Study of Re-Treatment With ALXN2220 in Patients With ATTR-CM | NCT07213583

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

NCT07213583

Sponsor

Neurimmune AG

Status

Active, not recruiting

Last Update Posted

Dec 19, 2025Actual

Enrollment

35Estimated

Phase

Phase 2

Conditions

Amyloid Transthyretin Cardiomyopathy

Interventions

ALXN2220

Countries

France
Germany
Netherlands
Spain

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

ID	Type	Description	Link
2025-520506-35-00	EU Trial (CTIS) Number

Status Module

Record Verification Date

Oct 2025

Overall Recruitment Status or Expanded Access Status

Active, not recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Aug 28, 2025Actual

Primary Completion Date

Oct 31, 2026Estimated

Completion Date

Oct 31, 2026Estimated

First Submitted Date

Sep 15, 2025

First Submission Date that Met QC Criteria

Oct 1, 2025

First Posted Date

Oct 9, 2025Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Dec 18, 2025

Last Update Posted Date

Dec 19, 2025Actual

Name	Class
Alexion Pharmaceuticals, Inc.	INDUSTRY

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

35Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
ALXN2220	Experimental	Participants will receive ALXN2220 via intravenous (IV) infusion during the Active Treatment Period	Drug: ALXN2220

Interventions

Name	Type	Description	Arm Group Labels	Other Names
ALXN2220	Drug	Participants will receive ALXN2220 via IV infusion.	ALXN2220

Outcomes Module

Primary Outcomes

Measure	Description	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 Outcomes

Measure	Description	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

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

Not provided

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Not provided

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Hôpital Henri Mondor		Créteil		94000	France
CHU de Rennes - Hôpital Pontchaillou

IPD Sharing Statement Module

Plan to Share IPD

Yes

Description

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

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D000686	Amyloidosis

Ancestor Terms

ID	Term
D057165	Proteostasis Deficiencies
D008659	Metabolic Diseases
D009750	Nutritional and Metabolic Diseases

Browse Leaves

Not provided

Browse Branches

Not provided