Pilot Study to Evaluate Levodopa as Treatment for Residual Amblyopia (ATS14)

This study has been completed.
Sponsor:
Collaborator:
Information provided by (Responsible Party):
Ray Kraker, Jaeb Center for Health Research
ClinicalTrials.gov Identifier:
NCT00789672
First received: October 27, 2008
Last updated: May 16, 2012
Last verified: May 2012
Results First Received: September 14, 2010  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition: Amblyopia
Interventions: Drug: levodopa/carbidopa
Device: patching

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Eligibility criteria included age 8 to <18 years, best-corrected visual acuity in the amblyopic eye between 67 and 18 letters inclusive measured with E-ETDRS, fellow eye best-corrected visual acuity of 78 letters or better, and the presence or history of strabismus and/or anisometropia.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
At enrollment, subjects were required to have been treated with at least 2 hours of patching per day of daily patching and while on that therapy, have had stable visual acuity (5 letters or one logMAR line of improvement since a previous visit at least 8 weeks earlier)

Reporting Groups
  Description
Lower Dose 0.51 mg Levodopa/Carbidopa Oral levodopa 0.51 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.
Higher Dose 0.76 mg Levodopa/Carbidopa Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.

Participant Flow:   Overall Study
    Lower Dose 0.51 mg Levodopa/Carbidopa     Higher Dose 0.76 mg Levodopa/Carbidopa  
STARTED     16     17  
COMPLETED     16     17  
NOT COMPLETED     0     0  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Lower Dose 0.51 mg Levodopa/Carbidopa Oral levodopa 0.51 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.
Higher Dose 0.76 mg Levodopa/Carbidopa Oral levodopa 0.76 mg/kg tid with carbidopa 0.17 mg/kg tid combined with 2 hours of daily patching, with a rapid taper of medication before a primary outcome exam.
Total Total of all reporting groups

Baseline Measures
    Lower Dose 0.51 mg Levodopa/Carbidopa     Higher Dose 0.76 mg Levodopa/Carbidopa     Total  
Number of Participants  
[units: participants]
  16     17     33  
Age  
[units: years]
Mean ± Standard Deviation
  11.3  ± 2.2     11.0  ± 2.2     11.1  ± 2.2  
Age, Customized [1]
[units: participants]
     
8 to <9     3     3     6  
9 to <10     2     3     5  
10 to <11     3     4     7  
11 to <12     3     1     4  
12 to <13     0     3     3  
13 to <14     3     1     4  
14 to <15     2     0     2  
15 to <16     0     2     2  
16 to <17     0     0     0  
17 to <18     0     0     0  
Gender  
[units: participants]
     
Female     10     9     19  
Male     6     8     14  
Region of Enrollment  
[units: participants]
     
United States     16     17     33  
Cause of Amblyopia  
[units: participants]
     
Strabismus     2     5     7  
Anisometropia     6     5     11  
Strabismus and anisometropia     8     7     15  
Distance Visual Acuity in Amblyopic Eye [2]
[units: participants]
     
20/400 (18 to 22 letters)     0     1     1  
20/320 (23 to 27 letters)     0     0     0  
20/250 (28 to 32 letters)     0     1     1  
20/200 (33 to 37 letters)     1     1     2  
20/160 (38 to 42 letters)     1     1     2  
20/125 (43 to 47 letters)     0     1     1  
20/100 (48 to 52 letters)     2     0     2  
20/80 (53 to 57 letters)     4     6     10  
20/63 (58 to 62 letters)     4     5     9  
20/50 (63 to 67 letters)     4     1     5  
Distance Visual Acuity in Fellow Eye [2]
[units: participants]
     
20/25 (78 to 82 letters)     4     4     8  
20/20 (83 to 87 letters)     6     8     14  
20/16 (88 to 92 letters)     5     5     10  
20/12 (93 to 97 letters)     1     0     1  
Race/Ethnicity  
[units: participants]
     
White     14     17     31  
African-American     1     0     1  
Hispanic or Latino     1     0     1  
Asian     0     0     0  
More than one race     0     0     0  
Unknown/Not reported     0     0     0  
Refractive Error in Amblyopic Eye (spherical equivalent/diopters) [3]
[units: participants]
     
<0.00D     0     1     1  
0 to < +1.00D     1     2     3  
+1.00 to <+2.00D     1     1     2  
+2.00 to <+3.00D     2     2     4  
+3.00 to <+4.00D     3     2     5  
greater than or equal to +4.00D     9     9     18  
Refractive Error in Fellow Eye (spherical equivalent/diopters) [3]
[units: participants]
     
