Ignite Creation Date:
2025-12-24 @ 10:22 PM
Ignite Modification Date:
2025-12-28 @ 10:28 AM
Study NCT ID:
NCT01536535
Status:
COMPLETED
Last Update Posted:
2019-09-20
First Post:
2012-02-16
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Predicting Response to Standardized Pediatric Colitis Therapy
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D003093', 'term': 'Colitis, Ulcerative'}], 'ancestors': [{'id': 'D003092', 'term': 'Colitis'}, {'id': 'D005759', 'term': 'Gastroenteritis'}, {'id': 'D005767', 'term': 'Gastrointestinal Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D015212', 'term': 'Inflammatory Bowel Diseases'}, {'id': 'D003108', 'term': 'Colonic Diseases'}, {'id': 'D007410', 'term': 'Intestinal Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D019804', 'term': 'Mesalamine'}, {'id': 'D000305', 'term': 'Adrenal Cortex Hormones'}, {'id': 'D011241', 'term': 'Prednisone'}, {'id': 'D003082', 'term': 'Colectomy'}], 'ancestors': [{'id': 'D062368', 'term': 'meta-Aminobenzoates'}, {'id': 'D062365', 'term': 'Aminobenzoates'}, {'id': 'D001565', 'term': 'Benzoates'}, {'id': 'D000146', 'term': 'Acids, Carbocyclic'}, {'id': 'D002264', 'term': 'Carboxylic Acids'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D000636', 'term': 'Aminosalicylic Acids'}, {'id': 'D012459', 'term': 'Salicylates'}, {'id': 'D062385', 'term': 'Hydroxybenzoates'}, {'id': 'D006880', 'term': 'Hydroxy Acids'}, {'id': 'D001555', 'term': 'Benzene Derivatives'}, {'id': 'D006841', 'term': 'Hydrocarbons, Aromatic'}, {'id': 'D006844', 'term': 'Hydrocarbons, Cyclic'}, {'id': 'D006838', 'term': 'Hydrocarbons'}, {'id': 'D010636', 'term': 'Phenols'}, {'id': 'D006728', 'term': 'Hormones'}, {'id': 'D006730', 'term': 'Hormones, Hormone Substitutes, and Hormone Antagonists'}, {'id': 'D011244', 'term': 'Pregnadienediols'}, {'id': 'D011245', 'term': 'Pregnadienes'}, {'id': 'D011278', 'term': 'Pregnanes'}, {'id': 'D013256', 'term': 'Steroids'}, {'id': 'D000072473', 'term': 'Fused-Ring Compounds'}, {'id': 'D011083', 'term': 'Polycyclic Compounds'}, {'id': 'D000099090', 'term': 'Surgical Procedures, Colorectal'}, {'id': 'D013505', 'term': 'Digestive System Surgical Procedures'}, {'id': 'D013514', 'term': 'Surgical Procedures, Operative'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'jhyams@connecticutchildrens.org', 'phone': '8605459532', 'title': 'Dr. Jeffrey Hyams', 'organization': 'Connecticut Childrens'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}}, 'adverseEventsModule': {'timeFrame': 'Adverse events were collected from Baseline through to the 52 week visit.', 'eventGroups': [{'id': 'EG000', 'title': 'Mild UC', 'description': 'Mild = Initiated on 5-Aminosalicylates (ASA) or on oral CS with PUCAI \\< 45', 'otherNumAtRisk': 178, 'deathsNumAtRisk': 178, 'otherNumAffected': 157, 'seriousNumAtRisk': 178, 'deathsNumAffected': 0, 'seriousNumAffected': 21}, {'id': 'EG001', 'title': 'Moderate to Severe UC', 'description': 'Moderate/Severe = Initiated on IV CS or oral CS with PUCAI ≥45', 'otherNumAtRisk': 250, 'deathsNumAtRisk': 250, 'otherNumAffected': 224, 'seriousNumAtRisk': 250, 'deathsNumAffected': 0, 'seriousNumAffected': 79}], 'otherEvents': [{'term': 'Abdominal Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 178, 'numAffected': 56}, {'groupId': 'EG001', 'numAtRisk': 250, 'numAffected': 71}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Diarrhea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 178, 'numAffected': 52}, {'groupId': 'EG001', 'numAtRisk': 250, 'numAffected': 71}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 178, 'numAffected': 21}, {'groupId': 'EG001', 'numAtRisk': 250, 'numAffected': 23}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 178, 'numAffected': 10}, {'groupId': 'EG001', 'numAtRisk': 250, 'numAffected': 17}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 178, 'numAffected': 7}, {'groupId': 'EG001', 'numAtRisk': 250, 'numAffected': 11}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Dyspepsia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 178, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 250, 'numAffected': 11}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 178, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 250, 'numAffected': 4}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Acne', 'stats': [{'groupId': 'EG000', 'numAtRisk': 178, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 250, 'numAffected': 11}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Clostridium