ClinicalTrials.gov record NCT07626476
Autologous BCMA-targeted CAR-T Cell Injection for Relapsed/Refractory Light Chain Amyloidosis
An Exploratory Clinical Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Autologous BCMA-targeted CAR-T Cell Injection in Participants With Relapsed/Refractory Light Chain Amyloidosis
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 open-label study gives one fixed-dose infusion of autologous BCMA-targeted CAR-T cells to adults with relapsed or refractory light-chain (AL) amyloidosis. Before infusion participants undergo blood collection for cell manufacture and short-course lymphodepleting chemotherapy; the CAR-T dose is 1.0×10^6 CAR-T cells/kg. Participants are followed closely for safety and response, with a main follow-up up to 2 years and longer-term visits afterwards.
What it is studying. The study is evaluating the safety profile, early signs of anti-disease activity (efficacy), and in‑body behavior of a one-time intravenous infusion of the participant's own BCMA-targeted CAR-T cells. It will also measure pharmacokinetics, pharmacodynamics and immune responses after infusion.
What the researchers hope to learn. To evaluate safety, preliminarily assess efficacy, and explore pharmacokinetics, pharmacodynamics and immunogenicity of autologous BCMA-targeted CAR-T cells in relapsed/refractory AL amyloidosis.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
Systemic light chain amyloidosis (AL amyloidosis) is the most common type of systemic amyloidosis, with diverse clinical manifestations and difficulties in diagnosis and treatment. AL amyloidosis may involve multiple organs; the kidney and heart are the most commonly involved organs. The treatment goal is to reduce monoclonal immunoglobulin light-chain levels, prevent further amyloid deposition in important organs, and alleviate or reverse organ dysfunction caused by amyloid deposition. The principal approach to achieve this goal is to eliminate the plasma-cell or B-cell clones producing abnormal light chains. For patients with relapsed/refractory AL amyloidosis, the protocol states that there is currently no suitable treatment method and that participation in clinical trials is recommended. This study evaluates targeted BCMA autologous CART cell injection in participants with relapsed/refractory light chain amyloidosis. The main purpose is to evaluate safety, preliminarily verify efficacy, and explore in vivo pharmacokinetics, pharmacodynamics, immunogenicity and related characteristics after infusion.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Biological
- Targeted BCMA Autologous CART Cell Injection
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- The participant must personally sign an ethics-committee-approved informed consent form before study start.
- Age ≥18 years.
- Pathologically confirmed light chain amyloidosis.
- Relapsed/refractory light chain amyloidosis previously treated with 2 or more lines of therapy.
- dFLC >50 mg/L.
- Expected survival ≥12 weeks.
- ECOG score ≤2.
- Diagnosis of AL amyloidosis must meet the following conditions: (1) clinical manifestations, physical examination, laboratory or imaging examinations confirm tissue or organ involvement; (2) tissue biopsy pathology confirms amyloid deposition, and the precursor protein of amyloid protein is immunoglobulin light chain or heavy and light chain; and the participant is relapsed/refractory.
- Female participants of childbearing potential should agree to use effective contraception from the date of signing informed consent until 365 days after infusion. Effective contraception is defined as abstinence or contraception using methods specified in the protocol with an annual failure rate <1%.
- Adequate organ function before enrollment, meeting all of the following:
- Absolute neutrophil count ≥1.0×10\^9/L; granulocyte colony-stimulating factor (G-CSF) support is permitted.
- Platelet count ≥50×10\^9/L.
- Hemoglobin ≥8 g/dL.
- Bilirubin ≤1.5×upper limit of normal (ULN), except biliary obstruction caused by tumor compression.
- ALT or AST ≤2.5×ULN; for participants with liver involvement, ≤5×ULN.
- Mayo 2004 stage I-IIIa.
- Stable coagulation function: INR ≤1.5 and APTT ≤1.2×ULN, except tumor-related anticoagulant therapy.
- Baseline oxygen saturation on room air >92%.
Exclusion criteria
- Participants who have received the following prior treatments:
- Prior gene therapy before enrollment.
- Live vaccine injection within 4 weeks before enrollment.
- Other interventional clinical study drug treatment within 12 weeks before leukapheresis.
- Central nervous system involvement or complete intestinal obstruction.
- Moderate or higher pleural effusion or ascites that is difficult to control with conventional treatment and requires continuous catheter drainage.
- Active malignancy within the past 5 years, unless it is a curable tumor and has been clearly cured.
- HBsAg positivity with abnormal peripheral blood HBV DNA testing; HCV antibody positivity with peripheral blood HCV RNA positivity; HIV antibody positivity; CMV DNA positivity; or positive syphilis RPR test.
- Uncontrolled active infection, except CTCAE grade <2 urogenital infection and upper respiratory tract infection.
- Severe heart disease, including but not limited to unstable angina, myocardial infarction within 6 months before screening, congestive heart failure (New York Heart Association [NYHA] class ≥III), positive six-minute walk test, interventricular septum and left ventricular posterior wall thickness >1.5 cm, or ventricular arrhythmia and atrioventricular block indicated by ambulatory electrocardiography.
- Hypertension that cannot be controlled by medication.
- Toxicity from prior treatment not recovered to baseline or ≤grade 1 according to NCI CTCAE v5.0, except alopecia and clinically insignificant laboratory abnormalities.
- Major surgery within 2 weeks before enrollment, or planned surgery during the waiting period before infusion or within 12 weeks after study treatment, except planned local anesthesia surgery.
- Solid organ transplantation.
- Pregnant or breastfeeding women.
- History of central nervous system disease, such as cerebral aneurysm, epilepsy, stroke, senile dementia or psychiatric disease, or disturbance of consciousness.
