Nctid:
NCT00001596
Payload:
{"FullStudy"=>{"Rank"=>473390, "Study"=>{"DerivedSection"=>{"MiscInfoModule"=>{"VersionHolder"=>"December 01, 2023", "RemovedCountryList"=>{"RemovedCountry"=>["Puerto Rico"]}}, "ConditionBrowseModule"=>{"ConditionMeshList"=>{"ConditionMesh"=>[{"ConditionMeshId"=>"D000011658", "ConditionMeshTerm"=>"Pulmonary Fibrosis"}, {"ConditionMeshId"=>"D000000417", "ConditionMeshTerm"=>"Albinism"}, {"ConditionMeshId"=>"D000022861", "ConditionMeshTerm"=>"Hermanski-Pudlak Syndrome"}, {"ConditionMeshId"=>"D000016115", "ConditionMeshTerm"=>"Albinism, Oculocutaneous"}, {"ConditionMeshId"=>"D000010981", "ConditionMeshTerm"=>"Platelet Storage Pool Deficiency"}, {"ConditionMeshId"=>"D000008661", "ConditionMeshTerm"=>"Metabolism, Inborn Errors"}, {"ConditionMeshId"=>"D000005355", "ConditionMeshTerm"=>"Fibrosis"}]}, "ConditionAncestorList"=>{"ConditionAncestor"=>[{"ConditionAncestorId"=>"D000010335", "ConditionAncestorTerm"=>"Pathologic Processes"}, {"ConditionAncestorId"=>"D000017563", 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"ConditionAncestorTerm"=>"Skin Diseases"}, {"ConditionAncestorId"=>"D000025861", "ConditionAncestorTerm"=>"Blood Coagulation Disorders, Inherited"}, {"ConditionAncestorId"=>"D000001778", "ConditionAncestorTerm"=>"Blood Coagulation Disorders"}, {"ConditionAncestorId"=>"D000006402", "ConditionAncestorTerm"=>"Hematologic Diseases"}, {"ConditionAncestorId"=>"D000001791", "ConditionAncestorTerm"=>"Blood Platelet Disorders"}, {"ConditionAncestorId"=>"D000006474", "ConditionAncestorTerm"=>"Hemorrhagic Disorders"}]}, "ConditionBrowseLeafList"=>{"ConditionBrowseLeaf"=>[{"ConditionBrowseLeafId"=>"M8175", "ConditionBrowseLeafName"=>"Fibrosis", "ConditionBrowseLeafAsFound"=>"Fibrosis", "ConditionBrowseLeafRelevance"=>"high"}, {"ConditionBrowseLeafId"=>"M16045", "ConditionBrowseLeafName"=>"Syndrome", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M3454", "ConditionBrowseLeafName"=>"Albinism", "ConditionBrowseLeafAsFound"=>"Albinism", "ConditionBrowseLeafRelevance"=>"high"}, {"ConditionBrowseLeafId"=>"M18283", "ConditionBrowseLeafName"=>"Albinism, Oculocutaneous", "ConditionBrowseLeafAsFound"=>"Oculocutaneous Albinism", "ConditionBrowseLeafRelevance"=>"high"}, {"ConditionBrowseLeafId"=>"M14202", "ConditionBrowseLeafName"=>"Pulmonary Fibrosis", "ConditionBrowseLeafAsFound"=>"Pulmonary Fibrosis", "ConditionBrowseLeafRelevance"=>"high"}, {"ConditionBrowseLeafId"=>"M11331", "ConditionBrowseLeafName"=>"Metabolism, Inborn Errors", "ConditionBrowseLeafAsFound"=>"Inborn Errors of Metabolism", "ConditionBrowseLeafRelevance"=>"high"}, {"ConditionBrowseLeafId"=>"M13561", "ConditionBrowseLeafName"=>"Platelet Storage Pool Deficiency", "ConditionBrowseLeafAsFound"=>"Platelet Storage Pool Deficiency", "ConditionBrowseLeafRelevance"=>"high"}, {"ConditionBrowseLeafId"=>"M22535", "ConditionBrowseLeafName"=>"Hermanski-Pudlak Syndrome", "ConditionBrowseLeafAsFound"=>"Hermansky-Pudlak Syndrome", "ConditionBrowseLeafRelevance"=>"high"}, {"ConditionBrowseLeafId"=>"M10858", "ConditionBrowseLeafName"=>"Lung Diseases", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M19503", "ConditionBrowseLeafName"=>"Lung Diseases, Interstitial", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M14667", "ConditionBrowseLeafName"=>"Respiratory Tract Diseases", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M23376", "ConditionBrowseLeafName"=>"Genetic Diseases, Inborn", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M11329", "ConditionBrowseLeafName"=>"Metabolic Diseases", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M7961", "ConditionBrowseLeafName"=>"Eye Diseases", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M18029", "ConditionBrowseLeafName"=>"Eye Diseases, Hereditary", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M3622", "ConditionBrowseLeafName"=>"Amino Acid Metabolism, Inborn Errors", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M15366", "ConditionBrowseLeafName"=>"Skin Diseases, Genetic", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M15364", "ConditionBrowseLeafName"=>"Skin Diseases", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M19451", "ConditionBrowseLeafName"=>"Hypopigmentation", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M13444", "ConditionBrowseLeafName"=>"Pigmentation Disorders", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M21667", "ConditionBrowseLeafName"=>"Hemostatic Disorders", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M4749", "ConditionBrowseLeafName"=>"Blood Coagulation Disorders", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M22785", "ConditionBrowseLeafName"=>"Blood Coagulation Disorders, Inherited", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M9180", "ConditionBrowseLeafName"=>"Hematologic Diseases", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M4762", "ConditionBrowseLeafName"=>"Blood Platelet Disorders", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M9250", "ConditionBrowseLeafName"=>"Hemorrhagic Disorders", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"T263", "ConditionBrowseLeafName"=>"Albinism", "ConditionBrowseLeafAsFound"=>"Albinism", "ConditionBrowseLeafRelevance"=>"high"}, {"ConditionBrowseLeafId"=>"T4204", "ConditionBrowseLeafName"=>"Oculocutaneous Albinism", "ConditionBrowseLeafAsFound"=>"Oculocutaneous Albinism", "ConditionBrowseLeafRelevance"=>"high"}, {"ConditionBrowseLeafId"=>"T4589", "ConditionBrowseLeafName"=>"Platelet Storage Pool Deficiency", "ConditionBrowseLeafAsFound"=>"Platelet Storage Pool Deficiency", "ConditionBrowseLeafRelevance"=>"high"}, {"ConditionBrowseLeafId"=>"T2785", "ConditionBrowseLeafName"=>"Hermansky-Pudlak