Ignite Creation Date:
2025-12-24 @ 10:31 PM
Ignite Modification Date:
2026-02-21 @ 6:44 PM
Study NCT ID:
NCT01663935
Status:
TERMINATED
Last Update Posted:
2019-06-11
First Post:
2012-08-09
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Vision Response to Dopamine Replacement
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000417', 'term': 'Albinism'}, {'id': 'D016115', 'term': 'Albinism, Oculocutaneous'}], 'ancestors': [{'id': 'D015785', 'term': 'Eye Diseases, Hereditary'}, {'id': 'D005128', 'term': 'Eye Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D000592', 'term': 'Amino Acid Metabolism, Inborn Errors'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D012873', 'term': 'Skin Diseases, Genetic'}, {'id': 'D017496', 'term': 'Hypopigmentation'}, {'id': 'D010859', 'term': 'Pigmentation Disorders'}, {'id': 'D012871', 'term': 'Skin Diseases'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C009265', 'term': 'carbidopa, levodopa drug combination'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'mcstruck@wisc.edu', 'phone': '608 263-6429', 'title': 'Dr Michael Struck', 'organization': 'University of Wisconsin, Madison'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}}, 'adverseEventsModule': {'timeFrame': 'Adverse event data was collected for each patient starting at baseline through the month 4 study follow up visit.', 'eventGroups': [{'id': 'EG000', 'title': 'Levodopa/Carbidopa 4mg/kg/Day', 'description': 'Treatment drug taken orally three times daily\n\nLevodopa/carbidopa: This study will have an intent to treat goal. Anyone that fits the inclusion criteria for the study will be entered and receive study drug.', 'otherNumAtRisk': 20, 'deathsNumAtRisk': 20, 'otherNumAffected': 0, 'seriousNumAtRisk': 20, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Visual Acuity Change', 'denoms': [{'units': 'Participants', 'counts': [{'value': '19', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Levodopa/Carbidopa 4mg/kg/Day', 'description': 'Treatment drug taken orally three times daily\n\nLevodopa/carbidopa: This study will have an intent to treat goal. Anyone that fits the inclusion criteria for the study will be entered and receive study drug.'}], 'classes': [{'categories': [{'measurements': [{'value': '.774', 'spread': '.207', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '3 months', 'description': 'Change in visual acuity from baseline to 3 months as measured in logMAR by Snellen or Sweep visual evoked potential (SVEP). logMar lower values equals better visual outcome.', 'unitOfMeasure': 'logMar', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'participants pre and post treatment per protocol'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'Contrast Sensitivity', 'denoms': [{'units': 'Participants', 'counts': [{'value': '19', 'groupId': 'OG000'}]}, {'units': 'eyes', 'counts': [{'value': '38', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Levodopa/Carbidopa 4mg/kg/Day', 'description': 'Treatment drug taken orally three times daily\n\nLevodopa/carbidopa: This study will have an intent to treat goal. Anyone that fits the inclusion criteria for the study will be entered and receive study drug.'}], 'classes': [{'categories': [{'measurements': [{'value': '23.7', 'spread': '21.6', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '3 months', 'description': 'Ancillary testing of visual/retinal function with contrast sensitivity testing. Contrast sensitivity testing measures how well the eyes can distinguish between finer and finer light increments compared to dark. This test is a chart with different capital letters organized in horizontal lines. The contrast decreases with each line. The person will move down the chart to determine the least level of contrast they can see.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'eyes', 'denomUnitsSelected': 'eyes', 'populationDescription': 'participants pre and post medication per protocol for contrast sensitivity. Range could be anywhere from a score of 0 to 75 for each eye. The higher the score the more letters they could see in low contrast.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Levodopa/Carbidopa 4mg/kg/Day', 'description': 'Treatment drug taken orally three times daily\n\nLevodopa/carbidopa: This study will have an intent to treat goal. Anyone that fits the inclusion criteria for the study will be entered and receive study drug.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '20'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '20'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '20', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Levodopa/Carbidopa 4mg/kg/Day', 'description': 'Treatment drug taken orally three times daily\n\nLevodopa/carbidopa: This study will have an intent to treat goal. Anyone that fits the inclusion criteria for the study will be entered and receive study drug.'