Nctid:
NCT00001151
Payload:
{"hasResults"=>true, "derivedSection"=>{"miscInfoModule"=>{"versionHolder"=>"2024-10-21"}, "conditionBrowseModule"=>{"meshes"=>[{"id"=>"D000012279", "term"=>"Rickets"}, {"id"=>"D000006996", "term"=>"Hypocalcemia"}], "ancestors"=>[{"id"=>"D000002128", "term"=>"Calcium Metabolism Disorders"}, {"id"=>"D000008659", "term"=>"Metabolic Diseases"}, {"id"=>"D000014883", "term"=>"Water-Electrolyte Imbalance"}, {"id"=>"D000001851", "term"=>"Bone Diseases, Metabolic"}, {"id"=>"D000001847", "term"=>"Bone Diseases"}, {"id"=>"D000009140", "term"=>"Musculoskeletal Diseases"}, {"id"=>"D000014808", "term"=>"Vitamin D Deficiency"}, {"id"=>"D000001361", "term"=>"Avitaminosis"}, {"id"=>"D000003677", "term"=>"Deficiency Diseases"}, {"id"=>"D000044342", "term"=>"Malnutrition"}, {"id"=>"D000009748", "term"=>"Nutrition Disorders"}], "browseLeaves"=>[{"id"=>"M15108", "name"=>"Rickets", "asFound"=>"Rickets", "relevance"=>"HIGH"}, {"id"=>"M10046", "name"=>"Hypocalcemia", "asFound"=>"Hypocalcemia", "relevance"=>"HIGH"}, {"id"=>"M11639", "name"=>"Metabolic Diseases", "relevance"=>"LOW"}, {"id"=>"M5391", "name"=>"Calcium Metabolism Disorders", "relevance"=>"LOW"}, {"id"=>"M17624", "name"=>"Water-Electrolyte Imbalance", "relevance"=>"LOW"}, {"id"=>"M5126", "name"=>"Bone Diseases", "relevance"=>"LOW"}, {"id"=>"M5130", "name"=>"Bone Diseases, Metabolic", "relevance"=>"LOW"}, {"id"=>"M12097", "name"=>"Musculoskeletal Diseases", "relevance"=>"LOW"}, {"id"=>"M17551", "name"=>"Vitamin D Deficiency", "relevance"=>"LOW"}, {"id"=>"M4660", "name"=>"Avitaminosis", "relevance"=>"LOW"}, {"id"=>"M6879", "name"=>"Deficiency Diseases", "relevance"=>"LOW"}, {"id"=>"M25306", "name"=>"Malnutrition", "relevance"=>"LOW"}, {"id"=>"M12684", "name"=>"Nutrition Disorders", "relevance"=>"LOW"}, {"id"=>"T5019", "name"=>"Rickets", "asFound"=>"Rickets", "relevance"=>"HIGH"}], "browseBranches"=>[{"name"=>"Musculoskeletal Diseases", "abbrev"=>"BC05"}, {"name"=>"Nutritional and Metabolic Diseases", "abbrev"=>"BC18"}, {"name"=>"All Conditions", "abbrev"=>"All"}, {"name"=>"Rare Diseases", "abbrev"=>"Rare"}]}, "interventionBrowseModule"=>{"meshes"=>[{"id"=>"D000004100", "term"=>"Dihydroxycholecalciferols"}, {"id"=>"D000002117", "term"=>"Calcitriol"}], "ancestors"=>[{"id"=>"D000014815", "term"=>"Vitamins"}, {"id"=>"D000018977", "term"=>"Micronutrients"}, {"id"=>"D000045505", "term"=>"Physiological Effects of Drugs"}, {"id"=>"D000050071", "term"=>"Bone Density Conservation Agents"}, {"id"=>"D000077264", "term"=>"Calcium-Regulating Hormones and Agents"}, {"id"=>"D000002120", "term"=>"Calcium Channel Agonists"}, {"id"=>"D000049990", "term"=>"Membrane Transport Modulators"}, {"id"=>"D000045504", "term"=>"Molecular Mechanisms of Pharmacological Action"}, {"id"=>"D000014662", "term"=>"Vasoconstrictor Agents"}], "browseLeaves"=>[{"id"=>"M17550", "name"=>"Vitamin D", "relevance"=>"LOW"}, {"id"=>"M6003", "name"=>"Cholecalciferol", "relevance"=>"LOW"}, {"id"=>"M8026", "name"=>"Ergocalciferols", "relevance"=>"LOW"}, {"id"=>"M7284", "name"=>"Dihydroxycholecalciferols", "asFound"=>"Mirabegron 50 mg", "relevance"=>"HIGH"}, {"id"=>"M5380", "name"=>"Calcitriol", "asFound"=>"Chef", "relevance"=>"HIGH"}, {"id"=>"M17558", "name"=>"Vitamins", "relevance"=>"LOW"}, {"id"=>"M21009", "name"=>"Micronutrients", "relevance"=>"LOW"}, {"id"=>"M16885", "name"=>"Trace