Trial Outcomes & Findings for Pilot Study to Evaluate Levodopa as Treatment for Residual Amblyopia (NCT NCT00789672)

Last Updated: 2016-07-13

Results Overview

Visual acuity was measured with the electronic early treatment diabetic retinopathy study (E-ETDRS) method and resulted in a letter score that could range from 0 to 97 letters, with 0 being the worst and 97 being the best. Letter scores are presented as Snellen Equivalents for presentation (i.e. 20/20 includes those with letter scores between 83 and 87 letters, 20/25 includes those with letter scores between 78 to 82 letters, etc.).

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

33 participants

Primary outcome timeframe

9 weeks after starting levodopa

Results posted on

2016-07-13

Participant Flow

Eligibility criteria included age 8 to \<18 years, best-corrected visual acuity in the amblyopic eye between 67 and 18 letters inclusive measured with E-ETDRS, fellow eye best-corrected visual acuity of 78 letters or better, and the presence or history of strabismus and/or anisometropia.

At enrollment, subjects were required to have been treated with at least 2 hours of patching per day of daily patching and while on that therapy, have had stable visual acuity (5 letters or one logMAR line of improvement since a previous visit at least 8 weeks earlier)

Participant milestones

Participant milestones
Measure	Lower Dose 0.51 mg Levodopa/Carbidopa Oral levodopa 0.51 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.	Higher Dose 0.76 mg Levodopa/Carbidopa Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.
Overall Study STARTED	16	17
Overall Study COMPLETED	16	17
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Pilot Study to Evaluate Levodopa as Treatment for Residual Amblyopia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Lower Dose 0.51 mg Levodopa/Carbidopa n=16 Participants Oral levodopa 0.51 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.	Higher Dose 0.76 mg Levodopa/Carbidopa n=17 Participants Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.	Total n=33 Participants Total of all reporting groups
Age, Continuous	11.3 years STANDARD_DEVIATION 2.2 • n=5 Participants	11.0 years STANDARD_DEVIATION 2.2 • n=7 Participants	11.1 years STANDARD_DEVIATION 2.2 • n=5 Participants
Age, Customized 8 to <9	3 participants n=5 Participants	3 participants n=7 Participants	6 participants n=5 Participants
Age, Customized 9 to <10	2 participants n=5 Participants	3 participants n=7 Participants	5 participants n=5 Participants
Age, Customized 10 to <11	3 participants n=5 Participants	4 participants n=7 Participants	7 participants n=5 Participants
Age, Customized 11 to <12	3 participants n=5 Participants	1 participants n=7 Participants	4 participants n=5 Participants
Age, Customized 12 to <13	0 participants n=5 Participants	3 participants n=7 Participants	3 participants n=5 Participants
Age, Customized 13 to <14	3 participants n=5 Participants	1 participants n=7 Participants	4 participants n=5 Participants
Age, Customized 14 to <15	2 participants n=5 Participants	0 participants n=7 Participants	2 participants n=5 Participants
Age, Customized 15 to <16	0 participants n=5 Participants	2 participants n=7 Participants	2 participants n=5 Participants
Age, Customized 16 to <17	0 participants n=5 Participants	0 participants n=7 Participants	0 participants n=5 Participants
Age, Customized 17 to <18	0 participants n=5 Participants	0 participants n=7 Participants	0 participants n=5 Participants
