Ignite Creation Date:
2025-12-25 @ 5:09 AM
Ignite Modification Date:
2025-12-26 @ 4:13 AM
Study NCT ID:
NCT04132427
Status:
COMPLETED
Last Update Posted:
2024-08-26
First Post:
2019-10-11
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
MTT for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'submissionTracking': {'firstMcpInfo': {'postDateStruct': {'date': '2024-04-18', 'type': 'ACTUAL'}}}}, 'conditionBrowseModule': {'meshes': [{'id': 'C537403', 'term': 'Pitt-Hopkins syndrome'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D014640', 'term': 'Vancomycin'}, {'id': 'C110422', 'term': 'magnesium citrate'}, {'id': 'D064307', 'term': 'Microbiota'}], 'ancestors': [{'id': 'D006020', 'term': 'Glycopeptides'}, {'id': 'D006001', 'term': 'Glycoconjugates'}, {'id': 'D002241', 'term': 'Carbohydrates'}, {'id': 'D010455', 'term': 'Peptides'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}, {'id': 'D008827', 'term': 'Microbiological Phenomena'}, {'id': 'D058448', 'term': 'Biota'}, {'id': 'D044822', 'term': 'Biodiversity'}, {'id': 'D017753', 'term': 'Ecosystem'}, {'id': 'D004777', 'term': 'Environment'}, {'id': 'D055669', 'term': 'Ecological and Environmental Phenomena'}, {'id': 'D001686', 'term': 'Biological Phenomena'}, {'id': 'D004778', 'term': 'Environment and Public Health'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'jim.adams@asu.edu', 'phone': '480 965-3316', 'title': 'James B. Adams', 'organization': 'Arizona State University'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}}, 'adverseEventsModule': {'timeFrame': 'AE data reported here is from the start of the treatment to the end of treatment (approximately 14 weeks).', 'description': 'We use standard definition of the Adverse events and Serious Adverse events.', 'eventGroups': [{'id': 'EG000', 'title': 'Group A: Treatment', 'description': 'Vancomycin, magnesium citrate, microbiota\n\nvancomycin, magnesium citrate, microbiota: 10 days of oral vancomycin, then 1 day of oral magnesium citrate, , then 4 days of high-dose oral microbiota, followed by 12 weeks of low-dose oral microbiota', 'otherNumAtRisk': 3, 'deathsNumAtRisk': 3, 'otherNumAffected': 3, 'seriousNumAtRisk': 3, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'Group B: Placebo', 'description': 'placebo vancomycin, real magnesium citrate (because it obviously empties the bowels) and placebo microbiota\n\nplacebo vancomycin, real magnesium citrate, placebo microbiota: 10 days of oral placebo vancomycin, then 1 day of oral real magnesium citrate, , then 4 days of high-dose oral placebo microbiota, followed by 12 weeks of low-dose oral placebo microbiota', 'otherNumAtRisk': 3, 'deathsNumAtRisk': 3, 'otherNumAffected': 3, 'seriousNumAtRisk': 3, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Hyperactivity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Irritability', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Gassy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Insomnia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Loss of appetite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Diarrhea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Redness- Genitalia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Change in Stool Color', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Abdominal Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Bloating', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 12.0'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Daily Stool Record (DSR(', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Group A: Treatment', 'description': 'Vancomycin, magnesium citrate, microbiota\n\nvancomycin, magnesium citrate, microbiota: 10 days of oral vancomycin, then 1 day of oral magnesium citrate, , then 4 days of high-dose oral microbiota, followed by 12 weeks of low-dose oral microbiota'}, {'id': 'OG001', 'title': 'Group B: Placebo', 'description': 'placebo vancomycin, real magnesium citrate (because it obviously empties the bowels) and placebo microbiota\n\nplacebo vancomycin, real magnesium citrate, placebo microbiota: 10 days of oral placebo vancomycin, then 1 day of oral real magnesium citrate, , then 4 days of high-dose oral placebo microbiota, followed by 12 weeks of low-dose oral placebo microbiota'}], 'classes': [{'categories': [{'measurements': [{'value': '-2.3', 'spread': '2.5', 'groupId': 'OG000'}, {'value': '0', 'spread': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'change in % abnormal days from baseline (for 2 weeks) vs. week 14 (2 weeks from week 13-14)', 'description': 'The DSR is a daily record of their bowel movements including Bristol Stool Form scale. It is rated as the number of days (out of 14 days) with an abnormal report (abnormal stool, no stool, or the use of a gastrointestinal treatment). A higher percentage indicates worse symptoms.', 'unitOfMeasure': 