Vision Response to Dopamine Replacement

This study is currently recruiting participants. (see Contacts and Locations)

Verified October 2015 by University of Wisconsin, Madison

Sponsor:

Information provided by (Responsible Party):

University of Wisconsin, Madison

ClinicalTrials.gov Identifier:

NCT01663935

First received: August 9, 2012

Last updated: October 9, 2015

Last verified: October 2015

History of Changes

Purpose

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	Intervention	Phase
Albinism Oculocutaneous Albinism	Drug: Levodopa/carbidopa	Phase 2


Study Type:	Interventional
Study Design:	Endpoint Classification: Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	Interventional Study of Levodopa Replacement on Retinal Function in Oculocutaneous Albinism

Resource links provided by NLM:

Genetics Home Reference related topics: Lenz microphthalmia syndrome N-acetylglutamate synthase deficiency argininosuccinic aciduria citrullinemia oculocutaneous albinism oculofaciocardiodental syndrome ornithine translocase deficiency

Drug Information available for: Dopamine Levodopa Dopamine hydrochloride Carbidopa

Genetic and Rare Diseases Information Center resources: Oculocutaneous Albinism Albinism Inborn Amino Acid Metabolism Disorder Hypomelanotic Disorder

U.S. FDA Resources

Further study details as provided by University of Wisconsin, Madison:

Primary Outcome Measures:

Visual Acuity Improvement [ Time Frame: 3 months ] [ Designated as safety issue: No ]
Change in visual acuity as measured in logMAR by Snellen or Sweep visual evoked potential (SVEP)

Secondary Outcome Measures:

Retinal Function [ Time Frame: 3 months ] [ Designated as safety issue: No ]
Electroretinography -- electro physiologic testing of retinal function

Other Outcome Measures:

Color and Contrast sensitivity [ Time Frame: 3 months ] [ Designated as safety issue: No ]
Ancillary testing of visual/retinal function


Estimated Enrollment:	50
Study Start Date:	August 2012
Estimated Study Completion Date:	October 2017
Estimated Primary Completion Date:	September 2017 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
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

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.

Eligibility


Ages Eligible for Study:	3 Years and older (Child, Adult, Senior)
Genders Eligible for Study:	Both
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

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01663935

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


Responsible Party:	University of Wisconsin, Madison
ClinicalTrials.gov Identifier:	NCT01663935 History of Changes
Other Study ID Numbers:	2012-0023
Study First Received:	August 9, 2012
Last Updated:	October 9, 2015
Health Authority:	United States: Institutional Review Board

Keywords provided by University of Wisconsin, Madison:


levodopa Albinism dopamine	oculocutaneous albinism vision retina function

Additional relevant MeSH terms:


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

ClinicalTrials.gov processed this record on September 29, 2016

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