Study of ALXN2220 Versus Placebo in Adults With ATTR-CM

Eligibility: for people ages 18-90 (full criteria)
Location: at UCSF
Dates: study started January 2024
completion around October 2028

Description

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.

Official Title

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)

Keywords

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

Eligibility

You can join if…

Open to people ages 18-90

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

You CAN'T join if...

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

Locations

Research Site accepting new patients
San Francisco California 94143 United States
Research Site accepting new patients
La Jolla California 92037 United States
Research Site accepting new patients
Irvine California 92614 United States
Research Site accepting new patients
Palo Alto California 94304 United States

Details

Status: accepting new patients
Start Date: January 2024
Completion Date: October 2028 (estimated)
Sponsor: Alexion Pharmaceuticals, Inc.
ID: NCT06183931
Phase: Phase 3 Cardiomyopathy Research Study
Study Type: Interventional
Participants: Expecting 1000 study participants
Last Updated: February 26, 2025