ClinicalTrials.gov record NCT04270058
TEGSEDI Pregnancy Surveillance Program
Pregnancy Surveillance Program of Women and Infants Exposed to TEGSEDI During Pregnancy
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This is a prospective safety surveillance study collecting pregnancy and infant outcomes for women with hATTR-PN who were exposed or not exposed to TEGSEDI (inotersen). Participants are enrolled into two cohorts (exposed and unexposed) and pediatric outcomes are followed through about 1 year of age. The study will estimate how often selected pregnancy, fetal/neonatal outcomes and pregnancy complications occur in each cohort.
What it is studying. The study aims to estimate the frequency of selected pregnancy outcomes, fetal and neonatal outcomes (including major congenital malformations and growth issues), and selected pregnancy complications in women with hATTR-PN who were exposed to at least one dose of TEGSEDI within 25 weeks before conception or during pregnancy, compared with pregnant women with hATTR-PN who were not exposed within that window.
What the researchers hope to learn. To provide safety surveillance data by estimating how often selected pregnancy outcomes, fetal/neonatal outcomes, and pregnancy complications occur in women with hATTR-PN exposed to TEGSEDI compared with unexposed women with hATTR-PN. The information is intended to inform understanding of pregnancy and infant outcomes after maternal exposure to TEGSEDI.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- inotersen
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- TEGSEDI exposed patients (Cohort 1) meeting the following criteria will be eligible for study entry:
- Pregnancies exposed to at least 1 dose of TEGSEDI within 25 weeks prior to conception or during pregnancy.
- Able and willing to provide informed consent.
- Unexposed patients (Cohort 2) meeting the following criteria will be eligible for study entry:
- Have a diagnosis of hATTR-PN during pregnancy.
- Have not been exposed to TEGSEDI within 25 weeks prior to conception or during pregnancy.
- Able and willing to provide informed consent.
Exclusion criteria
- None
Study description and what participation involves
The registry record has no detailed description beyond the summary above.
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants will have information collected about their pregnancy and about their baby up to 1 year of age. The study separates people into two groups (those exposed to at least one dose of TEGSEDI within the exposure window and those not exposed) and will record pregnancy outcomes (for example live birth, spontaneous abortion, stillbirth, elective abortion, preterm birth) and fetal/neonatal outcomes (for example major and minor congenital malformations, small for gestational age, failure to thrive, and postnatal development). The registry lists a follow-up timeframe of “10 years or 12 months after the last live birth whichever is later” for the primary outcome data. The record does not provide details on specific study visits, tests, samples, questionnaires, or other procedures.
How the study is structured. A prospective observational cohort study with two groups: (1) pregnant people exposed to at least one dose of TEGSEDI (inotersen) within 25 weeks before conception or during pregnancy, and (2) pregnant people with hATTR-PN not exposed within that period. Data collected will include pregnancy outcomes and infant follow-up through about 1 year of age; the registry notes outcome tracking for up to 10 years or 12 months after the last live birth, whichever is later. The record lists a target duration of 21 months for individual participation.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
- The registry says recruitment has not started. When is it expected to open, and can I register interest now?
- No sites are listed on the registry yet. Where will the study take place?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on ATTRv amyloidosis
This trial is also listed on this site under wild-type ATTR amyloidosis.
Important dates
- Study start
- December 2025 (estimated)
- Primary completion
- Nov 15, 2030 (estimated)
- Study completion
- Nov 15, 2030 (estimated)
- Target enrollment
- 20 participants (estimated)
- Last updated on registry
- Aug 19, 2025
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Akcea Therapeutics
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Ionis Pharmaceuticals 📞 (844) 962-4787 ✉ ionisNCT04270058study@clinicaltrialmedia.com
Participating sites
Study locations have not been posted on ClinicalTrials.gov yet. This study is not yet recruiting, so sites may be added when recruitment begins. The central study contact above may be able to tell you about planned sites.
Terms used on this page · explanations by MedicalUpdates.ai
- Cohort
- A group of people who share a characteristic, such as a diagnosis, and are studied together. 'Prospective' means they are followed forward from enrolment; 'retrospective' means existing records are reviewed.
- informed consent
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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