Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program

May 28, 2026 updated by: Alnylam Pharmaceuticals

Patisiran-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.

Study Overview

Status

Recruiting

Conditions

Study Type

Observational

Enrollment (Estimated)

10

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • France
      • Nantes, France
        • Recruiting
        • Clinical Trial Site
  • Germany
      • Münster, Germany
        • Recruiting
        • Clinical Trial Site
  • Italy
      • Pavia, Italy
        • Recruiting
        • Clinical Trial Site
  • Netherlands
      • Groningen, Netherlands
        • Recruiting
        • Clinical Trial Site
  • Portugal
      • Lisbon, Portugal
        • Recruiting
        • Clinical Trial Site
  • Spain
      • Madrid, Spain
        • Recruiting
        • Clinical Trial Site
  • United States
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • Recruiting
        • Clinical Trial Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Any woman exposed to patisiran-LNP 12 weeks prior to their last menstrual period (LMP) or at any time during pregnancy.

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
From 12 weeks prior to last dose of patisiran through one year after birth (Up to 21 months)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
From 12 weeks prior to last dose of patisiran through one year after birth (Up to 21 months)
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.
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
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.
From 12 weeks prior to last dose of patisiran through one year after birth (Up to 21 months)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alnylam Pharmaceuticals

Investigators

  • Study Director: Medical Director, Alnylam Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 1, 2020

Primary Completion (Estimated)

October 12, 2030

Study Completion (Estimated)

October 12, 2030

Study Registration Dates

First Submitted

July 14, 2021

First Submitted That Met QC Criteria

September 7, 2021

First Posted (Actual)

September 10, 2021

Study Record Updates

Last Update Posted (Actual)

June 1, 2026

Last Update Submitted That Met QC Criteria

May 28, 2026

Last Verified

May 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ALN-TTR02-010

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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