ClinicalTrials.gov record NCT07789483
An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of ALXN2220 in Adults With Transthyretin Amyloid Cardiomyopathy
An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of ALXN2220 in Adult Participants With Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This open-label extension studies long-term safety, tolerability, and efficacy of ALXN2220 in people with transthyretin amyloid cardiomyopathy who completed Study ALXN2220-ATTRCM-301. Participants receive ALXN2220 by IV infusion every 4 weeks for a minimum of 12 months and up to 24 months or until commercial access. The study tracks side effects, mortality and cardiovascular events, symptom and function scores, and immune responses to the drug.
What it is studying. The study is testing the long-term safety and effects of intravenous ALXN2220 given in addition to standard care in adults with ATTR-CM who completed Study 301. It will collect data on treatment-emergent adverse events and on clinical outcomes such as death, cardiovascular events, symptom and quality-of-life measures (KCCQ-OS), functional capacity (6-minute walk test), and immune responses to the drug over up to 24 months.
What the researchers hope to learn. To evaluate long-term safety and tolerability of ALXN2220 (percentage with treatment-emergent adverse events). To assess long-term effects on all-cause mortality, cardiovascular clinical events, time-to-event outcomes, symptoms and health-related quality of life (KCCQ-OS), functional capacity (6-minute walk test), and immunogenicity (anti-drug and neutralizing antibodies) up to 24 months.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- ALXN2220
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- 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.
Exclusion criteria
- 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.
Study description and what participation involves
The registry record has no detailed description beyond the summary above.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants give informed consent and receive ALXN2220 as an IV infusion every 4 weeks for a minimum of 12 months and up to 24 months (or until they have access to commercially available ALXN2220). They attend periodic study visits during which the study team records side effects and major clinical events (including deaths and cardiovascular events) and monitors safety. Participants complete questionnaires about symptoms and quality of life (KCCQ) and perform a six-minute walk test to measure functional capacity. Blood samples will be collected to assess immune responses to ALXN2220 (anti-drug antibodies and neutralizing antibodies).
How the study is structured. This is an open-label, single-group extension study. Eligible participants who completed the blinded treatment period of Study 301 receive ALXN2220 by IV infusion every 4 weeks for at least 12 months and up to 24 months (or until they have access to commercially available ALXN2220). There is one treatment arm, no masking, and outcomes are followed for up to 24 months, including safety (treatment-emergent adverse events) and several clinical and functional measures.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- The registry says recruitment has not started. When is it expected to open, and can I register interest now?
- The listed sites are in 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, United Kingdom, United States. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on wild-type ATTR amyloidosis
Important dates
- Study start
- Nov 23, 2027 (estimated)
- Primary completion
- Jul 11, 2030 (estimated)
- Study completion
- Jul 11, 2030 (estimated)
- Target enrollment
- 1180 participants (estimated)
- Last updated on registry
- Aug 28, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Alexion Pharmaceuticals, Inc., Sponsor 📞 1-855-752-2356 ✉ clinicaltrials@alexion.com
Participating sites (195)
- 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
Terms used on this page · explanations by MedicalUpdates.ai
- Blinded
- Who does not know which intervention a participant is receiving. It can apply to participants, the people giving the treatment, those assessing results, or those analysing the data.
- Informed Consent
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- legally authorized representative
- A person who is legally allowed to make decisions, including consent to research, on behalf of someone who cannot consent for themselves.
- Sponsor
- The organisation or person that initiates the study and has authority over it. The sponsor may be a company, hospital, university, government agency or individual, and is not necessarily the organisation that funds the study.
- investigational
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
- 5 half-lives
- The time it takes for the amount of a drug in the body to fall by half. 'Five half-lives' is often used as the point at which a previous drug has essentially left the body.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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