Ignite Creation Date:
2025-12-25 @ 1:18 AM
Ignite Modification Date:
2026-02-13 @ 7:59 AM
Study NCT ID:
NCT03801993
Status:
COMPLETED
Last Update Posted:
2024-10-15
First Post:
2019-01-04
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Multicenter Study of Patient-reported Gastrointestinal Symptoms in People With Cystic Fibrosis
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D003550', 'term': 'Cystic Fibrosis'}, {'id': 'D003248', 'term': 'Constipation'}], 'ancestors': [{'id': 'D010182', 'term': 'Pancreatic Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D008171', 'term': 'Lung Diseases'}, {'id': 'D012140', 'term': 'Respiratory Tract Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D007232', 'term': 'Infant, Newborn, Diseases'}, {'id': 'D012817', 'term': 'Signs and Symptoms, Digestive'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'cgoss@uw.edu', 'phone': '(206) 987-5725', 'title': 'Dr. Christopher H. Goss M.D., M.Sc.', 'organization': "Seattle Children's Hospital"}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}}, 'adverseEventsModule': {'timeFrame': 'Adverse Events, including Deaths and Serious Adverse Experiences, were not collected or monitored as part of this study.', 'description': 'Adverse Events, including Deaths and Serious Adverse Experiences, were not collected or monitored as part of this study.', 'eventGroups': [{'id': 'EG000', 'title': 'All Subjects', 'description': 'Subjects with a diagnosis of Cystic Fibrosis (CF) who meet all the inclusion and none of the exclusion criteria will be eligible for participation in this study.', 'otherNumAtRisk': 0, 'deathsNumAtRisk': 0, 'otherNumAffected': 0, 'seriousNumAtRisk': 0, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Number of Participants Who Fully Completed at Least One ePRO', 'denoms': [{'units': 'Participants', 'counts': [{'value': '169', 'groupId': 'OG000'}, {'value': '233', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Age <18', 'description': 'Age \\<18 years at baseline among All Enrolled'}, {'id': 'OG001', 'title': 'Age >=18', 'description': 'Age \\>= 18 years at baseline among All Enrolled'}], 'classes': [{'title': 'Week 1', 'categories': [{'measurements': [{'value': '133', 'groupId': 'OG000'}, {'value': '196', 'groupId': 'OG001'}]}]}, {'title': 'Week 2', 'categories': [{'measurements': [{'value': '130', 'groupId': 'OG000'}, {'value': '194', 'groupId': 'OG001'}]}]}, {'title': 'Week 4', 'categories': [{'measurements': [{'value': '125', 'groupId': 'OG000'}, {'value': '187', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '1 month', 'description': 'Number participants with scheduled outside-the-clinic assessments who fully completed at least one of the four PROs. A "fully complete" questionnaire is defined as having responded to all questions with no missing responses.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Number of Participants With Period-Prevalence of Constipation', 'denoms': [{'units': 'Participants', 'counts': [{'value': '95', 'groupId': 'OG000'}, {'value': '151', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Age <18 With Complete Follow-up', 'description': 'Participants under 18 years old at baseline who fully completed the disease-specific questionnaire at baseline and all follow-up weeks were considered.'}, {'id': 'OG001', 'title': 'Age >=18 With Complete Follow-up', 'description': 'Participants 18 years or older at baseline who fully completed the disease-specific questionnaire at baseline and all follow-up weeks were considered.'}], 'classes': [{'categories': [{'measurements': [{'value': '18', 'groupId': 'OG000'}, {'value': '35', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '1 month', 'description': 'This table summarizes the period-prevalence of protocol-defined constipation. For each follow-up response, constipation was defined by the protocol as having fewer than 3 bowel movements and/or Bristol Stool Scale of Type 1 or 2 (hard lumps) in the past 7 days. Period prevalence is defined as occurrence of the symptom at any time from baseline to week 4. Only participants who fully completed the disease-specific questionnaire at baseline and all follow-up weeks were considered.