AKCEA-TTR-LRx for Transthyretin Amyloid Cardiomyopathy

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
Transthyretin Amyloid CardiomyopathyAKCEA-TTR-LRx - Drug
Eligibility
18 - 90
All Sexes
What conditions do you have?
Select

Study Summary

This trial is testing a new drug compared to placebo (a "sugar pill" with no medicine) to see if it can help people with a certain heart condition.

Eligible Conditions
  • Transthyretin Amyloid Cardiomyopathy

Treatment Effectiveness

Effectiveness Progress

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

Study Objectives

2 Primary · 11 Secondary · Reporting Duration: Baseline up to Week 140

Baseline to Week 120
CV Clinical Events at Week 120
CV Mortality at Week 120
Change From Baseline in the 6MWT Distance at Week 120
Change From Baseline in the Kansas City Cardiomyopathy Questionnaire (KCCQ) Scores at Week 120
Composite Outcome of Cardiovascular (CV) Mortality and recurrent CV clinical events at Week 120
Rate of All-Cause Mortality at Week 120
Rate of CV Clinical Events at Week 120
Baseline to Week 121
Change From Baseline in the 6-minute Walk Test (6MWT) Distance at Week 121
Change From Baseline in the Kansas City Cardiomyopathy Questionnaire (KCCQ) Scores at Week 121
Week 140
All-Cause Mortality up to Week 140
CV Clinical Events up to Week 140
CV Mortality up to Week 140
Composite Outcome of Cardiovascular (CV) Mortality and Recurrent CV Clinical Events up to Week 140

Trial Safety

Safety Progress

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

Trial Design

3 Treatment Groups

Eplontersen
1 of 3
AKCEA-TTR-LRx
1 of 3
Placebo
1 of 3

Experimental Treatment

Non-Treatment Group

1400 Total Participants · 3 Treatment Groups

Primary Treatment: AKCEA-TTR-LRx · Has Placebo Group · Phase 3

Eplontersen
Drug
Experimental Group · 1 Intervention: Eplontersen · Intervention Types: Drug
AKCEA-TTR-LRx
Drug
Experimental Group · 1 Intervention: AKCEA-TTR-LRx · Intervention Types: Drug
Placebo
Drug
PlaceboComparator Group · 1 Intervention: Placebo · Intervention Types: Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
AKCEA-TTR-LRx
Not yet FDA approved
Eplontersen
2020
Completed Phase 1
~30

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: baseline up to week 140

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

Eligibility Criteria

Age 18 - 90 · All Participants · 5 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
A thickened septum is present on an echocardiogram.
You are non-pregnant and non-lactating, and either surgically sterile or post-menopausal or abstinent
You are male and you are either sterile or you are not engaged in sexual relations with a woman of child-bearing potential.