Sex: Female, Male Female	10 Participants n=5 Participants	9 Participants n=7 Participants	19 Participants n=5 Participants
Sex: Female, Male Male	6 Participants n=5 Participants	8 Participants n=7 Participants	14 Participants n=5 Participants
Region of Enrollment United States	16 participants n=5 Participants	17 participants n=7 Participants	33 participants n=5 Participants
Cause of Amblyopia Strabismus	2 participants n=5 Participants	5 participants n=7 Participants	7 participants n=5 Participants
Cause of Amblyopia Anisometropia	6 participants n=5 Participants	5 participants n=7 Participants	11 participants n=5 Participants
Cause of Amblyopia Strabismus and anisometropia	8 participants n=5 Participants	7 participants n=7 Participants	15 participants n=5 Participants
Distance Visual Acuity in Amblyopic Eye 20/400 (18 to 22 letters)	0 participants n=5 Participants	1 participants n=7 Participants	1 participants n=5 Participants
Distance Visual Acuity in Amblyopic Eye 20/320 (23 to 27 letters)	0 participants n=5 Participants	0 participants n=7 Participants	0 participants n=5 Participants
Distance Visual Acuity in Amblyopic Eye 20/250 (28 to 32 letters)	0 participants n=5 Participants	1 participants n=7 Participants	1 participants n=5 Participants
Distance Visual Acuity in Amblyopic Eye 20/200 (33 to 37 letters)	1 participants n=5 Participants	1 participants n=7 Participants	2 participants n=5 Participants
Distance Visual Acuity in Amblyopic Eye 20/160 (38 to 42 letters)	1 participants n=5 Participants	1 participants n=7 Participants	2 participants n=5 Participants
Distance Visual Acuity in Amblyopic Eye 20/125 (43 to 47 letters)	0 participants n=5 Participants	1 participants n=7 Participants	1 participants n=5 Participants
Distance Visual Acuity in Amblyopic Eye 20/100 (48 to 52 letters)	2 participants n=5 Participants	0 participants n=7 Participants	2 participants n=5 Participants
Distance Visual Acuity in Amblyopic Eye 20/80 (53 to 57 letters)	4 participants n=5 Participants	6 participants n=7 Participants	10 participants n=5 Participants
Distance Visual Acuity in Amblyopic Eye 20/63 (58 to 62 letters)	4 participants n=5 Participants	5 participants n=7 Participants	9 participants n=5 Participants
Distance Visual Acuity in Amblyopic Eye 20/50 (63 to 67 letters)	4 participants n=5 Participants	1 participants n=7 Participants	5 participants n=5 Participants
Distance Visual Acuity in Fellow Eye 20/25 (78 to 82 letters)	4 participants n=5 Participants	4 participants n=7 Participants	8 participants n=5 Participants
Distance Visual Acuity in Fellow Eye 20/20 (83 to 87 letters)	6 participants n=5 Participants	8 participants n=7 Participants	14 participants n=5 Participants
Distance Visual Acuity in Fellow Eye 20/16 (88 to 92 letters)	5 participants n=5 Participants	5 participants n=7 Participants	10 participants n=5 Participants
Distance Visual Acuity in Fellow Eye 20/12 (93 to 97 letters)	1 participants n=5 Participants	0 participants n=7 Participants	1 participants n=5 Participants
Race/Ethnicity White	14 participants n=5 Participants	17 participants n=7 Participants	31 participants n=5 Participants
Race/Ethnicity African-American	1 participants n=5 Participants	0 participants n=7 Participants	1 participants n=5 Participants
Race/Ethnicity Hispanic or Latino	1 participants n=5 Participants	0 participants n=7 Participants	1 participants n=5 Participants
Race/Ethnicity Asian	0 participants n=5 Participants	0 participants n=7 Participants	0 participants n=5 Participants