difficile', 'stats': [{'groupId': 'EG000', 'numAtRisk': 178, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 250, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Pancreatitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 178, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 250, 'numAffected': 2}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}], 'seriousEvents': [{'term': 'Hospitalized for UC complications', 'stats': [{'groupId': 'EG000', 'numAtRisk': 178, 'numAffected': 17}, {'groupId': 'EG001', 'numAtRisk': 250, 'numAffected': 69}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Pancreatitus acute', 'stats': [{'groupId': 'EG000', 'numAtRisk': 178, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 250, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Nephrolithiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 178, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 250, 'numAffected': 2}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Clostridium difficile colitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 178, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 250, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Dehydration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 178, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 250, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Depression', 'stats': [{'groupId': 'EG000', 'numAtRisk': 178, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 250, 'numAffected': 0}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Generalised tonic-clonic seizure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 178, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 250, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 178, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 250, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Hypersensitivity to mesalamine severe headache and pleuritic chest pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 178, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 250, 'numAffected': 1}], 'organSystem': 'Immune system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Intentional Overdose', 'stats': [{'groupId': 'EG000', 'numAtRisk': 178, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 250, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Intestinal anastomosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 178, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 250, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Syncope', 'stats': [{'groupId': 'EG000', 'numAtRisk': 178, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 250, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Vasculitic rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 178, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 250, 'numAffected': 1}], 'organSystem': 'Immune system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '1'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Number of Participants With Corticosteroid Free Remission (SFR)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '163', 'groupId': 'OG000'}, {'value': '237', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Mild UC', 'description': 'Mild = Initiated on mesalazine, or on oral CS with PUCAI \\< 45'}, {'id': 'OG001', 'title': 'Moderate to Severe UC', 'description': 'Moderate/Severe = Initiated on IV CS, or oral CS with PUCAI ≥45'}], 'classes': [{'categories': [{'measurements': [{'value': '80', 'groupId': 'OG000'}, {'value': '70', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '52 weeks', 'description': 'Week 52 CS-free remission: Number of participants with a PUCAI \\< 10 and no corticosteroids (CS) for 28 days without additional therapy or colectomy. Participants in both groups received corticosteroids, biologics, or colectomies, if symptomatic.\n\nThe Pediatric Ulcerative Colitis Activity Index (PUCAI) is a non-invasive disease activity index. The index measures include abdominal pain, rectal bleeding, stool consistency, number of stools per 24 hours, nocturnal stools, and activity level. A total score less than 10 indicates remission, Mild disease activity is 10-30, Moderate 35-60, Severe disease activity 65-85.