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AL AmyloidosisPhase 2NCT07055724

Study of Eque-cel CAR-T Therapy in Newly Diagnosed Severe AL Amyloidosis

A Phase 2 study testing Eque-cel CAR-T Therapy in people with AL Amyloidosis, run by Nanjing IASO Biotechnology Co., Ltd..

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
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Trial summary

The goal of this clinical trial is to learn if Equecabtagene Autoleucel(Eque-cel), a Chimeric Antigen Receptor T-cell (CAR-T) therapy, works to treat severe Light Chain (AL) Amyloidosis in newly diagnosed adults with Mayo Stage IIIb. It will also learn about the safety and effects of Eque-cel. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
AL Amyloidosis
What is being tested?
Eque-cel CAR-T Therapy
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
17 participants
Where
No sites listed yet
Study dates
Jul 2025to Jun 2027
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

  • Subjects aged ≥18 years, regardless of gender.
  • Performance status: Eastern Cooperative Oncology Group (ECOG) performance status score ≤2.
  • Clinically diagnosed with newly diagnosed AL (light chain) amyloidosis, classified as Mayo Stage IIIb per the revised 2004 Mayo Clinic staging system at screening, with serum N-terminal pro-brain natriuretic peptide (NT-proBNP) >8500 ng/L and serum cardiac troponin T (cTnT) >0.035 μg/L or cardiac troponin I (cTnI) >0.01 g/L.
  • Presence of measurable hematologic disease at screening, defined as at least one of the following:
  • Difference in free light chains (dFLC) >4 mg/dL, or
  • Involved free light chain (iFLC) >4 mg/dL with abnormal kappa/lambda (κ/λ) ratio, or
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  • Serum protein electrophoresis (SPEP) M protein >0.5 g/dL.
  • Histopathological diagnosis of amyloidosis based on polarized light microscopy of Congo red-stained tissue specimens showing green birefringence, with confirmation of AL-derived amyloid deposits by at least one of the following methods:
  • Immunohistochemistry/immunofluorescence,
  • Mass spectrometry,
  • Electron microscopy for characteristic appearance/immunoelectron microscopy.
  • Presence of clonal plasma cells in bone marrow, detectable by light microscopy or flow cytometry.
  • Presence of M protein in blood or urine, with exclusion of multiple myeloma, Waldenström macroglobulinemia, or other lymphoplasmacytic proliferative disorders.
  • Organ involvement: At least one organ affected (kidney, heart, liver, nervous system, gastrointestinal tract, lung, soft tissue) as per consensus guidelines.
  • Expected survival time ≥12 weeks.
  • Subjects must have adequate organ function, meeting all of the following laboratory criteria prior to enrollment:
  • Complete blood count: Absolute neutrophil count (ANC) ≥1×10⁹/L; absolute lymphocyte count (ALC) ≥0.3×10⁹/L.
  • Hemoglobin >60 g/L (no red blood cell [RBC] transfusion within 7 days prior to testing; use of recombinant human erythropoietin allowed).
  • Liver function: Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤2.5× upper limit of normal (ULN); total serum bilirubin ≤1.5× ULN.
  • Renal function: Creatinine clearance (CrCl) ≥30 mL/min as calculated by the Cockcroft-Gault formula.
  • Coagulation function: Fibrinogen ≥1.0 g/L; activated partial thromboplastin time (APTT) ≤1.5× ULN; prothrombin time (PT) ≤1.5× ULN.
  • Blood oxygen saturation >91%.
  • Echocardiogram showing left ventricular ejection fraction (LVEF) ≥40%.
  • Systolic blood pressure (SBP) ≥90 mmHg (oral vasopressor support allowed).
  • Subjects and their spouses agree to use effective contraceptive methods (tools or medications) from the time of signing the informed consent form until one year after CAR-T cell infusion.
  • Subjects voluntarily sign the informed consent form (ICF).

