Does levodopa improve vision in albinism? Results of a randomized, controlled clinical trial

C. Gail Summers, John E. Connett, Ann M. Holleschau, Jennifer L. Anderson, Inge De Becker, Brian S. Mckay, Murray H. Brilliant

Research output: Contribution to journalArticlepeer-review

18 Scopus citations


Background: Dopamine is an intermediate product in the biosynthesis of melanin pigment, which is absent or reduced in albinism. Animal research has shown that supplying a precursor to dopamine, levodopa, may improve visual acuity in albinism by enhancing neural networks. This study examines the safety and effectiveness of levodopa on best-corrected visual acuity in human subjects with albinism. Design: Prospective, randomized, placebo-controlled, double-masked clinical trial conducted at the University of Minnesota. Participants: Forty-five subjects with albinism. Methods: Subjects with albinism were randomly assigned to one of three treatment arms: levodopa 0.76mg/kg with 25% carbidopa, levodopa 0.51mg/kg with 25% carbidopa, or placebo and followed for 20 weeks, with best-corrected visual acuity measured at enrollment, and at weeks 5, 10, 15, and 20 after enrollment. Side-effects were recorded with a symptom survey. Blood was drawn for genotyping. Main Outcome Measures: Side-effects and best-corrected visual acuity 20 weeks after enrolment. Results: All subjects had at least one mutation found in a gene known to cause albinism. Mean age was 14.5 years (range: 3.5 to 57.8 years). Follow up was 100% and compliance was good. Minor side-effects were reported; there were no serious adverse events. There was no statistically significant improvement in best-corrected visual acuity after 20 weeks with either dose of levodopa. Conclusions: Levodopa, in the doses used in this trial and for the time course of administration, did not improve visual acuity in subjects with albinism.

Original languageEnglish (US)
Pages (from-to)713-721
Number of pages9
JournalClinical and Experimental Ophthalmology
Issue number8
StatePublished - Nov 1 2014


  • Albinism
  • Levodopa
  • Randomized controlled trial
  • Visual acuity

ASJC Scopus subject areas

  • Ophthalmology


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