Ignite Creation Date:
2025-12-26 @ 10:46 PM
Ignite Modification Date:
2026-03-05 @ 8:48 AM
Study NCT ID:
NCT02366312
Status:
COMPLETED
Last Update Posted:
2021-02-18
First Post:
2015-02-05
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
A Phase 2, Single-center, Single-arm, Open Label Trial of Vismodegib in Patients With Keratocystic Odontogenic Tumors
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009807', 'term': 'Odontogenic Cysts'}], 'ancestors': [{'id': 'D007570', 'term': 'Jaw Cysts'}, {'id': 'D001845', 'term': 'Bone Cysts'}, {'id': 'D003560', 'term': 'Cysts'}, {'id': 'D009369', 'term': 'Neoplasms'}, {'id': 'D007571', 'term': 'Jaw Diseases'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D009057', 'term': 'Stomatognathic Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C538724', 'term': 'HhAntag691'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'brianl.schmidt@nyu.edu', 'phone': '212-998-8900', 'title': 'Dr. Brian Schmidt', 'organization': 'NYU College of Dentistry'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}, 'limitationsAndCaveats': {'description': 'Enrollment for the study closed early by the sponsor due to low enrollment number. Only two subjects were enrolled and received the study treatment.'}}, 'adverseEventsModule': {'timeFrame': 'Up to three years', 'eventGroups': [{'id': 'EG000', 'title': 'NBCCS-associated KCOT', 'description': 'Nevoid Basal Cell Carcinoma Syndrome (NBCCS)-associated Keratocystic Odontogenic Tumor (KCOT) Group', 'otherNumAtRisk': 1, 'deathsNumAtRisk': 1, 'otherNumAffected': 1, 'seriousNumAtRisk': 1, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'Sporadic KCOT', 'description': 'Sporadic Keratocystic Odontogenic Tumor (KCOT) Group', 'otherNumAtRisk': 1, 'deathsNumAtRisk': 1, 'otherNumAffected': 1, 'seriousNumAtRisk': 1, 'deathsNumAffected': 0, 'seriousNumAffected': 1}], 'otherEvents': [{'term': 'Pain', 'notes': 'Right jaw pain / non serious / possibly related', 'stats': [{'groupId': 'EG000', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Earache', 'notes': 'Non-serious / unlikely related', 'stats': [{'groupId': 'EG000', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Sore Throat', 'notes': 'non-serious / unlikely related', 'stats': [{'groupId': 'EG000', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Cramping', 'notes': 'Right hand cramping / non-serious / possibly related to study drug', 'stats': [{'groupId': 'EG000', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'spasm', 'notes': 'Left Quadricep Spasm / non-serious / probably related to study drug', 'stats': [{'groupId': 'EG000', 'numAtRisk': 1, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Allergic reaction', 'notes': 'right face / non-serious / not related', 'stats': [{'groupId': 'EG000', 'numAtRisk': 1, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Spasm', 'notes': 'Both legs / non-serious / probably related', 'stats': [{'groupId': 'EG000', 'numAtRisk': 1, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'cramp', 'notes': 'finger cramping / non-serious / probably related', 'stats': [{'groupId': 'EG000', 'numAtRisk': 1, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hair loss', 'notes': 'non-serious / possibly related', 'stats': [{'groupId': 'EG000', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Pain', 'notes': 'epigastric pain / non-serious / unlikely related', 'stats': [{'groupId': 'EG000', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Facial numbness', 'notes': 'Right side / non-serious / probably related', 'stats': [{'groupId': 'EG000', 'numAtRisk': 1, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Leg Cramp', 'notes': 'worsening of leg cramps / non-serious / probably related', 'stats': [{'groupId': 'EG000', 'numAtRisk': 1, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Chest Discomfort', 'notes': 'non-serious / not related / possible related', 'stats': [{'groupId': 'EG000', 'numAtRisk': 1, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Cold symptoms', 'notes': 'non-serious / not related', 'stats': [{'groupId': 'EG000', 'numAtRisk': 1, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'GI upset', 'notes': 'Non serious / Not related / possibly related', 'stats': [{'groupId': 'EG000', 'numAtRisk': 1, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Upper Respiratory Infection', 'notes': 'non-serious / unlikely related', 