Hereditary Transthyretin-mediated (hATTR) AmyloidosisNCT05040373
Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program
An observational study of Hereditary Transthyretin-mediated (hATTR) Amyloidosis and Polyneuropathy, run by Alnylam Pharmaceuticals at 7 sites in 7 countries.
Trial 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. Read more about this study
- Who is it for?
- Any age, women only
- Conditions the study is for
- Hereditary Transthyretin-mediated (hATTR) Amyloidosis, Polyneuropathy
- How many people
- 10 participants
- Where
- 7 sitesin 7 countries
- Study dates
- 1 Aug 2020to 12 Oct 2030
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- Women only
- No healthy volunteers
- Child, Adult, Older adult
Study population: Any woman exposed to patisiran-LNP 12 weeks prior to their last menstrual period (LMP) or at any time during pregnancy.
Who may be able to join
- 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
Who cannot join
- There are no exclusion criteria for participation in this program.
Trial locations and contacts
7 sites in 7 countries. Contact the study team or a site near you to ask about taking part.
7 sites in 7 countries
United States1 site, 1 recruiting
- Recruiting
Clinical Trial Site
Iowa City, Iowa, 52242
About this study
Official title: Patisiran-LNP Pregnancy Surveillance Program
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.
- Keywords
- Hereditary neuropathic amyloidosis, Familial amyloidosis, Amyloidosis, Amyloidosis senile, Cardiac amyloidosis
- Medical subject headings
- Amyloidosis, Polyneuropathies, Amyloid Neuropathies, Familial, Amyloidosis, Familial
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
Minor congenital malformations will be classified classified according to the EUROCAT and MACDP classification systems.
Measured: From 12 weeks prior to last dose of patisiran through one year after birth (Up to 21 months)
Prevalence of Pregnancy Outcomes
Pregnancy outcomes are defined as live birth, spontaneous abortions, stillbirths, elective abortions, molar or pregnancy, ectopic pregnancy, preterm births, and maternal death.
Measured: 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
Other adverse fetal/neonatal/infant outcomes are defined as low birth weight, failure to thrive, small for gestational age, postnatal growth and development, neonatal, and perinatal, or infant death.
Measured: From 12 weeks prior to last dose of patisiran through one year after birth (Up to 21 months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Sep 2021
- Study start1 Aug 2020
- Primary completion12 Oct 2030 (estimated)
- Study completion12 Oct 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary Transthyretin-mediated (hATTR) AmyloidosisStudies of the same condition, open and under way.
- More trials run by Alnylam PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Alnylam Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Alnylam Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05040373
- Sponsor's protocol ID
- ALN-TTR02-010
- Other IDs
- EUPAS36021
- Status last verified
- Oct 2026
- First submitted
- 14 Jul 2021
- Last updated
- 8 Oct 2026
- Results posted
- No
- Individual data sharing
- No