Vision Response to Dopamine Replacement

Study Description

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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.

Condition or disease	Intervention/treatment	Phase
Albinism; Oculocutaneous Albinism	Drug: Levodopa/carbidopa	Phase 2

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 ERG testing and 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 SVEP after 3 months of treatment is the primary outcome. Patients include OCA1a patients, OCA1b, OCA2, and unclassified 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 Design

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Study Type :	Interventional (Clinical Trial)
Estimated Enrollment :	50 participants
Intervention Model:	Single Group Assignment
Masking:	None (Open Label)
Primary Purpose:	Treatment
Official Title:	Interventional Study of Levodopa Replacement on Retinal Function in Oculocutaneous Albinism
Study Start Date :	August 2012
Estimated Primary Completion Date :	September 2017
Estimated Study Completion Date :	October 2017

Arms and Interventions

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Arm	Intervention/treatment
Levodopa/carbidopa 4mg/kg/day; Treatment drug taken orally three times daily	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.; Other Names: Sinemet; Atamet; Parcopa

Outcome Measures

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Primary Outcome Measures :

1. Visual Acuity Improvement [ Time Frame: 3 months ]
   Change in visual acuity as measured in logMAR by Snellen or Sweep visual evoked potential (SVEP)

Secondary Outcome Measures :

1. Retinal Function [ Time Frame: 3 months ]
   Electroretinography -- electro physiologic testing of retinal function

Other Outcome Measures:

1. Color and Contrast sensitivity [ Time Frame: 3 months ]
   Ancillary testing of visual/retinal function

Eligibility Criteria

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Ages Eligible for Study:	3 Years and older (Child, Adult, Senior)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No

Criteria

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

Contacts and Locations

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Contacts

Contact: Michael C Struck, MD	608-263-9859	mcstruck@wisc.edu
Contact: Angie Wealti	608-265-7557	Angie Wealti <wealti@ophth.wisc.edu>

Locations

United States, Wisconsin
University of Wisconsin	Recruiting
Madison, Wisconsin, United States, 53705
Contact: Angie Wealti 608-265-7557 Angie Wealti <wealti@ophth.wisc.edu>
Principal Investigator: Michael C Struck, MD

Sponsors and Collaborators

University of Wisconsin, Madison

Investigators

Principal Investigator:	Michael C Struck, MD	University of Wisconsin, Madison

More Information

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Responsible Party:	University of Wisconsin, Madison
ClinicalTrials.gov Identifier:	NCT01663935 History of Changes
Other Study ID Numbers:	2016-1576; 2012-0023 ( Other Identifier: Study team )
First Posted:	August 14, 2012
Last Update Posted:	May 4, 2017
Last Verified:	May 2017

Keywords provided by University of Wisconsin, Madison:

levodopa; Albinism; dopamine	oculocutaneous albinism; vision; retina function

Additional relevant MeSH terms:

Albinism; Albinism, Oculocutaneous; Eye Diseases, Hereditary; Eye Diseases; Genetic Diseases, Inborn; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Skin Diseases, Genetic; Hypopigmentation; Pigmentation Disorders; Skin Diseases; Metabolic Diseases; Levodopa; Carbidopa	Dopamine; Antiparkinson Agents; Anti-Dyskinesia Agents; Dopamine Agents; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Physiological Effects of Drugs; Cardiotonic Agents; Sympathomimetics; Autonomic Agents; Peripheral Nervous System Agents; Protective Agents; Aromatic Amino Acid Decarboxylase Inhibitors; Enzyme Inhibitors