<0.00D     1     0     1  
0 to < +1.00D     6     10     16  
+1.00 to <+2.00D     5     3     8  
+2.00 to <+3.00D     2     3     5  
+3.00 to <+4.00D     2     1     3  
greater than or equal to +4.00D     0     0     0  
Mean (SD) Distance Visual Acuity in Amblyopic Eye [4]
[units: letters]
Mean ± Standard Deviation
  56.2  ± 8.8     50.5  ± 12.2     53.2  ± 10.9  
Mean (SD) Distance Visual Acuity in Fellow Eye [4]
[units: letters]
Mean ± Standard Deviation
  86.4  ± 4.5     85.5  ± 3.3     85.9  ± 3.9  
Mean (SD) Intereye Acuity Difference [5]
[units: letters]
Mean ± Standard Deviation
  30.2  ± 11.7     35.1  ± 13.6     32.7  ± 12.7  
Mean (SD) Spherical Equivalent Refractive Error in Amblyopic Eye [3]
[units: diopters]
Mean ± Standard Deviation
  4.05  ± 1.92     3.28  ± 1.93     3.65  ± 1.93  
Mean (SD) Spherical Equivalent Refractive Error in Fellow Eye [3]
[units: diopters]
Mean ± Standard Deviation
  1.20  ± 1.17     1.04  ± 0.99     1.12  ± 1.07  
Weight in Kilograms  
[units: kilograms]
Median ( Full Range )
  47  
  ( 25 to 71 )  
  34  
  ( 23 to 74 )  
  42  
  ( 23 to 74 )  
[1] Age at enrollment in years
[2] Visual acuity was measured with the electronic early treatment diabetic retinopathy study (E-ETDRS) method and resulted in a letter score that could range from 0 to 97 letters, with 0 being the worst and 97 being the best. Letter scores are presented as Snellen Equivalents for presentation (i.e. 20/20 includes those with letter scores between 83 and 87 letters, 20/25 includes those with letter scores between 78 to 82 letters, etc.).
[3] Spherical equivalent in diopters defined from cycloplegic refraction as sphere plus half the cylinder value
[4] Visual acuity was measured with the electronic early treatment diabetic retinopathy study (E-ETDRS) method and resulted in a letter score that could range from 0 to 97 letters, with 0 being the worst and 97 being the best.
[5] Visual acuity was measured with the electronic early treatment diabetic retinopathy study (E-ETDRS) method and resulted in a letter score in each eye that could range from 0 to 97 letters. Intereye difference calculated as the difference in letters between the sound eye and amblyopic eye (positive difference indicates sound eye better).



  Outcome Measures
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1.  Primary:   Distribution of Amblyopic Eye Visual Acuity Letter Scores at 9 Weeks After Starting Levodopa   [ Time Frame: 9 weeks after starting levodopa ]

2.  Primary:   Mean Amblyopic Eye Visual Acuity Letter Scores at 9 Weeks After Starting Levodopa   [ Time Frame: 9 weeks after starting levodopa ]

3.  Primary:   Distribution of Change in Amblyopic Eye Visual Acuity Scores at 9 Weeks After Starting Levodopa   [ Time Frame: baseline to 9 weeks ]

4.  Primary:   Mean Change in Amblyopic Eye Visual Acuity Letter Scores at 9 Weeks After Starting Levodopa   [ Time Frame: baseline to 9 weeks ]

5.  Primary:   Tolerability of Study Medication-Adverse Event Reporting   [ Time Frame: 24 weeks ]

6.  Secondary:   Distribution of Amblyopic Eye Visual Acuity Letter Scores at 4 Weeks After Enrollment   [ Time Frame: 4 weeks after enrollment ]

7.  Secondary:   Mean Amblyopic Eye Visual Acuity Letter Scores at 4 Weeks Post Enrollment   [ Time Frame: 4 weeks after enrollment ]

8.  Secondary:   Distribution of Amblyopic Eye Visual Acuity Letter Scores at 10 Weeks After Stopping Levodopa   [ Time Frame: 10 weeks after stopping levodopa ]

9.  Secondary:   Mean Amblyopic Eye Visual Acuity Letter Scores at 10 Weeks After Stopping Levodopa   [ Time Frame: 10 weeks after stopping levodopa ]

10.  Secondary:   Distribution of Change in Amblyopic Eye Visual Acuity Scores at 4 Weeks Post Enrollment   [ Time Frame: enrollment to 4 weeks ]

11.  Secondary:   Mean Change in Amblyopic Eye Visual Acuity Letter Scores at 4 Weeks Post Enrollment   [ Time Frame: enrollment to 4 weeks ]

12.  Secondary:   Distribution of Change in Amblyopic Eye Visual Acuity Scores at 10 Weeks After Stopping Levodopa   [ Time Frame: baseline to 10 weeks after stopping levodopa ]

13.  Secondary:   Mean Change in Amblyopic Eye Visual Acuity Letter Scores at 10 Weeks After Stopping Levodopa   [ Time Frame: baseline to 10 weeks after stopping levodopa ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
Without a patching-only control group, no conclusions can be made regarding efficacy, safety, or side effects with this treatment. Placebo controlled trial is needed to see if levodopa can augment occlusion therapy in the treatment of amblyopia.


  More Information
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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: Raymond Kraker, MSPH
Organization: Jaeb Center for Health Research
phone: 813-875-8690
e-mail: pedig@jaeb.org


No publications provided


Responsible Party: Ray Kraker, Jaeb Center for Health Research
ClinicalTrials.gov Identifier: NCT00789672     History of Changes
Other Study ID Numbers: NEI-141, 2U10EY011751
Study First Received: October 27, 2008
Results First Received: September 14, 2010
Last Updated: May 16, 2012
Health Authority: United States: Food and Drug Administration