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A Phase 2 Multicenter Study to Test the Effects of ALXN2220 on Markers of Disease Severity and the Safety of Re-Treatment in Patients with Transthyretin Amyloid Cardiomyopathy
EU CTIS ID: 2025-520506-35-00
What this study is testing
To evaluate the pharmacodynamics of re-treatment with ALXN2220
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1. Must have received at least one dose of ALXN2220 in Study NI006-101 and, in the opinion of the Investigator, tolerated the study drug
- 3. Able to understand and sign an informed consent form prior to initiation of any study procedures, and willing and able to comply with all study procedures
- 4. Women of childbearing potential (WOCBP) must have a negative serum pregnancy test at screening and must agree to use highly effective physician-approved contraception from screening to 5 months after ending study participation.
- 5. Male participants are eligible to participate if they agree to the following during the study intervention period and for at least 7 months after the last dose of study intervention: • Refrain from donating fresh unwashed semen Plus, either: • Be abstinent from heterosexual intercourse as their preferred and usual lifestyle (abstinent on a long term and persistent basis) and agree to remain abstinent OR • Must agree to use a male condom and should also be advised of the benefit for a female partner to use a highly effective method of contraception as a condom may break or leak when having sexual intercourse with a WOCBP who is not currently pregnant.
- 2. Male or female participants aged ≥18 years at the time of obtaining informed consent
You likely can't join if
- 1. Suspected or known intolerance/allergy to proteins or any components of the study drug
- 10. Hemoglobin < 8 g/dL for women or < 9 g/dL for men measured at screening
- 11. Platelet count < 100 x 109/L or other disorder associated with clinically significant thrombocytopenia measured at screening
- 12. History of bleeding diathesis or coagulopathy (e.g., antiphospholipid antibody syndrome, congenital disorders such as hemophilia A, B, and Von Willebrand disease)
- 13. History of human immunodeficiency virus (HIV) or positive HIV antibody test at screening
- 14. Alanine transaminase (ALT) > 2.5 × upper limit of normal (ULN) measured at screening
See the full eligibility criteria
Who can join
- 1. Must have received at least one dose of ALXN2220 in Study NI006-101 and, in the opinion of the Investigator, tolerated the study drug
- 3. Able to understand and sign an informed consent form prior to initiation of any study procedures, and willing and able to comply with all study procedures
- 4. Women of childbearing potential (WOCBP) must have a negative serum pregnancy test at screening and must agree to use highly effective physician-approved contraception from screening to 5 months after ending study participation.
- 5. Male participants are eligible to participate if they agree to the following during the study intervention period and for at least 7 months after the last dose of study intervention: • Refrain from donating fresh unwashed semen Plus, either: • Be abstinent from heterosexual intercourse as their preferred and usual lifestyle (abstinent on a long term and persistent basis) and agree to remain abstinent OR • Must agree to use a male condom and should also be advised of the benefit for a female partner to use a highly effective method of contraception as a condom may break or leak when having sexual intercourse with a WOCBP who is not currently pregnant.
- 2. Male or female participants aged ≥18 years at the time of obtaining informed consent
What rules you out
- 1. Suspected or known intolerance/allergy to proteins or any components of the study drug
- 10. Hemoglobin < 8 g/dL for women or < 9 g/dL for men measured at screening
- 11. Platelet count < 100 x 109/L or other disorder associated with clinically significant thrombocytopenia measured at screening
- 12. History of bleeding diathesis or coagulopathy (e.g., antiphospholipid antibody syndrome, congenital disorders such as hemophilia A, B, and Von Willebrand disease)
- 13. History of human immunodeficiency virus (HIV) or positive HIV antibody test at screening
- 14. Alanine transaminase (ALT) > 2.5 × upper limit of normal (ULN) measured at screening
- 15. Total bilirubin > 2.5 × ULN measured at screening
- 16. Current unstable liver or biliary disease per Investigator’s assessment defined by the presence of ascites, encephalopathy, coagulopathy, hypoalbuminemia, esophageal or gastric varices, persistent jaundice, or cirrhosis. Participants with severe hepatic impairment (e.g., Child Pugh Class C) are not eligible. Participants with hepatitis B (presence of HbsAg and detectable HBV Deoxyribonucleic acid (DNA)) or hepatitis C (presence of HCV antibodies and detectable HCV viral load) are not eligible
- 17. Lymphoma, leukemia, or any malignancy or clonal stem cell disorder (myelodysplastic syndrome, polycythemia vera, essential thrombocytopenia, myelofibrosis) within the past 5 years
- 18. Respiratory insufficiency requiring continuous daytime oxygen therapy
- 19. Participants with renal failure requiring dialysis or who have an estimated glomerular filtration rate (eGFR) by Chronic Kidney Disease epidemiology collaboration (CKD-EPI) formula < 20 mL/min/1.73 m2 measured at Screening
- 2. Treatment or study discontinuation in Study NI006-101 due to a treatment-related adverse event that was serious, severe or life-threatening (on Common terminology criteria for adverse events (CTCAE) scale)
- 20. Use of disallowed medication
- 21. Prior treatment with an anti-ATTR antibody other than ALXN2220/NI006 or with TTR-directed gene editing (nexiguran-ziclumeran)
- 22. Polyneuropathy secondary to ATTR amyloidosis requiring a wheelchair (i.e., Polyneuropathy disability score IV)
- 23. Known medical or psychological condition(s) or risk factor that, in the opinion of the Investigator or Sponsor, might interfere with the participant’s full participation in the study, pose any additional risk for the participant, or confound the assessment of the participant or outcome of the study
- 3. Participation in another investigational clinical study or intake of investigational drug within 30 calendar days or 5 half-lives of Day 1, whichever is longer
- 4. Any new or uncontrolled condition after completion of Study NI006-101 that could make the participant unsuitable for participation in this study, per Investigator’s assessment
- 5. Acute coronary syndrome, unstable angina, stroke, transient ischemic attack, coronary revascularization, cardiac device implantation, cardiac valve repair, or major cardiac surgery within 3 months of screening
- 6. Uncontrolled hypertension (resting systolic blood pressure (BP) > 160 mm Hg or diastolic BP > 100 mm Hg at screening)
- 7. Resting systolic BP < 90 mm Hg or symptomatic orthostatic hypotension, despite appropriate treatment, at screening per Investigator’s assessment
- 8. Uncontrolled clinically significant cardiac arrhythmia, per Investigator’s assessment
- 9. Active listing for organ transplantation at time of ICF signature
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 65+ years, 18-64 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.