Study of ALXN2220 Versus Placebo in Adults With ATTR-CM

Overview

About this study

The purpose of this study is to assess the effectiveness 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 composite endpoint of all-cause mortality (ACM) and total cardiovascular (CV) clinical events.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

- Centrally confirmed diagnosis of ATTR-CM with either wild-type or variant TTR genotype

- End-diastolic interventricular septal wall thickness ≥ 11 mm for women or ≥ 12 mm for
men 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

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Eligibility last updated 1/9/23. Questions regarding updates should be directed to the study team contact.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Jacksonville, Fla.

Mayo Clinic principal investigator

Jose Nativi Nicolau, M.D.

Open for enrollment

Contact information:

Clinical Studies Unit

(904) 953-2255

More information

Publications

Publications are currently not available