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants who fully completed the disease-specific questionnaire at baseline and all follow-up weeks'}, {'type': 'SECONDARY', 'title': 'Mean Patient Reported Outcome (PRO) Scores', 'denoms': [{'units': 'Participants', 'counts': [{'value': '169', 'groupId': 'OG000'}, {'value': '233', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Age <18', 'description': 'Age \\<18 years at baseline among All Enrolled'}, {'id': 'OG001', 'title': 'Age >=18', 'description': 'Age \\>= 18 years at baseline among All Enrolled'}], 'classes': [{'title': 'PAC-SYM Total Score', 'categories': [{'measurements': [{'value': '0.49', 'spread': '0.45', 'groupId': 'OG000'}, {'value': '0.64', 'spread': '0.55', 'groupId': 'OG001'}]}]}, {'title': 'PAGI-SYM Total Score', 'categories': [{'measurements': [{'value': '0.49', 'spread': '0.56', 'groupId': 'OG000'}, {'value': '0.78', 'spread': '0.66', 'groupId': 'OG001'}]}]}, {'title': 'PAC-QOL Total Score', 'categories': [{'measurements': [{'value': '0.58', 'spread': '0.47', 'groupId': 'OG000'}, {'value': '0.73', 'spread': '0.59', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'At Visit 1 (1 day)', 'description': 'Mean PRO scores (PAC-SYM score range: 0-4, PAGI-SYM score range: 0-5, PAC-QOL score range: 0-4) at time of enrollment (Visit 1) where lower scores correspond to less symptom severity.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Number of Participants Treated for GI Symptoms', 'denoms': [{'units': 'Participants', 'counts': [{'value': '169', 'groupId': 'OG000'}, {'value': '233', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Age <18', 'description': 'Age \\<18 years at baseline among All Enrolled'}, {'id': 'OG001', 'title': 'Age >=18', 'description': 'Age \\>= 18 years at baseline among All Enrolled'}], 'classes': [{'title': 'Initiating or Receiving GI Treatment', 'categories': [{'measurements': [{'value': '167', 'groupId': 'OG000'}, {'value': '228', 'groupId': 'OG001'}]}]}, {'title': 'GI Treatment for Constipation', 'categories': [{'measurements': [{'value': '88', 'groupId': 'OG000'}, {'value': '78', 'groupId': 'OG001'}]}]}, {'title': 'GI Treatment for Non-Constipation', 'categories': [{'measurements': [{'value': '166', 'groupId': 'OG000'}, {'value': '228', 'groupId': 'OG001'}]}]}, {'title': 'Use GI Tube', 'categories': [{'measurements': [{'value': '24', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}]}]}, {'title': 'On Azithromycin', 'categories': [{'measurements': [{'value': '60', 'groupId': 'OG000'}, {'value': '131', 'groupId': 'OG001'}]}]}, {'title': 'On drugs used in diabetes', 'categories': [{'measurements': [{'value': '12', 'groupId': 'OG000'}, {'value': '59', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'At Visit 1 (1 day)', 'description': 'Number of enrolled participants receiving treatment for GI symptoms at Visit 1', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'All Enrolled', 'description': 'Eligible participant who fully completed at least one ePRO at Baseline Visit'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'comment': 'With 400 subjects, this study will allow estimation of variability (between and within subject) in 160 pediatric subjects and 240 adult subjects.', 'achievements': [{'groupId': 'FG000', 'numSubjects': '402'}]}, {'type': 'Week 1', 'comment': 'At least one fully completed ePRO at Week 1', 'achievements': [{'groupId': 'FG000', 'numSubjects': '329'}]}, {'type': 'Week 2', 'comment': 'At least one fully completed ePRO at Week 2', 'achievements': [{'groupId': 'FG000', 'numSubjects': '324'}]}, {'type': 