Syndrome", "ConditionBrowseLeafAsFound"=>"Hermansky-Pudlak Syndrome", "ConditionBrowseLeafRelevance"=>"high"}, {"ConditionBrowseLeafId"=>"T3036", "ConditionBrowseLeafName"=>"Inborn Amino Acid Metabolism Disorder", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"T2945", "ConditionBrowseLeafName"=>"Hypomelanotic Disorder", "ConditionBrowseLeafRelevance"=>"low"}]}, "ConditionBrowseBranchList"=>{"ConditionBrowseBranch"=>[{"ConditionBrowseBranchName"=>"Symptoms and General Pathology", "ConditionBrowseBranchAbbrev"=>"BC23"}, {"ConditionBrowseBranchName"=>"All Conditions", "ConditionBrowseBranchAbbrev"=>"All"}, {"ConditionBrowseBranchName"=>"Eye Diseases", "ConditionBrowseBranchAbbrev"=>"BC11"}, {"ConditionBrowseBranchName"=>"Diseases and Abnormalities at or Before Birth", "ConditionBrowseBranchAbbrev"=>"BC16"}, {"ConditionBrowseBranchName"=>"Skin and Connective Tissue Diseases", "ConditionBrowseBranchAbbrev"=>"BC17"}, {"ConditionBrowseBranchName"=>"Nutritional and Metabolic Diseases", "ConditionBrowseBranchAbbrev"=>"BC18"}, {"ConditionBrowseBranchName"=>"Respiratory Tract (Lung and Bronchial) Diseases", "ConditionBrowseBranchAbbrev"=>"BC08"}, {"ConditionBrowseBranchName"=>"Blood and Lymph Conditions", "ConditionBrowseBranchAbbrev"=>"BC15"}, {"ConditionBrowseBranchName"=>"Heart and Blood Diseases", "ConditionBrowseBranchAbbrev"=>"BC14"}, {"ConditionBrowseBranchName"=>"Rare Diseases", "ConditionBrowseBranchAbbrev"=>"Rare"}]}}, "InterventionBrowseModule"=>{"InterventionMeshList"=>{"InterventionMesh"=>[{"InterventionMeshId"=>"C000093844", "InterventionMeshTerm"=>"Pirfenidone"}]}, "InterventionAncestorList"=>{"InterventionAncestor"=>[{"InterventionAncestorId"=>"D000000700", "InterventionAncestorTerm"=>"Analgesics"}, {"InterventionAncestorId"=>"D000018689", "InterventionAncestorTerm"=>"Sensory System Agents"}, {"InterventionAncestorId"=>"D000018373", "InterventionAncestorTerm"=>"Peripheral Nervous System Agents"}, {"InterventionAncestorId"=>"D000045505", "InterventionAncestorTerm"=>"Physiological Effects of Drugs"}, {"InterventionAncestorId"=>"D000000894", "InterventionAncestorTerm"=>"Anti-Inflammatory Agents, Non-Steroidal"}, {"InterventionAncestorId"=>"D000018712", "InterventionAncestorTerm"=>"Analgesics, Non-Narcotic"}, {"InterventionAncestorId"=>"D000000893", "InterventionAncestorTerm"=>"Anti-Inflammatory Agents"}, {"InterventionAncestorId"=>"D000018501", "InterventionAncestorTerm"=>"Antirheumatic Agents"}, {"InterventionAncestorId"=>"D000000970", "InterventionAncestorTerm"=>"Antineoplastic Agents"}]}, "InterventionBrowseLeafList"=>{"InterventionBrowseLeaf"=>[{"InterventionBrowseLeafId"=>"M194750", "InterventionBrowseLeafName"=>"Pirfenidone", "InterventionBrowseLeafAsFound"=>"Disturbances", "InterventionBrowseLeafRelevance"=>"high"}, {"InterventionBrowseLeafId"=>"M3722", "InterventionBrowseLeafName"=>"Analgesics", "InterventionBrowseLeafRelevance"=>"low"}, {"InterventionBrowseLeafId"=>"M3907", "InterventionBrowseLeafName"=>"Anti-Inflammatory Agents", "InterventionBrowseLeafRelevance"=>"low"}, {"InterventionBrowseLeafId"=>"M3908", "InterventionBrowseLeafName"=>"Anti-Inflammatory Agents, Non-Steroidal", "InterventionBrowseLeafRelevance"=>"low"}, {"InterventionBrowseLeafId"=>"M20476", "InterventionBrowseLeafName"=>"Analgesics, Non-Narcotic", "InterventionBrowseLeafRelevance"=>"low"}, {"InterventionBrowseLeafId"=>"M20294", "InterventionBrowseLeafName"=>"Antirheumatic Agents", "InterventionBrowseLeafRelevance"=>"low"}]}, "InterventionBrowseBranchList"=>{"InterventionBrowseBranch"=>[{"InterventionBrowseBranchName"=>"Anti-Inflammatory Agents", "InterventionBrowseBranchAbbrev"=>"Infl"}, {"InterventionBrowseBranchName"=>"Antineoplastic Agents", "InterventionBrowseBranchAbbrev"=>"ANeo"}, {"InterventionBrowseBranchName"=>"Antirheumatic Agents", "InterventionBrowseBranchAbbrev"=>"ARhu"}, {"InterventionBrowseBranchName"=>"Analgesics", "InterventionBrowseBranchAbbrev"=>"Analg"}, {"InterventionBrowseBranchName"=>"All Drugs and Chemicals", "InterventionBrowseBranchAbbrev"=>"All"}]}}}, "ResultsSection"=>{"MoreInfoModule"=>{"PointOfContact"=>{"PointOfContactEMail"=>"gahlw@mail.nih.gov", "PointOfContactPhone"=>"301-402-2739", "PointOfContactTitle"=>"Dr. William A. Gahl", "PointOfContactOrganization"=>"National Human Genome Research Institute"}, "CertainAgreement"=>{"AgreementPISponsorEmployee"=>"Yes", "AgreementRestrictiveAgreement"=>"No"}}, "AdverseEventsModule"=>{"EventGroupList"=>{"EventGroup"=>[{"EventGroupId"=>"EG000", "EventGroupTitle"=>"Pirfenidone", "EventGroupDescription"=>"Subjects received pirfenidone 801 mg (3 pills of 267 mg each), three times daily.", "EventGroupOtherNumAtRisk"=>"23", "EventGroupOtherNumAffected"=>"18", "EventGroupSeriousNumAtRisk"=>"23", "EventGroupSeriousNumAffected"=>"6"}, {"EventGroupId"=>"EG001", "EventGroupTitle"=>"Placebo", "EventGroupDescription"=>"Subjects received placebo (3 pills), three times daily.", "EventGroupOtherNumAtRisk"=>"12", "EventGroupOtherNumAffected"=>"9", "EventGroupSeriousNumAtRisk"=>"12", "EventGroupSeriousNumAffected"=>"2"}]}, "OtherEventList"=>{"OtherEvent"=>[{"OtherEventTerm"=>"Abdominal pain", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"23", "OtherEventStatsNumEvents"=>"0", "OtherEventStatsNumAffected"=>"0"}, {"OtherEventStatsGroupId"=>"EG001", "OtherEventStatsNumAtRisk"=>"12", "OtherEventStatsNumEvents"=>"2", "OtherEventStatsNumAffected"=>"2"}]}, "OtherEventOrganSystem"=>"Gastrointestinal disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (11.0)"}, {"OtherEventTerm"=>"Arthalgia", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"23", "OtherEventStatsNumEvents"=>"1", "OtherEventStatsNumAffected"=>"1"}, {"OtherEventStatsGroupId"=>"EG001", "OtherEventStatsNumAtRisk"=>"12", "OtherEventStatsNumEvents"=>"0", "OtherEventStatsNumAffected"=>"0"}]}, "OtherEventOrganSystem"=>"Musculoskeletal