Nctid:
NCT00001213
Payload:
{"FullStudy"=>{"Rank"=>474417, "Study"=>{"DerivedSection"=>{"MiscInfoModule"=>{"VersionHolder"=>"December 08, 2023"}, "ConditionBrowseModule"=>{"ConditionMeshList"=>{"ConditionMesh"=>[{"ConditionMeshId"=>"D000003554", "ConditionMeshTerm"=>"Cystinosis"}]}, "ConditionAncestorList"=>{"ConditionAncestor"=>[{"ConditionAncestorId"=>"D000016464", "ConditionAncestorTerm"=>"Lysosomal Storage Diseases"}, {"ConditionAncestorId"=>"D000008661", "ConditionAncestorTerm"=>"Metabolism, Inborn Errors"}, {"ConditionAncestorId"=>"D000030342", "ConditionAncestorTerm"=>"Genetic Diseases, Inborn"}, {"ConditionAncestorId"=>"D000008659", "ConditionAncestorTerm"=>"Metabolic Diseases"}]}, "ConditionBrowseLeafList"=>{"ConditionBrowseLeaf"=>[{"ConditionBrowseLeafId"=>"M8031", "ConditionBrowseLeafName"=>"Fanconi Syndrome", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M6449", "ConditionBrowseLeafName"=>"Cystinosis", "ConditionBrowseLeafAsFound"=>"Cystinosis", "ConditionBrowseLeafRelevance"=>"high"}, {"ConditionBrowseLeafId"=>"M18561", "ConditionBrowseLeafName"=>"Lysosomal Storage Diseases", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M11331", "ConditionBrowseLeafName"=>"Metabolism, Inborn Errors", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M23376", "ConditionBrowseLeafName"=>"Genetic Diseases, Inborn", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M11329", "ConditionBrowseLeafName"=>"Metabolic Diseases", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"T1714", "ConditionBrowseLeafName"=>"Cystinosis", "ConditionBrowseLeafAsFound"=>"Cystinosis", "ConditionBrowseLeafRelevance"=>"high"}, {"ConditionBrowseLeafId"=>"T2272", "ConditionBrowseLeafName"=>"Fanconi Syndrome", "ConditionBrowseLeafRelevance"=>"low"}]}, "ConditionBrowseBranchList"=>{"ConditionBrowseBranch"=>[{"ConditionBrowseBranchName"=>"Urinary Tract, Sexual Organs, and Pregnancy Conditions", "ConditionBrowseBranchAbbrev"=>"BXS"}, {"ConditionBrowseBranchName"=>"Diseases and Abnormalities at or Before Birth", "ConditionBrowseBranchAbbrev"=>"BC16"}, {"ConditionBrowseBranchName"=>"All Conditions", "ConditionBrowseBranchAbbrev"=>"All"}, {"ConditionBrowseBranchName"=>"Nutritional and Metabolic Diseases", "ConditionBrowseBranchAbbrev"=>"BC18"}, {"ConditionBrowseBranchName"=>"Rare Diseases", "ConditionBrowseBranchAbbrev"=>"Rare"}]}}, "InterventionBrowseModule"=>{"InterventionMeshList"=>{"InterventionMesh"=>[{"InterventionMeshId"=>"D000003543", "InterventionMeshTerm"=>"Cysteamine"}]}, "InterventionAncestorList"=>{"InterventionAncestor"=>[{"InterventionAncestorId"=>"D000065104", "InterventionAncestorTerm"=>"Cystine Depleting Agents"}, {"InterventionAncestorId"=>"D000045504", "InterventionAncestorTerm"=>"Molecular Mechanisms of Pharmacological Action"}]}, "InterventionBrowseLeafList"=>{"InterventionBrowseLeaf"=>[{"InterventionBrowseLeafId"=>"M12504", "InterventionBrowseLeafName"=>"Ophthalmic Solutions", "InterventionBrowseLeafRelevance"=>"low"}, {"InterventionBrowseLeafId"=>"M21550", "InterventionBrowseLeafName"=>"Pharmaceutical Solutions", "InterventionBrowseLeafRelevance"=>"low"}, {"InterventionBrowseLeafId"=>"M6438", "InterventionBrowseLeafName"=>"Cysteamine", "InterventionBrowseLeafAsFound"=>"Legumes", "InterventionBrowseLeafRelevance"=>"high"}]}, "InterventionBrowseBranchList"=>{"InterventionBrowseBranch"=>[{"InterventionBrowseBranchName"=>"Pharmaceutical Solutions", "InterventionBrowseBranchAbbrev"=>"PhSol"}, {"InterventionBrowseBranchName"=>"All Drugs and Chemicals", "InterventionBrowseBranchAbbrev"=>"All"}]}}}, "ResultsSection"=>{"MoreInfoModule"=>{"PointOfContact"=>{"PointOfContactEMail"=>"bishopra@nei.nih.gov", "PointOfContactPhone"=>"301-402-3771", "PointOfContactTitle"=>"Rachel J. Bishop, MD, Principal Investigator, National Eye Institute", "PointOfContactOrganization"=>"National Institutes of Health"}, "CertainAgreement"=>{"AgreementPISponsorEmployee"=>"Yes", "AgreementRestrictiveAgreement"=>"No"}}, "AdverseEventsModule"=>{"EventGroupList"=>{"EventGroup"=>[{"EventGroupId"=>"EG000", "EventGroupTitle"=>"Cysteamine Topical Solution", "EventGroupDescription"=>"Cysteamine topical solution administered hourly while awake in both