'Week 4', 'comment': 'At least one fully completed ePRO at Week 4', 'achievements': [{'groupId': 'FG000', 'numSubjects': '312'}]}, {'type': 'COMPLETED', 'comment': 'Fully completed at least one ePRO at all Follow-up Weeks', 'achievements': [{'groupId': 'FG000', 'numSubjects': '254'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '148'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '169', 'groupId': 'BG000'}, {'value': '233', 'groupId': 'BG001'}, {'value': '402', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Age <18', 'description': 'Age \\<18 years at baseline among All Enrolled'}, {'id': 'BG001', 'title': 'Age >=18', 'description': 'Age \\>= 18 years at baseline among All Enrolled'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '169', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '169', 'groupId': 'BG002'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '233', 'groupId': 'BG001'}, {'value': '233', 'groupId': 'BG002'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '10.28', 'groupId': 'BG000', 'lowerLimit': '2.09', 'upperLimit': '17.83'}, {'value': '26.23', 'groupId': 'BG001', 'lowerLimit': '18.04', 'upperLimit': '61.13'}, {'value': '19.36', 'groupId': 'BG002', 'lowerLimit': '2.09', 'upperLimit': '61.13'}]}]}], 'paramType': 'MEDIAN', 'unitOfMeasure': 'years', 'dispersionType': 'FULL_RANGE'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '75', 'groupId': 'BG000'}, {'value': '118', 'groupId': 'BG001'}, {'value': '193', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '94', 'groupId': 'BG000'}, {'value': '115', 'groupId': 'BG001'}, {'value': '209', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '12', 'groupId': 'BG000'}, {'value': '6', 'groupId': 'BG001'}, {'value': '18', 'groupId': 'BG002'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '157', 'groupId': 'BG000'}, {'value': '226', 'groupId': 'BG001'}, {'value': '383', 'groupId': 'BG002'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Black or African American', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '5', 'groupId': 'BG001'}, {'value': '6', 'groupId': 'BG002'}]}, {'title': 'White', 'measurements': [{'value': '164', 'groupId': 'BG000'}, {'value': '218', 'groupId': 'BG001'}, {'value': '382', 'groupId': 'BG002'}]}, {'title': 'More than one race', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '7', 'groupId': 'BG002'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Genotype', 'classes': [{'categories': [{'title': 'F508 Heterozygous', 'measurements': [{'value': '64', 'groupId': 'BG000'}, {'value': '78', 'groupId': 'BG001'}, {'value': '142', 'groupId': 'BG002'}]}, {'title': 'F508 Homozygous', 'measurements': [{'value': '89', 'groupId': 'BG000'}, {'value': '126', 'groupId': 'BG001'}, {'value': '215', 'groupId': 'BG002'}]}, {'title': 'Other/Unknown', 'measurements': [{'value': '16', 'groupId': 'BG000'}, {'value': '29', 'groupId': 'BG001'}, {'value': '45', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'description': 'Genotype was extracted by medical record review reporting prior results of tests for the diagnosis of cystic fibrosis. The variant F508del is common, and participants having one copies (F508del Heterozygous), no copies (Other), or without genetic test results (Unknown) have more heterogeneous phenotypes than those with two copies (F508del Homozygous).', 'unitOfMeasure': 'Participants'}, {'title': 'Mutation Class', 'classes': [{'categories': [{'title': 'I-III (more severe)', 'measurements': [{'value': '129', 'groupId': 'BG000'}, {'value': '185', 'groupId': 'BG001'}, {'value': '314', 'groupId': 'BG002'}]}, {'title': 'IV-V (less severe)', 'measurements': [{'value': '13', 'groupId': 'BG000'}, {'value': '17', 'groupId': 'BG001'}, {'value': '30', 'groupId': 'BG002'}]}, {'title': 'Unknown', 'measurements': [{'value': '27', 'groupId': 'BG000'}, {'value': '31', 'groupId': 