Safety and Efficacy of Umbilical Cord Blood-Derived Mesenchymal Stem Cells in the Treatment of Long-Term Cytopenia After CAR-T Therapy

NCT07212335 | Recruiting Early Phase 1 DMC

• 1 other identifier: IIT2025028
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

This study is a single arm, open label, exploratory clinical study aimed at evaluating the efficacy, and safety of allogeneic umbilical cord blood-derived mesenchymal stem cells in the treatment of long-term cytopenia after CAR-T therapy.

Conditions

Immune Effector Cell Associated Hematotoxicity

Keywords

ICAHT; hematotoxicity; CAR-T; cytopenia

Outcome Measures

Primary Outcomes (1)

• Number of Participants with Treatment-Related Adverse Events as Assessed by CTCAE v5.0

  From recruitment to 24 weeks after the last treatment.

Secondary Outcomes (5)

• Complete Response (CR) rate

  At Week 4 and Week 8 after the completion of MSC infusion

• Partial Response (PR) rate

  At Week 4 and Week 8 after the completion of MSC infusion

• Overall Response (OR) rate

  At Week 4 and Week 8 after the completion of MSC infusion

• Response time

  From the first MSC infusion to 8th week after the last MSC infusion

• Duration of Relief（DOR）

  from the date of achiving CR/PR to the earliest occurrence of hematopoietic indices falling below the CR/PR criteria or the end of the preset 24-week follow-up period after the first MSC infusion.

Study Arms (1)

MSC

EXPERIMENTAL

Mesenchymal stem cells: intravenous infusion, 2×10⁶ cells/kg body weight/week (once weekly), with 1 to 4 administrations based on different dosage groups.

Biological: Mesenchymal Stem Cell Infusion

Interventions

Mesenchymal Stem Cell Infusion BIOLOGICAL

Mesenchymal stem cells: intravenous infusion, 2×10⁶ cells/kg body weight/week (once weekly), with 1 to 4 administrations based on different dosage groups.

MSC

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients must meet all the following criteria to be enrolled in this study:
• Voluntarily participate in the study and sign the informed consent form;
• Aged ≥ 18 years, regardless of gender;
• Patients with acute lymphoblastic leukemia (ALL), lymphoma, or myeloma who still have severe cytopenia (meeting any of the following conditions: absolute neutrophil count ≤ 1×10⁹/L; platelet count ≤ 30×10⁹/L; hemoglobin ≤ 70 g/dL) 3 weeks after CAR-T cell infusion;
• ECOG performance status score ≤ 2;
• Estimated survival time ≥ 6 months;
• For female patients of childbearing potential, a negative pregnancy test result is required. Female patients of childbearing potential and male patients must use highly effective contraceptive measures during the study period and for 4 months/6 months after the discontinuation of treatment, respectively.

You may not qualify if:

