Actively Recruiting

FEMALE
ID05040373

Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program

Led by Alnylam Pharmaceuticals · Updated on 2026-06-01

10

Participants Needed

7

Research Sites

N/A

Total Duration

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AI-Summary

What this Trial Is About

This research aims to collect and evaluate pregnancy outcomes and complications in women exposed to patisiran-LNP, a treatment used for hereditary transthyretin-mediated (hATTR) amyloidosis and polyneuropathy. The study focuses on monitoring the effects on fetal, neonatal, and infant health after exposure during pregnancy. The program observes pregnant women who have taken commercial patisiran-LNP (ONPATTRO) either within 12 weeks before their last menstrual period (LMP) or at any time during pregnancy. There are no intervention groups or treatments assigned in this observational study, as it monitors outcomes related to prior exposure. Participants will be followed from the time of exposure through pregnancy and up to one year after birth, totaling up to 21 months. Researchers will track the prevalence of major and minor congenital malformations, pregnancy outcomes, and other adverse fetal, neonatal, or infant outcomes. The study gathers data to better understand the safety and effects of patisiran-LNP during pregnancy.

CONDITIONS

Brief Title

Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program

Who Can Participate

FEMALE

Eligibility Criteria

Eligible

You may qualify if you...

  • Documentation that the patient was exposed to patisiran-LNP at any point starting from 12 weeks before last menstrual period or at any point during pregnancy
Not Eligible

You will not qualify if you...

History of severe allergic reactions to study medication Currently pregnant or breastfeeding Recent participation in another clinical trial within the last 30 days Presence of uncontrolled medical conditions that could affect safety

AI-Screening

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Surveillance

Duration - Up to 21 months from 12 weeks prior to last dose through one year after birth

Participants who were exposed to patisiran-LNP during pregnancy or in the 12 weeks prior are observed for pregnancy and infant outcomes.

Periodic visits based on pregnancy and infant assessments

Trial Site Locations

Total: 7 locations

1

Clinical Trial Site

Iowa City, Iowa, United States, 52242

Actively Recruiting

2

Clinical Trial Site

Nantes, France

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3

Clinical Trial Site

Münster, Germany

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4

Clinical Trial Site

Pavia, Italy

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5

Clinical Trial Site

Groningen, Netherlands

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6

Clinical Trial Site

Lisbon, Portugal

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7

Clinical Trial Site

Madrid, Spain

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Research Team

A

Alnylam Clinical Trial Information Line

A

Alnylam Clinical Trial Information Line

How is the study designed?

Study Type

OBSERVATIONAL

Masking

N/A

Allocation

N/A

Model

N/A

Primary Purpose

N/A

Number of Arms

1

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