TEGSEDI Pregnancy Surveillance Program

NCT04270058 | Not Yet Recruiting

• 2 other identifiers: TEG4005EUPAS41839
• Study Type: observational
• Target: 20
• Locations: 0 countries
• Sites: N/A

Brief 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.

Conditions

Hereditary Transthyretin-mediated Amyloidosis With Polyneropathy; Pregnancy

Keywords

Hereditary transthyretin amyloidosis with polyneuropathy; Hereditary transthyretin amyloidosis; hATTR; hATTR-PN; Rare disease

Outcome Measures

Primary Outcomes (2)

• 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

  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

  10 years or 12 months after the last live birth whichever is later

Study Arms (2)

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.

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.

Interventions

inotersen DRUG

TEGSEDI injection, for subcutaneous use

Cohort 1

Eligibility Criteria

• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

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.

You may qualify if:

• 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.

You may not qualify if:

• None

Sponsors & Collaborators

• Akcea Therapeutics: lead

MeSH Terms

Conditions

Amyloidosis, Hereditary, Transthyretin-Related; Rare Diseases

Interventions

Inotersen

Condition Hierarchy (Ancestors)

Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Central Study Contacts

Ionis Pharmaceuticals

CONTACT

(844) 962-4787; ionisNCT04270058study@clinicaltrialmedia.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 21 Months
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 7, 2020
• First Posted: February 17, 2020
• Study Start: December 1, 2025
• Primary Completion (Estimated): November 15, 2030
• Study Completion (Estimated): November 15, 2030
• Last Updated: August 19, 2025

Record last verified: 2025-08