Ignite Creation Date:
2025-12-24 @ 7:55 PM
Ignite Modification Date:
2026-04-02 @ 1:47 AM
Study NCT ID:
NCT03075904
Status:
TERMINATED
Last Update Posted:
2020-02-05
First Post:
2017-03-06
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
A Safety and Dose-Finding Study of SYNT001 in Subjects With Pemphigus (Vulgaris or Foliaceus)
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D010392', 'term': 'Pemphigus'}], 'ancestors': [{'id': 'D012872', 'term': 'Skin Diseases, Vesiculobullous'}, {'id': 'D012871', 'term': 'Skin Diseases'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D001327', 'term': 'Autoimmune Diseases'}, {'id': 'D007154', 'term': 'Immune System Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000708950', 'term': 'orilanolimab'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'clinicaltrials@alexion.com', 'phone': '855-752-2356', 'title': 'Alexion Pharmaceuticals, Inc.', 'organization': 'Alexion Pharmaceuticals, Inc.'}, 'certainAgreement': {'otherDetails': 'The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding study results for a period that is less than or equal to 60 days from the date that the communication is submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot unilaterally extend the embargo.', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'Baseline through Day 112', 'eventGroups': [{'id': 'EG000', 'title': 'Cohort 1: ALXN1830', 'description': 'Participants received 5 doses of ALXN1830 10 mg/kg administered weekly.', 'otherNumAtRisk': 8, 'deathsNumAtRisk': 8, 'otherNumAffected': 7, 'seriousNumAtRisk': 8, 'deathsNumAffected': 0, 'seriousNumAffected': 1}], 'otherEvents': [{'term': 'Dry eye', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}, {'term': 'Malaise', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}, {'term': 'Hepatic cyst', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}, {'term': 'Herpes simplex', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}, {'term': 'Staphylococcal infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}, {'term': 'Tinea barbae', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}, {'term': 'Infusion related reaction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}, {'term': 'Urine analysis abnormal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 6}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}, {'term': 'Sinus headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}, {'term': 'Acute kidney injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}, {'term': 'Hyperhidrosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}, {'term': 'Pallor', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}], 'seriousEvents': [{'term': 'Disease progression', 'notes': 'Resolved and was considered to be not related to the study drug by the Investigator.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}, {'term': 'Acute kidney injury', 'notes': 'Resolved and was considered to be not related to the study drug by the Investigator.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Count Of Participants Reporting Treatment-emergent Adverse Events (TEAEs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 1: ALXN1830', 'description': 'Participants received 5 doses of ALXN1830 10 mg/kg administered weekly.'}], 'classes': [{'title': 'TEAEs', 'categories': [{'measurements': [{'value': '7', 'groupId': 'OG000'}]}]}, {'title': 'Serious TEAEs', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Discontinuations due to TEAEs', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Deaths', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Day 1 (after first dose) through Day 112', 'description': 'A TEAE was defined as any adverse event (AE) that starts on or after the first dose of study drug or occurs prior to the first dose and worsens in severity on or after the first dose of study drug, during the Treatment Period and Follow-up Period. A TEAE was considered "serious" (Grade 3) if, in the view of either the investigator or sponsor, it resulted in any of the following outcomes: death, life-threatening adverse drug event, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect, or an event that may have jeopardized the participant and may have required medical or surgical intervention to prevent one of the previously listed outcomes. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received at least 1 dose of study drug.'}, {'type': 'SECONDARY', 'title': 'Maximum Percent Reduction Of Mean Total Immunoglobulin G (IgG) Levels From Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 1: ALXN1830', 'description': 'Participants received 5 doses of ALXN1830 10 mg/kg administered weekly.'