New study tracks pregnancy safety of rare disease drug
NCT ID NCT05040373
First seen Jun 27, 2026 · Last updated Sep 09, 2026 · Updated 2 times
Summary
This study monitors pregnancy outcomes in women who took patisiran-LNP for hereditary ATTR amyloidosis. Researchers will track birth defects, pregnancy complications, and infant health. The goal is to better understand the drug's safety during pregnancy, not to test a new treatment.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Participants
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About 10 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Aug 2020
- Expected to finish
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Oct 2030
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
Who is studied
Any woman exposed to patisiran-LNP 12 weeks prior to their last menstrual period (LMP) or at any time during pregnancy.
- Ages
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Children (under 18), adults (18 to 64) and older adults (65 and over)
- Sex
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Female participants only
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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 * Germany Only: Patients must be treated per the summary of product characteristics (SmPC) for any approved treatment for ATTR amyloidosis Exclusion Criteria: * There are no exclusion criteria for participation in this program.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
7 sites in 7 countries. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Clinical Trial Site
RECRUITINGIowa City, Iowa, 52242, United States
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Clinical Trial Site
RECRUITINGNantes, France
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Clinical Trial Site
RECRUITINGMünster, Germany
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Clinical Trial Site
RECRUITINGPavia, Italy
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Clinical Trial Site
RECRUITINGGroningen, Netherlands
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Clinical Trial Site
RECRUITINGLisbon, Portugal
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Clinical Trial Site
RECRUITINGMadrid, Spain
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