eyes\n\nCysteamine", "EventGroupOtherNumAtRisk"=>"328", "EventGroupOtherNumAffected"=>"257", "EventGroupSeriousNumAtRisk"=>"328", "EventGroupSeriousNumAffected"=>"11"}]}, "OtherEventList"=>{"OtherEvent"=>[{"OtherEventTerm"=>"Photophobia", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"239", "OtherEventStatsNumAffected"=>"239"}]}, "OtherEventOrganSystem"=>"Eye disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Conjunctival hyperaemia", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"69", "OtherEventStatsNumAffected"=>"69"}]}, "OtherEventOrganSystem"=>"Eye disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Eye pain", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"66", "OtherEventStatsNumAffected"=>"66"}]}, "OtherEventOrganSystem"=>"Eye disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Ocular hyperaemia", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"52", "OtherEventStatsNumAffected"=>"52"}]}, "OtherEventOrganSystem"=>"Eye disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Eye irritation", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"49", "OtherEventStatsNumAffected"=>"49"}]}, "OtherEventOrganSystem"=>"Eye disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Lacrimation increased", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"23", "OtherEventStatsNumAffected"=>"23"}]}, "OtherEventOrganSystem"=>"Eye disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Optic disc disorder", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"19", "OtherEventStatsNumAffected"=>"19"}]}, "OtherEventOrganSystem"=>"Eye disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Vision blurred", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"20", "OtherEventStatsNumAffected"=>"20"}]}, "OtherEventOrganSystem"=>"Eye disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Keratitis", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"19", "OtherEventStatsNumAffected"=>"19"}]}, "OtherEventOrganSystem"=>"Eye disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Dry eye", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"14", "OtherEventStatsNumAffected"=>"14"}]}, "OtherEventOrganSystem"=>"Eye disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Eye pruritis", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"13", "OtherEventStatsNumAffected"=>"13"}]}, "OtherEventOrganSystem"=>"Eye disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Conjunctivitis", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"12", "OtherEventStatsNumAffected"=>"12"}]}, "OtherEventOrganSystem"=>"Eye disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Eyelid oedema", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"12", "OtherEventStatsNumAffected"=>"12"}]}, "OtherEventOrganSystem"=>"Eye disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Retinal disorder", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"11", "OtherEventStatsNumAffected"=>"11"}]}, "OtherEventOrganSystem"=>"Eye disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Blindness", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"9", "OtherEventStatsNumAffected"=>"9"}]}, "OtherEventOrganSystem"=>"Eye disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Corneal epithelium disorder", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"6", "OtherEventStatsNumAffected"=>"6"}]}, "OtherEventOrganSystem"=>"Eye disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Blepharitis", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"5", "OtherEventStatsNumAffected"=>"5"}]}, "OtherEventOrganSystem"=>"Eye disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Erythema of eyelid", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"5", "OtherEventStatsNumAffected"=>"5"}]}, "OtherEventOrganSystem"=>"Eye disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Eye swelling", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"3", "OtherEventStatsNumAffected"=>"3"}]}, "OtherEventOrganSystem"=>"Eye disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Conjunctival oedema", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"2", "OtherEventStatsNumAffected"=>"2"}]}, "OtherEventOrganSystem"=>"Eye disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Eye oedema", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"1", "OtherEventStatsNumAffected"=>"1"}]}, "OtherEventOrganSystem"=>"Eye disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Eyelid cyst", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"1", "OtherEventStatsNumAffected"=>"1"}]}, "OtherEventOrganSystem"=>"Eye disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Eyelid irritation", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"1", "OtherEventStatsNumAffected"=>"1"}]}, "OtherEventOrganSystem"=>"Eye disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Ulcerative keratitis", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"1", "OtherEventStatsNumAffected"=>"1"}]}, "OtherEventOrganSystem"=>"Eye disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Headache", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"94", "OtherEventStatsNumAffected"=>"94"}]}, "OtherEventOrganSystem"=>"Nervous system disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Visual field defect", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"35", "OtherEventStatsNumAffected"=>"35"}]}, "OtherEventOrganSystem"=>"Nervous system disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Benign intracranial hypertension", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"4", "OtherEventStatsNumAffected"=>"4"}]}, "OtherEventOrganSystem"=>"Nervous system disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Instillation site irritation", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"33", "OtherEventStatsNumAffected"=>"33"}]}, "OtherEventOrganSystem"=>"General disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Instillation site pain", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"22", "OtherEventStatsNumAffected"=>"22"}]}, "OtherEventOrganSystem"=>"General disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Adverse drug reaction", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"7", "OtherEventStatsNumAffected"=>"7"}]}, "OtherEventOrganSystem"=>"General disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Instillation site erythema", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"5", "OtherEventStatsNumAffected"=>"5"}]}, "OtherEventOrganSystem"=>"General disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Swelling", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"2", "OtherEventStatsNumAffected"=>"2"}]}, "OtherEventOrganSystem"=>"General disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Drug ineffective", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"1", "OtherEventStatsNumAffected"=>"1"}]}, "OtherEventOrganSystem"=>"Eye disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Instillation site lacrimation", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"1", "OtherEventStatsNumAffected"=>"1"}]}, "OtherEventOrganSystem"=>"General disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Instillation site reaction", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"1", "OtherEventStatsNumAffected"=>"1"}]}, "OtherEventOrganSystem"=>"General disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Eye infection", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"6", "OtherEventStatsNumAffected"=>"6"}]}, "OtherEventOrganSystem"=>"Infections and infestations", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Conjunctivitis ineffective", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"3", "OtherEventStatsNumAffected"=>"3"}]}, "OtherEventOrganSystem"=>"Infections and infestations", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Hordeolum", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"3", "OtherEventStatsNumAffected"=>"3"}]}, "OtherEventOrganSystem"=>"Infections and infestations", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Vomiting", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"8", "OtherEventStatsNumAffected"=>"8"}]}, "OtherEventOrganSystem"=>"Gastrointestinal disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Abdominal pain", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"1", "OtherEventStatsNumAffected"=>"1"}]}, "OtherEventOrganSystem"=>"Gastrointestinal disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Drug hypersensitivity", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"2", "OtherEventStatsNumAffected"=>"2"}]}, "OtherEventOrganSystem"=>"Immune system disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}, {"OtherEventTerm"=>"Visual evoked potentials abnormal", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"328", "OtherEventStatsNumEvents"=>"1", "OtherEventStatsNumAffected"=>"1"}]}, "OtherEventOrganSystem"=>"Investigations", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (9.0)"}]}, "EventsTimeFrame"=>"Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially.", "SeriousEventList"=>{"SeriousEvent"=>[{"SeriousEventTerm"=>"Optic disc disorder", "SeriousEventStatsList"=>{"SeriousEventStats"=>[{"SeriousEventStatsGroupId"=>"EG000", "SeriousEventStatsNumAtRisk"=>"328", "SeriousEventStatsNumEvents"=>"1", "SeriousEventStatsNumAffected"=>"1"}]}, "SeriousEventOrganSystem"=>"Eye disorders", "SeriousEventAssessmentType"=>"Systematic Assessment", "SeriousEventSourceVocabulary"=>"MedDRA (9.0)"}, {"SeriousEventTerm"=>"Renal failure", "SeriousEventStatsList"=>{"SeriousEventStats"=>[{"SeriousEventStatsGroupId"=>"EG000", "SeriousEventStatsNumAtRisk"=>"328", "SeriousEventStatsNumEvents"=>"2", "SeriousEventStatsNumAffected"=>"2"}]}, "SeriousEventOrganSystem"=>"Renal and urinary disorders", "SeriousEventAssessmentType"=>"Systematic Assessment", "SeriousEventSourceVocabulary"=>"MedDRA (9.0)"}, {"SeriousEventTerm"=>"Blindness", "SeriousEventStatsList"=>{"SeriousEventStats"=>[{"SeriousEventStatsGroupId"=>"EG000", "SeriousEventStatsNumAtRisk"=>"328", "SeriousEventStatsNumEvents"=>"1", "SeriousEventStatsNumAffected"=>"1"}]}, "SeriousEventOrganSystem"=>"Eye disorders", "SeriousEventAssessmentType"=>"Systematic Assessment", "SeriousEventSourceVocabulary"=>"MedDRA (9.0)"}, {"SeriousEventTerm"=>"Benign intracranial hypertension", "SeriousEventStatsList"=>{"SeriousEventStats"=>[{"SeriousEventStatsGroupId"=>"EG000", "SeriousEventStatsNumAtRisk"=>"328", "SeriousEventStatsNumEvents"=>"8", "SeriousEventStatsNumAffected"=>"8"}]}, "SeriousEventOrganSystem"=>"Nervous system disorders", "SeriousEventAssessmentType"=>"Systematic Assessment", "SeriousEventSourceVocabulary"=>"MedDRA (9.0)"}, {"SeriousEventTerm"=>"Gastrointestinal infection", "SeriousEventStatsList"=>{"SeriousEventStats"=>[{"SeriousEventStatsGroupId"=>"EG000", "SeriousEventStatsNumAtRisk"=>"328", "SeriousEventStatsNumEvents"=>"1", "SeriousEventStatsNumAffected"=>"1"}]}, "SeriousEventOrganSystem"=>"Infections and infestations", "SeriousEventAssessmentType"=>"Systematic Assessment", "SeriousEventSourceVocabulary"=>"MedDRA (9.0)"}, {"SeriousEventTerm"=>"Intestinal obstruction", "SeriousEventStatsList"=>{"SeriousEventStats"=>[{"SeriousEventStatsGroupId"=>"EG000", "SeriousEventStatsNumAtRisk"=>"328", "SeriousEventStatsNumEvents"=>"1", "SeriousEventStatsNumAffected"=>"1"}]}, "SeriousEventOrganSystem"=>"Gastrointestinal disorders", "SeriousEventAssessmentType"=>"Systematic Assessment", "SeriousEventSourceVocabulary"=>"MedDRA (9.0)"}]}, "EventsDescription"=>"Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).", "EventsFrequencyThreshold"=>"0"}, "OutcomeMeasuresModule"=>{"OutcomeMeasureList"=>{"OutcomeMeasure"=>[{"OutcomeClassList"=>{"OutcomeClass"=>[{"OutcomeCategoryList"=>{"OutcomeCategory"=>[{"OutcomeMeasurementList"=>{"OutcomeMeasurement"=>[{"OutcomeMeasurementValue"=>"257", "OutcomeMeasurementGroupId"=>"OG000"}]}}]}}]}, "OutcomeDenomList"=>{"OutcomeDenom"=>[{"OutcomeDenomUnits"=>"Participants", "OutcomeDenomCountList"=>{"OutcomeDenomCount"=>[{"OutcomeDenomCountValue"=>"328", "OutcomeDenomCountGroupId"=>"OG000"}]}}]}, "OutcomeGroupList"=>{"OutcomeGroup"=>[{"OutcomeGroupId"=>"OG000", "OutcomeGroupTitle"=>"Cysteamine Topical Solution", "OutcomeGroupDescription"=>"Cysteamine topical solution administered hourly while awake in both eyes\n\nCysteamine"}]}, "OutcomeMeasureType"=>"Primary", "OutcomeMeasureTitle"=>"Number of Participants With Serious and Non-Serious Adverse Events", "OutcomeMeasureParamType"=>"Number", "OutcomeMeasureTimeFrame"=>"Any Time Point up to 27 Years", "OutcomeMeasureDescription"=>"Since efficacy of ophthalmic cysteamine was established and a New Drug Application (NDA) filed, the post-hoc primary outcome measure is the evaluation of safety information. There was no specified time frame for this outcome measure, as safety data was being collected until the drug became available for commercial purchase in May 