Recruiting

Patisiran

Sponsor:

Alnylam Pharmaceuticals

Code:

NCT05040373

Conditions

Hereditary Transthyretin-mediated (hATTR) Amyloidosis

Polyneuropathy

Eligibility Criteria

Sex: Female

Age: 0+

Healthy Volunteers: Not accepted

Study Details

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

Study ID

NCT05040373

Start date

Aug 1, 2020

Status verified date

May, 2026

Completion date

Oct 12, 2030

Anticipated

Primary completion date

Oct 12, 2030

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: Female

Age: 0+

Healthy Volunteers: Not accepted

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 Design

Enrollment

10 participants

Anticipated

Interventions and Outcome Measures

Arms

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.

Primary outcome measure

  • 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) ]

Central Contacts and Locations

Central contacts

Alnylam Clinical Trial Information Line

1-877-ALNYLAMclinicaltrials@alnylam.com

Alnylam Clinical Trial Information Line

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

Locations

Clinical Trial Site

Recruiting

Iowa City, Iowa, United States, 52242

More Information

Sponsor

Alnylam Pharmaceuticals

Last update posted

Jun 1, 2026

Last verified

May, 2026

Keywords

  • Hereditary neuropathic amyloidosis
  • Familial amyloidosis
  • Amyloidosis
  • Amyloidosis senile
  • Cardiac amyloidosis

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Alnylam Pharmaceuticals on 2026-06-01.