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'PRIMARY', 'title': 'Number of Participants Who Needed Additional Therapy or Colectomy', 'denoms': [{'units': 'Participants', 'counts': [{'value': '163', 'groupId': 'OG000'}, {'value': '237', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Mild UC Disease', 'description': 'Mild = Initiated on mesalazine, or on oral CS with PUCAI \\< 45'}, {'id': 'OG001', 'title': 'Moderate to Severe UC', 'description': 'Moderate/Severe = Initiated on IV CS, or oral CS with PUCAI ≥45'}], 'classes': [{'categories': [{'measurements': [{'value': '46', 'groupId': 'OG000'}, {'value': '151', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Within 52 weeks', 'description': 'Number of participants who needed additional therapy or colectomy. Additional therapy included Anti-Tumour Necrosis Factor alpha (TNFα), Calcineurin inhibitor, Immunomodulator', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Number of Participants Receiving a Colectomy', 'denoms': [{'units': 'Participants', 'counts': [{'value': '163', 'groupId': 'OG000'}, {'value': '237', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Mild UC Disease', 'description': 'Mild = Initiated on mesalazine, or on oral CS with PUCAI \\< 45'}, {'id': 'OG001', 'title': 'Moderate to Severe UC', 'description': 'Moderate/Severe = Initiated on IV CS, or oral CS with PUCAI ≥45'}], 'classes': [{'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '23', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Within 52 weeks', 'description': 'Number of participants who received a colectomy within 52 weeks', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Mild UC Disease', 'description': 'Mild = Initiated on mesalazine, or on oral CS with Pediatric Ulcerative Colitis Activity Index (PUCAI) \\< 45'}, {'id': 'FG001', 'title': 'Moderate to Severe UC', 'description': 'Moderate/Severe = Initiated on IV CS, or oral CS with PUCAI ≥45'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '178'}, {'groupId': 'FG001', 'numSubjects': '250'}]}, {'type': 'COMPLETED', 'achievements': [{'comment': 'Patients either completed week 52 visit or had early colectomy or had additional therapy.', 'groupId': 'FG000', 'numSubjects': '163'}, {'comment': 'Patients either completed week 52 visit or had early colectomy or had additional therapy.', 'groupId': 'FG001', 'numSubjects': '237'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '15'}, {'groupId': 'FG001', 'numSubjects': '13'}]}], 'dropWithdraws': [{'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '10'}, {'groupId': 'FG001', 'numSubjects': '5'}]}, {'type': 'Lost to Follow-up', 'reasons': [{'groupId': 'FG000', 'numSubjects': '4'}, {'groupId': 'FG001', 'numSubjects': '6'}]}, {'type': 'Physician Decision', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Participant moved', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}]}], 'recruitmentDetails': 'Recruitment and enrollment began in July 2012 and ended in April 2015. Final study visits were in April 2016 at which time all study patients had a minimum of one year follow up. Patients were recruited from 29 centers in the United States and Canada.', 'preAssignmentDetails': 'Participants were consented at diagnostic colonoscopy obtaining biological specimens (DNA, serum, rectal tissue, stool for microbiome). After diagnosis of Ulcerative Colitis, they began treatment dictated by initial disease severity and provider/patient choice. Three patients were untreated.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '178', 'groupId': 'BG000'}, {'value': '250', 'groupId': 'BG001'}, {'value': '428', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Mild UC', 'description': 'Mild = Initiated on mesalamine, or on oral CS with PUCAI \\< 45'}, {'id': 'BG001', 'title': 'Moderate to Severe UC', 'description': 'Moderate/Severe = Initiated on IV CS, or oral CS with PUCAI ≥45'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '178', 'groupId': 'BG000'}, {'value': '250', 'groupId': 'BG001'}, {'value': '428', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '12.6', 'spread': '3.4', 'groupId': 'BG000'}, {'value': '12.7', 'spread': '3.2', 'groupId': 'BG001'}, {'value': '12.7', 'spread': '3.3', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '178', 'groupId': 'BG000'}, {'value': '250', 'groupId': 'BG001'}, {'value': '428', 'groupId': 'BG002'}]}], 'categories': [{'title': 'Female', 'measurements': [{'value': '88', 'groupId': 'BG000'}, {'value': '124', 'groupId': 'BG001'}, {'value': '212', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '90', 'groupId': 'BG000'}, {'value': '126', 'groupId': 'BG001'}, {'value': '216', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race and Ethnicity Not Collected', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants', 'populationDescription': 'Race and Ethnicity were not collected from any participant.'