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Recruiting NCT05040373

Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program

Observational Hereditary Transthyretin-mediated (hATTR) Amyloidosis Polyneuropathy

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
This is an observational study: the protocol does not assign a study treatment.
Who it may be relevant to
Registry conditions: Hereditary Transthyretin-mediated (hATTR) Amyloidosis, Polyneuropathy. Basic parameters: No limits · Female.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, France, Germany, Italy, Netherlands +2
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Official title

Patisiran-LNP Pregnancy Surveillance Program

Overview

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.

Primary outcome measures

  • 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)]
Secondary outcome measures (3)
  • 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)]
  • Prevalence of Pregnancy Outcomes [Time frame: 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)]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Cohort

Study locations

United States · 1 center
  • Clinical Trial Site — Iowa City
France · 1 center
  • Clinical Trial Site — Nantes
Germany · 1 center
  • Clinical Trial Site — Münster
Italy · 1 center
  • Clinical Trial Site — Pavia
Netherlands · 1 center
  • Clinical Trial Site — Groningen
Portugal · 1 center
  • Clinical Trial Site — Lisbon
Spain · 1 center
  • Clinical Trial Site — Madrid

Identifiers

NCT: NCT05040373 · ALN-TTR02-010

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