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
- Are there results?
- Not yet: the study is under way
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
Study at a glance
From the registry record, last updated by the sponsor on 8 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Patisiran-LNP Pregnancy Surveillance Program
- ClinicalTrials.gov ID
- NCT05040373
- Acronym / sponsor ID
- ALN-TTR02-010
- Keywords
- Hereditary neuropathic amyloidosis, Familial amyloidosis, Amyloidosis, Amyloidosis senile, Cardiac amyloidosis
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Alnylam PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 1 Aug 2020 (actual)
- Primary completion
- 12 Oct 2030 (estimated)
- Study completion
- 12 Oct 2030 (estimated)
- First posted
- 10 Sep 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 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. |
Who may qualify
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- 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.
Key inclusion criteria (2 listed)
- 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
Key exclusion criteria (1 listed)
- There are no exclusion criteria for participation in this program.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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)
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.
3 secondary outcomes
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)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Oct 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Medical Director study director
Alnylam Pharmaceuticals
7 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hereditary Transthyretin-mediated (hATTR) Amyloidosis and Polyneuropathy, run by Alnylam Pharmaceuticals at 7 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 8 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States, France, Germany, Italy, Netherlands and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Alnylam Clinical Trial Information Line (1-877-ALNYLAM, clinicaltrials@alnylam.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the 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)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Oct 2026.