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2015-02-02', 'size': 645248, 'label': 'Study Protocol and Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'Prot_SAP_000.pdf', 'typeAbbrev': 'Prot_SAP', 'uploadDate': '2019-02-15T17:36', 'hasProtocol': True}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE4'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 431}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2012-07-10', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2019-09', 'completionDateStruct': {'date': '2018-04-30', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2019-09-04', 'studyFirstSubmitDate': '2012-02-16', 'resultsFirstSubmitDate': '2019-08-06', 'studyFirstSubmitQcDate': '2012-02-16', 'lastUpdatePostDateStruct': {'date': '2019-09-20', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2019-08-06', 'studyFirstPostDateStruct': {'date': '2012-02-22', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2019-08-28', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2018-04-30', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants With Corticosteroid Free Remission (SFR)', 'timeFrame': '52 weeks', 'description': 'Week 52 CS-free remission: Number of participants with a PUCAI \\< 10 and no corticosteroids (CS) for 28 days without additional therapy or colectomy. Participants in both groups received corticosteroids, biologics, or colectomies, if symptomatic.\n\nThe Pediatric Ulcerative Colitis Activity Index (PUCAI) is a non-invasive disease activity index. The index measures include abdominal pain, rectal bleeding, stool consistency, number of stools per 24 hours, nocturnal stools, and activity level. A total score less than 10 indicates remission, Mild disease activity is 10-30, Moderate 35-60, Severe disease activity 65-85.'}, {'measure': 'Number of Participants Who Needed Additional Therapy or Colectomy', 'timeFrame': 'Within 52 weeks', 'description': 'Number of participants who needed additional therapy or colectomy. Additional therapy included Anti-Tumour Necrosis Factor alpha (TNFα), Calcineurin inhibitor, Immunomodulator'}], 'secondaryOutcomes': [{'measure': 'Number of Participants Receiving a Colectomy', 'timeFrame': 'Within 52 weeks', 'description': 'Number of participants who received a colectomy within 52 weeks'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Ulcerative Colitis', 'mesalamine', 'corticosteroids', 'PROTECT'], 'conditions': ['Ulcerative Colitis']}, 'referencesModule': {'references': [{'pmid': '28939374', 'type': 'RESULT', 'citation': 'Hyams JS, Davis S, Mack DR, Boyle B, Griffiths AM, LeLeiko NS, Sauer CG, Keljo DJ, Markowitz J, Baker SS, Rosh J, Baldassano RN, Patel A, Pfefferkorn M, Otley A, Heyman M, Noe J, Oliva-Hemker M, Rufo P, Strople J, Ziring D, Guthery SL, Sudel B, Benkov K, Wali P, Moulton D, Evans J, Kappelman MD, Marquis A, Sylvester FA, Collins MH, Venkateswaran S, Dubinsky M, Tangpricha V, Spada KL, Britt A, Saul B, Gotman N, Wang J, Serrano J, Kugathasan S, Walters T, Denson LA. Factors associated with early outcomes following standardised therapy in children with ulcerative colitis (PROTECT): a multicentre inception cohort study. Lancet Gastroenterol Hepatol. 2017 Dec;2(12):855-868. doi: 10.1016/S2468-1253(17)30252-2. Epub 2017 Sep 20.'}, {'pmid': '31373712', 'type': 'DERIVED', 'citation': 'Carmody JK, Plevinsky J, Peugh JL, Denson LA, Hyams JS, Lobato D, LeLeiko NS, Hommel KA. Longitudinal non-adherence predicts treatment escalation in paediatric ulcerative colitis. Aliment Pharmacol Ther. 2019 Oct;50(8):911-918. doi: 10.1111/apt.15445. Epub 2019 Aug 2.'}, {'pmid': '30935734', 'type': 'DERIVED', 'citation': 'Hyams JS, Davis Thomas S, Gotman N, Haberman Y, Karns R, Schirmer M, Mo A, Mack DR, Boyle B, Griffiths AM, LeLeiko NS, Sauer CG, Keljo DJ, Markowitz J, Baker SS, Rosh J, Baldassano RN, Patel A, Pfefferkorn M, Otley A, Heyman M, Noe J, Oliva-Hemker M, Rufo PA, Strople J, Ziring D, Guthery SL, Sudel B, Benkov K, Wali P, Moulton D, Evans J, Kappelman MD, Marquis MA, Sylvester FA, Collins MH, Venkateswaran S, Dubinsky M, Tangpricha V, Spada KL, Saul B, Wang J, Serrano J, Hommel K, Marigorta UM, Gibson G, Xavier RJ, Kugathasan S, Walters T, Denson LA. Clinical and biological predictors of response to standardised paediatric colitis therapy (PROTECT): a multicentre inception cohort study. Lancet. 