Study of ALXN2220 Versus Placebo in Adults With ATTR-CM (DepleTTR-CM)

February 20, 2026 updated by: Alexion Pharmaceuticals, Inc.

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)

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.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

1181

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Argentina
      • Buenos Aires, Argentina, 1425
        • Research Site
      • CABA, Argentina, C1025ABI
        • Research Site
      • Ciudad de Buenos Aires, Argentina, C1181ACH
        • Research Site
      • Ciudad de Buenos Aires, Argentina, C1428
        • Research Site
      • Córdoba, Argentina, X5016KEH
        • Research Site
  • Australia
      • Bedford Park, Australia, 5042
        • Research Site
      • Box Hill, Australia, 3128
        • Research Site
      • Brisbane, Australia, 4102
        • Research Site
      • Darlinghurst, Australia, 2010
        • Research Site
      • Joondalup, Australia, 6027
        • Research Site
      • Melbourne, Australia, 3004
        • Research Site
      • Westmead, Australia, 2145
        • Research Site
  • Austria
      • Graz, Austria, 8036
        • Research Site
      • Innsbruck, Austria, 6020
        • Research Site
      • Vienna, Austria, 1090
        • Research Site
      • Vienna, Austria, 1160
        • Research Site
      • Wels, Austria, 4600
        • Research Site
  • Belgium
      • Bruges, Belgium, 8000
        • Research Site
      • Brussels, Belgium, 1070
        • Research Site
      • Genk, Belgium, 3600
        • Research Site
  • Brazil
      • Aracaju, Brazil, 49015-380
        • Research Site
      • Campinas, Brazil, 13083
        • Research Site
      • Fortaleza, Brazil, 60430-375
        • Research Site
      • Porto Alegre, Brazil, 90560-030
        • Research Site
      • Ribeirão Preto, Brazil, 14051-140
        • Research Site
      • Ribeirão Preto, Brazil, 14026-020
        • Research Site
      • Rio de Janeiro, Brazil, 20551-030
        • Research Site
      • Rio de Janeiro, Brazil, 22270-005
        • Research Site
      • São Paulo, Brazil, 05403-000
        • Research Site
      • São Paulo, Brazil, 04012-909
        • Research Site
      • São Paulo, Brazil, 01323-001
        • Research Site
      • São Paulo, Brazil, 05652-900
        • Research Site
  • Canada
    • Alberta
      • Calgary, Alberta, Canada, T2N 1N4
        • Research Site
    • Manitoba
      • Winnipeg, Manitoba, Canada, R2H 2A6
        • Research Site
    • Ontario
      • London, Ontario, Canada, N6C 2R5
        • Research Site
      • Toronto, Ontario, Canada, M5G 2C4
        • Research Site
    • Quebec
      • Chicoutimi, Quebec, Canada, G7H 5H6
        • Research Site
      • Montreal, Quebec, Canada, H1T 1C8
        • Research Site
      • Montreal, Quebec, Canada, H2X 1R9
        • Research Site
      • Rimouski, Quebec, Canada, G5L 5T1
        • Research Site
  • China
      • Beijing, China, 100730
        • Research Site
      • Beijing, China, 100034
        • Research Site
      • Beijing, China, 100037
        • Research Site
      • Beijing, China, 100191
        • Research Site
      • Changsha, China, 430033
        • Research Site
      • Chengdu, China, 610000
        • Research Site
      • Chengdu, China, 610072
        • Research Site
      • Chongqing, China, 400042
        • Research Site
      • Fuzhou, China, 350005
        • Research Site
      • Guangzhou, China, 510080
        • Research Site
      • Guiyang, China, 550044
        • Research Site
      • Hangzhou, China, 310003
        • Research Site
      • Hangzhou, China, 310009
        • Research Site
      • Harbin, China, 150080
        • Research Site
      • Hefei, China, 230001
        • Research Site
      • Jinan, China, 250021
        • Research Site
      • Nanjing, China, 210029
        • Research Site
      • Shanghai, China, 201114
        • Research Site
      • Tianjin, China, 300457
        • Research Site
      • Wuhan, China, 430030
        • Research Site
      • Xi'an, China, 710061
        • Research Site
  • Czechia
      • Prague, Czechia, 128 08
        • Research Site
      • Prague, Czechia, 140 00
        • Research Site
  • Denmark
      • Aarhus, Denmark, 8200
        • Research Site
      • Odense C, Denmark, 5000
        • Research Site
  • France
      • Bron, France, 69677
        • Research Site
      • Chambray-lès-Tours, France, 37170
        • Research Site
      • Créteil, France, 94010
        • Research Site
      • Marseille, France, 13005
        • Research Site
      • Montpellier, France, 34295
        • Research Site
      • Paris, France, 75018
        • Research Site
      • Paris, France, 75651
        • Research Site
      • Pessac, France, 33604
        • Research Site
      • Poitiers, France, 86021
        • Research Site
      • Rennes, France, 35033
        • Research Site
      • Saint-Herblain, France, 44093
        • Research Site
      • Toulouse, France, 31059
        • Research Site
  • Germany
      • Berlin, Germany, 10117
        • Research Site
      • Cologne, Germany, 50937
        • Research Site
      • Essen, Germany, 45147
        • Research Site
      • Giessen, Germany, 35392
        • Research Site
      • Hamburg, Germany, 22767
        • Research Site
      • Hanover, Germany, 30625
        • Research Site
      • Heidelberg, Germany, 69120
        • Research Site
      • Homburg, Germany, 66421
        • Research Site
      • Kiel, Germany, 24105
        • Research Site
      • Leipzig, Germany, 04103
        • Research Site
      • München, Germany, 81377
        • Research Site
      • Münster, Germany, 48149
        • Research Site
      • Würzburg, Germany, 97078
        • Research Site
  • Greece
      • Athens, Greece, 11528
        • Research Site
  • Ireland
      • Dublin, Ireland, 8
        • Research Site
  • Israel
      • Beer Yaakov, Israel, 70300
        • Research Site
      • Haifa, Israel, 34362
        • Research Site
      • Haifa, Israel, 31096
        • Research Site
      • Jerusalem, Israel, 91120
        • Research Site
      • Jerusalem, Israel, 9372212
        • Research Site
      • Petah Tikva, Israel, 4941492
        • Research Site
      • Rehovot, Israel, 76100
        • Research Site
  • Italy
      • Bologna, Italy, 40138
        • Research Site
      • Ferrara, Italy, 44124
        • Research Site
      • Florence, Italy, 50134
        • Research Site
      • Forlì, Italy, 47121
        • Research Site
      • Messina, Italy, 98124
        • Research Site
      • Milan, Italy, 20122
        • Research Site
      • Napoli, Italy, 80131
        • Research Site
      • Padua, Italy, 35128
        • Research Site
      • Pavia, Italy, 27100
        • Research Site
      • Pisa, Italy, 56124
        • Research Site
      • Roma, Italy, 00189
        • Research Site
      • Torino, Italy, 10154
        • Research Site
      • Trieste, Italy, 34128
        • Research Site
  • Japan
      • Bunkyō City, Japan, 113-8431
        • Research Site
      • Kumamoto, Japan, 860-8556
        • Research Site
