Ignite Creation Date:
2025-12-24 @ 1:49 PM
Ignite Modification Date:
2026-01-04 @ 12:24 PM
Study NCT ID:
NCT00676195
Status:
COMPLETED
Last Update Posted:
2017-03-29
First Post:
2008-05-07
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
An Open-Label Study of N-Acetyl Cysteine in Children With Autism
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D001321', 'term': 'Autistic Disorder'}], 'ancestors': [{'id': 'D000067877', 'term': 'Autism Spectrum Disorder'}, {'id': 'D002659', 'term': 'Child Development Disorders, Pervasive'}, {'id': 'D065886', 'term': 'Neurodevelopmental Disorders'}, {'id': 'D001523', 'term': 'Mental Disorders'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D000111', 'term': 'Acetylcysteine'}], 'ancestors': [{'id': 'D003545', 'term': 'Cysteine'}, {'id': 'D000603', 'term': 'Amino Acids, Sulfur'}, {'id': 'D013457', 'term': 'Sulfur Compounds'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D000596', 'term': 'Amino Acids'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'hardanay@stanford.edu', 'phone': '650-736-1235', 'title': 'Antonio Hardan, MD', 'organization': 'Stanford University School of Medicine'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}, 'limitationsAndCaveats': {'description': 'This is an open-label trial with a small sample size.'}}, 'adverseEventsModule': {'timeFrame': 'Baseline and 4, 8 and 12 Weeks.', 'eventGroups': [{'id': 'EG000', 'title': 'N-Acetyl Cysteine', 'description': 'N-Acetyl Cysteine: Dosage of orally administered N-Acetyl Cysteine is as follows:\n\nDays 1-30: 900 mg, once per day Days 31-60: 900 mg, twice per day Days 61-90: 900 mg, three times per day', 'otherNumAtRisk': 24, 'otherNumAffected': 19, 'seriousNumAtRisk': 24, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Nausea/Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 24, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Diarrhea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 24, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Decreased Appetite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 24, 'numEvents': 6, 'numAffected': 5}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Depressive Affect', 'stats': [{'groupId': 'EG000', 'numAtRisk': 24, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Nasal Congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 24, 'numEvents': 6, 'numAffected': 6}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Insomnia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 24, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Decreased Motor Activity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 24, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Grinding Teeth', 'stats': [{'groupId': 'EG000', 'numAtRisk': 24, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Number of Participants With Adverse Events Reported on the Dosage Record and Treatment Emergent Symptom Scale (DOTES)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '24', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'N-Acetyl Cysteine', 'description': 'N-Acetyl Cysteine: Dosage of orally administered N-Acetyl Cysteine is as follows:\n\nDays 1-30: 900 mg, once per day Days 31-60: 900 mg, twice per day Days 61-90: 900 mg, three times per day'}], 'classes': [{'categories': [{'measurements': [{'value': '17', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '4, 8, and 12 weeks', 'description': 'Number of Participants with adverse events reported on the Dosage Record and Treatment Emergent Symptom Scale (DOTES) during the course of the study as measured at time points (4, 8, and 12 weeks).', 'unitOfMeasure': 'number of participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Social Responsiveness Scale (SRS)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Open-Label'}], 'timeFrame': '12 weeks', 'reportingStatus': 'POSTED', 'populationDescription': 'No analyses were conducted since it is an open-label trial and data was analyzed in the double-blind phase. Additionally, the main reason for the this phase is to allow participants who were on the placebo during the double-blind phase of NCT00627705 to receive the compound.'}, {'type': 'SECONDARY', 'title': 'Sensory Profile Questionnaire (SPQ)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Open-Label'}], 'timeFrame': '12 weeks', 'reportingStatus': 'POSTED', 'populationDescription': 'No analyses were conducted since it is an open-label trial and data was analyzed in the double-blind phase. Additionally, the main reason for the this phase is to allow participants who were on the placebo during the double-blind phase of NCT00627705 to receive the compound.'}, {'type': 'SECONDARY', 'title': 'Irritability Subscale of the Aberrant Behavior Checklist (ABC)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Open-Label'}], 'timeFrame': '4, 8, and 12 weeks', 'reportingStatus': 'POSTED', 'populationDescription': 'No analyses were conducted since it is an open-label trial and data was analyzed in the double-blind phase. Additionally, the main reason for the this phase is to allow participants who were on the placebo during the double-blind phase of NCT00627705 to receive the compound.'}, {'type': 'SECONDARY', 'title': 'Glutathione Metabolism Intermediates in Peripheral Blood Measured by High-performance Liquid Chromatography', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Open-Label'}], 'timeFrame': '12 weeks', 'reportingStatus': 'POSTED', 'populationDescription': 'No analyses were conducted since it is an open-label trial and data was analyzed in the double-blind phase. Additionally, the main reason for the this phase is to allow participants who were on the placebo during the double-blind phase of NCT00627705 to receive the compound.