and connective tissue disorders", "OtherEventAssessmentType"=>"Non-systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (11.0)"}, {"OtherEventTerm"=>"Chest pain", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"23", "OtherEventStatsNumEvents"=>"3", "OtherEventStatsNumAffected"=>"3"}, {"OtherEventStatsGroupId"=>"EG001", "OtherEventStatsNumAtRisk"=>"12", "OtherEventStatsNumEvents"=>"1", "OtherEventStatsNumAffected"=>"1"}]}, "OtherEventOrganSystem"=>"Cardiac disorders", "OtherEventAssessmentType"=>"Non-systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (11.0)"}, {"OtherEventTerm"=>"Dizziness", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"23", "OtherEventStatsNumEvents"=>"4", "OtherEventStatsNumAffected"=>"4"}, {"OtherEventStatsGroupId"=>"EG001", "OtherEventStatsNumAtRisk"=>"12", "OtherEventStatsNumEvents"=>"0", "OtherEventStatsNumAffected"=>"0"}]}, "OtherEventOrganSystem"=>"Nervous system disorders", "OtherEventAssessmentType"=>"Non-systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (11.0)"}, {"OtherEventTerm"=>"Drug eruption", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"23", "OtherEventStatsNumEvents"=>"2", "OtherEventStatsNumAffected"=>"2"}, {"OtherEventStatsGroupId"=>"EG001", "OtherEventStatsNumAtRisk"=>"12", "OtherEventStatsNumEvents"=>"0", "OtherEventStatsNumAffected"=>"0"}]}, "OtherEventOrganSystem"=>"Immune system disorders", "OtherEventAssessmentType"=>"Non-systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (11.0)"}, {"OtherEventTerm"=>"Dyspepsia", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"23", "OtherEventStatsNumEvents"=>"12", "OtherEventStatsNumAffected"=>"12"}, {"OtherEventStatsGroupId"=>"EG001", "OtherEventStatsNumAtRisk"=>"12", "OtherEventStatsNumEvents"=>"6", "OtherEventStatsNumAffected"=>"6"}]}, "OtherEventOrganSystem"=>"Gastrointestinal disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (11.0)"}, {"OtherEventTerm"=>"Fatigue", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"23", "OtherEventStatsNumEvents"=>"2", "OtherEventStatsNumAffected"=>"2"}, {"OtherEventStatsGroupId"=>"EG001", "OtherEventStatsNumAtRisk"=>"12", "OtherEventStatsNumEvents"=>"0", "OtherEventStatsNumAffected"=>"0"}]}, "OtherEventOrganSystem"=>"General disorders", "OtherEventAssessmentType"=>"Non-systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (11.0)"}, {"OtherEventTerm"=>"Myalgia", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"23", "OtherEventStatsNumEvents"=>"0", "OtherEventStatsNumAffected"=>"0"}, {"OtherEventStatsGroupId"=>"EG001", "OtherEventStatsNumAtRisk"=>"12", "OtherEventStatsNumEvents"=>"1", "OtherEventStatsNumAffected"=>"1"}]}, "OtherEventOrganSystem"=>"Musculoskeletal and connective tissue disorders", "OtherEventAssessmentType"=>"Non-systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (11.0)"}, {"OtherEventTerm"=>"Nausea", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"23", "OtherEventStatsNumEvents"=>"4", "OtherEventStatsNumAffected"=>"4"}, {"OtherEventStatsGroupId"=>"EG001", "OtherEventStatsNumAtRisk"=>"12", "OtherEventStatsNumEvents"=>"1", "OtherEventStatsNumAffected"=>"1"}]}, "OtherEventOrganSystem"=>"Gastrointestinal disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (11.0)"}, {"OtherEventTerm"=>"Palpitations", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"23", "OtherEventStatsNumEvents"=>"3", "OtherEventStatsNumAffected"=>"3"}, {"OtherEventStatsGroupId"=>"EG001", "OtherEventStatsNumAtRisk"=>"12", "OtherEventStatsNumEvents"=>"0", "OtherEventStatsNumAffected"=>"0"}]}, "OtherEventOrganSystem"=>"Cardiac disorders", "OtherEventAssessmentType"=>"Non-systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (11.0)"}, {"OtherEventTerm"=>"Photosensitivity reaction", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"23", "OtherEventStatsNumEvents"=>"2", "OtherEventStatsNumAffected"=>"2"}, {"OtherEventStatsGroupId"=>"EG001", "OtherEventStatsNumAtRisk"=>"12", "OtherEventStatsNumEvents"=>"0", "OtherEventStatsNumAffected"=>"0"}]}, "OtherEventOrganSystem"=>"Skin and subcutaneous tissue disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (11.0)"}, {"OtherEventTerm"=>"Rash", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"23", "OtherEventStatsNumEvents"=>"2", "OtherEventStatsNumAffected"=>"2"}, {"OtherEventStatsGroupId"=>"EG001", "OtherEventStatsNumAtRisk"=>"12", "OtherEventStatsNumEvents"=>"2", "OtherEventStatsNumAffected"=>"2"}]}, "OtherEventOrganSystem"=>"Skin and subcutaneous tissue disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (11.0)"}, {"OtherEventTerm"=>"Somnolence", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"23", "OtherEventStatsNumEvents"=>"1", "OtherEventStatsNumAffected"=>"1"}, {"OtherEventStatsGroupId"=>"EG001", "OtherEventStatsNumAtRisk"=>"12", "OtherEventStatsNumEvents"=>"0", "OtherEventStatsNumAffected"=>"0"}]}, "OtherEventOrganSystem"=>"Nervous system disorders", "OtherEventAssessmentType"=>"Non-systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (11.0)"}, {"OtherEventTerm"=>"Transaminases increased", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"23", "OtherEventStatsNumEvents"=>"1", "OtherEventStatsNumAffected"=>"1"}, {"OtherEventStatsGroupId"=>"EG001", "OtherEventStatsNumAtRisk"=>"12", "OtherEventStatsNumEvents"=>"0", "OtherEventStatsNumAffected"=>"0"}]}, "OtherEventOrganSystem"=>"Investigations", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (11.0)"}, {"OtherEventTerm"=>"Urticaria", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"23", "OtherEventStatsNumEvents"=>"0", "OtherEventStatsNumAffected"=>"0"}, {"OtherEventStatsGroupId"=>"EG001", "OtherEventStatsNumAtRisk"=>"12", "OtherEventStatsNumEvents"=>"1", "OtherEventStatsNumAffected"=>"1"}]}, "OtherEventOrganSystem"=>"Immune