'days', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'PRIMARY', 'title': 'Safety Measures', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Group A: Treatment', 'description': 'Vancomycin, magnesium citrate, microbiota\n\nvancomycin, magnesium citrate, microbiota: 10 days of oral vancomycin, then 1 day of oral magnesium citrate, , then 4 days of high-dose oral microbiota, followed by 12 weeks of low-dose oral microbiota'}, {'id': 'OG001', 'title': 'Group B: Placebo', 'description': 'placebo vancomycin, real magnesium citrate (because it obviously empties the bowels) and placebo microbiota\n\nplacebo vancomycin, real magnesium citrate, placebo microbiota: 10 days of oral placebo vancomycin, then 1 day of oral real magnesium citrate, , then 4 days of high-dose oral placebo microbiota, followed by 12 weeks of low-dose oral placebo microbiota'}], 'classes': [{'categories': [{'measurements': [{'value': '8', 'groupId': 'OG000'}, {'value': '10', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'weeks 0-14', 'description': 'number of adverse events and serious adverse events likely associated with treatment', 'unitOfMeasure': 'number of AEs among all participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'CGI for GI Disorders', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Group A: Treatment', 'description': 'Vancomycin, magnesium citrate, microbiota\n\nvancomycin, magnesium citrate, microbiota: 10 days of oral vancomycin, then 1 day of oral magnesium citrate, , then 4 days of high-dose oral microbiota, followed by 12 weeks of low-dose oral microbiota'}, {'id': 'OG001', 'title': 'Group B: Placebo', 'description': 'placebo vancomycin, real magnesium citrate (because it obviously empties the bowels) and placebo microbiota\n\nplacebo vancomycin, real magnesium citrate, placebo microbiota: 10 days of oral placebo vancomycin, then 1 day of oral real magnesium citrate, , then 4 days of high-dose oral placebo microbiota, followed by 12 weeks of low-dose oral placebo microbiota'}], 'classes': [{'categories': [{'measurements': [{'value': '-1', 'spread': '1', 'groupId': 'OG000'}, {'value': '-.67', 'spread': '1.15', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'change in score between baseline and week 14', 'description': 'Clinical Global Impressions of GI Disorders, including severity, change in severity, and side effects. The severity is rated on a scale of 1-7, with 1 being normal and 7 being the worst possible.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'CGI for PTHS Symptoms', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Group A: Treatment', 'description': 'Vancomycin, magnesium citrate, microbiota\n\nvancomycin, magnesium citrate, microbiota: 10 days of oral vancomycin, then 1 day of oral magnesium citrate, , then 4 days of high-dose oral microbiota, followed by 12 weeks of low-dose oral microbiota'}, {'id': 'OG001', 'title': 'Group B: Placebo', 'description': 'placebo vancomycin, real magnesium citrate (because it obviously empties the bowels) and placebo microbiota\n\nplacebo vancomycin, real magnesium citrate, placebo microbiota: 10 days of oral placebo vancomycin, then 1 day of oral real magnesium citrate, , then 4 days of high-dose oral placebo microbiota, followed by 12 weeks of low-dose oral placebo microbiota'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'spread': '0', 'groupId': 'OG000'}, {'value': '-.33', 'spread': '.58', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'change in score between baseline and week 14', 'description': 'Clinical Global Impressions of Pitt Hopkins Syndrome symptoms, including severity, change in severity, and side effects. The severity is rated on a scale of 1-7, with 1 being normal and 7 being the worst possible.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'PGI-PTHS', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Group A: Treatment', 'description': 'Vancomycin, magnesium citrate, microbiota\n\nvancomycin, magnesium citrate, microbiota: 10 days of oral vancomycin, then 1 day of oral magnesium citrate, , then 4 days of high-dose oral microbiota, followed by 12 weeks of low-dose oral microbiota'}, {'id': 'OG001', 'title': 'Group B: Placebo', 'description': 'placebo vancomycin, real magnesium citrate (because it obviously empties the bowels) and placebo microbiota\n\nplacebo vancomycin, real magnesium citrate, placebo microbiota: 10 days of oral placebo vancomycin, then 1 day of oral real magnesium citrate, , then 4 days of high-dose oral placebo microbiota, followed by 12 weeks of low-dose oral placebo microbiota'}], 'classes': [{'categories': [{'measurements': [{'value': '.68', 'spread': '.97', 'groupId': 'OG000'}, {'value': '.29', 'spread': '.44', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'change in score between baseline and week 14', 'description': 'Parent Global Impressions