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Evaluating ALXN2220 for treating heart disease caused by amyloidosis

A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Amyloid Depleter ALXN2220 in Adult Participants With Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

Phase 3 Interventional Alexion Pharmaceuticals, Inc. · NCT06183931

This study is testing a new treatment called ALXN2220 to see if it can help adults with heart disease caused by amyloidosis feel better and live longer compared to a placebo.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment1000 (estimated)
Ages18 Years to 90 Years
SexAll
SponsorAlexion Pharmaceuticals, Inc. Industry-sponsored
Locations250 sites (Irvine, California and 249 other locations)
Trial IDNCT06183931 on ClinicalTrials.gov

What this trial studies

This study aims to assess the efficacy of ALXN2220 compared to a placebo in adults diagnosed with Transthyretin Amyloid Cardiomyopathy (ATTR-CM). Participants will be monitored for occurrences of all-cause mortality and cardiovascular clinical events. The study includes adults with specific echocardiographic and biomarker criteria, ensuring a targeted approach to treatment. The trial is designed to provide insights into the potential benefits of ALXN2220 in managing this serious condition.

Who should consider this trial

Good fit: Ideal candidates are adults diagnosed with ATTR-CM who have specific echocardiographic findings and elevated NT-proBNP levels.

Not a fit: Patients with other forms of amyloidosis or severe cardiac conditions may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly reduce mortality and improve cardiovascular outcomes for patients with ATTR-CM.

How similar studies have performed: Previous studies have shown promise in treating ATTR-CM with similar approaches, indicating potential for success.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Diagnosis of ATTR-CM with either wild-type or variant TTR genotype
* End-diastolic interventricular septal wall thickness ≥ 12 mm on echocardiography measured at Screening
* NT-proBNP \> 2000 pg/mL at Screening
* Treatment with a loop diuretic for at least 30 days prior to Screening
* History of heart failure NYHA Class II-IV at Screening
* Life expectancy of ≥ 6 months as per the Investigator's judgment
* Males and females of childbearing ability must use contraception

Exclusion Criteria:

* Known leptomeningeal amyloidosis
* Known light chain (AL) or secondary amyloidosis (AA), or any other form of systemic amyloidosis
* Acute coronary syndrome, unstable angina, stroke, transient ischemic attack, coronary revascularization, cardiac device implantation, cardiac valve repair, or major surgery within 3 months of Screening
* Uncontrolled clinically significant cardiac arrhythmia, per Investigator's assessment
* LVEF \< 30% on echocardiography
* Renal failure requiring dialysis or an eGFR \< 20 mL/min/1.73 m2 at Screening
* Polyneuropathy with PND score IV

Where this trial is running

Irvine, California and 249 other locations

+200 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Transthyretin Amyloid CardiomyopathyAmyloid DepleterALXN2220All-cause mortalityCardiovascularTransthyretin amyloidTransthyretinN-terminal pro b-type natriuretic peptide
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.