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

Phase 2

Completed

• Conditions: Albinism
• Interventions: Drug: Placebo; Drug: Levodopa

• Registration Number: NCT01176435
• Lead Sponsor: University of Minnesota

Brief 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.

Study Timeline

• Study First Submitted: 2010-08-04
• Study First Submitted QC: 2010-08-04
• Study First Posted: 2010-08-06
• Study Start: 2010-09
• Primary Completion: 2014-12
• Study Completion: 2014-12
• Results First Submitted: 2016-06-22
• Status Verified: 2018-04
• Results First Submitted QC: 2018-04-30
• Last Update Submitted: 2018-04-30
• Results First Posted: 2018-05-02
• Last Update Posted: 2018-05-02

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 45

Inclusion Criteria

• Age 3 to 60 years with albinism

Exclusion Criteria

• Glaucoma or at increased risk of glaucoma
• History of dystonia
• History of melanoma
• Planning to undergo eye muscle surgery during study time frame
• Undergoing vision therapy
• Taking iron supplements or vitamins with iron
• Taking medication for ADHD
• Known liver or gastrointestinal disease
• Previous treatment with levodopa
• Psychological problems
• Ocular abnormalities other than those associated with albinism
• Pregnant, nursing or planning to become pregnant during study
• Known allergy to levodopa/carbidopa

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	Solution taken orally three times a day.
0.51 mg/kg L-DOPA	Levodopa	Solution taken orally three times a day.
0.76 mg/kg L-DOPA	Levodopa	Solution taken orally three times a day.

Primary Outcome Measures

Name	Time	Method
Improved Vision	20 weeks	Binocular best-corrected visual acuity-The visual acuity test is used to determine the smallest letters you can read on a standardized chart (Snellen chart) or a card held 20 feet (6 meters) away. Special charts are used when testing at distances shorter than 20 feet (6 meters). Ranges are 20/10 vision to 20/200 vision. 20/10 being the best and 20/200 being the worse.

Trial Locations

Locations (1)

University of Minnesota Eye Clinic; 🇺🇸 Minneapolis, Minnesota, United States