'stats': [{'groupId': 'EG000', 'numAtRisk': 1, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Loss of taste', 'notes': 'non-serious, possibly related', 'stats': [{'groupId': 'EG000', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Toes cramping', 'notes': 'non-serious, possible related', 'stats': [{'groupId': 'EG000', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Loose stool', 'notes': 'non-serious, possible related', 'stats': [{'groupId': 'EG000', 'numAtRisk': 1, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'lower extremity pain', 'notes': 'both sides, non-serious, possibly related', 'stats': [{'groupId': 'EG000', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Right facial pain', 'notes': 'non serious, possibly related', 'stats': [{'groupId': 'EG000', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 1, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Left sided muscle ache', 'notes': 'non-serious, not related', 'stats': [{'groupId': 'EG000', 'numAtRisk': 1, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Sacral injury', 'notes': 'non-serious , not related', 'stats': [{'groupId': 'EG000', 'numAtRisk': 1, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Nasopharyn lesion', 'notes': 'non serious, possibly related', 'stats': [{'groupId': 'EG000', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'bilateral lower extremities swelling', 'notes': 'non-serious , unlikely related', 'stats': [{'groupId': 'EG000', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Endocrine disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}], 'seriousEvents': [{'term': 'High grade Newuorendocrine Carcinoma of head/neck', 'notes': 'Serious but not related to study drug', 'stats': [{'groupId': 'EG000', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'KCOT Volume (Greatest Dimension)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'NBCCS-associated KCOT', 'description': 'Nevoid Basal Cell Carcinoma Syndrome (NBCCS)-associated Keratocystic Odontogenic Tumor (KCOT) Group'}, {'id': 'OG001', 'title': 'Sporadic KCOT', 'description': 'Sporadic Keratocystic Odontogenic Tumor (KCOT) Group'}], 'classes': [{'title': 'Baseline', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '3.8', 'groupId': 'OG001'}]}]}, {'title': '6 month (In treatment)', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2.6', 'groupId': 'OG001'}]}]}, {'title': '1 Year (In treatment)', 'categories': [{'measurements': [{'value': '2.3', 'groupId': 'OG000'}, {'value': '2.0', 'groupId': 'OG001'}]}]}, {'title': '2 Year (post-treatment)', 'categories': [{'measurements': [{'value': '2.2', 'groupId': 'OG000'}, {'value': '1.9', 'groupId': 'OG001'}]}]}, {'title': '3 Year (post-treatment)', 'categories': [{'measurements': [{'value': '2.0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline, 6 month (in treatment), 1 year (in treatment), 2 Year (post-treatment), 3 Year (post-treatment)', 'description': 'To evaluate the efficacy of GDC-0449 in reducing KCOT size (shrinkage) in NBCCS-associated KCOT and sporadic KCOT patients following 6 to 12 months of ingestion of 150 mg/day, up to 1 year of treatment and up to 2 years of post- treatment follow-up.', 'unitOfMeasure': 'centimeter', 'reportingStatus': 'POSTED', 'populationDescription': 'Subjects received ERIVEDGE® (vismodegib) 150 mg capsule orally once daily for 12 months.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'NBCCS-associated KCOT', 'description': 'Nevoid Basal Cell Carcinoma Syndrome (NBCCS)-associated Keratocystic Odontogenic Tumor (KCOT) Group'}, {'id': 'FG001', 'title': 'Sporadic KCOT', 'description': 'Sporadic Keratocystic Odontogenic Tumor (KCOT) Group'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '1'}, {'comment': 'The subject did not attend the 2-year post-treatment visit (last visit).', 'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}], 'dropWithdraws': [{'type': 'Lost to Follow-up', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}]}], 'recruitmentDetails': 'Subjects were recruited at the NYU Oral cancer center between mmyy and mmyy. The first participant was enrolled in mmyy and the last subject was enrolled in mmyy.', 'preAssignmentDetails': 'Of 3 enrolled subjects, 2 met inclusion criteria and received treatment.