of Pitt Hopkins Syndrome symptoms. This is a rating of 29 symptoms, and an average is computed. The scale ranges from -3 (much worse) to +3 (much better).', 'unitOfMeasure': '-3 to +3', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'GSRS', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Group A: Treatment', 'description': 'Vancomycin, magnesium citrate, microbiota\n\nvancomycin, magnesium citrate, microbiota: 10 days of oral vancomycin, then 1 day of oral magnesium citrate, , then 4 days of high-dose oral microbiota, followed by 12 weeks of low-dose oral microbiota'}, {'id': 'OG001', 'title': 'Group B: Placebo', 'description': 'placebo vancomycin, real magnesium citrate (because it obviously empties the bowels) and placebo microbiota\n\nplacebo vancomycin, real magnesium citrate, placebo microbiota: 10 days of oral placebo vancomycin, then 1 day of oral real magnesium citrate, , then 4 days of high-dose oral placebo microbiota, followed by 12 weeks of low-dose oral placebo microbiota'}], 'classes': [{'categories': [{'measurements': [{'value': '-2.3', 'spread': '.87', 'groupId': 'OG000'}, {'value': '-1.07', 'spread': '1.91', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'change in score between baseline and week 14', 'description': 'Gastrointestinal Symptom Rating Scale. This is a 15-item questionnaire, with each item rated on a scale of 1 (no symptoms) to 7 (very severe discomfort). The average score for all 15 items is reported here.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'FLACC', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Group A: Treatment', 'description': 'Vancomycin, magnesium citrate, microbiota\n\nvancomycin, magnesium citrate, microbiota: 10 days of oral vancomycin, then 1 day of oral magnesium citrate, , then 4 days of high-dose oral microbiota, followed by 12 weeks of low-dose oral microbiota'}, {'id': 'OG001', 'title': 'Group B: Placebo', 'description': 'placebo vancomycin, real magnesium citrate (because it obviously empties the bowels) and placebo microbiota\n\nplacebo vancomycin, real magnesium citrate, placebo microbiota: 10 days of oral placebo vancomycin, then 1 day of oral real magnesium citrate, , then 4 days of high-dose oral placebo microbiota, followed by 12 weeks of low-dose oral placebo microbiota'}], 'classes': [{'categories': [{'measurements': [{'value': '-4.0', 'spread': '2.65', 'groupId': 'OG000'}, {'value': '-2.67', 'spread': '2.08', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'change in score between baseline and week 14', 'description': 'Revised Face Legs Activity Crying Consolability Pain Questionnaire for Children with Cognitive Impairment (FLACC). This is a rating scale of 5 symptoms of pain, with each item rated on a scale of 0 (no symptom) to 2 (maximum symptom), and the scores for each item are summed to create a total score (zero to 10).', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Group A: Treatment', 'description': 'Vancomycin, magnesium citrate, microbiota\n\nvancomycin, magnesium citrate, microbiota: 10 days of oral vancomycin, then 1 day of oral magnesium citrate, , then 4 days of high-dose oral microbiota, followed by 12 weeks of low-dose oral microbiota'}, {'id': 'FG001', 'title': 'Group B: Placebo', 'description': 'placebo vancomycin, real magnesium citrate (because it obviously empties the bowels) and placebo microbiota\n\nplacebo vancomycin, real magnesium citrate, placebo microbiota: 10 days of oral placebo vancomycin, then 1 day of oral real magnesium citrate, , then 4 days of high-dose oral placebo microbiota, followed by 12 weeks of low-dose oral placebo microbiota'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '3'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '3'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '6', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Group A: Treatment', 'description': 'Vancomycin, magnesium citrate, microbiota\n\nvancomycin, magnesium citrate, microbiota: 10 days of oral vancomycin, then 1 day of oral magnesium citrate, , then 4 days of high-dose oral microbiota, followed by 12 weeks of low-dose oral microbiota'}, {'id': 'BG001', 'title': 'Group B: Placebo', 'description': 'placebo vancomycin, real magnesium citrate (because it obviously empties the bowels) and placebo microbiota\n\nplacebo vancomycin, real magnesium citrate, placebo microbiota: 10 days of oral placebo vancomycin, then 1 day of oral real magnesium citrate, , then 4 days of high-dose oral placebo microbiota, followed by 12 weeks of low-dose oral placebo microbiota'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '6', 'groupId': 'BG002'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', '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': '13.0', 'spread': '3.6', 'groupId': 'BG000'}, {'value': '12.7', 'spread': '3.1', 'groupId': 'BG001'}, {'value': '12.8', 