In vivo CAR-T therapy for relapsed or refractory B‑cell leukemias, lymphomas, and multiple myeloma

Intracellularly Prepared Chimeric Antigen Receptor T-cell Therapy Targeting CD19 for the Treatment of Relapsed/Refractory Hematological Malignancies

This trial will try a single in‑body (intravenous) CAR‑T infusion to treat adults with relapsed or refractory B‑cell acute lymphoblastic leukemia, B‑cell non‑Hodgkin lymphoma, or multiple myeloma.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 24 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Chongqing Precision Biotech Co., Ltd (Industry-sponsored)
• Drugs / interventions: chemotherapy, immunotherapy, CAR-T, Chimeric Antigen Receptor
• Locations: 1 site (Kunming, Yunnan)
• Trial ID: NCT07239323 on ClinicalTrials.gov

What this trial studies

This open-label, single-arm Phase 1 study gives adults with relapsed or refractory B-ALL, B‑cell non‑Hodgkin lymphoma, or multiple myeloma a single intravenous dose of an in vivo CAR‑T preparation. Participants will be hospitalized for observation after infusion and monitored closely for safety, tolerability, and early signs of anti-tumor activity. Laboratory values, organ function, and disease status will be followed during screening and at scheduled visits, with adverse events recorded. Subjects will be followed for up to two years to determine durability of disease control and late effects.

Who should consider this trial

Good fit: Adults (≥18 years) with confirmed relapsed or refractory B‑ALL, B‑cell lymphoma, or multiple myeloma who meet the study's blood count, renal function, and ECOG 0–2 criteria are appropriate candidates.

Not a fit: Patients with inadequate blood counts or renal function below the protocol thresholds, active uncontrolled infections, or who cannot commit to hospitalization and long‑term follow‑up are unlikely to benefit.

Why it matters

Potential benefit: If successful, this approach could offer a faster, off‑the‑shelf CAR‑T option that reduces the time and complexity of current manufactured CAR‑T therapies.

How similar studies have performed: Most approved CAR‑T therapies to date use ex vivo manufactured cells; in vivo CAR‑T delivery is a novel approach with limited early clinical data and mainly preclinical support.

Eligibility criteria

Inclusion Criteria:

1. Age ≥ 18 years old, gender unrestricted;
2. Confirmed diagnosis of relapsed/refractory malignant hematological tumors, including B-ALL, B-cell lymphoma and multiple myeloma;
3. ECOG performance status score 0-2, with an expected survival period of ≥ 3 months;
4. Blood routine test results during the screening period meet the following criteria:

   ① Hemoglobin ≥ 6 g/dL (no red blood cell transfusion within 1 week before screening), recombinant human erythropoietin (rhEPO) is allowed; for patients meeting the hemoglobin ≥ 6 g/dL criterion, red blood cell transfusion can be used to maintain hemoglobin ≥ 6 g/dL;
   * Absolute neutrophil count (ANC) ≥ 600/μL (no use of granulocyte colony-stimulating factor \[G-CSF\] within 1 week before screening, or no use of pegylated G-CSF within 2 weeks before screening); ③ Platelet count ≥ 50,000/μL; ④ Lymphocyte count ≥ 500/μL;
5. Normal renal function during the screening period: creatinine clearance rate (CrCl) ≥ 45 mL/min (calculated using the Cockcroft-Gault formula);
6. Liver function during the screening period meets the following criteria:

   ① Alanine aminotransferase (ALT), aspartate aminotransferase (AST) ≤ 3.0 × ULN;

   ② Total bilirubin (TBIL) ≤ 2.0 × ULN (except for congenital hyperbilirubinemia such as Gilbert's syndrome, direct bilirubin can be relaxed to ≤ 1.5 × ULN);
7. Cardiac function during the screening period meets the following criteria:

   ① Left ventricular ejection fraction (LVEF) ≥ 40% (measured by echocardiography or MUGA scan);

   ② No clinically significant pericardial effusion;

   ③ No clinically significant electrocardiogram (ECG) abnormalities;
8. Pulmonary function during the screening period meets the following criteria: blood oxygen saturation (SpO₂) ≥ 90%;
9. Women of childbearing age must have a negative pregnancy test during the screening period and before drug administration, and must not be in the lactation period;
10. Men and women of childbearing age must agree to take effective contraceptive measures and not donate reproductive cells (including sperm or eggs) from the time of signing the informed consent form until 1 year after the end of study drug administration;
11. The subject or their legally authorized representative has signed the informed consent form (ICF), indicating their understanding of the purpose and procedures of the study and their voluntary participation in this study.

Exclusion Criteria:

1. Other anti-tumor treatments within the screening period (judged by the investigator comprehensively):

   ① Received chemotherapy, targeted therapy or immunotherapy within 5 half-lives before administration;

   ② Received radiotherapy within 4 weeks before administration (if the radiotherapy target area covers ≤ 5% of bone marrow reserve, the time limit for radiotherapy completion is not restricted);
2. History of hematopoietic stem cell transplantation: Received allogeneic or autologous hematopoietic stem cell transplantation within 3 months before administration;
3. History of other malignant tumors (except for this disease), except for the following situations:

   ① Received radical treatment and had no known active disease for ≥ 2 years before enrollment;

   ② Had fully treated non-melanoma skin cancer in the past and had no active lesions at present;
4. Received treatment related to vesicular stomatitis virus glycoprotein (VSVG) pseudotyped virus in the past;
5. Had severe and uncontrolled infections (bacterial, viral, fungal, etc.) within the screening period;
6. Clinically significant cardiac diseases:

   * Had symptomatic heart failure or other serious cardiac diseases (such as severe arrhythmia);

     * Had New York Heart Association (NYHA) Class III-IV congestive heart failure; ③ Had a myocardial infarction or received coronary artery bypass grafting (CABG) / coronary artery stent implantation within 6 months before signing the informed consent;

       * Had clinically significant ventricular arrhythmia or a history of unexplained syncope; ⑤ Had a history of syncope (excluding cases caused by vasovagal reactions or dehydration); ⑥ Had a history of severe non-ischemic cardiomyopathy;
7. Other clinically significant diseases, including but not limited to:

   * Primary immunodeficiency; ② Had a stroke or seizure within 6 months before screening;

     * Had clear clinical evidence of dementia or mental status changes; ④ Had Parkinson's disease, Parkinson-like movement disorders or a history of the above;
8. Had undergone surgery within 2 weeks before administration, or planned to undergo surgery within 2 weeks after administration (local anesthesia surgery excluded);
9. Had received live attenuated vaccines within 1 month before administration;
10. Had a history of severe allergic reactions to this product or its formulation components;
11. Patients who were not suitable for establishing intravenous access;
12. The investigator believed that there were other conditions that made the patient unsuitable for participating in this study.

Where this trial is running

Kunming, Yunnan

• 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China — Kunming, Yunnan, China (Recruiting)

Study contacts

• Principal investigator: Wang Sanbin, MD — 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
• Study coordinator: Wang Sanbin, PhD

Conditions B-Acute Lymphoblastic Leukemia; B Cell Non-Hodgkin's Lymphoma; Multiple Myeloma; in Vivo; CAR-T; malignant hematological tumors