
| SE Refractive Error in Amblyopic Eye at Randomization | 4.5 diopters STANDARD_DEVIATION 2.3 | 4.0 diopters STANDARD_DEVIATION 2.6 | 4.2 diopters STANDARD_DEVIATION 2.5 |
| SE Refractive Error in Fellow Eye at Randomization | 1.6 diopters STANDARD_DEVIATION 1.7 | 1.9 diopters STANDARD_DEVIATION 2 | 1.8 diopters STANDARD_DEVIATION 1.9 |
| Sex: Female, Male Female | 24 Participants | 40 Participants | 64 Participants |
| Sex: Female, Male Male | 25 Participants | 50 Participants | 75 Participants |
| Visual Acuity in the Amblyopic Eye at Randomization 20/100 to <20/200 (38-52 letters) | 13 participants | 34 participants | 47 participants |
| Visual Acuity in the Amblyopic Eye at Randomization 20/200 or worse (<=37 letters) | 9 participants | 7 participants | 16 participants |
| Visual Acuity in the Amblyopic Eye at Randomization 20/50 (63-67 letters) | 13 participants | 14 participants | 27 participants |
| Visual Acuity in the Amblyopic Eye at Randomization 20/63 (58-62 letters) | 6 participants | 20 participants | 26 participants |
| Visual Acuity in the Amblyopic Eye at Randomization 20/80 (53-57 letters) | 8 participants | 15 participants | 23 participants |
| Visual Acuity in the Amblyopic Eye at Randomization | 51.7 letters STANDARD_DEVIATION 11.9 | 52.8 letters STANDARD_DEVIATION 9.8 | 52.4 letters STANDARD_DEVIATION 10.5 |
| Visual Acuity in the Fellow Eye at Randomization 20/12 (93-97 letters) | 2 participants | 6 participants | 8 participants |
| Visual Acuity in the Fellow Eye at Randomization 20/16 (88-92 letters) | 23 participants | 34 participants | 57 participants |
| Visual Acuity in the Fellow Eye at Randomization 20/20 (83-87 letters) | 20 participants | 35 participants | 55 participants |
| Visual Acuity in the Fellow Eye at Randomization 20/25 (78-82 letters) | 4 participants | 15 participants | 19 participants |
| Visual Acuity in the Fellow Eye at Randomization | 87.3 letters STANDARD_DEVIATION 3.5 | 86.7 letters STANDARD_DEVIATION 4.1 | 86.9 letters STANDARD_DEVIATION 3.9 |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 37 / 90 | 17 / 49 |
| serious Total, serious adverse events | 0 / 90 | 0 / 49 |
Outcome results
Primary
Distribution of Amblyopic Eye Visual Acuity Change From Baseline
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.
Time frame: 18 weeks after enrollment
Population: 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.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity Change From Baseline | 10-14 letters worse | 0 participants |
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity Change From Baseline | 5-9 letters worse | 2 participants |
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity Change From Baseline | within 4 letters | 35 participants |
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity Change From Baseline | 5-9 letters better | 36 participants |
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity Change From Baseline | 10-14 letters better | 10 participants |
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity Change From Baseline | >= 15 letters better | 3 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity Change From Baseline | 10-14 letters better | 1 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity Change From Baseline | 10-14 letters worse | 0 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity Change From Baseline | 5-9 letters better | 19 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity Change From Baseline | 5-9 letters worse | 1 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity Change From Baseline | >= 15 letters better | 1 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity Change From Baseline | within 4 letters | 23 participants |
Primary
Mean Amblyopic Eye Visual Acuity Change From Baseline
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.
