Phase 2 N=33 Randomized Triple-blind Treatment

Pilot Study to Evaluate Levodopa as Treatment for Residual Amblyopia

Amblyopia

Bottom Line

View on ClinicalTrials.gov: NCT00789672 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

Mar 2011

Primary outcome: Primary: Distribution of Amblyopic Eye Visual Acuity Letter Scores at 9 Weeks After Starting Levodopa — 2; 5; 2; 0 participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: levodopa/carbidopa (Drug); patching (Device)
Age: Pediatric · 8+ yrs
Sex: All
Sponsor: Jaeb Center for Health Research
Primary completion: Sep 2009

Outcome Measures

Outcome	Result	p-value
PRIMARY Distribution of Amblyopic Eye Visual Acuity Letter Scores at 9 Weeks After Starting Levodopa	2; 5; 2; 0; 1; 1	—
PRIMARY Mean Amblyopic Eye Visual Acuity Letter Scores at 9 Weeks After Starting Levodopa	59.9; 56.5	—
PRIMARY Distribution of Change in Amblyopic Eye Visual Acuity Scores at 9 Weeks After Starting Levodopa	0; 0; 0; 0; 0; 1	—
PRIMARY Mean Change in Amblyopic Eye Visual Acuity Letter Scores at 9 Weeks After Starting Levodopa	3.8; 6.1	—
PRIMARY Tolerability of Study Medication-Adverse Event Reporting	6; 6; 3; 6; 1; 3	—
SECONDARY Distribution of Amblyopic Eye Visual Acuity Letter Scores at 4 Weeks After Enrollment	2; 4; 2; 1; 1; 3	—
SECONDARY Mean Amblyopic Eye Visual Acuity Letter Scores at 4 Weeks Post Enrollment	59.1; 54.3	—
SECONDARY Distribution of Amblyopic Eye Visual Acuity Letter Scores at 10 Weeks After Stopping Levodopa	2; 5; 0; 1; 3; 2	—
SECONDARY Mean Amblyopic Eye Visual Acuity Letter Scores at 10 Weeks After Stopping Levodopa	60.6; 53.9	—
SECONDARY Distribution of Change in Amblyopic Eye Visual Acuity Scores at 4 Weeks Post Enrollment	0; 0; 0; 0; 0; 0	—
SECONDARY Mean Change in Amblyopic Eye Visual Acuity Letter Scores at 4 Weeks Post Enrollment	2.9; 3.8	—
SECONDARY Distribution of Change in Amblyopic Eye Visual Acuity Scores at 10 Weeks After Stopping Levodopa	0; 0; 0; 0; 0; 1	—
SECONDARY Mean Change in Amblyopic Eye Visual Acuity Letter Scores at 10 Weeks After Stopping Levodopa	4.9; 3.5	—

Summary

This pilot study is being conducted as a prelude to a randomized trial to compare levodopa/carbidopa plus patching versus patching alone. The purpose of the pilot study is to demonstrate recruitment potential, to provide prospective data on the tolerability of levodopa as a treatment for amblyopia, to provide limited data on its safety, to provide limited data on it's efficiency, and to provide data to assist in selecting a dose to use in a subsequent phase 3 randomized trial. In addition, this study will provide the opportunity for investigators to gain experience in using levodopa prior to a randomized trial.

Eligibility Criteria

Inclusion Criteria

Age 8 to < 18 years old
Amblyopia associated with strabismus, anisometropia, or both
Visual acuity in the amblyopic eye 18 to 67 letters inclusive (20/50 to 20/400)
Visual acuity in the sound eye ≥ 78 letters (20/25 or better)
Current amblyopia treatment of at least 2 hours patching per day
No improvement in best-corrected amblyopic eye visual acuity between two consecutive visits at least 4 weeks apart using the same testing method and optimal spectacle correction (if needed), with no improvement of more than 4 letters or one logMAR line.

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
Strabismus surgery planned within 16 weeks
Known allergy to levodopa-carbidopa
History of dystonic reactions
Current requirement to take oral iron supplements including multivitamins containing iron during the 8 weeks of treatment with levodopa-carbidopa
Current use of antihypertensive, anti-depressant medications, phenothiazines, butyrophenones, risperidone and isoniazid, non-specific monoamine oxidase inhibitors
Current use of medication for the treatment of attention deficit hyperactivity disorder
Known gastrointestinal or liver disease
History of melanoma
Known psychological problems
Known skin reactions to patch or bandage adhesives
Prior levodopa treatment
Current treatment with topical atropine
Females who are pregnant, lactating, or intending to become pregnant within the next 16 weeks.
A negative urine pregnancy test will be required for all females who have experienced menarche.
Requirements regarding the establishment of pregnancy and monitoring of pregnancy over the course of the study as defined by each individual Institutional Review Board may supersede these criteria.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00789672). 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.