system disorders", "OtherEventAssessmentType"=>"Non-systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (11.0)"}, {"OtherEventTerm"=>"Vertigo", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"23", "OtherEventStatsNumEvents"=>"1", "OtherEventStatsNumAffected"=>"1"}, {"OtherEventStatsGroupId"=>"EG001", "OtherEventStatsNumAtRisk"=>"12", "OtherEventStatsNumEvents"=>"0", "OtherEventStatsNumAffected"=>"0"}]}, "OtherEventOrganSystem"=>"Ear and labyrinth disorders", "OtherEventAssessmentType"=>"Non-systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (11.0)"}, {"OtherEventTerm"=>"Vomiting", "OtherEventStatsList"=>{"OtherEventStats"=>[{"OtherEventStatsGroupId"=>"EG000", "OtherEventStatsNumAtRisk"=>"23", "OtherEventStatsNumEvents"=>"0", "OtherEventStatsNumAffected"=>"0"}, {"OtherEventStatsGroupId"=>"EG001", "OtherEventStatsNumAtRisk"=>"12", "OtherEventStatsNumEvents"=>"2", "OtherEventStatsNumAffected"=>"2"}]}, "OtherEventOrganSystem"=>"Gastrointestinal disorders", "OtherEventAssessmentType"=>"Systematic Assessment", "OtherEventSourceVocabulary"=>"MedDRA (11.0)"}]}, "EventsTimeFrame"=>"Adverse event data were collected over the entire period of the double-blind portion of the study, 4 years.", "SeriousEventList"=>{"SeriousEvent"=>[{"SeriousEventTerm"=>"Blood creatinine phosphokinase increased", "SeriousEventStatsList"=>{"SeriousEventStats"=>[{"SeriousEventStatsGroupId"=>"EG000", "SeriousEventStatsNumAtRisk"=>"23", "SeriousEventStatsNumEvents"=>"1", "SeriousEventStatsNumAffected"=>"1"}, {"SeriousEventStatsGroupId"=>"EG001", "SeriousEventStatsNumAtRisk"=>"12", "SeriousEventStatsNumEvents"=>"1", "SeriousEventStatsNumAffected"=>"1"}]}, "SeriousEventOrganSystem"=>"Investigations", "SeriousEventAssessmentType"=>"Non-systematic Assessment", "SeriousEventSourceVocabulary"=>"MedDRA (11.0)"}, {"SeriousEventTerm"=>"Chest pain", "SeriousEventStatsList"=>{"SeriousEventStats"=>[{"SeriousEventStatsGroupId"=>"EG000", "SeriousEventStatsNumAtRisk"=>"23", "SeriousEventStatsNumEvents"=>"2", "SeriousEventStatsNumAffected"=>"2"}, {"SeriousEventStatsGroupId"=>"EG001", "SeriousEventStatsNumAtRisk"=>"12", "SeriousEventStatsNumEvents"=>"0", "SeriousEventStatsNumAffected"=>"0"}]}, "SeriousEventOrganSystem"=>"Cardiac disorders", "SeriousEventAssessmentType"=>"Non-systematic Assessment", "SeriousEventSourceVocabulary"=>"MedDRA (11.0)"}, {"SeriousEventTerm"=>"Deep vein thrombosis", "SeriousEventStatsList"=>{"SeriousEventStats"=>[{"SeriousEventStatsGroupId"=>"EG000", "SeriousEventStatsNumAtRisk"=>"23", "SeriousEventStatsNumEvents"=>"1", "SeriousEventStatsNumAffected"=>"1"}, {"SeriousEventStatsGroupId"=>"EG001", "SeriousEventStatsNumAtRisk"=>"12", "SeriousEventStatsNumEvents"=>"0", "SeriousEventStatsNumAffected"=>"0"}]}, "SeriousEventOrganSystem"=>"Vascular disorders", "SeriousEventAssessmentType"=>"Non-systematic Assessment", "SeriousEventSourceVocabulary"=>"MedDRA (11.0)"}, {"SeriousEventTerm"=>"Haematochezia", "SeriousEventStatsList"=>{"SeriousEventStats"=>[{"SeriousEventStatsGroupId"=>"EG000", "SeriousEventStatsNumAtRisk"=>"23", "SeriousEventStatsNumEvents"=>"1", "SeriousEventStatsNumAffected"=>"1"}, {"SeriousEventStatsGroupId"=>"EG001", "SeriousEventStatsNumAtRisk"=>"12", "SeriousEventStatsNumEvents"=>"0", "SeriousEventStatsNumAffected"=>"0"}]}, "SeriousEventOrganSystem"=>"Gastrointestinal disorders", "SeriousEventAssessmentType"=>"Non-systematic Assessment", "SeriousEventSourceVocabulary"=>"MedDRA (11.0)"}, {"SeriousEventTerm"=>"Respiratory failure", "SeriousEventStatsList"=>{"SeriousEventStats"=>[{"SeriousEventStatsGroupId"=>"EG000", "SeriousEventStatsNumAtRisk"=>"23", "SeriousEventStatsNumEvents"=>"2", "SeriousEventStatsNumAffected"=>"2"}, {"SeriousEventStatsGroupId"=>"EG001", "SeriousEventStatsNumAtRisk"=>"12", "SeriousEventStatsNumEvents"=>"1", "SeriousEventStatsNumAffected"=>"1"}]}, "SeriousEventOrganSystem"=>"Respiratory, thoracic and mediastinal disorders", "SeriousEventAssessmentType"=>"Non-systematic Assessment", "SeriousEventSourceVocabulary"=>"MedDRA (11.0)"}]}, "EventsFrequencyThreshold"=>"0"}, "OutcomeMeasuresModule"=>{"OutcomeMeasureList"=>{"OutcomeMeasure"=>[{"OutcomeClassList"=>{"OutcomeClass"=>[{"OutcomeCategoryList"=>{"OutcomeCategory"=>[{"OutcomeMeasurementList"=>{"OutcomeMeasurement"=>[{"OutcomeMeasurementValue"=>"-23.52", "OutcomeMeasurementSpread"=>"24.32", "OutcomeMeasurementGroupId"=>"OG000"}, {"OutcomeMeasurementValue"=>"-20.93", "OutcomeMeasurementSpread"=>"25.59", "OutcomeMeasurementGroupId"=>"OG001"}]}}]}}]}, "OutcomeDenomList"=>{"OutcomeDenom"=>[{"OutcomeDenomUnits"=>"Participants", "OutcomeDenomCountList"=>{"OutcomeDenomCount"=>[{"OutcomeDenomCountValue"=>"11", "OutcomeDenomCountGroupId"=>"OG000"}, {"OutcomeDenomCountValue"=>"3", "OutcomeDenomCountGroupId"=>"OG001"}]}}]}, "OutcomeGroupList"=>{"OutcomeGroup"=>[{"OutcomeGroupId"=>"OG000", "OutcomeGroupTitle"=>"Pirfenidone", "OutcomeGroupDescription"=>"Subjects received pirfenidone 801 mg (3 pills of 267 mg each), three times daily."}, {"OutcomeGroupId"=>"OG001", "OutcomeGroupTitle"=>"Placebo", 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"BaselineGroupList"=>{"BaselineGroup"=>[{"BaselineGroupId"=>"BG000", "BaselineGroupTitle"=>"Pirfenidone", "BaselineGroupDescription"=>"Subjects received pirfenidone 801 mg (3 pills of 267 mg each), three times daily."}, {"BaselineGroupId"=>"BG001", "BaselineGroupTitle"=>"Placebo", "BaselineGroupDescription"=>"Subjects received placebo (3 pills), three times daily."}, {"BaselineGroupId"=>"BG002", "BaselineGroupTitle"=>"Total", "BaselineGroupDescription"=>"Total of all reporting groups"}]}, "BaselineMeasureList"=>{"BaselineMeasure"=>[{"BaselineClassList"=>{"BaselineClass"=>[{"BaselineCategoryList"=>{"BaselineCategory"=>[{"BaselineCategoryTitle"=>"<=18 years", "BaselineMeasurementList"=>{"BaselineMeasurement"=>[{"BaselineMeasurementValue"=>"0", "BaselineMeasurementGroupId"=>"BG000"}, {"BaselineMeasurementValue"=>"0", "BaselineMeasurementGroupId"=>"BG001"}, {"BaselineMeasurementValue"=>"0", "BaselineMeasurementGroupId"=>"BG002"}]}}, {"BaselineCategoryTitle"=>"Between 18 and 65 years", 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{"BaselineClassList"=>{"BaselineClass"=>[{"BaselineCategoryList"=>{"BaselineCategory"=>[{"BaselineMeasurementList"=>{"BaselineMeasurement"=>[{"BaselineMeasurementValue"=>"72.77", "BaselineMeasurementSpread"=>"8.12", "BaselineMeasurementGroupId"=>"BG000"}, {"BaselineMeasurementValue"=>"73.53", "BaselineMeasurementSpread"=>"10.09", "BaselineMeasurementGroupId"=>"BG001"}, {"BaselineMeasurementValue"=>"73.03", "BaselineMeasurementSpread"=>"8.70", "BaselineMeasurementGroupId"=>"BG002"}]}}]}}]}, "BaselineMeasureTitle"=>"Forced Vital Capacity", "BaselineMeasureParamType"=>"Mean", "BaselineMeasureDescription"=>"Forced Vital Capacity (FVC) is the volume of air that can be forcibly blown out from the lungs after full inspiration. It is recorded as the percentage of predicted volume (predicted FVC volume is calculated based on subject's height, age, sex, and weight). FVC normal range is 80-120% of predicted.", "BaselineMeasureUnitOfMeasure"=>"% of predicted", "BaselineMeasureDispersionType"=>"Standard Deviation"}, {"BaselineClassList"=>{"BaselineClass"=>[{"BaselineCategoryList"=>{"BaselineCategory"=>[{"BaselineMeasurementList"=>{"BaselineMeasurement"=>[{"BaselineMeasurementValue"=>"73.84", "BaselineMeasurementSpread"=>"12.07", "BaselineMeasurementGroupId"=>"BG000"}, {"BaselineMeasurementValue"=>"73.28", "BaselineMeasurementSpread"=>"10.09", "BaselineMeasurementGroupId"=>"BG001"}, {"BaselineMeasurementValue"=>"73.65", "BaselineMeasurementSpread"=>"11.28", "BaselineMeasurementGroupId"=>"BG002"}]}}]}}]}, "BaselineMeasureTitle"=>"Total Lung Capacity", "BaselineMeasureParamType"=>"Mean", "BaselineMeasureDescription"=>"Total Lung Capacity (TLC) is the volume in the lungs at maximal inflation. TLC is recorded as the percentage of predicted volume based on subject's height, age, sex, and weight. TLC normal range is 80-120% of predicted.", "BaselineMeasureUnitOfMeasure"=>"% of predicted volume", "BaselineMeasureDispersionType"=>"Standard Deviation"}, {"BaselineClassList"=>{"BaselineClass"=>[{"BaselineCategoryList"=>{"BaselineCategory"=>[{"BaselineMeasurementList"=>{"BaselineMeasurement"=>[{"BaselineMeasurementValue"=>"67.82", "BaselineMeasurementSpread"=>"15.94", "BaselineMeasurementGroupId"=>"BG000"}, {"BaselineMeasurementValue"=>"66.83", "BaselineMeasurementSpread"=>"16.60", "BaselineMeasurementGroupId"=>"BG001"}, {"BaselineMeasurementValue"=>"67.48", "BaselineMeasurementSpread"=>"15.93", "BaselineMeasurementGroupId"=>"BG002"}]}}]}}]}, "BaselineMeasureTitle"=>"Adjusted Diffusing Capacity of the Lung for Carbon Monoxide", "BaselineMeasureParamType"=>"Mean", "BaselineMeasureDescription"=>"Adjusted Diffusing Capacity of the lung for carbon monoxide (DLCOa) measures gas uptake during a single inspiration in a standard time, adjusted for subject's hemoglobin levels. DLCOa is recorded as a percentage of predicted volume, and normal range is 75-125% of predicted.", "BaselineMeasureUnitOfMeasure"=>"% of predicted volume", "BaselineMeasureDispersionType"=>"Standard Deviation"}, {"BaselineClassList"=>{"BaselineClass"=>[{"BaselineCategoryList"=>{"BaselineCategory"=>[{"BaselineMeasurementList"=>{"BaselineMeasurement"=>[{"BaselineMeasurementValue"=>"517.39", "BaselineMeasurementSpread"=>"112.79", "BaselineMeasurementGroupId"=>"BG000"}, {"BaselineMeasurementValue"=>"526.87", "BaselineMeasurementSpread"=>"103.00", "BaselineMeasurementGroupId"=>"BG001"}, {"BaselineMeasurementValue"=>"520.65", "BaselineMeasurementSpread"=>"107.94", "BaselineMeasurementGroupId"=>"BG002"}]}}]}}]}, "BaselineMeasureTitle"=>"6 Minute Walk Test", "BaselineMeasureParamType"=>"Mean", "BaselineMeasureDescription"=>"6 minute walk test (6MWT) measures the distance that a patient can quickly walk on a flat hard surface in a period of six minutes.", "BaselineMeasureUnitOfMeasure"=>"meters", "BaselineMeasureDispersionType"=>"Standard Deviation"}]}}}, "ProtocolSection"=>{"DesignModule"=>{"PhaseList"=>{"Phase"=>["Phase 2"]}, "StudyType"=>"Interventional", "DesignInfo"=>{"DesignAllocation"=>"Randomized", "DesignMaskingInfo"=>{"DesignMasking"=>"Quadruple", "DesignWhoMaskedList"=>{"DesignWhoMasked"=>["Participant", "Care Provider", "Investigator", "Outcomes Assessor"]}}, "DesignPrimaryPurpose"=>"Treatment", "DesignInterventionModel"=>"Parallel Assignment"}, "EnrollmentInfo"=>{"EnrollmentType"=>"Actual", "EnrollmentCount"=>"35"}}, "StatusModule"=>{"OverallStatus"=>"Completed", "StartDateStruct"=>{"StartDate"=>"September 2005"}, "ExpandedAccessInfo"=>{"HasExpandedAccess"=>"No"}, "StatusVerifiedDate"=>"September 2017", "CompletionDateStruct"=>{"CompletionDate"=>"May 9, 2016", "CompletionDateType"=>"Actual"}, "LastUpdateSubmitDate"=>"September 14, 2017", "StudyFirstSubmitDate"=>"November 3, 1999", "ResultsFirstSubmitDate"=>"December 12, 2011", "StudyFirstSubmitQCDate"=>"November 3, 1999", "LastUpdatePostDateStruct"=>{"LastUpdatePostDate"=>"October 16, 2017", "LastUpdatePostDateType"=>"Actual"}, "ResultsFirstSubmitQCDate"=>"January 24, 2012", "StudyFirstPostDateStruct"=>{"StudyFirstPostDate"=>"November 4, 1999", "StudyFirstPostDateType"=>"Estimate"}, "ResultsFirstPostDateStruct"=>{"ResultsFirstPostDate"=>"January 26, 2012", "ResultsFirstPostDateType"=>"Estimate"}, "PrimaryCompletionDateStruct"=>{"PrimaryCompletionDate"=>"September 2009", "PrimaryCompletionDateType"=>"Actual"}}, "OutcomesModule"=>{"PrimaryOutcomeList"=>{"PrimaryOutcome"=>[{"PrimaryOutcomeMeasure"=>"Change in Forced Vital Capacity (36 Months)", "PrimaryOutcomeTimeFrame"=>"Measured at baseline and 36 months", "PrimaryOutcomeDescription"=>"Change from baseline in the Forced Vital Capacity (FVC) measurement at 36 months. FVC is the volume of air that can be forcibly blown out from the lungs after full inspiration. FVC is recorded as the percentage of predicted volume (predicted FVC volume is calculated based on subject's height, age, sex, and weight)."}]}, "SecondaryOutcomeList"=>{"SecondaryOutcome"=>[{"SecondaryOutcomeMeasure"=>"Change in Forced Vital Capacity (12 Months)", "SecondaryOutcomeTimeFrame"=>"Measured at baseline and 12 months", "SecondaryOutcomeDescription"=>"Change from baseline in the Forced Vital Capacity (FVC) measurement at 12 months. FVC is the volume of air that can be forcibly blown out from the lungs after full inspiration. FVC is recorded as the percentage of predicted volume (predicted FVC volume is calculated based on subject's height, age, sex, and weight)."}, {"SecondaryOutcomeMeasure"=>"Change in Total Lung Capacity (36 Months)", "SecondaryOutcomeTimeFrame"=>"Measured at baseline and 36 months", "SecondaryOutcomeDescription"=>"Change from baseline in Total Lung Capacity (TLC) measured at 36 months. TLC is the volume in the lungs at maximal inflation. TLC is recorded as the percentage of predicted volume based on subject's height, age, sex, and weight."}, {"SecondaryOutcomeMeasure"=>"Change in Total Lung Capacity (12 Months)", "SecondaryOutcomeTimeFrame"=>"Measured at baseline and 12 months", "SecondaryOutcomeDescription"=>"Change from baseline in Total Lung Capacity (TLC) measured at 12 months. TLC is the volume in the lungs at maximal inflation. TLC is recorded as the percentage of predicted volume based on subject's height, age, sex, and weight."}, {"SecondaryOutcomeMeasure"=>"Change in Adjusted Diffusing Capacity of the Lung for Carbon Monoxide (36 Months)", "SecondaryOutcomeTimeFrame"=>"Measured at baseline and 36 months", "SecondaryOutcomeDescription"=>"Change from baseline in adjusted Diffusing Capacity of the lung for carbon monoxide (DLCOa) measured at 36 months. DLCOa measures gas uptake during a single inspiration in a standard time, adjusted for subject's hemoglobin levels."}, {"SecondaryOutcomeMeasure"=>"Change in Adjusted Diffusing Capacity of the Lung for Carbon Monoxide (12 Months)", "SecondaryOutcomeTimeFrame"=>"Measured at baseline and 12 months", "SecondaryOutcomeDescription"=>"Change from baseline in adjusted Diffusing Capacity of the lung for carbon monoxide (DLCOa) measured at 12 months. DLCOa measures gas uptake during a single inspiration in a standard time, adjusted for subject's hemoglobin levels."}, {"SecondaryOutcomeMeasure"=>"Change in 6 Minute Walk Test (36 Months)", "SecondaryOutcomeTimeFrame"=>"Measured at baseline and 36 months", "SecondaryOutcomeDescription"=>"Change from baseline of the 6 minute walk test (6MWT) at 36 months. The 6MWT measures the distance that a patient can quickly walk on a flat hard surface in a period of six minutes."}, {"SecondaryOutcomeMeasure"=>"Change in 6 Minute Walk Test (12 Months)", "SecondaryOutcomeTimeFrame"=>"Measured at baseline and 12 months", "SecondaryOutcomeDescription"=>"Change from baseline of the 6 minute walk test (6MWT) at 12 months. The 6MWT measures the distance that a patient can quickly walk on a flat hard surface in a period of six minutes."}]}}, "OversightModule"=>{"OversightHasDMC"=>"Yes"}, "ConditionsModule"=>{"KeywordList"=>{"Keyword"=>["Albinism", "Platelet Storage Pool Deficiency", "Pulmonary Fibrosis", "Hermansky-Pudlak Syndrome"]}, "ConditionList"=>{"Condition"=>["Albinism", "Inborn Errors of Metabolism", "Oculocutaneous Albinism", "Platelet Storage Pool Deficiency", "Pulmonary Fibrosis"]}}, "ReferencesModule"=>{"ReferenceList"=>{"Reference"=>[{"ReferencePMID"=>"11455388", "ReferenceType"=>"background", "ReferenceCitation"=>"Anikster Y, Huizing M, White J, Shevchenko YO, Fitzpatrick DL, Touchman JW, Compton JG, Bale SJ, Swank RT, Gahl WA, Toro JR. Mutation of a new gene causes a unique form of Hermansky-Pudlak syndrome in a genetic isolate of central Puerto Rico. Nat Genet. 2001 Aug;28(4):376-80. doi: 10.1038/ng576."}, {"ReferencePMID"=>"9562579", "ReferenceType"=>"background", "ReferenceCitation"=>"Gahl WA, Brantly M, Kaiser-Kupfer MI, Iwata F, Hazelwood S, Shotelersuk V, Duffy LF, Kuehl EM, Troendle J, Bernardini I. Genetic defects and clinical characteristics of patients with a form of oculocutaneous albinism (Hermansky-Pudlak syndrome). N Engl J Med. 1998 Apr 30;338(18):1258-64. doi: 10.1056/NEJM199804303381803."}, {"ReferencePMID"=>"12126938", "ReferenceType"=>"background", "ReferenceCitation"=>"Gahl WA, Brantly M, Troendle J, Avila NA, Padua A, Montalvo C, Cardona H, Calis KA, Gochuico B. Effect of pirfenidone on the pulmonary fibrosis of Hermansky-Pudlak syndrome. Mol Genet Metab. 2002 Jul;76(3):234-42. doi: 10.1016/s1096-7192(02)00044-6."}]}, "SeeAlsoLinkList"=>{"SeeAlsoLink"=>[{"SeeAlsoLinkURL"=>"http://clinicalstudies.info.nih.gov/cgi/detail.cgi?B_1997-HG-0085.html", "SeeAlsoLinkLabel"=>"NIH Clinical Center Detailed Web Page"}]}}, "DescriptionModule"=>{"BriefSummary"=>"Hermansky-Pudlak Syndrome (HPS) is an inherited disease that results in decreased pigmentation (oculocutaneous albinism), bleeding problems due to a platelet abnormality (platelet storage pool defect), and storage of an abnormal fat-protein compound (lysosomal accumulation of ceroid lipofuscin).\n\nThe disease can cause poor functioning of the lungs, intestine, kidneys, or heart. The most serious complication of the disease is pulmonary fibrosis and typically causes death in patients 40 - 50 years old. The disorder is common in Puerto Rico, where many of the clinical research studies on the disease have been conducted. Neither the full extent of the disease nor the basic cause of the disease is known. There is no known treatment for HPS.\n\nThe drug pirfenidone blocks the biochemical process of inflammation and has been reported to slow or reverse pulmonary fibrosis in animal systems.\n\nIn this study researchers will select up to 40 HPS patients diagnosed with pulmonary fibrosis. The patients will be randomly divided into 2 groups. The patients will not know if they are taking pirfenidone or a placebo \"sugar pill\".\n\nGroup one will be patients who will receive pirfenidone.\nGroup two will be patients who will receive a placebo \"sugar pill\"\n\nThe major outcome measurement of the therapy will be a change in the lung function (forced vital capacity). The study will be stopped if one therapy proves to be more effective than the other.", "DetailedDescription"=>"Hermansky-Pudlak Syndrome (HPS) is a rare autosomal recessive disease consisting of oculocutaneous albinism and a platelet storage pool defect. The most serious complication of this disorder, which is common in Puerto Rico, is pulmonary fibrosis, generally fatal in the fourth or fifth decade. There is no treatment for the pulmonary disease of HPS, which resembles idiopathic pulmonary fibrosis. However, a drug called pirfenidone has antifibrotic effects in animal models of lung fibrosis. Pirfenidone is an IND drug initially provided by Marnac, Inc.; InterMune, Inc., now holds the license. Pirfenidone inhibits cytokine-induced inflammation. Reported side effects include gastrointestinal upset, a photosensitivity rash, and palpitations. Between 1997 and 2001, we performed a randomized, placebo-controlled trial under this protocol that found pirfenidone to be safe and efficacious when analyzed using a repeated measures model. Using a random coefficients model, however, the data were definitive only in the restricted group of subjects whose initial forced vital capacity was greater than 50% of predicted. Because the repeated measures analysis had been chosen a priori as the optimal model, the DSMB stopped the study and directed that all patients receive pirfenidone. (Of the 23 original patients, 3 are still receiving pirfenidone under this protocol.)\n\nNow, to prove efficacy of pirfenidone, we are conducting a block-randomized, placebo-controlled, double-blind trial involving up to 40 HPS patients whose forced vital capacity is 51-85% of predicted. For every patient randomly assigned to the placebo group, two will receive pirfenidone. Patients are largely drawn from the Puerto Rican population and are simultaneously enrolled in clinical protocol 95-HG-193. They are admitted to the NIH Clinical Center for 2-3 day admissions every 4 months. The primary efficacy variable is change in forced vital capacity, determined on every admission. Secondary efficacy variables are also examined. A CT scan of the chest and bone densitometry are performed. After 4 years of patient accrual, 35 patients were enrolled; the original statistical analysis plan (SAP) called for 39 patients to be enrolled within one year. The NHGRI DSMB revised the original SAP to perform an interim data analysis 12 months after 30 patients were enrolled, i.e., in May of 2009. That analysis directed the study to stop due to futility. However, this protocol will continue to provide pirfenidone to the three original protocol patients still enrolled, and to any pirfenidone-treated patients who choose to undergo pulmonary lavage to help us determine the effects of pirfenidone on the cytokine profile of alveolar macrophages. The lavages would require enrollment in a separate protocol. The treatment drug will be stopped immediately for all placebo patients and for pirfenidone patients who do not plan to enroll in the lavage protocol. Pirfenidone treatment will stop just after the lavage is performed on patients who do enroll in the lavage protocol, 04-HG-0211. All patients will be invited to continue to come to the NIH annually under the HPS natural history protocol, 95-HG-0193."}, "EligibilityModule"=>{"Gender"=>"All", "MinimumAge"=>"18 years", "StdAgeList"=>{"StdAge"=>["Adult", "Older Adult"]}, "HealthyVolunteers"=>"No", "EligibilityCriteria"=>"INCLUSION CRITERIA\n\nFor the portion of the protocol involving continuations of pirfenidone treatment, the criteria are simply previous enrollment in 97-HG-0085.\n\nFor enrollment in the new clinical trial, the inclusion criteria involve enrollment in protocol 95-HG-0193, \"Clinical and Basic Investigations into Hermansky-Pudlak Syndrome\". This itself requires a diagnosis of HPS based upon molecular grounds or the electron microscopic demonstration of deficiency of platelet dense bodies. In addition, for protocol 97-HG-0085, patients must:\n\nBe over 18 years of age.\nHave an FVC greater than 50 percent and less than or equal to 85 percent of predicted OR a hemoglobin-corrected DL(co) greater than 35 percent and less than or equal to 80 percent of predicted, with no evidence of a pulmonary embolism.\nHave evidence of reduced exercise tolerance lasting longer than one week on either the St. George's Hospital Respiratory Questionnaire or the Dyspnea Perception Scale.\nFEV(1)/FVC greater than 80 percent of predicted after bronchodilators.\nNo evidence of improvement in pulmonary fibrosis within the past year defined as an FVC increased by 10 percent or a DL(co) increased by 15 percent.\nDistance walked greater than or equal to 150 meters (492 feet) with oxygen saturation greater than or equal to 83 percent on less than or equal to 6 L/min. of oxygen during the 6-Minute Walk Test (6MWT).\nBe available, willing, and able to come to the NIH Clinical Center for admission every 4 months for three years.\n\nEXCLUSION CRITERIA\n\nHistory of clinically significant environmental exposure known to cause pulmonary fibrosis (including but not limited to drugs, asbestos, beryllium, radiation, domestic birds).\nAn explanation for interstitial lung disease other than HPS, including but not limited to radiation, sarcoidosis, hypersensitivity pneumonitis, bronchiolitis obliterans organizing pneumonia, cancer.\nDiagnosis of any connective tissue disease including but not limited to scleroderma, systemic lupus erythematosus, rheumatoid arthritis.\nListing on a lung transplantation waiting list.\nPregnancy or lactation\nCigarette smoking in the past 6 months\nHistory of ethanol abuse or recreational drug use in the past two years\nHistory of human immunodeficiency virus (HIV) or chronic viral hepatitis infection\nChronic use of high-dose steroids (greater than 10 mg prednisone/day)\nPrior use of pirfenidone\nUse of any of the following within 28 days of enrollment: investigational therapy, cytotoxic/immunosuppressive agents other than corticosteroids (including but not limited to azathioprine, cyclosphosphamide, methotrexate, cyclosporine); cytokine modulators (including but not limited to etanercept and infliximab); therapies targeted to treat pulmonary fibrosis (including but not limited to D-penicillamine, colchicine, interferon gamma-1b, bosentan, N-acetylcysteine\nAny severe medical complication including but not be limited to uncontrolled seizures, repeated transient ischemic attacks, abnormal mental status, severe ataxia, uncontrolled migraine headaches, diplopia, repeated episodes of syncope, untreated clinical depression, recent myocardial infarction (past 6 months), unstable angina, clinically relevant arrhythmias, uncontrolled hypotension or hypertension (systolic blood pressure less than 80 or greater than 180 mm Hg), myocarditis, hepatomegaly (liver greater than 3 cm below the right costal margin), renal glomerular impairment (creatinine clearance less than 35 ml/min/1.73 m2, pancreatitis, toxic thyroiditis, malignancy (except basal cell carcinoma)\nMedications with a high frequency of life threatening side effects\nSignificant laboratory abnormalities, including but not limited to serum potassium less than 3.0 or greater than 5.4 mEq/L, SGPT greater than 100 U/L, CK greater than 700 U/L, hemoglobin less than 9.0 g/dL, platelets less than 70 k/mm3, leucocyte count less than 2.0 k/microliter, or cholesterol greater than 400 mg/dL.\nFor women of child bearing age, failure to have an effective method of birth control."}, "IdentificationModule"=>{"NCTId"=>"NCT00001596", "BriefTitle"=>"Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome", "Organization"=>{"OrgClass"=>"NIH", "OrgFullName"=>"National Institutes of Health Clinical Center (CC)"}, "OfficialTitle"=>"Therapeutic Clinical Trial of Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome", "OrgStudyIdInfo"=>{"OrgStudyId"=>"970085"}, "SecondaryIdInfoList"=>{"SecondaryIdInfo"=>[{"SecondaryId"=>"97-HG-0085"}]}}, "ArmsInterventionsModule"=>{"ArmGroupList"=>{"ArmGroup"=>[{"ArmGroupType"=>"Active Comparator", "ArmGroupLabel"=>"Pirfenidone", "ArmGroupDescription"=>"Subjects received pirfenidone 801 mg (3 pills of 267 mg each), three times daily.", "ArmGroupInterventionList"=>{"ArmGroupInterventionName"=>["Drug: Pirfenidone"]}}, {"ArmGroupType"=>"Placebo Comparator", "ArmGroupLabel"=>"Placebo", "ArmGroupDescription"=>"Subjects received placebo (3 pills), three times daily.", "ArmGroupInterventionList"=>{"ArmGroupInterventionName"=>["Drug: Placebo"]}}]}, "InterventionList"=>{"Intervention"=>[{"InterventionName"=>"Pirfenidone", "InterventionType"=>"Drug", "InterventionDescription"=>"Treatment with pirfenidone 801 mg (3 pills of 267 mg each), three times daily.", "InterventionOtherNameList"=>{"InterventionOtherName"=>["Deskar", "Esbriet"]}, "InterventionArmGroupLabelList"=>{"InterventionArmGroupLabel"=>["Pirfenidone"]}}, {"InterventionName"=>"Placebo", "InterventionType"=>"Drug", "InterventionDescription"=>"Placebo (3 pills), three times daily.", "InterventionOtherNameList"=>{"InterventionOtherName"=>["Inactive matching placebo"]}, "InterventionArmGroupLabelList"=>{"InterventionArmGroupLabel"=>["Placebo"]}}]}}, "ContactsLocationsModule"=>{"LocationList"=>{"Location"=>[{"LocationZip"=>"20892", "LocationCity"=>"Bethesda", "LocationState"=>"Maryland", "LocationCountry"=>"United States", "LocationFacility"=>"National Institutes of Health Clinical Center, 9000 Rockville Pike"}]}}, "SponsorCollaboratorsModule"=>{"LeadSponsor"=>{"LeadSponsorName"=>"William Gahl, M.D.", "LeadSponsorClass"=>"NIH"}, "CollaboratorList"=>{"Collaborator"=>[{"CollaboratorName"=>"National Human Genome Research Institute (NHGRI)", "CollaboratorClass"=>"NIH"}]}, "ResponsibleParty"=>{"ResponsiblePartyType"=>"Sponsor-Investigator", "ResponsiblePartyInvestigatorTitle"=>"Clinical Director, National Human Genome Research Institute", "ResponsiblePartyInvestigatorFullName"=>"William Gahl, M.D.", "ResponsiblePartyInvestigatorAffiliation"=>"National Human Genome Research Institute (NHGRI)"}}}}}}