Elements", "relevance"=>"LOW"}, {"id"=>"M5381", "name"=>"Calcium", "relevance"=>"LOW"}, {"id"=>"M9789", "name"=>"Hormones", "relevance"=>"LOW"}, {"id"=>"M5398", "name"=>"Calcium, Dietary", "relevance"=>"LOW"}, {"id"=>"M17409", "name"=>"Vasoconstrictor Agents", "relevance"=>"LOW"}, {"id"=>"T442", "name"=>"Cholecalciferol", "relevance"=>"LOW"}, {"id"=>"T479", "name"=>"Vitamin D3", "relevance"=>"LOW"}, {"id"=>"T440", "name"=>"Calciferol", "relevance"=>"LOW"}, {"id"=>"T478", "name"=>"Vitamin D2", "relevance"=>"LOW"}, {"id"=>"T445", "name"=>"Ergocalciferol", "relevance"=>"LOW"}], "browseBranches"=>[{"name"=>"Bone Density Conservation Agents", "abbrev"=>"BDCA"}, {"name"=>"Micronutrients", "abbrev"=>"Micro"}, {"name"=>"All Drugs and Chemicals", "abbrev"=>"All"}, {"name"=>"Vasoconstrictor Agents", "abbrev"=>"VaCoAg"}, {"name"=>"Vitamins", "abbrev"=>"Vi"}]}}, "resultsSection"=>{"moreInfoModule"=>{"pointOfContact"=>{"email"=>"marxs@mail.nih.gov", "phone"=>"3014965051", "title"=>"Stephen J. Marx, M.D./National Institute of Diabetes and Digestive and Kidney Diseases", "organization"=>"NIH"}, "certainAgreement"=>{"piSponsorEmployee"=>true}}, "adverseEventsModule"=>{"eventGroups"=>[{"id"=>"EG000", "title"=>"Group 1", "description"=>"Patient has rickets (cild) or osteomalacia (adult) of bone", "otherNumAtRisk"=>6, "otherNumAffected"=>0, "seriousNumAtRisk"=>6, "seriousNumAffected"=>0}], "frequencyThreshold"=>"5"}, "outcomeMeasuresModule"=>{"outcomeMeasures"=>[{"type"=>"PRIMARY", "title"=>"Number of Participants With Normal Serum Calcium Concentrations", "denoms"=>[{"units"=>"Participants", "counts"=>[{"value"=>"6", "groupId"=>"OG000"}]}], "groups"=>[{"id"=>"OG000", "title"=>"Group 1", "description"=>"Patient has rickets (cild) or osteomalacia (adult) of bone"}], "classes"=>[{"categories"=>[{"measurements"=>[{"value"=>"6", "groupId"=>"OG000", "lowerLimit"=>"8", "upperLimit"=>"11"}]}]}], "paramType"=>"NUMBER", "timeFrame"=>"1 year average", "description"=>"Normal calcium concentration 8.2-10.6 mg/dL", "unitOfMeasure"=>"participants", "reportingStatus"=>"POSTED"}]}, "participantFlowModule"=>{"groups"=>[{"id"=>"FG000", "title"=>"Group 1", "description"=>"Patient has rickets (child) or osteomalacia (adult) of bone"}], "periods"=>[{"title"=>"Overall Study", "milestones"=>[{"type"=>"STARTED", "achievements"=>[{"groupId"=>"FG000", "numSubjects"=>"6"}]}, {"type"=>"COMPLETED", "achievements"=>[{"groupId"=>"FG000", "numSubjects"=>"1"}]}, {"type"=>"NOT COMPLETED", "achievements"=>[{"groupId"=>"FG000", "numSubjects"=>"5"}]}]}]}, "baselineCharacteristicsModule"=>{"denoms"=>[{"units"=>"Participants", "counts"=>[{"value"=>"6", "groupId"=>"BG000"}]}], "groups"=>[{"id"=>"BG000", "title"=>"Group 1", "description"=>"Patient has rickets (cild) or osteomalacia (adult) of bone"}], "measures"=>[{"title"=>"Age, Continuous", "classes"=>[{"categories"=>[{"measurements"=>[{"value"=>"15", "groupId"=>"BG000", "lowerLimit"=>"5", "upperLimit"=>"50"}]}]}], "paramType"=>"MEAN", "unitOfMeasure"=>"years", "dispersionType"=>"FULL_RANGE"}, {"title"=>"Sex: Female, Male", "classes"=>[{"categories"=>[{"title"=>"Female", "measurements"=>[{"value"=>"3", "groupId"=>"BG000"}]}, {"title"=>"Male", "measurements"=>[{"value"=>"3", "groupId"=>"BG000"}]}]}], "paramType"=>"COUNT_OF_PARTICIPANTS", "unitOfMeasure"=>"Participants"}, {"title"=>"Region