AlbinismPhase 2NCT01176435
Trial of L-DOPA as a Treatment to Improve Vision in Albinism
A Phase 2 study testing Levodopa in people with Albinism, run by University of Minnesota at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 3 to 60 years
- Conditions the study is for
- Albinism
- What is being tested?
- Levodopa
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 45 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2010to Dec 2014
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age 3 to 60 years with albinism
Who cannot join
- 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
Show all 13Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Minnesota Eye Clinic
Minneapolis, Minnesota, 55455
About this study
Official title: Clinical Trial to Evaluate Levodopa as Treatment to Improve Vision in Individuals With Albinism
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.
Read the 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.
- Conditions
- Albinism
- Being tested
- Levodopa, Placebo
- Keywords
- Levodopa, Albinism, Vision
- Medical subject headings
- Albinism
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LevodopaDrug
Solution taken orally three times a day.
LevodopaDrug
Solution taken orally three times a day.
PlaceboDrug
Solution taken orally three times a day.
| Study group | Role | What they receive |
|---|---|---|
| 0.76 mg/kg L-DOPA | Active comparator | Solution taken orally three times a day. Receives: Drug: Levodopa |
| 0.51 mg/kg L-DOPA | Active comparator | Solution taken orally three times a day. Receives: Drug: Levodopa |
| Placebo | Placebo comparator | Solution taken orally three times a day. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 20 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Aug 2010 (estimated)
- Study startSep 2010
- Primary completionDec 2014
- Study completionDec 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for AlbinismStudies of the same condition, open and under way.
- More trials run by University of MinnesotaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 45 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Minnesota
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Gail Summers, M.D., Principal investigator, University of Minnesota
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01176435
- Sponsor's protocol ID
- 0912M75653
- Status last verified
- Apr 2018
- First submitted
- 4 Aug 2010
- Last updated
- 2 May 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 May 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Trial to Evaluate Levodopa as Treatment to Improve Vision in Individuals With Albinism
- ClinicalTrials.gov ID
- NCT01176435
- Acronym / sponsor ID
- 0912M75653
- Conditions
- Albinism
- Keywords
- Levodopa, Albinism, Vision
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 45 participants (actual)
- Lead sponsor
- University of MinnesotaUniversity, hospital or other
- Study start
- Sep 2010
- Primary completion
- Dec 2014 (actual)
- Study completion
- Dec 2014 (actual)
- First posted
- 6 Aug 2010 (estimated)
- Results
- Posted 2 May 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| 0.76 mg/kg L-DOPA | Active comparator | Drug: LevodopaSolution taken orally three times a day. |
| 0.51 mg/kg L-DOPA | Active comparator | Drug: LevodopaSolution taken orally three times a day. |
| Placebo | Placebo comparator | Drug: PlaceboSolution taken orally three times a day. |
Who may qualify
- Ages
- 3 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (1 listed)
- Age 3 to 60 years with albinism
Key exclusion criteria (13 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LevodopaSolution taken orally three times a day. | Drug | 0.76 mg/kg L-DOPA |
| LevodopaSolution taken orally three times a day. | Drug | 0.51 mg/kg L-DOPA |
| PlaceboSolution taken orally three times a day. | Drug | Placebo |
Primary outcome
Improved Vision
Time frame: 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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 May 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Gail Summers, M.D. principal investigator
University of Minnesota
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Levodopa in people with Albinism, run by University of Minnesota at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 2 May 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 2 May 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Improved Vision (time frame: 20 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 May 2018.