Study of ALXN2220 Versus Placebo in Adults With ATTR-CM

NCT ID: NCT06183931

Last Updated: 2025-12-17

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

1160 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-01-11

Study Completion Date

2027-10-05

Brief Summary

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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.

Detailed Description

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Conditions

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Transthyretin Amyloid Cardiomyopathy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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ALXN2220

Participants will receive weight-based dose of ALXN2220 via intravenous (IV) infusion every 4 weeks (q4w) for at least 24 months up to a maximum of 48 months.

Group Type EXPERIMENTAL

ALXN2220

Intervention Type DRUG

Participants will receive ALXN2220 via IV infusion.

Placebo

Participants will receive placebo via IV infusion q4w for at least 24 months up to a maximum of 48 months.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Participants will receive placebo via IV infusion.

Interventions

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ALXN2220

Participants will receive ALXN2220 via IV infusion.

Intervention Type DRUG

Placebo

Participants will receive placebo via IV infusion.

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Maximum Eligible Age

90 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Alexion Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Research Site

Irvine, California, United States

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La Jolla, California, United States

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Palo Alto, California, United States

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San Francisco, California, United States

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Aurora, Colorado, United States

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Washington D.C., District of Columbia, United States

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Jacksonville, Florida, United States

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Weston, Florida, United States

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Atlanta, Georgia, United States

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Atlanta, Georgia, United States

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Chicago, Illinois, United States

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Indianapolis, Indiana, United States

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Boston, Massachusetts, United States

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Boston, Massachusetts, United States

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Kansas City, Missouri, United States

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St Louis, Missouri, United States

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New York, New York, United States

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New York, New York, United States

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Stony Brook, New York, United States

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Chapel Hill, North Carolina, United States

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Durham, North Carolina, United States

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Cleveland, Ohio, United States

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Columbus, Ohio, United States

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Portland, Oregon, United States

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Danville, Pennsylvania, United States

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Philadelphia, Pennsylvania, United States

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Charleston, South Carolina, United States

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Greenville, South Carolina, United States

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Memphis, Tennessee, United States

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Fort Worth, Texas, United States

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Houston, Texas, United States

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Salt Lake City, Utah, United States

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Falls Church, Virginia, United States

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Richmond, Virginia, United States

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Seattle, Washington, United States

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Madison, Wisconsin, United States

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Buenos Aires, , Argentina

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CABA, , Argentina

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Ciudad de Buenos Aires, , Argentina

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Ciudad de Buenos Aires, , Argentina

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Córdoba, , Argentina

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Bedford Park, , Australia

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Box Hill, , Australia

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Brisbane, , Australia

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Darlinghurst, , Australia

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Joondalup, , Australia

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Melbourne, , Australia

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Westmead, , Australia

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Graz, , Austria

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Innsbruck, , Austria

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Vienna, , Austria

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Vienna, , Austria

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Wels, , Austria

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Bruges, , Belgium

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Brussels, , Belgium

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Genk, , Belgium

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Aracaju, , Brazil

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Campinas, , Brazil

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Fortaleza, , Brazil

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Porto Alegre, , Brazil

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Ribeirão Preto, , Brazil

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Ribeirão Preto, , Brazil

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Rio de Janeiro, , Brazil

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Rio de Janeiro, , Brazil

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São Paulo, , Brazil

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São Paulo, , Brazil

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São Paulo, , Brazil

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São Paulo, , Brazil

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Calgary, Alberta, Canada

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Winnipeg, Manitoba, Canada

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London, Ontario, Canada

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Toronto, Ontario, Canada

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Chicoutimi, Quebec, Canada

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Montreal, Quebec, Canada

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Montreal, Quebec, Canada

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Rimouski, Quebec, Canada

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Beijing, , China

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Beijing, , China

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Beijing, , China

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Beijing, , China

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Changsha, , China

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Chengdu, , China

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Chengdu, , China

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Chongqing, , China

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Fuzhou, , China

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Guangzhou, , China

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Guiyang, , China

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Hangzhou, , China

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Hangzhou, , China

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Harbin, , China

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Hefei, , China

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Jinan, , China

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Nanjing, , China

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Shanghai, , China

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Tianjin, , China

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Wuhan, , China

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Xi'an, , China

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Prague, , Czechia

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Prague, , Czechia

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Aarhus, , Denmark

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Odense C, , Denmark

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Bron, , France

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Chambray-lès-Tours, , France

