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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
University of Minnesota - Clinical and Translational Science Institute
ClinicalTrials.gov Identifier:
NCT01176435
First received: August 4, 2010
Last updated: September 23, 2013
Last verified: September 2013
  Purpose

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 Intervention Phase
Albinism
Drug: Levodopa
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment
Official Title: Clinical Trial to Evaluate Levodopa as Treatment to Improve Vision in Individuals With Albinism

Resource links provided by NLM:


Further study details as provided by University of Minnesota - Clinical and Translational Science Institute:

Primary Outcome Measures:
  • Improved vision [ Time Frame: Week 55 ] [ Designated as safety issue: No ]

Estimated Enrollment: 45
Study Start Date: September 2010
Estimated Study Completion Date: December 2013
Estimated Primary Completion Date: December 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Placebo Comparator: 0.76 mg/kg L-DOPA
Solution taken orally three times a day.
Drug: Levodopa
Solution taken orally three times a day.
Placebo 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: Levodopa
Solution taken orally three times a day.

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.

  Eligibility

Ages Eligible for Study:   3 Years to 60 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01176435

Locations
United States, Minnesota
University of Minnesota Eye Clinic
Minneapolis, Minnesota, United States, 55455
Sponsors and Collaborators
University of Minnesota - Clinical and Translational Science Institute
Investigators
Principal Investigator: Gail Summers, M.D. University of Minnesota - Clinical and Translational Science Institute
  More Information

No publications provided

Responsible Party: University of Minnesota - Clinical and Translational Science Institute
ClinicalTrials.gov Identifier: NCT01176435     History of Changes
Other Study ID Numbers: 0912M75653
Study First Received: August 4, 2010
Last Updated: September 23, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by University of Minnesota - Clinical and Translational Science Institute:
Levodopa
Albinism
Vision

Additional relevant MeSH terms:
Albinism
Eye Diseases, Hereditary
Eye Diseases
Genetic Diseases, Inborn
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Skin Diseases, Genetic
Hypopigmentation
Pigmentation Disorders
Skin Diseases
Metabolic Diseases
Levodopa
Antiparkinson Agents
Anti-Dyskinesia Agents
Central Nervous System Agents
Therapeutic Uses
Pharmacologic Actions
Dopamine Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on September 30, 2014