Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program

NCT05040373 | Recruiting

• 1 other identifier: ALN-TTR02-010
• Study Type: observational
• Target: 10
• Locations: 7 countries
• Sites: 7

Brief Summary

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.

Conditions

Hereditary Transthyretin-mediated (hATTR) Amyloidosis; Polyneuropathy

Keywords

Hereditary neuropathic amyloidosis; Familial amyloidosis; Amyloidosis; Amyloidosis senile; Cardiac amyloidosis

Outcome Measures

Primary Outcomes (1)

• Prevalence of Major Congenital Malformations

  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 Outcomes (3)

• Prevalence of Minor Congenital Malformations

  From 12 weeks prior to last dose of patisiran through one year after birth (Up to 21 months)

• Prevalence of Pregnancy Outcomes

  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

  From 12 weeks prior to last dose of patisiran through one year after birth (Up to 21 months)

Study Arms (1)

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.

Eligibility Criteria

• Sex: female
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• Documentation that the patient was exposed to patisiran-LNP at any point starting from 12 weeks before LMP or at any point during pregnancy

Sponsors & Collaborators

• Alnylam Pharmaceuticals: lead

Study Sites (7)

Clinical Trial Site

Iowa City, Iowa, 52242, United States

RECRUITING

Clinical Trial Site

Nantes, France

RECRUITING

Clinical Trial Site

Münster, Germany

RECRUITING

Clinical Trial Site

Pavia, Italy

RECRUITING

Clinical Trial Site

Groningen, Netherlands

RECRUITING

Clinical Trial Site

Lisbon, Portugal

RECRUITING

Clinical Trial Site

Madrid, Spain

RECRUITING

MeSH Terms

Conditions

Amyloidosis; Polyneuropathies; Amyloid Neuropathies, Familial; Amyloidosis, Familial

Condition Hierarchy (Ancestors)

Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases; Peripheral Nervous System Diseases; Neuromuscular Diseases; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Amyloid Neuropathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors

Study Officials

• Medical Director

  Alnylam Pharmaceuticals

  STUDY DIRECTOR

Central Study Contacts

Alnylam Clinical Trial Information Line

CONTACT

1-877-ALNYLAM; clinicaltrials@alnylam.com

Alnylam Clinical Trial Information Line

CONTACT

1-877-256-9526; clinicaltrials@alnylam.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 14, 2021
• First Posted: September 10, 2021
• Study Start: August 1, 2020
• Primary Completion (Estimated): October 12, 2030
• Study Completion (Estimated): October 12, 2030
• Last Updated: April 24, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1); IT (1); PT (1); US (1); DE (1); NL (1); ES (1)