Time frame: 18 weeks after enrollment
Population: 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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Levodopa/Carbidopa | Mean Amblyopic Eye Visual Acuity Change From Baseline | 5.2 letters | Standard Deviation 5.3 |
| Placebo | Mean Amblyopic Eye Visual Acuity Change From Baseline | 3.8 letters | Standard Deviation 5 |
Comparison: With 129 participants, assuming a 1-sided type I error rate of 4.85%, there was 96% power to detect a difference in mean visual acuity between treatment groups at 18 weeks adjusted for baseline and for 1 interim analysis for futility if the true difference was 5 letters with SD of 7 letters and 82% power if the true difference was 3.75 lettersp-value: 0.0695% CI: [-0.4, 3.3]ANCOVA
Secondary
Amblyopia Resolution at 10 Weeks
Treatment group comparisons adjusted for baseline acuity scores using logistic regression of the proportion of subjects with 20/25 or better visual acuity.
Time frame: 10 weeks after enrollment
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Levodopa/Carbidopa | Amblyopia Resolution at 10 Weeks | 20/25 or better | 0 participants |
| Levodopa/Carbidopa | Amblyopia Resolution at 10 Weeks | 20/32 or worse | 88 participants |
| Placebo | Amblyopia Resolution at 10 Weeks | 20/25 or better | 0 participants |
| Placebo | Amblyopia Resolution at 10 Weeks | 20/32 or worse | 48 participants |
Secondary
Amblyopia Resolution at 16 Weeks
Treatment group comparisons adjusted for baseline acuity scores using logistic regression of the proportion of subjects with 20/25 or better visual acuity.
Time frame: 16 weeks after enrollment
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Levodopa/Carbidopa | Amblyopia Resolution at 16 Weeks | 20/25 or better | 1 participants |
| Levodopa/Carbidopa | Amblyopia Resolution at 16 Weeks | 20/32 or worse | 86 participants |
| Placebo | Amblyopia Resolution at 16 Weeks | 20/25 or better | 0 participants |
| Placebo | Amblyopia Resolution at 16 Weeks | 20/32 or worse | 46 participants |
Secondary
Amblyopia Resolution at 18 Weeks
Treatment group comparisons adjusted for baseline acuity scores using logistic regression of the proportion of subjects with 20/25 or better visual acuity.
Time frame: 18 weeks after enrollment
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Levodopa/Carbidopa | Amblyopia Resolution at 18 Weeks | 20/25 or better | 0 participants |
| Levodopa/Carbidopa | Amblyopia Resolution at 18 Weeks | 20/32 or worse | 86 participants |
| Placebo | Amblyopia Resolution at 18 Weeks | 20/25 or better | 0 participants |
| Placebo | Amblyopia Resolution at 18 Weeks | 20/32 or worse | 45 participants |
Secondary
Amblyopia Resolution at 26 Weeks
Treatment group comparisons adjusted for baseline acuity scores using logistic regression of the proportion of subjects with 20/25 or better visual acuity.
Time frame: 26 weeks after enrollment
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Levodopa/Carbidopa | Amblyopia Resolution at 26 Weeks | 20/25 or better | 0 participants |
| Levodopa/Carbidopa | Amblyopia Resolution at 26 Weeks | 20/32 or worse | 86 participants |
| Placebo | Amblyopia Resolution at 26 Weeks | 20/25 or better | 0 participants |
| Placebo | Amblyopia Resolution at 26 Weeks | 20/32 or worse | 45 participants |
Secondary
Amblyopia Resolutionat 4 Weeks
Treatment group comparisons adjusted for baseline acuity scores using logistic regression of the proportion of subjects with 20/25 or better visual acuity.
Time frame: 4 weeks after enrollment
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Levodopa/Carbidopa | Amblyopia Resolutionat 4 Weeks | 20/25 or better | 0 participants |
| Levodopa/Carbidopa | Amblyopia Resolutionat 4 Weeks | 20/32 or worse | 88 participants |
| Placebo | Amblyopia Resolutionat 4 Weeks | 20/25 or better | 0 participants |
| Placebo | Amblyopia Resolutionat 4 Weeks | 20/32 or worse | 47 participants |
Secondary
Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 10 Weeks
Treatment group comparisons of the proportion of subjects who have improved from baseline by 10 or more letters.
