Phase 3
N=139
Levodopa for the Treatment of Residual Amblyopia
Amblyopia
Bottom Line
View on ClinicalTrials.gov: NCT01190813 ↗Enrolled (actual)
139
Serious AEs
0.0%
Results posted
Feb 2015
Primary outcome: Primary: Distribution of Amblyopic Eye Visual Acuity Change From Baseline — 0; 0; 2; 1 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Levodopa/Carbidopa (Drug); Placebo (Drug); Patching (Other)
- Age
- Pediatric · 7+ yrs
- Sex
- All
- Sponsor
- Jaeb Center for Health Research
- Primary completion
- Jan 2014
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Distribution of Amblyopic Eye Visual Acuity Change From Baseline |
0; 0; 2; 1; 35; 23 | — |
| PRIMARY Mean Amblyopic Eye Visual Acuity Change From Baseline |
5.2; 3.8 | 0.06 |
| SECONDARY Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 18 Weeks |
13; 2 | 0.06 |
| SECONDARY Distribution of Amblyopic Eye Visual Acuity at 18 Weeks |
0; 0; 5; 1; 7; 6 | — |
| SECONDARY Mean Amblyopic Eye Visual Acuity at 18 Weeks |
58.7; 54.8 | — |
| SECONDARY Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 4 Weeks |
4; 2; 84; 45 | — |
| SECONDARY Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 10 Weeks |
10; 9; 78; 39 | — |
| SECONDARY Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 16 Weeks |
15; 10; 72; 36 | — |
| SECONDARY Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 26 Weeks |
17; 5; 69; 40 | — |
| SECONDARY Amblyopia Resolutionat 4 Weeks |
0; 0; 88; 47 | — |
| SECONDARY Amblyopia Resolution at 10 Weeks |
0; 0; 88; 48 | — |
| SECONDARY Amblyopia Resolution at 16 Weeks |
1; 0; 86; 46 | — |
| SECONDARY Amblyopia Resolution at 18 Weeks |
0; 0; 86; 45 | — |
| SECONDARY Amblyopia Resolution at 26 Weeks |
0; 0; 86; 45 | — |
| SECONDARY Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 4 Weeks |
0; 0; 6; 2; 55; 28 | — |
| SECONDARY Mean Change in Amblyopic Eye Visual Acuity From Baseline at 4 Weeks |
2.2; 2.5 | 0.65 |
| SECONDARY Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 10 Weeks |
1; 0; 4; 1; 43; 30 | — |
| SECONDARY Mean Change in Amblyopic Eye Visual Acuity From Baseline at 10 Weeks |
3.8; 3.7 | 0.44 |
| SECONDARY Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 16 Weeks |
0; 0; 1; 0; 41; 26 | — |
| SECONDARY Mean Change in Amblyopic Eye Visual Acuity From Baseline at 16 Weeks |
5.1; 4.2 | 0.20 |
| SECONDARY Distribution of Amblyopic Eye Visual Acuity Change From Baseline at 26 Weeks |
3; 1; 36; 23; 30; 16 | — |
| SECONDARY Mean Change in Amblyopic Eye Visual Acuity From Baseline at 26 Weeks |
5.0; 4.2 | 0.17 |
| SECONDARY Distribution of Fellow Eye Visual Acuity at 18 Weeks |
12; 4; 44; 22; 22; 16 | — |
| SECONDARY Mean Fellow Eye Visual Acuity at 18 Weeks |
88.1; 88.0 | — |
| SECONDARY Distribution of Fellow Eye Visual Acuity Change From Baseline at 18 Weeks |
0; 0; 0; 0; 4; 3 | — |
| SECONDARY Mean Fellow Eye Visual Acuity Change From Baseline at 18 Weeks |
1.5; 0.5 | — |
| SECONDARY Mean Child Symptom Survey Score at Enrollment |
1.60; 1.73 | — |
| SECONDARY Mean Child Symptom Survey Score at 4 Weeks |
1.42; 1.51 | — |
| SECONDARY Mean Child Symptom Survey Score at 10 Weeks |
1.33; 1.42 | — |
| SECONDARY Mean Child Symptom Survey Score at 16 Weeks |
1.25; 1.38 | — |
| SECONDARY Mean Child Symptom Survey Score at 18 Weeks |
1.17; 1.28 | — |
| SECONDARY Mean Child Symptom Survey Score at 26 Weeks |
1.23; 1.33 | — |
| SECONDARY Mean Parent Symptom Survey Score at Enrollment |
1.42; 1.44 | — |
| SECONDARY Mean Parent Symptom Survey Score at 4 Weeks |
1.29; 1.26 | — |
| SECONDARY Mean Parent Symptom Survey Score at 10 Weeks |
