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 (part of Ionis Pharmaceuticals, Inc.) · 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.
82 studies on the registry are indexed under Amyloidosis, Hereditary, Transthyretin-Related; 28 are open to participants now.
This study's planned enrollment of 20 is below the median of 100 across 40 observational studies indexed under Amyloidosis, Hereditary, Transthyretin-Related.
Browse Amyloidosis, Hereditary, Transthyretin-Related studies →Akcea Therapeutics is the lead sponsor of 15 studies on the registry; 1 is open to participants now.
Of its 9 completed or terminated interventional studies of FDA-regulated products, 8 (89%) have results posted.
Counted across the registry records on this site, refreshed daily.
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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
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
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Start is estimated for Dec 2025; primary completion for Nov 2030, 59 months later.
Of 25 observational studies under Amyloidosis, Hereditary, Transthyretin-Related, half reached primary completion within 93 months of starting (the median), counting those still running; a quarter within 29 and three quarters within 97. This study's planned 59 months is below that.
How these figures are worked out, and which studies are left out of them.
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 →