NCT05071300Phase 3Active not recruiting

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

Study sponsor: Ionis Pharmaceuticals, Inc. · IONS

Compare the available registry edits below. Primary completion is the date data collection ends for the main study measures; it does not tell you when results will be published.

Open ClinicalTrials.gov record CSV export · Pro

Primary completion2029-08Estimated

Enrollment · participants151Actual

Source updated2025-12-04Last update listed in the registry record

Version history

Changes to this trial

Last 12 months

Pro unlocks the full available history and CSV export. View Pro features

No recorded changes are available for this view and time period. Our history may be incomplete; this does not establish that the trial stayed unchanged.

Study details

Conditions studied

Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

Treatments or interventions studied

EplontersenDrugAlso known as AKCEA-TTR-LRx, ION-682884, IONIS-TTR-LRx

Primary outcomes · main study measures

Read the study measures

measure: Change From Baseline in Platelet Count; timeFrame: Baseline to Week 181; measure: Number of Participants With Clinically Significant Changes From Baseline in Renal Function; timeFrame: Baseline to Week 181; measure: Number of Participants with Clinically Significant Changes from Baseline in Transaminases; timeFrame: Baseline to Week 181; measure: Change From Baseline in Adverse Events; timeFrame: Baseline to Week 181; measure: Change From Baseline in Number of Concomitant Medications Used; timeFrame: Baseline to Week 181; measure: Number of Participants With Clinically Significant Changes From Baseline in Vital Signs; timeFrame: Baseline to Week 181; measure: Change From Baseline in Body Weight; timeFrame: Baseline to Week 181; measure: Number of Participants With Clinically Significant Changes From Baseline in Physical Examination Findings; timeFrame: Baseline to Week 181; measure: Number of Participants With Clinically Significant Changes From Baseline in Clinical Laboratory Tests; timeFrame: Baseline to Week 181; measure: Number of Participants With Clinically Significant Changes From Baseline in Electrocardiogram (ECG) Parameters; timeFrame: Baseline to Week 181; measure: Number of Participants With Clinically Significant Changes From Baseline in Thyroid Panel Tests; timeFrame: Baseline to Week 181; measure: Number of Participants With Clinically Significant Changes From Baseline in Coagulation Tests; timeFrame: Baseline to Week 181; measure: Number of Participants With Clinically Significant Changes From Baseline in Inflammatory Panel Tests; timeFrame: Baseline to Week 181; measure: Number of Participants With Clinically Significant Changes From Baseline in Complement and Immunogenicity Tests; timeFrame: Baseline to Week 181

History coverage

Research sample: selected versions from May 2023 onward plus a preceding baseline where available. Only selected fields were retained; endpoint measures were truncated to 300 characters. This is not complete trial or sponsor history. Daily snapshots begin when collection starts.

How to read this history