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| ID | Type | Description | Link |
|---|---|---|---|
| 2012-0023 | Other Identifier | Study team |
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Lack of funding
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Levodopa/carbidopa 4mg/kg/day | Other | Treatment drug taken orally three times daily |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Levodopa/carbidopa | Drug | 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. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Visual Acuity Change | 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. | 3 months |
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| Measure | Description | Time Frame |
|---|---|---|
| Contrast Sensitivity | Ancillary testing of visual/retinal function with contrast sensitivity testing. Contrast sensitivity testing measures how well the eyes can distinguish between finer and finer light increments compared to dark. This test is a chart with different capital letters organized in horizontal lines. The contrast decreases with each line. The person will move down the chart to determine the least level of contrast they can see. |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Michael C Struck, MD | University of Wisconsin, Madison | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Wisconsin | Madison | Wisconsin | 53705 | United States |
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| ID | Title | Description |
|---|---|---|
| FG000 | Levodopa/Carbidopa 4mg/kg/Day | Treatment drug taken orally three times daily 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. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Levodopa/Carbidopa 4mg/kg/Day | Treatment drug taken orally three times daily 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. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Visual Acuity Change | 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. | participants pre and post treatment per protocol | Posted | Mean | Standard Deviation | logMar | 3 months |
|
|
Adverse event data was collected for each patient starting at baseline through the month 4 study follow up visit.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Levodopa/Carbidopa 4mg/kg/Day | Treatment drug taken orally three times daily 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. |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr Michael Struck | University of Wisconsin, Madison | 608 263-6429 | mcstruck@wisc.edu |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Sep 5, 2017 | Mar 27, 2019 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D000417 | Albinism |
| D016115 | Albinism, Oculocutaneous |
| ID | Term |
|---|---|
| D015785 | Eye Diseases, Hereditary |
| D005128 | Eye Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| C009265 | carbidopa, levodopa drug combination |
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|
| 3 months |
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Participants |
|
|
| Other Pre-specified | Contrast Sensitivity | Ancillary testing of visual/retinal function with contrast sensitivity testing. Contrast sensitivity testing measures how well the eyes can distinguish between finer and finer light increments compared to dark. This test is a chart with different capital letters organized in horizontal lines. The contrast decreases with each line. The person will move down the chart to determine the least level of contrast they can see. | participants pre and post medication per protocol for contrast sensitivity. Range could be anywhere from a score of 0 to 75 for each eye. The higher the score the more letters they could see in low contrast. | Posted | Mean | Standard Deviation | score on a scale | 3 months | eyes | eyes |
|
|
|
| 0 |
| 20 |
| 0 |
| 20 |
| 0 |
| 20 |
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| D000592 | Amino Acid Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D012873 | Skin Diseases, Genetic |
| D017496 | Hypopigmentation |
| D010859 | Pigmentation Disorders |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |