ClinicalTrials.gov record NCT07055724

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

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 17, 2026

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

StatusNot yet recruitingThe study has been registered but has not started enrolling. Sites and contacts may not be listed yet.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 2A study that tests whether a treatment has the intended effect and continues to check safety, usually in a larger group than phase 1.
Age18 years old and older
SexAll
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This Phase II, single-arm study gives all participants a single infusion of Eque-cel CAR‑T cells after collecting their blood cells and giving short lymphodepletion. It seeks to measure hematologic response and safety in newly diagnosed Mayo Stage IIIb AL amyloidosis. Participants are monitored closely for 24 weeks after infusion and followed for long-term outcomes for up to 15 years. The trial plans to enroll up to 17 people.

What it is studying. The study tests whether one dose of Eque-cel (a BCMA-targeted CAR‑T cell therapy) produces a hematologic very good partial response (VGPR) or better by Day 90, and it also measures safety, organ responses, progression-free and overall survival.

What the researchers hope to learn. To measure the proportion of patients achieving hematologic VGPR or better by Day 90 after Eque-cel infusion; to assess safety and side effects; to evaluate organ response rates, progression-free survival, and overall survival over the follow-up period.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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? 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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Biological
  • Eque-cel CAR-T Therapy

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • 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
  • 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).

Exclusion criteria

  • 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.
  • 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.

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Show the detailed description from the registry

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.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants provide blood cells via leukapheresis for manufacturing their personalized CAR‑T product, may receive optional bridging therapy if needed, get short-course lymphodepleting chemotherapy (fludarabine and cyclophosphamide), and receive a single infusion of Eque‑cel CAR‑T. They will be monitored closely for at least 24 weeks after infusion (early safety and response checks) and then followed by long-term checkups for up to 15 years. Monitoring includes regular clinical assessments and laboratory/organ tests per the protocol. Participants must follow contraception requirements and complete informed consent.

How the study is structured. Interventional Phase II, open-label, single-group study. All enrolled participants have their blood cells collected to manufacture personalized Eque-cel, may receive short bridging therapy if needed, get lymphodepletion medicines, then receive one infusion of Eque-cel at the study dose. Participants undergo close monitoring for adverse events and response for 24 weeks, then periodic long-term follow-up (up to 15 years). The study is planned as a single-center trial with up to 17 participants.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The registry says recruitment has not started. When is it expected to open, and can I register interest now?
  • No sites are listed on the registry yet. Where will the study take place?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on AL amyloidosis

Clinical trials69 trials listed for AL amyloidosisEvery trial the registry lists for AL amyloidosis, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in AL amyloidosis researchLatest monthly update: September 2026. Includes: Daratumumab alone. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
July 2025 (estimated)
Primary completion
June 2027 (estimated)
Study completion
June 2027 (estimated)
Target enrollment
17 participants (estimated)
Last updated on registry
Jul 9, 2025
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Nanjing IASO Biotechnology Co., Ltd.

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites

Study locations have not been posted on ClinicalTrials.gov yet. This study is not yet recruiting, so sites may be added when recruitment begins. The central study contact above may be able to tell you about planned sites.

Terms used on this page · explanations by MedicalUpdates.ai

Chimeric Antigen Receptor
A treatment in which a person's own immune cells (T cells) are collected, modified in a laboratory to recognise a target, and returned to the body.
very good partial response
A response category used in some blood conditions meaning the disease marker has fallen a great deal but not disappeared. The exact definition depends on the condition and the study.
Performance status
A scale from 0 to 5 that describes how a person's condition affects daily activity. 0 means fully active; 1 means restricted in strenuous activity; 2 means able to self-care but unable to work; higher numbers mean more limitation.
at screening
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
free light chains
Small pieces of antibody protein made by plasma cells and measured in a blood test. In some conditions, such as AL amyloidosis, abnormal free light chains are the source of the disease.
dFLC
The difference between the abnormal ('involved') and normal ('uninvolved') free light chain levels in the blood. Used in some conditions to decide whether disease is measurable and to track response.
enrollment
The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
Absolute neutrophil count
The number of neutrophils, a type of white blood cell that fights infection, in a blood sample. Studies often require it to be above a set level.
upper limit of normal
The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
Creatinine clearance
A measure of kidney function, calculated from a blood creatinine level together with age, weight and sex.
Echocardiogram
An ultrasound scan of the heart. A transesophageal echocardiogram is done through a probe passed into the food pipe for a closer view.
informed consent form
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
corticosteroids
Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
prior therapy
Treatment a person has already had for the condition. A 'line of therapy' is one course of treatment; studies often require, or limit, the number of prior lines.
baseline
Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
relapsed/refractory
A condition that has not responded to a treatment. 'Relapsed' means it responded and then came back; 'refractory' means it did not respond in the first place.
single-arm
All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
open-label
No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
randomization
Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
blinding
Who does not know which intervention a participant is receiving. It can apply to participants, the people giving the treatment, those assessing results, or those analysing the data.
follow-up
The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
primary endpoint
The main result the study is designed to measure and report on. Other results, called secondary outcomes, are also collected.
endpoints
Something the study measures to judge its results, such as a symptom score, a blood test or survival. Outcomes are grouped as primary, secondary and sometimes exploratory.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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