Race/Ethnicity More than one race	0 participants n=5 Participants	0 participants n=7 Participants	0 participants n=5 Participants
Race/Ethnicity Unknown/Not reported	0 participants n=5 Participants	0 participants n=7 Participants	0 participants n=5 Participants
Refractive Error in Amblyopic Eye (spherical equivalent/diopters) <0.00D	0 participants n=5 Participants	1 participants n=7 Participants	1 participants n=5 Participants
Refractive Error in Amblyopic Eye (spherical equivalent/diopters) 0 to < +1.00D	1 participants n=5 Participants	2 participants n=7 Participants	3 participants n=5 Participants
Refractive Error in Amblyopic Eye (spherical equivalent/diopters) +1.00 to <+2.00D	1 participants n=5 Participants	1 participants n=7 Participants	2 participants n=5 Participants
Refractive Error in Amblyopic Eye (spherical equivalent/diopters) +2.00 to <+3.00D	2 participants n=5 Participants	2 participants n=7 Participants	4 participants n=5 Participants
Refractive Error in Amblyopic Eye (spherical equivalent/diopters) +3.00 to <+4.00D	3 participants n=5 Participants	2 participants n=7 Participants	5 participants n=5 Participants
Refractive Error in Amblyopic Eye (spherical equivalent/diopters) greater than or equal to +4.00D	9 participants n=5 Participants	9 participants n=7 Participants	18 participants n=5 Participants
Refractive Error in Fellow Eye (spherical equivalent/diopters) <0.00D	1 participants n=5 Participants	0 participants n=7 Participants	1 participants n=5 Participants
Refractive Error in Fellow Eye (spherical equivalent/diopters) 0 to < +1.00D	6 participants n=5 Participants	10 participants n=7 Participants	16 participants n=5 Participants
Refractive Error in Fellow Eye (spherical equivalent/diopters) +1.00 to <+2.00D	5 participants n=5 Participants	3 participants n=7 Participants	8 participants n=5 Participants
Refractive Error in Fellow Eye (spherical equivalent/diopters) +2.00 to <+3.00D	2 participants n=5 Participants	3 participants n=7 Participants	5 participants n=5 Participants
Refractive Error in Fellow Eye (spherical equivalent/diopters) +3.00 to <+4.00D	2 participants n=5 Participants	1 participants n=7 Participants	3 participants n=5 Participants
Refractive Error in Fellow Eye (spherical equivalent/diopters) greater than or equal to +4.00D	0 participants n=5 Participants	0 participants n=7 Participants	0 participants n=5 Participants
Mean (SD) Distance Visual Acuity in Amblyopic Eye	56.2 letters STANDARD_DEVIATION 8.8 • n=5 Participants	50.5 letters STANDARD_DEVIATION 12.2 • n=7 Participants	53.2 letters STANDARD_DEVIATION 10.9 • n=5 Participants
Mean (SD) Distance Visual Acuity in Fellow Eye	86.4 letters STANDARD_DEVIATION 4.5 • n=5 Participants	85.5 letters STANDARD_DEVIATION 3.3 • n=7 Participants	85.9 letters STANDARD_DEVIATION 3.9 • n=5 Participants
Mean (SD) Intereye Acuity Difference	30.2 letters STANDARD_DEVIATION 11.7 • n=5 Participants	35.1 letters STANDARD_DEVIATION 13.6 • n=7 Participants	32.7 letters STANDARD_DEVIATION 12.7 • n=5 Participants
Mean (SD) Spherical Equivalent Refractive Error in Amblyopic Eye	4.05 diopters STANDARD_DEVIATION 1.92 • n=5 Participants	3.28 diopters STANDARD_DEVIATION 1.93 • n=7 Participants	3.65 diopters STANDARD_DEVIATION 1.93 • n=5 Participants
Mean (SD) Spherical Equivalent Refractive Error in Fellow Eye	1.20 diopters STANDARD_DEVIATION 1.17 • n=5 Participants	1.04 diopters STANDARD_DEVIATION 0.99 • n=7 Participants	1.12 diopters STANDARD_DEVIATION 1.07 • n=5 Participants
Weight in Kilograms	47 kilograms n=5 Participants	34 kilograms n=7 Participants	42 kilograms n=5 Participants