2019 Apr 27;393(10182):1708-1720. doi: 10.1016/S0140-6736(18)32592-3. Epub 2019 Mar 29.'}]}, 'descriptionModule': {'briefSummary': 'This is a multi-center, open-label study to determine the safety and effectiveness (how well it works) of two standardized treatments called "mesalamine" (Pentasa®) and "prednisone" in children with newly diagnosed Ulcerative Colitis (UC). Standardized treatments are types of treatments agreed upon and used by many qualified doctors. The medications being used in this study are considered "standard of care". Currently the ways in which these medicines are used (doses, frequency of dosing) may vary from site to site. This study will determine response to a standardized way of giving these medicines.\n\nThis study will also identify biomarkers for ulcerative colitis. Biomarkers are things that doctors can find in blood, stool, or bowel tissue that indicate how much inflammation there is in the bowel, how the inflammation is produced, and whether the inflammation is responding to treatment. Collecting response and remission (free of symptoms) information on these standardized treatments and the "biomarkers" can possibly help doctors create a model, or plan to know which children with UC may respond quickly, or which children may develop complications.', 'detailedDescription': "Ulcerative Colitis (UC) denotes a phenotype of chronic inflammatory bowel disease (IBD), where inflammation is localized to the colonic mucosa, and extends from the rectum proximally in varying extents. The disorder is thought to result from an inappropriate activation of the mucosal immune system by antigens derived from both the host epithelium and the enteric flora, in genetically susceptible individuals. UC is strikingly heterogeneous with respect to age of onset, anatomical extent and disease course, with some experiencing chronically active severe disease, while others have intermittent periods of clinical remission and disease exacerbation. Patients' therapeutic responses vary. The reasons underlying such variability are not well understood. Although it has been widely hypothesized that several genes may influence the development of UC, and modify its phenotypic expression and severity, to date there are few confirmed examples of such relationships.\n\nIt has been postulated that the 5-aminosalicylate drugs exert their anti-inflammatory effect locally at the intestinal mucosa. Mechanisms likely include inhibition of 5-lipoxygenase resulting in decreased production of leukotriene B4, scavenging of reactive oxygen metabolites, prevention of up-regulation of leukocyte adhesion molecules, and inhibition of Interleukin 1 (IL-1) synthesis. Though multiple studies have shown the efficacy of aminosalicylates in inducing and maintaining remission in adults with UC, there are few data in children.\n\nCorticosteroids (CS) have been the mainstay of treatment of severe UC since efficacy was first demonstrated in the 1955 randomized controlled trial by Truelove and Witts. Recent practice guidelines developed in adults support their use because of rapid onset of action and significant efficacy though CS dependency is noted. Though no controlled data on their use have been reported in children they are frequently used in this population. A recent report from investigators leading the prospective Pediatric IBD Collaborative Research Group Registry described 97 subjects with a diagnosis of UC and a minimum of 1 year follow-up; 79% received CS. At diagnosis 81% of CS treated patients had moderate/severe disease, and 81% had pancolitis. Clinically inactive disease, determined by physician global assessment, was noted in 60% at 3 months following CS therapy, but by one year 45% were considered CS dependent despite the frequent use of immunomodulators (IM). Among those children with initially moderate to severe disease in clinical remission at 3 months, about two-thirds had stopped the CS by one year."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '17 Years', 'minimumAge': '4 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Age ≥ 4years and ≤17 years at initiation of therapy (achieved 4th birthday, not yet 18th)\n* Weight ≥15 kg\n* New diagnosis of ulcerative colitis established by standard clinical, endoscopic, and histologic features at the PROTECT study site\n* Colitis extending beyond the rectosigmoid (Paris classification E2, E3, or E4)\\[144\\]. If a patient is seriously ill and the clinician does not advance the colonoscope beyond the sigmoid colon but the clinical condition of the patient highly suggests more extensive disease then that patient is eligible for study.