'spread': '3.0', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '5', 'groupId': 'BG002'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', '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': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', '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': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'White', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '6', 'groupId': 'BG002'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '6', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Daily Stool Record', 'classes': [{'categories': [{'measurements': [{'value': '14', 'spread': '0', 'groupId': 'BG000'}, {'value': '14', 'spread': '0', 'groupId': 'BG001'}, {'value': '14', 'spread': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'Number of abnormal days out of 14 days', 'dispersionType': 'STANDARD_DEVIATION'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2022-03-28', 'size': 619648, 'label': 'Study Protocol and Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'Prot_SAP_000.pdf', 'typeAbbrev': 'Prot_SAP', 'uploadDate': '2024-02-05T12:11', 'hasProtocol': True}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR'], 'maskingDescription': 'Part 1 is double-blind Parts 2 and 3 are single-blind (outcomes assessor is blinded)'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'Part 1: Randomized, double-blind, placebo-controlled Part 2: Treatment group enters observation, placebo group switched to treatment Part 3: Long-term observation'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 6}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2019-09-30', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-03', 'completionDateStruct': {'date': '2022-04-15', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-07-31', 'studyFirstSubmitDate': '2019-10-11', 'resultsFirstSubmitDate': '2024-03-25', 'studyFirstSubmitQcDate': '2019-10-17', 'lastUpdatePostDateStruct': {'date': '2024-08-26', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2024-07-31', 'studyFirstPostDateStruct': {'date': '2019-10-18', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2024-08-26', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-03-17', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Daily Stool Record (DSR(', 'timeFrame': 'change in % abnormal days from baseline (for 2 weeks) vs. week 14 (2 weeks from week 13-14)', 'description': 'The DSR is a daily record of their bowel movements including Bristol Stool Form scale. It is rated as the number of days (out of 14 days) with an abnormal report (abnormal stool, no stool, or the use of a gastrointestinal treatment). A higher percentage indicates worse symptoms.'}, {'measure': 'Safety Measures', 'timeFrame': 'weeks 0-14', 'description': 'number of adverse events and serious adverse events likely associated with treatment'}], 'secondaryOutcomes': [{'measure': 'CGI for GI Disorders', 'timeFrame': 'change in score between baseline and week 14', 'description': 'Clinical Global Impressions of GI Disorders, including severity, change in severity, and side effects. The severity is rated on a scale of 1-7, with 1 being normal and 7 being the worst possible.'}, {'measure': 'CGI for PTHS Symptoms', 'timeFrame': 'change in score between baseline and week 14', 'description': 'Clinical Global Impressions of Pitt Hopkins Syndrome symptoms, including severity, change in severity, and side effects. The severity is rated on a scale of 1-7, with 1 being normal and 7 being the worst possible.'}, {'measure': 'PGI-PTHS', 'timeFrame': 'change in score between baseline and week 14', 'description': 'Parent Global Impressions of Pitt Hopkins Syndrome symptoms. This is a rating of 29 symptoms, and an average is computed. The scale ranges from -3 (much worse) to +3 (much better).'}, {'measure': 'GSRS', 'timeFrame': 'change in score between baseline and week 14', 'description': 'Gastrointestinal Symptom Rating Scale. This is a 15-item questionnaire, with each item rated on a scale of 1 (no symptoms) to 7 (very severe discomfort). The average score for all 15 items is reported here.'}, {'measure': 'FLACC', 'timeFrame': 'change in score between baseline and week 14', 'description': 'Revised Face Legs Activity Crying Consolability Pain Questionnaire for Children with Cognitive Impairment (FLACC). This is a rating scale of 5 symptoms of pain, with each item rated on a scale of 0 (no symptom) to 2 (maximum symptom), and the scores for each item are summed to create a total score (zero to 10).'