2013.", "OutcomeMeasureUnitOfMeasure"=>"participants", "OutcomeMeasureReportingStatus"=>"Posted"}, {"OutcomeClassList"=>{"OutcomeClass"=>[{"OutcomeCategoryList"=>{"OutcomeCategory"=>[{"OutcomeMeasurementList"=>{"OutcomeMeasurement"=>[{"OutcomeMeasurementValue"=>"98", "OutcomeMeasurementGroupId"=>"OG000"}]}}]}}]}, "OutcomeDenomList"=>{"OutcomeDenom"=>[{"OutcomeDenomUnits"=>"Participants", "OutcomeDenomCountList"=>{"OutcomeDenomCount"=>[{"OutcomeDenomCountValue"=>"161", "OutcomeDenomCountGroupId"=>"OG000"}]}}, {"OutcomeDenomUnits"=>"eyes", "OutcomeDenomCountList"=>{"OutcomeDenomCount"=>[{"OutcomeDenomCountValue"=>"321", "OutcomeDenomCountGroupId"=>"OG000"}]}}]}, "OutcomeGroupList"=>{"OutcomeGroup"=>[{"OutcomeGroupId"=>"OG000", "OutcomeGroupTitle"=>"Cysteamine Topical Solution", "OutcomeGroupDescription"=>"Cysteamine topical solution administered hourly while awake in both eyes\n\nCysteamine"}]}, "OutcomeMeasureType"=>"Primary", "OutcomeMeasureTitle"=>"Number of Eyes With a Corneal Cystine Crystal Score (CCCS) Response", "OutcomeMeasureParamType"=>"Number", "OutcomeMeasureTimeFrame"=>"Any Time Point Up to 19 Years", "OutcomeMeasureDescription"=>"Response is defined as a decrease from baseline of at least 1 in Corneal Cystine Crystal Score (CCCS) at any time on study when baseline CCCS is greater than or equal to 1, or CCCS does not increase at least 1 at any time on study when baseline CCCS is less than 1.\n\nThe CCCS is based on a library of slit-lamp photographs of corneas with increasing crystal densities (0-3). Slit-lamp photos were to be taken to assess the extent of the corneal crystal accumulation. To minimize bias when assessing the extent of corneal crystal accumulation, photos were centrally graded at the National Eye Institute (NEI) where each photo was graded independently by masked graders. If more than one CCCS was recorded in a given study year, the highest (worst) CCCS value was used for that year.\n\nThe results were obtained from a combined analyses of the NIH cysteamine studies evaluating various cysteamine ophthalmic solution formulations from 1986 through 2005.", "OutcomeMeasureUnitOfMeasure"=>"eyes", "OutcomeMeasureReportingStatus"=>"Posted", "OutcomeMeasureTypeUnitsAnalyzed"=>"eyes", "OutcomeMeasureDenomUnitsSelected"=>"Participants", "OutcomeMeasurePopulationDescription"=>"One hundred sixty-one (161) participants were analyzed in the pre-specified intent-to-treat population [defined as patients who received study medication (between 1986 and 2005), and had a baseline and a post-baseline CCCS value]. After 2005, all participants enrolled received open-label treatment and only safety data was obtained."}]}}, "ParticipantFlowModule"=>{"FlowGroupList"=>{"FlowGroup"=>[{"FlowGroupId"=>"FG000", "FlowGroupTitle"=>"Cysteamine Topical Solution", "FlowGroupDescription"=>"Cysteamine topical solution administered hourly while awake in both eyes\n\nCysteamine"}]}, "FlowPeriodList"=>{"FlowPeriod"=>[{"FlowPeriodTitle"=>"Overall Study", "FlowMilestoneList"=>{"FlowMilestone"=>[{"FlowMilestoneType"=>"STARTED", "FlowAchievementList"=>{"FlowAchievement"=>[{"FlowAchievementGroupId"=>"FG000", "FlowAchievementNumSubjects"=>"328"}]}}, {"FlowMilestoneType"=>"COMPLETED", "FlowAchievementList"=>{"FlowAchievement"=>[{"FlowAchievementGroupId"=>"FG000", "FlowAchievementNumSubjects"=>"310"}]}}, {"FlowMilestoneType"=>"NOT COMPLETED", "FlowAchievementList"=>{"FlowAchievement"=>[{"FlowAchievementGroupId"=>"FG000", "FlowAchievementNumSubjects"=>"18"}]}}]}, "FlowDropWithdrawList"=>{"FlowDropWithdraw"=>[{"FlowReasonList"=>{"FlowReason"=>[{"FlowReasonGroupId"=>"FG000", "FlowReasonNumSubjects"=>"18"}]}, "FlowDropWithdrawType"=>"Death"}]}}]}}, "BaselineCharacteristicsModule"=>{"BaselineDenomList"=>{"BaselineDenom"=>[{"BaselineDenomUnits"=>"Participants", "BaselineDenomCountList"=>{"BaselineDenomCount"=>[{"BaselineDenomCountValue"=>"328", "BaselineDenomCountGroupId"=>"BG000"}]}}]}, "BaselineGroupList"=>{"BaselineGroup"=>[{"BaselineGroupId"=>"BG000", "BaselineGroupTitle"=>"Cysteamine Topical Solution", "BaselineGroupDescription"=>"Cysteamine topical solution administered hourly while awake in both