Trial Outcomes & Findings for Trial of L-DOPA as a Treatment to Improve Vision in Albinism (NCT NCT01176435)
NCT ID: NCT01176435
Last Updated: 2018-05-02
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
45 participants
Primary outcome timeframe
20 weeks
Results posted on
2018-05-02
Participant Flow
Participant milestones
| Measure |
0.76 mg/kg L-DOPA
Solution taken orally three times a day.
Levodopa: Solution taken orally three times a day.
|
0.51 mg/kg L-DOPA
Solution taken orally three times a day.
Levodopa: Solution taken orally three times a day.
|
Placebo
Solution taken orally three times a day.
Levodopa: Solution taken orally three times a day.
|
|---|---|---|---|
|
Overall Study
STARTED
|
15
|
15
|
15
|
|
Overall Study
COMPLETED
|
15
|
15
|
15
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Trial of L-DOPA as a Treatment to Improve Vision in Albinism
Baseline characteristics by cohort
| Measure |
0.76 mg/kg L-DOPA
n=15 Participants
Solution taken orally three times a day.
Levodopa: Solution taken orally three times a day.
|
0.51 mg/kg L-DOPA
n=15 Participants
Solution taken orally three times a day.
Levodopa: Solution taken orally three times a day.
|
Placebo
n=15 Participants
Solution taken orally three times a day.
Levodopa: Solution taken orally three times a day.
|
Total
n=45 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
12.5 years
n=5 Participants
|
19.7 years
n=7 Participants
|
10.6 years
n=5 Participants
|
14.5 years
n=4 Participants
|
|
Sex: Female, Male
Female
|
11 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
24 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
21 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
4 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
13 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
39 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Region of Enrollment
United States
|
15 participants
n=5 Participants
|
15 participants
n=7 Participants
|
15 participants
n=5 Participants
|
45 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: 20 weeksBinocular 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.
Outcome measures
| Measure |
0.76 mg/kg L-DOPA
n=15 Participants
Solution taken orally three times a day.
Levodopa: Solution taken orally three times a day.
|
0.51 mg/kg L-DOPA
n=15 Participants
Solution taken orally three times a day.
Levodopa: Solution taken orally three times a day.
|
Placebo
n=15 Participants
Solution taken orally three times a day.
Levodopa: Solution taken orally three times a day.
|
|---|---|---|---|
|
Improved Vision
|
0.67 logMAR
Interval 0.2 to 1.0
|
0.55 logMAR
Interval 0.2 to 0.88
|
0.53 logMAR
Interval 0.2 to 0.9
|
Adverse Events
0.76 mg/kg L-DOPA
Serious events: 0 serious events
Other events: 11 other events
Deaths: 0 deaths
0.51 mg/kg L-DOPA
Serious events: 0 serious events
Other events: 7 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
0.76 mg/kg L-DOPA
n=15 participants at risk
Solution taken orally three times a day.
Levodopa: Solution taken orally three times a day.
|
0.51 mg/kg L-DOPA
n=15 participants at risk
Solution taken orally three times a day.
Levodopa: Solution taken orally three times a day.
|
Placebo
n=15 participants at risk
Solution taken orally three times a day.
Levodopa: Solution taken orally three times a day.
|
|---|---|---|---|
|
Nervous system disorders
Headache
|
6.7%
1/15 • Number of events 1
|
13.3%
2/15 • Number of events 2
|
0.00%
0/15
|
|
Gastrointestinal disorders
Loss of Appetite
|
6.7%
1/15 • Number of events 1
|
6.7%
1/15 • Number of events 1
|
6.7%
1/15 • Number of events 1
|
|
Cardiac disorders
Dizzy when standing quickly
|
13.3%
2/15 • Number of events 2
|
0.00%
0/15
|
6.7%
1/15 • Number of events 1
|
|
General disorders
Dry mouth
|
13.3%
2/15 • Number of events 2
|
0.00%
0/15
|
0.00%
0/15
|
|
Gastrointestinal disorders
Nausea
|
6.7%
1/15 • Number of events 1
|
13.3%
2/15 • Number of events 2
|
0.00%
0/15
|
|
General disorders
Sleepiness
|
6.7%
1/15 • Number of events 1
|
6.7%
1/15 • Number of events 1
|
6.7%
1/15 • Number of events 1
|
|
Endocrine disorders
Thirsty
|
6.7%
1/15 • Number of events 1
|
0.00%
0/15
|
0.00%
0/15
|
|
Infections and infestations
Rash
|
0.00%
0/15
|
0.00%
0/15
|
6.7%
1/15 • Number of events 1
|
|
General disorders
Fatigue
|
13.3%
2/15 • Number of events 2
|
0.00%
0/15
|
6.7%
1/15 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Swelling of hand
|
0.00%
0/15
|
6.7%
1/15 • Number of events 1
|
0.00%
0/15
|
|
Nervous system disorders
Hyperactivity
|
0.00%
0/15
|
0.00%
0/15
|
6.7%
1/15 • Number of events 1
|
|
Nervous system disorders
Twitch/Tremor
|
0.00%
0/15
|
0.00%
0/15
|
6.7%
1/15 • Number of events 1
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place