of Enrollment", "classes"=>[{"title"=>"United States", "categories"=>[{"measurements"=>[{"value"=>"6", "groupId"=>"BG000"}]}]}], "paramType"=>"NUMBER", "unitOfMeasure"=>"participants"}]}}, "protocolSection"=>{"designModule"=>{"phases"=>["PHASE2"], "studyType"=>"INTERVENTIONAL", "designInfo"=>{"allocation"=>"NA", "maskingInfo"=>{"masking"=>"NONE"}, "primaryPurpose"=>"TREATMENT", "interventionModel"=>"SINGLE_GROUP"}, "enrollmentInfo"=>{"type"=>"ACTUAL", "count"=>6}}, "statusModule"=>{"whyStopped"=>"Terminated because of lack of drug supply", "overallStatus"=>"TERMINATED", "startDateStruct"=>{"date"=>"1976-03"}, "expandedAccessInfo"=>{"hasExpandedAccess"=>false}, "statusVerifiedDate"=>"2013-06", "completionDateStruct"=>{"date"=>"2009-10", "type"=>"ACTUAL"}, "lastUpdateSubmitDate"=>"2013-11-21", "studyFirstSubmitDate"=>"1999-11-03", "resultsFirstSubmitDate"=>"2010-11-12", "studyFirstSubmitQcDate"=>"1999-11-03", "lastUpdatePostDateStruct"=>{"date"=>"2013-11-25", "type"=>"ESTIMATED"}, "resultsFirstSubmitQcDate"=>"2013-11-21", "studyFirstPostDateStruct"=>{"date"=>"1999-11-04", "type"=>"ESTIMATED"}, "resultsFirstPostDateStruct"=>{"date"=>"2013-11-25", "type"=>"ESTIMATED"}, "primaryCompletionDateStruct"=>{"date"=>"2009-10", "type"=>"ACTUAL"}}, "outcomesModule"=>{"primaryOutcomes"=>[{"measure"=>"Number of Participants With Normal Serum Calcium Concentrations", "timeFrame"=>"1 year average", "description"=>"Normal calcium concentration 8.2-10.6 mg/dL"}]}, "oversightModule"=>{"oversightHasDmc"=>false}, "conditionsModule"=>{"keywords"=>["Calciferol", "Hypocalcemia", "Rickets"], "conditions"=>["Hypocalcemia", "Rickets"]}, "referencesModule"=>{"references"=>[{"pmid"=>"6893458", "type"=>"BACKGROUND", "citation"=>"Marx SJ, Swart EG Jr, Hamstra AJ, DeLuca HF. Normal intrauterine development of the fetus of a woman receiving extraordinarily high doses of 1,25-dihydroxyvitamin D3. J Clin Endocrinol Metab. 1980 Nov;51(5):1138-42. doi: 10.1210/jcem-51-5-1138."}, {"pmid"=>"4357855", "type"=>"BACKGROUND", "citation"=>"Fraser D, Kooh SW, Kind HP, Holick MF, Tanaka Y, DeLuca HF. Pathogenesis of hereditary vitamin-D-dependent rickets. An inborn error of vitamin D metabolism involving defective conversion of 25-hydroxyvitamin D to 1 alpha,25-dihydroxyvitamin D. N Engl J Med. 1973 Oct 18;289(16):817-22. doi: 10.1056/NEJM197310182891601. No abstract available."}, {"pmid"=>"233695", "type"=>"BACKGROUND", "citation"=>"Marx SJ, Spiegel AM, Brown EM, Gardner DG, Downs RW Jr, Attie M, Hamstra AJ, DeLuca HF. A familial syndrome of decrease in sensitivity to 1,25-dihydroxyvitamin D. J Clin Endocrinol Metab. 1978 Dec;47(6):1303-10. doi: 10.1210/jcem-47-6-1303."}]}, "descriptionModule"=>{"briefSummary"=>"Vitamin D in the diet undergoes changes in the liver and kidneys to several forms. Patients suffering from disorders with Vitamin D resistance are unable to absorb calcium from food. Patients diagnosed with these disorders will be evaluated and treated with high doses of another form of Vitamin D (1,25-dihydroxyvitamin D3). Patients will be monitored and observed throughout the study to avoid experiencing side effects from the medication.", "detailedDescription"=>"Patients with extreme resistance to 1,25-dihydroxyvitamin D will be evaluated and treated with high doses of 1,25-dihydroxyvitamin D3.