Time frame: 10 weeks after enrollment
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Levodopa/Carbidopa | Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 10 Weeks | Improved 10 or more letters | 10 participants |
| Levodopa/Carbidopa | Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 10 Weeks | Not improved 10 or more letters | 78 participants |
| Placebo | Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 10 Weeks | Improved 10 or more letters | 9 participants |
| Placebo | Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 10 Weeks | Not improved 10 or more letters | 39 participants |
95% CI: [-25, 10]
Secondary
Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 16 Weeks
Treatment group comparisons of the proportion of subjects who have improved from baseline by 10 or more letters.
Time frame: 16 weeks after enrollment
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Levodopa/Carbidopa | Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 16 Weeks | Improved 10 or more letters | 15 participants |
| Levodopa/Carbidopa | Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 16 Weeks | Not improved 10 or more letters | 72 participants |
| Placebo | Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 16 Weeks | Improved 10 or more letters | 10 participants |
| Placebo | Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 16 Weeks | Not improved 10 or more letters | 36 participants |
95% CI: [-22, 13]
Secondary
Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 18 Weeks
Treatment group comparisons of the proportion of subjects who have improved from baseline by 10 or more letters.
Time frame: 18 weeks after enrollment
Population: 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
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Levodopa/Carbidopa | Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 18 Weeks | 13 participants |
| Placebo | Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 18 Weeks | 2 participants |
Comparison: A sample size of 129 participants provided 80% power with 1-sided type I error rate of 5% to reject the hypothesis of no difference between groups if the proportion improved was 30% in the levodopa group compared with 10% in the placebo group.p-value: 0.0695% CI: [-7, 28]Fisher Exact
Secondary
Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 26 Weeks
Treatment 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.
Time frame: 26 weeks after enrollment
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Levodopa/Carbidopa | Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 26 Weeks | Improved 10 or more letters | 17 participants |
| Levodopa/Carbidopa | Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 26 Weeks | Not improved 10 or more letters | 69 participants |
| Placebo | Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 26 Weeks | Improved 10 or more letters | 5 participants |
| Placebo | Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 26 Weeks | Not improved 10 or more letters | 40 participants |
Secondary
Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 4 Weeks
Treatment 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.
Time frame: 4 weeks after enrollment
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Levodopa/Carbidopa | Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 4 Weeks | Improved 10 or more letters | 4 participants |
| Levodopa/Carbidopa | Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 4 Weeks | Did not improve 10 or more letters | 84 participants |
| Placebo | Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 4 Weeks | Improved 10 or more letters | 2 participants |
| Placebo | Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 4 Weeks | Did not improve 10 or more letters | 45 participants |
95% CI: [-17, 18]
Secondary
Distribution of Amblyopic Eye Visual Acuity at 18 Weeks
Time frame: 18 weeks after enrollment
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity at 18 Weeks | 20/32 (73-77 letters) | 5 participants |
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity at 18 Weeks | 20/80 (53-57 letters) | 12 participants |
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity at 18 Weeks | 20/50 (63-67 letters) | 20 participants |
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity at 18 Weeks | 20/100 (48-52 letters) | 17 participants |
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity at 18 Weeks | 20/40 (68-72 letters) | 7 participants |
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity at 18 Weeks | 20/125 (43-47 letters) | 4 participants |
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity at 18 Weeks | 20/63 (58-62 letters) | 18 participants |
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity at 18 Weeks | 20/160 or worse (<=42 letters) | 3 participants |
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity at 18 Weeks | 20/25 (78-82 letters) | 0 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity at 18 Weeks | 20/160 or worse (<=42 letters) | 7 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity at 18 Weeks | 20/25 (78-82 letters) | 0 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity at 18 Weeks | 20/32 (73-77 letters) | 1 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity at 18 Weeks | 20/40 (68-72 letters) | 6 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity at 18 Weeks | 20/50 (63-67 letters) | 5 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity at 18 Weeks | 20/63 (58-62 letters) | 9 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity at 18 Weeks | 20/80 (53-57 letters) | 5 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity at 18 Weeks | 20/100 (48-52 letters) | 9 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity at 18 Weeks | 20/125 (43-47 letters) | 3 participants |
Secondary
Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 10 Weeks
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.
Time frame: 10 weeks after enrollment
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 10 Weeks | 10-14 letters worse | 1 participants |
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 10 Weeks | 5-9 letters worse | 4 participants |
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 10 Weeks | within 4 letters | 43 participants |
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 10 Weeks | 5-9 letters better | 30 participants |
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 10 Weeks | 10-14 letters better | 7 participants |
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 10 Weeks | >= 15 letters better | 3 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 10 Weeks | 10-14 letters better | 9 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 10 Weeks | 10-14 letters worse | 0 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 10 Weeks | 5-9 letters better | 8 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 10 Weeks | 5-9 letters worse | 1 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 10 Weeks | >= 15 letters better | 0 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 10 Weeks | within 4 letters | 30 participants |
Secondary
Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 16 Weeks
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.
Time frame: 16 weeks after enrollment
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 16 Weeks | 5-9 letters better | 30 participants |
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 16 Weeks | 5-9 letters worse | 1 participants |
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 16 Weeks | 10-14 letters better | 9 participants |
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 16 Weeks | 10-14 letters worse | 0 participants |
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 16 Weeks | >= 15 letters better | 6 participants |
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 16 Weeks | within 4 letters | 41 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 16 Weeks | >= 15 letters better | 0 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 16 Weeks | 10-14 letters worse | 0 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 16 Weeks | 5-9 letters worse | 0 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 16 Weeks | 5-9 letters better | 10 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 16 Weeks | 10-14 letters better | 10 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 16 Weeks | within 4 letters | 26 participants |
Secondary
Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 26 Weeks
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.
Time frame: 26 weeks after enrollment
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 26 Weeks | within 4 letters | 36 participants |
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 26 Weeks | 10-14 letters better | 14 participants |
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 26 Weeks | 5-9 letters better | 30 participants |
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 26 Weeks | >= 15 letters better | 3 participants |
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 26 Weeks | 5-9 letters worse | 3 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 26 Weeks | >= 15 letters better | 0 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 26 Weeks | 5-9 letters worse | 1 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 26 Weeks | within 4 letters | 23 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 26 Weeks | 5-9 letters better | 16 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 26 Weeks | 10-14 letters better | 5 participants |
Secondary
Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 4 Weeks
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.
Time frame: 4 weeks after enrollment
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 4 Weeks | 10-14 letters worse | 0 participants |
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 4 Weeks | 5-9 letters worse | 6 participants |
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 4 Weeks | within 4 letters | 55 participants |
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 4 Weeks | 5-9 letters better | 23 participants |
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 4 Weeks | 10-14 letters better | 4 participants |
| Levodopa/Carbidopa | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 4 Weeks | >= 15 letters better | 0 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 4 Weeks | 10-14 letters better | 1 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 4 Weeks | 10-14 letters worse | 0 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 4 Weeks | 5-9 letters better | 15 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 4 Weeks | 5-9 letters worse | 2 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 4 Weeks | >= 15 letters better | 1 participants |
| Placebo | Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 4 Weeks | within 4 letters | 28 participants |
Secondary
Distribution of Fellow Eye Visual Acuity at 18 Weeks
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 18 weeks after enrollment, adjusted for baseline acuity.
Time frame: 18 weeks after enrollment
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Levodopa/Carbidopa | Distribution of Fellow Eye Visual Acuity at 18 Weeks | 20/16 (88-92 letters) | 44 participants |
| Levodopa/Carbidopa | Distribution of Fellow Eye Visual Acuity at 18 Weeks | 20/25 (78-82 letters) | 8 participants |
| Levodopa/Carbidopa | Distribution of Fellow Eye Visual Acuity at 18 Weeks | 20/20 (83-87 letters) | 22 participants |
| Levodopa/Carbidopa | Distribution of Fellow Eye Visual Acuity at 18 Weeks | 20/32 (73-77 letters) | 1 participants |
| Levodopa/Carbidopa | Distribution of Fellow Eye Visual Acuity at 18 Weeks | 20/12 (93-97 letters) | 12 participants |
| Placebo | Distribution of Fellow Eye Visual Acuity at 18 Weeks | 20/32 (73-77 letters) | 0 participants |
| Placebo | Distribution of Fellow Eye Visual Acuity at 18 Weeks | 20/12 (93-97 letters) | 4 participants |
| Placebo | Distribution of Fellow Eye Visual Acuity at 18 Weeks | 20/16 (88-92 letters) | 22 participants |
| Placebo | Distribution of Fellow Eye Visual Acuity at 18 Weeks | 20/20 (83-87 letters) | 16 participants |
| Placebo | Distribution of Fellow Eye Visual Acuity at 18 Weeks | 20/25 (78-82 letters) | 3 participants |
Secondary
Distribution of Fellow Eye Visual Acuity at 26 Weeks
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.
