RN1201injection for Relapsed/Refractory CD19+/BCMA+ Hematologic Malignancies

NCT ID: NCT07113496

Last Updated: 2025-08-13

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE1
• Total Enrollment: 27 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-08-31
• Study Completion Date: 2027-12-31

Brief Summary

This single-arm, dose-escalation exploratory trial evaluates the safety and efficacy of Allogeneic CAR-T (UCAR-T) cell therapy in patients with relapsed or refractory CD19+/BCMA+ hematologic malignancies, including those with minimal residual disease (MRD). Eligible patients will receive lymphodepletion followed by a single infusion of UCAR-T cells, either post-transplant or without transplantation depending on disease status. The trial assesses overall response and disease control rates, treatment-emergent adverse events, and in vivo behavior of UCAR-T cells.

Detailed Description

This single-arm, dose-escalation exploratory trial evaluates the safety and efficacy of Allogeneic CAR-T (UCAR-T) cell therapy in patients with relapsed or refractory CD19+/BCMA+ hematologic malignancies, including those with minimal residual disease (MRD). Eligible patients will receive lymphodepletion followed by a single infusion of UCAR-T cells, either post-transplant or without transplantation depending on disease status.Primary endpoints include treatment-emergent adverse events (TEAEs) and dose-limiting toxicities (DLTs). Secondary endpoints include objective response rate (ORR), disease control rate (DCR), pharmacokinetics, and pharmacodynamics of UCAR-T. This study aims to provide initial evidence for the safety and anti-tumor activity of UCAR-T in CD19+/BCMA+ hematologic malignancies.

Conditions

Relapsed or Refractory B-cell Hematologic Malignancies; B-cell Acute Lymphoblastic Leukemia (B-ALL); Multiple Myeloma (MM); Plasmablastic Lymphoma; Relapsed or Refractory CD19+/BCMA+ Hematologic Malignancies; Mature B-Cell Lymphoma

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Allogeneic CAR-T cell therapy

RN1201 cells injection will be infused via intravenously

Group Type: EXPERIMENTAL

Allogeneic CAR-T

Intervention Type: BIOLOGICAL

Patients will receive lymphodepletion chemotherapy followed by a single intravenous infusion of Allogeneic CAR-T cells. In select cases, CAR-T infusion may be administered post-autologous hematopoietic stem cell transplantation (auto-HSCT)

Interventions

Allogeneic CAR-T

Patients will receive lymphodepletion chemotherapy followed by a single intravenous infusion of Allogeneic CAR-T cells. In select cases, CAR-T infusion may be administered post-autologous hematopoietic stem cell transplantation (auto-HSCT)

Intervention Type: BIOLOGICAL

Eligibility Criteria

Inclusion Criteria

1. Voluntary participation with signed informed consent.

2. Pathologically confirmed CD19-positive and/or B-cell maturation antigen (BCMA)-positive hematologic malignancy according to the WHO 2017 classification, including but not limited to multiple myeloma, B-cell acute lymphoblastic leukemia (B-ALL), mature B-cell lymphomas, and plasmablastic lymphoma.

3. Relapsed/refractory disease defined as failure to achieve complete remission after standard therapy, or relapse after an initial response during treatment or follow-up.

4. Measurable disease required:

1. For B-ALL: persistent minimal residual disease (MRD) positivity despite hematologic remission.

2. For lymphoma: at least one measurable lesion ≥1.5 cm in longest diameter per IWG revised criteria.

3. For multiple myeloma: positive immunofixation electrophoresis or presence of extramedullary disease.

5. Age ≥18 years; both sexes eligible.

6. Expected survival ≥12 weeks.

7. Adequate organ function (exceptions for disease-related impairment are at the investigator's discretion):

1. Total bilirubin <2× upper limit of normal (ULN); serum creatinine <ULN; ALT and AST <3× ULN.

2. Absolute neutrophil count ≥0.5×10⁹/L; platelets ≥20×10⁹/L (no requirement if marrow involvement is documented).

3. Eastern Cooperative Oncology Group (ECOG) performance status 0-3.

4. Left ventricular ejection fraction (LVEF) ≥50%.

Exclusion Criteria

1. Known hypersensitivity, allergy, intolerance, or contraindication to CD19/BCMA-UCAR-T or any study drugs (fludarabine, cyclophosphamide, tocilizumab).

2. Genetic syndromes: Fanconi, Kostmann, Shwachman, or any documented bone-marrow failure syndrome.

3. Active or uncontrolled infection requiring IV antibiotics; evidence of severe active infection.

4. NYHA Class III or IV heart failure (unless clearly secondary to the underlying malignancy).

5. Central Nervous System (CNS) disorders unrelated to the primary hematologic malignancy.

6. Prior malignancy except adequately treated carcinoma in situ of skin, cervix, lung, or other non-active tumors.

7. Significant bleeding diathesis (e.g., gastrointestinal (GI) bleeding, coagulopathy, hypersplenism).

8. History of significant cardiac disease within the past 3 months that, in the investigator's judgment, renders the patient unable to tolerate study participation..

9. Pregnancy, lactation, or planned pregnancy within 6 months.

10. Any condition that, in the investigator's opinion, may increase risk or interfere with study results.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

The First Affiliated Hospital with Nanjing Medical University

OTHER

Sponsor Role: lead

Responsible Party

Lei Fan

Director of lymphoma center

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Lei Fan

Role: PRINCIPAL_INVESTIGATOR

The First Affiliated Hospital with Nanjing Medical University

Locations

The First Affiliated Hospital with Nanjing Medical University

Nanjing, Jiangsu, China

Countries

China

Central Contacts

Lei Fan

Role: CONTACT

fanlei@jsph.org.cn

086+025-68306124

Facility Contacts

Lei Fan

Role: primary

fanlei@jsph.org.cn

086+025-68306124

Other Identifiers

RN1201-Onc

Identifier Type: -

Identifier Source: org_study_id