Who cannot join

  • Subjects who have received any of the following treatments prior to enrollment:
  • Gene therapy before enrollment;
  • Live vaccine within 4 weeks before enrollment;
  • Other interventional clinical trial drugs within 12 weeks before leukapheresis.
  • Positive for hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) with detectable hepatitis B virus (HBV) DNA in peripheral blood; positive for hepatitis C virus (HCV) antibody with detectable HCV RNA in peripheral blood; positive for human immunodeficiency virus (HIV) antibody; positive for cytomegalovirus (CMV) DNA; positive for syphilis testing.
  • Use of therapeutic doses of corticosteroids (defined as prednisone or equivalent >20 mg/day) within 7 days before screening, though physiological replacement, topical, and inhaled steroids are permitted.
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  • Presence of uncontrolled active infection within 7 days before peripheral blood mononuclear cell (PBMC) collection (excluding genitourinary system infections and upper respiratory tract infections <CTCAE Grade 2).
  • Receipt of any systemic therapy for AL amyloidosis within 14 days before PBMC collection.
  • Undergoing major surgery within 14 days before PBMC collection, or planning surgery within 2 weeks after study treatment (subjects planning local anesthesia surgery are eligible to participate).
  • Unresolved non-hematologic toxicity from prior therapy to baseline or ≤Grade 1 (per NCI-CTCAE v5.0, excluding alopecia and Grade 2 peripheral neuropathy).
  • Known life-threatening allergic reaction, hypersensitivity, or intolerance to cellular products or their components.
  • Subjects with suspected or confirmed symptoms of central nervous system (CNS) involvement by plasma cell tumors.
  • Bone marrow plasma cells >30% with clinical symptoms of multiple myeloma accompanied by osteolytic bone lesions.
  • Severe active cardiovascular disease with significant clinical symptoms despite treatment, including but not limited to unstable angina, myocardial infarction, and severe arrhythmia (excluding those with implanted implantable cardioverter-defibrillator [ICD] or pacemaker).
  • Subjects with bleeding or severe thrombosis symptoms as judged by the investigator, or with genetic/acquired bleeding and severe thrombosis conditions (including hemophilia, coagulopathy, thrombocytopenia, splenomegaly), or currently receiving thrombolytic or anticoagulant therapy.
  • Subjects with hypertension that cannot be controlled by medication.
  • Subjects who have received solid organ transplantation.
  • Subjects with a history of central nervous system disorders (e.g., cerebral aneurysm, epilepsy, stroke, dementia, psychosis) or consciousness impairment.
  • Subjects with unstable systemic diseases as judged by the investigator, including but not limited to severe liver, kidney, or metabolic diseases requiring medical treatment.
  • Diagnosis of malignancy other than multiple myeloma within 5 years before screening, excluding adequately treated cervical carcinoma in situ, basal cell or squamous cell skin cancer, localized prostate cancer post-radical surgery, or ductal carcinoma in situ of the breast post-radical surgery.
  • Participation in another interventional clinical trial within 1 month before signing the ICF.
  • Women who are pregnant, breastfeeding, or planning to become pregnant.
  • Subjects who do not agree to or have not completed the signing of the ICF.
  • Other conditions deemed unsuitable for enrollment by the investigator.

Trial locations and contacts

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

Liu Peng contact

86-021-64041990

liu.peng@zs-hospital.sh.cn

Li Jing contact

86-021-64041990

About this study

Official title: Exploratory Clinical Study of Fully Human BCMA Chimeric Antigen Receptor Autologous T-cell Injection (Equecabtagene Autoleucel, Eque-cel) for the Treatment of Newly Diagnosed Mayo Stage IIIb AL Amyloidosis Patients

The goal of this clinical trial is to learn if Equecabtagene Autoleucel(Eque-cel), a Chimeric Antigen Receptor T-cell (CAR-T) therapy, works to treat severe Light Chain (AL) Amyloidosis in newly diagnosed adults with Mayo Stage IIIb. It will also learn about the safety and effects of Eque-cel. The main questions it aims to answer are:

Does Eque-cel lead to hematologic remission (achieving a very good partial response or better) in AL amyloidosis? How safe is Eque-cel for these patients, and what side effects might occur?

Read more

Participants will:

Undergo blood cell collection to create personalized Eque-cel therapy. Receive pre-treatment to prepare their body for the therapy (lymphodepletion). Receive a single infusion of Eque-cel. Be monitored closely for 24 weeks after infusion, followed by long-term checkups for up to 15 years.

Read the detailed description

This exploratory clinical trial investigates the efficacy and safety of Eque-cel , a fully human B-cell Maturation Antigen (BCMA)-targeted CAR-T cell therapy, in treating newly diagnosed Mayo Stage IIIb AL amyloidosis, a rare and severe systemic disorder characterized by abnormal plasma cell proliferation and amyloid fibril deposition leading to organ dysfunction. Current treatments, such as high-dose chemotherapy with stem cell transplantation or daratumumab-based regimens, fail to meet the urgent needs of Stage IIIb patients due to severe cardiac involvement, poor tolerability, and lack of approved standard therapies, necessitating rapid, low-cardiotoxicity interventions. Eque-cel, approved for relapsed/refractory multiple myeloma in China, has shown promise in prior studies with high hematologic response rates and rapid action, offering a potential solution for this high-risk population.

This study is an interventional clinical trial focused on treating newly diagnosed Mayo Stage IIIb AL Amyloidosis patients. It is a Phase II exploratory study aimed at initially validating the efficacy and safety of Eque-cel. The study employs a single-arm, open-label design, with all participants receiving Eque-cel CAR-T cell therapy (at a dose of 1.0×10⁶ CAR-T cells/kg), without randomization or blinding, to directly assess treatment outcomes. The trial is conducted at a single center (Zhongshan Hospital, Fudan University) and plans to enroll up to 17 participants. The process includes peripheral blood mononuclear cell (PBMC) collection, optional bridging therapy, lymphodepletion preconditioning, and cell infusion, followed by a 24-week primary follow-up period and long-term monitoring for up to 15 years. The study aims to complete patient enrollment within 2 years. The primary endpoint is to evaluate the hematologic response rate post-treatment, while secondary endpoints include safety and survival-related metrics. Through innovative CAR-T therapy, this study seeks to address unmet clinical needs and provide new treatment hope for this high-risk population.

Conditions
AL Amyloidosis
Being tested
Eque-cel CAR-T Therapy
Keywords
Eque-cel, CAR-T, Mayo Stage IIIb
Medical subject headings
Immunoglobulin Light-chain Amyloidosis

What participants receive

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

  • Eque-cel CAR-T TherapyBiological

    Participants in this arm receive a single infusion of Eque-cel at a dose of 1.0 x 10^6 CAR-T cells/kg following lymphodepletion preconditioning with fludarabine and cyclophosphamide. Eque-cel is a fully human BCMA-targeted CAR-T cell therapy designed to recognize and eliminate BCMA-expressing cells.

Study groups in NCT07055724
Study groupRoleWhat they receive
Eque-cel ArmExperimentalEque-cel : a dose of 1.0 x 10^6 CAR-T cells/kg
Receives: Biological: Eque-cel CAR-T Therapy

What is being measured

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

  1. Proportion of Patients Achieving Hematologic VGPR or Better by Day 90

    This measure assesses the effectiveness of Eque-cel CAR-T therapy by calculating the percentage of patients who achieve a Very Good Partial Response (VGPR) or better within 90 days post-infusion. Hematologic response indicates a significant reduction in AL amyloidosis blood markers, like abnormal light chains, based on international consensus guidelines. Blood samples are tested at Day 90 using lab methods such as serum free light chain (FLC) and protein electrophoresis (SPEP) to confirm VGPR, reflecting disease control in Mayo Stage IIIb patients.

    Measured: From Eque-cel infusion to Day 90 post-infusion

Secondary outcomes (5)

  1. Complete Hematologic Response Rate (CHR)

    The proportion and time to achieving complete hematologic response as defined by the international consensus criteria for amyloidosis.

    Measured: From Eque-cel infusion to 24 months

  2. Major Organ Deterioration-Progression-Free Survival (MOD-PFS)

    This composite endpoint is defined as the time from randomization to the first occurrence of any of the following events (whichever comes first)：

    1. Death;
    2. Clinical manifestation of heart failure: defined as dyspnea at rest (for at least 3 consecutive days) solely due to amyloid cardiomyopathy, or the need for heart transplantation, left ventricular assist device (LVAD), or intra-aortic balloon pump (IABP);
    3. Clinical manifestation of renal failure: defined as progression to end-stage renal disease (requiring hemodialysis or kidney transplantation); hematologic progression as per consensus criteria: from CHR, any detectable monoclonal protein or abnormal free light chain ratio (ratio must double); from PR, a 50% increase in serum M protein to >0.5 g/dL or a 50% increase in urine M protein to >200 mg/day (measurable peak required); a 50% increase in free light chains to >100 mg/L.

    Measured: From the time of Eque-cel infusion up to 24 months

  3. Progression-Free Survival (PFS)

    Defined as the time from screening/enrollment to the first documentation of hematologic disease progression, organ (cardiac, renal, or hepatic) progression, or death from any cause (whichever occurs first), based on laboratory results and determined by international consensus criteria.

    Measured: From the time of Eque-cel infusion up to 24 months

  4. Organ Response Rate (OrRR)

    The proportion of patients with baseline organ involvement achieving a response in each respective organ within 24 months.

    Measured: From the time of Eque-cel infusion up to 24 months

  5. Overall Survival (OS)

    Defined as the time from screening/enrollment to the date of death from any cause.

    Measured: From the time of Eque-cel infusion up to 24 months

Timeline

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

  1. First posted9 Jul 2025
  2. Study startJul 2025 (estimated)
  3. Primary completionJun 2027 (estimated)
  4. Study completionJun 2027 (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 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
17 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07055724
Sponsor's protocol ID
CT103ACI003
Status last verified
Jul 2025
First submitted
26 Jun 2025
Last updated
9 Jul 2025
Results posted
No
Data monitoring committee
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 2 study testing Eque-cel CAR-T Therapy in people with AL Amyloidosis, run by Nanjing IASO Biotechnology Co., Ltd..

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 26 inclusion and 24 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 9 Jul 2025. 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 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?

Contact Liu Peng (86-021-64041990, liu.peng@zs-hospital.sh.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Proportion of Patients Achieving Hematologic VGPR or Better by Day 90 (time frame: From Eque-cel infusion to Day 90 post-infusion). 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 NCT07055724.

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 9 Jul 2025; AzTrials last refreshed its copy on 26 Oct 2026.

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