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| ID | Type | Description | Link |
|---|---|---|---|
| 2U10EY011751 | U.S. NIH Grant/Contract | View source |
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| Name | Class |
|---|---|
| National Eye Institute (NEI) | NIH |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Levodopa/Carbidopa | Active Comparator | Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid |
|
| Placebo | Placebo Comparator | Oral placebo tid |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Levodopa/Carbidopa | Drug | Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid |
| |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 18 weeks after enrollment |
| 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. | 18 weeks after enrollment |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 18 weeks after enrollment |
| Distribution of Amblyopic Eye Visual Acuity at 18 Weeks |
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Inclusion Criteria:
Age 7 to 12
Amblyopia associated with strabismus, anisometropia, or both
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:
Current amblyopia treatment (other than spectacles)
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:
Requirements for spectacle correction:
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.
Eye examination within 6 months prior to enrollment
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
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:
Myopia more than -6.00 D (spherical equivalent) in either eye.
Current vision therapy or orthoptics
Ocular cause for reduced visual acuity
Prior intraocular or refractive surgery
History of narrow-angle glaucoma
Bronchial asthma or severe pulmonary disease
Strabismus surgery planned within 26 weeks
Known allergy to levodopa or carbidopa
History of dystonic reactions
Current use of oral iron supplements including multivitamins containing iron during treatment with levodopa-carbidopa
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
Known liver disease
History of melanoma
Known psychological problems
Known skin reactions to patch or bandage adhesives
Prior levodopa treatment
Treatment with topical ophthalmic atropine within the past 12 weeks
A physician-prescribed diet high in protein
Females who are pregnant, lactating, or intend to become pregnant within the next 34 weeks.
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| Name | Affiliation | Role |
|---|---|---|
| Michael X Repka, MD | Jaeb Center for Health Research | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Wilmer Eye Institute | Baltimore | Maryland | 21287 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 25676904 | Result | Pediatric Eye Disease Investigator Group; Repka MX, Kraker RT, Dean TW, Beck RW, Siatkowski RM, Holmes JM, Beauchamp CL, Golden RP, Miller AM, Verderber LC, Wallace DK. A randomized trial of levodopa as treatment for residual amblyopia in older children. Ophthalmology. 2015 May;122(5):874-81. doi: 10.1016/j.ophtha.2015.01.002. Epub 2015 Feb 9. |
| Label | URL |
|---|---|
| PubMed abstract | View source |
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A de-identified dataset is available on the PEDIG public website at http://pedig.jaeb.org/Studies.aspx?RecID=196
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Between September 2010 and October 2013, 139 participants from 27 sites were randomly assigned to levodopa (n=90) or placebo (n=49).
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| ID | Title | Description |
|---|---|---|
| FG000 | 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 |
| FG001 | Placebo | Oral placebo tid Placebo: Oral placebo tid Patching: Two hours of daily patching |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | 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 |
| BG001 | Placebo |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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. | 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. | Posted | Number | participants | 18 weeks after enrollment |
|
From enrollment to last follow-up visit (26 weeks)
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Headache | General disorders | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Raymond Kraker, M.S.P.H., Director of PEDIG Coordinating Center | Jaeb Center for Health Research | 813-975-8690 | rkraker@jaeb.org |
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| ID | Term |
|---|---|
| D000550 | Amblyopia |
| ID | Term |
|---|---|
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D014786 | Vision Disorders |
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| ID | Term |
|---|---|
| C009265 | carbidopa, levodopa drug combination |
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| Placebo |
| Drug |
Oral placebo tid |
|
| Patching | Other | Two hours of daily patching |
|
| 18 weeks after enrollment |
| 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. | 18 weeks after enrollment |
| 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. | 4 weeks after enrollment |
| 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. | 10 weeks after enrollment |
| 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. | 16 weeks after enrollment |
| 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. | 26 weeks after enrollment |
| 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. | 4 weeks after enrollment |
| 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. | 10 weeks after enrollment |
| 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. | 16 weeks after enrollment |
| 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. | 18 weeks after enrollment |
| 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. | 26 weeks after enrollment |
| 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. | 4 weeks after enrollment |
| 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. | 4 weeks after enrollment |
| 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. | 10 weeks after enrollment |
| 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. | 10 weeks after enrollment |
| 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. | 16 weeks after enrollment |
| 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. | 16 weeks after enrollment |
| 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. | 26 weeks after enrollment |
| 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. | 26 weeks after enrollment |
| 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. | 18 weeks after enrollment |
| 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. | 18 weeks after enrollment |
| 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. | 18 weeks after enrollment |
| 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. | 18 weeks after enrollment |
| 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). | At enrollment |
| 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). | 4 weeks after enrollment |
| 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). | 10 weeks after enrollment |