- Other systemic disease judged unstable by the investigator, including but not limited to severe hepatic, renal or metabolic disease requiring medication.
- Known life-threatening allergic reaction, hypersensitivity or intolerance to the CAR-T cell product or its components.
- Bleeding or severe thrombosis as judged by the investigator, hereditary/acquired bleeding or severe thrombosis, including hemophilia, coagulopathy, thrombocytopenia and hypersplenism, or current thrombolytic or anticoagulant therapy.
- Other conditions that the investigator considers unsuitable for enrollment.
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Show the detailed description from the registry
This is an open-label, fixed-dose exploratory clinical study to observe the safety and efficacy of targeted BCMA autologous CART cell injection in participants with relapsed/refractory light chain amyloidosis. The study is expected to enroll 30 participants and uses a fixed dose of 1.0×10\^6 CAR-T/kg. The safety observation period is 28 days after CAR-T administration. The study process includes screening, PBMC leukapheresis, pre-lymphodepletion examination, lymphodepleting conditioning, infusion of targeted BCMA autologous CART cell injection with the infusion day defined as Day 0, and the main follow-up period. Before infusion, participants receive fludarabine plus cyclophosphamide lymphodepleting conditioning from Day -5 to Day -3 for three consecutive days. The recommended regimen is cyclophosphamide 250 mg/m²/day by intravenous infusion once daily for three days and fludarabine 25 mg/m²/day by intravenous infusion once daily for three days. The investigator may adjust the conditioning regimen according to renal function and creatinine clearance, such as replacing fludarabine with bendamustine or reducing the dose. The targeted BCMA autologous CART cell injection is infused once on Day 0 by intravenous injection and completed within 30 minutes. If an adverse event occurs after conditioning and before infusion and the investigator determines that infusion should be suspended, infusion may be delayed until the adverse event recovers to a state allowing infusion. Before infusion, the investigator should reassess whether repeated lymphodepleting conditioning is needed. The main follow-up period lasts from infusion of targeted BCMA autologous CART cell injection until the participant meets the criteria for exit from main follow-up or completes the 2-year main follow-up visit at Day 720. Efficacy assessments are performed at Day 28, Day 60 and Day 90, and then every 90 days until the end of main follow-up. Participants who complete the 2-year main follow-up enter long-term follow-up. Participants who meet criteria to exit main follow-up enter long-term visits until death, withdrawal from the study, or 15 years after cell infusion.
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.
After informed consent and screening, participants undergo leukapheresis to collect their own immune cells. They then receive lymphodepleting chemotherapy (typically fludarabine plus cyclophosphamide for 3 days, with dose adjustments possible) followed by a single intravenous infusion of BCMA-targeted autologous CAR‑T cells at a fixed dose. There is close safety monitoring (including a 28-day safety observation) and scheduled follow-up visits for blood tests and efficacy assessments at Day 28, Day 60, Day 90 and then every 90 days up to a 2-year main follow-up; long-term follow-up may continue per protocol. Infusion may be delayed if an adverse event occurs after conditioning, and conditioning can be adjusted for kidney function.
How the study is structured. An interventional, open-label single-group study. Participants give blood for leukapheresis to make autologous CAR-T cells, receive a short course of lymphodepleting chemotherapy (commonly fludarabine plus cyclophosphamide, with possible adjustments), then receive a single IV infusion of BCMA-targeted CAR-T at a fixed dose of 1.0×10^6 CAR-T/kg. Safety and efficacy assessments occur at set visits (early checks at Day 28, Day 60, Day 90, then about every 90 days) during a main follow-up of up to 2 years and longer-term monitoring thereafter.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
- The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on AL amyloidosis
Important dates
- Study start
- Dec 16, 2024
- Primary completion
- Dec 15, 2027 (estimated)
- Study completion
- Dec 15, 2029 (estimated)
- Target enrollment
- 30 participants (estimated)
- Last updated on registry
- Jun 4, 2026
- 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
- Beijing Boren Hospital
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.
- Yajing Zhang 📞 +86 18501333856 ✉ yajing_cart66@126.com
Participating sites (1)
- Beijing GoBroad Boren HospitalRecruiting
Beijing, Fengtai District, 100070 · China
Terms used on this page · explanations by MedicalUpdates.ai
- 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.
- pharmacokinetics
- How the body absorbs, distributes, breaks down and removes a drug. It is studied by measuring drug levels or related substances at set times after a dose.
- pharmacodynamics
- What a drug does to the body, for example how it changes a blood marker or a symptom, and how that relates to the dose.
- 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.
- lines of 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.
- 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.
- ECOG score
- 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.
- biopsy
- Removal of a small piece of tissue so it can be examined under a microscope. 'Biopsy-proven' or 'histologically confirmed' means the diagnosis was confirmed by examining tissue under a microscope.
- childbearing potential
- Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
- 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.
- Baseline
- Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
- study drug
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
- before screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- six-minute walk test
- A test of how far a person can walk on a flat surface in six minutes. It is used to measure stamina and to track change over time.
- CAR-T
- 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.
- 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.
- open-label
- No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
- follow-up
- The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
- creatinine clearance
- A measure of kidney function, calculated from a blood creatinine level together with age, weight and sex.
- adverse event
- Any unwanted medical problem that happens during a study, whether or not it was caused by the treatment. A 'serious' adverse event is one that meets specific criteria, such as causing death, being life-threatening, requiring or prolonging hospital admission, causing significant disability, or another medically important outcome.
- long-term follow-up
- Observation that continues for years after the study treatment, often required for gene and cell therapies.
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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