Light Chain AmyloidosisNCT06983951
Exploratory Clinical Study on the Safety and Efficacy of Targeted BCMA Autologous CART Cell Injection in Subjects With Recurrent/Refractory Light Chain Amyloidosis
A study testing BCMA CART in people with Light Chain Amyloidosis, run by Beijing Boren Hospital at 1 site in China.
Trial summary
This study is a multicenter, open label, fixed dose exploratory clinical trial with an expected enrollment of 30 subjects. The main objective is to evaluate the safety of targeted BCMA autologous CART cell injection in the treatment of recurrent or refractory light chain amyloidosis in subjects, preliminarily verify… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Light Chain Amyloidosis
- What is being tested?
- BCMA CART
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteChina
- Study dates
- 20 Feb 2025to 30 Sep 2027
- Phase
- Interventional
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
- Participants must meet all inclusion criteria, and only those who do not meet any exclusion criteria can be enrolled
- Participants must personally sign the informed consent form approved by the ethics committee in writing before the start of the study;
- The age of the subject is ≥ 18 years old;
- Diagnosed with light chain amyloidosis through pathological examination;
- Subjects with recurrent/refractory light chain amyloidosis who have previously received second-line or higher treatment;
- dFLC > 50mg/L
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- Expected survival period ≥ 12 weeks;
- ECOG score ≤ 2 points;
- Female subjects with fertility should agree to take effective contraceptive measures from the date of signing the informed consent form until 365 days after reinfusion. Effective contraceptive measures are defined as abstinence or using contraceptive methods with an annual failure rate of less than 1% as specified in the plan;
- Prior to enrollment, participants must have appropriate organ function and meet all of the following test results:
- 9.1 Absolute neutrophil count ≥ 1.0 × 109/L [allowed to use granulocyte colony-stimulating factor (G-CSF) support]; 9.2 Platelet count ≥ 50 × 109/L; 9.3 Hemoglobin ≥ 8 g/dl; 9.4 Bilirubin value ≤ 1.5 x upper limit of normal (ULN); 9.5 ALT or AST ≤ 2.5 times the upper limit of normal (ULN) (with liver involvement ≤ 5 times the upper limit of normal); 9.6 Mayo 2004 Stage I-IIIa participants; 9.7 Stable coagulation function: INR ≤ 1.5, APTT ≤ 1.2 x upper limit of normal (ULN); Basic blood oxygen saturation is greater than 92% in indoor natural air environment.
Who cannot join
- -
- Subjects who meet any of the following criteria will be excluded:
- Subjects who have received the following previous treatments:
- 1.1 Individuals who have received gene therapy prior to enrollment; 1.2 Subjects who received live vaccines within 4 weeks prior to enrollment; 1.3 Received other intervention clinical drug treatments within 12 weeks prior to single collection;
- Patients with central involvement or complete intestinal obstruction;
- Patients with moderate to severe pleural and peritoneal effusion who are difficult to control with conventional treatment and require continuous catheterization and drainage;
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- Active malignant tumors within the past 5 years, unless they are curable tumors that have been significantly cured, such as basal or squamous cell carcinoma, cervical or breast carcinoma in situ, etc;
- Subjects with positive hepatitis B B surface antigen (HBsAg) and abnormal detection of HBV DNA in peripheral blood (abnormal detection of HBV DNA is defined as: quantitative detection of HBV DNA is higher than the detection limit of the testing center or higher than the normal reference value range of the testing center or positive qualitative detection of HBV DNA); Individuals with positive hepatitis C virus (HCV) antibodies and positive hepatitis C virus (HCV) RNA in peripheral blood; Individuals who are HIV antibody positive; Individuals who test positive for Cytomegalovirus (CMV) DNA; Positive RPR results in syphilis testing;
- There are uncontrollable active infections (excluding CTCAE grade 2 urinary and reproductive system infections and upper respiratory tract infections);
- Severe heart disease: including but not limited to unstable angina, myocardial infarction (within 6 months before screening), congestive heart failure (NYHA classification ≥ III), 24-hour dynamic electrocardiogram showing ventricular arrhythmia and atrioventricular block, positive six minute walk test, interventricular septum and left ventricular posterior wall thickness>1.5cm;
- Hypertensive subjects who cannot be controlled by drug treatment;
- Previous treatment toxicity reactions have not improved to baseline or ≤ grade 1 (NCI-CTCAE v5.0 version, except for hair loss and clinically insignificant laboratory test abnormalities);
- Have undergone major surgery within 2 weeks prior to enrollment, or plan to undergo surgery during the waiting period for reinfusion or within 12 weeks after receiving study treatment (excluding planned local anesthesia surgery);
- Solid organ transplant recipients;
- Pregnant or lactating women;
- Subjects with previous central nervous system disorders (such as cerebral aneurysms, epilepsy, stroke, senile dementia, mental illness, etc.) or consciousness disorders;
- Other researchers have identified unstable systemic diseases, including but not limited to severe liver, kidney, or metabolic diseases that require drug treatment;
- Known to have life-threatening allergic reactions, hypersensitivity reactions, or intolerance to cellular preparations or their components;
- Patients diagnosed by researchers as having bleeding, severe thrombosis, or genetic/acquired bleeding and severe thrombosis (including hemophilia, coagulation dysfunction, thrombocytopenia, splenomegaly, etc.), or patients undergoing thrombolytic or anticoagulant therapy; Researchers believe that there are other situations that are not suitable for inclusion.
Trial locations and contacts
1 site in 1 country.
China1 site
Beijing Gobroad Brond Hospital
Beijing, Beijing Municipality, 100070
About this study
Official title: Exploratory Clinical Study Evaluating the Safety and Efficacy of Targeted BCMA Autologous CART Cell Injection in Subjects With Recurrent/Refractory Light Chain Amyloidosis
This study is a multicenter, open label, fixed dose exploratory clinical trial with an expected enrollment of 30 subjects. The main objective is to evaluate the safety of targeted BCMA autologous CART cell injection in the treatment of recurrent or refractory light chain amyloidosis in subjects, preliminarily verify the effectiveness of targeted BCMA autologous CART cell injection in the treatment of recurrent or refractory light chain amyloidosis in subjects, and explore the pharmacokinetic, pharmacodynamic, and immunogenic characteristics of targeted BCMA autologous CART cell injection after reinfusion.
- Conditions
- Light Chain Amyloidosis
- Being tested
- BCMA CART
- 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.
BCMA CARTGenetic
The cell product used in this study is a targeted BCMA autologous CART cell injection.
Research drug name: Targeted BCMA Autologous CART Cell Injection Drug code: BJBR-BCMA-006 Administration method: single intravenous injection
| Study group | Role | What they receive |
|---|---|---|
| BCMA CART | Experimental | Not described Receives: Genetic: BCMA CART |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency Of AES, SAES
Measured: 0 day to 24 months after treatment
Dose Limiting Toxicity
Measured: 0~28 day after treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 May 2025
- Study start20 Feb 2025
- Primary completion30 Sep 2027 (estimated)
- Study completion30 Sep 2027 (estimated)
Why it stopped: Due to the promulgation of State Council Order No. 818, clinical trials of this category are prohibited.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Light Chain AmyloidosisStudies 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Beijing Boren Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- yajing zhang, MD/PhD, Principal investigator, Beijing Gaobo Boren Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06983951
- Sponsor's protocol ID
- BRYY-IIT-LCYJ-2024-025
- Status last verified
- Feb 2025
- First submitted
- 19 Feb 2025
- Last updated
- 13 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No