An observational study in Hereditary Transthyretin-mediated Amyloidosis With Polyneropathy and Pregnancy, sponsored by Akcea Therapeutics. Not yet recruiting. Open to female participants. Per ClinicalTrials.gov, last updated 2025-08-19.
Sponsored by Akcea Therapeutics · Observational
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.
There will be 2 cohorts:
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.
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.
The 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.
TEGSEDI exposed patients (Cohort 1) meeting the following criteria will be eligible for study entry:
Unexposed patients (Cohort 2) meeting the following criteria will be eligible for study entry:
Exclusion Criteria:
None
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.
Drug: inotersen
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.
TEGSEDI injection, for subcutaneous use
Frequency of Selected Pregnancy and Fetal/Neonatal Outcomes
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) * pregnancy outcomes include live births, spontaneous abortions, stillbirths, elective abortions, preterm birth * fetal/neonatal outcomes include major and minor congenital malformations, small for gestational age, failure to thrive, and postnatal development
Time frame: 10 years or 12 months after the last live birth whichever is later
Frequency of Selected Pregnancy Complications
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
Time frame: 10 years or 12 months after the last live birth whichever is later
No study locations are listed for this record.
This study is not yet recruiting, as verified in Aug 2025. You cannot join it, but the record below documents what was studied.
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Amyloidosis, Hereditary, Transthyretin-Related→
Akcea Therapeutics