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['microbiota transfer therapy', 'fecal transplant', 'fecal microbiota transplant', 'intestinal microbiota'], 'conditions': ['Pitt Hopkins Syndrome']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'https://autism.asu.edu', 'label': 'link for our research group'}]}, 'descriptionModule': {'briefSummary': 'The investigators propose to investigate Microbiota Transfer Therapy (MTT) for treating patients with Pitt Hopkins Syndrome (PTHS) and gastrointestinal problems similar to Irritable Bowel Syndrome (IBS). MTT involves a combination of 10 days of oral vancomycin (an antibiotic to kill pathogenic bacteria), followed by a bowel cleanse, followed by 12 weeks of Fecal Microbiota (FM).', 'detailedDescription': 'For children ages 5-17 years with PTHS and gastrointestinal problems, a Phase 2 clinical trial will evaluate the safety, tolerability, and efficacy of MTT.\n\nRandomized, double-blind, placebo-Controlled Treatment (14 weeks)\n\nThe trial will be a randomized, double-blind, placebo-controlled trial which will include a 10-day treatment with oral vancomycin (or placebo), then 1 day of magnesium citrate to cleanse the bowel of vancomycin and bacteria/feces (all participants, since its bowel-emptying effect cannot be blinded), followed by oral administration of FM (or placebo). An initial high dose of FM (or placebo) for two days will be followed by a lower maintenance dose of FM (or placebo) for 12 weeks.\n\nGroup A: real treatment Group B: placebo vancomycin, real magnesium citrate, placebo FM'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '17 Years', 'minimumAge': '7 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n1. Children ages 7-17 years with Pitt Hopkins Syndrome (verified by genetic testing)\n2. GI disorder as defined below that has lasted for at least 2 years.\n3. No changes in medications, supplements, diet, or therapies in last 2 months, and no intention to change them during the Parts 1 and 2 of the clinical trial.\n4. Ability to swallow pills (without chewing)\n5. Review of last two years of medical records by the study physician.\n\nExclusion Criteria:\n\n1. Antibiotics in last 3 months\n2. Probiotics in last 2 months, or fecal transplant in last 12 months\n3. Tube feeding\n4. Severe gastrointestinal problems that require immediate treatment (life-threatening)\n5. Ulcerative Colitis, Crohn's Disease, diagnosed Celiac Disease, Eosinophilic Gastroenteritis, or similar conditions\n6. Unstable, poor health (based on study physician's opinion)\n7. Recent or scheduled surgeries\n8. Current participation in other clinical trials\n9. Females who are pregnant or who are at risk of pregnancy and sexually active without effective birth control.\n10. Allergy or intolerance to vancomycin or magnesium citrate\n11. Clinically significant abnormalities at baseline on two blood safety tests: Comprehensive Metabolic Panel, and Complete Blood Count with Differential.\n12. Evidence of significant impairment of immune system, or taking medications that can compromise the immune system, and thus increase risk if exposed to multiple-drug resistant bacteria."}, 'identificationModule': {'nctId': 'NCT04132427', 'briefTitle': 'MTT for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders', 'organization': {'class': 'OTHER', 'fullName': 'Arizona State University'}, 'officialTitle': 'Microbiota Transfer Therapy for Children With Both Pitt Hopkins Syndrome and Gastrointestinal Disorders', 'orgStudyIdInfo': {'id': 'IRB#: 120190263'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Group A: Treatment', 'description': 'Vancomycin, magnesium citrate, microbiota', 'interventionNames': ['Combination Product: vancomycin, magnesium citrate, microbiota']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Group B: Placebo', 'description': 'placebo vancomycin, real magnesium citrate (because it obviously empties the bowels) and placebo microbiota', 'interventionNames': ['Combination Product: placebo vancomycin, real magnesium citrate, placebo microbiota']}], 'interventions': [{'name': 'vancomycin, magnesium citrate, microbiota', 'type': 'COMBINATION_PRODUCT', 'description': '10 days of oral vancomycin, then 1 day of oral magnesium citrate, , then 4 days of high-dose oral microbiota, followed by 12 weeks of low-dose oral microbiota', 'armGroupLabels': ['Group A: Treatment']}, {'name': 'placebo vancomycin, real magnesium citrate, placebo microbiota', 'type': 'COMBINATION_PRODUCT', 'description': '10 days of oral placebo vancomycin, then 1 day of oral real magnesium citrate, , then 4 days of high-dose oral placebo microbiota, followed by 12 weeks of low-dose oral placebo microbiota', 'armGroupLabels': ['Group B: Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '85284', 'city': 'Tempe', 'state': 'Arizona', 'country': 'United States', 'facility': 'Arizona State University', 'geoPoint': {'lat': 33.41477, 'lon': -111.90931}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Arizona State University', 'class': 'OTHER'}, 'collaborators': [{'name': 'Pitt Hopkins Research Foundation', 'class': 'OTHER'}], 'responsibleParty': {'type': 'SPONSOR'}}}}