An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of ALXN2220 in Adults With Transthyretin Amyloid Cardiomyopathy
LTSAFE-ALXN
An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of ALXN2220 in Adult Participants With Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
4 other identifiers
interventional
1,180
26 countries
188
Brief Summary
The purpose of Study ALXN2220-ATTRCM-302 is to evaluate the long-term safety, tolerability and efficacy of ALXN2220 in adult participants with ATTR-CM treated with ALXN2220 in addition to SoC who have completed Study ALXN2220-ATTRCM-301.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_3
Started Nov 2027
Typical duration for phase_3
188 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
August 18, 2026
CompletedFirst Posted
Study publicly available on registry
August 27, 2026
CompletedStudy Start
First participant enrolled
November 23, 2027
ExpectedPrimary Completion
Last participant's last visit for primary outcome
July 11, 2030
Study Completion
Last participant's last visit for all outcomes
July 11, 2030
August 27, 2026
August 1, 2026
2.6 years
August 18, 2026
August 24, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Percentage of participants with TEAEs
To evaluate the long-term safety and tolerability of ALXN2220 in participants with ATTR-CM
Up to 24 months
Secondary Outcomes (9)
Total occurrence of ACM and CV clinical events
Up to 24 months
Total occurrence of CV clinical events
Up to 24 months
Time to ACM
Up to 24 months
Time to first CV clinical event or ACM
Up to 24 months
Time to CVM
Up to 24 months
- +4 more secondary outcomes
Study Arms (1)
ALXN2220
EXPERIMENTALEligible participants will receive ALXN2220 administered IV q4w for a minimum of 12 months and up to 24 months or until participant has access to commercially available ALXN2220 for ATTR-CM, whichever occurs first.
Interventions
Eligibility Criteria
You may qualify if:
- Type of Participant: Participants who have completed the Blinded Treatment Period (including the PC visit) of Study 301.
- Sex and Contraceptive/Barrier Requirements: Male and/or female (according to their reproductive organs and functions assigned by chromosomal complement).
- Informed Consent: Participant, or legally authorized representative where relevant and allowed by law, is willing and able to comply with all study requirements and to provide signed informed consent.
You may not qualify if:
- Participants who permanently discontinued study intervention in Study 301
- Participants who withdrew full consent during Study 301.
- Medical Conditions: Suspected or known intolerance/allergy to proteins or any components of ALXN2220. Has had a heart transplant or permanent CMAD implantation. Has any new or worsening of existing medical or psychological condition or risk factor that in the opinion of the Investigator or Sponsor might jeopardize participant's safety or compliance with the protocol, or pose any additional risk for the participant, or confound the assessment of the participant or outcome of the study.
- Prior/Concurrent/Future Clinical Study Experience: Current or future participation in another investigational clinical study or intake of investigational drug within 30 calendar days or 5 half-lives of the study intervention, whichever is longer, before signing the ICF, except for study intervention used in Study 301.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (195)
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Irvine, California, 92614, United States
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La Jolla, California, 92037, United States
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Palo Alto, California, 94304, United States
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San Francisco, California, 94143, United States
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Aurora, Colorado, 80045, United States
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Washington D.C., District of Columbia, 20010, United States
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Jacksonville, Florida, 32224, United States
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Weston, Florida, 33331, United States
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Atlanta, Georgia, 30309, United States
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Atlanta, Georgia, 30322, United States
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Indianapolis, Indiana, 46202, United States
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Boston, Massachusetts, 02115, United States
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Boston, Massachusetts, 02118, United States
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Kansas City, Missouri, 64111, United States
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St Louis, Missouri, 63110, United States
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New York, New York, 10016, United States
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New York, New York, 10032, United States
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Stony Brook, New York, 11794, United States
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Chapel Hill, North Carolina, 27599, United States
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Durham, North Carolina, 27705, United States
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Cleveland, Ohio, 44195, United States
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Columbus, Ohio, 43210, United States
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Portland, Oregon, 97239, United States
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Danville, Pennsylvania, 17822, United States
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Philadelphia, Pennsylvania, 19104, United States
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Charleston, South Carolina, 29425, United States
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Greenville, South Carolina, 29605, United States
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Memphis, Tennessee, 38120, United States
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Fort Worth, Texas, 76104, United States
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Houston, Texas, 77030, United States
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Salt Lake City, Utah, 84132, United States
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Falls Church, Virginia, 22042, United States
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Richmond, Virginia, 23298, United States
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Seattle, Washington, 98195, United States
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Madison, Wisconsin, 53705, United States
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Buenos Aires, Buenos Aires, 1425, Argentina
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CABA, Buenos Aires, C1025ABI, Argentina
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Ciudad de Buenos Aires, Buenos Aires F.D., C1181ACH, Argentina