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'NBCCS-associated KCOT', 'description': 'Nevoid Basal Cell Carcinoma Syndrome (NBCCS)-associated Keratocystic Odontogenic Tumor (KCOT) Group'}, {'id': 'BG001', 'title': 'Sporadic KCOT', 'description': 'Sporadic Keratocystic Odontogenic Tumor (KCOT) Group'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '2', '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': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'White - Not Hispanic or Latino', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}, {'title': 'Hispanic or Latino', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}, {'title': 'African American', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}, {'title': 'Asian/Pacific Islander', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}, {'title': 'Other', 'categories': [{'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': '1', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Primary KCOT Tumor size at Baseline', 'classes': [{'categories': [{'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '3.8', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'description': 'Volumetric CT Scan to capture primary KCOT lesion size (greatest dimension) at baseline', 'unitOfMeasure': 'centimeter'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2016-10-14', 'size': 565132, 'label': 'Study Protocol and Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'Prot_SAP_000.pdf', 'typeAbbrev': 'Prot_SAP', 'uploadDate': '2020-10-05T13:02', 'hasProtocol': True}, {'date': '2016-10-20', 'size': 317967, 'label': 'Informed Consent Form', 'hasIcf': True, 'hasSap': False, 'filename': 'ICF_001.pdf', 'typeAbbrev': 'ICF', 'uploadDate': '2020-10-05T13:05', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 2}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2015-10-27', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-02', 'completionDateStruct': {'date': '2019-08', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2021-02-01', 'studyFirstSubmitDate': '2015-02-05', 'resultsFirstSubmitDate': '2020-12-21', 'studyFirstSubmitQcDate': '2015-02-18', 'lastUpdatePostDateStruct': {'date': '2021-02-18', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2021-02-01', 'studyFirstPostDateStruct': {'date': '2015-02-19', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2021-02-18', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2019-08', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'KCOT Volume (Greatest Dimension)', 'timeFrame': 'Baseline, 6 month (in treatment), 1 year (in treatment), 2 Year (post-treatment), 3 Year (post-treatment)', 'description': 'To evaluate the efficacy of GDC-0449 in reducing KCOT size (shrinkage) in NBCCS-associated KCOT and sporadic KCOT patients following 6 to 12 months of ingestion of 150 mg/day, up to 1 year of treatment and up to 2 years of post- treatment follow-up.'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Keratocystic odontogenic tumor', 'KCOT'], 'conditions': ['Keratocystic Odontogenic Tumor']}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to determine how well a daily dose of 150 mg of Erivedge (vismodegib) reduces Keratocystic odontogenic tumor (KCOT) size, and to evaluate the safety of this dose. Erivedge is FDA-approved for use in adults with a specific type of skin cancer. However, the drug is experimental for patients with KCOT.', 'detailedDescription': 'This is a single-center, interventional, single-arm, open-label, two-cohort clinical trial. A total of 20 patients will be enrolled (10 with NBCCS-associated KCOT and 10 with sporadic KCOT) over a period of 2 years. This is a 3 year study where each patient will undergo up to 1 year of treatment and up to 2 years of post-treatment follow-up.\n\nAll patients will be assessed for safety and efficacy of the study drug GDC-0449 (Vismodegib).