eyes\n\nCysteamine"}]}, "BaselineMeasureList"=>{"BaselineMeasure"=>[{"BaselineClassList"=>{"BaselineClass"=>[{"BaselineCategoryList"=>{"BaselineCategory"=>[{"BaselineMeasurementList"=>{"BaselineMeasurement"=>[{"BaselineMeasurementValue"=>"13.1", "BaselineMeasurementSpread"=>"10.04", "BaselineMeasurementGroupId"=>"BG000"}]}}]}}]}, "BaselineMeasureTitle"=>"Age, Continuous", "BaselineMeasureParamType"=>"Mean", "BaselineMeasureDescription"=>"Although 328 participants were initially enrolled, the mean and standard deviation were only obtained for 322 participants.", "BaselineMeasureUnitOfMeasure"=>"years", "BaselineMeasureDispersionType"=>"Standard Deviation"}, {"BaselineClassList"=>{"BaselineClass"=>[{"BaselineClassTitle"=>"Female", "BaselineCategoryList"=>{"BaselineCategory"=>[{"BaselineMeasurementList"=>{"BaselineMeasurement"=>[{"BaselineMeasurementValue"=>"153", "BaselineMeasurementGroupId"=>"BG000"}]}}]}}, {"BaselineClassTitle"=>"Male", "BaselineCategoryList"=>{"BaselineCategory"=>[{"BaselineMeasurementList"=>{"BaselineMeasurement"=>[{"BaselineMeasurementValue"=>"173", "BaselineMeasurementGroupId"=>"BG000"}]}}]}}]}, "BaselineMeasureTitle"=>"Gender", "BaselineMeasureParamType"=>"Number", "BaselineMeasureDescription"=>"Gender was captured for 326 out of 328 participants, the gender of two participants is unknown.", "BaselineMeasureUnitOfMeasure"=>"participants"}]}}}, "ProtocolSection"=>{"DesignModule"=>{"PhaseList"=>{"Phase"=>["Phase 2"]}, "StudyType"=>"Interventional", "DesignInfo"=>{"DesignAllocation"=>"N/A", "DesignMaskingInfo"=>{"DesignMasking"=>"None (Open Label)"}, "DesignPrimaryPurpose"=>"Treatment", "DesignInterventionModel"=>"Single Group Assignment"}, "EnrollmentInfo"=>{"EnrollmentType"=>"Actual", "EnrollmentCount"=>"328"}}, "StatusModule"=>{"OverallStatus"=>"Completed", "StartDateStruct"=>{"StartDate"=>"April 1986"}, "ExpandedAccessInfo"=>{"HasExpandedAccess"=>"No"}, "StatusVerifiedDate"=>"June 2014", "CompletionDateStruct"=>{"CompletionDate"=>"July 2013", "CompletionDateType"=>"Actual"}, "LastUpdateSubmitDate"=>"June 20, 2014", "StudyFirstSubmitDate"=>"November 3, 1999", "ResultsFirstSubmitDate"=>"June 20, 2014", "StudyFirstSubmitQCDate"=>"November 3, 1999", "LastUpdatePostDateStruct"=>{"LastUpdatePostDate"=>"July 22, 2014", "LastUpdatePostDateType"=>"Estimate"}, "ResultsFirstSubmitQCDate"=>"June 20, 2014", "StudyFirstPostDateStruct"=>{"StudyFirstPostDate"=>"November 4, 1999", "StudyFirstPostDateType"=>"Estimate"}, "ResultsFirstPostDateStruct"=>{"ResultsFirstPostDate"=>"July 22, 2014", "ResultsFirstPostDateType"=>"Estimate"}, "PrimaryCompletionDateStruct"=>{"PrimaryCompletionDate"=>"July 2013", "PrimaryCompletionDateType"=>"Actual"}}, "OutcomesModule"=>{"PrimaryOutcomeList"=>{"PrimaryOutcome"=>[{"PrimaryOutcomeMeasure"=>"Number of Participants With Serious and Non-Serious Adverse Events", "PrimaryOutcomeTimeFrame"=>"Any Time Point up to 27 Years", "PrimaryOutcomeDescription"=>"Since efficacy of ophthalmic cysteamine was established and a New Drug Application (NDA) filed, the post-hoc primary outcome measure is the evaluation of safety information. There was no specified time frame for this outcome measure, as safety data was being collected until the drug became available for commercial purchase in May 2013."}, {"PrimaryOutcomeMeasure"=>"Number of Eyes With a Corneal Cystine Crystal Score (CCCS) Response", "PrimaryOutcomeTimeFrame"=>"Any Time Point Up to 19 Years", "PrimaryOutcomeDescription"=>"Response is defined as a decrease from baseline of at least 1 in Corneal Cystine Crystal Score (CCCS) at any time on study when baseline CCCS is greater than or equal to 1, or CCCS does not increase at least 1 at any time on study when baseline CCCS is less than 1.\n\nThe CCCS is based on a library of slit-lamp photographs of corneas with increasing crystal densities (0-3). Slit-lamp photos were to be taken to assess the extent of the corneal crystal accumulation. To minimize bias when assessing the extent of corneal crystal accumulation, photos were centrally graded at the National Eye Institute (NEI) where each photo was graded independently by masked graders. If more than one CCCS was recorded in a given study year, the highest (worst) CCCS value was used for that year.\n\nThe results were obtained from a combined analyses of the NIH cysteamine studies evaluating various cysteamine ophthalmic solution formulations from 1986 through 2005."