Patisiran-LNP Pregnancy Surveillance Program

Alnylam Pharmaceuticals8 sites in 7 countries10 target enrollmentAugust 1, 2020

ConditionsHereditary Transthyretin-mediated (hATTR) Amyloidosis Polyneuropathy

Overview

Phase: N/A
Intervention: Patisiran
Conditions: Hereditary Transthyretin-mediated (hATTR) Amyloidosis
Sponsor: Alnylam Pharmaceuticals
Enrollment: 10
Locations: 8
Primary Endpoint: Prevalence of Major Congenital Malformations
Status: Recruiting
Last Updated: 8 days ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

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.

Registry

clinicaltrials.gov

Start Date

August 1, 2020

End Date

October 12, 2030

Last Updated

8 days ago

Study Type

Observational

Sex

Female

Investigators

Sponsor

Alnylam Pharmaceuticals

Responsible Party

Sponsor

Eligibility Criteria

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

Exclusion Criteria

•There are no exclusion criteria for participation in this program.

Arms & Interventions

Patisiran

Pregnant women exposed to commercial patisiran-LNP (ONPATTRO) during the 12 weeks prior to their last menstrual period (LMP) or at any time during pregnancy.

Outcomes

Primary Outcomes

Prevalence of Major Congenital Malformations

Time Frame: 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 Outcomes

Prevalence of Minor Congenital Malformations(From 12 weeks prior to last dose of patisiran through one year after birth (Up to 21 months))
Prevalence of Pregnancy Outcomes(From 12 weeks prior to last dose of patisiran through one year after birth (Up to 21 months))
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))