\n* Disease activity by PUCAI of ≥10 at diagnosis\n* No therapy previously initiated to treat the newly diagnosed ulcerative colitis\n* Stool culture negative for routine enteric pathogens (Salmonella, Shigella, Campylobacter, E. coli 0157:H7) and Clostridium difficile toxin. Recent successful treatment for Clostridium difficile does not exclude a patient if toxin now absent. However, the patient must be a minimum of 5 weeks from the time treatment was started at the time toxin is absent.\n* Stool study negative for enteric parasites (ova and parasites)\n* Parent/guardian consent and patient assent\n* Ability to remain in follow-up for a minimum of one year from diagnosis\n* Female patients of child bearing age must have a negative urine pregnancy test and practice acceptable contraception (e.g., abstinence, intramuscular or hormonal contraception, two barrier methods (e.g., condom, diaphragm, or spermicide), intrauterine device, verbal report of the partner with history of vasectomy, or be surgically sterile). All female patients of childbearing potential (post-menarche) will undergo urine pregnancy testing at screening and must not be lactating.\n\nExclusion Criteria:\n\n* Clinical, endoscopic, radiologic, or histologic evidence suggesting Crohn's disease (CD) consistent with Paris and North American Society for Pediatric Gastroenterology, Hepatology and Nutrition (NASPGHAN) criteria \\[144, 145\\]\n* A previous diagnosis of inflammatory bowel disease for which treatment was given\n* Evidence of any active enteric infection at the time of study entry\n* Use of any oral CS for non-gastrointestinal indication within the past 4 weeks (e.g., asthma). Use of inhaled CS does not exclude a patient.\n* History of use of IM or anti-TNFα agent for other medical conditions (e.g., juvenile rheumatoid arthritis) within the past 6 months\n* Use of Accutane within the past 4 weeks\n* Use of any investigational drug within the past four weeks\n* Use of any 5-aminosalicylate within the past 4 weeks\n* Pregnancy\n* Subjects with poorly controlled medical conditions (e.g. diabetes, congestive heart failure)\n* Proctitis or proctosigmoiditis only (Paris classification E1) on colonoscopic evaluation\n* Chronic renal disease (BUN and serum creatinine \\>1.5 times the upper normal limit)\n* Hepatic disease (AST or Alkaline phosphatase (ALP) greater than 3 times the upper normal limit in the absence of concomitant liver disease associated with IBD following full evaluation)\n* History of allergy or hypersensitivity to salicylates, aminosalicylates, or any component of the Pentasa capsule.\n* History of coexisting chronic illness or evidence of significant organic or psychiatric disease on medical history or physical examination, which, in the Investigator's opinion, would prevent participation in the study\n* History or presence of any condition causing malabsorption or an effect on gastrointestinal (GI) motility, or history of extensive small bowel resection (greater than half the length of the small intestine).\n* The finding of Helicobacter pylori at the time of evaluation does not exclude the patient from the study. Whether to treat this patient for Helicobacter pylori and when will be left to the discretion of the site."}, 'identificationModule': {'nctId': 'NCT01536535', 'acronym': 'PROTECT', 'briefTitle': 'Predicting Response to Standardized Pediatric Colitis Therapy', 'organization': {'class': 'OTHER', 'fullName': "Connecticut Children's Medical Center"}, 'officialTitle': 'Multicenter Open-label Study Evaluating the Safety and Efficacy of Standardized Initial Therapy Using Either Mesalamine or Corticosteroids Then Mesalamine to Treat Children and Adolescents With Newly Diagnosed Ulcerative Colitis.', 'orgStudyIdInfo': {'id': 'U01DK095745-01', 'link': 'https://reporter.nih.gov/quickSearch/U01DK095745-01', 'type': 'NIH'}, 'secondaryIdInfos': [{'id': '1U34DK090804', 'link': 'https://reporter.nih.gov/quickSearch/1U34DK090804', 'type': 'NIH'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Mild UC', 'description': 'Mild = Initiated on mesalazine, or on oral CS with Pediatric Ulcerative Colitis Activity Index (PUCAI) \\< 45\n\nPatients can be treated with any of the therapies noted below:\n\nMesalazine: doses is rounded to the nearest 500mg increment, maximum dose of 75 mg/kg/day Oral corticosteroids:1-1.5 mg/kg/day, rounded up to the nearest 5 mg value\n\nIV corticosteroids:\n\nAdditional Therapies:\n\nCalcineurin inhibitor (cyclosporine, tacrolimus) or anti-Tumour Necrosis Factor alpha (TNFα) therapy: These therapies may also be instituted if in the judgment of the attending physician is needed.