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

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

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT07213583

Enrollment

Registered

2025-10-09

Start date

2025-08-28

Completion date

2026-10-31

Last updated

2025-12-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Amyloid Transthyretin Cardiomyopathy

Keywords

Amyloid Depleter, ALXN2220, Transthyretin Amyloid Cardiomyopathy, Transthyretin amyloid (ATTR), Transthyretin (TTR), Amyloidosis, ATTR-CM

Brief summary

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.

Detailed description

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.

Interventions

DRUGALXN2220

Participants will receive ALXN2220 via IV infusion.

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame
Change from Baseline to Week 48 in ECV measured by cMRI	Baseline, Week 48
Change from Baseline to Week 48 in Heart Retention/Whole Body Retention measured by scintigraphy	Baseline, Week 48

Secondary

Measure	Time frame
Incidence of TEAEs, including serious TEAEs	From Baseline up to Week 48
ALXN2220 serum concentrations	From Baseline up to Week 48
Incidence of Anti-Drug Antibodies	From Baseline up to Week 48

Countries

France, Germany, Netherlands, Spain

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026