Recruiting

TEGSEDI

Sponsor:

Akcea Therapeutics

Code:

NCT04270058

Conditions

Hereditary Transthyretin-mediated Amyloidosis With Polyneropathy

Pregnancy

Eligibility Criteria

Sex: Female

Age: 0+

Healthy Volunteers: Not accepted

Interventions

inotersen

Study Details

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

Study ID

NCT04270058

Start date

Jan 15, 2020

Status verified date

Apr, 2025

Completion date

Nov 15, 2030

Anticipated

Primary completion date

Nov 15, 2030

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: Female

Age: 0+

Healthy Volunteers: Not accepted

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

Enrollment

20 participants

Anticipated

Interventions and Outcome Measures

Arms

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.

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

TEGSEDI injection, for subcutaneous use

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 ]
  • Frequency of Selected Pregnancy Complications [ Time Frame: 10 years or 12 months after the last live birth whichever is later ]

Central Contacts and Locations

Central contacts

Ionis Pharmaceuticals

(844) 962-4787ionisNCT04270058study@clinicaltrialmedia.com

Amy Miller, RPh, Pharm D.

877-465-7510tegsedipregnancy@ubc.com

Locations

TEGSEDI Pregnancy Coordinating Center

Recruiting

Morgantown, West Virginia, United States, 26508

Contacts

Amy Miller, RPh, PharmD

877-465-7510tegsedipregnancy@ubc.com

More Information

Sponsor

Akcea Therapeutics

Last update posted

Apr 24, 2025

Last verified

Apr, 2025

Keywords

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

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Akcea Therapeutics on 2025-04-24.