'BG001'}, {'value': '58', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'description': 'Each of a patient\'s allele genotype variants were matched to traditional CFTR mutation classifications: Protein production (Class I), Protein processing (Class II), Gating (Class III), Conduction (Class IV), and Insufficient protein (Class V). The maximum class was assigned to the genotype pair and grouped by severity, with Class I-III "more severe" and Class IV-V "less severe". Unmatched variants were assigned class Unknown. F508del for example is Class II, so a participant heterozygous for F508del would have mutation Class II or greater, depending on the other allele variant\'s class.', 'unitOfMeasure': 'Participants'}, {'title': 'Sweat Chloride at Diagnosis', 'classes': [{'categories': [{'measurements': [{'value': '91.00', 'spread': '22.08', 'groupId': 'BG000'}, {'value': '97.53', 'spread': '18.05', 'groupId': 'BG001'}, {'value': '94.73', 'spread': '20.11', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'mEq/L', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Pancreatic Sufficiency', 'classes': [{'categories': [{'title': 'Insufficient', 'measurements': [{'value': '149', 'groupId': 'BG000'}, {'value': '204', 'groupId': 'BG001'}, {'value': '353', 'groupId': 'BG002'}]}, {'title': 'Sufficient', 'measurements': [{'value': '20', 'groupId': 'BG000'}, {'value': '29', 'groupId': 'BG001'}, {'value': '49', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Modulator Use', 'classes': [{'categories': [{'title': 'None', 'measurements': [{'value': '87', 'groupId': 'BG000'}, {'value': '87', 'groupId': 'BG001'}, {'value': '174', 'groupId': 'BG002'}]}, {'title': 'Ivacaftor', 'measurements': [{'value': '10', 'groupId': 'BG000'}, {'value': '10', 'groupId': 'BG001'}, {'value': '20', 'groupId': 'BG002'}]}, {'title': 'Lumacaftor/Ivacaftor', 'measurements': [{'value': '55', 'groupId': 'BG000'}, {'value': '38', 'groupId': 'BG001'}, {'value': '93', 'groupId': 'BG002'}]}, {'title': 'Tezacaftor/Ivacaftor', 'measurements': [{'value': '16', 'groupId': 'BG000'}, {'value': '87', 'groupId': 'BG001'}, {'value': '103', 'groupId': 'BG002'}]}, {'title': 'Triple Combination', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '10', 'groupId': 'BG001'}, {'value': '11', 'groupId': 'BG002'}]}, {'title': 'Ivacaftor and Triple Combination', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2018-11-19', 'size': 529457, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2023-10-30T16:18', 'hasProtocol': True}, {'date': '2019-12-16', 'size': 2730978, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2023-10-30T16:17', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 402}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2019-05-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-10', 'completionDateStruct': {'date': '2019-09-17', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-10-09', 'studyFirstSubmitDate': '2019-01-04', 'resultsFirstSubmitDate': '2023-11-07', 'studyFirstSubmitQcDate': '2019-01-09', 'lastUpdatePostDateStruct': {'date': '2024-10-15', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2024-10-09', 'studyFirstPostDateStruct': {'date': '2019-01-14', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2024-10-15', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2019-09-17', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants Who Fully Completed at Least One ePRO', 'timeFrame': '1 month', 'description': 'Number participants with scheduled outside-the-clinic assessments who fully completed at least one of the four PROs. A "fully complete" questionnaire is defined as having responded to all questions with no missing responses.'