• Patients with any of the following conditions are prohibited from enrolling in this study:
• Having received other anti-tumor treatments (including but not limited to chemotherapy, radiotherapy, targeted therapy, immunotherapy, or hematopoietic stem cell transplantation) that may affect the hematopoietic system or blood cell count within 1 month before the screening period after CAR-T cell infusion;
• Significant bone marrow infiltration by tumor cells during the screening period (for ALL: bone marrow morphological examination showing leukemia cell proportion \> 5%; for multiple myeloma (MM) and lymphoma: bone marrow flow cytometry showing positive minimal residual disease (MRD), or bone marrow pathological immunohistochemistry showing lymphoma/clonal plasma cell infiltration);
• Presence of any of the following conditions within 1 week before the first dose administration: infection with hemodynamic instability (requiring vasoactive drug support); deep fungal infection confirmed by imaging or microbiology (e.g., invasive aspergillosis, bloodstream infection, etc.); Pneumocystis jirovecii pneumonia, active tuberculosis, viremia (cytomegalovirus, parvovirus B19, etc.), and viral pneumonia (cytomegalovirus, COVID-19 virus, influenza virus, adenovirus, parainfluenza virus, etc.); as well as other severe infections that may affect hematopoiesis as judged by the investigator;
• Serum creatinine or blood urea nitrogen ≥ 1.5 times the upper limit of normal (ULN);
• Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≥ 3 times ULN; total bilirubin ≥ 1.5 times ULN;
• Other severe and/or uncontrolled diseases, or conditions that may affect study participation as judged by the investigator, including but not limited to: severe cardiac rhythm or conduction abnormalities (e.g., ventricular arrhythmias requiring clinical intervention, second-degree to third-degree atrioventricular block, etc.), corrected QT interval using Fridericia's formula (QTcF) \> 480 ms on electrocardiogram; New York Heart Association (NYHA) heart function classification of Grade Ⅲ-Ⅳ; uncontrolled diabetes mellitus (glycated hemoglobin \[HbA1c\] \> 9%); refractory hypertension; chronic obstructive pulmonary disease (forced expiratory volume in 1 second \[FEV1\] \< 50% of predicted value), etc.;
• History of arteriovenous thrombosis or atherosclerosis;
• Positive for anti-human immunodeficiency virus (HIV) antibody or anti-Treponema pallidum specific antibody; positive for hepatitis B surface antigen (HBsAg); or positive for hepatitis B core antibody with hepatitis B virus deoxyribonucleic acid (HBV-DNA) \> ULN; or hepatitis C virus ribonucleic acid (HCV-RNA) \> ULN;
• A history of or current malignant solid tumor (except cured non-invasive basal cell or squamous cell carcinoma of the skin and/or other cured carcinoma in situ; except other malignant tumors that have achieved clinical cure for \> 5 years with no recurrence within 5 years);
• Within 6 months after allogeneic hematopoietic stem cell transplantation, or donor cell chimerism rate ≤ 95%, or presence of active acute graft-versus-host disease (aGVHD) of Grade Ⅱ or higher, or moderate to severe chronic graft-versus-host disease (cGVHD);
• Having received a live vaccine within 4 weeks before the first dose administration, or planning to receive any live vaccine during the study period;
• Pregnant or lactating female patients;
• Patients with mental disorders;
• Participation in any other study drug trial (including vaccine trials) or exposure to other study drugs within 4 weeks or 5 half-lives (whichever is longer) before the first dose administration;

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead

Study Sites (1)

Chinese Academy of Medical Sciences Hospital of Hematology (Chinese Academy of Medical Sciences Institute of Hematology)

Tianjin, 300020, China

RECRUITING

MeSH Terms

Conditions

Cytopenia

Condition Hierarchy (Ancestors)

Hematologic Diseases; Hemic and Lymphatic Diseases

Central Study Contacts

Zhenzhen Wang

CONTACT

22-23608095; wangzhenzhen@ihcams.ac.cn

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: The study is divided into two phases: dose escalation phase and dose expansion phase.The dose escalation phase adopted a "3+3" design, with MTD (Maximum Tolerated Dose) determined by DLT (Dose-Limiting Toxicity) incidence. There are three groups: A, B, and C, with 3 subjects in each group. Group A: intravenous infusion of 2 × 10⁶ cells/kg body weight once; Group B: intravenous infusion of 2 × 10⁶ cells/kg body weight, once a week for 2 times; Group C: intravenous infusion of 2.0 × 10⁶ cells/kg body weight, once a week for 4 times. No DLT in three subjects means proceeding to the next higher dose group.One DLT in three subjects requires enrolling three more subjects.No DLT in these additions allows advancing to the higher dose group.The trial terminates when two or more DLTs occur in three or six subjects, with the current dose defined as MTD. In the dose expansion phase, investigators will select one specific dose test group (either A, B, or C) and expand the sample size by 6 subjects.

Study Record Dates

• First Submitted: September 30, 2025
• First Posted: October 8, 2025
• Study Start: October 1, 2025
• Primary Completion (Estimated): September 30, 2027
• Study Completion (Estimated): September 30, 2028
• Last Updated: February 11, 2026

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• Time Frame: 12 months to 36 months after study completion
• Access Criteria: Upon request to PI

Locations

CN (1)