Ignite Creation Date:
2025-12-24 @ 6:44 PM
Ignite Modification Date:
2025-12-30 @ 8:34 PM
Study NCT ID:
NCT04543357
Status:
TERMINATED
Last Update Posted:
2025-02-26
First Post:
2020-08-07
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
A Study to Evaluate AAV9 Neutralizing Antibody Seroconversion in Household Contacts.
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006679', 'term': 'HIV Seropositivity'}], 'ancestors': [{'id': 'D015658', 'term': 'HIV Infections'}, {'id': 'D000086982', 'term': 'Blood-Borne Infections'}, {'id': 'D003141', 'term': 'Communicable Diseases'}, {'id': 'D007239', 'term': 'Infections'}, {'id': 'D015229', 'term': 'Sexually Transmitted Diseases, Viral'}, {'id': 'D012749', 'term': 'Sexually Transmitted Diseases'}, {'id': 'D016180', 'term': 'Lentivirus Infections'}, {'id': 'D012192', 'term': 'Retroviridae Infections'}, {'id': 'D012327', 'term': 'RNA Virus Infections'}, {'id': 'D014777', 'term': 'Virus Diseases'}, {'id': 'D000091662', 'term': 'Genital Diseases'}, {'id': 'D000091642', 'term': 'Urogenital Diseases'}, {'id': 'D007153', 'term': 'Immunologic Deficiency Syndromes'}, {'id': 'D007154', 'term': 'Immune System Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D001800', 'term': 'Blood Specimen Collection'}], 'ancestors': [{'id': 'D013048', 'term': 'Specimen Handling'}, {'id': 'D019411', 'term': 'Clinical Laboratory Techniques'}, {'id': 'D019937', 'term': 'Diagnostic Techniques and Procedures'}, {'id': 'D003933', 'term': 'Diagnosis'}, {'id': 'D011677', 'term': 'Punctures'}, {'id': 'D013514', 'term': 'Surgical Procedures, Operative'}, {'id': 'D008919', 'term': 'Investigative Techniques'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'ClinicalTrials.gov_Inquiries@pfizer.com', 'phone': '1-800-718-1021', 'title': 'Pfizer ClinicalTrials.gov Call Center', 'organization': 'Pfizer Inc.'}, 'certainAgreement': {'otherDetails': 'Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'Adverse events were monitored from Screening (Day -48 to Day -1) up to Day 56.', 'description': 'MedDRA v26', 'eventGroups': [{'id': 'EG000', 'title': 'Participants With Potential Fordadistrogene Movaparvovec Exposure', 'description': 'People working or living in the same household of patients who were enrolled in an interventional study of fordadistrogene movaparvovec (including study C3391003 \\[NCT04281485\\] and C3391002 \\[not listed on ClinicalTrials.gov\\]) were enrolled.', 'otherNumAtRisk': 8, 'deathsNumAtRisk': 8, 'otherNumAffected': 0, 'seriousNumAtRisk': 8, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Number of Participants (Previously Seronegative for Neutralizing Antibodies [NAbs] to AAV9) Who Developed NAb to AAV9 at Day 28 After the Interventional Study Patient Was Dosed', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Participants With Potential Fordadistrogene Movaparvovec Exposure', 'description': 'People working or living in the same household of patients who were enrolled in an interventional study of fordadistrogene movaparvovec (including study C3391003 \\[NCT04281485\\] and C3391002 \\[not listed on ClinicalTrials.gov\\]) were enrolled.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Day 28 after the interventional study participant was dosed', 'description': 'Development of NAb to AAV9 was defined by an increase of \\>=6-fold above Baseline titer in participants with a detectable, but negative test (ie, titer \\>=1 and \\<4) for NAb to AAV9 at Baseline. If there was an undetectable titer at Baseline (ie, titer \\<1), then development of NAb to AAV9 was defined by a NAb to AAV9 titer \\>=6.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': '"Number of participants analyzed" signifies number of participants evaluable for the outcome measure: Participants with a NAb titer undetectable (ie, titer \\<1) or detectable, but negative test (ie, titer \\>=1 and \\<4) at Baseline were included in the analysis.'}, {'type': 'SECONDARY', 'title': 'Number of Participants (Previously Seronegative for Anti-Drug Antibody [ADA] to AAV9) Who Developed ADA to AAV9 at Day 28 After the Interventional Study Patient Was Dosed', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Participants With Potential Fordadistrogene Movaparvovec Exposure', 'description': 'People working or living in the same household of patients who were enrolled in an interventional study of fordadistrogene movaparvovec (including study C3391003 \\[NCT04281485\\] and C3391002 \\[not listed on ClinicalTrials.gov\\]) were enrolled.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Day 28 after the interventional study participant was dosed', 'description': 'Development of ADA to AAV9 was defined by a titer ≥300 in participants with a negative test (ie, titer \\<50) for ADA to AAV9 at Baseline.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': '"Number of participants analyzed" signifies number of participants evaluable for the outcome measure: Participants with an ADA titer negative at Baseline were included in the analysis.'}, {'type': 'PRIMARY', 'title': 'Number of Participants (Previously Seronegative for NAb to AAV9) Who Developed NAb to AAV9 at Day 56 After the Interventional Study Patient Was Dosed', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Participants With Potential Fordadistrogene Movaparvovec Exposure', 'description': 'People working or living in the same household of patients who were enrolled in an interventional study of fordadistrogene movaparvovec (including study C3391003 \\[NCT04281485\\] and C3391002 \\[not listed on ClinicalTrials.gov\\]) were enrolled.