}], 'secondaryOutcomes': [{'measure': 'Number of Participants With Period-Prevalence of Constipation', 'timeFrame': '1 month', 'description': 'This table summarizes the period-prevalence of protocol-defined constipation. For each follow-up response, constipation was defined by the protocol as having fewer than 3 bowel movements and/or Bristol Stool Scale of Type 1 or 2 (hard lumps) in the past 7 days. Period prevalence is defined as occurrence of the symptom at any time from baseline to week 4. Only participants who fully completed the disease-specific questionnaire at baseline and all follow-up weeks were considered.'}, {'measure': 'Mean Patient Reported Outcome (PRO) Scores', 'timeFrame': 'At Visit 1 (1 day)', 'description': 'Mean PRO scores (PAC-SYM score range: 0-4, PAGI-SYM score range: 0-5, PAC-QOL score range: 0-4) at time of enrollment (Visit 1) where lower scores correspond to less symptom severity.'}, {'measure': 'Number of Participants Treated for GI Symptoms', 'timeFrame': 'At Visit 1 (1 day)', 'description': 'Number of enrolled participants receiving treatment for GI symptoms at Visit 1'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Cystic Fibrosis', 'CF', 'GI', 'nutrition', 'quality of life', 'bowel', 'constipation', 'gastrointestinal'], 'conditions': ['Cystic Fibrosis']}, 'referencesModule': {'references': [{'pmid': '36319569', 'type': 'DERIVED', 'citation': 'Moshiree B, Freeman AJ, Vu PT, Khan U, Ufret-Vincenty C, Heltshe SL, Goss CH, Schwarzenberg SJ, Freedman SD, Borowitz D, Sathe M; GALAXY Study Group. Multicenter prospective study showing a high gastrointestinal symptom burden in cystic fibrosis. J Cyst Fibros. 2023 Mar;22(2):266-274. doi: 10.1016/j.jcf.2022.10.006. Epub 2022 Oct 29.'}]}, 'descriptionModule': {'briefSummary': 'This is a prospective, multicenter, observational study designed to collect gastrointestinal related data in patients with Cystic Fibrosis (CF).', 'detailedDescription': 'There are currently no large, multicenter prospective clinical trials examining management of constipation or other gastrointestinal (GI) symptoms in people with cystic fibrosis (CF). Current recommendations in the CF literature are largely based on expert consensus and opinions. Yet, constipation and other GI symptoms are crucial factors in quality of life (QOL) and maintenance of optimal nutritional state in people with CF. This study will use GI-symptomatology questionnaires to understand the multiple overlapping GI symptoms in people with CF.\n\nEligible subjects will be consented and enrolled in the study at the Enrollment Visit. At the visit, the subject or parent/guardian will complete the patient reported outcome surveys (PROs) using a mobile device (e.g., smartphone or tablet). The same questionnaires will be completed on a mobile device outside the clinic three additional times. The PROs will consist of four questionnaires: Patient Assessment of Constipation Symptoms (PAC-SYM), Patient Assessment of Gastrointestinal Symptoms (PAGI-SYM), Patient Assessment of Constipation Quality of Life (PAC-QOL) and a disease-specific questionnaire (Bristol Stool Scale and questions about fecal incontinence, and stool quality and frequency).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '2 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Subjects with a diagnosis of Cystic Fibrosis (CF) who meet all the inclusion and none of the exclusion criteria will be eligible for participation in this study.', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n1. All genders ≥ 2 years of age at time of consent\n2. Documentation of a Cystic Fibrosis (CF) diagnosis as evidenced by one or more clinical features consistent with the CF phenotype and one or more of the following criteria:\n\n * Sweat chloride equal to or greater than 60 milliequivalent (mEq)/liter by quantitative pilocarpine iontophoresis test (QPIT)\n * Two well-characterized mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene\n * Abnormal nasal potential difference (NPD) (change in NPD in response to a low chloride solution and isoproterenol of less than -5 mV)\n3. Enrolled in the Cystic Fibrosis Foundation Patient Registry (subjects may enroll in the Registry at Enrollment Visit if not previously enrolled)\n4. Written informed consent (and assent when applicable) obtained from subject or subject's legal representative\n5. Willing to complete questionnaires on mobile device\n6. Able to use the Medidata Patient Cloud mobile application for completing the questionnaires\n\nExclusion Criteria:\n\n1. Presence of a condition or abnormality that, in the opinion of the Investigator, would complicate interpretation of study outcome data or interfere with achieving the study objectives\n2. Presence of a pulmonary exacerbation at the Enrollment Visit\n3. Hospitalization for distal intestinal obstruction syndrome (DIOS) within the 28 days prior to the Enrollment Visit\n4. Current gastrointestinal (GI) or abdominal/pelvic malignancy\n5. Abdominal or pelvic surgery within the 28 days prior to the Enrollment Visit\n6. At the time of the Enrollment Visit, planned abdominal or pelvic surgery or bowel cleanout in the 28 days after the Enrollment Visit\n7. Initiation of new CFTR modulator therapy within the 4 weeks prior to the Enrollment Visit\n8. Intent to initiate new CFTR modulator therapy within 28 days of the Enrollment Visit"}, 'identificationModule': {'nctId': 'NCT03801993', 'acronym': 'GALAXY', 'briefTitle': 'Multicenter Study of Patient-reported Gastrointestinal Symptoms in People With Cystic Fibrosis', 'organization': {'class': 'OTHER', 'fullName': "Seattle Children's Hospital"}, 'officialTitle': 'Multicenter Study of Patient-reported Gastrointestinal Symptoms in People With Cystic Fibrosis (GALAXY-OB-18)', 'orgStudyIdInfo': {'id': 'GALAXY-OB-18'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'All Subjects', 'description': 'Subjects with a diagnosis of Cystic Fibrosis (CF) who meet all the inclusion and none of the exclusion criteria will be eligible for participation in this study.'}]}, 'contactsLocationsModule': {'locations': [{'zip': '94304', 'city': 'Palo Alto', 'state': 'California', 'country': 'United States', 'facility': 'Stanford University Medical Center', 'geoPoint': {'lat': 37.44188, 'lon': -122.14302}}, {'zip': '06106', 'city': 'Hartford', 'state': 'Connecticut', 'country': 'United States', 'facility': 'Central Connecticut Cystic Fibrosis Center', 'geoPoint': {'lat': 41.76371, 'lon': -72.68509}}, {'zip': '20816', 'city': 'Washington D.C.', 'state': 'District of Columbia', 'country': 'United States', 'facility': "Children's National Medical Center", 'geoPoint': {'lat': 38.89511, 'lon': -77.03637}}, {'zip': '32514', 'city': 'Pensacola', 'state': 'Florida', 'country': 'United States', 'facility': "Nemours Children's Clinic - Pensacola", 'geoPoint': {'lat': 30.42131, 'lon': -87.21691}}, {'zip': '33701', 'city': 'St. Petersburg', 'state': 'Florida', 'country': 'United States', 'facility': "All Children's Hospital", 'geoPoint': {'lat': 27.77086, 'lon': -82.67927}}, {'zip': '30322', 'city': 'Atlanta', 'state': 'Georgia', 'country': 'United States', 'facility': "Children's Healthcare of Atlanta and Emory University", 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'zip': '60611', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': 'Northwestern University', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '61637', 'city': 'Peoria', 'state': 'Illinois', 'country': 'United States', 'facility': 'OSF Saint Francis Medical Center', 'geoPoint': {'lat': 40.69365, 'lon': -89.58899}}, {'zip': '46202', 'city': 'Indianapolis', 'state': 'Indiana', 'country': 'United States', 'facility': 'Riley Hospital for Children', 'geoPoint': {'lat': 39.76838, 'lon': -86.15804}}, {'zip': '40506-9983', 'city': 'Lexington', 'state': 'Kentucky', 'country': 'United States', 'facility': 'University of Kentucky', 'geoPoint': {'lat': 37.98869, 'lon': -84.47772}}, {'zip': '04102', 'city': 