Study of ALXN2220 Versus Placebo in Adults With ATTR-CM
DepleTTR-CM
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)
2 other identifiers
interventional
1,181
26 countries
207
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_3
Started Jan 2024
Typical duration for phase_3
207 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
November 15, 2023
CompletedFirst Posted
Study publicly available on registry
December 28, 2023
CompletedStudy Start
First participant enrolled
January 11, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 7, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
October 5, 2027
February 23, 2026
February 1, 2026
3.4 years
November 15, 2023
February 20, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Total Occurrence of ACM and CV clinical events during the Blinded Treatment Period
Baseline up to the end study (up to Month 48)
Secondary Outcomes (5)
Change From Baseline in Kansas City Cardiomyopathy Questionnaire-Overall Score (KCCQ-OS) at Month 24
Baseline, Month 24
Time to CV-related Mortality
Baseline up to the end of study (up to Month 48)
Change from Baseline in Six-minute Walk Test (6MWT) at Month 24
Baseline, Month 24
Total Occurrences of CV Clinical Events
Baseline up to the end of study (up to Month 48)
Time to ACM
Baseline up to the end of study (up to Month 48)
Study Arms (2)
ALXN2220
EXPERIMENTALParticipants 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.
Placebo
PLACEBO COMPARATORParticipants will receive placebo via IV infusion q4w for at least 24 months up to a maximum of 48 months.
Interventions
Eligibility Criteria
You may qualify if:
- 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 may not qualify 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (212)
Research Site
Irvine, California, 92614, United States
Research Site
La Jolla, California, 92037, United States
Research Site
Palo Alto, California, 94304, United States
Research Site
San Francisco, California, 94143, United States
Research Site
Aurora, Colorado, 80045, United States
Research Site
Washington D.C., District of Columbia, 20010, United States
Research Site
Jacksonville, Florida, 32224, United States
Research Site
Weston, Florida, 33331, United States
Research Site
Atlanta, Georgia, 30309, United States
Research Site
Atlanta, Georgia, 30322, United States
Research Site
Chicago, Illinois, 60611, United States
Research Site
Indianapolis, Indiana, 46202, United States
Research Site
Boston, Massachusetts, 02115, United States
Research Site
Boston, Massachusetts, 02118, United States
Research Site
Kansas City, Missouri, 64111, United States
Research Site
St Louis, Missouri, 63110, United States
Research Site
New York, New York, 10016, United States
Research Site
New York, New York, 10032, United States
Research Site
Stony Brook, New York, 11794, United States
Research Site
Chapel Hill, North Carolina, 27599, United States
Research Site
Durham, North Carolina, 27705, United States
Research Site
Cleveland, Ohio, 44195, United States
Research Site
Columbus, Ohio, 43210, United States
Research Site
Portland, Oregon, 97239, United States
Research Site
Danville, Pennsylvania, 17822, United States
Research Site
Philadelphia, Pennsylvania, 19104, United States
Research Site
Charleston, South Carolina, 29425, United States
Research Site
Greenville, South Carolina, 29605, United States
Research Site
Memphis, Tennessee, 38120, United States
Research Site
Fort Worth, Texas, 76104, United States
Research Site
Houston, Texas, 77030, United States
Research Site
Salt Lake City, Utah, 84132, United States
Research Site
Falls Church, Virginia, 22042, United States
Research Site
Richmond, Virginia, 23298, United States
Research Site
Seattle, Washington, 98195, United States