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '15', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '5', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '9', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '11', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'Race/Ethnicity', 'categories': [{'title': 'Non hispanic or latino/ white', 'measurements': [{'value': '16', 'groupId': 'BG000'}]}, {'title': 'non hispanic or latino/ asian', 'measurements': [{'value': '3', 'groupId': 'BG000'}]}, {'title': 'hispanic or latino', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '19', 'groupId': 'BG000'}]}]}, {'title': 'Canada', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2017-09-05', 'size': 231433, 'label': 'Study Protocol and Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'Prot_SAP_000.pdf', 'typeAbbrev': 'Prot_SAP', 'uploadDate': '2019-03-27T16:42', 'hasProtocol': True}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 20}}, 'statusModule': {'whyStopped': 'Lack of funding', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2012-10-17', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2019-05', 'completionDateStruct': {'date': '2018-04-12', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2019-05-17', 'studyFirstSubmitDate': '2012-08-09', 'resultsFirstSubmitDate': '2019-04-09', 'studyFirstSubmitQcDate': '2012-08-13', 'lastUpdatePostDateStruct': {'date': '2019-06-11', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2019-05-17', 'studyFirstPostDateStruct': {'date': '2012-08-14', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2019-06-11', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2018-04-12', 'type': 'ACTUAL'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Contrast Sensitivity', 'timeFrame': '3 months', 'description': 'Ancillary testing of visual/retinal function with contrast sensitivity testing. Contrast sensitivity testing measures how well the eyes can distinguish between finer and finer light increments compared to dark. This test is a chart with different capital letters organized in horizontal lines. The contrast decreases with each line. The person will move down the chart to determine the least level of contrast they can see.'}], 'primaryOutcomes': [{'measure': 'Visual Acuity Change', 'timeFrame': '3 months', 'description': 'Change in visual acuity from baseline to 3 months as measured in logMAR by Snellen or Sweep visual evoked potential (SVEP). logMar lower values equals better visual outcome.'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['levodopa', 'Albinism', 'dopamine', 'oculocutaneous albinism', 'vision', 'retina function'], 'conditions': ['Albinism', 'Oculocutaneous Albinism']}, 'descriptionModule': {'briefSummary': 'The purpose of the study is to evaluate and document physiologic and functional changes in visual performance and retinal function of patients diagnosed with albinism (a dopamine deficiency state) following a trial of oral Levodopa/carbidopa treatment.', 'detailedDescription': 'In this study the investigators propose that the retina itself in albinism is deficient in dopamine, and vision improvement will occur as a result of improved retinal function in response to the deficient neurotransmitter dopamine. This study has a pretest-posttest design in order to determine if improvement in vision is in response to replacement of deficiency (dopamine). The electroretinography (ERG) testing and optical coherence tomography (OCT) will be critical determinants to confirm vision improvement as a result of improved retinal function, but are not primary outcome data. Main outcome measures will be collected at pre-treatment, 1 month, 3 months, and 4 months. Change in visual acuity as measured in logMAR by Snellen or sweep visual evoked potential (SVEP) after 3 months of treatment is the primary outcome. Patients include OCA1a patients, OCA1b, OCA2, and unclassified Oculocutaneous albinism (OCA). OCA1a patients clinically are known to have the worst vision, and physiologically have the lowest (or absent) levels of tyrosinase function (Dopamine Production). All patients will be treated with Levodopa/carbidopa 4mg/kg/day in three divided doses.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '3 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* clinical diagnosis of oculocutaneous albinism\n* age over 3 and weight over 25 lbs.\n\nExclusion Criteria:\n\n* ocular only albinism\n* ocular pathology other than albinism\n* neurologic disease, history of myocardial infarction, history of clinical depression, pregnancy'}, 'identificationModule': {'nctId': 'NCT01663935', 'briefTitle': 'Vision Response to Dopamine Replacement', 'organization': {'class': 'OTHER', 'fullName': 'University of Wisconsin, Madison'}, 'officialTitle': 'Interventional Study of Levodopa Replacement on Retinal Function in Oculocutaneous Albinism', 'orgStudyIdInfo': {'id': '2016-1576'}, 'secondaryIdInfos': [{'id': '2012-0023', 'type': 'OTHER', 'domain': 'Study team'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'OTHER', 'label': 'Levodopa/carbidopa 4mg/kg/day', 'description': 'Treatment drug taken orally three times daily', 'interventionNames': ['Drug: Levodopa/carbidopa']}], 'interventions': [{'name': 'Levodopa/carbidopa', 'type': 'DRUG', 'otherNames': ['Sinemet', 'Atamet', 'Parcopa'], 'description': 'This study will have an intent to treat goal. Anyone that fits the inclusion criteria for the study will be entered and receive study drug.', 'armGroupLabels': ['Levodopa/carbidopa 4mg/kg/day']}]}, 'contactsLocationsModule': {'locations': [{'zip': '53705', 'city': 'Madison', 'state': 'Wisconsin', 'country': 'United States', 'facility': 'University of Wisconsin', 'geoPoint': {'lat': 43.07305, 'lon': -89.40123}}], 'overallOfficials': [{'name': 'Michael C Struck, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'University of Wisconsin, Madison'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University of Wisconsin, Madison', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}