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This project will evaluate the effect of two doses of levodopa (L-DOPA) in a randomized, placebo-controlled, double-masked clinical trial to see if vision can be improved in individuals with albinism. The hypothesis is that providing L-DOPA to the retinas of these individuals may increase melanin pigment production. Increased melanin has previously been shown to be associated with improved vision.
A group of 45 individuals with the clinical findings of oculocutaneous albinism (OCA) will be randomly assigned to one of 3 treatment groups: treatment with 0.76 mg/kg/d with 25% carbidopa, 0.51 mg/kg/d levodopa with 25% carbidopa [divided into 3 doses/d), or placebo. Subjects will be between ages 3 and 60 years. Blood will be drawn to determine the mutation(s) in the genes that causes OCA. Primary outcome will be binocular best-corrected visual acuity measured with the EVA. Enrollment and 20 week examination will be complete eye exam with fundus photos. At weeks 5, 10, and 15, exams will include just vital signs and BCVA. At all visits, a review of potential side effects will be conducted. Between visits, subjects will be contacted to determine if any side effects have occurred. The study will remain double masked until the last study examination on the last subject has been performed. At that time, the data will be statistically analyzed and subjects will be informed re: treatment assignment, mutations found, and the study results.](streamdown:incomplete-link)
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 0.76 mg/kg L-DOPA | Active Comparator | Solution taken orally three times a day. |
|
| 0.51 mg/kg L-DOPA | Active Comparator | Solution taken orally three times a day. |
|
| Placebo | Placebo Comparator | Solution taken orally three times a day. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Levodopa | Drug | Solution taken orally three times a day. |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Improved Vision | Binocular best-corrected visual acuity-The visual acuity test is used to determine the smallest letters you can read on a standardized chart (Snellen chart) or a card held 20 feet (6 meters) away. Special charts are used when testing at distances shorter than 20 feet (6 meters). Ranges are 20/10 vision to 20/200 vision. 20/10 being the best and 20/200 being the worse. | 20 weeks |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Gail Summers, M.D. | University of Minnesota | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Minnesota Eye Clinic | Minneapolis | Minnesota | 55455 | United States |
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| ID | Title | Description |
|---|---|---|
| FG000 | 0.76 mg/kg L-DOPA | Solution taken orally three times a day. Levodopa: Solution taken orally three times a day. |
| FG001 | 0.51 mg/kg L-DOPA | Solution taken orally three times a day. Levodopa: Solution taken orally three times a day. |
| FG002 | Placebo | Solution taken orally three times a day. Levodopa: Solution taken orally three times a day. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | 0.76 mg/kg L-DOPA | Solution taken orally three times a day. Levodopa: Solution taken orally three times a day. |
| BG001 | 0.51 mg/kg L-DOPA | Solution taken orally three times a day. Levodopa: Solution taken orally three times a day. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| 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 | Improved Vision | Binocular best-corrected visual acuity-The visual acuity test is used to determine the smallest letters you can read on a standardized chart (Snellen chart) or a card held 20 feet (6 meters) away. Special charts are used when testing at distances shorter than 20 feet (6 meters). Ranges are 20/10 vision to 20/200 vision. 20/10 being the best and 20/200 being the worse. | Posted | Mean | 95% Confidence Interval | logMAR | 20 weeks |
|
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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 | 0.76 mg/kg L-DOPA | Solution taken orally three times a day. Levodopa: Solution taken orally three times a day. |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Headache | Nervous system disorders | MEDra | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| C. Gail Summers, MD | University of Minnesota | 612-625-6469 | summe001@umn.edu |
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| ID | Term |
|---|---|
| D000417 | Albinism |
| 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 |
|---|---|
| D007980 | Levodopa |
| ID | Term |
|---|---|
| D004295 | Dihydroxyphenylalanine |
| D002395 | Catecholamines |
| D000588 | Amines |
| D009930 | Organic Chemicals |
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| Levodopa |
| Drug |
Solution taken orally three times a day. |
|
| Placebo | Drug | Solution taken orally three times a day. |
|
| BG002 | Placebo | Solution taken orally three times a day. Levodopa: Solution taken orally three times a day. |
| BG003 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| OG002 | Placebo | Solution taken orally three times a day. Levodopa: Solution taken orally three times a day. |
|
|
| 0 |
| 15 |
| 11 |
| 15 |
| EG001 | 0.51 mg/kg L-DOPA | Solution taken orally three times a day. Levodopa: Solution taken orally three times a day. | 0 | 15 | 7 | 15 |
| EG002 | Placebo | Solution taken orally three times a day. Levodopa: Solution taken orally three times a day. | 0 | 15 | 5 | 15 |
| Loss of Appetite | Gastrointestinal disorders | MedRa | Systematic Assessment |
|
| Dizzy when standing quickly | Cardiac disorders | MedRa | Systematic Assessment |
|
| Dry mouth | General disorders | MedRa | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | Medra | Systematic Assessment |
|
| Sleepiness | General disorders | Medra | Systematic Assessment |
|
| Thirsty | Endocrine disorders | Medra | Systematic Assessment |
|
| Rash | Infections and infestations | Medra | Systematic Assessment |
|
| Fatigue | General disorders | Medra | Systematic Assessment |
|
| Swelling of hand | Musculoskeletal and connective tissue disorders | Medra | Systematic Assessment |
|
| Hyperactivity | Nervous system disorders | Medra | Systematic Assessment |
|
| Twitch/Tremor | Nervous system disorders | Medra | Systematic Assessment |
|
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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 |
| D002396 |
| Catechols |
| D010636 | Phenols |
| D001555 | Benzene Derivatives |
| D006841 | Hydrocarbons, Aromatic |
| D006844 | Hydrocarbons, Cyclic |
| D006838 | Hydrocarbons |
| D010649 | Phenylalanine |
| D024322 | Amino Acids, Aromatic |
| D000598 | Amino Acids, Cyclic |
| D000596 | Amino Acids |
| D000602 | Amino Acids, Peptides, and Proteins |
| D014443 | Tyrosine |