Research Site
Madison, Wisconsin, 53705, United States
Research Site
Buenos Aires, 1425, Argentina
Research Site
CABA, C1025ABI, Argentina
Research Site
Ciudad de Buenos Aires, C1181ACH, Argentina
Research Site
Ciudad de Buenos Aires, C1428, Argentina
Research Site
Córdoba, X5016KEH, Argentina
Research Site
Bedford Park, 5042, Australia
Research Site
Box Hill, 3128, Australia
Research Site
Brisbane, 4102, Australia
Research Site
Darlinghurst, 2010, Australia
Research Site
Joondalup, 6027, Australia
Research Site
Melbourne, 3004, Australia
Research Site
Westmead, 2145, Australia
Research Site
Graz, 8036, Austria
Research Site
Innsbruck, 6020, Austria
Research Site
Vienna, 1090, Austria
Research Site
Vienna, 1160, Austria
Research Site
Wels, 4600, Austria
Research Site
Bruges, 8000, Belgium
Research Site
Brussels, 1070, Belgium
Research Site
Genk, 3600, Belgium
Research Site
Aracaju, 49015-380, Brazil
Research Site
Campinas, 13083, Brazil
Research Site
Fortaleza, 60430-375, Brazil
Research Site
Porto Alegre, 90560-030, Brazil
Research Site
Ribeirão Preto, 14026-020, Brazil
Research Site
Ribeirão Preto, 14051-140, Brazil
Research Site
Rio de Janeiro, 20551-030, Brazil
Research Site
Rio de Janeiro, 22270-005, Brazil
Research Site
São Paulo, 01323-001, Brazil
Research Site
São Paulo, 04012-909, Brazil
Research Site
São Paulo, 05403-000, Brazil
Research Site
São Paulo, 05652-900, Brazil
Research Site
Calgary, Alberta, T2N 1N4, Canada
Research Site
Winnipeg, Manitoba, R2H 2A6, Canada
Research Site
London, Ontario, N6C 2R5, Canada
Research Site
Toronto, Ontario, M5G 2C4, Canada
Research Site
Chicoutimi, Quebec, G7H 5H6, Canada
Research Site
Montreal, Quebec, H1T 1C8, Canada
Research Site
Montreal, Quebec, H2X 1R9, Canada
Research Site
Rimouski, Quebec, G5L 5T1, Canada
Research Site
Beijing, 100034, China
Research Site
Beijing, 100037, China
Research Site
Beijing, 100191, China
Research Site
Beijing, 100730, China
Research Site
Changsha, 430033, China
Research Site
Chengdu, 610000, China
Research Site
Chengdu, 610072, China
Research Site
Chongqing, 400042, China
Research Site
Fuzhou, 350005, China
Research Site
Guangzhou, 510080, China
Research Site
Guiyang, 550044, China
Research Site
Hangzhou, 310003, China
Research Site
Hangzhou, 310009, China
Research Site
Harbin, 150080, China
Research Site
Hefei, 230001, China
Research Site
Jinan, 250021, China
Research Site
Nanjing, 210029, China
Research Site
Shanghai, 201114, China
Research Site
Tianjin, 300457, China
Research Site
Wuhan, 430030, China
Research Site
Xi'an, 710061, China
Research Site
Prague, 128 08, Czechia
Research Site
Prague, 140 00, Czechia
Research Site
Aarhus, 8200, Denmark
Research Site
Odense C, 5000, Denmark
Research Site
Bron, 69677, France
Research Site
Chambray-lès-Tours, 37170, France
Research Site
Créteil, 94010, France
Research Site
Marseille, 13005, France
Research Site
Montpellier, 34295, France
Research Site
Paris, 75018, France
Research Site
Paris, 75651, France
Research Site
Pessac, 33604, France
Research Site
Poitiers, 86021, France
Research Site
Rennes, 35033, France
Research Site
Saint-Herblain, 44093, France
Research Site
Toulouse, 31059, France
Research Site
Berlin, 10117, Germany
Research Site
Cologne, 50937, Germany
Research Site
Essen, 45147, Germany
Research Site
Giessen, 35392, Germany
Research Site
Hamburg, 22767, Germany
Research Site
Hanover, 30625, Germany
Research Site
Heidelberg, 69120, Germany
Research Site
Homburg, 66421, Germany
Research Site
Kiel, 24105, Germany
Research Site
Leipzig, 04103, Germany
Research Site
München, 81377, Germany
Research Site
Münster, 48149, Germany
Research Site
Würzburg, 97078, Germany
Research Site
Athens, 11528, Greece
Research Site
Dublin, 8, Ireland
Research Site