PRIMARY outcome

Timeframe: 9 weeks after starting levodopa

Outcome measures

Outcome measures
Measure	Lower Dose 0.51 mg Levodopa/Carbidopa n=16 Participants Oral levodopa 0.51 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.	Higher Dose 0.76 mg Levodopa/Carbidopa n=17 Participants Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.
Distribution of Amblyopic Eye Visual Acuity Letter Scores at 9 Weeks After Starting Levodopa <20/100 (<47 letters)	2 participants	5 participants
Distribution of Amblyopic Eye Visual Acuity Letter Scores at 9 Weeks After Starting Levodopa 20/100 (47 to 52 letters)	2 participants	0 participants
Distribution of Amblyopic Eye Visual Acuity Letter Scores at 9 Weeks After Starting Levodopa 20/80 (53 to 57)	1 participants	1 participants
Distribution of Amblyopic Eye Visual Acuity Letter Scores at 9 Weeks After Starting Levodopa 20/63 (58 to 62)	3 participants	5 participants
Distribution of Amblyopic Eye Visual Acuity Letter Scores at 9 Weeks After Starting Levodopa 20/50 (63 to 67)	4 participants	3 participants
Distribution of Amblyopic Eye Visual Acuity Letter Scores at 9 Weeks After Starting Levodopa 20/40 (68-72 letters)	3 participants	2 participants
Distribution of Amblyopic Eye Visual Acuity Letter Scores at 9 Weeks After Starting Levodopa 20/32 (73 to 77 letters)	1 participants	1 participants

PRIMARY outcome

Timeframe: 9 weeks after starting levodopa

Outcome measures

Outcome measures
Measure	Lower Dose 0.51 mg Levodopa/Carbidopa n=16 Participants Oral levodopa 0.51 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.	Higher Dose 0.76 mg Levodopa/Carbidopa n=17 Participants Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.
Mean Amblyopic Eye Visual Acuity Letter Scores at 9 Weeks After Starting Levodopa	59.9 letters Standard Deviation 9.7	56.5 letters Standard Deviation 12.7

PRIMARY outcome

Timeframe: baseline to 9 weeks

Visual acuity was measured with the electronic early treatment diabetic retinopathy study (E-ETDRS) method and resulted in a letter score that could range from 0 to 97 letters, with 0 being the worst and 97 the best. A difference was calculated as the difference in letters between baseline and outcome with positive difference indicating improvement in acuity.

Outcome measures

Outcome measures
Measure	Lower Dose 0.51 mg Levodopa/Carbidopa n=16 Participants Oral levodopa 0.51 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.	Higher Dose 0.76 mg Levodopa/Carbidopa n=17 Participants Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.
Distribution of Change in Amblyopic Eye Visual Acuity Scores at 9 Weeks After Starting Levodopa >=15 letters worse	0 participants	0 participants
Distribution of Change in Amblyopic Eye Visual Acuity Scores at 9 Weeks After Starting Levodopa 10 to 14 letters worse	0 participants	0 participants
Distribution of Change in Amblyopic Eye Visual Acuity Scores at 9 Weeks After Starting Levodopa 5 to 9 letters worse	0 participants	1 participants
Distribution of Change in Amblyopic Eye Visual Acuity Scores at 9 Weeks After Starting Levodopa Within plus or minus 4 letters	9 participants	5 participants
Distribution of Change in Amblyopic Eye Visual Acuity Scores at 9 Weeks After Starting Levodopa 5 to 9 letters better	5 participants	6 participants
Distribution of Change in Amblyopic Eye Visual Acuity Scores at 9 Weeks After Starting Levodopa 10 to 14 letters better	2 participants	4 participants
Distribution of Change in Amblyopic Eye Visual Acuity Scores at 9 Weeks After Starting Levodopa >=15 letters better	0 participants	1 participants

PRIMARY outcome

Timeframe: baseline to 9 weeks

Visual acuity was measured with the electronic early treatment diabetic retinopathy study (E-ETDRS) method and resulted in a letter score that could range from 0 to 97 letters, with 0 being the worst and 97 the best. A difference was calculated as the difference in letters between baseline and outcome with positive difference indicating improvement in acuity.

Outcome measures

Outcome measures
Measure	Lower Dose 0.51 mg Levodopa/Carbidopa n=16 Participants Oral levodopa 0.51 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.	Higher Dose 0.76 mg Levodopa/Carbidopa n=17 Participants Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.
Mean Change in Amblyopic Eye Visual Acuity Letter Scores at 9 Weeks After Starting Levodopa	3.8 letters Standard Deviation 3.5	6.1 letters Standard Deviation 5.6

PRIMARY outcome

Timeframe: 24 weeks

Number of adverse events reported throughout entire study.