Time frame: 26 weeks after enrollment
Population: 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.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Levodopa/Carbidopa | Distribution of Fellow Eye Visual Acuity at 26 Weeks | 20/16 (88-92 letters) | 34 participants |
| Levodopa/Carbidopa | Distribution of Fellow Eye Visual Acuity at 26 Weeks | 20/25 (78-82 letters) | 9 participants |
| Levodopa/Carbidopa | Distribution of Fellow Eye Visual Acuity at 26 Weeks | 20/20 (83-87 letters) | 28 participants |
| Levodopa/Carbidopa | Distribution of Fellow Eye Visual Acuity at 26 Weeks | 20/32 (73-77 letters) | 0 participants |
| Levodopa/Carbidopa | Distribution of Fellow Eye Visual Acuity at 26 Weeks | 20/12 (93-97 letters) | 15 participants |
| Placebo | Distribution of Fellow Eye Visual Acuity at 26 Weeks | 20/32 (73-77 letters) | 1 participants |
| Placebo | Distribution of Fellow Eye Visual Acuity at 26 Weeks | 20/12 (93-97 letters) | 7 participants |
| Placebo | Distribution of Fellow Eye Visual Acuity at 26 Weeks | 20/16 (88-92 letters) | 22 participants |
| Placebo | Distribution of Fellow Eye Visual Acuity at 26 Weeks | 20/20 (83-87 letters) | 13 participants |
| Placebo | Distribution of Fellow Eye Visual Acuity at 26 Weeks | 20/25 (78-82 letters) | 2 participants |
Secondary
Distribution of Fellow Eye Visual Acuity Change From Baseline at 18 Weeks
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.
Time frame: 18 weeks after enrollment
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Levodopa/Carbidopa | Distribution of Fellow Eye Visual Acuity Change From Baseline at 18 Weeks | >=15 letters worse | 0 participants |
| Levodopa/Carbidopa | Distribution of Fellow Eye Visual Acuity Change From Baseline at 18 Weeks | 10-14 letters worse | 0 participants |
| Levodopa/Carbidopa | Distribution of Fellow Eye Visual Acuity Change From Baseline at 18 Weeks | 5-9 letters worse | 4 participants |
| Levodopa/Carbidopa | Distribution of Fellow Eye Visual Acuity Change From Baseline at 18 Weeks | within 4 letters | 68 participants |
| Levodopa/Carbidopa | Distribution of Fellow Eye Visual Acuity Change From Baseline at 18 Weeks | 5-9 letters better | 15 participants |
| Levodopa/Carbidopa | Distribution of Fellow Eye Visual Acuity Change From Baseline at 18 Weeks | 10-14 letters better | 0 participants |
| Placebo | Distribution of Fellow Eye Visual Acuity Change From Baseline at 18 Weeks | 5-9 letters better | 3 participants |
| Placebo | Distribution of Fellow Eye Visual Acuity Change From Baseline at 18 Weeks | >=15 letters worse | 0 participants |
| Placebo | Distribution of Fellow Eye Visual Acuity Change From Baseline at 18 Weeks | within 4 letters | 39 participants |
| Placebo | Distribution of Fellow Eye Visual Acuity Change From Baseline at 18 Weeks | 10-14 letters worse | 0 participants |
| Placebo | Distribution of Fellow Eye Visual Acuity Change From Baseline at 18 Weeks | 10-14 letters better | 0 participants |
| Placebo | Distribution of Fellow Eye Visual Acuity Change From Baseline at 18 Weeks | 5-9 letters worse | 3 participants |
Secondary
Distribution of Fellow Eye Visual Acuity Change From Baseline at 26 Weeks
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.
