Ignite Creation Date:
2025-12-25 @ 12:14 AM
Ignite Modification Date:
2026-02-27 @ 1:27 AM
Study NCT ID:
NCT04270058
Status:
NOT_YET_RECRUITING
Last Update Posted:
2025-08-19
First Post:
2020-02-07
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
TEGSEDI Pregnancy Surveillance Program
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['United States']}, 'conditionBrowseModule': {'meshes': [{'id': 'C567782', 'term': 'Amyloidosis, Hereditary, Transthyretin-Related'}, {'id': 'D035583', 'term': 'Rare Diseases'}], 'ancestors': [{'id': 'D020969', 'term': 'Disease Attributes'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000629536', 'term': 'Inotersen'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 20}, 'targetDuration': '21 Months', 'patientRegistry': True}, 'statusModule': {'overallStatus': 'NOT_YET_RECRUITING', 'startDateStruct': {'date': '2025-12', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-08', 'completionDateStruct': {'date': '2030-11-15', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-08-14', 'studyFirstSubmitDate': '2020-02-07', 'studyFirstSubmitQcDate': '2020-02-12', 'lastUpdatePostDateStruct': {'date': '2025-08-19', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2020-02-17', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2030-11-15', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Frequency of Selected Pregnancy and Fetal/Neonatal Outcomes', 'timeFrame': '10 years or 12 months after the last live birth whichever is later', 'description': 'Estimate the frequency of selected pregnancy and fetal/neonatal outcomes through 1 year of age in women who were exposed to at least 1 dose of TEGSEDI (Cohort 1) within 25 weeks prior to conception or during pregnancy, with the exposure window of interest for major congenital malformations being the first trimester, and in the unexposed cohort of pregnant women (Cohort 2) who have a diagnosis of hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN)\n\n* pregnancy outcomes include live births, spontaneous abortions, stillbirths, elective abortions, preterm birth\n* fetal/neonatal outcomes include major and minor congenital malformations, small for gestational age, failure to thrive, and postnatal development'}, {'measure': 'Frequency of Selected Pregnancy Complications', 'timeFrame': '10 years or 12 months after the last live birth whichever is later', 'description': 'Estimate the frequency of selected pregnancy complications in women who were exposed to TEGSEDI (Cohort 1) within 25 weeks prior to conception or during pregnancy and in the unexposed cohort of pregnant women (Cohort 2) who have a diagnosis of hATTR-PN'}]}, 'oversightModule': {'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Hereditary transthyretin amyloidosis with polyneuropathy', 'Hereditary transthyretin amyloidosis', 'hATTR', 'hATTR-PN', 'Rare disease'], 'conditions': ['Hereditary Transthyretin-mediated Amyloidosis With Polyneropathy', 'Pregnancy']}, 'descriptionModule': {'briefSummary': 'This is a worldwide safety surveillance study of pregnancy outcomes in women with hATTR-PN who may have been exposed or were not exposed to TEGSEDI prior to or during the pregnancy and of pediatric outcomes up to 1 year of age.'}, 'eligibilityModule': {'sex': 'FEMALE', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'genderBased': True, 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'There will be 2 cohorts:\n\nCohort 1 will be pregnant patients who have been exposed to at least 1 dose of TEGSEDI within 25 weeks prior to conception or during pregnancy.\n\nCohort 2 will be pregnant patients who have hATTR-PN, who were not exposed to TEGSEDI or have not received TEGSEDI within the previous 25 weeks prior to conception.\n\nThe exposed cohort will include pregnant patients from any country where TEGSEDI is marketed and/or clinical trials, post-market studies and registries are being conducted or expanded access programs are available.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\nTEGSEDI exposed patients (Cohort 1) meeting the following criteria will be eligible for study entry:\n\n1. Pregnancies exposed to at least 1 dose of TEGSEDI within 25 weeks prior to conception or during pregnancy.\n2. Able and willing to provide informed consent.\n\nUnexposed patients (Cohort 2) meeting the following criteria will be eligible for study entry:\n\n1. Have a diagnosis of hATTR-PN during pregnancy.\n2. Have not been exposed to TEGSEDI within 25 weeks prior to conception or during pregnancy.\n3. Able and willing to provide informed consent.\n\nExclusion Criteria:\n\nNone'}, 'identificationModule': {'nctId': 'NCT04270058', 'briefTitle': 'TEGSEDI Pregnancy Surveillance Program', 'organization': {'class': 'INDUSTRY', 'fullName': 'Akcea Therapeutics'}, 'officialTitle': 'Pregnancy Surveillance Program of Women and Infants Exposed to TEGSEDI During Pregnancy', 'orgStudyIdInfo': {'id': 'TEG4005'}, 'secondaryIdInfos': [{'id': 'EUPAS41839', 'type': 'OTHER', 'domain': 'EU PAS'}]}, 'armsInterventionsModule': {'armGroups': [{'label': 'Cohort 1', 'description': 'Cohort 1 will be pregnant patients who have been exposed to at least 1 dose of TEGSEDI within 25 weeks prior to conception or during pregnancy.', 'interventionNames': ['Drug: inotersen']}, {'label': 'Cohort 2', 'description': 'Cohort 2 will be pregnant patients who have hATTR-PN, who were not exposed to TEGSEDI or have not received TEGSEDI within the previous 25 weeks prior to conception.'}], 'interventions': [{'name': 'inotersen', 'type': 'DRUG', 'description': 'TEGSEDI injection, for subcutaneous use', 'armGroupLabels': ['Cohort 1']}]}, 'contactsLocationsModule': {'centralContacts': [{'name': 'Ionis Pharmaceuticals', 'role': 'CONTACT', 'email': 'ionisNCT04270058study@clinicaltrialmedia.com', 'phone': '(844) 962-4787'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Akcea Therapeutics', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}