Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program

Recruiting

• Conditions: Polyneuropathy; Hereditary Transthyretin-mediated (hATTR) Amyloidosis

• Registration Number: NCT05040373
• Lead Sponsor: Alnylam Pharmaceuticals

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2020-08-01
• Study First Submitted: 2021-07-14
• Study First Submitted QC: 2021-09-07
• Study First Posted: 2021-09-10
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-09
• Last Update Posted: 2025-05-13
• Primary Completion: 2030-10-12
• Study Completion: 2030-10-12

Recruitment & Eligibility

• Status: RECRUITING
• Sex: Female
• Target Recruitment: 10

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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.

Trial Locations

Locations (1)

Clinical Trial Site; 🇪🇸 Madrid, Spain