An observational study in Transthyretin Amyloidosis, sponsored by AstraZeneca. Recruiting at 1 site in Germany. Per ClinicalTrials.gov, last updated 2026-08-27.
Sponsored by AstraZeneca · Observational
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Among 17 studies in Amyloidosis, Hereditary, Transthyretin-Related with a site in Germany — 7 open to participants now, 10 under way, 3 of them phase 3. Amyloidosis, Hereditary, Transthyretin-Related · Germany.
Among the 17 studies in Amyloidosis, Hereditary, Transthyretin-Related with a site in Germany, this one is the 9th largest by planned enrollment of the 10 observational ones that give a count, is one of 3 that started in 2025, is one of 10 observational ones, and was the 15th registered.
82 studies on the registry are indexed under Amyloidosis, Hereditary, Transthyretin-Related; 28 are open to participants now.
Browse Amyloidosis, Hereditary, Transthyretin-Related studies →7 of the 17 studies in Amyloidosis, Hereditary, Transthyretin-Related with a site in Germany are open now. Recruiting first, the newest first:
AstraZeneca · Sites in Germany, United States, Canada and 3 more countries
Ages 18+
Bayer · Sites in Germany, Austria, Belgium and 14 more countries
Ages 18+
Eidos Therapeutics, a BridgeBio company · Sites in Germany, United States, Argentina and 21 more countries
Ages 18–75
AstraZeneca · Sites in Germany, United States, Canada and 3 more countries
Ages 18–130
Intellia Therapeutics · Sites in Germany, United States, Argentina and 23 more countries
Ages 18–90
A study can be open while its sites in Germany have not opened yet or have stopped. Only those recruiting at a site in Germany →
Of the 22 Amyloidosis, Hereditary, Transthyretin-Related studies recruiting now, 15 (68%) set no upper age limit and 1 (5%) stops at 75 or younger; 2 take children under 18; 4 take healthy volunteers.
This study: all sexes · no healthy volunteers.
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17 studies; the count open now beside each. This study's row is marked.
First in 2008; this study’s year is marked. 3 started in 2025.
This study's planned enrollment of 10 is below the median of 100 across 40 observational studies indexed under Amyloidosis, Hereditary, Transthyretin-Related.
Median enrollment 225 across the 7 interventional studies, 1,250 across the 10 observational. Of the 6 that completed or were terminated, 5 (83%) have results posted.
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16 of 17 have an industry lead sponsor. The 9 most frequent:
AstraZeneca is the lead sponsor of 3,503 studies on the registry; 273 are open to participants now.
Of its 369 completed or terminated interventional studies of FDA-regulated products, 178 (48%) have results posted.
AstraZeneca has 6 studies filed under Amyloidosis, Hereditary, Transthyretin-Related — fewer than ten, so there is no comparison to draw.
None of the studies under way reaches primary completion within a year.
Among the 17 studies in Amyloidosis, Hereditary, Transthyretin-Related with a site in Germany, from their registry updates:
Nothing recorded among them since 2026-09-25.
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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.
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.
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
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Exclusion Criteria:
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.
Time frame: November 2025 - December 2035
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
Time frame: November 2025-December 2035
Plan to share: 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
Started Nov 2025 (actual). Primary completion is estimated for Dec 2035, 121 months after the start.
Of 25 observational studies under Amyloidosis, Hereditary, Transthyretin-Related, half reached primary completion within 93 months of starting (the median), counting those still running; a quarter within 29 and three quarters within 97. This study's planned 121 months is above that.
How these figures are worked out, and which studies are left out of them.
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Amyloidosis, Hereditary, Transthyretin-Related →