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
- Are there results?
- Not yet: the study is under way
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.
Study at a glance
From the registry record, last updated by the sponsor on 7 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT07213583
- Acronym / sponsor ID
- NI006-102
- Conditions
- Amyloid Transthyretin Cardiomyopathy
- Keywords
- Amyloid Depleter, ALXN2220, Transthyretin Amyloid Cardiomyopathy, Transthyretin amyloid (ATTR), Transthyretin (TTR), Amyloidosis, ATTR-CM
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 35 participants (estimated)
- Lead sponsor
- Neurimmune AGIndustry (pharma, biotech, devices)
- Collaborators
- Alexion Pharmaceuticals, Inc.
- Study start
- 28 Aug 2025 (actual)
- Primary completion
- 31 Oct 2026 (estimated)
- Study completion
- 31 Oct 2026 (estimated)
- First posted
- 9 Oct 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ALXN2220 | Experimental | Drug: ALXN2220Participants will receive ALXN2220 via intravenous (IV) infusion during the Active Treatment Period |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- Must have received at least one dose of ALXN2220 in Study NI006-101
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ALXN2220Participants will receive ALXN2220 via IV infusion. | Drug | ALXN2220 |
Primary outcome
Change from Baseline to Week 48 in ECV measured by cMRI
Time frame: Baseline, Week 48
Change from Baseline to Week 48 in Heart Retention/Whole Body Retention measured by scintigraphy
Time frame: Baseline, Week 48
3 secondary outcomes
Incidence of TEAEs, including serious TEAEs
Time frame: From Baseline up to Week 48
ALXN2220 serum concentrations
Time frame: From Baseline up to Week 48
Incidence of Anti-Drug Antibodies
Time frame: From Baseline up to Week 48
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Oct 2026.
- Individual participant dataSharing planned: Yes. Neurimmune has a public commitment to allow requests for study data and will be supplying protocol, results and plain language summaries.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
6 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ALXN2220 in people with Amyloid Transthyretin Cardiomyopathy, run by Neurimmune AG at 6 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 7 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in France, Germany, Netherlands, Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from Baseline to Week 48 in ECV measured by cMRI (time frame: Baseline, Week 48). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Oct 2026.