Outcome measures

Outcome measures
Measure	Lower Dose 0.51 mg Levodopa/Carbidopa n=16 Participants Oral levodopa 0.51 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.	Higher Dose 0.76 mg Levodopa/Carbidopa n=17 Participants Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.
Tolerability of Study Medication-Adverse Event Reporting Headache	6 events	6 events
Tolerability of Study Medication-Adverse Event Reporting Sinus infection	1 events	0 events
Tolerability of Study Medication-Adverse Event Reporting Cold/Upper Respiratory Infection/Cough	3 events	6 events
Tolerability of Study Medication-Adverse Event Reporting Rash	1 events	3 events
Tolerability of Study Medication-Adverse Event Reporting Flu	2 events	1 events
Tolerability of Study Medication-Adverse Event Reporting Nausea/Vomiting	2 events	1 events
Tolerability of Study Medication-Adverse Event Reporting Fatigue/Sleepiness	3 events	1 events
Tolerability of Study Medication-Adverse Event Reporting Dizziness/light-headedness	3 events	0 events
Tolerability of Study Medication-Adverse Event Reporting Conjunctivitis	0 events	2 events
Tolerability of Study Medication-Adverse Event Reporting Muscle pain	0 events	2 events
Tolerability of Study Medication-Adverse Event Reporting Stomach ache	0 events	2 events
Tolerability of Study Medication-Adverse Event Reporting Ear ache	1 events	0 events
Tolerability of Study Medication-Adverse Event Reporting Fever	1 events	0 events
Tolerability of Study Medication-Adverse Event Reporting Loss of appetite	2 events	0 events
Tolerability of Study Medication-Adverse Event Reporting Nightmare	1 events	0 events
Tolerability of Study Medication-Adverse Event Reporting Knee injury	1 events	0 events
Tolerability of Study Medication-Adverse Event Reporting Weight loss	1 events	0 events
Tolerability of Study Medication-Adverse Event Reporting Constipation	1 events	0 events
Tolerability of Study Medication-Adverse Event Reporting Finger injury	0 events	1 events
Tolerability of Study Medication-Adverse Event Reporting Pulled muscle	0 events	1 events

SECONDARY outcome

Timeframe: 4 weeks after enrollment

Visual acuity was measured with the electronic early treatment diabetic retinopathy study (E-ETDRS) method and resulted in a letter score that could range from 0 to 97 letters, with 0 being the worst and 97 the best. Letter scores are presented as Snellen Equivalents for presentation (i.e. 20/20 includes those with letter scores between 83 and 87 letters, 20/25 includes those with letter scores between 78 to 82 letters, etc.).

Outcome measures

Outcome measures
Measure	Lower Dose 0.51 mg Levodopa/Carbidopa n=16 Participants Oral levodopa 0.51 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.	Higher Dose 0.76 mg Levodopa/Carbidopa n=17 Participants Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.
Distribution of Amblyopic Eye Visual Acuity Letter Scores at 4 Weeks After Enrollment <20/100 (<47 letters)	2 participants	4 participants
Distribution of Amblyopic Eye Visual Acuity Letter Scores at 4 Weeks After Enrollment 20/100 (47 to 52 letters)	2 participants	1 participants
Distribution of Amblyopic Eye Visual Acuity Letter Scores at 4 Weeks After Enrollment 20/80 (53 to 57)	1 participants	3 participants
Distribution of Amblyopic Eye Visual Acuity Letter Scores at 4 Weeks After Enrollment 20/63 (58 to 62)	4 participants	5 participants
Distribution of Amblyopic Eye Visual Acuity Letter Scores at 4 Weeks After Enrollment 20/50 (63 to 67)	4 participants	3 participants
Distribution of Amblyopic Eye Visual Acuity Letter Scores at 4 Weeks After Enrollment 20/40 (68-72 letters)	3 participants	1 participants
Distribution of Amblyopic Eye Visual Acuity Letter Scores at 4 Weeks After Enrollment 20/32 (73 to 77 letters)	0 participants	0 participants