}], 'classes': [{'categories': [{'measurements': [{'value': '57.3', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline through Day 112', 'description': 'Pharmacodynamic samples were collected for analysis throughout the study. The maximum percent reduction of mean serum total IgG levels from Baseline observed during the study is presented.', 'unitOfMeasure': 'percentage of reduction', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received at least 1 dose of study drug and had postdose pharmacodynamic data available.'}, {'type': 'SECONDARY', 'title': 'Maximum Percent Reduction In Mean Pemphigus Disease Area Index (PDAI) Total Activity Score From Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 1: ALXN1830', 'description': 'Participants received 5 doses of ALXN1830 10 mg/kg administered weekly.'}], 'classes': [{'categories': [{'measurements': [{'value': '45.7', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline through Day 112', 'description': 'Pemphigus severity and disease activity was measured using the PDAI in regions where a validated questionnaire was available. The PDAI was administered during Treatment Period and Follow-up Period. PDAI total activity was comprised of scores for the skin, mucous membrane, and scalp subscales. The investigator determined a PDAI score as 0 to 250 points for total activity score (0 to 120 for skin, 0 to 10 for scalp, and 0 to 120 for mucosa). A higher score indicated higher impact on skin disease. The maximum percent reduction in PDAI total activity score from Baseline observed during the study is presented.', 'unitOfMeasure': 'percentage of reduction', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received at least 1 dose of study drug and had postdose pharmacodynamic data available.'}, {'type': 'SECONDARY', 'title': 'Maximum Percent Reduction Of Mean Circulating Immune Complexes (CIC) Levels From Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 1: ALXN1830', 'description': 'Participants received 5 doses of ALXN1830 10 mg/kg administered weekly.'}], 'classes': [{'categories': [{'measurements': [{'value': '51.4', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline through Day 112', 'description': 'Pharmacodynamic samples were collected for analysis throughout the study. The maximum percent reduction of mean CIC levels from Baseline observed during the study is presented.', 'unitOfMeasure': 'percentage of reduction', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received at least 1 dose of study drug and had postdose pharmacodynamic data available.'}, {'type': 'SECONDARY', 'title': 'Maximum Percent Reduction Of Mean Anti-Desmoglein (Dsg) 1 And 3 Antibodies From Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 1: ALXN1830', 'description': 'Participants received 5 doses of ALXN1830 10 mg/kg administered weekly.'}], 'classes': [{'title': 'anti-Dsg 1', 'categories': [{'measurements': [{'value': '8.9', 'groupId': 'OG000'}]}]}, {'title': 'anti-Dsg 3', 'categories': [{'measurements': [{'value': '20.4', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline through Day 112', 'description': 'Pharmacodynamic samples were collected for analysis throughout the study. The maximum percent reduction of mean anti-Dsg 1 and 3 antibodies from Baseline observed during the study is presented.', 'unitOfMeasure': 'percentage of reduction', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received at least 1 dose of study drug and had postdose pharmacodynamic data available.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Cohort 1: ALXN1830', 'description': 'Participants received 5 doses of ALXN1830 10 mg/kg administered weekly.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '8'}]}, {'type': 'Received at Least 1 Dose of Study Drug', 'achievements': [{'groupId': 'FG000', 'numSubjects': '8'}]}, {'type': 'COMPLETED', 'comment': 'All 8 participants received all 5 weekly doses of ALXN1830 10 mg/kg in the Treatment Period.', 'achievements': [{'groupId': 'FG000', 'numSubjects': '4'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '4'}]}], 'dropWithdraws': [{'type': 'Physician Decision', 'reasons': [{'groupId': 'FG000', 'numSubjects': '3'}]}, {'type': 'Need for Medication not Permitted', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}]}], 'preAssignmentDetails': 'This study was terminated after the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy were characterized at a single dose level (10 milligram/kilogram \\[mg/kg\\]) in Cohort 1, before any participants were enrolled in Cohort 2. This results disclosure is for Cohort 1 only.