Transthyretin AmyloidosisEPPRONCT07205666
The Eplontersen Pregnancy and Lactation Outcomes Study
An observational study of Transthyretin Amyloidosis, run by AstraZeneca at 1 site in Germany.
Trial summary
The overall objective of this DPSS is to describe the occurrence of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant associated with exposure to eplontersen during pregnancy and/or lactation. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Transthyretin Amyloidosis
- How many people
- 10 participants
- Where
- 1 siteGermany
- Study dates
- 30 Nov 2025to 30 Dec 2035
- 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
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: The study population will include all PV reports of pregnant and/or lactating individuals diagnosed with an indication approved for use of eplontersen who administered at least one dose of eplontersen during pregnancy or lactation
Who may be able to join
- all pregnancy and/or lactation cases with exposure to eplontersen and a diagnosis of an approved indication for treatment with eplontersen
- all adverse event reports in infants in the first 12 months of age that are or can be linked to pregnancy or lactation reports in individuals previously diagnosed with an approved indication and exposed to eplontersen during pregnancy or lactaction
Who cannot join
- all case reports considered invalid (i.e. minimum data is not provided at first report nor follow-up), or where reporter indicates that they do not wish to be contacted to obtain follow-up information, or the reporter/patient cannot be identified
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
AstraZeneca Clinical Study Information Center contact
About this study
Official title: The Eplontersen Pregnancy and Lactation Outcomes Study (EPPRO): A Descriptive Safety Study of Pregnant and Lactating Individuals and Their Offspring Exposed to Eplontersen
The overall objective of this DPSS is to describe the occurrence of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant associated with exposure to eplontersen during pregnancy and/or lactation.
Read the detailed description
Currently, there are no clinical studies of eplontersen use in pregnant individuals. Although it is expected that exposure to eplontersen during pregnancy or lactation is very rare, it is important to capture as many exposed cases as possible. This descriptive pregnancy safety study (DPSS) will add to the current body of knowledge regarding the safety of eplontersen exposure during pregnancy and lactation and maternal, fetal, and infant outcomes following exposure to eplontersen. The study is designed to fulfill health authorities' post-marketing requirements. This DPSS will analyze secondary data on cases of eplontersen-exposed pregnant and lactating individuals collected through a PRegnancy outcomes Intensive Monitoring (PRIM) enhanced pharmacovigilance (PV) approach utilizing AstraZeneca's PV Global Safety Database (PV-Argus). Data sources:
All eplontersen-exposed cases reported to AstraZeneca PV-Argus as notifiable events related to pregnancy and lactation; and cases reported from clinical trials, spontaneous post-marketing reports, post-marketing observational studies, patient-oriented programs, published literature, and personal communication by healthcare providers will be eligible for inclusion in the analysis. Data on eplontersen-exposed cases will be captured and processed using a pre-specified follow-up schedule and questionnaires. In case of missing data, there will be several attempts to collect data using all contact methods supplied by the reporters at the time of initial report.
Study size:
This DPSS is descriptive in nature, and a formal sample size calculation is not performed. Feasibility calculations indicate that the DPSS may capture five exposed pregnancies over the study period. Data analysis:
Analyses will be conducted in accordance with the study objectives, table/listing shells, and applicable guidelines. Demographics, medical and obstetric history, and disease characteristics will be summarized using descriptive statistics. The outcomes will be reported as a proportion and 95% confidence interval (if applicable) and calculated by dividing the number of cases of the outcome by the appropriate denominator for the particular outcome. If fewer than ten cases are reported, formal analyses will not be performed, and reporting will be limited to case narratives.
- Conditions
- Transthyretin Amyloidosis
- Keywords
- Eplontersen, pregnancy, Wainua, Wainzua
- Medical subject headings
- Amyloidosis, Hereditary, Transthyretin-Related
What is being measured
The primary outcome is the main question the study is designed to answer.
Major congenital malformations
All major congenital malformations diagnosed in utero, at birth or during the the first year of life. Considering the small number of patients anticipated to be included in this study, it is possible that only case narratives will be developed. However, if at least ten exposed pregnancies are reported, the data will be summarized quantitatively, using relevant measures. Major congenital malformation will be reported as numbers and proportions. When presented, the 95% confidence intervals (CIs) for proportion of the outcome of interest will be constructed using the exact (Clopper-Pearson) method.
Measured: November 2025 - December 2035
Secondary outcomes (1)
composite outcome of pregnancy and infant adverse outcomes ( except major congenital malformation
pregnancy complications, spontaneous abortion, stillbirth, induced abortion, preterm birth, small for gestational age, postnatal growth deficiencies, infant development delay, neonatal mortality, infant mortality, as well as any other infant adverse events
Measured: November 2025-December 2035
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Oct 2025
- Study start30 Nov 2025
- Primary completion30 Dec 2035 (estimated)
- Study completion30 Dec 2035 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Transthyretin AmyloidosisStudies of the same condition, open and under way.
- More trials run by AstraZenecaThe 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
- Case only
- Time perspective
- Prospective
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- AstraZeneca
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- ICON plc
- Responsible party
- Sponsor
- Investigators
- Myriam Alexander, PHD, Principal investigator, AstraZeneca
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07205666
- Sponsor's protocol ID
- D8451R00002
- Acronym
- EPPRO
- Status last verified
- Aug 2026
- First submitted
- 17 Sep 2025
- Last updated
- 27 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided: The enhanced pharmacovigilance approach used in the study involves extraction of targeted safety questionnaire for pregnant or breastfeeding women exposed to eplontersen and reporting to AZ as part of routine pharmacovigilance
Publications
Geissbuhler Y, Rezaallah B, Moore A. An alternative to product-specific pregnancy registries? PRIM; PRegnancy outcomes Intensive Monitoring. Reprod Toxicol. 2020 Jun;94:13-21. doi: 10.1016/j.reprotox.2020.03.004. Epub 2020 Mar 10.
