Levodopa for the Treatment of Residual Amblyopia

NCT01190813 | Completed Phase 3 Results DMC

• 2 other identifiers: ATS 172U10EY011751
• Study Type: interventional
• Target: 139
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Amblyopia

Keywords

amblyopia, levodopa

Outcome Measures

Primary Outcomes (2)

• 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

Secondary Outcomes (41)

• Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 18 Weeks

  18 weeks after enrollment

• Distribution of Amblyopic Eye Visual Acuity at 18 Weeks

  18 weeks after enrollment

• Mean Amblyopic Eye Visual Acuity at 18 Weeks

  18 weeks after enrollment

• Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 4 Weeks

  4 weeks after enrollment

• Amblyopic Eye Visual Acuity Improvement Treatment Group Comparison at 10 Weeks

  10 weeks after enrollment

Study Arms (2)

Levodopa/Carbidopa

ACTIVE COMPARATOR

Levodopa 0.76 mg/kg with Carbidopa 0.17 mg/kg tid

Drug: Levodopa/Carbidopa; Other: Patching

Placebo

PLACEBO COMPARATOR

Oral placebo tid

Drug: Placebo; Other: Patching

Interventions

Levodopa/Carbidopa DRUG

Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid

Levodopa/Carbidopa

Placebo DRUG

Oral placebo tid

Placebo

Patching OTHER

Two hours of daily patching

Levodopa/Carbidopa; Placebo

Eligibility Criteria

• Age: 7 Years - 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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)
• 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.

You may not qualify if:

• 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 attention deficit hyperactivity disorder
• Known liver disease
• History of melanoma
• Known psychological problems

Sponsors & Collaborators

• Jaeb Center for Health Research: lead
• National Eye Institute (NEI): collaborator

Study Sites (1)

Wilmer Eye Institute

Baltimore, Maryland, 21287, United States

Location

Related Publications (1)

• 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.

  PMID: 25676904 RESULT

Related Links

• PubMed abstract
• PubMed Central HHS Public Access - Full Text

MeSH Terms

Conditions

Amblyopia

Interventions

carbidopa, levodopa drug combination

Condition Hierarchy (Ancestors)

Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Vision Disorders; Sensation Disorders; Neurologic Manifestations; Eye Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Raymond Kraker, M.S.P.H., Director of PEDIG Coordinating Center
• Organization: Jaeb Center for Health Research

rkraker@jaeb.org

813-975-8690

Study Officials

• Michael X Repka, MD

  Jaeb Center for Health Research

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 17, 2010
• First Posted: August 30, 2010
• Study Start: September 1, 2010
• Primary Completion: January 1, 2014
• Study Completion: April 1, 2014
• Last Updated: June 2, 2016
• Results First Posted: February 13, 2015

Record last verified: 2016-05

Data Sharing

• IPD Sharing: Will share

Locations

US (1)