Trial of L-DOPA as a Treatment to Improve Vision in Albinism

• Sponsor: University of Minnesota (Other)
• Overall Status: Completed
• CT.gov ID: NCT01176435
• Collaborator: (none)
• Enrollment: 45
• Location: 1
• Arms: 3
• Duration (Months): 51
• Patients Per Site Per Month: 0.9

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
Albinism	Drug: Levodopa; Drug: Levodopa; Drug: Placebo	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

• Study Type: Interventional
• Actual Enrollment: 45 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Double (Participant, Investigator)
• Primary Purpose: Treatment
• Official Title: Clinical Trial to Evaluate Levodopa as Treatment to Improve Vision in Individuals With Albinism
• Study Start Date: Sep 1, 2010
• Actual Primary Completion Date: Dec 1, 2014
• Actual Study Completion Date: Dec 1, 2014

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

1. 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

• Ages Eligible for Study: 3 Years to 60 Years
• Sexes Eligible for Study: All
• Accepts 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

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.

• Responsible Party: University of Minnesota
• ClinicalTrials.gov Identifier: NCT01176435
• Other Study ID Numbers: 0912M75653
• First Posted: Aug 6, 2010
• Last Update Posted: May 2, 2018
• Last Verified: Apr 1, 2018

• Keywords provided by University of Minnesota: Levodopa; Albinism; Vision
• Additional relevant MeSH terms: Albinism; Levodopa

Study Results

Participant Flow

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

1. Primary Outcome

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

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

[Not Specified]

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.