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NCT Number: NCT07213583

Study of Re-Treatment With ALXN2220 in Patients With ATTR-CM

This is a Phase 2, single-arm, multicenter study to evaluate the pharmacodynamics and safety of re-treatment with ALXN2220 in previous participants of Study NI006-101.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Amyloid Transthyretin Cardiomyopathy Amyloidosis Metabolic Diseases Nutritional and Metabolic Diseases Proteostasis Deficiencies

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Hôpital Henri Mondor, Créteil, France

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About this study

This is a Phase 2, single arm, multicenter study to evaluate the pharmacodynamics and safety of re-treatment with ALXN2220 in the patient population of previous participants in Study NI006-101.

The study will also explore changes in cardiac structure and function, and clinical function of this dosing regimen, as well as changes in cardiac amyloid load, structure and function during the treatment-pause since conclusion of Study NI006-101. The study consists of a Screening Period and a 48 week Active Treatment Period.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Must have received at least one dose of ALXN2220 in Study NI006-101

Exclusion criteria

  • Suspected or known intolerance/allergy to proteins or any components of the study drug
  • 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)
  • Any new or uncontrolled condition after completion of Study NI006-101 that could make the participant unsuitable for participation in this study

Treatment and study plan

ALXN2220

Drug

Participants will receive ALXN2220 via IV infusion.

Primary outcomes

  1. Change from Baseline to Week 48 in ECV measured by cMRI

    Time frame: Baseline, Week 48

  2. Change from Baseline to Week 48 in Heart Retention/Whole Body Retention measured by scintigraphy

    Time frame: Baseline, Week 48

Secondary outcomes

  1. Incidence of TEAEs, including serious TEAEs

    Time frame: From Baseline up to Week 48

  2. ALXN2220 serum concentrations

    Time frame: From Baseline up to Week 48

  3. Incidence of Anti-Drug Antibodies

    Time frame: From Baseline up to Week 48

Sponsors and collaborators

Lead sponsor

Neurimmune AG

Industry

Collaborators

  • Alexion Pharmaceuticals, Inc.

Registry information

Official study title

A Phase 2, Single Arm, Multicenter Study to Evaluate the Pharmacodynamics and Safety of Re-Treatment With ALXN2220 in Patients With Transthyretin Amyloid Cardiomyopathy

Important dates

Study start
2025
Primary completion
2026
Study completion
2026
First posted
Oct 9, 2025
Registry last updated
Dec 19, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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