Vision Response to Dopamine Replacement

Phase 2

Terminated

• Conditions: Albinism; Oculocutaneous Albinism
• Interventions: Drug: Levodopa/carbidopa

• Registration Number: NCT01663935
• Lead Sponsor: University of Wisconsin, Madison

Brief 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.

Study Timeline

• Study First Submitted: 2012-08-09
• Study First Submitted QC: 2012-08-13
• Study First Posted: 2012-08-14
• Study Start: 2012-10-17
• Primary Completion: 2018-04-12
• Study Completion: 2018-04-12
• Results First Submitted: 2019-04-09
• Status Verified: 2019-05
• Results First Submitted QC: 2019-05-17
• Last Update Submitted: 2019-05-17
• Results First Posted: 2019-06-11
• Last Update Posted: 2019-06-11

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

• clinical diagnosis of oculocutaneous albinism
• age over 3 and weight over 25 lbs.

Exclusion Criteria

• ocular only albinism
• ocular pathology other than albinism
• neurologic disease, history of myocardial infarction, history of clinical depression, pregnancy

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Levodopa/carbidopa 4mg/kg/day	Levodopa/carbidopa	Treatment drug taken orally three times daily

Primary Outcome Measures

Name	Time	Method
Visual Acuity Change	3 months	Change in visual acuity from baseline to 3 months as measured in logMAR by Snellen or Sweep visual evoked potential (SVEP). logMar lower values equals better visual outcome.

Trial Locations

Locations (1)

University of Wisconsin; 🇺🇸 Madison, Wisconsin, United States