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

NCT ID: NCT01176435

Last Updated: 2018-05-02

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 45 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2010-09-30
• Study Completion Date: 2014-12-31

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.

Conditions

Albinism

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

0.76 mg/kg L-DOPA

Solution taken orally three times a day.

Group Type: ACTIVE_COMPARATOR

Levodopa

Intervention Type: DRUG

Solution taken orally three times a day.

0.51 mg/kg L-DOPA

Solution taken orally three times a day.

Group Type: ACTIVE_COMPARATOR

Levodopa

Intervention Type: DRUG

Solution taken orally three times a day.

Placebo

Solution taken orally three times a day.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Solution taken orally three times a day.

Interventions

Levodopa

Solution taken orally three times a day.

Intervention Type: DRUG

Levodopa

Solution taken orally three times a day.

Intervention Type: DRUG

Placebo

Solution taken orally three times a day.

Intervention Type: DRUG

Eligibility Criteria

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

• Minimum Eligible Age: 3 Years
• Maximum Eligible Age: 60 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University of Minnesota

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Gail Summers, M.D.

Role: PRINCIPAL_INVESTIGATOR

University of Minnesota

Locations

University of Minnesota Eye Clinic

Minneapolis, Minnesota, United States

Countries

United States

Other Identifiers

0912M75653

Identifier Type: -

Identifier Source: org_study_id