Amyloid Transthyretin CardiomyopathyPhase 2NCT07213583
Study of Re-Treatment With ALXN2220 in Patients With ATTR-CM
A Phase 2 study testing ALXN2220 in people with Amyloid Transthyretin Cardiomyopathy, run by Neurimmune AG at 6 sites in 4 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyloid Transthyretin Cardiomyopathy
- What is being tested?
- ALXN2220
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 35 participants
- Where
- 6 sitesin 4 countries
- Study dates
- 28 Aug 2025to 31 Oct 2026
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Must have received at least one dose of ALXN2220 in Study NI006-101
Who cannot join
- 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
Trial locations and contacts
6 sites in 4 countries.
6 sites in 4 countries
France3 sites
Hôpital Henri Mondor
Créteil, 94000
CHU de Rennes - Hôpital Pontchaillou
Rennes, 35033
CHU Toulouse - Hôpital Rangueil
Toulouse, 31059
Spain1 site
Hospital Universitario Puerta de Hierro Majadahonda
Majadahonda, 28222
About this study
Official 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
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.
Read the 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.
- Conditions
- Amyloid Transthyretin Cardiomyopathy
- Being tested
- ALXN2220
- Keywords
- Amyloid Depleter, ALXN2220, Transthyretin Amyloid Cardiomyopathy, Transthyretin amyloid (ATTR), Transthyretin (TTR), Amyloidosis, ATTR-CM
- Medical subject headings
- Amyloidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALXN2220Drug
Participants will receive ALXN2220 via IV infusion.
| Study group | Role | What they receive |
|---|---|---|
| ALXN2220 | Experimental | Participants will receive ALXN2220 via intravenous (IV) infusion during the Active Treatment Period Receives: Drug: ALXN2220 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from Baseline to Week 48 in ECV measured by cMRI
Measured: Baseline, Week 48
Change from Baseline to Week 48 in Heart Retention/Whole Body Retention measured by scintigraphy
Measured: Baseline, Week 48
Secondary outcomes (3)
Incidence of TEAEs, including serious TEAEs
Measured: From Baseline up to Week 48
ALXN2220 serum concentrations
Measured: From Baseline up to Week 48
Incidence of Anti-Drug Antibodies
Measured: From Baseline up to Week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Oct 2025
- Study start28 Aug 2025
- Primary completion31 Oct 2026 (estimated)
- Study completion31 Oct 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyloid Transthyretin CardiomyopathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 35 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Neurimmune AG
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Alexion Pharmaceuticals, Inc.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07213583
- Sponsor's protocol ID
- NI006-102
- Other IDs
- 2025-520506-35-00, 2025
- Status last verified
- Oct 2026
- First submitted
- 15 Sep 2025
- Last updated
- 7 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Neurimmune has a public commitment to allow requests for study data and will be supplying protocol, results and plain language summaries.