This study is in progress, not accepting new patients

Study of ALXN2220 Versus Placebo in Adults With ATTR-CM

a study on Cardiomyopathy

Summary

Eligibility
for people ages 18-90 (full criteria)
Location
at Irvine, California and other locations
Dates
study started
study ends around
Principal Investigator
by Roxana Ghashghaei
Headshot of Roxana Ghashghaei
Roxana Ghashghaei

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

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
    Irvine California 92614 United States
  • Research Site
    La Jolla California 92037 United States

Lead Scientist at UC Irvine

  • Roxana Ghashghaei
    Associate Clinical Professor, Medicine, School of Medicine. Authored (or co-authored) 18 research publications

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Alexion Pharmaceuticals, Inc.
ID
NCT06183931
Phase
Phase 3 Cardiomyopathy Research Study
Study Type
Interventional
Participants
About 1181 people participating
Last Updated