NCT01663935

Vision Response to Dopamine Replacement

Interventional Study of Levodopa Replacement on Retinal Function in Oculocutaneous Albinism

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 20 (actual)
• Sponsor: University of Wisconsin, Madison · Academic / Other
• Sex: All
• Age: 3 Years
• Healthy volunteers: Not accepted

Summary

The purpose of the study is to evaluate and document physiologic and functional changes in visual performance and retinal function of patients diagnosed with albinism (a dopamine deficiency state) following a trial of oral Levodopa/carbidopa treatment.

Detailed description

In this study the investigators propose that the retina itself in albinism is deficient in dopamine, and vision improvement will occur as a result of improved retinal function in response to the deficient neurotransmitter dopamine. This study has a pretest-posttest design in order to determine if improvement in vision is in response to replacement of deficiency (dopamine). The electroretinography (ERG) testing and optical coherence tomography (OCT) will be critical determinants to confirm vision improvement as a result of improved retinal function, but are not primary outcome data. Main outcome measures will be collected at pre-treatment, 1 month, 3 months, and 4 months. Change in visual acuity as measured in logMAR by Snellen or sweep visual evoked potential (SVEP) after 3 months of treatment is the primary outcome. Patients include OCA1a patients, OCA1b, OCA2, and unclassified Oculocutaneous albinism (OCA). OCA1a patients clinically are known to have the worst vision, and physiologically have the lowest (or absent) levels of tyrosinase function (Dopamine Production). All patients will be treated with Levodopa/carbidopa 4mg/kg/day in three divided doses.

Conditions

• Albinism
• Oculocutaneous Albinism

Interventions

Type	Name	Description
DRUG	Levodopa/carbidopa	This study will have an intent to treat goal. Anyone that fits the inclusion criteria for the study will be entered and receive study drug.

Timeline

• Start date: 2012-10-17
• Primary completion: 2018-04-12
• Completion: 2018-04-12
• First posted: 2012-08-14
• Last updated: 2019-06-11
• Results posted: 2019-06-11

Locations

1 site across 1 country: United States