SECONDARY outcome

Timeframe: 4 weeks after enrollment

Outcome measures

Outcome measures
Measure	Lower Dose 0.51 mg Levodopa/Carbidopa n=16 Participants Oral levodopa 0.51 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.	Higher Dose 0.76 mg Levodopa/Carbidopa n=17 Participants Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.
Mean Amblyopic Eye Visual Acuity Letter Scores at 4 Weeks Post Enrollment	59.1 letters Standard Deviation 9.2	54.3 letters Standard Deviation 12.9

SECONDARY outcome

Timeframe: 10 weeks after stopping levodopa

Visual acuity was measured with the electronic early treatment diabetic retinopathy study (E-ETDRS) method and resulted in a letter score that could range from 0 to 97 letters, with 0 being the worst and 97 the best. Letter scores are presented as Snellen Equivalents for presentation (i.e. 20/20 includes those with letter scores between 83 and 87 letters, 20/25 includes those with letter scores between 78 to 82 letters, etc.).

Outcome measures

Outcome measures
Measure	Lower Dose 0.51 mg Levodopa/Carbidopa n=16 Participants Oral levodopa 0.51 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.	Higher Dose 0.76 mg Levodopa/Carbidopa n=17 Participants Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.
Distribution of Amblyopic Eye Visual Acuity Letter Scores at 10 Weeks After Stopping Levodopa <20/100 (<47 letters)	2 participants	5 participants
Distribution of Amblyopic Eye Visual Acuity Letter Scores at 10 Weeks After Stopping Levodopa 20/100 (47 to 52 letters)	0 participants	1 participants
Distribution of Amblyopic Eye Visual Acuity Letter Scores at 10 Weeks After Stopping Levodopa 20/80 (53 to 57)	3 participants	2 participants
Distribution of Amblyopic Eye Visual Acuity Letter Scores at 10 Weeks After Stopping Levodopa 20/63 (58 to 62)	4 participants	3 participants
Distribution of Amblyopic Eye Visual Acuity Letter Scores at 10 Weeks After Stopping Levodopa 20/50 (63 to 67)	2 participants	6 participants
Distribution of Amblyopic Eye Visual Acuity Letter Scores at 10 Weeks After Stopping Levodopa 20/40 (68-72 letters)	1 participants	0 participants
Distribution of Amblyopic Eye Visual Acuity Letter Scores at 10 Weeks After Stopping Levodopa 20/32 (73 to 77 letters)	3 participants	0 participants

SECONDARY outcome

Timeframe: 10 weeks after stopping levodopa

Outcome measures

Outcome measures
Measure	Lower Dose 0.51 mg Levodopa/Carbidopa n=16 Participants Oral levodopa 0.51 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.	Higher Dose 0.76 mg Levodopa/Carbidopa n=17 Participants Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.
Mean Amblyopic Eye Visual Acuity Letter Scores at 10 Weeks After Stopping Levodopa	60.6 letters Standard Deviation 11.1	53.9 letters Standard Deviation 11.4

SECONDARY outcome

Timeframe: enrollment to 4 weeks

Visual acuity was measured with the electronic early treatment diabetic retinopathy study (E-ETDRS) method and resulted in a letter score that could range from 0 to 97 letters, with 0 being the worst and 97 the best. A difference was calculated as the difference in letters between baseline and outcome with positive difference indicating improvement in acuity.

