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

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Neurimmune AG

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Cardiovascular Diseases [C14]

Trial duration

30 Jun 2025 → ongoing

Decision date (initial)

2025-06-27

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Funding sources

Neurimmune AG, Alexion Pharmaceuticals, Inc

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Others, Pharmacodynamic, Pharmacokinetic

To evaluate the pharmacodynamics of re-treatment with ALXN2220

Secondary objectives 3

1. To evaluate the safety and tolerability of re-treatment with ALXN2220
2. To evaluate the PK of ALXN2220
3. To evaluate the immunogenicity of re-treatment with ALXN2220

Conditions and MedDRA coding

Adults with Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

Regulatory references

Plan to share IPD

Yes

IPD plan description

Neurimmune has a public commitment to allow requests for study data and will be supplying protocol, results and plain language summaries.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. 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
2. 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
3. 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.
4. 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.
5. 2. Male or female participants aged ≥18 years at the time of obtaining informed consent

Exclusion criteria 23

1. 1. Suspected or known intolerance/allergy to proteins or any components of the study drug
2. 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)
3. 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. 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. 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. 6. Uncontrolled hypertension (resting systolic blood pressure (BP) > 160 mm Hg or diastolic BP > 100 mm Hg at screening)
7. 7. Resting systolic BP < 90 mm Hg or symptomatic orthostatic hypotension, despite appropriate treatment, at screening per Investigator’s assessment
8. 8. Uncontrolled clinically significant cardiac arrhythmia, per Investigator’s assessment
9. 9. Active listing for organ transplantation at time of ICF signature
10. 10. Hemoglobin < 8 g/dL for women or < 9 g/dL for men measured at screening
11. 11. Platelet count < 100 x 109/L or other disorder associated with clinically significant thrombocytopenia measured at screening
12. 12. History of bleeding diathesis or coagulopathy (e.g., antiphospholipid antibody syndrome, congenital disorders such as hemophilia A, B, and Von Willebrand disease)
13. 13. History of human immunodeficiency virus (HIV) or positive HIV antibody test at screening
14. 14. Alanine transaminase (ALT) > 2.5 × upper limit of normal (ULN) measured at screening
15. 15. Total bilirubin > 2.5 × ULN measured at screening
16. 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. 17. Lymphoma, leukemia, or any malignancy or clonal stem cell disorder (myelodysplastic syndrome, polycythemia vera, essential thrombocytopenia, myelofibrosis) within the past 5 years
18. 18. Respiratory insufficiency requiring continuous daytime oxygen therapy
19. 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
20. 20. Use of disallowed medication
21. 21. Prior treatment with an anti-ATTR antibody other than ALXN2220/NI006 or with TTR-directed gene editing (nexiguran-ziclumeran)
22. 22. Polyneuropathy secondary to ATTR amyloidosis requiring a wheelchair (i.e., Polyneuropathy disability score IV)
23. 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

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. Change from baseline to Visit 16 in cMRI
2. Change from baseline to Visit 16 in scintigraphy

Secondary endpoints 6

1. Incidence of TEAEs, including serious TEAEs
2. Change from baseline by visit for vital signs and physical examination
3. Change from baseline by visit in clinical laboratory parameters
4. Change from baseline by visit for ECG parameters
5. ALXN2220 serum concentrations
6. ADA incidence, response category and titer

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Neurimmune AG

Sponsor organisation

Neurimmune AG

Address

Wagistrasse 18

City

Schlieren

Postcode

8952

Country

Switzerland

Scientific contact point

Organisation

Neurimmune AG

Contact name

Peter C. Kahr, MD

Public contact point

Organisation

Neurimmune AG

Contact name

VP Clinical Development

Third parties 3

Locations

4 EU/EEA countries · 6 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 23 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

7 submissions — initial application plus substantial / non-substantial modifications