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Light Chain Cardiac AmyloidosisPhase 1/2NCT07504289

CAR-NK Therapy for Cardiac Amyloidosis

A Phase 1/2 study testing CAR-NK in people with Light Chain Cardiac Amyloidosis, run by Second Affiliated Hospital, Zhejiang University, School of Medicine.

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Sites and contacts
Official record
Search all trials

Trial summary

Relapsed/refractory (R/R) light chain cardiac amyloidosis is associated with a poor prognosis, and cellular immunotherapy constitutes a crucial therapeutic modality for these patients. Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Light Chain Cardiac Amyloidosis
What is being tested?
CAR-NK
Type of treatment
Surgery or procedure
Could I receive a placebo?
No, everyone receives an active treatment
How many people
36 participants
Where
No sites listed yet
Study dates
7 Mar 2026to 6 Mar 2029
Phase
Phase 1/2
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 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Voluntarily participate in this study and sign the informed consent form.
  • Aged 18 to 75 years, of either sex.
  • Pathologically confirmed amyloidosis, with positive Congo red staining of tissue specimens, green birefringent material observed under polarizing microscope, or characteristic electron microscopic findings.
  • Presence of measurable light chain amyloidosis lesions meeting at least one of the following criteria:
  • 1）Serum M-protein ≥ 0.5 g/dL (detected by routine serum protein electrophoresis and immunofixation electrophoresis).
  • 2）Abnormal κ/λ ratio, with the difference between involved and uninvolved free light chains (dFLC) ≥ 50 mg/L.
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  • 5. Confirmed cardiac involvement by amyloidosis, meeting at least one of the following criteria; extramyocardial organ involvement is permitted:
  • Echocardiography: Mean ventricular wall thickness > 12 mm with no other identifiable cardiac etiologies.
  • Elevated NT-ProBNP level (> 332 ng/L) without concomitant renal failure or atrial fibrillation.
  • 6. Having received at least one course of first-line therapy based on bortezomib or CD38 monoclonal antibody with suboptimal hematological response, meeting at least one of the following criteria:
  • Failure to achieve partial response (PR) after 1 treatment cycle.
  • Failure to achieve very good partial response (VGPR) after 2 treatment cycles. 7. Estimated overall survival ≥ 12 weeks. 8. Eastern Cooperative Oncology Group (ECOG) performance status score of 0 to 3.
  • 9. Sufficient organ function reserve excluding the heart, meeting all the following criteria:
  • Peripheral blood neutrophil count ≥ 1000/μL, hemoglobin ≥ 7 g/dL, platelet count ≥ 50,000/μL; red blood cell or platelet transfusion is permitted within 1 week prior to screening.
  • Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 2.5 × upper normal limit (UNL).
  • Serum total bilirubin ≤ 1.5 × UNL.
  • Estimated glomerular filtration rate (eGFR) ≥ 20 mL/min/1.73 m², calculated by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula.
  • Baseline oxygen saturation > 92% in a natural indoor air environment. 10. A prior history of one hematopoietic stem cell transplantation is permitted.
  • 11. At least 3 weeks have elapsed since the completion of approved therapeutic interventions for AL cardiac amyloidosis (e.g., systemic chemotherapy, immunotherapy) prior to the administration of the study drug.
  • 12. Female subjects of childbearing potential must have a negative pregnancy test and agree to adopt effective contraceptive measures during the trial period.

Who cannot join

  • NT-ProBNP ≥ 8500 ng/L;
  • Heart failure of New York Heart Association (NYHA) functional class IIIB or IV ;
  • Heart failure judged by the investigator to be caused by ischemic heart disease (e.g., a previous myocardial infarction with documented elevated cardiac enzymes and electrocardiographic changes) or uncorrected valvular heart disease, rather than primarily by AL amyloidosis;
  • Hospitalization for unstable angina or myocardial infarction within 6 months prior to the first dose, or percutaneous coronary intervention with recent stent implantation within 6 months, or coronary artery bypass grafting within 6 months;
  • For subjects with congestive heart failure, hospitalization for cardiovascular-related diseases within 4 weeks prior to screening;
  • Baseline corrected QT interval by Fridericia's formula (QTcF) > 500 milliseconds on a 12-lead electrocardiogram at screening. Subjects with a permanent pacemaker implanted may be enrolled regardless of their calculated QTc interval;
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  • Supine systolic blood pressure < 90 mmHg, or symptomatic orthostatic hypotension (defined as a decrease in systolic blood pressure > 20 mmHg upon standing despite pharmacotherapy (e.g., midodrine, fludrocortisone) and no hypovolemia);
  • A history of hypersensitivity to any component of the cellular product;
  • A history of other malignant neoplasms;
  • Receipt of BCMA-targeted therapy, including antibody-drug conjugates (ADCs), bispecific antibodies, and cellular therapy, within 3 months prior to screening;
  • Receipt of gene therapy within 3 months prior to screening;
  • Active infections requiring treatment (excluding uncomplicated urinary tract infections and bacterial pharyngitis); prophylactic antibiotic, antiviral, and antifungal therapy is permitted, however;
  • Subjects infected with hepatitis B (HBsAg-positive with HBV-DNA < 10³ copies/mL is not an exclusion criterion) or hepatitis C virus (including virus carriers), syphilis, and other acquired or congenital immunodeficiency diseases, including but not limited to human immunodeficiency virus (HIV) infection;
  • Unresolved toxicities from prior antineoplastic therapy (toxicities per CTCAE Version 5.0 not recovered to ≤ Grade 1, except for fatigue, anorexia, and alopecia);
  • Subjects with a history of epilepsy or other central nervous system diseases;
  • Lactating women who are unwilling to discontinue breastfeeding;
  • Any other condition that the investigator deems may increase the risk to the subject or interfere with the trial results.