Beer Yaakov, 70300, Israel
Research Site
Haifa, 31096, Israel
Research Site
Haifa, 34362, Israel
Research Site
Jerusalem, 91120, Israel
Research Site
Jerusalem, 9372212, Israel
Research Site
Petah Tikva, 4941492, Israel
Research Site
Rehovot, 76100, Israel
Research Site
Bologna, 40138, Italy
Research Site
Ferrara, 44124, Italy
Research Site
Florence, 50134, Italy
Research Site
Forlì, 47121, Italy
Research Site
Messina, 98124, Italy
Research Site
Milan, 20122, Italy
Research Site
Naples, 80131, Italy
Research Site
Padua, 35128, Italy
Research Site
Pavia, 27100, Italy
Research Site
Pisa, 56124, Italy
Research Site
Roma, 00189, Italy
Research Site
Torino, 10154, Italy
Research Site
Trieste, 34128, Italy
Research Site
Bunkyō City, 113-8431, Japan
Research Site
Kumamoto, 860-8556, Japan
Research Site
Kurume-shi, 830-0011, Japan
Research Site
Kyoto, 602-8566, Japan
Research Site
Matsumoto-shi, 390-8621, Japan
Research Site
Sapporo, 060-8543, Japan
Research Site
Shinjuku-ku, 160-8582, Japan
Research Site
Suita-shi, 564-8565, Japan
Research Site
Groningen, 9713 GZ, Netherlands
Research Site
Maastricht, 6229 HX, Netherlands
Research Site
Rotterdam, 3015 GD, Netherlands
Research Site
Utrecht, 3584 CX, Netherlands
Research Site
Lørenskog, N-1478, Norway
Research Site
Oslo, 0372, Norway
Research Site
Trondheim, 7030, Norway
Research Site
Gdansk, 80-952, Poland
Research Site
Poznan, 61-848, Poland
Research Site
Warsaw, 02-097, Poland
Research Site
Busan, 49241, South Korea
Research Site
Seoul, 03080, South Korea
Research Site
Seoul, 03722, South Korea
Research Site
Seoul, 06351, South Korea
Research Site
Seoul, 06591, South Korea
Research Site
Seoul, 5505, South Korea
Research Site
Wŏnju, 26426, South Korea
Research Site
A Coruña, 15009, Spain
Research Site
Barcelona, 8035, Spain
Research Site
El Palmar, 30120, Spain
Research Site
Jaén, 23007, Spain
Research Site
L'Hospitalet de Llobregat, 08907, Spain
Research Site
Madrid, 28027, Spain
Research Site
Majadahonda, 28222, Spain
Research Site
Málaga, 29010, Spain
Research Site
Palma de Mallorca, 07198, Spain
Research Site
Pamplona, 31008, Spain
Research Site
Salamanca, 37007, Spain
Research Site
Santiago de Compostela-Coruña, 15706, Spain
Research Site
Valencia, 46010, Spain
Research Site
Gothenburg, 413 45, Sweden
Research Site
Lund, 22242, Sweden
Research Site
Skellefteå, 93186, Sweden
Research Site
Stockholm, 171 64, Sweden
Research Site
Basel, 4031, Switzerland
Research Site
Bern, 3010, Switzerland
Research Site
Lucerne, 6000, Switzerland
Research Site
Sankt Gallen, 9007, Switzerland
Research Site
Zurich, 8091, Switzerland
Research Site
New Taipei City, 220, Taiwan
Research Site
Taichung, 404, Taiwan
Research Site
Taichung, 40705, Taiwan
Research Site
Taipei, 10002, Taiwan
Research Site
Taipei, 11217, Taiwan
Research Site
Ankara, 06230, Turkey (Türkiye)
Research Site
Dinar, 03400, Turkey (Türkiye)
Research Site
Eskişehir, 26480, Turkey (Türkiye)
Research Site
Istanbul, 34303, Turkey (Türkiye)
Research Site
Mersin, 33079, Turkey (Türkiye)
Research Site
Birmingham, B15 2SQ, United Kingdom
Research Site
Cardiff, CF15 9SS, United Kingdom
Research Site
Glasgow, G20 0SP, United Kingdom
Research Site
Hexham, NE46 1QJ, United Kingdom
Research Site
London, NW3 2QG, United Kingdom
Research Site
London, SE1 1YR, United Kingdom
Research Site
Manchester, M15 6SE, United Kingdom
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 15, 2023
First Posted
December 28, 2023
Study Start
January 11, 2024
Primary Completion (Estimated)
June 7, 2027
Study Completion (Estimated)
October 5, 2027
Last Updated
February 23, 2026
Record last verified: 2026-02
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL, SAP, CSR
Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.