}]}}, "OversightModule"=>{"OversightHasDMC"=>"No"}, "ConditionsModule"=>{"KeywordList"=>{"Keyword"=>["Cysteamine Eye Drops", "Nephropathic Cystinosis", "Corneal Crystals", "Benzalkonium", "Toxicity", "Efficacy", "Cystinosis"]}, "ConditionList"=>{"Condition"=>["Cystinosis"]}}, "ReferencesModule"=>{"ReferenceList"=>{"Reference"=>[{"ReferencePMID"=>"3821824", "ReferenceType"=>"background", "ReferenceCitation"=>"Kaiser-Kupfer MI, Fujikawa L, Kuwabara T, Jain S, Gahl WA. Removal of corneal crystals by topical cysteamine in nephropathic cystinosis. N Engl J Med. 1987 Mar 26;316(13):775-9. doi: 10.1056/NEJM198703263161304."}, {"ReferencePMID"=>"2185723", "ReferenceType"=>"background", "ReferenceCitation"=>"Kaiser-Kupfer MI, Gazzo MA, Datiles MB, Caruso RC, Kuehl EM, Gahl WA. A randomized placebo-controlled trial of cysteamine eye drops in nephropathic cystinosis. Arch Ophthalmol. 1990 May;108(5):689-93. doi: 10.1001/archopht.1990.01070070075038."}, {"ReferencePMID"=>"3541536", "ReferenceType"=>"background", "ReferenceCitation"=>"Gahl WA. Cystinosis coming of age. Adv Pediatr. 1986;33:95-126. No abstract available."}, {"ReferencePMID"=>"11001803", "ReferenceType"=>"background", "ReferenceCitation"=>"Gahl WA, Kuehl EM, Iwata F, Lindblad A, Kaiser-Kupfer MI. Corneal crystals in nephropathic cystinosis: natural history and treatment with cysteamine eyedrops. Mol Genet Metab. 2000 Sep-Oct;71(1-2):100-20. doi: 10.1006/mgme.2000.3062."}, {"ReferencePMID"=>"9758713", "ReferenceType"=>"background", "ReferenceCitation"=>"Iwata F, Kuehl EM, Reed GF, McCain LM, Gahl WA, Kaiser-Kupfer MI. A randomized clinical trial of topical cysteamine disulfide (cystamine) versus free thiol (cysteamine) in the treatment of corneal cystine crystals in cystinosis. Mol Genet Metab. 1998 Aug;64(4):237-42. doi: 10.1006/mgme.1998.2725."}]}}, "DescriptionModule"=>{"BriefSummary"=>"Cystinosis is an inherited disease that results in poor growth and kidney disease, among other things. The damage to the kidneys and other organs is thought to be due to accumulation of cystine inside the cells of various body tissues. This chemical also accumulates in the cornea-the covering of the eye over the pupil and iris. After 10 to 20 years, the corneas of some patients become so packed with crystals that the surfaces may become irregular, occasionally causing small, painful breaks.\n\nPatients enrolled in a NIH study on cystinosis are receiving the drug cysteamine. Taken by mouth, this drug reduces cystine in some tissues, but not in the cornea. This study began in 1986 to test whether cysteamine eye drops could prevent or reduce corneal cystine crystals in these patients. The drops have been very effective in removing crystals and reducing pain in patients who take the medication as directed. Patients who do not take the medication as prescribed do not benefit.\n\nAfter the effectiveness of the drops was proven, the main purpose was modified to continue to evaluate the long-term safety and effectiveness of cysteamine eye drops for treating cystine crystals in the corneas of patients with cystinosis until the drops are approved by the Food and Drug Administration (FDA). When the New Drug Application (NDA) for the Sigma-Tau standard formulation is granted, this protocol will be terminated.", "DetailedDescription"=>"Protocol 86-EI-0062 began as a randomized, double-masked, placebo controlled study to evaluate the efficacy and safety of 0.5% topical cysteamine but was subsequently amended as a natural history protocol. Additional protocols conducted at the National Eye Institute (NEI) at the National Institutes of Health (NIH) began after this protocol and tested various formulations of cysteamine topical solution for efficacy and safety in patients with cystinosis. Subjects from these NIH protocols testing various formulations were ultimately transferred to this natural history protocol for open-label treatment once it was established that the formulation within this study was the most effective. All subjects enrolled in this protocol received the most effective cysteamine topical solution formulation in both eyes. The control was defined as the natural course of corneal crystal accumulation in patients with cystinosis. The efficacy data were obtained from all of the studies conducted at NIH evaluating various cysteamine ophthalmic solution formulations from 1986 until 2005. The safety data were collected from 1986 until the termination of this protocol in July 2013.