Outcome measures

Outcome measures
Measure	Lower Dose 0.51 mg Levodopa/Carbidopa n=16 Participants Oral levodopa 0.51 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.	Higher Dose 0.76 mg Levodopa/Carbidopa n=17 Participants Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.
Distribution of Change in Amblyopic Eye Visual Acuity Scores at 4 Weeks Post Enrollment >=15 letters worse	0 participants	0 participants
Distribution of Change in Amblyopic Eye Visual Acuity Scores at 4 Weeks Post Enrollment 10 to 14 letters worse	0 participants	0 participants
Distribution of Change in Amblyopic Eye Visual Acuity Scores at 4 Weeks Post Enrollment 5 to 9 letters worse	0 participants	0 participants
Distribution of Change in Amblyopic Eye Visual Acuity Scores at 4 Weeks Post Enrollment Within plus or minus 4 letters	11 participants	9 participants
Distribution of Change in Amblyopic Eye Visual Acuity Scores at 4 Weeks Post Enrollment 5 to 9 letters better	5 participants	7 participants
Distribution of Change in Amblyopic Eye Visual Acuity Scores at 4 Weeks Post Enrollment 10 to 14 letters better	0 participants	1 participants
Distribution of Change in Amblyopic Eye Visual Acuity Scores at 4 Weeks Post Enrollment >=15 letters better	0 participants	0 participants

SECONDARY outcome

Timeframe: enrollment to 4 weeks

Visual acuity was measured with the electronic early treatment diabetic retinopathy study (E-ETDRS) method and resulted in a letter score that could range from 0 to 97 letters, with 0 being the worst and 97 the best. A difference was calculated as the difference in letters between baseline and outcome with positive difference indicating improvement in acuity.

Outcome measures

Outcome measures
Measure	Lower Dose 0.51 mg Levodopa/Carbidopa n=16 Participants Oral levodopa 0.51 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.	Higher Dose 0.76 mg Levodopa/Carbidopa n=17 Participants Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.
Mean Change in Amblyopic Eye Visual Acuity Letter Scores at 4 Weeks Post Enrollment	2.9 letters Standard Deviation 2.8	3.8 letters Standard Deviation 3.6

SECONDARY outcome

Timeframe: baseline to 10 weeks after stopping levodopa

Visual acuity was measured with the electronic early treatment diabetic retinopathy study (E-ETDRS) method and resulted in a letter score that could range from 0 to 97 letters. A difference was calculated as the difference in letters between baseline and outcome with positive difference indicating improvement in acuity.

Outcome measures

Outcome measures
Measure	Lower Dose 0.51 mg Levodopa/Carbidopa n=16 Participants Oral levodopa 0.51 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.	Higher Dose 0.76 mg Levodopa/Carbidopa n=17 Participants Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.
Distribution of Change in Amblyopic Eye Visual Acuity Scores at 10 Weeks After Stopping Levodopa >=15 letters worse	0 participants	0 participants
Distribution of Change in Amblyopic Eye Visual Acuity Scores at 10 Weeks After Stopping Levodopa 10 to 14 letters worse	0 participants	0 participants
Distribution of Change in Amblyopic Eye Visual Acuity Scores at 10 Weeks After Stopping Levodopa 5 to 9 letters worse	0 participants	1 participants
Distribution of Change in Amblyopic Eye Visual Acuity Scores at 10 Weeks After Stopping Levodopa Within plus or minus 4 letters	7 participants	8 participants
Distribution of Change in Amblyopic Eye Visual Acuity Scores at 10 Weeks After Stopping Levodopa 5 to 9 letters better	6 participants	7 participants
Distribution of Change in Amblyopic Eye Visual Acuity Scores at 10 Weeks After Stopping Levodopa 10 to 14 letters better	2 participants	1 participants
Distribution of Change in Amblyopic Eye Visual Acuity Scores at 10 Weeks After Stopping Levodopa >=15 letters better	0 participants	0 participants

SECONDARY outcome

Timeframe: baseline to 10 weeks after stopping levodopa

Visual acuity was measured with the electronic early treatment diabetic retinopathy study (E-ETDRS) method and resulted in a letter score that could range from 0 to 97 letters, with 0 being the worst and 97 the best. A difference was calculated as the difference in letters between baseline and outcome with positive difference indicating improvement in acuity.

