ClinicalTrials.gov record NCT05040373

Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 17, 2026

Patisiran-LNP Pregnancy Surveillance Program

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeObservationalA study in which researchers do not assign participants to a treatment or other intervention. They observe or measure what happens, which may include study visits, tests, questionnaires or a review of medical records.
SexFemale
LocationFrance, Germany, Italy, Netherlands, Portugal, Spain, United States
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This prospective observational program follows women exposed to patisiran-LNP (including commercial ONPATTRO) before or during pregnancy. It collects information on major and minor congenital malformations, pregnancy outcomes, and other adverse fetal/neonatal/infant outcomes. Outcomes are recorded from exposure starting 12 weeks before last menstrual period or during pregnancy through one year after birth (up to 21 months).

What it is studying. The study is collecting and evaluating pregnancy, fetal, neonatal, and infant outcomes after exposure to patisiran-LNP.

What the researchers hope to learn. To collect and evaluate the prevalence of major and minor congenital malformations, pregnancy outcomes, and other adverse fetal/neonatal/infant outcomes after exposure to patisiran-LNP.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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
  • Germany Only: Patients must be treated per the summary of product characteristics (SmPC) for any approved treatment for ATTR amyloidosis

Exclusion criteria

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

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

If you take part, researchers will collect and evaluate information about your pregnancy, any pregnancy complications, and your baby's health after birth. They will follow outcomes from 12 weeks before the last dose of patisiran through one year after birth (up to 21 months). The study will record major and minor congenital malformations (classified using EUROCAT and MACDP), pregnancy outcomes (for example live birth, spontaneous abortion, stillbirth, elective abortion, molar or ectopic pregnancy, preterm birth, and maternal death), and other infant outcomes (such as low birth weight, failure to thrive, small for gestational age, postnatal growth and development, and neonatal or infant death). The registry record does not list specific study visits, tests, or sample collections beyond collecting and evaluating these outcome data.

How the study is structured. This is a prospective observational cohort. Women who were exposed to patisiran-LNP before or during pregnancy are followed and outcomes are recorded; the study does not assign treatments. Key outcomes include prevalence of major and minor congenital malformations, pregnancy outcomes (for example live birth, spontaneous abortion, stillbirth, elective abortion, molar or ectopic pregnancy, preterm birth, maternal death), and other adverse fetal/neonatal/infant outcomes, measured from exposure through one year after birth (up to 21 months).

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The registry lists this study under Hereditary Transthyretin-mediated (hATTR) Amyloidosis; Polyneuropathy. Does it apply to my diagnosis?
  • This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
  • The listed sites are in France, Germany, Italy, Netherlands, Portugal, Spain, United States. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on wild-type ATTR amyloidosis

This trial is also listed on this site under ATTRv amyloidosis.

Clinical trials121 trials listed for wild-type ATTR amyloidosisEvery trial the registry lists for wild-type ATTR amyloidosis, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in wild-type ATTR amyloidosis researchLatest monthly update: September 2026. Includes: Frailty and support needs. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Aug 1, 2020
Primary completion
Oct 12, 2030 (estimated)
Study completion
Oct 12, 2030 (estimated)
Target enrollment
10 participants (estimated)
Last updated on registry
Oct 8, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Alnylam Pharmaceuticals

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (7)

  • Clinical Trial SiteRecruiting

    Iowa City, Iowa, 52242 · United States

  • Clinical Trial SiteRecruiting

    Nantes · France

  • Clinical Trial SiteRecruiting

    Münster · Germany

  • Clinical Trial SiteRecruiting

    Pavia · Italy

  • Clinical Trial SiteRecruiting

    Groningen · Netherlands

  • Clinical Trial SiteRecruiting

    Lisbon · Portugal

  • Clinical Trial SiteRecruiting

    Madrid · Spain

Terms used on this page · explanations by MedicalUpdates.ai

exclusion criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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