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

University of Minnesota (UMN)

Status and phase: Completed; Phase 2

Conditions: Albinism

Treatments: Drug: Placebo; Drug: Levodopa

Study type: Interventional

Funder types: Other

Identifiers

NCT01176435

0912M75653

Details and patient eligibility

About

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.

Full 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.

Enrollment: 45 patients

Sex: All

Ages: 3 to 60 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

---

45 participants in 3 patient groups, including a placebo group

0.76 mg/kg L-DOPA

Active Comparator group

Description:

Solution taken orally three times a day.

Treatment:

Drug: Levodopa

Drug: Levodopa

0.51 mg/kg L-DOPA

Active Comparator group

Description:

Solution taken orally three times a day.

Treatment:

Drug: Levodopa

Drug: Levodopa

Placebo

Placebo Comparator group

Description:

Solution taken orally three times a day.

Treatment:

Drug: Placebo