Hereditary Transthyretin-mediated Amyloidosis With Polyneropathy, Pregnancy
Conditions
Keywords
Hereditary transthyretin amyloidosis with polyneuropathy, Hereditary transthyretin amyloidosis, hATTR, hATTR-PN, Rare disease
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.
Interventions
DRUGinotersen
TEGSEDI injection, for subcutaneous use
Sponsors
Akcea Therapeutics
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
FEMALE
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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 |
Contacts
Primary ContactIonis Pharmaceuticals
Outcome results
None listed