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Cohort 1: ALXN1830', 'description': 'Participants received 5 doses of ALXN1830 10 mg/kg administered weekly.'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '51.4', 'spread': '16.43', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '5', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '3', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '8', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '3', 'groupId': 'BG000'}]}, {'title': 'White', 'measurements': [{'value': '5', 'groupId': 'BG000'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Type of Pemphigus', 'classes': [{'title': 'Vulgaris', 'categories': [{'measurements': [{'value': '7', 'groupId': 'BG000'}]}]}, {'title': 'Foliaceus', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age at Diagnosis of Pemphigus', 'classes': [{'categories': [{'measurements': [{'value': '41.3', 'spread': '18.90', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}], 'populationDescription': 'All participants who were enrolled in the study.'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2019-03-25', 'size': 1141576, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_000.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2020-01-15T12:33', 'hasProtocol': False}, {'date': '2018-09-18', 'size': 13380427, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_001.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2020-01-16T14:11', 'hasProtocol': True}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 8}}, 'statusModule': {'whyStopped': 'Study objectives achieved', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2017-07-18', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2020-01', 'completionDateStruct': {'date': '2019-01-16', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-01-16', 'studyFirstSubmitDate': '2017-03-06', 'resultsFirstSubmitDate': '2020-01-16', 'studyFirstSubmitQcDate': '2017-03-08', 'lastUpdatePostDateStruct': {'date': '2020-02-05', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2020-01-16', 'studyFirstPostDateStruct': {'date': '2017-03-09', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2020-02-05', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2019-01-16', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Count Of Participants Reporting Treatment-emergent Adverse Events (TEAEs)', 'timeFrame': 'Day 1 (after first dose) through Day 112', 'description': 'A TEAE was defined as any adverse event (AE) that starts on or after the first dose of study drug or occurs prior to the first dose and worsens in severity on or after the first dose of study drug, during the Treatment Period and Follow-up Period. A TEAE was considered "serious" (Grade 3) if, in the view of either the investigator or sponsor, it resulted in any of the following outcomes: death, life-threatening adverse drug event, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect, or an event that may have jeopardized the participant and may have required medical or surgical intervention to prevent one of the previously listed outcomes. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module.'}], 'secondaryOutcomes': [{'measure': 'Maximum Percent Reduction Of Mean Total Immunoglobulin G (IgG) Levels From Baseline', 'timeFrame': 'Baseline through Day 112', 'description': 'Pharmacodynamic samples were collected for analysis throughout the study. The maximum percent reduction of mean serum total IgG levels from Baseline observed during the study is presented.'}, {'measure': 'Maximum Percent Reduction In Mean Pemphigus Disease Area Index (PDAI) Total Activity Score From Baseline', 'timeFrame': 'Baseline through Day 112', 'description': 'Pemphigus severity and disease activity was measured using the PDAI in regions where a validated questionnaire was available. The PDAI was administered during Treatment Period and Follow-up Period. PDAI total activity was comprised of scores for the skin, mucous membrane, and scalp subscales. The investigator determined a PDAI score as 0 to 250 points for total activity score (0 to 120 for skin, 0 to 10 for scalp, and 0 to 120 for mucosa). A higher score indicated higher impact on skin disease. The maximum percent reduction in PDAI total activity score from Baseline observed during the study is presented.'}, {'measure': 'Maximum Percent Reduction Of Mean Circulating Immune Complexes (CIC) Levels From Baseline', 'timeFrame': 'Baseline through Day 112', 'description': 'Pharmacodynamic samples were collected for analysis throughout the study. The maximum percent reduction of mean CIC levels from Baseline observed during the study is presented.'