\n\nOBJECTIVE:\n\nThe free thiol cysteamine depletes cystinotic leukocytes and other cells of cystine, whose accumulation is considered the cause of organ damage in cystinosis. This organ damage involves most tissues of the body. Cysteamine therapy improved growth and stabilized renal function in pre-renal transplant cystinosis, without substantial toxicity but there was no noticeable effect on cystine crystal accumulation in the cornea, most likely because of inadequate local cysteamine concentration in the cornea. Previous studies have shown the safety of cysteamine 0.5% topical solution in benzalkonium chloride and its efficacy in resolving the cystine corneal crystals. The main purpose of this protocol is to maintain topical cysteamine treatment in patients with nephropathic cystinosis until the drops are approved by the FDA. When the NDA for the Sigma-Tau standard formulation is granted, the present protocol (86-EI-0062) will be terminated.\n\nSTUDY POPULATION:\n\nUp to 350 adults and children over two years old, who have a confirmed diagnosis of cystinosis will be enrolled.\n\nSTUDY DESIGN:\n\nThis is an open label treatment protocol. Eligible subjects will receive drops of cysteamine 0.5% topical solution in benzalkonium chloride hourly while awake in both eyes. They will undergo an eye examination at their baseline visit. They will take cysteamine eye drops in both eyes every hour during waking hours. They will return to the NIH Clinical Center for a follow-up safety eye examination one year after the baseline visit, and then every two years thereafter until the drug is available commercially.\n\nOUTCOME MEASURES:\n\nThe initial pre-specified primary outcome measure was the reduction of cystine corneal crystals. The post-hoc primary outcome measure (after the protocol was modified) was the collection of safety data."}, "EligibilityModule"=>{"Gender"=>"All", "MinimumAge"=>"2 years", "StdAgeList"=>{"StdAge"=>["Child", "Adult", "Older Adult"]}, "HealthyVolunteers"=>"No", "EligibilityCriteria"=>"INCLUSION CRITERIA:\n\nPatients must have a documented clinical diagnosis of cystinosis.\nPatients should be 2 years old or older.\n\nEXCLUSION CRITERIA:\n\nInability to travel to NIH for a baseline examination, after 1 year, and every two years thereafter for follow-up\nInability to cooperate for slit-lamp examination"}, "IdentificationModule"=>{"NCTId"=>"NCT00001213", "BriefTitle"=>"Cysteamine Eye Drops to Treat Corneal Crystals in Cystinosis", "Organization"=>{"OrgClass"=>"NIH", "OrgFullName"=>"National Institutes of Health Clinical Center (CC)"}, "OfficialTitle"=>"Trial of Topical Cysteamine in the Treatment of Corneal Cystine Crystal Accumulation in Cystinosis", "OrgStudyIdInfo"=>{"OrgStudyId"=>"860062"}, "SecondaryIdInfoList"=>{"SecondaryIdInfo"=>[{"SecondaryId"=>"86-EI-0062"}]}}, "ArmsInterventionsModule"=>{"ArmGroupList"=>{"ArmGroup"=>[{"ArmGroupType"=>"Experimental", "ArmGroupLabel"=>"Cysteamine topical solution", "ArmGroupDescription"=>"Cysteamine topical solution administered hourly while awake in both eyes", "ArmGroupInterventionList"=>{"ArmGroupInterventionName"=>["Drug: Cysteamine"]}}]}, "InterventionList"=>{"Intervention"=>[{"InterventionName"=>"Cysteamine", "InterventionType"=>"Drug", "InterventionArmGroupLabelList"=>{"InterventionArmGroupLabel"=>["Cysteamine topical solution"]}}]}}, "ContactsLocationsModule"=>{"LocationList"=>{"Location"=>[{"LocationZip"=>"20892", "LocationCity"=>"Bethesda", "LocationState"=>"Maryland", "LocationCountry"=>"United States", "LocationFacility"=>"National Institutes of Health Clinical Center, 9000 Rockville Pike"}]}, "OverallOfficialList"=>{"OverallOfficial"=>[{"OverallOfficialName"=>"Rachel J Bishop, M.D.", "OverallOfficialRole"=>"Principal Investigator", "OverallOfficialAffiliation"=>"National Eye Institute (NEI)"}]}}, "SponsorCollaboratorsModule"=>{"LeadSponsor"=>{"LeadSponsorName"=>"National Eye Institute (NEI)", "LeadSponsorClass"=>"NIH"}, "ResponsibleParty"=>{"ResponsiblePartyType"=>"Sponsor"}}}}}}
