Eplontersen for Transthyretin Amyloid Cardiomyopathy

Phase-Based Progress Estimates
Transthyretin Amyloid CardiomyopathyEplontersen - Drug
All Sexes
What conditions do you have?

Study Summary

The purpose of this study is to evaluate the safety and tolerability of extended dosing with eplontersen in participants with ATTR-CM.

Eligible Conditions
  • Transthyretin Amyloid Cardiomyopathy

Treatment Effectiveness

Effectiveness Progress

2 of 3
This is further along than 85% of similar trials

Study Objectives

9 Primary · 7 Secondary · Reporting Duration: Baseline up to 36 months

Month 36
Change From Baseline in 5 Level EuroQol - 5 Dimension (EQ-5D-5L)
Change From Baseline in 6-minute Walk Test (6MWT)
Change From Baseline in ALT
Change From Baseline in AST
Change From Baseline in Biomarker: High Sensitivity Cardiac Troponin T (hs-cTnT)
Change From Baseline in Biomarker: N-terminal prohormone of Brain Natriuretic Peptide (NT-proBNP)
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR)
Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ)
Change From Baseline in Platelet Count
Change From Baseline in Short Form Health Survey Questionnaire (SF-36)
Change From Baseline in Transthyretin (TTR) Serum Levels
Change From Baseline in Urine Protein Creatinine Ratio (UPCR)
Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAE)
Number of Participants With Clinically Significant Changes From Baseline in Electrocardiogram (ECG) Parameters
Number of Participants With Clinically Significant Changes From Baseline in Thyroid-Stimulating Hormone (TSH)
Percentage of Participants With Anti-Drug Antibodies (ADA)

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Trial Design

1 Treatment Group

1 of 1

Experimental Treatment

1400 Total Participants · 1 Treatment Group

Primary Treatment: Eplontersen · No Placebo Group · Phase 3

Experimental Group · 1 Intervention: Eplontersen · Intervention Types: Drug
First Studied
Drug Approval Stage
How many patients have taken this drug
Completed Phase 1

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: baseline up to 36 months

Who is running the clinical trial?

Ionis Pharmaceuticals, Inc.Lead Sponsor
140 Previous Clinical Trials
13,410 Total Patients Enrolled
1 Trials studying Transthyretin Amyloid Cardiomyopathy
80 Patients Enrolled for Transthyretin Amyloid Cardiomyopathy
AstraZenecaIndustry Sponsor
3,988 Previous Clinical Trials
91,526,362 Total Patients Enrolled

Eligibility Criteria

Age 18+ · All Participants · 3 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You have either satisfactorily completed the Treatment Period and End of Treatment Visit for ION-682884-CS2 OR you are diagnosed with ATTR-CM and participated adequately in ISIS 420915 - CS101 as judged by the Investigator and Sponsor.
The investigator is prepared to administer open-label eplontersen therapy.
You are willing to take vitamin A supplements as instructed.