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Córdoba, Córdoba Province, X5016KEH, Argentina
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Ciudad de Buenos Aires, C1428, Argentina
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Darlinghurst, New South Wales, 2010, Australia
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Westmead, New South Wales, 2145, Australia
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Brisbane, Queensland, 4102, Australia
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Bedford Park, South Australia, 5042, Australia
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Box Hill, Victoria, 3128, Australia
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Melbourne, Victoria, 3004, Australia
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Joondalup, Western Australia, 6027, Australia
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Graz, 8036, Austria
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Innsbruck, 6020, Austria
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Vienna, 1090, Austria
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Vienna, 1160, Austria
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Wels, 4600, Austria
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Bruges, West-Vlaanderen, 8000, Belgium
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Genk, 3600, Belgium
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Fortaleza, Ceará, 60430-375, Brazil
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Rio de Janeiro, Rio de Janeiro, 20551-030, Brazil
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Rio de Janeiro, Rio de Janeiro, 22270-005, Brazil
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Porto Alegre, Rio Grande do Sul, 90560-030, Brazil
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Campinas, São Paulo, 13083, Brazil
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Ribeirão Preto, São Paulo, 14026-020, Brazil
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Ribeirão Preto, São Paulo, 14051-140, Brazil
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São Paulo, São Paulo, 04012-909, Brazil
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São Paulo, São Paulo, 05403-000, Brazil
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São Paulo, São Paulo, 05652-900, Brazil
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Calgary, Alberta, T2N 1N4, Canada
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Winnipeg, Manitoba, R2H 2A6, Canada
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London, Ontario, N6A 5A5, Canada
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Chicoutimi, Quebec, G7H 5H6, Canada
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Montr Al, Quebec, H1T 1C8, Canada
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Montr Al, Quebec, H2X 1R9, Canada
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Rimouski, Quebec, G5L 5T1, Canada
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Beijing, Beijing Municipality, 100037, China
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Beijing, Beijing Municipality, 100191, China
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Chongqing, Chongqing Municipality, 400042, China
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Guangzhou, Guangdong, 510080, China
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Guiyang, Guizhou, 550044, China
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Harbin, Heilongjiang, 150080, China
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Wuhan, Hubei, 430030, China
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Changsha, Hunan, 430033, China
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Nanjing, Jiangsu, 210029, China
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Xi'an, Shaanxi, 710061, China
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Jinan, Shangdong, 250021, China
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Shanghai, Shanghai Municipality, 201114, China
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Hangzhou, Zhejiang, 310003, China
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Hangzhou, Zhejiang, 310009, China
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Beijing, 100730, China
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Tianjin, 300457, China
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Praha, Central Bohemia, 140 00, Czechia
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Prague, 128 08, Czechia
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Aarhus, 8200, Denmark
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Odense C, 5000, Denmark
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Bron, 69677, France
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Chambray-lès-Tours, 37170, France
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Créteil, 94010, France
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Marseille, 13005, France
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Montpellier, 34295, France
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Paris, 75018, France
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Paris, 75651, France
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Pessac, 33604, France
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Poitiers, 86021, France
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Rennes, 35033, France
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Saint-Herblain, 44093, France
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Toulouse, 31059, France
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Berlin, Deutschalnd, 10117, Germany
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Hanover, Lower Saxony, 30625, Germany
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Münster, Munster, 48149, Germany
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Cologne, North Rhine-Westphalia, 50937, Germany
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Homburg, Saarland, 66421, Germany
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Leipzig, Saxony, 04103, Germany
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Kiel, Schleswig-Holstein, 24105, Germany
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Essen, 45147, Germany
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Gie En, 35392, Germany
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Hamburg, 22767, Germany
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Heidelberg, 69120, Germany
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M Nchen, 81377, Germany
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W Rzburg, 97078, Germany
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Athens, Attica, 11528, Greece
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Dublin, 8, Ireland
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Beer Yaakov, 70300, Israel
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Haifa, 31096, Israel
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Jerusalem, 91120, Israel
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Rehovot, 76100, Israel
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Bologna, Bologna, 40138, Italy
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Forlì, Emilia-Romagna, 47121, Italy
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Florence, Florence, 50134, Italy
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Rome, Lazio, 00189, Italy
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Messina, Messina, 98124, Italy
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Naples, Napoli, 80131, Italy
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Padova, Padova, 35128, Italy
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Pisa, PI, 56124, Italy
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Torino, TO, 10154, Italy
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Trieste, TS, 34128, Italy
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Ferrara, 44124, Italy
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Milan, 20122, Italy
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Pavia, 27100, Italy
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Kurume-shi, Fukuoka, 830-0011, Japan
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Sapporo, Hokkaido, 060-8543, Japan
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Kumamoto, Kumamoto, 860-8556, Japan
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Kyoto, Kyoto, 602-8566, Japan
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Matsumoto-shi, Nagano, 390-8621, Japan
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Suita-shi, Osaka, 564-8565, Japan
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Bunkyo-ku, Tokyo, 113-8431, Japan
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Shinjuku-ku, Tokyo, 160-8582, Japan
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Groningen, 9713 GZ, Netherlands
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Maastricht, 6229 HX, Netherlands
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Rotterdam, 3015 GD, Netherlands
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Utrecht, 3584 CX, Netherlands
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Trondheim, Trøndelag, 7030, Norway
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Lorenskog, N-1478, Norway
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Oslo, 0372, Norway
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Warsaw, Masovian Voivodeship, 02-097, Poland
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Gdansk, Pomeranian Voivodeship, 80-952, Poland
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Poznan, 61-848, Poland
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Seoul, Seoul, 03080, South Korea
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Seoul, Seoul, 03722, South Korea
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Seoul, Seoul, 06351, South Korea
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Seoul, Seoul, 06591, South Korea
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Seoul, Seoul, 5505, South Korea
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Busan, 49241, South Korea
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Jaén, Andalusia, 23007, Spain
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Málaga, Andalusia, 29010, Spain
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Palma de Mallorca, Balearic Islands, 07198, Spain
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Salamanca, Castille and León, 37007, Spain
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Barcelona, Catalonia, 8035, Spain
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L'Hospitalet de Llobregat, Catalonia, 08907, Spain
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Valencia, Comunidad Valencia, 46010, Spain
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A Coruña, Galicia, 15009, Spain
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Santiago de Compostela-Coru A, Galicia, 15706, Spain
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Madrid, Madrid, 28027, Spain
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Majadahonda, Madrid, 28222, Spain
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El Palmar, Murcia, 30120, Spain
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Pamplona, Navarre, 31008, Spain
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Skellefte, Skelleftea, 93186, Sweden
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Lund, Skåne County, 22242, Sweden
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Gothenburg, 413 45, Sweden
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Stockholm, 171 64, Sweden
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Basel, 4031, Switzerland
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Bern, 3010, Switzerland
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Lucerne, 6000, Switzerland
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Sankt Gallen, 9007, Switzerland
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Zurich, 8091, Switzerland
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Taichung, Taichung, 404, Taiwan
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Taichung, Taichung, 40705, Taiwan
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Taipei, Taipei, 10002, Taiwan
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Taipei, Taiwan, 11217, Taiwan
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Mersin, Mersin, 33079, Turkey (Türkiye)
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Dinar, 03400, Turkey (Türkiye)
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Eskişehir, 26480, Turkey (Türkiye)
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Istanbul, 34303, Turkey (Türkiye)
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Birmingham, Edgbaston, B16 8QQ, United Kingdom
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London, GREATER LONDON, NW3 2QG, United Kingdom
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London, London, SE1 1YR, United Kingdom
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Glasgow, Scotland, G20 0SP, United Kingdom
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Cardiff, CF15 9SS, United Kingdom
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Manchester, M15 6SE, United Kingdom
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NA
- Masking
- NONE
- Masking Details
- Study 302 is a single arm, OLE study. All participants will receive open-label ALXN2220 upon entry into the OLE study. No IMP masking or blinding procedures will be implemented or maintained during the OLE study.
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 18, 2026
First Posted
August 27, 2026
Study Start (Estimated)
November 23, 2027
Primary Completion (Estimated)
July 11, 2030
Study Completion (Estimated)
July 11, 2030
Last Updated
August 27, 2026
Record last verified: 2026-08
Data Sharing
- IPD Sharing
- Will share
Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.