Trial locations and contacts

No sites are listed yet. Contact the study team or a site near you to ask about taking part.

Yang Xu contact

0571-87783679

yxu@zju.edu.cn

About this study

Official title: Clinical Study of CAR-NK Cells Targeting BCMA/CD19 in the Treatment of Refractory/Relapsed Light-Chain Cardiac Amyloidosis

Relapsed/refractory (R/R) light chain cardiac amyloidosis is associated with a poor prognosis, and cellular immunotherapy constitutes a crucial therapeutic modality for these patients. The efficacy and safety of CAR-T therapy have been reported in relevant studies; however, CAR-T manufacturing requires a lengthy timeline, and the leukapheresis procedure places an additional cardiac burden on patients. CAR-NK therapy boasts superior safety profiles compared with CAR-T therapy, and natural killer (NK) cells feature a wide range of sources. Investigators have accumulated prior experience in the clinical application of CAR-NK therapy, and has also achieved the successful development and preclinical application of CD19/BCMA dual-target CAR-T products. Furthermore, in the institution of the Investigator, there are dozens of newly diagnosed and more than 100 follow-up patients with AL cardiac amyloidosis each year. Investigators propose to initiate a phase I/II prospective clinical study to assess the safety and efficacy of umbilical cord blood-derived BCMA/CD19-targeted CAR-NK cell therapy for participants with relapsed/refractory light chain cardiac amyloidosis.

Being tested
CAR-NK

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CAR-NKProcedure or surgery

    Also called CD19/BCMA dual-target Chimeric Antigen Receptor Natural Killer Cells

    R/R AL cardiac amyloidosis patients in the CD19/BCMA dual-target CAR-NK therapy arm

Study groups in NCT07504289
Study groupRoleWhat they receive
Experimental armExperimentalNot described
Receives: Procedure: CAR-NK

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. safty and efficacy

    The overall incidence and severity of all adverse events were calculated using CTCAE Version 5.0. The incidence of treatment-emergent adverse events (TEAEs) and abnormal laboratory findings possibly or definitely related to CB CAR-NK-BCMA/CD19, as well as dose-limiting toxicities (DLTs), were assessed for any occurrence from the initiation of study treatment up to Day 28.

    Definition of Dose-Limiting Toxicity (DLT):Toxic events related to CB CAR-NK-BCMA/CD19 treatment occurring within 28 days post-infusion include:

    Grade ≥4 cytokine release syndrome (CRS) or immune effector cell-associated neurotoxicity syndrome (ICANS); Grade 3 CRS or ICANS with a duration of ≥7 days; Other Grade ≥3 hematological or non-hematological toxic reactions. The treatment efficacy in participants is evaluated in accordance with the LYRIC Efficacy Evaluation Criteria (2016).

    Measured: Starting on Day 0 (CB CAR-NK-BCMA/CD19 infusion), participants will return to the outpatient clinic at the following intervals:• Days 1, 4, 7, 10, 14, 21, 28• Month 2 (±1 week)• Month 3 (±1 week)

  2. the safety and efficacy of umbilical cord blood-derived CAR-NK cells targeting BCMA/CD19 (CB CAR-NK-BCMA/CD19) in the treatment of patients with light chain cardiac amyloidosis

    General condition of participants, ECOG performance status, symptoms and physical signs, complete blood count (CBC), serum biochemistry, ferritin, coagulation function, immunofixation electrophoresis (IFE), free light chains (FLC), cardiac biomarker tests (high-sensitivity troponin, BNP or NT-proBNP), electrocardiogram (ECG), echocardiography, immune function tests, etc.; peripheral blood collection for detection of B-cell, cytokine and CAR expansion; efficacy assessments on Days 14 and 28 including IFE, κ/λ free light chains (κ/λ FLC), and cardiac biomarker tests (high-sensitivity troponin, BNP or NT-proBNP).peripheral blood collection for detection of T-cell, cytokine and CAR expansion; assessment of comorbidities.

    Measured: 1.Follow-up visits will be conducted on Days 1, 4, 7, 10, 14, 21, and 28 post-infusion (treatment phase). 2. Within the first year: Assessments will be performed once monthly From Year 1 to Year 2: Assessments will be performed once every 3 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted31 Mar 2026
  2. Study start7 Mar 2026 (estimated)
  3. Primary completion6 Mar 2028 (estimated)
  4. Study completion6 Mar 2029 (estimated)

Resources

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 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
36 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07504289
Sponsor's protocol ID
2026-0335
Status last verified
Mar 2026
First submitted
18 Mar 2026
Last updated
31 Mar 2026
Results posted
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing CAR-NK in people with Light Chain Cardiac Amyloidosis, run by Second Affiliated Hospital, Zhejiang University, School of Medicine.

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 20 inclusion and 17 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: opening soon, as of the sponsor's last update on 31 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.

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 combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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?

Contact Yang Xu (0571-87783679, yxu@zju.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

safty and efficacy (time frame: Starting on Day 0 (CB CAR-NK-BCMA/CD19 infusion), participants will return to the outpatient clinic at the following intervals:• Days 1, 4, 7, 10, 14, 21, 28• Month 2 (±1 week)• Month 3 (±1 week)). 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?

On the ClinicalTrials.gov record for NCT07504289.

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 31 Mar 2026.

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