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'N-Acetyl Cysteine', 'description': 'N-Acetyl Cysteine: Dosage of orally administered N-Acetyl Cysteine is as follows:\n\nDays 1-30: 900 mg, once per day Days 31-60: 900 mg, twice per day Days 61-90: 900 mg, three times per day'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '24'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '21'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '3'}]}], 'dropWithdraws': [{'type': 'Lost to Follow-up', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}, {'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}, {'type': 'Physician Decision', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '24', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'N-Acetyl Cysteine', 'description': 'N-Acetyl Cysteine: Dosage of orally administered N-Acetyl Cysteine is as follows:\n\nDays 1-30: 900 mg, once per day Days 31-60: 900 mg, twice per day Days 61-90: 900 mg, three times per day'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '24', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '22', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '24', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 24}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2008-06'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2017-02', 'completionDateStruct': {'date': '2010-06', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2017-02-08', 'studyFirstSubmitDate': '2008-05-07', 'resultsFirstSubmitDate': '2016-08-12', 'studyFirstSubmitQcDate': '2008-05-09', 'lastUpdatePostDateStruct': {'date': '2017-03-29', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2017-02-08', 'studyFirstPostDateStruct': {'date': '2008-05-12', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2017-03-29', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2010-06', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants With Adverse Events Reported on the Dosage Record and Treatment Emergent Symptom Scale (DOTES)', 'timeFrame': '4, 8, and 12 weeks', 'description': 'Number of Participants with adverse events reported on the Dosage Record and Treatment Emergent Symptom Scale (DOTES) during the course of the study as measured at time points (4, 8, and 12 weeks).'}], 'secondaryOutcomes': [{'measure': 'Social Responsiveness Scale (SRS)', 'timeFrame': '12 weeks'}, {'measure': 'Sensory Profile Questionnaire (SPQ)', 'timeFrame': '12 weeks'}, {'measure': 'Irritability Subscale of the Aberrant Behavior Checklist (ABC)', 'timeFrame': '4, 8, and 12 weeks'}, {'measure': 'Glutathione Metabolism Intermediates in Peripheral Blood Measured by High-performance Liquid Chromatography', 'timeFrame': '12 weeks'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['N-Acetyl Cysteine'], 'conditions': ['Autistic Disorder']}, 'descriptionModule': {'briefSummary': 'The purpose of the study is to test the tolerability and efficacy of N-Acetyl Cysteine (NAC) in children with Autism.', 'detailedDescription': "N-Acetyl Cysteine (NAC) is a compound that increases the levels of Glutathione, the body's main antioxidant. Glutathione is a compound in the blood that is part of a natural defense system (the antioxidant system). Anti-oxidants protect the body from damage caused by internal toxins called free radicals. It is possible that children with Autism tend to have lower levels of glutathione, an important compound in our bodies that helps combat the effects of toxic free radicals. We hope that by studying the antioxidant system in more detail, we will increase our understanding of the reasons why people develop Autism so that we can design better ways to treat individuals with this condition. This study is meant to test the safety tolerability of N-Acetyl Cysteine and its effectiveness in the treatment of behavioral difficulties in children with autism. It will also examine the possible benefit of this agent in improving the core deficits in autism such as social deficits."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '12 Years', 'minimumAge': '3 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\nSubjects will be eligible for this study if they participated in the Double-blind, randomized, placebo controlled study of N-Acetyl Cysteine in Autism study at Stanford University and meet all of the following criteria:\n\n1. Outpatients between 3.0 and 12.11 years of age inclusive\n2. Males and females who are physically healthy\n3. diagnosis of autism based on Diagnostic and Statistical Manual (DSM-IV-TR) criteria, the Autism Diagnostic Interview-Revised, and expert clinical evaluation\n4. Clinical Global Impression Severity rating of 4\n5. Care provider who can reliably bring subject to clinic visits, can provide trustworthy ratings, and interacts with subject on a regular basis\n6. Ability of subject to swallow the compound\n7. Stable concomitant medications for at least 2 weeks\n8. No planned changes in psychosocial interventions during the open-label N-Acetyl Cysteine trial\n\nExclusion Criteria:\n\n1. Diagnostic and Statistical Manual (DSM-IV-TR) diagnosis of schizophrenia, schizoaffective disorder, or psychotic disorder, not otherwise specified\n2. Active medical problems: unstable seizures, significant physical illness (e.g., serious liver or renal pathology)\n3. Pregnancy or sexually active females\n4. Subjects taking antioxidant agents and glutathione prodrugs will be excluded from the study except if they have been off these compounds for at least 4 weeks'}, 'identificationModule': {'nctId': 'NCT00676195', 'briefTitle': 'An Open-Label Study of N-Acetyl Cysteine in Children With Autism', 'organization': {'class': 'OTHER', 'fullName': 'Stanford University'}, 'officialTitle': 'An Open-Label Study of N-Acetyl Cysteine in Autism', 'orgStudyIdInfo': {'id': 'SU-05062008-1139'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'N-Acetyl Cysteine', 'description': 'Dosage of orally administered N-Acetyl Cysteine is as follows:\n\nDays 1-30: 900 mg, once per day Days 31-60: 900 mg, twice per day Days 61-90: 900 mg, three times per day', 'interventionNames': ['Drug: N-Acetyl Cysteine']}], 'interventions': [{'name': 'N-Acetyl Cysteine', 'type': 'DRUG', 'otherNames': ['NAC'], 'description': 'Dosage of orally administered N-Acetyl Cysteine is as follows:\n\nDays 1-30: 900 mg, once per day Days 31-60: 900 mg, twice per day Days 61-90: 900 mg, three times per day', 'armGroupLabels': ['N-Acetyl Cysteine']}]}, 'contactsLocationsModule': {'locations': [{'zip': '94305', 'city': 'Stanford', 'state': 'California', 'country': 'United States', 'facility': 'Stanford University School of Medicine', 'geoPoint': {'lat': 37.42411, 'lon': -122.16608}}], 'overallOfficials': [{'name': 'Antonio Hardan, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Stanford University'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'YES'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Stanford University', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Associate Professor', 'investigatorFullName': 'Antonio Hardan', 'investigatorAffiliation': 'Stanford University'}}}}