TEGSEDI Pregnancy Surveillance Program

Recruiting

• Conditions: Hereditary Transthyretin-mediated Amyloidosis With Polyneropathy; Pregnancy
• Interventions: Drug: inotersen

• Registration Number: NCT04270058
• Lead Sponsor: Akcea Therapeutics

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2020-01-15
• Study First Submitted: 2020-02-07
• Study First Submitted QC: 2020-02-12
• Study First Posted: 2020-02-17
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-23
• Last Update Posted: 2025-04-24
• Primary Completion: 2030-11-15
• Study Completion: 2030-11-15

Recruitment & Eligibility

• Status: RECRUITING
• Sex: Female
• Target Recruitment: 20

Inclusion Criteria

TEGSEDI exposed patients (Cohort 1) meeting the following criteria will be eligible for study entry:

1. Pregnancies exposed to at least 1 dose of TEGSEDI within 25 weeks prior to conception or during pregnancy.
2. Able and willing to provide informed consent.

Unexposed patients (Cohort 2) meeting the following criteria will be eligible for study entry:

1. Have a diagnosis of hATTR-PN during pregnancy.
2. Have not been exposed to TEGSEDI within 25 weeks prior to conception or during pregnancy.
3. Able and willing to provide informed consent.

Exclusion Criteria

None

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Cohort 1	inotersen	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.

Primary Outcome Measures

Name	Time	Method
Frequency of Selected Pregnancy and Fetal/Neonatal Outcomes	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	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

Trial Locations

Locations (1)

TEGSEDI Pregnancy Coordinating Center; 🇺🇸 Morgantown, West Virginia, United States