\n\nImmunomodulator or biologic therapy: thiopurine then dosing of azathioprine:2.5-3 mg/kg/day; 6-MP at 1-1.5 mg/kg/day Colectomy', 'interventionNames': ['Drug: Mesalazine', 'Drug: IV Corticosteroid', 'Drug: Oral Corticosteroids', 'Other: Additional Therapies', 'Procedure: Colectomy']}, {'type': 'EXPERIMENTAL', 'label': 'Moderate to Severe UC', 'description': 'Moderate/Severe = Initiated on IV CS, or oral CS with PUCAI ≥45\n\nPatients can be treated with any of the therapies noted below:\n\nMesalazine: doses is rounded to the nearest 500mg increment, maximum dose of 75 mg/kg/day Oral corticosteroids:1-1.5 mg/kg/day, rounded up to the nearest 5 mg value\n\nIV corticosteroids:\n\nAdditional Therapies:\n\nCalcineurin inhibitor (cyclosporine, tacrolimus) or anti-TNFα therapy: These therapies may also be instituted if in the judgment of the attending physician is needed.\n\nImmunomodulator or biologic therapy: thiopurine then dosing of azathioprine:2.5-3 mg/kg/day; 6-Mercaptopurine (MP) at 1-1.5 mg/kg/day Colectomy', 'interventionNames': ['Drug: Mesalazine', 'Drug: IV Corticosteroid', 'Drug: Oral Corticosteroids', 'Other: Additional Therapies', 'Procedure: Colectomy']}], 'interventions': [{'name': 'Mesalazine', 'type': 'DRUG', 'otherNames': ['Pentasa'], 'description': 'Mesalazine (Pentasa) comes in 500mg capsules, and doses will need to be rounded to the nearest 500mg increment, with a maximum dose of 76 mg/kg/day. The average dose for the pediatric population will be approximately 70 mg/kg/day. Patients will be allowed to escalate to the final dose over 4 days to minimize side-effects such as headache.', 'armGroupLabels': ['Mild UC', 'Moderate to Severe UC']}, {'name': 'IV Corticosteroid', 'type': 'DRUG', 'otherNames': ['Prednisone'], 'description': 'Treatment with IV Corticosteroid', 'armGroupLabels': ['Mild UC', 'Moderate to Severe UC']}, {'name': 'Oral Corticosteroids', 'type': 'DRUG', 'otherNames': ['Prednisone'], 'description': 'Treatment with oral corticosteroids', 'armGroupLabels': ['Mild UC', 'Moderate to Severe UC']}, {'name': 'Additional Therapies', 'type': 'OTHER', 'description': 'Anti-TNFα, Calcineurin inhibitor, Immunomodulator', 'armGroupLabels': ['Mild UC', 'Moderate to Severe UC']}, {'name': 'Colectomy', 'type': 'PROCEDURE', 'description': 'Colectomy', 'armGroupLabels': ['Mild UC', 'Moderate to Severe UC']}]}, 'contactsLocationsModule': {'locations': [{'zip': '90095', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'UCLA Medical Center', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '94143', 'city': 'San Francisco', 'state': 'California', 'country': 'United States', 'facility': 'University of California at San Francisco', 'geoPoint': {'lat': 37.77493, 'lon': -122.41942}}, {'zip': '06106', 'city': 'Hartford', 'state': 'Connecticut', 'country': 'United States', 'facility': "Connecticut Children's Medical Center", 'geoPoint': {'lat': 41.76371, 'lon': -72.68509}}, {'zip': '32207', 'city': 'Jacksonville', 'state': 'Florida', 'country': 'United States', 'facility': "Nemours Children's Clinic", 'geoPoint': {'lat': 30.33218, 'lon': -81.65565}}, {'zip': '30322', 'city': 'Atlanta', 'state': 'Georgia', 'country': 'United States', 'facility': "Emory Children's Center", 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'zip': '60611-2605', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': "Ann & Robert H. Lurie Children's Hospital of Chicago", 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '46202', 'city': 'Indianapolis', 'state': 'Indiana', 'country': 'United States', 'facility': "Riley Children's Hospital", 'geoPoint': {'lat': 39.76838, 'lon': -86.15804}}, {'zip': '21287', 'city': 'Baltimore', 'state': 'Maryland', 'country': 'United States', 'facility': "John Hopkins Children's Hospital", 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': "Children's Hospital of Boston", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '55454', 'city': 'Minneapolis', 'state': 'Minnesota', 'country': 'United States', 'facility': 'University of Minnesota Medical Center', 'geoPoint': {'lat': 44.97997, 'lon': -93.26384}}, {'zip': '07962', 'city': 