TEGSEDI Pregnancy Surveillance Program

Sponsor

Akcea Therapeutics (Industry)

Overall Status

Recruiting

CT.gov ID

NCT04270058

Collaborator

(none)

Enrollment

Location

106.5

Anticipated Duration (Months)

0.2

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Hereditary Transthyretin-mediated Amyloidosis With Polyneropathy Pregnancy	Drug: inotersen

Study Design

Study Type:

Observational [Patient Registry]

Anticipated Enrollment :

20 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

Pregnancy Surveillance Program of Women and Infants Exposed to TEGSEDI During Pregnancy

Anticipated Study Start Date :

Dec 31, 2021

Anticipated Primary Completion Date :

Nov 15, 2030

Anticipated Study Completion Date :

Nov 15, 2030

Arms and Interventions

Arm	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.

Arm

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.

Outcome Measures

Primary Outcome Measures

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

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A and Older

Sexes Eligible for Study:

Female

Accepts Healthy Volunteers:

Inclusion Criteria:

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.

Exclusion Criteria:

None

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	TEGSEDI Pregnancy Coordinating Center	Morgantown	West Virginia	United States	26505

Sponsors and Collaborators

Akcea Therapeutics

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Akcea Therapeutics

ClinicalTrials.gov Identifier:

NCT04270058

Other Study ID Numbers:

TEG4005

First Posted:

Feb 17, 2020

Last Update Posted:

Dec 17, 2021

Last Verified:

Jul 1, 2021

Studies a U.S. FDA-regulated Drug Product:

Yes

Studies a U.S. FDA-regulated Device Product:

Keywords provided by Akcea Therapeutics

Hereditary transthyretin amyloidosis with polyneuropathy

Hereditary transthyretin amyloidosis

hATTR

hATTR-PN

Rare disease

Additional relevant MeSH terms:

Amyloidosis

Study Results

No Results Posted as of Dec 17, 2021