'Portland', 'state': 'Maine', 'country': 'United States', 'facility': 'Maine Medical Center', 'geoPoint': {'lat': 43.65737, 'lon': -70.2589}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': "Boston Children's Hospital, Brigham & Women's Hospital", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '01655', 'city': 'Worcester', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'University of Massachusetts Memorial Health Care', 'geoPoint': {'lat': 42.26259, 'lon': -71.80229}}, {'zip': '49503', 'city': 'Grand Rapids', 'state': 'Michigan', 'country': 'United States', 'facility': "Helen DeVos Children's Hospital", 'geoPoint': {'lat': 42.96336, 'lon': -85.66809}}, {'zip': '55455', 'city': 'Minneapolis', 'state': 'Minnesota', 'country': 'United States', 'facility': 'The Minnesota Cystic Fibrosis Center', 'geoPoint': {'lat': 44.97997, 'lon': -93.26384}}, {'zip': '63104', 'city': 'St Louis', 'state': 'Missouri', 'country': 'United States', 'facility': "SSM Health Cardinal Glennon Children's Hospital", 'geoPoint': {'lat': 38.62727, 'lon': -90.19789}}, {'zip': '08903', 'city': 'New Brunswick', 'state': 'New Jersey', 'country': 'United States', 'facility': 'Rutgers - Robert Wood Johnson Medical School', 'geoPoint': {'lat': 40.48622, 'lon': -74.45182}}, {'zip': '28203', 'city': 'Charlotte', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Atrium Health Pulmonary Care', 'geoPoint': {'lat': 35.22709, 'lon': -80.84313}}, {'zip': '27103', 'city': 'Winston-Salem', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Wake Forest University Baptist Medical Center', 'geoPoint': {'lat': 36.09986, 'lon': -80.24422}}, {'zip': '43205', 'city': 'Columbus', 'state': 'Ohio', 'country': 'United States', 'facility': "Nationwide Children's Hospital", 'geoPoint': {'lat': 39.96118, 'lon': -82.99879}}, {'zip': '73104', 'city': 'Oklahoma City', 'state': 'Oklahoma', 'country': 'United States', 'facility': 'Oklahoma Cystic Fibrosis Center', 'geoPoint': {'lat': 35.46756, 'lon': -97.51643}}, {'zip': '97239', 'city': 'Portland', 'state': 'Oregon', 'country': 'United States', 'facility': 'Oregon Health Sciences University', 'geoPoint': {'lat': 45.52345, 'lon': -122.67621}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': "Children's Hospital of Philadelphia", 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '75247', 'city': 'Dallas', 'state': 'Texas', 'country': 'United States', 'facility': "University of Texas Southwestern / Children's Health", 'geoPoint': {'lat': 32.78306, 'lon': -96.80667}}, {'zip': '84113', 'city': 'Salt Lake City', 'state': 'Utah', 'country': 'United States', 'facility': 'Intermountain Cystic Fibrosis Center', 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'zip': '05401', 'city': 'Burlington', 'state': 'Vermont', 'country': 'United States', 'facility': "The Children's Specialty Center Fletcher Allen Health Care", 'geoPoint': {'lat': 44.47588, 'lon': -73.21207}}], 'overallOfficials': [{'name': 'A. Jay Freeman, MD, MSc', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': "Emory University/Children's Healthcare"}, {'name': 'Baharak Moshiree, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'University of North Carolina Charlotte, Atrium Health'}, {'name': 'Meghana Sathe, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': "University of Texas Southwestern/Children's Health"}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Chris Goss', 'class': 'OTHER'}, 'collaborators': [{'name': 'Emory University', 'class': 'OTHER'}, {'name': 'University of North Carolina, Charlotte', 'class': 'OTHER'}, {'name': 'University of Texas', 'class': 'OTHER'}, {'name': 'Cystic Fibrosis Foundation', 'class': 'OTHER'}], 'responsibleParty': {'type': 'SPONSOR_INVESTIGATOR', 'investigatorTitle': 'Professor of Medicine and Pediatrics', 'investigatorFullName': 'Chris Goss', 'investigatorAffiliation': "Seattle Children's Hospital"}}}}