PubMed 32169556
Study at a glance
From the registry record, last updated by the sponsor on 27 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Eplontersen Pregnancy and Lactation Outcomes Study (EPPRO): A Descriptive Safety Study of Pregnant and Lactating Individuals and Their Offspring Exposed to Eplontersen
- ClinicalTrials.gov ID
- NCT07205666
- Acronym / sponsor ID
- EPPRO / D8451R00002
- Conditions
- Transthyretin Amyloidosis
- Keywords
- Eplontersen, pregnancy, Wainua, Wainzua
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- AstraZenecaIndustry (pharma, biotech, devices)
- Collaborators
- ICON plc
- Study start
- 30 Nov 2025 (actual)
- Primary completion
- 30 Dec 2035 (estimated)
- Study completion
- 30 Dec 2035 (estimated)
- First posted
- 3 Oct 2025 (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 overall objective of this DPSS is to describe the occurrence of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant associated with exposure to eplontersen during pregnancy and/or lactation.
Detailed description
Currently, there are no clinical studies of eplontersen use in pregnant individuals. Although it is expected that exposure to eplontersen during pregnancy or lactation is very rare, it is important to capture as many exposed cases as possible. This descriptive pregnancy safety study (DPSS) will add to the current body of knowledge regarding the safety of eplontersen exposure during pregnancy and lactation and maternal, fetal, and infant outcomes following exposure to eplontersen. The study is designed to fulfill health authorities' post-marketing requirements. This DPSS will analyze secondary data on cases of eplontersen-exposed pregnant and lactating individuals collected through a PRegnancy outcomes Intensive Monitoring (PRIM) enhanced pharmacovigilance (PV) approach utilizing AstraZeneca's PV Global Safety Database (PV-Argus). Data sources:
All eplontersen-exposed cases reported to AstraZeneca PV-Argus as notifiable events related to pregnancy and lactation; and cases reported from clinical trials, spontaneous post-marketing reports, post-marketing observational studies, patient-oriented programs, published literature, and personal communication by healthcare providers will be eligible for inclusion in the analysis. Data on eplontersen-exposed cases will be captured and processed using a pre-specified follow-up schedule and questionnaires. In case of missing data, there will be several attempts to collect data using all contact methods supplied by the reporters at the time of initial report.
Study size:
This DPSS is descriptive in nature, and a formal sample size calculation is not performed. Feasibility calculations indicate that the DPSS may capture five exposed pregnancies over the study period. Data analysis:
Analyses will be conducted in accordance with the study objectives, table/listing shells, and applicable guidelines. Demographics, medical and obstetric history, and disease characteristics will be summarized using descriptive statistics. The outcomes will be reported as a proportion and 95% confidence interval (if applicable) and calculated by dividing the number of cases of the outcome by the appropriate denominator for the particular outcome. If fewer than ten cases are reported, formal analyses will not be performed, and reporting will be limited to case narratives.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- The study population will include all PV reports of pregnant and/or lactating individuals diagnosed with an indication approved for use of eplontersen who administered at least one dose of eplontersen during pregnancy or lactation
Key inclusion criteria (2 listed)
- all pregnancy and/or lactation cases with exposure to eplontersen and a diagnosis of an approved indication for treatment with eplontersen
- all adverse event reports in infants in the first 12 months of age that are or can be linked to pregnancy or lactation reports in individuals previously diagnosed with an approved indication and exposed to eplontersen during pregnancy or lactaction
Key exclusion criteria (1 listed)
- all case reports considered invalid (i.e. minimum data is not provided at first report nor follow-up), or where reporter indicates that they do not wish to be contacted to obtain follow-up information, or the reporter/patient cannot be identified
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
Major congenital malformations
Time frame: November 2025 - December 2035
All major congenital malformations diagnosed in utero, at birth or during the the first year of life. Considering the small number of patients anticipated to be included in this study, it is possible that only case narratives will be developed. However, if at least ten exposed pregnancies are reported, the data will be summarized quantitatively, using relevant measures. Major congenital malformation will be reported as numbers and proportions. When presented, the 95% confidence intervals (CIs) for proportion of the outcome of interest will be constructed using the exact (Clopper-Pearson) method.
1 secondary outcome
composite outcome of pregnancy and infant adverse outcomes ( except major congenital malformation
Time frame: November 2025-December 2035
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 27 Aug 2026.
- Results publication: PubMed 32169556Geissbuhler Y, Rezaallah B, Moore A. An alternative to product-specific pregnancy registries? PRIM; PRegnancy outcomes Intensive Monitoring. Reprod Toxicol. 2020 Jun;94:13-21. doi: 10.1016/j.reprotox.2020.03.004. Epub 2020 Mar 10.
- Individual participant dataSharing planned: Undecided. The enhanced pharmacovigilance approach used in the study involves extraction of targeted safety questionnaire for pregnant or breastfeeding women exposed to eplontersen and reporting to AZ as part of routine pharmacovigilance
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A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
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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.
AstraZeneca Clinical Study Information Center contact
Myriam Alexander, PHD principal investigator
AstraZeneca
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Transthyretin Amyloidosis, run by AstraZeneca at 1 site in Germany.
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 27 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Germany. 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 AstraZeneca Clinical Study Information Center (1-877-240-9479, information.center@astrazeneca.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Major congenital malformations (time frame: November 2025 - December 2035). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT07205666, which also lists 1 linked publication.
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 27 Aug 2026.