Hereditary Transthyretin-mediated Amyloidosis With PolyneropathyNCT04270058
TEGSEDI Pregnancy Surveillance Program
An observational study of Hereditary Transthyretin-mediated Amyloidosis With Polyneropathy and Pregnancy, run by Akcea Therapeutics.
Trial summary
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. Read more about this study
- Who is it for?
- Any age, women only
- Conditions the study is for
- Hereditary Transthyretin-mediated Amyloidosis With Polyneropathy, Pregnancy
- What is being tested?
- inotersen
- Type of treatment
- Injection or infusion
- How many people
- 20 participants
- Where
- No sites listed yet
- Study dates
- Dec 2025to 15 Nov 2030
- How long will I take part?
- 21 Months
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- Women only
- No healthy volunteers
- Child, Adult, Older adult
Study population: 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.
Who may be able to join
- 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.
Who cannot join
- None
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Pregnancy Surveillance Program of Women and Infants Exposed to TEGSEDI During Pregnancy
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.
- Being tested
- inotersen
- Keywords
- Hereditary transthyretin amyloidosis with polyneuropathy, Hereditary transthyretin amyloidosis, hATTR, hATTR-PN, Rare disease
- Medical subject headings
- Amyloidosis, Hereditary, Transthyretin-Related, Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
inotersenDrug
TEGSEDI injection, for subcutaneous use
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | 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. Receives: Drug: inotersen | |
| Cohort 2 | 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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
Measured: 10 years or 12 months after the last live birth whichever is later
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Feb 2020
- Study startDec 2025 (estimated)
- Primary completion15 Nov 2030 (estimated)
- Study completion15 Nov 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary Transthyretin-mediated Amyloidosis With PolyneropathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 20 participants (estimated)
- Target duration
- 21 Months
Sponsor and investigators
- Lead sponsor
- Akcea Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04270058
- Sponsor's protocol ID
- TEG4005
- Other IDs
- EUPAS41839
- Status last verified
- Aug 2025
- First submitted
- 7 Feb 2020
- Last updated
- 19 Aug 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pregnancy Surveillance Program of Women and Infants Exposed to TEGSEDI During Pregnancy
- ClinicalTrials.gov ID
- NCT04270058
- Acronym / sponsor ID
- TEG4005
- Keywords
- Hereditary transthyretin amyloidosis with polyneuropathy, Hereditary transthyretin amyloidosis, hATTR, hATTR-PN, Rare disease
- Study type
- Observational
- Recruitment status
- Not yet recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Akcea TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- Dec 2025 (estimated)
- Primary completion
- 15 Nov 2030 (estimated)
- Study completion
- 15 Nov 2030 (estimated)
- First posted
- 17 Feb 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 21 Months
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | – | Drug: inotersenCohort 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 | – | –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. |
Who may qualify
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- 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.
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (1 listed)
- None
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| inotersenTEGSEDI injection, for subcutaneous use | Drug | Cohort 1 |
Primary outcome
Frequency of Selected Pregnancy and Fetal/Neonatal Outcomes
Time frame: 10 years or 12 months after the last live birth whichever is later
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
Frequency of Selected Pregnancy Complications
Time frame: 10 years or 12 months after the last live birth whichever is later
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Aug 2025.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Questions about this trial
What is this clinical trial studying?
An observational study of Hereditary Transthyretin-mediated Amyloidosis With Polyneropathy and Pregnancy, run by Akcea Therapeutics.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 19 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Ionis Pharmaceuticals ((844) 962-4787, ionisNCT04270058study@clinicaltrialmedia.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Frequency of Selected Pregnancy and Fetal/Neonatal Outcomes (time frame: 10 years or 12 months after the last live birth whichever is later). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Aug 2025; AzTrials last refreshed its copy on 27 Oct 2026.