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Créteil, , France

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Marseille, , France

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Montpellier, , France

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Paris, , France

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Paris, , France

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Pessac, , France

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Poitiers, , France

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Rennes, , France

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Saint-Herblain, , France

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Toulouse, , France

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Berlin, , Germany

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Cologne, , Germany

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Essen, , Germany

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Giessen, , Germany

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Hamburg, , Germany

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Hanover, , Germany

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Heidelberg, , Germany

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Homburg, , Germany

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Kiel, , Germany

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Leipzig, , Germany

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München, , Germany

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Münster, , Germany

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Würzburg, , Germany

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Athens, , Greece

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Dublin, , Ireland

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Beer Yaakov, , Israel

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Haifa, , Israel

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Haifa, , Israel

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Jerusalem, , Israel

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Jerusalem, , Israel

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Petah Tikva, , Israel

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Rehovot, , Israel

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Bologna, , Italy

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Ferrara, , Italy

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Florence, , Italy

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Forlì, , Italy

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Messina, , Italy

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Milan, , Italy

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Napoli, , Italy

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Padua, , Italy

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Pavia, , Italy

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Pisa, , Italy

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Roma, , Italy

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Torino, , Italy

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Trieste, , Italy

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Bunkyō City, , Japan

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Kumamoto, , Japan

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Kurume-shi, , Japan

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Kyoto, , Japan

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Matsumoto-shi, , Japan

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Sapporo, , Japan

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Shinjuku-ku, , Japan

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Suita-shi, , Japan

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Groningen, , Netherlands

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Maastricht, , Netherlands

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Rotterdam, , Netherlands

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Utrecht, , Netherlands

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Lørenskog, , Norway

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Oslo, , Norway

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Trondheim, , Norway

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Gdansk, , Poland

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Poznan, , Poland

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Warsaw, , Poland

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Busan, , South Korea

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Seoul, , South Korea

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Seoul, , South Korea

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Seoul, , South Korea

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Seoul, , South Korea

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Seoul, , South Korea

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Wŏnju, , South Korea

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A Coruña, , Spain

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Barcelona, , Spain

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El Palmar, , Spain

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Jaén, , Spain

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L'Hospitalet de Llobregat, , Spain

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Madrid, , Spain

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Majadahonda, , Spain

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Málaga, , Spain

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Palma de Mallorca, , Spain

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Pamplona, , Spain

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Salamanca, , Spain

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Santiago de Compostela-Coruña, , Spain

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Valencia, , Spain

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Gothenburg, , Sweden

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Lund, , Sweden

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Skellefteå, , Sweden

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Stockholm, , Sweden

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Basel, , Switzerland

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Bern, , Switzerland

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Lucerne, , Switzerland

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Sankt Gallen, , Switzerland

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Zurich, , Switzerland

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New Taipei City, , Taiwan

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Taichung, , Taiwan

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Taichung, , Taiwan

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Taipei, , Taiwan

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Taipei, , Taiwan

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Ankara, , Turkey (Türkiye)

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Dinar, , Turkey (Türkiye)

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Eskişehir, , Turkey (Türkiye)

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Istanbul, , Turkey (Türkiye)

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Mersin, , Turkey (Türkiye)

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Birmingham, , United Kingdom

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Cardiff, , United Kingdom

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Glasgow, , United Kingdom

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Hexham, , United Kingdom

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London, , United Kingdom

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London, , United Kingdom

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Manchester, , United Kingdom

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Countries

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United States 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

Other Identifiers

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D6810C00001

Identifier Type: -

Identifier Source: org_study_id