Pregnancy Surveillance Program of Women and Infants Exposed to TEGSEDI During Pregnancy
Trial Snapshot
- Phase
- Not Applicable
- Status
- Not yet recruiting
- Sponsor
- Akcea Therapeutics
- Enrollment
- 20
- Primary Endpoint
- Frequency of Selected Pregnancy and Fetal/Neonatal Outcomes
Study Overview
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.
Study Design
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Eligibility Criteria
- Sex
- Female
- Accepts Healthy Volunteers
- No
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
- Not provided
Arms & Interventions
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.
Intervention: inotersen (Drug)
Outcomes
Primary Outcomes
Frequency of Selected Pregnancy and Fetal/Neonatal Outcomes
Time Frame: 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
Time Frame: 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
Secondary Outcomes
No secondary outcomes reported