ALXN2220 for Transthyretin amyloid cardiomyopathy (ATTR-CM)

Inclusion criteria

• {"criterion_text":"- 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\n- 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\n- 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.\n- 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.\n- 2. Male or female participants aged ≥18 years at the time of obtaining informed consent"}

Exclusion criteria

• {"criterion_text":"- 1. Suspected or known intolerance/allergy to proteins or any components of the study drug\n- 10. Hemoglobin < 8 g/dL for women or < 9 g/dL for men measured at screening\n- 11. Platelet count < 100 x 109/L or other disorder associated with clinically significant thrombocytopenia measured at screening\n- 12. History of bleeding diathesis or coagulopathy (e.g., antiphospholipid antibody syndrome, congenital disorders such as hemophilia A, B, and Von Willebrand disease)\n- 13. History of human immunodeficiency virus (HIV) or positive HIV antibody test at screening\n- 14. Alanine transaminase (ALT) > 2.5 × upper limit of normal (ULN) measured at screening\n- 15. Total bilirubin > 2.5 × ULN measured at screening\n- 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\n- 17. Lymphoma, leukemia, or any malignancy or clonal stem cell disorder (myelodysplastic syndrome, polycythemia vera, essential thrombocytopenia, myelofibrosis) within the past 5 years\n- 18. Respiratory insufficiency requiring continuous daytime oxygen therapy\n- 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\n- 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)\n- 20. Use of disallowed medication\n- 21. Prior treatment with an anti-ATTR antibody other than ALXN2220/NI006 or with TTR-directed gene editing (nexiguran-ziclumeran)\n- 22. Polyneuropathy secondary to ATTR amyloidosis requiring a wheelchair (i.e., Polyneuropathy disability score IV)\n- 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\n- 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\n- 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\n- 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\n- 6. Uncontrolled hypertension (resting systolic blood pressure (BP) > 160 mm Hg or diastolic BP > 100 mm Hg at screening)\n- 7. Resting systolic BP < 90 mm Hg or symptomatic orthostatic hypotension, despite appropriate treatment, at screening per Investigator’s assessment\n- 8. Uncontrolled clinically significant cardiac arrhythmia, per Investigator’s assessment\n- 9. Active listing for organ transplantation at time of ICF signature"}

Primary endpoints

• {"endpoint_text":"- Change from baseline to Visit 16 in cMRI","definition_or_measurement_approach":"Change from baseline to Visit 16 as measured by cardiac MRI (cMRI) assessments (as stated)"}
• {"endpoint_text":"- Change from baseline to Visit 16 in scintigraphy","definition_or_measurement_approach":"Change from baseline to Visit 16 as measured by scintigraphy assessments (as stated)"}

Secondary endpoints

• {"endpoint_text":"- Incidence of TEAEs, including serious TEAEs","definition_or_measurement_approach":"Incidence of treatment-emergent adverse events, including serious TEAEs (as stated)"}
• {"endpoint_text":"- Change from baseline by visit for vital signs and physical examination","definition_or_measurement_approach":"Change from baseline assessed by scheduled visits for vital signs and physical examination (as stated)"}
• {"endpoint_text":"- Change from baseline by visit in clinical laboratory parameters","definition_or_measurement_approach":"Change from baseline assessed by scheduled visits for clinical laboratory parameters (as stated)"}
• {"endpoint_text":"- Change from baseline by visit for ECG parameters","definition_or_measurement_approach":"Change from baseline assessed by scheduled visits for ECG parameters (as stated)"}
• {"endpoint_text":"- ALXN2220 serum concentrations","definition_or_measurement_approach":"Serum concentrations of ALXN2220 measured (pharmacokinetic sampling) (as stated)"}
• {"endpoint_text":"- ADA incidence, response category and titer","definition_or_measurement_approach":"Incidence of anti-drug antibodies (ADA), response category and titer (as stated)"}

Netherlands

Earliest CTIS Part Ii Submission Date

19-06-2025

Latest Decision Or Authorization Date

24-06-2025

Processing Time Days

5

Number Of Sites

1

Number Of Participants

5

Spain

Earliest CTIS Part Ii Submission Date

11-06-2025

Latest Decision Or Authorization Date

24-06-2025

Processing Time Days

13

Number Of Sites

1

Number Of Participants

4

Germany

Earliest CTIS Part Ii Submission Date

28-03-2025

Latest Decision Or Authorization Date

26-06-2025

Processing Time Days

90

Number Of Sites

1

Number Of Participants

10

France

Earliest CTIS Part Ii Submission Date

13-05-2025

Latest Decision Or Authorization Date

27-06-2025

Processing Time Days

45

Number Of Sites

3

Number Of Participants

16

Primary sponsor

Full Name

Neurimmune AG

Organisation Type

Pharmaceutical company

Country Of Registered Address

Switzerland

Contract research organisations

Name

Icon Clinical Research Limited

Responsibilities

codes:1,10,11,12,2,5,6,7,8; contact CTIS-Biotech@iconplc.com

Name

SGS Analytics Germany GmbH

Responsibilities

Clinical chemistry, Serology / endocrinology, Biomarker analysis, sample logistics; contact de.hn.muc.info@sgs.com

Name

QPS Netherlands B.V.

Responsibilities

PK and ADA analysis; contact office-nl@qps.com

Third parties

• {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"codes:1,10,11,12,2,5,6,7,8","organisation_type":"Pharmaceutical company"}
• {"country":"Germany","full_name":"SGS Analytics Germany GmbH","duties_or_roles":"Clinical chemistry, Serology / endocrinology, Biomarker analysis, sample logistics","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"Netherlands","full_name":"QPS Netherlands B.V.","duties_or_roles":"PK and ADA analysis","organisation_type":"Pharmaceutical company"}