      • Kurume-shi, Japan, 830-0011
        • Research Site
      • Kyoto, Japan, 602-8566
        • Research Site
      • Matsumoto-shi, Japan, 390-8621
        • Research Site
      • Sapporo, Japan, 060-8543
        • Research Site
      • Shinjuku-ku, Japan, 160-8582
        • Research Site
      • Suita-shi, Japan, 564-8565
        • Research Site
  • Netherlands
      • Groningen, Netherlands, 9713 GZ
        • Research Site
      • Maastricht, Netherlands, 6229 HX
        • Research Site
      • Rotterdam, Netherlands, 3015 GD
        • Research Site
      • Utrecht, Netherlands, 3584 CX
        • Research Site
  • Norway
      • Lørenskog, Norway, N-1478
        • Research Site
      • Oslo, Norway, 0372
        • Research Site
      • Trondheim, Norway, 7030
        • Research Site
  • Poland
      • Gdansk, Poland, 80-952
        • Research Site
      • Poznan, Poland, 61-848
        • Research Site
      • Warsaw, Poland, 02-097
        • Research Site
  • South Korea
      • Busan, South Korea, 49241
        • Research Site
      • Seoul, South Korea, 03080
        • Research Site
      • Seoul, South Korea, 03722
        • Research Site
      • Seoul, South Korea, 06351
        • Research Site
      • Seoul, South Korea, 06591
        • Research Site
      • Seoul, South Korea, 5505
        • Research Site
      • Wŏnju, South Korea, 26426
        • Research Site
  • Spain
      • A Coruña, Spain, 15009
        • Research Site
      • Barcelona, Spain, 8035
        • Research Site
      • El Palmar, Spain, 30120
        • Research Site
      • Jaén, Spain, 23007
        • Research Site
      • L'Hospitalet de Llobregat, Spain, 08907
        • Research Site
      • Madrid, Spain, 28027
        • Research Site
      • Majadahonda, Spain, 28222
        • Research Site
      • Málaga, Spain, 29010
        • Research Site
      • Palma de Mallorca, Spain, 07198
        • Research Site
      • Pamplona, Spain, 31008
        • Research Site
      • Salamanca, Spain, 37007
        • Research Site
      • Santiago de Compostela-Coruña, Spain, 15706
        • Research Site
      • Valencia, Spain, 46010
        • Research Site
  • Sweden
      • Gothenburg, Sweden, 413 45
        • Research Site
      • Lund, Sweden, 22242
        • Research Site
      • Skellefteå, Sweden, 93186
        • Research Site
      • Stockholm, Sweden, 171 64
        • Research Site
  • Switzerland
      • Basel, Switzerland, 4031
        • Research Site
      • Bern, Switzerland, 3010
        • Research Site
      • Lucerne, Switzerland, 6000
        • Research Site
      • Sankt Gallen, Switzerland, 9007
        • Research Site
      • Zurich, Switzerland, 8091
        • Research Site
  • Taiwan
      • New Taipei City, Taiwan, 220
        • Research Site
      • Taichung, Taiwan, 40705
        • Research Site
      • Taichung, Taiwan, 404
        • Research Site
      • Taipei, Taiwan, 10002
        • Research Site
      • Taipei, Taiwan, 11217
        • Research Site
  • Turkey (Türkiye)
      • Ankara, Turkey (Türkiye), 06230
        • Research Site
      • Dinar, Turkey (Türkiye), 03400
        • Research Site
      • Eskişehir, Turkey (Türkiye), 26480
        • Research Site
      • Istanbul, Turkey (Türkiye), 34303
        • Research Site
      • Mersin, Turkey (Türkiye), 33079
        • Research Site
  • United Kingdom
      • Birmingham, United Kingdom, B15 2SQ
        • Research Site
      • Cardiff, United Kingdom, CF15 9SS
        • Research Site
      • Glasgow, United Kingdom, G20 0SP
        • Research Site
      • Hexham, United Kingdom, NE46 1QJ
        • Research Site
      • London, United Kingdom, NW3 2QG
        • Research Site
      • London, United Kingdom, SE1 1YR
        • Research Site
      • Manchester, United Kingdom, M15 6SE
        • Research Site
  • United States
    • California
      • Irvine, California, United States, 92614
        • Research Site
      • La Jolla, California, United States, 92037
        • Research Site
      • Palo Alto, California, United States, 94304
        • Research Site
      • San Francisco, California, United States, 94143
        • Research Site
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Research Site
    • District of Columbia
      • Washington D.C., District of Columbia, United States, 20010
        • Research Site
    • Florida
      • Jacksonville, Florida, United States, 32224
        • Research Site
      • Weston, Florida, United States, 33331
        • Research Site
    • Georgia
      • Atlanta, Georgia, United States, 30309
        • Research Site
      • Atlanta, Georgia, United States, 30322
        • Research Site
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Research Site
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Research Site
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Research Site
      • Boston, Massachusetts, United States, 02118
        • Research Site
    • Missouri
      • Kansas City, Missouri, United States, 64111
        • Research Site
      • St Louis, Missouri, United States, 63110
        • Research Site
    • New York
      • New York, New York, United States, 10016
        • Research Site
      • New York, New York, United States, 10032
        • Research Site
      • Stony Brook, New York, United States, 11794
        • Research Site
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27599
        • Research Site
      • Durham, North Carolina, United States, 27705
        • Research Site
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Research Site
      • Columbus, Ohio, United States, 43210
        • Research Site
    • Oregon
      • Portland, Oregon, United States, 97239
        • Research Site
    • Pennsylvania
      • Danville, Pennsylvania, United States, 17822
        • Research Site
      • Philadelphia, Pennsylvania, United States, 19104
        • Research Site
    • South Carolina
      • Charleston, South Carolina, United States, 29425
        • Research Site
      • Greenville, South Carolina, United States, 29605
        • Research Site
    • Tennessee
      • Memphis, Tennessee, United States, 38120
        • Research Site
    • Texas
      • Fort Worth, Texas, United States, 76104
        • Research Site
      • Houston, Texas, United States, 77030
        • Research Site
    • Utah
      • Salt Lake City, Utah, United States, 84132
        • Research Site
    • Virginia
      • Falls Church, Virginia, United States, 22042
        • Research Site
      • Richmond, Virginia, United States, 23298
        • Research Site
    • Washington
      • Seattle, Washington, United States, 98195
        • Research Site
    • Wisconsin
      • Madison, Wisconsin, United States, 53705
        • Research Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 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.
Drug: ALXN2220
Participants will receive ALXN2220 via IV infusion.
Placebo Comparator: Placebo
Participants will receive placebo via IV infusion q4w for at least 24 months up to a maximum of 48 months.
Drug: Placebo
Participants will receive placebo via IV infusion.