'Morristown', 'state': 'New Jersey', 'country': 'United States', 'facility': "Goryeb Children's Hospital / Atlantic Health", 'geoPoint': {'lat': 40.79677, 'lon': -74.48154}}, {'zip': '14222', 'city': 'Buffalo', 'state': 'New York', 'country': 'United States', 'facility': "Women and Children's Hospital of Buffalo", 'geoPoint': {'lat': 42.88645, 'lon': -78.87837}}, {'zip': '11040', 'city': 'New Hyde Park', 'state': 'New York', 'country': 'United States', 'facility': "Cohen Children's Medical Center", 'geoPoint': {'lat': 40.7351, 'lon': -73.68791}}, {'zip': '10029', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Mt Sinai Hospital', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '10032', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': "Morgan Stanley Children's Hospital", 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '13210', 'city': 'Syracuse', 'state': 'New York', 'country': 'United States', 'facility': "Golisano Children's Hospital SUNY Upstate Medical University", 'geoPoint': {'lat': 43.04812, 'lon': -76.14742}}, {'zip': '27599', 'city': 'Chapel Hill', 'state': 'North Carolina', 'country': 'United States', 'facility': 'University of North Carolina at Chapel HIll', 'geoPoint': {'lat': 35.9132, 'lon': -79.05584}}, {'zip': '45229', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'facility': "Cincinnati Children's Hospital Medical Center", 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '43205', 'city': 'Columbus', 'state': 'Ohio', 'country': 'United States', 'facility': "Nationwide Children's Hospital", 'geoPoint': {'lat': 39.96118, 'lon': -82.99879}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': "Children's Hospital of Philadelphia", 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '15224', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'country': 'United States', 'facility': "Children's Hospital of Pittsburgh of UPMC", 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}, {'zip': '02903', 'city': 'Providence', 'state': 'Rhode Island', 'country': 'United States', 'facility': "Hasbro Children's Hospital", 'geoPoint': {'lat': 41.82399, 'lon': -71.41283}}, {'zip': '37232', 'city': 'Nashville', 'state': 'Tennessee', 'country': 'United States', 'facility': "Monroe Carell Jr. Children's Hospital at Vanderbilt", 'geoPoint': {'lat': 36.16589, 'lon': -86.78444}}, {'zip': '75235', 'city': 'Dallas', 'state': 'Texas', 'country': 'United States', 'facility': 'UT Southwestern', 'geoPoint': {'lat': 32.78306, 'lon': -96.80667}}, {'zip': '84132', 'city': 'Salt Lake City', 'state': 'Utah', 'country': 'United States', 'facility': "Primary Children's Medical Center (University of Utah)", 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'zip': '53226', 'city': 'Milwaukee', 'state': 'Wisconsin', 'country': 'United States', 'facility': 'Medical College of Wisconsin', 'geoPoint': {'lat': 43.0389, 'lon': -87.90647}}, {'zip': 'B3k6r8', 'city': 'Halifax', 'state': 'Nova Scotia', 'country': 'Canada', 'facility': 'IWK Health Centre', 'geoPoint': {'lat': 44.64269, 'lon': -63.57688}}, {'zip': 'K1H8L1', 'city': 'Ottawa', 'state': 'Ontario', 'country': 'Canada', 'facility': "Children's Hospital of Eastern Ontario", 'geoPoint': {'lat': 45.41117, 'lon': -75.69812}}, {'zip': 'M5G1X8', 'city': 'Toronto', 'state': 'Ontario', 'country': 'Canada', 'facility': 'Hospital for Sick Children', 'geoPoint': {'lat': 43.70643, 'lon': -79.39864}}], 'overallOfficials': [{'name': 'Jeffrey Hyams, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': "Connecticut Children's Medical Center"}, {'name': 'Lee Denson, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': "Children's Hospital Medical Center, Cincinnati"}, {'name': 'Sonia Davis, DrPH', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Collaborative Studies Coordinating Center - UNC-CH'}]}, 'ipdSharingStatementModule': {'infoTypes': ['STUDY_PROTOCOL', 'SAP', 'CSR', 'ANALYTIC_CODE'], 'timeFrame': 'Data will be shared 6 months after the primary publication of the manuscript appears on line.', 'ipdSharing': 'YES', 'description': 'Data will be shared 6 months after the primary publication of the manuscript appears on line.', 'accessCriteria': 'NIH repository list serve to be determined'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': "Connecticut Children's Medical Center", 'class': 'OTHER'}, 'collaborators': [{'name': 'National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)', 'class': 'NIH'}], 'responsibleParty': {'type': 'SPONSOR'}}}}