Study of ALXN2220 Versus Placebo in Adults With ATTR-CM

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)

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT06183931

Acronym

DepleTTR-CM

Enrollment

1181

Registered

2023-12-28

Start date

2024-01-11

Completion date

2028-04-05

Last updated

2026-08-10

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Transthyretin Amyloid Cardiomyopathy

Keywords

Amyloid Depleter, ALXN2220, All-cause mortality (ACM), Cardiovascular (CV), Transthyretin Amyloid Cardiomyopathy, Transthyretin amyloid (ATTR), Transthyretin (TTR), N-terminal pro b-type natriuretic peptide (NT-proBNP), NT-proBNP, Amyloidosis, ATTR-CM

Brief summary

The primary objective of this study is to access the efficacy of ALXN2220 in the treatment of adult participants with ATTR-CM by evaluating the difference between the ALXN2220 and placebo groups as assessed by the total occurrences of all-cause mortality (ACM) and cardiovascular (CV) clinical events.

Interventions

DRUGALXN2220

Participants will receive ALXN2220 via IV infusion.

DRUGPlacebo

Participants will receive placebo via IV infusion.

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)

Eligibility

Sex/Gender

ALL

Age

18 Years to 90 Years

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame
Total Occurrence of ACM and CV clinical events during the Blinded Treatment Period	Baseline up to the end of study (up to Month 48)

Secondary

Measure	Time frame
Change From Baseline in Kansas City Cardiomyopathy Questionnaire-Overall Score (KCCQ-OS) at Month 30	Baseline, Month 30
Time to CV-related Mortality	Baseline up to the end of study (up to Month 48)
Change from Baseline in Six-minute Walk Test (6MWT) at Month 30	Baseline, Month 30
Total Occurrences of CV Clinical Events	Baseline up to the end of study (up to Month 48)
Time to ACM	Baseline up to the end of study (up to Month 48)

Countries

Argentina, Australia, Austria, Belgium, Brazil, Canada, China, Czechia, Denmark, France, Germany, Greece, Ireland, Israel, Italy, Japan, Netherlands, Norway, Poland, South Korea, Spain, Sweden, Switzerland, Taiwan, Turkey (Türkiye), United Kingdom, United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Aug 11, 2026