\n\nPlan: In previously untreated patients the study will be divided into a control and one or more treatment periods. During the control period, parathyroid status will be assessed by parameters nos. 1\\& 2 (below). In previously treated patients maintenance vitamin D will be gradually replaced with 1,25(OH)2D3. This will be accomplished by withdrawal of vitamin D and institution of 1,25(OH)2D3 when the serum calcium shows a downward trend.\n\n1,25(OH)2D3 as 0.25 or 0.5 ug capsules (though IND 20,889) or as a solution of I microgram per ml will be administered orally. In most cases, because of consideration of time and expense, the cooperation of the patient's local physician will be enlisted. The following will be monitored:\n\n1. Serum calcium, phosphorus,alkaline phosphatase,creatinine at twice weekly intervals. After a maintenance dose has been established, this will be decreased to a monthly, and subsequently 3-6 monthly interval.\n2. Urine calcium, phosphorus,creatinine and cAMP before therapy and, when appropriate, during therapy.\n\nThe dose of 1,25(OH)2D3 will be 0.125 to 50.0 ug/day. Serum calcium will not be allowed to rise above the normal range (2.0 -2.4 mM at NIH). Should hypercalcemia occur, appropriate treatment will be initiated and the drug dosage will be decreased."}, "eligibilityModule"=>{"sex"=>"ALL", "stdAges"=>["CHILD", "ADULT", "OLDER_ADULT"], "healthyVolunteers"=>false, "eligibilityCriteria"=>"* INCLUSION CRITERIA:\n\nPatients with hereditary resistance to calcitrol."}, "identificationModule"=>{"nctId"=>"NCT00001151", "briefTitle"=>"Studies With 1,25-Dihydroxycholecalciferol", "organization"=>{"class"=>"NIH", "fullName"=>"National Institutes of Health Clinical Center (CC)"}, "officialTitle"=>"Studies With 1,25-Dihydroxycholecalciferol", "orgStudyIdInfo"=>{"id"=>"760081"}, "secondaryIdInfos"=>[{"id"=>"76-DK-0081", "type"=>"OTHER", "domain"=>"76-DK-0081"}]}, "armsInterventionsModule"=>{"armGroups"=>[{"type"=>"EXPERIMENTAL", "label"=>"Drug treatment", "description"=>"1,25-Dihydroxycholecalciferol 5 ug orally per day for 2 years", "interventionNames"=>["Drug: 1,25-Dihydroxycholecalciferol"]}], "interventions"=>[{"name"=>"1,25-Dihydroxycholecalciferol", "type"=>"DRUG", "description"=>"Usual doses of 1,25-dihydroycholecalciferol are 0.25-1 ug per day. All patients in this study received very high doses or 5-20 ug per day.", "armGroupLabels"=>["Drug treatment"]}]}, "contactsLocationsModule"=>{"locations"=>[{"zip"=>"20892", "city"=>"Bethesda", "state"=>"Maryland", "country"=>"United States", "facility"=>"National Institutes of Health Clinical Center, 9000 Rockville Pike", "geoPoint"=>{"lat"=>38.98067, "lon"=>-77.10026}}], "overallOfficials"=>[{"name"=>"Stephen J Marx, MD", "role"=>"PRINCIPAL_INVESTIGATOR", "affiliation"=>"NIDDK/NIH"}]}, "sponsorCollaboratorsModule"=>{"leadSponsor"=>{"name"=>"National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)", "class"=>"NIH"}, "responsibleParty"=>{"type"=>"PRINCIPAL_INVESTIGATOR", "investigatorTitle"=>"Chief of the Metabolic Diseases Branch of NIDDK/NIH", "investigatorFullName"=>"Stephen Marx, M.D.", "investigatorAffiliation"=>"National Institutes of Health Clinical Center (CC)"}}}}