| 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). | 16 weeks after enrollment |
| 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). | 18 weeks after enrollment |
| 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). | 26 weeks after enrollment |
| 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). | At enrollment |
| 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). | 4 weeks after enrollment |
| 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). | 10 weeks after enrollment |
| 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). | 16 weeks after enrollment |
| 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). | 18 weeks after enrollment |
| 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). | 26 weeks after enrollment |
| Mean Systemic Adverse Events | Enrollment through 26 weeks |
| 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. | 26 weeks after enrollment |
| 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. | 26 weeks after enrollment |
| 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. | 26 weeks after enrollment |
| 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. | 26 weeks after enrollment |
| PubMed Central HHS Public Access - Full Text | View source |
| Refused study treatment after randomized |
|
| Adverse Event |
|
Oral placebo tid
Placebo: Oral placebo tid
Patching: Two hours of daily patching
| BG002 | Total | Total of all reporting groups |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Age, Customized | Number | participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Number | participants |
|
| Region of Enrollment | Number | participants |
|
| Cause of Amblyopia | Number | participants |
|
| Patching Duration at Randomization | Number | participants |
|
| Visual Acuity in the Amblyopic Eye at Randomization | Number | participants |
|
| Visual Acuity in the Amblyopic Eye at Randomization | Mean | Standard Deviation | letters |
|
| Visual Acuity in the Fellow Eye at Randomization | Number | participants |
|
| Visual Acuity in the Fellow Eye at Randomization | Mean | Standard Deviation | letters |
|
| Intraocular Visual Acuity Difference at Randomization | Mean | Standard Deviation | letters |
|
| SE Refractive Error in Amblyopic Eye at Randomization | Mean | Standard Deviation | diopters |
|
| SE Refractive Error in Fellow Eye at Randomization | Mean | Standard Deviation | diopters |
|
| Anisometropia at Randomization | Mean | Standard Deviation | diopters |
|
| 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 |
| OG001 | Placebo | Oral placebo tid Placebo: Oral placebo tid Patching: Two hours of daily patching |
|
|
| 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. | 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 | Posted | Number | participants | 18 weeks after enrollment |
|
|
|
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| Secondary | Distribution of Amblyopic Eye Visual Acuity at 18 Weeks | Posted | Number | participants | 18 weeks after enrollment |
|
|
|
| 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. | Posted | Mean | Standard Deviation | letters | 18 weeks after enrollment |
|
|
|
| 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. | Posted | Number | participants | 4 weeks after enrollment |
|
|
|
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| 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. | Posted | Number | participants | 10 weeks after enrollment |
|
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|
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| 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. | Posted | Number | participants | 16 weeks after enrollment |
|
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|
|
| 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. | Posted | Number | participants | 26 weeks after enrollment |
|
|
|
| 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. | Posted | Number | participants | 4 weeks after enrollment |
|
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| 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. | Posted | Number | participants | 10 weeks after enrollment |
|
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| 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. | Posted | Number | participants | 16 weeks after enrollment |
|
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| 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. | Posted | Number | participants | 18 weeks after enrollment |
|
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| 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. | Posted | Number | participants | 26 weeks after enrollment |
|
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| 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. | Posted | Number | participants | 4 weeks after enrollment |
|
|
|
| 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. | Posted | Mean | Standard Deviation | letters | 4 weeks after enrollment |
|
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|
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| 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. | Posted | Number | participants | 10 weeks after enrollment |
|
|
|
| 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. | Posted | Mean | Standard Deviation | letters | 10 weeks after enrollment |
|
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| 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. | Posted | Number | participants | 16 weeks after enrollment |
|
|
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| 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. | Posted | Mean | Standard Deviation | letters | 16 weeks after enrollment |
|
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| 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. | Posted | Number | participants | 26 weeks after enrollment |
|
|
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| 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. | Posted | Mean | Standard Deviation | letters | 26 weeks after enrollment |
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| 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. | Posted | Number | participants | 18 weeks after enrollment |
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| 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. | Posted | Mean | Standard Deviation | letters | 18 weeks after enrollment |
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| 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. | Posted | Number | participants | 18 weeks after enrollment |
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| 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. | 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. | Posted | Mean | Standard Deviation | letters | 18 weeks after enrollment |
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| 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. | Posted | Mean | Standard Deviation | letters | 18 weeks after enrollment |
