Transthyretin Amyloid CardiomyopathyPhase 3DepleTTR-CMNCT06183931
Study of ALXN2220 Versus Placebo in Adults With ATTR-CM
A Phase 3 study testing ALXN2220 in people with Transthyretin Amyloid Cardiomyopathy, run by Alexion Pharmaceuticals, Inc. at 213 sites in 27 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 90 years
- Conditions the study is for
- Transthyretin Amyloid Cardiomyopathy
- What is being tested?
- ALXN2220
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 1,182 participants
- Where
- 213 sitesin 27 countries
- Study dates
- 11 Jan 2024to 5 Apr 2028
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 90 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
213 sites in 27 countries.
213 sites in 27 countries
United States36 sites
Research Site
Atlanta, Georgia, 30309
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Atlanta, Georgia, 30322
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Aurora, Colorado, 80045
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Boston, Massachusetts, 02115
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Boston, Massachusetts, 02118
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Chapel Hill, North Carolina, 27599
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Charleston, South Carolina, 29425
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Chicago, Illinois, 60611
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Cleveland, Ohio, 44195
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Columbus, Ohio, 43210
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Danville, Pennsylvania, 17822
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Durham, North Carolina, 27705
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Falls Church, Virginia, 22042
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Fort Worth, Texas, 76104
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Greenville, South Carolina, 29605
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Houston, Texas, 77030
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Indianapolis, Indiana, 46202
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Irvine, California, 92614
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Jacksonville, Florida, 32224
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Kansas City, Missouri, 64111
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La Jolla, California, 92037
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Madison, Wisconsin, 53705
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Memphis, Tennessee, 38120
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New York, New York, 10016
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New York, New York, 10032
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Palo Alto, California, 94304
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Philadelphia, Pennsylvania, 19104
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Portland, Oregon, 97239
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Richmond, Virginia, 23298
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Salt Lake City, Utah, 84132
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San Francisco, California, 94143
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Seattle, Washington, 98195
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St Louis, Missouri, 63110
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Stony Brook, New York, 11794
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Washington D.C., District of Columbia, 20010
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Weston, Florida, 33331
China21 sites
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Beijing, 100034
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Beijing, 100037
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Beijing, 100191
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Beijing, 100730
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Changsha, 430033
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Chengdu, 610000
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Chengdu, 610072
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Chongqing, 400042
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Fuzhou, 350005
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Guangzhou, 510080
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Guiyang, 550044
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Hangzhou, 310003
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Hangzhou, 310009
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Harbin, 150080
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Hefei, 230001
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Jinan, 250021
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Nanjing, 210029
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Shanghai, 201114
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Tianjin, 300457
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Wuhan, 430030
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Xi'an, 710061
Germany13 sites
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Berlin, 10117
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Cologne, 50937
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Essen, 45147
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Giessen, 35392
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Hamburg, 22767
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Hanover, 30625
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Heidelberg, 69120
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Homburg, 66421
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Kiel, 24105
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Leipzig, 04103
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München, 81377
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Münster, 48149
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Würzburg, 97078
Italy13 sites
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Bologna, 40138
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Ferrara, 44124
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Florence, 50134
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Forlì, 47121
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Messina, 98124
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Milan, 20122
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Naples, 80131
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Padova, 35128
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Pavia, 27100
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Pisa, 56124
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Roma, 00189
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Torino, 10154
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Trieste, 34128
Spain13 sites
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A Coruña, 15009
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Barcelona, 8035
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El Palmar, 30120
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Jaén, 23007
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L'Hospitalet de Llobregat, 08907
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Madrid, 28027
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Majadahonda, 28222
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Málaga, 29010
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Palma de Mallorca, 07198
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Pamplona, 31008
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Salamanca, 37007
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Santiago de Compostela-Coruña, 15706
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Valencia, 46010
Brazil12 sites
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Aracaju, 49015-380
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Campinas, 13083
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Fortaleza, 60430-375
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Porto Alegre, 90560-030
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Ribeirão Preto, 14026-020
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Ribeirão Preto, 14051-140
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Rio de Janeiro, 20551-030
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Rio de Janeiro, 22270-005
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São Paulo, 01323-001
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São Paulo, 04012-909
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São Paulo, 05403-000
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São Paulo, 05652-900
France12 sites
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Bron, 69677
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Chambray-lès-Tours, 37170
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Créteil, 94010
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Marseille, 13005
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Montpellier, 34295
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Paris, 75018
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Paris, 75651
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Pessac, 33604
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Poitiers, 86021
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Rennes, 35033
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Saint-Herblain, 44093
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Toulouse, 31059
Canada8 sites
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Calgary, Alberta, T2N 1N4
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Chicoutimi, Quebec, G7H 5H6
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London, Ontario, N6A 5A5
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Montreal, Quebec, H1T 1C8
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Montreal, Quebec, H2X 1R9
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Rimouski, Quebec, G5L 5T1
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Toronto, Ontario, M5G 2C4
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Winnipeg, Manitoba, R2H 2A6
Japan8 sites
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Bunkyō City, 113-8431
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Kumamoto, 860-8556
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Kurume-shi, 830-0011
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Kyoto, 602-8566
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Matsumoto-shi, 390-8621
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Sapporo, 060-8543
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Shinjuku-ku, 160-8582
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Suita-shi, 564-8565
Australia7 sites
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Bedford Park, 5042
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Box Hill, 3128
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Brisbane, 4102
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Darlinghurst, 2010
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Joondalup, 6027
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Melbourne, 3004
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Westmead, 2145
Israel7 sites
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Beer Yaakov, 70300
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Haifa, 31096
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Haifa, 34362
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Jerusalem, 91120
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Jerusalem, 9372212
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Petah Tikva, 4941492
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Rehovot, 76100
South Korea7 sites
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Busan, 49241
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Seoul, 03080
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Seoul, 03722
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Seoul, 06351
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Seoul, 06591
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Seoul, 5505
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Wŏnju, 26426
United Kingdom7 sites
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Birmingham, B15 2SQ
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Cardiff, CF15 9SS
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Glasgow, G20 0SP
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Hexham, NE46 1QJ
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London, NW3 2QG
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London, SE1 1YR
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Manchester, M15 6SE
Argentina5 sites
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Buenos Aires, 1425
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CABA, C1025ABI
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Ciudad de Buenos Aires, C1181ACH
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Ciudad de Buenos Aires, C1428
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Córdoba, X5016KEH
Austria5 sites
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Graz, 8036
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Innsbruck, 6020
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Vienna, 1090
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Vienna, 1160
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Wels, 4600
Switzerland5 sites
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Basel, 4031
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Bern, 3010
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Lucerne, 6000
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Sankt Gallen, 9007
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Zurich, 8091
Taiwan5 sites
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New Taipei City, 220
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Taichung, 404
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Taichung, 40705
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Taipei, 10002
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Taipei, 11217
Turkey (Türkiye)5 sites
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Ankara, 06230
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Dinar, 03400
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Eskişehir, 26480
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Istanbul, 34303
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Mersin, 33079
Netherlands4 sites
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Groningen, 9713 GZ
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Maastricht, 6229 HX
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Rotterdam, 3015 GD
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Utrecht, 3584 CX
Sweden4 sites
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Gothenburg, 413 45
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Lund, 22242
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Skellefteå, 93186
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Stockholm, 171 64
Belgium3 sites
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Bruges, 8000
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Brussels, 1070
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Genk, 3600
Denmark3 sites
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Aarhus, 8200
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Copenhagen, DK-2100
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Odense C, 5000
Norway3 sites
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Lørenskog, N-1478
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Oslo, 0372
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Trondheim, 7030
Poland3 sites
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Gdansk, 80-952
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Poznan, 61-848
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Warsaw, 02-097
About this study
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)
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.
- Conditions
- Transthyretin Amyloid Cardiomyopathy
- Being tested
- ALXN2220, Placebo
- Keywords
- Amyloid Depleter, ALXN2220, All-cause mortality (ACM), Cardiovascular (CV), Transthyretin Amyloid Cardiomyopathy, Transthyretin amyloid (ATTR), Transthyretin (TTR), N-terminal pro b-type natriuretic peptide (NT-proBNP), NT-proBNP, Amyloidosis, ATTR-CM
- Medical subject headings
- Amyloidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALXN2220Drug
Participants will receive ALXN2220 via IV infusion.
PlaceboDrug
Participants will receive placebo via IV infusion.
| Study group | Role | What they receive |
|---|---|---|
| ALXN2220 | Experimental | Participants will receive weight-based dose of ALXN2220 via intravenous (IV) infusion every 4 weeks (q4w) for at least 30 months up to a maximum of 48 months. Receives: Drug: ALXN2220 |
| Placebo | Placebo comparator | Participants will receive placebo via IV infusion q4w for at least 30 months up to a maximum of 48 months. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Total Occurrence of ACM and CV clinical events during the Blinded Treatment Period
Measured: Baseline up to the end of study (up to Month 48)
Secondary outcomes (5)
Change From Baseline in Kansas City Cardiomyopathy Questionnaire-Overall Score (KCCQ-OS) at Month 30
Measured: Baseline, Month 30
Time to CV-related Mortality
Measured: Baseline up to the end of study (up to Month 48)
Change from Baseline in Six-minute Walk Test (6MWT) at Month 30
Measured: Baseline, Month 30
Total Occurrences of CV Clinical Events
Measured: Baseline up to the end of study (up to Month 48)
Time to ACM
Measured: Baseline up to the end of study (up to Month 48)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Dec 2023
- Study start11 Jan 2024
- Primary completion6 Dec 2027 (estimated)
- Study completion5 Apr 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Transthyretin Amyloid CardiomyopathyStudies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 1,182 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06183931
- Sponsor's protocol ID
- D6810C00001
- Other IDs
- ALXN2220-ATTRCM-301
- Acronym
- DepleTTR-CM
- Status last verified
- Jul 2026
- First submitted
- 15 Nov 2023
- Last updated
- 22 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.
Publications
Beltran J, Bolton S, Patel P, Sharma R. Patient engagement in protocol development for rare disease clinical trials: a framework. Res Involv Engagem. 2026 Oct 9;12(1):167. doi: 10.1186/s40900-026-00985-3.
PubMed 42855714