Pregnancy Surveillance Program of Women and Infants Exposed to TEGSEDI During Pregnancy (TEG4005)

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Other
• If ‘other’, further details on the scope of the study:
  Pregnancy Surveillance Program

Non-interventional study

• Non-interventional study design:
  Cohort

  Other
• Non-interventional study design, other:
  Pregnancy surveillance program

Study drug and medical condition

• Name of medicine:
  TEGSEDI
• Additional medical condition(s):
  Pregnancy Hereditary Transthyretin Amyloidosis with Polyneuropathy (hATTR-PN)

Population studied

• Age groups:
  Preterm newborn infants (0 – 27 days)

  Term newborn infants (0 – 27 days)

  Infants and toddlers (28 days – 23 months)

  Adults (18 to < 46 years)

  Adults (46 to < 65 years)
• Special population of interest:
  Pregnant women
• Estimated number of subjects:
  20

Study design details

• Study design:
  This is a worldwide observational pregnancy surveillance program (PSP) of pregnancy exposures to TEGSEDI. The study is designed to estimate the frequency of selected pregnancy, fetal/neonatal and pregnancy complications outcomes from the TEGSEDI exposed and unexposed patients.;
• Main study objective:
  The objectives of this PSP are to:; • 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 hATTRPN; - 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; • 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
• Setting:
  The study will actively pursue and attempt to capture all pregnancies that meet the eligibility criteria that occur worldwide.
• Outcomes:
  Primary Outcome Measures:; 1.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; [Time Frame: 10 years or 12 months after the last live birth whichever is later]; 2.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; [Time Frame: 10 years or 12 months after the last live birth whichever is later]
• Data analysis plan:
  The primary outcomes of interest are major congenital malformations (MCMs) as reviewed and classified using both the conventions of the Metropolitan Atlanta Congenital Defects Program (MACDP) (Correa, 2007) and European Registration of Congenital Anomalies and Twins (EUROCAT) definitions. For these outcomes, the prevalence will be summarized using all live births as the denominator in computing the rates. A 2-sided 95% confidence interval for these rates will be calculated using exact (Clopper-Pearson) methods. Analysis will be repeated by trimester of first exposure and by the timing of exposure beginning prior to or during pregnancy, with the exposure window of interest for MCMs being the first trimester.