Long-term follow-up of ALXN2220 in adults with transthyretin amyloid heart disease
Continues ALXN2220 treatment in adults with transthyretin amyloid cardiomyopathy who finished an earlier study.
Key facts
- Who runs it
- Alexion Pharmaceuticals, Inc.
- Where
- Prague and Praha, CzechiaSee the 2 sitesAlso in 26 other countries, 172 sites
- Trial code
- Registry title
- An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of ALXN2220 in Adults With Transthyretin Amyloid Cardiomyopathy
- Status
- Not yet recruiting
- Phase
- Phase 3
- People taking part
- 1,180 people, planned
- Starts
- 23 Nov 2027
- Expected to end
- 11 Jul 2030
- Type of study
- Interventional
- Treatments tested
- ALXN2220
- Record updated
- 28 August 2026
Who can take part
AI summary
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for adults with transthyretin amyloid cardiomyopathy (ATTR-CM) who finished the blinded treatment period of Study 301. They will receive ALXN2220 along with standard care.
- Age
- Adults (no exact age range is given in the record).
- Sex
- Men and women can join.
- Disease stage
- Transthyretin amyloid cardiomyopathy (ATTR-CM).
- Earlier treatment
- Must have completed the Blinded Treatment Period of Study 301. Must not have permanently stopped the study treatment in Study 301.
Main reasons for not taking part
- Stopped study treatment for good in Study 301
- Withdrew full consent during Study 301
- Known or suspected allergy to proteins or any part of ALXN2220
- Had a heart transplant or a permanent CMAD implant
- Took part in another study or took an investigational drug within 30 days or 5 half-lives
The registry criteria, word for word
Show all 9 criteriaShow less
Other criteria
- 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
Where in Czechia
Also in 26 other countries, 172 sites
- PragueHospital not named in the registryContact: Alexion Pharmaceuticals, Inc., SponsorNo longer taking part
- Praha, Central BohemiaHospital not named in the registryContact: Alexion Pharmaceuticals, Inc., SponsorNo longer taking part
About the trial
What the trial wants to find out
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.
The group in the trial
- ALXN2220Treatment being tested
Eligible 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.
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorData and sources
- Registry record
- Listed on ClinicalTrials.gov and on the EU's official list of trials (CTIS).
- Official title
- An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of ALXN2220 in Adult Participants With Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
- Plain-language title
- Long-term follow-up of ALXN2220 in adults with transthyretin amyloid heart diseaseGenerated from the registry record. The registry title is the official one.
- AI summary
- From the trial's eligibility criteria. Read by machine on 13 November 2026.
- Record updated
- 28 August 2026