1.22; 1.30 | — |
| SECONDARY Mean Parent Symptom Survey Score at 16 Weeks |
1.24; 1.25 | — |
| SECONDARY Mean Parent Symptom Survey Score at 18 Weeks |
1.17; 1.17 | — |
| SECONDARY Mean Parent Symptom Survey Score at 26 Weeks |
1.19; 1.22 | — |
| SECONDARY Mean Systemic Adverse Events |
1.28; 1.55 | — |
| SECONDARY Distribution of Fellow Eye Visual Acuity at 26 Weeks |
15; 7; 34; 22; 28; 13 | — |
| SECONDARY Mean Fellow Eye Visual Acuity at 26 Weeks |
88.0; 88.2 | — |
| SECONDARY Distribution of Fellow Eye Visual Acuity Change From Baseline at 26 Weeks |
1; 0; 0; 0; 2; 4 | — |
| SECONDARY Mean Fellow Eye Visual Acuity Change From Baseline at 26 Weeks |
1.4; 0.8 | — |
Summary
The purpose of this study is to evaluate the efficacy and safety of oral levodopa and patching versus oral placebo and patching as treatment for residual amblyopia in children 7 to <13 years old with visual acuity of 20/50 to 20/400 in the amblyopic eye.
Eligibility Criteria
Inclusion Criteria
- Age 7 to 12
- Amblyopia associated with strabismus, anisometropia, or both
- Criteria for strabismus: One of the following criteria must be met: Heterotropia at distance and/or near fixation on examination (with or without spectacles); History of strabismus surgery; Documented history of strabismus which is no longer present (which in the judgment of the investigator could have caused amblyopia)
- Criteria for anisometropia: One of the following criteria must be met: ≥0.50 D difference between eyes in spherical equivalent; ≥1.50 D difference between eyes in astigmatism in any meridian
- Visual acuity, measured in each eye (amblyopic eye without cycloplegia) within 7 days prior to enrollment using the E-ETDRS protocol by a study certified visual acuity tester as follows:
- Visual acuity in the amblyopic eye 18 to 67 letters inclusive (20/50 to 20/400)
- Visual acuity in the fellow eye ≥78 letters (20/25 or better)
- Current amblyopia treatment (other than spectacles)
- 12 weeks of at least 2 hours of occlusion per day prescribed for the fellow eye during the immediate pre-enrollment period.
- While on current treatment, visual acuity has not improved one line (5 letters) or more since a non-study visit at least 6 weeks ago. Both acuity measurements to define no improvement must have been done using the same testing method.
- Treatment with atropine at any time during this pre-enrollment period is not allowed.
- Any treatment prior to the current patching episode with stable acuity is acceptable.
- 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:
- Spherical equivalent must be within 0.50 D of fully correcting the anisometropia.
- Hypermetropia of 3.00D or more must be corrected.
- Hypermetropia must not be under corrected by more than 1.50 D spherical equivalent, and reduction in plus sphere must be symmetric in the two eyes.
- Cylinder power in both eyes must be within 0.50 D of fully correcting the astigmatism.
- Cylinder axis in both eyes is within 6 degrees of the axis in the spectacles when cylinder power is ≥1.00 D.
- Myopia of amblyopic eye greater than 0.50 D by spherical equivalent must be corrected, and the glasses must not under correct the myopia by more than 0.25 D or overcorrect it by more than 0.50 D.
- 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
- nystagmus per se does not exclude the subject if the above visual acuity criteria are met
- 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 atte
Data sourced from ClinicalTrials.gov (NCT01190813). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.