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure
Time Frame
Change From Baseline in Kansas City Cardiomyopathy Questionnaire-Overall Score (KCCQ-OS) at Month 24
Time Frame: Baseline, Month 24
Baseline, Month 24
Time to CV-related Mortality
Time Frame: Baseline up to the end of study (up to Month 48)
Baseline up to the end of study (up to Month 48)
Change from Baseline in Six-minute Walk Test (6MWT) at Month 24
Time Frame: Baseline, Month 24
Baseline, Month 24
Time to ACM
Time Frame: Baseline up to the end of study (up to Month 48)
Baseline up to the end of study (up to Month 48)
Total Occurrences of CV Clinical Events
Time Frame: Baseline up to the end of study (up to Month 48)
Baseline up to the end of study (up to Month 48)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alexion Pharmaceuticals, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 11, 2024

Primary Completion (Estimated)

June 7, 2027

Study Completion (Estimated)

October 5, 2027

Study Registration Dates

First Submitted

November 15, 2023

First Submitted That Met QC Criteria

December 14, 2023

First Posted (Actual)

December 28, 2023

Study Record Updates

Last Update Posted (Actual)

February 23, 2026

Last Update Submitted That Met QC Criteria

February 20, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • D6810C00001
  • ALXN2220-ATTRCM-301 (Other Identifier: Alexion)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • CSR

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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