Time frame: 26 weeks after enrollment
Population: 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.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Levodopa/Carbidopa | Distribution of Fellow Eye Visual Acuity Change From Baseline at 26 Weeks | >=15 letters worse | 1 participants |
| Levodopa/Carbidopa | Distribution of Fellow Eye Visual Acuity Change From Baseline at 26 Weeks | 10-14 letters worse | 0 participants |
| Levodopa/Carbidopa | Distribution of Fellow Eye Visual Acuity Change From Baseline at 26 Weeks | 5-9 letters worse | 2 participants |
| Levodopa/Carbidopa | Distribution of Fellow Eye Visual Acuity Change From Baseline at 26 Weeks | within 4 letters | 74 participants |
| Levodopa/Carbidopa | Distribution of Fellow Eye Visual Acuity Change From Baseline at 26 Weeks | 5-9 letters better | 7 participants |
| Levodopa/Carbidopa | Distribution of Fellow Eye Visual Acuity Change From Baseline at 26 Weeks | 10-14 letters better | 2 participants |
| Placebo | Distribution of Fellow Eye Visual Acuity Change From Baseline at 26 Weeks | 5-9 letters better | 6 participants |
| Placebo | Distribution of Fellow Eye Visual Acuity Change From Baseline at 26 Weeks | >=15 letters worse | 0 participants |
| Placebo | Distribution of Fellow Eye Visual Acuity Change From Baseline at 26 Weeks | within 4 letters | 35 participants |
| Placebo | Distribution of Fellow Eye Visual Acuity Change From Baseline at 26 Weeks | 10-14 letters worse | 0 participants |
| Placebo | Distribution of Fellow Eye Visual Acuity Change From Baseline at 26 Weeks | 10-14 letters better | 0 participants |
| Placebo | Distribution of Fellow Eye Visual Acuity Change From Baseline at 26 Weeks | 5-9 letters worse | 4 participants |
Secondary
Mean Amblyopic Eye Visual Acuity at 18 Weeks
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.
Time frame: 18 weeks after enrollment
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Levodopa/Carbidopa | Mean Amblyopic Eye Visual Acuity at 18 Weeks | 58.7 letters | Standard Deviation 8.9 |
| Placebo | Mean Amblyopic Eye Visual Acuity at 18 Weeks | 54.8 letters | Standard Deviation 12.3 |
Secondary
Mean Change in Amblyopic Eye Visual Acuity From Baseline at 10 Weeks
A 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.
Time frame: 10 weeks after enrollment
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Levodopa/Carbidopa | Mean Change in Amblyopic Eye Visual Acuity From Baseline at 10 Weeks | 3.8 letters | Standard Deviation 4.9 |
| Placebo | Mean Change in Amblyopic Eye Visual Acuity From Baseline at 10 Weeks | 3.7 letters | Standard Deviation 4.9 |
p-value: 0.4495% CI: [-1.6, 1.8]ANCOVA
Secondary
Mean Change in Amblyopic Eye Visual Acuity From Baseline at 16 Weeks
A 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.
Time frame: 16 weeks after enrollment
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Levodopa/Carbidopa | Mean Change in Amblyopic Eye Visual Acuity From Baseline at 16 Weeks | 5.1 letters | Standard Deviation 6.3 |
| Placebo | Mean Change in Amblyopic Eye Visual Acuity From Baseline at 16 Weeks | 4.2 letters | Standard Deviation 4.9 |
p-value: 0.295% CI: [-1.2, 2.9]ANCOVA
Secondary
Mean Change in Amblyopic Eye Visual Acuity From Baseline at 26 Weeks
A 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.
