Phase 3
Completed N=139
Levodopa for the Treatment of Residual Amblyopia
Source: ClinicalTrials.gov NCT01190813 ↗Enrolled (actual)
139
Serious AEs
0.0%
Results posted
Feb 2015
Primary outcomePrimary: Distribution of Amblyopic Eye Visual Acuity Change From Baseline — 0; 0; 2; 1 participants
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.
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 | — |
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. Informational only — not medical advice.