TEGSEDI Pregnancy Surveillance Program

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.

Study Overview

• Status: Not yet recruiting
• Conditions: Pregnancy; Hereditary Transthyretin-mediated Amyloidosis With Polyneropathy
• Intervention / Treatment: Drug: inotersen

• Study Type: Observational
• Enrollment (Estimated): 20

Contacts and Locations

• Study Contact: Name: Ionis Pharmaceuticals; Phone Number: (844) 962-4787; Email: ionisNCT04270058study@clinicaltrialmedia.com

Study Locations

• United States
  • West Virginia
    • Morgantown, West Virginia, United States, 26508
      • Recruiting
      • TEGSEDI Pregnancy Coordinating Center
      • Contact: Amy Miller, RPh, PharmD; Phone Number: 877-465-7510; Email: tegsedipregnancy@ubc.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• 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.

Description

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 Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
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; TEGSEDI injection, for subcutaneous use
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 the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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

Collaborators and Investigators

• Sponsor: Akcea Therapeutics

Study record dates

Study Major Dates

• Study Start (Estimated): December 1, 2025
• Primary Completion (Estimated): November 15, 2030
• Study Completion (Estimated): November 15, 2030

Study Registration Dates

• First Submitted: February 7, 2020
• First Submitted That Met QC Criteria: February 12, 2020
• First Posted (Actual): February 17, 2020

Study Record Updates

• Last Update Posted (Actual): August 19, 2025
• Last Update Submitted That Met QC Criteria: August 14, 2025
• Last Verified: August 1, 2025

More Information

Terms related to this study

• Keywords: Rare disease; hATTR-PN; hATTR; Hereditary transthyretin amyloidosis; Hereditary transthyretin amyloidosis with polyneuropathy
• Additional Relevant MeSH Terms: Metabolic Diseases; Proteostasis Deficiencies; Amyloidosis
• Other Study ID Numbers: TEG4005; EUPAS41839 (Other Identifier: EU PAS)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No