A Study to Assess the Long-Term Safety and Efficacy of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
← catalyst calendarNCT05071300 · readout ≤ 1,108 d
Sponsored by Ionis Pharmaceuticals, Inc. (industry) · IONS — their whole pipeline →.
Phase
Phase 3
Status
Active, not recruiting
Study type
Interventional
Design
Na · None · Treatment
Enrollment
151actual
Sites
33
Countries
Argentina, Australia, Brazil +10
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2022-01-04 | actual | When the study began enrolling |
| Primary completion | Aug 2029 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | Aug 2029 | estimated | The whole study's end, after follow-up |
| First posted | 2021-10-08 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2025-12-04 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Intervention
- Drug: Eplontersen — also filed as AKCEA-TTR-LRx, ION-682884, IONIS-TTR-LRx
Primary outcomes
what the primary-completion date above is the date OFChange From Baseline in Platelet Count
measured Baseline to Week 181
Number of Participants With Clinically Significant Changes From Baseline in Renal Function
measured Baseline to Week 181
Number of Participants with Clinically Significant Changes from Baseline in Transaminases
measured Baseline to Week 181
Change From Baseline in Adverse Events
measured Baseline to Week 181
Change From Baseline in Number of Concomitant Medications Used
measured Baseline to Week 181
Number of Participants With Clinically Significant Changes From Baseline in Vital Signs
measured Baseline to Week 181
Change From Baseline in Body Weight
measured Baseline to Week 181
Number of Participants With Clinically Significant Changes From Baseline in Physical Examination Findings
measured Baseline to Week 181
Number of Participants With Clinically Significant Changes From Baseline in Clinical Laboratory Tests
measured Baseline to Week 181
Number of Participants With Clinically Significant Changes From Baseline in Electrocardiogram (ECG) Parameters
measured Baseline to Week 181
Number of Participants With Clinically Significant Changes From Baseline in Thyroid Panel Tests
measured Baseline to Week 181
Number of Participants With Clinically Significant Changes From Baseline in Coagulation Tests
measured Baseline to Week 181
Number of Participants With Clinically Significant Changes From Baseline in Inflammatory Panel Tests
measured Baseline to Week 181
Number of Participants With Clinically Significant Changes From Baseline in Complement and Immunogenicity Tests
measured Baseline to Week 181
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