Albinism
Conditions
Keywords
Levodopa, Albinism, Vision
Brief summary
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.
Detailed description
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.
Interventions
DRUGLevodopa
Solution taken orally three times a day.
DRUGPlacebo
Solution taken orally three times a day.
Sponsors
University of Minnesota
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
3 Years to 60 Years
Healthy volunteers
No
Inclusion criteria
* Age 3 to 60 years with albinism
Exclusion criteria
* Glaucoma or at increased risk of glaucoma * History of dystonia * History of melanoma * Planning to undergo eye muscle surgery during study time frame * Undergoing vision therapy * Taking iron supplements or vitamins with iron * Taking medication for ADHD * Known liver or gastrointestinal disease * Previous treatment with levodopa * Psychological problems * Ocular abnormalities other than those associated with albinism * Pregnant, nursing or planning to become pregnant during study * Known allergy to levodopa/carbidopa
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Improved Vision | 20 weeks | 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. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| 0.76 mg/kg L-DOPA Solution taken orally three times a day.
Levodopa: Solution taken orally three times a day. | 15 |
| 0.51 mg/kg L-DOPA Solution taken orally three times a day.
Levodopa: Solution taken orally three times a day. | 15 |
| Placebo Solution taken orally three times a day.
Levodopa: Solution taken orally three times a day. | 15 |
| Total | 45 |
Baseline characteristics
| Characteristic | 0.76 mg/kg L-DOPA | Total | Placebo | 0.51 mg/kg L-DOPA |
|---|---|---|---|---|
| Age, Continuous | 12.5 years | 14.5 years | 10.6 years | 19.7 years |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 1 Participants | 2 Participants | 1 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants | 4 Participants | 2 Participants | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 13 Participants | 39 Participants | 12 Participants | 14 Participants |
| Region of Enrollment United States | 15 participants | 45 participants | 15 participants | 15 participants |
| Sex: Female, Male Female | 11 Participants | 24 Participants | 5 Participants | 8 Participants |
| Sex: Female, Male Male | 4 Participants | 21 Participants | 10 Participants | 7 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — | — / — |
| other Total, other adverse events | 11 / 15 | 7 / 15 | 5 / 15 |
| serious Total, serious adverse events | 0 / 15 | 0 / 15 | 0 / 15 |
Outcome results
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.
Time frame: 20 weeks
| Arm | Measure | Value (MEAN) |
|---|---|---|
| 0.76 mg/kg L-DOPA | Improved Vision | 0.67 logMAR |
| 0.51 mg/kg L-DOPA | Improved Vision | 0.55 logMAR |
| Placebo | Improved Vision | 0.53 logMAR |