Outcome measures

Outcome measures
Measure	Lower Dose 0.51 mg Levodopa/Carbidopa n=16 Participants Oral levodopa 0.51 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.	Higher Dose 0.76 mg Levodopa/Carbidopa n=17 Participants Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.
Mean Change in Amblyopic Eye Visual Acuity Letter Scores at 10 Weeks After Stopping Levodopa	4.9 letters Standard Deviation 3.7	3.5 letters Standard Deviation 4.7

Adverse Events

Lower Dose 0.51 mg Levodopa/Carbidopa

Serious events: 0 serious events

Other events: 8 other events

Deaths: 0 deaths

Higher Dose 0.76 mg Levodopa/Carbidopa

Serious events: 0 serious events

Other events: 11 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Additional Information

Raymond Kraker, MSPH

Jaeb Center for Health Research

Phone: 813-875-8690

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Lower Dose 0.51 mg Levodopa/Carbidopa n=16 participants at risk Oral levodopa 0.51 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.	Higher Dose 0.76 mg Levodopa/Carbidopa n=17 participants at risk Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.
General disorders Headache	18.8% 3/16 • Number of events 6 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.	17.6% 3/17 • Number of events 6 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.
Respiratory, thoracic and mediastinal disorders Cold/Upper Respiratory Infection/Cough	18.8% 3/16 • Number of events 3 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.	17.6% 3/17 • Number of events 6 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.
Skin and subcutaneous tissue disorders Rash	6.2% 1/16 • Number of events 1 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.	17.6% 3/17 • Number of events 3 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.
General disorders Flu	12.5% 2/16 • Number of events 2 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.	5.9% 1/17 • Number of events 1 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.
Gastrointestinal disorders Nausea/Vomiting	12.5% 2/16 • Number of events 2 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.	5.9% 1/17 • Number of events 1 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.
General disorders Fatique/Sleepiness	12.5% 2/16 • Number of events 3 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.	5.9% 1/17 • Number of events 1 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.
General disorders Dizziness/Light-headedness	6.2% 1/16 • Number of events 3 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.	0.00% 0/17 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.
Eye disorders Conjunctivitis	0.00% 0/16 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.	5.9% 1/17 • Number of events 2 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.
Musculoskeletal and connective tissue disorders Muscle Pain	0.00% 0/16 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.	5.9% 1/17 • Number of events 2 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.
Gastrointestinal disorders Stomache Ache	0.00% 0/16 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.	11.8% 2/17 • Number of events 2 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.
Ear and labyrinth disorders Ear Ache	6.2% 1/16 • Number of events 1 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.	0.00% 0/17 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.
General disorders Fever	6.2% 1/16 • Number of events 1 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.	0.00% 0/17 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.
General disorders Loss of Appetite	6.2% 1/16 • Number of events 2 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.	0.00% 0/17 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.
General disorders Nightmare	6.2% 1/16 • Number of events 1 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.	0.00% 0/17 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.
Musculoskeletal and connective tissue disorders Knee Injury	6.2% 1/16 • Number of events 1 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.	0.00% 0/17 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.
Respiratory, thoracic and mediastinal disorders Sinus Infection	6.2% 1/16 • Number of events 1 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.	0.00% 0/17 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.
Metabolism and nutrition disorders Weight Loss	6.2% 1/16 • Number of events 1 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.	0.00% 0/17 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.
Gastrointestinal disorders Constipation	6.2% 1/16 • Number of events 1 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.	0.00% 0/17 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.
Musculoskeletal and connective tissue disorders Finger injury	0.00% 0/16 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.	5.9% 1/17 • Number of events 1 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.
Musculoskeletal and connective tissue disorders Pulled Muscle	0.00% 0/16 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.	5.9% 1/17 • Number of events 1 • Up to 6 months Adverse events were systematically collected at each office visit (4wks post-enrollment, 9 wks after starting levodopa, and 10 wks after stopping levodopa) and on each phone call (2wks and 7wks post-enrollment)throughout the study.