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Day 56 after the interventional study participant was dosed', 'description': 'Development of NAb to AAV9 was defined by an increase of \\>=6-fold above Baseline titer in participants with a detectable, but negative test (ie, titer \\>=1 and \\<4) for NAb to AAV9 at Baseline. If there was an undetectable titer at Baseline (ie, titer \\<1), then development of NAb to AAV9 was defined by a NAb to AAV9 titer \\>=6.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': '"Number of participants analyzed" signifies number of participants evaluable for the outcome measure: Participants with a NAb titer undetectable (ie, titer \\<1) or detectable, but negative test (ie, titer \\>=1 and \\<4) at Baseline were included in the analysis.'}, {'type': 'SECONDARY', 'title': 'Number of Participants (Previously Seronegative for Anti-Drug Antibody [ADA] to AAV9) Who Developed ADA to AAV9 at Day 56 After the Interventional Study Patient Was Dosed', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Participants With Potential Fordadistrogene Movaparvovec Exposure', 'description': 'People working or living in the same household of patients who were enrolled in an interventional study of fordadistrogene movaparvovec (including study C3391003 \\[NCT04281485\\] and C3391002 \\[not listed on ClinicalTrials.gov\\]) were enrolled.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Day 56 after the interventional study participant was dosed', 'description': 'Development of ADA to AAV9 was defined by a titer ≥300 in participants with a negative test (ie, titer \\<50) for ADA to AAV9 at Baseline.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': '"Number of participants analyzed" signifies number of participants evaluable for the outcome measure: Participants with an ADA titer negative at Baseline were included in the analysis.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Participants With Potential Fordadistrogene Movaparvovec Exposure', 'description': 'People working or living in the same household of patients who were enrolled in an interventional study of fordadistrogene movaparvovec (including study C3391003 \\[NCT04281485\\] and C3391002 \\[not listed on ClinicalTrials.gov\\]) were enrolled.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '8'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '8'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}], 'preAssignmentDetails': 'This was a low-interventional study to assess development of neutralizing Antibodies (NAbs) to adeno associated virus serotype 9 (AAV9) in household contacts of Duchenne Muscular Dystrophy (DMD) Patients Treated with fordadistrogene movaparvovec.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Participants With Potential Fordadistrogene Movaparvovec Exposure', 'description': 'People working or living in the same household of patients who were enrolled in an interventional study of fordadistrogene movaparvovec (including study C3391003 \\[NCT04281485\\] and C3391002 \\[not listed on ClinicalTrials.gov\\]) were enrolled.'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '41.0', 'spread': '9.35', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'Years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '6', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '7', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}], 'populationDescription': 'All participants who signed the informed consent document, and 1) who were household members of interventional study participants (including siblings) who were assigned to fordadistrogene movaparvovec or placebo and received a single dose of study treatment and 2) who had at least one day of contact with the interventional study participant during the first 2 weeks.'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2022-04-05', 'size': 13483202, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2025-02-05T05:07', 'hasProtocol': True}, {'date': '2024-05-17', 'size': 329478, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2025-02-05T04:56', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'bioSpec': {'retention': 'SAMPLES_WITHOUT_DNA', 'description': 'Blood'}, 'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'OTHER'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 8}, 'patientRegistry': False}, 'statusModule': {'whyStopped': 'Study was terminated due to lack of efficacy in the Phase 3 study C3391003, Pfizer has decided to terminate any future dosing of fordadistrogene movaparvovec.', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2022-08-17', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-02', 'completionDateStruct': {'date': '2024-02-27', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-02-05', 'studyFirstSubmitDate': '2020-08-07', 'resultsFirstSubmitDate': '2025-02-05', 'studyFirstSubmitQcDate': '2020-09-02', 'lastUpdatePostDateStruct': {'date': '2025-02-26', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2025-02-05', 'studyFirstPostDateStruct': {'date': '2020-09-10', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2025-02-26', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2024-02-27', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants (Previously Seronegative for Neutralizing Antibodies [NAbs] to AAV9) Who Developed NAb to AAV9 at Day 28 After the Interventional Study Patient Was Dosed', 'timeFrame': 'Day 28 after the interventional study participant was dosed', 'description': 'Development of NAb to AAV9 was defined by an increase of \\>=6-fold above Baseline titer in participants with a detectable, but negative test (ie, titer \\>=1 and \\<4) for NAb to AAV9 at Baseline. If there was an undetectable titer at Baseline (ie, titer \\<1), then development of NAb to AAV9 was defined by a NAb to AAV9 titer \\>=6.'