Time frame: 26 weeks after enrollment
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Levodopa/Carbidopa | Mean Change in Amblyopic Eye Visual Acuity From Baseline at 26 Weeks | 5.0 letters | Standard Deviation 5.7 |
| Placebo | Mean Change in Amblyopic Eye Visual Acuity From Baseline at 26 Weeks | 4.2 letters | Standard Deviation 4.7 |
p-value: 0.17ANCOVA
Secondary
Mean Change in Amblyopic Eye Visual Acuity From Baseline at 4 Weeks
A treatment group comparison of the mean amblyopic eye visual acuity at 4 weeks after enrollment, adjusted for baseline acuity.
Time frame: 4 weeks after enrollment
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Levodopa/Carbidopa | Mean Change in Amblyopic Eye Visual Acuity From Baseline at 4 Weeks | 2.2 letters | Standard Deviation 4.1 |
| Placebo | Mean Change in Amblyopic Eye Visual Acuity From Baseline at 4 Weeks | 2.5 letters | Standard Deviation 4.7 |
p-value: 0.6595% CI: [-1.9, 1.3]ANCOVA
Secondary
Mean Child Symptom Survey Score at 10 Weeks
A 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).
Time frame: 10 weeks after enrollment
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Levodopa/Carbidopa | Mean Child Symptom Survey Score at 10 Weeks | 1.33 units on a scale | Standard Deviation 0.31 |
| Placebo | Mean Child Symptom Survey Score at 10 Weeks | 1.42 units on a scale | Standard Deviation 0.37 |
Secondary
Mean Child Symptom Survey Score at 16 Weeks
A 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).
Time frame: 16 weeks after enrollment
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Levodopa/Carbidopa | Mean Child Symptom Survey Score at 16 Weeks | 1.25 units on a scale | Standard Deviation 0.27 |
| Placebo | Mean Child Symptom Survey Score at 16 Weeks | 1.38 units on a scale | Standard Deviation 0.36 |
Secondary
Mean Child Symptom Survey Score at 18 Weeks
A 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).
Time frame: 18 weeks after enrollment
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Levodopa/Carbidopa | Mean Child Symptom Survey Score at 18 Weeks | 1.17 units on a scale | Standard Deviation 0.22 |
| Placebo | Mean Child Symptom Survey Score at 18 Weeks | 1.28 units on a scale | Standard Deviation 0.32 |
Secondary
Mean Child Symptom Survey Score at 26 Weeks
A 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).
Time frame: 26 weeks after enrollment
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Levodopa/Carbidopa | Mean Child Symptom Survey Score at 26 Weeks | 1.23 units on a scale | Standard Deviation 0.26 |
| Placebo | Mean Child Symptom Survey Score at 26 Weeks | 1.33 units on a scale | Standard Deviation 0.32 |
Secondary
Mean Child Symptom Survey Score at 4 Weeks
A 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).
Time frame: 4 weeks after enrollment
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Levodopa/Carbidopa | Mean Child Symptom Survey Score at 4 Weeks | 1.42 units on a scale | Standard Deviation 0.37 |
| Placebo | Mean Child Symptom Survey Score at 4 Weeks | 1.51 units on a scale | Standard Deviation 0.42 |
Secondary
Mean Child Symptom Survey Score at Enrollment
A 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).
Time frame: At enrollment
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Levodopa/Carbidopa | Mean Child Symptom Survey Score at Enrollment | 1.60 units on a scale | Standard Deviation 0.37 |
| Placebo | Mean Child Symptom Survey Score at Enrollment | 1.73 units on a scale | Standard Deviation 0.45 |
Secondary
Mean Fellow Eye Visual Acuity at 18 Weeks
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.
Time frame: 18 weeks after enrollment
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Levodopa/Carbidopa | Mean Fellow Eye Visual Acuity at 18 Weeks | 88.1 letters | Standard Deviation 4.4 |
| Placebo | Mean Fellow Eye Visual Acuity at 18 Weeks | 88.0 letters | Standard Deviation 3.2 |
Secondary
Mean Fellow Eye Visual Acuity at 26 Weeks
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.
