Hereditary Transthyretin-mediated (hATTR) Amyloidosis, Polyneuropathy
Conditions
Keywords
Hereditary neuropathic amyloidosis, Familial amyloidosis, Amyloidosis, Amyloidosis senile, Cardiac amyloidosis
Brief summary
The purpose of this study is to collect and evaluate pregnancy outcomes, pregnancy complications, and fetal/neonatal/infant outcomes in women exposed to patisiran-LNP.
Interventions
None listed
Sponsors
Alnylam Pharmaceuticals
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
FEMALE
Healthy volunteers
No
Inclusion criteria
* Documentation that the patient was exposed to patisiran-LNP at any point starting from 12 weeks before LMP or at any point during pregnancy * Germany Only: Patients must be treated per the summary of product characteristics (SmPC) for any approved treatment for ATTR amyloidosis
Exclusion criteria
* There are no
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Prevalence of Major Congenital Malformations | From 12 weeks prior to last dose of patisiran through one year after birth (Up to 21 months) | Major congenital malformations will be classified according to the European Concerted Action on Congenital Anomalies and Twins (EUROCAT) and Metropolitan Atlanta Congenital Defects Program (MACDP) classification systems. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Prevalence of Minor Congenital Malformations | From 12 weeks prior to last dose of patisiran through one year after birth (Up to 21 months) | Minor congenital malformations will be classified classified according to the EUROCAT and MACDP classification systems. |
| Prevalence of Pregnancy Outcomes | From 12 weeks prior to last dose of patisiran through one year after birth (Up to 21 months) | Pregnancy outcomes are defined as live birth, spontaneous abortions, stillbirths, elective abortions, molar or pregnancy, ectopic pregnancy, preterm births, and maternal death. |
| Prevalence of Other Adverse Fetal/Neonatal/Infant Outcomes | From 12 weeks prior to last dose of patisiran through one year after birth (Up to 21 months) | Other adverse fetal/neonatal/infant outcomes are defined as low birth weight, failure to thrive, small for gestational age, postnatal growth and development, neonatal, and perinatal, or infant death. |
Countries
France, Germany, Italy, Netherlands, Portugal, Spain, United States
Contacts
CONTACTAlnylam Clinical Trial Information Line
STUDY_DIRECTORMedical Director
Alnylam Pharmaceuticals
Outcome results
None listed