Pilot Study to Evaluate Levodopa as Treatment for Residual Amblyopia

NCT00789672 | Completed Phase 2 Results DMC

• 2 other identifiers: NEI-1412U10EY011751
• Study Type: interventional
• Target: 33
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Amblyopia

Keywords

Amblyopia; Patching; Levodopa

Outcome Measures

Primary Outcomes (5)

• Distribution of Amblyopic Eye Visual Acuity Letter Scores at 9 Weeks After Starting 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 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.).

  9 weeks after starting levodopa

• Mean Amblyopic Eye Visual Acuity Letter Scores at 9 Weeks After Starting 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 being the best.

  9 weeks after starting levodopa

• Distribution of Change in Amblyopic Eye Visual Acuity Scores at 9 Weeks After Starting 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.

  baseline to 9 weeks

• Mean Change in Amblyopic Eye Visual Acuity Letter Scores at 9 Weeks After Starting 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.

  baseline to 9 weeks

• Tolerability of Study Medication-Adverse Event Reporting

  Number of adverse events reported throughout entire study.

  24 weeks

Secondary Outcomes (8)

• Distribution of Amblyopic Eye Visual Acuity Letter Scores at 4 Weeks After Enrollment

  4 weeks after enrollment

• Mean Amblyopic Eye Visual Acuity Letter Scores at 4 Weeks Post Enrollment

  4 weeks after enrollment

• Distribution of Amblyopic Eye Visual Acuity Letter Scores at 10 Weeks After Stopping Levodopa

  10 weeks after stopping levodopa

• Mean Amblyopic Eye Visual Acuity Letter Scores at 10 Weeks After Stopping Levodopa

  10 weeks after stopping levodopa

• Distribution of Change in Amblyopic Eye Visual Acuity Scores at 4 Weeks Post Enrollment

  enrollment to 4 weeks

Study Arms (2)

Lower Dose (3-1) levodopa/carbidopa

ACTIVE COMPARATOR

Oral levodopa 0.51 mg/kg tid with carbidopa 0.17 mg/kg tid (3 to 1 formulation) combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam 9 weeks after starting medication.

Device: patching; Drug: levodopa/carbidopa

Higher Dose (4.5-1) levodopa/carbidopa

ACTIVE COMPARATOR

Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid (approximately 4.5 to 1 formulation) combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam 9 weeks after starting medication.

Drug: levodopa/carbidopa; Device: patching

Interventions

levodopa/carbidopa DRUG

Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid (approximately 4.5 to 1 formulation)

Higher Dose (4.5-1) levodopa/carbidopa

patching DEVICE

2 hours daily patching

Also known as: Coverlet, 3M Opticlude, Ortopad®

Higher Dose (4.5-1) levodopa/carbidopa; Lower Dose (3-1) levodopa/carbidopa

Eligibility Criteria

• Age: 8 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

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
• 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

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)

• Repka MX, Kraker RT, Beck RW, Atkinson CS, Bacal DA, Bremer DL, Davis PL, Gearinger MD, Glaser SR, Hoover DL, Laby DM, Morrison DG, Rogers DL, Sala NA, Suh DW, Wheeler MB; Pediatric Eye Disease Investigator Group. Pilot study of levodopa dose as treatment for residual amblyopia in children aged 8 years to younger than 18 years. Arch Ophthalmol. 2010 Sep;128(9):1215-7. doi: 10.1001/archophthalmol.2010.178. No abstract available.

  PMID: 20837811 RESULT

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

Limitations and Caveats

Without a patching-only control group, no conclusions can be made regarding efficacy, safety, or side effects with this treatment. Placebo controlled trial is needed to see if levodopa can augment occlusion therapy in the treatment of amblyopia.

Results Point of Contact

• Title: Raymond Kraker, MSPH
• Organization: Jaeb Center for Health Research

pedig@jaeb.org

813-875-8690

Study Officials

• Michael X. Repka, M.D.

  Wilmer Eye Institute

  STUDY CHAIR

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director, PEDIG Coordinating Center

Study Record Dates

• First Submitted: October 27, 2008
• First Posted: November 13, 2008
• Study Start: January 1, 2009
• Primary Completion: September 1, 2009
• Study Completion: December 1, 2009
• Last Updated: July 13, 2016
• Results First Posted: March 23, 2011

Record last verified: 2016-07

Locations

US (1)