Time frame: 26 weeks after enrollment
Population: 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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Levodopa/Carbidopa | Mean Fellow Eye Visual Acuity at 26 Weeks | 88.0 letters | Standard Deviation 4.5 |
| Placebo | Mean Fellow Eye Visual Acuity at 26 Weeks | 88.2 letters | Standard Deviation 4.1 |
Secondary
Mean Fellow Eye Visual Acuity Change From Baseline at 18 Weeks
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.
Time frame: 18 weeks after enrollment
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Levodopa/Carbidopa | Mean Fellow Eye Visual Acuity Change From Baseline at 18 Weeks | 1.5 letters | Standard Deviation 3 |
| Placebo | Mean Fellow Eye Visual Acuity Change From Baseline at 18 Weeks | 0.5 letters | Standard Deviation 3.1 |
Secondary
Mean Fellow Eye Visual Acuity Change From Baseline at 26 Weeks
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.
Time frame: 26 weeks after enrollment
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Levodopa/Carbidopa | Mean Fellow Eye Visual Acuity Change From Baseline at 26 Weeks | 1.4 letters | Standard Deviation 3.6 |
| Placebo | Mean Fellow Eye Visual Acuity Change From Baseline at 26 Weeks | 0.8 letters | Standard Deviation 3.3 |
Secondary
Mean Parent Symptom Survey Score at 10 Weeks
A 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).
Time frame: 10 weeks after enrollment
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Levodopa/Carbidopa | Mean Parent Symptom Survey Score at 10 Weeks | 1.22 units on a scale | Standard Deviation 0.22 |
| Placebo | Mean Parent Symptom Survey Score at 10 Weeks | 1.30 units on a scale | Standard Deviation 0.25 |
Secondary
Mean Parent Symptom Survey Score at 16 Weeks
A 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).
Time frame: 16 weeks after enrollment
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Levodopa/Carbidopa | Mean Parent Symptom Survey Score at 16 Weeks | 1.24 units on a scale | Standard Deviation 0.27 |
| Placebo | Mean Parent Symptom Survey Score at 16 Weeks | 1.25 units on a scale | Standard Deviation 0.22 |
Secondary
Mean Parent Symptom Survey Score at 18 Weeks
A 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).
Time frame: 18 weeks after enrollment
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Levodopa/Carbidopa | Mean Parent Symptom Survey Score at 18 Weeks | 1.17 units on a scale | Standard Deviation 0.21 |
| Placebo | Mean Parent Symptom Survey Score at 18 Weeks | 1.17 units on a scale | Standard Deviation 0.2 |
Secondary
Mean Parent Symptom Survey Score at 26 Weeks
A 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).
Time frame: 26 weeks after enrollment
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Levodopa/Carbidopa | Mean Parent Symptom Survey Score at 26 Weeks | 1.19 units on a scale | Standard Deviation 0.24 |
| Placebo | Mean Parent Symptom Survey Score at 26 Weeks | 1.22 units on a scale | Standard Deviation 0.29 |
Secondary
Mean Parent Symptom Survey Score at 4 Weeks
A 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).
Time frame: 4 weeks after enrollment
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Levodopa/Carbidopa | Mean Parent Symptom Survey Score at 4 Weeks | 1.29 units on a scale | Standard Deviation 0.27 |
| Placebo | Mean Parent Symptom Survey Score at 4 Weeks | 1.26 units on a scale | Standard Deviation 0.27 |
Secondary
Mean Parent Symptom Survey Score at Enrollment
A 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).
Time frame: At enrollment
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Levodopa/Carbidopa | Mean Parent Symptom Survey Score at Enrollment | 1.42 units on a scale | Standard Deviation 0.34 |
| Placebo | Mean Parent Symptom Survey Score at Enrollment | 1.44 units on a scale | Standard Deviation 0.29 |
Secondary
Mean Systemic Adverse Events
Time frame: Enrollment through 26 weeks
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Levodopa/Carbidopa | Mean Systemic Adverse Events | 1.28 events | Standard Deviation 1.39 |
| Placebo | Mean Systemic Adverse Events | 1.55 events | Standard Deviation 1.9 |