TEGSEDI Pregnancy Surveillance Program

Study Overview
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Brief Summary
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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.

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

Conditions
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Hereditary Transthyretin-mediated Amyloidosis With Polyneropathy, Pregnancy

Intervention / Treatment
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* Drug: inotersen


Participation Criteria
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Eligibility Criteria
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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
Sexes Eligible for Study
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Female

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Cohort 1<br>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<br>* TEGSEDI injection, for subcutaneous use<br>|
| Cohort 2<br>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?
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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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Hereditary transthyretin amyloidosis with polyneuropathy, Hereditary transthyretin amyloidosis, hATTR, hATTR-PN, Rare disease