A Phase 2 interventional study of Levodopa and Levodopa in Albinism, sponsored by University of Minnesota. Completed at 1 site in United States. Open to participants aged 3 Years to 60 Years. Per ClinicalTrials.gov, last updated 2018-05-02.
Sponsored by University of Minnesota · Phase 2, Interventional, and Treatment
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.
17 studies on the registry are indexed under Albinism; 8 are open to participants now.
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Exclusion Criteria:
Solution taken orally three times a day.
Drug: Levodopa
Solution taken orally three times a day.
Drug: Levodopa
Solution taken orally three times a day.
Drug: Placebo
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
| Milestone | 0.76 mg/kg L-DOPA | 0.51 mg/kg L-DOPA | Placebo |
|---|---|---|---|
| Started | 15 | 15 | 15 |
| Completed | 15 | 15 | 15 |
| Not completed | 0 | 0 | 0 |
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.
| logMAR | 0.76 mg/kg L-DOPA | 0.51 mg/kg L-DOPA | Placebo |
|---|---|---|---|
| Improved Vision | 0.67 (0.20 to 1.0) | 0.55 (0.20 to 0.88) | 0.53 (0.20 to 0.90) |
Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| 0.76 mg/kg L-DOPA | — | 0/15 (0%) | 11/15 (73.3%) |
| 0.51 mg/kg L-DOPA | — | 0/15 (0%) | 7/15 (46.7%) |
| Placebo | — | 0/15 (0%) | 5/15 (33.3%) |
| Event | 0.76 mg/kg L-DOPA | 0.51 mg/kg L-DOPA | Placebo |
|---|---|---|---|
| HeadacheNervous system disorders | 1/15 | 2/15 | 0/15 |
| Dizzy when standing quicklyCardiac disorders | 2/15 | 0/15 | 1/15 |
| Dry mouthGeneral disorders | 2/15 | 0/15 | 0/15 |
| NauseaGastrointestinal disorders | 1/15 | 2/15 | 0/15 |
| FatigueGeneral disorders | 2/15 | 0/15 | 1/15 |
| Loss of AppetiteGastrointestinal disorders | 1/15 | 1/15 | 1/15 |
| SleepinessGeneral disorders | 1/15 | 1/15 | 1/15 |
| ThirstyEndocrine disorders | 1/15 | 0/15 | 0/15 |
| RashInfections and infestations | 0/15 | 0/15 | 1/15 |
| Swelling of handMusculoskeletal and connective tissue disorders | 0/15 | 1/15 | 0/15 |
| Age, Continuous(years) | 0.76 mg/kg L-DOPA | 0.51 mg/kg L-DOPA | Placebo | Total |
|---|---|---|---|---|
| Mean | 12.5 (3.5 to 57) | 19.7 (4 to 49.3) | 10.6 (4 to 18.1) | 14.5 (3.5 to 57) |
| Sex: Female, Male(Participants) | 0.76 mg/kg L-DOPA | 0.51 mg/kg L-DOPA | Placebo | Total |
|---|---|---|---|---|
| Female | 11 | 8 | 5 | 24 |
| Male | 4 | 7 | 10 | 21 |
| Race (NIH/OMB)(Participants) | 0.76 mg/kg L-DOPA | 0.51 mg/kg L-DOPA | Placebo | Total |
|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 0 |
| Asian | 1 | 0 | 1 | 2 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 |
| Black or African American | 1 | 1 | 2 | 4 |
| White | 13 | 14 | 12 | 39 |
| More than one race | 0 | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 0 | 0 |
| Region of Enrollment(participants) | 0.76 mg/kg L-DOPA | 0.51 mg/kg L-DOPA | Placebo | Total |
|---|---|---|---|---|
| United States | 15 | 15 | 15 | 45 |
This study is completed, as verified in Apr 2018. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Levodopa