Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program

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.

Recruiting

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Key information

Conditions

Hereditary Transthyretin-mediated (hATTR) Amyloidosis Amyloid Neuropathies Amyloid Neuropathies, Familial Amyloidosis Amyloidosis, Familial Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Nutritional and Metabolic Diseases Peripheral Nervous System Diseases Polyneuropathies Polyneuropathy Proteostasis Deficiencies

Sex eligibility

Female

Study type

Observational

Primary location

Clinical Trial Site, Nantes, France

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

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

Time frame: 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

Time frame: 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

Time frame: 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.

Study contacts

Contact information is provided by the study sponsor or research team.

Alnylam Clinical Trial Information Line

CONTACT

[email protected]

1-877-ALNYLAM

Alnylam Clinical Trial Information Line

CONTACT

[email protected]

1-877-256-9526

Sponsors and collaborators

Lead sponsor

Alnylam Pharmaceuticals

Industry

Registry information

Official study title

Patisiran-LNP Pregnancy Surveillance Program

Important dates

Study start: 2020
Primary completion: 2030
Study completion: 2030
First posted: Sep 10, 2021
Registry last updated: Jun 1, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed