Clinical Trials You Should Know About...
Vision Response to Dopamine Replacement Trial - OCA
The Vision of Children provided initial funding for research by Michael Struck, MD, based at the University of Wisconsin Madison. Dr. Struck is currently recruiting patients for a human clinical trial he is conducting to study a particular treatment for oculocutaneous albinism (OCA). The following information provides additional details about the study including who is eligible to participate in the trial. Please contact their office directly with any questions (contact information indicated at the bottom of this page).
Purpose: The purpose of this 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 investigator proposes 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 pre-test/post-test design in order to determine if improvement in vision is in response to replacement of deficiency (dopamine). The electroretinogram (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, 3 months, and 4 months. Change in visual acuity as measured in logMAR by Snellen or sweep visually evoked potential (SVEP) after 3 months of treatment is the primary outcome.
Subjects include OCA1a, OCA1b, OCA2, and unclassified OCA patients. OCA1a patients clinically are known to have lower visual acuity, and physiologically have the lowest (or absent) levels of tyrosinase function (Dopamine Production). All patients will be treated with Levodopa/Carbidopa 4 mg/kg/day in three divided doses.
- Clinical diagnosis of oculocutaneous albinism
- Ages 3 years and older
- Over 25 pounds
- Informed consent and/or assent by subject and/or guardian
- Ocular only albinism
- Ocular pathology other than albinism
- History of neurologic disease or impairment (specifically seizures)
- History of depression diagnosed by a clinician
- History of myocardial infarction (heart attack)
- History of ulcers
- Currently pregnant or nursing, or planning to get pregnant in the next 4 months
- Currently taking a disallowed medication such as monoamine oxidase inhibitors; coordinator will check medications against this list