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

NCT07213583 | Active Not Recruiting Phase 2

• 2 other identifiers: NI006-1022025-520506-35-00
• Study Type: interventional
• Target: 35
• Locations: 4 countries
• Sites: 6

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.

Conditions

Amyloid Transthyretin Cardiomyopathy

Keywords

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

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (3)

• 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

Study Arms (1)

ALXN2220

EXPERIMENTAL

Participants will receive ALXN2220 via intravenous (IV) infusion during the Active Treatment Period

Drug: ALXN2220

Interventions

ALXN2220 DRUG

Participants will receive ALXN2220 via IV infusion.

ALXN2220

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• Neurimmune AG: lead
• Alexion Pharmaceuticals, Inc.: collaborator

Study Sites (6)

Hôpital Henri Mondor

Créteil, 94000, France

Location

CHU de Rennes - Hôpital Pontchaillou

Rennes, 35033, France

Location

CHU Toulouse - Hôpital Rangueil

Toulouse, 31059, France

Location

Universitätsklinikum Heidelberg

Heidelberg, 69120, Germany

Location

University Medical Center Groningen

Groningen, 9713, Netherlands

Location

Hospital Universitario Puerta de Hierro Majadahonda

Majadahonda, 28222, Spain

Location

MeSH Terms

Conditions

Amyloidosis

Condition Hierarchy (Ancestors)

Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 15, 2025
• First Posted: October 9, 2025
• Study Start: August 28, 2025
• Primary Completion (Estimated): October 31, 2026
• Study Completion (Estimated): October 31, 2026
• Last Updated: December 19, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will share

Locations

NL (1); DE (1); ES (1); FR (3)