Trial of L-DOPA as a Treatment to Improve Vision in Albinism
Study Details
Study Description
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.
Condition or Disease | Intervention/Treatment | Phase |
---|---|---|
|
Phase 2 |
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.
Study Design
Arms and Interventions
Arm | Intervention/Treatment |
---|---|
Active Comparator: 0.76 mg/kg L-DOPA Solution taken orally three times a day. |
Drug: Levodopa
Solution taken orally three times a day.
|
Active Comparator: 0.51 mg/kg L-DOPA Solution taken orally three times a day. |
Drug: Levodopa
Solution taken orally three times a day.
|
Placebo Comparator: Placebo Solution taken orally three times a day. |
Drug: Placebo
Solution taken orally three times a day.
|
Outcome Measures
Primary Outcome Measures
- 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.
Eligibility Criteria
Criteria
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
Contacts and Locations
Locations
Site | City | State | Country | Postal Code | |
---|---|---|---|---|---|
1 | University of Minnesota Eye Clinic | Minneapolis | Minnesota | United States | 55455 |
Sponsors and Collaborators
- University of Minnesota
Investigators
- Principal Investigator: Gail Summers, M.D., University of Minnesota
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- 0912M75653
Study Results
Participant Flow
Recruitment Details | |
---|---|
Pre-assignment Detail |
Arm/Group Title | 0.76 mg/kg L-DOPA | 0.51 mg/kg L-DOPA | Placebo |
---|---|---|---|
Arm/Group Description | Solution taken orally three times a day. Levodopa: Solution taken orally three times a day. | Solution taken orally three times a day. Levodopa: Solution taken orally three times a day. | Solution taken orally three times a day. Levodopa: Solution taken orally three times a day. |
Period Title: Overall Study | |||
STARTED | 15 | 15 | 15 |
COMPLETED | 15 | 15 | 15 |
NOT COMPLETED | 0 | 0 | 0 |
Baseline Characteristics
Arm/Group Title | 0.76 mg/kg L-DOPA | 0.51 mg/kg L-DOPA | Placebo | Total |
---|---|---|---|---|
Arm/Group Description | Solution taken orally three times a day. Levodopa: Solution taken orally three times a day. | Solution taken orally three times a day. Levodopa: Solution taken orally three times a day. | Solution taken orally three times a day. Levodopa: Solution taken orally three times a day. | Total of all reporting groups |
Overall Participants | 15 | 15 | 15 | 45 |
Age (years) [Mean (Full Range) ] | ||||
Mean (Full Range) [years] |
12.5
|
19.7
|
10.6
|
14.5
|
Sex: Female, Male (Count of Participants) | ||||
Female |
11
73.3%
|
8
53.3%
|
5
33.3%
|
24
53.3%
|
Male |
4
26.7%
|
7
46.7%
|
10
66.7%
|
21
46.7%
|
Race (NIH/OMB) (Count of Participants) | ||||
American Indian or Alaska Native |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
Asian |
1
6.7%
|
0
0%
|
1
6.7%
|
2
4.4%
|
Native Hawaiian or Other Pacific Islander |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
Black or African American |
1
6.7%
|
1
6.7%
|
2
13.3%
|
4
8.9%
|
White |
13
86.7%
|
14
93.3%
|
12
80%
|
39
86.7%
|
More than one race |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
Unknown or Not Reported |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
Region of Enrollment (participants) [Number] | ||||
United States |
15
100%
|
15
100%
|
15
100%
|
45
100%
|
Outcome Measures
Title | Improved Vision |
---|---|
Description | 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 |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | 0.76 mg/kg L-DOPA | 0.51 mg/kg L-DOPA | Placebo |
---|---|---|---|
Arm/Group Description | Solution taken orally three times a day. Levodopa: Solution taken orally three times a day. | Solution taken orally three times a day. Levodopa: Solution taken orally three times a day. | Solution taken orally three times a day. Levodopa: Solution taken orally three times a day. |
Measure Participants | 15 | 15 | 15 |
Mean (95% Confidence Interval) [logMAR] |
0.67
|
0.55
|
0.53
|
Adverse Events
Time Frame | ||||||
---|---|---|---|---|---|---|
Adverse Event Reporting Description | ||||||
Arm/Group Title | 0.76 mg/kg L-DOPA | 0.51 mg/kg L-DOPA | Placebo | |||
Arm/Group Description | Solution taken orally three times a day. Levodopa: Solution taken orally three times a day. | Solution taken orally three times a day. Levodopa: Solution taken orally three times a day. | Solution taken orally three times a day. Levodopa: Solution taken orally three times a day. | |||
All Cause Mortality |
||||||
0.76 mg/kg L-DOPA | 0.51 mg/kg L-DOPA | Placebo | ||||
Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
Total | / (NaN) | / (NaN) | / (NaN) | |||
Serious Adverse Events |
||||||
0.76 mg/kg L-DOPA | 0.51 mg/kg L-DOPA | Placebo | ||||
Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
Total | 0/15 (0%) | 0/15 (0%) | 0/15 (0%) | |||
Other (Not Including Serious) Adverse Events |
||||||
0.76 mg/kg L-DOPA | 0.51 mg/kg L-DOPA | Placebo | ||||
Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
Total | 11/15 (73.3%) | 7/15 (46.7%) | 5/15 (33.3%) | |||
Cardiac disorders | ||||||
Dizzy when standing quickly | 2/15 (13.3%) | 2 | 0/15 (0%) | 0 | 1/15 (6.7%) | 1 |
Endocrine disorders | ||||||
Thirsty | 1/15 (6.7%) | 1 | 0/15 (0%) | 0 | 0/15 (0%) | 0 |
Gastrointestinal disorders | ||||||
Loss of Appetite | 1/15 (6.7%) | 1 | 1/15 (6.7%) | 1 | 1/15 (6.7%) | 1 |
Nausea | 1/15 (6.7%) | 1 | 2/15 (13.3%) | 2 | 0/15 (0%) | 0 |
General disorders | ||||||
Dry mouth | 2/15 (13.3%) | 2 | 0/15 (0%) | 0 | 0/15 (0%) | 0 |
Sleepiness | 1/15 (6.7%) | 1 | 1/15 (6.7%) | 1 | 1/15 (6.7%) | 1 |
Fatigue | 2/15 (13.3%) | 2 | 0/15 (0%) | 0 | 1/15 (6.7%) | 1 |
Infections and infestations | ||||||
Rash | 0/15 (0%) | 0 | 0/15 (0%) | 0 | 1/15 (6.7%) | 1 |
Musculoskeletal and connective tissue disorders | ||||||
Swelling of hand | 0/15 (0%) | 0 | 1/15 (6.7%) | 1 | 0/15 (0%) | 0 |
Nervous system disorders | ||||||
Headache | 1/15 (6.7%) | 1 | 2/15 (13.3%) | 2 | 0/15 (0%) | 0 |
Hyperactivity | 0/15 (0%) | 0 | 0/15 (0%) | 0 | 1/15 (6.7%) | 1 |
Twitch/Tremor | 0/15 (0%) | 0 | 0/15 (0%) | 0 | 1/15 (6.7%) | 1 |
Limitations/Caveats
More Information
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title | C. Gail Summers, MD |
---|---|
Organization | University of Minnesota |
Phone | 612-625-6469 |
summe001@umn.edu |
- 0912M75653