}, {'measure': 'Number of Participants (Previously Seronegative for NAb to AAV9) Who Developed NAb to AAV9 at Day 56 After the Interventional Study Patient Was Dosed', 'timeFrame': 'Day 56 after the interventional study participant was dosed', 'description': 'Development of NAb to AAV9 was defined by an increase of \\>=6-fold above Baseline titer in participants with a detectable, but negative test (ie, titer \\>=1 and \\<4) for NAb to AAV9 at Baseline. If there was an undetectable titer at Baseline (ie, titer \\<1), then development of NAb to AAV9 was defined by a NAb to AAV9 titer \\>=6.'}], 'secondaryOutcomes': [{'measure': 'Number of Participants (Previously Seronegative for Anti-Drug Antibody [ADA] to AAV9) Who Developed ADA to AAV9 at Day 28 After the Interventional Study Patient Was Dosed', 'timeFrame': 'Day 28 after the interventional study participant was dosed', 'description': 'Development of ADA to AAV9 was defined by a titer ≥300 in participants with a negative test (ie, titer \\<50) for ADA to AAV9 at Baseline.'}, {'measure': 'Number of Participants (Previously Seronegative for Anti-Drug Antibody [ADA] to AAV9) Who Developed ADA to AAV9 at Day 56 After the Interventional Study Patient Was Dosed', 'timeFrame': 'Day 56 after the interventional study participant was dosed', 'description': 'Development of ADA to AAV9 was defined by a titer ≥300 in participants with a negative test (ie, titer \\<50) for ADA to AAV9 at Baseline.'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['seroconversion', 'viral shedding', 'DNA vector shedding'], 'conditions': ['Household Contacts']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'https://pmiform.com/clinical-trial-info-request?StudyID=C3391007', 'label': 'To obtain contact information for a study center near you, click here.'}]}, 'descriptionModule': {'briefSummary': 'This study will include male and female participants who live or work in the same household as a patient in one of the fordadistrogene movaparvovec interventional studies. Up to 5 participants from the same household may be enrolled. The objective is to estimate the likelihood of NAb seroconversion to AAV9 in household contacts of a patient in one of the interventional studies who is treated with fordadistrogene movaparvovec gene therapy.', 'detailedDescription': 'This single center study will include approximately 50 to 250 participants and is designed to estimate the likelihood of NAb seroconversion to AAV9 because of exposure to shed viral vector material released by a DMD patient treated with fordadistrogene movaparvovec in an interventional study. Eligible participants will undergo a blood draw provided by a Home Health Care Vendor at three home visits.\n\nThe total duration of participation in this study is about 4 months, including up to 48 days for the screening/baseline period and about 56 days after the DMD patient of the same household is dosed with the investigational gene therapy in the interventional study.\n\nTo maintain the blind in the blinded interventional studies, all C3391007 study participants and investigators as well as the sponsor will remain blinded to the Cohort assignment of the interventional study patient until the interventional study becomes unblinded.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '0 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'This single center study will include approximately 50 to 250 participants who live or work in the same household as a DMD patient in one of the fordadistrogene movaparvovec interventional studies. Up to 5 participants from the same household may be enrolled.', 'healthyVolunteers': True, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Males or females who weigh at least 9 kg.\n* Anticipated to be living or working in the same household as a patient in one of the fordadistrogene movaparvovec interventional studies for at least 4 months.\n* Anticipated to have \\> 10 hours of contact per week and expected to have direct contact with the interventional study patient.\n* The interventional patient is dosed in the interventional study.\n\nExclusion Criteria:\n\n* Prior treatment with gene therapy utilizing AAV vectors of any serotype.'}, 'identificationModule': {'nctId': 'NCT04543357', 'briefTitle': 'A Study to Evaluate AAV9 Neutralizing Antibody Seroconversion in Household Contacts.', 'organization': {'class': 'INDUSTRY', 'fullName': 'Pfizer'}, 'officialTitle': 'A Low-Interventional Study Of AAV9 Neutralizing Antibody Seroconversion in Household Contacts of Participants Within the C3391003 Clinical Trial', 'orgStudyIdInfo': {'id': 'C3391007'}, 'secondaryIdInfos': [{'id': 'ARISEN', 'type': 'OTHER', 'domain': 'Alias Study Number'}]}, 'armsInterventionsModule': {'armGroups': [{'label': 'All participants', 'description': 'Household contacts of a DMD patient in an interventional study of fordadistrogene movaparvovec.', 'interventionNames': ['Other: Blood Draw']}], 'interventions': [{'name': 'Blood Draw', 'type': 'OTHER', 'description': 'Blood Samples for NAb and ADA to AAV9', 'armGroupLabels': ['All participants']}]}, 'contactsLocationsModule': {'locations': [{'zip': '21205', 'city': 'Baltimore', 'state': 'Maryland', 'country': 'United States', 'facility': 'Kennedy Krieger Institute', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}], 'overallOfficials': [{'name': 'Pfizer CT.gov Call Center', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Pfizer'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO', 'description': "Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical\\_trials/trial\\_data\\_and\\_results/data\\_requests."}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Pfizer', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}