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| 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). | Posted | Mean | Standard Deviation | units on a scale | At enrollment |
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| 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). | Posted | Mean | Standard Deviation | units on a scale | 4 weeks after enrollment |
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| 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). | Posted | Mean | Standard Deviation | units on a scale | 10 weeks after enrollment |
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| 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). | Posted | Mean | Standard Deviation | units on a scale | 16 weeks after enrollment |
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| 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). | Posted | Mean | Standard Deviation | units on a scale | 18 weeks after enrollment |
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| 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). | Posted | Mean | Standard Deviation | units on a scale | 26 weeks after enrollment |
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| 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). | Posted | Mean | Standard Deviation | units on a scale | At enrollment |
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| 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). | Posted | Mean | Standard Deviation | units on a scale | 4 weeks after enrollment |
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| 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). | Posted | Mean | Standard Deviation | units on a scale | 10 weeks after enrollment |
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| 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). | Posted | Mean | Standard Deviation | units on a scale | 16 weeks after enrollment |
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| 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). | Posted | Mean | Standard Deviation | units on a scale | 18 weeks after enrollment |
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| 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). | Posted | Mean | Standard Deviation | units on a scale | 26 weeks after enrollment |
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| Secondary | Mean Systemic Adverse Events | Posted | Mean | Standard Deviation | events | Enrollment through 26 weeks |
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| 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. | 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. | Posted | Number | participants | 26 weeks after enrollment |
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| 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. | 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. | Posted | Mean | Standard Deviation | letters | 26 weeks after enrollment |
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| 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. | 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. | Posted | Number | participants | 26 weeks after enrollment |
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| 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. | Posted | Mean | Standard Deviation | letters | 26 weeks after enrollment |
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| 0 |
| 90 |
| 37 |
| 90 |
| EG001 | Placebo | Oral placebo tid Placebo: Oral placebo tid Patching: Two hours of daily patching | 0 | 49 | 17 | 49 |
| Nausea | General disorders | Non-systematic Assessment |
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| Cough | General disorders | Non-systematic Assessment |
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| Gastroenteritis viral | Gastrointestinal disorders | Non-systematic Assessment |
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| Nasopharyngitis | General disorders | Non-systematic Assessment |
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| Pharyngitis streptococcal | General disorders | Non-systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | Non-systematic Assessment |
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| Dry eye | Eye disorders | Non-systematic Assessment |
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| Eye irritation | Eye disorders | Non-systematic Assessment |
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Not provided
Not provided
| D012678 | Sensation Disorders |
| D009461 | Neurologic Manifestations |
| D005128 | Eye Diseases |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
| 20/40 (68-72 letters) |
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| 20/50 (63-67 letters) |
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| 20/63 (58-62 letters) |
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| 20/80 (53-57 letters) |
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| 20/100 (48-52 letters) |
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| 20/125 (43-47 letters) |
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| 20/160 or worse (<=42 letters) |
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| within 4 letters |
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| 5-9 letters better |
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| 10-14 letters better |
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| >= 15 letters better |
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| within 4 letters |
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| 5-9 letters better |
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| 10-14 letters better |
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| >= 15 letters better |
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| within 4 letters |
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| 5-9 letters better |
|
| 10-14 letters better |
|
| >= 15 letters better |
|
| 5-9 letters better |
|
| 10-14 letters better |
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| >= 15 letters better |
|
| 20/20 (83-87 letters) |
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| 20/25 (78-82 letters) |
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| 20/32 (73-77 letters) |
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| 5-9 letters worse |
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| within 4 letters |
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| 5-9 letters better |
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| 10-14 letters better |
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| Superiority or Other |
| 20/20 (83-87 letters) |
|
| 20/25 (78-82 letters) |
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| 20/32 (73-77 letters) |
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| 5-9 letters worse |
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| within 4 letters |
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| 5-9 letters better |
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| 10-14 letters better |
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