}, {'measure': 'Maximum Percent Reduction Of Mean Anti-Desmoglein (Dsg) 1 And 3 Antibodies From Baseline', 'timeFrame': 'Baseline through Day 112', 'description': 'Pharmacodynamic samples were collected for analysis throughout the study. The maximum percent reduction of mean anti-Dsg 1 and 3 antibodies from Baseline observed during the study is presented.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Pemphigus', 'Pemphigus Vulgaris', 'Pemphigus Foliaceus']}, 'referencesModule': {'references': [{'pmid': '34126109', 'type': 'DERIVED', 'citation': 'Werth VP, Culton DA, Concha JSS, Graydon JS, Blumberg LJ, Okawa J, Pyzik M, Blumberg RS, Hall RP 3rd. Safety, Tolerability, and Activity of ALXN1830 Targeting the Neonatal Fc Receptor in Chronic Pemphigus. J Invest Dermatol. 2021 Dec;141(12):2858-2865.e4. doi: 10.1016/j.jid.2021.04.031. Epub 2021 Jun 12.'}]}, 'descriptionModule': {'briefSummary': 'This was a multicenter, open-label safety study to determine the dose regimen of SYNT001 (ALXN1830) administered intravenously in participants with pemphigus (vulgaris or foliaceus).', 'detailedDescription': 'This study planned to evaluate 2 cohorts: up to 8 participants to receive 5 weekly intravenous (IV) doses of ALXN1830 at 10 milligram/kilogram (mg/kg) (Cohort 1) and up to 12 participants to receive 3 x 30 mg/kg weekly doses of ALXN1830 IV (loading) followed by 5 x 10 mg/kg doses of ALXN1830 IV every other week or 10 weekly doses of ALXN1830 IV (maintenance) (Cohort 2).\n\nThis study was terminated after the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy were characterized in participants with pemphigus at a single dose level (10 mg/kg) in Cohort 1, before any participants were enrolled in Cohort 2.\n\nThe study consisted of 3 periods: Screening, Treatment, and Follow-Up.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\nParticipants must have meet the following criteria to be included:\n\n* Were willing and able to read, understand and sign an informed consent form\n* Documented diagnosis of pemphigus vulgaris or foliaceus\n* Were required to use medically acceptable contraception\n\nExclusion Criteria:\n\nParticipants meeting any of the following criteria were excluded:\n\n* Were unable or unwilling to comply with the protocol\n* Active non-hematologic malignancy or history of non-hematologic malignancy in the 3 years prior to screening (exclusive of non-melanoma skin cancer and cervical cancer in situ)\n* Positive for human immunodeficiency virus (HIV) or hepatitis C antibody\n* Positive for hepatitis B surface antigen\n* IV immunoglobulin treatment within 30 days of screening\n* Any exposure to an investigational drug or device within the 30 days prior to screening\n* Plasmapheresis or immunoadsorption within 30 days of screening\n* Participant had any current medical condition that, in the opinion of the Investigator, may have compromised their safety or compliance, preclude successful conduct of the study, or interfere with interpretation of the results'}, 'identificationModule': {'nctId': 'NCT03075904', 'briefTitle': 'A Safety and Dose-Finding Study of SYNT001 in Subjects With Pemphigus (Vulgaris or Foliaceus)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Alexion Pharmaceuticals, Inc.'}, 'officialTitle': 'A Phase 1B/2, Multicenter, Open-Label, Safety, and Dose-Finding Study of SYNT001 in Subjects With Pemphigus (Vulgaris or Foliaceus)', 'orgStudyIdInfo': {'id': 'SYNT001-103'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Cohort 1: ALXN1830', 'description': 'Participants received 5 doses of ALXN1830 10 mg/kg administered weekly.', 'interventionNames': ['Drug: ALXN1830']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 2: ALXN1830', 'description': 'Participants were to receive 3 doses of ALXN1830 30 mg/kg administered weekly (loading) followed by 5 doses of ALXN1830 10 mg/kg administered every other week or 10 weekly doses of ALXN1830 IV (maintenance).', 'interventionNames': ['Drug: ALXN1830']}], 'interventions': [{'name': 'ALXN1830', 'type': 'DRUG', 'otherNames': ['SYNT001'], 'description': 'Administered via IV infusion.', 'armGroupLabels': ['Cohort 1: ALXN1830', 'Cohort 2: ALXN1830']}]}, 'contactsLocationsModule': {'locations': [{'zip': '27516', 'city': 'Chapel Hill', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Alexion Study Site', 'geoPoint': {'lat': 35.9132, 'lon': -79.05584}}, {'zip': '27710', 'city': 'Durham', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Alexion Study Site', 'geoPoint': {'lat': 35.99403, 'lon': -78.89862}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'Alexion Study Site', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Alexion Pharmaceuticals, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}