NCT01176435

Trial of L-DOPA as a Treatment to Improve Vision in Albinism

Clinical Trial to Evaluate Levodopa as Treatment to Improve Vision in Individuals With Albinism

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 45 (actual)
• Sponsor: University of Minnesota · Academic / Other
• Sex: All
• Age: 3 Years – 60 Years
• Healthy volunteers: Not accepted

Summary

This project will evaluate the effect of two doses of levodopa (L-DOPA) in a randomized, placebo-controlled, double-masked clinical trial to see if vision can be improved in individuals with albinism. The hypothesis is that providing L-DOPA to the retinas of these individuals may increase melanin pigment production. Increased melanin has previously been shown to be associated with improved vision.

Detailed description

A group of 45 individuals with the clinical findings of oculocutaneous albinism (OCA) will be randomly assigned to one of 3 treatment groups: treatment with 0.76 mg/kg/d with 25% carbidopa, 0.51 mg/kg/d levodopa with 25% carbidopa \[divided into 3 doses/d), or placebo. Subjects will be between ages 3 and 60 years. Blood will be drawn to determine the mutation(s) in the genes that causes OCA. Primary outcome will be binocular best-corrected visual acuity measured with the EVA. Enrollment and 20 week examination will be complete eye exam with fundus photos. At weeks 5, 10, and 15, exams will include just vital signs and BCVA. At all visits, a review of potential side effects will be conducted. Between visits, subjects will be contacted to determine if any side effects have occurred. The study will remain double masked until the last study examination on the last subject has been performed. At that time, the data will be statistically analyzed and subjects will be informed re: treatment assignment, mutations found, and the study results.

Conditions

• Albinism

Interventions

Type	Name	Description
DRUG	Levodopa	Solution taken orally three times a day.
DRUG	Levodopa	Solution taken orally three times a day.
DRUG	Placebo	Solution taken orally three times a day.

Timeline

• Start date: 2010-09-01
• Primary completion: 2014-12-01
• Completion: 2014-12-01
• First posted: 2010-08-06
• Last updated: 2018-05-02
• Results posted: 2018-05-02

Locations

1 site across 1 country: United States