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Males and females, 18 years of age and above at the time the informed consent form is signed;\n* Able to understand and sign the Informed Consent Form and other necessary paperwork prior to initiation of study procedures;\n* Able to communicate with the investigator/study site personnel, understand and comply with the study requirements, and willing to return for specified visits at the appointed time;\n* Patients who have received prior treatment for their KCOT and with a diagnosis of recurrent (maxillary or mandibular) sporadic KCOT or NBCCS-associated KCOT (single or multiple);\n* Diagnosis of KCOT will be done by past pathology report or by biopsy at the study site, if applicable;\n* Willingness to consent to biopsy of the lesion, if needed;\n* Willingness to delay excision of the target tumor site, unless evidence of disease progression or lack of drug tolerability;\n* Willingness to donate blood for genetic testing;\n* For female patients of childbearing potential, agreement to use two acceptable methods of birth control, including one barrier method during the study and 7 months after discontinuation of study drug;\n* For males with female partners of childbearing potential, agreement to use a male condom (with spermicide) and to advise their female partners to use an acceptable method of birth control during the study and for 2 months after the discontinuation of the study drug;\n* Agreement not to donate blood/blood products during the study and for 7 months after the discontinuation of the study drug;\n* For males not to donate sperm products or semen during treatment and for 2 months after the discontinuation of the study drug;\n* Able and willing to swallow pill;\n* No malabsorption syndrome or other condition that would interfere with enteral absorption;\n* At least 4 weeks since last chemotherapy, investigational therapy, radiotherapy or major surgical procedure and recovered from the first study drug administration;\n* KCOT measures at least 1 cm in one dimension on pretreatment volumetric CT scan;\n* No clinically significant abnormalities with clinical laboratory assessments;\n\nExclusion Criteria:\n\n* Concurrent anti-tumor therapy;\n* Completion of the most recent anti-tumor therapy (including Vismodegib) less than 4 weeks prior to the initiation of treatment (first study drug administration);\n* Uncontrolled medical illness;\n* Pregnancy or lactation; female patients who are planning to become pregnant for the duration of the study and 7 months post-treatment;\n* Inability or unwillingness to swallow capsules;\n* Any medical or psychological illness or condition preventing adequate consent;\n* History of significant atherosclerotic disease, including the following:\n\n * Coronary artery disease (i.e., myocardial infarction within the past year or unstable angina);\n * Documented carotid atheroma;\n* Known HIV infection;\n* Current alcohol abuse;\n* History of resistance to vismodegib (patients who previous received vismodegib for BCC and had no clinical response will be excluded).'}, 'identificationModule': {'nctId': 'NCT02366312', 'acronym': 'KCOT', 'briefTitle': 'A Phase 2, Single-center, Single-arm, Open Label Trial of Vismodegib in Patients With Keratocystic Odontogenic Tumors', 'organization': {'class': 'OTHER', 'fullName': 'NYU College of Dentistry'}, 'officialTitle': 'A Phase 2, Single-center, Single-arm, Open Label Trial of Vismodegib in Patients With Keratocystic Odontogenic Tumors', 'orgStudyIdInfo': {'id': '15-00254'}, 'secondaryIdInfos': [{'id': 'ML28859', 'type': 'OTHER', 'domain': 'Genentech'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'vismodegib', 'description': 'The 150-mg vismodegib drug product is a hard gelatin capsule formulation for oral administration. This study involves one year of treatment with Erivedge (150 mg/day) plus two years of follow-up.', 'interventionNames': ['Drug: vismodegib']}], 'interventions': [{'name': 'vismodegib', 'type': 'DRUG', 'otherNames': ['ERIVEDGE®, GDC-0449'], 'description': 'vismodegib is a synthetic, small molecule inhibitor of the sonic Hh pathway, which is involved in tumorigenesis, thus providing a strong rationale for its use in the treatment of a variety of cancers.', 'armGroupLabels': ['vismodegib']}]}, 'contactsLocationsModule': {'locations': [{'zip': '10010', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'NYU Bluestone Center For Cllinical Research', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}], 'overallOfficials': [{'name': 'Brian L Schmidt, DDS, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'NYU College of Dentistry'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'NYU College of Dentistry', 'class': 'OTHER'}, 'collaborators': [{'name': 'Genentech, Inc.', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Director, Bluestone Center for Clinical Research', 'investigatorFullName': 'Brian L. Schmidt', 'investigatorAffiliation': 'NYU College of Dentistry'}}}}