Phase 1 Study of Chemotherapy Plus HLA-mismatched GPBMC Infusion Bridging to Allo-HSCT for R/R Leukemia

NCT07297173 | Recruiting Phase 1

• 1 other identifier: Allo-GPBMC to Allo-HSCT
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

Brief Summary

This is a Phase 1 clinical study designed to evaluate the safety, tolerability, and preliminary efficacy of chemotherapy combining with HLA-mismatched G-CSF mobilized peripheral blood mononuclear cell (GPBMC) infusion as a bridging therapy to allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients with relapsed and refractory (R/R) leukemia.

Conditions

Relapsed Leukemia; Refractory Leukemia

Keywords

HLA-mismatched GPBMC; allo-HSCT; relapsed leukemia; refractory leukemia

Outcome Measures

Primary Outcomes (3)

• Graft-Versus-Host Disease (GVHD)

  Including acute GVHD (aGVHD) and chronic GVHD (cGVHD), assessed per international universal criteria (e.g., Glucksberg classification).

  Measured up to 2 years after the last participant is enrolled

• Infections

  Covering bacterial, viral, fungal infections (e.g., pneumonia, sepsis, cytomegalovirus infection), confirmed based on clinical symptoms, laboratory tests, and etiological evidence.

  Measured up to 2 years after the last participant is enrolled

• Hematological Toxicity

  Including neutropenia, thrombocytopenia, anemia, etc., graded according to the Common Terminology Criteria for Adverse Events (CTCAE) standards.

  Measured up to 2 years after the last participant is enrolled

Secondary Outcomes (4)

• Progression-Free Survival (PFS)

  Measured up to 4 years after the last participant is enrolled

• Graft-Versus-Host Disease-Free, Relapse-Free Survival (GRFS)

  Measured up to 4 years after the last participant is enrolled

• Overall Survival (OS)

  Measured up to 4 years after the last participant is enrolled

• Treatment-Related Mortality (TRM)

  Measured up to 2 years after the last participant is enrolled

Study Arms (1)

Cohort 1

EXPERIMENTAL

This cohort includes patients with relapsed and refractory leukemia. Patients firstly receive chemotherapy per center standard, followed by HLA-mismatched GPBMC infusion. Secondly, patients receive HLA-matched sibling/haploidentical/unrelated HSCT.

Procedure: Chemotherapy; Biological: HLA-mismatched GPBMC infusion; Procedure: Allogeneic Stem Cell Transplantation

Interventions

Chemotherapy PROCEDURE

Available chemotherapy regimens include but not limited to FLAG (fludarabine, cytarabine, G-CSF), DAV (daunorubicin, cytarabine, venetoclax), IAV (idarubicin, cytarabine, venetoclax), VDCP (vincristine, daunorubicin, cyclophosphamide, prednisone), hyper-CVAD A (cyclophosphamide, vincristine, doxorubicin, dexamethasone), FC (fludarabine and cyclophosphamide), et al.

Cohort 1

HLA-mismatched GPBMC infusion BIOLOGICAL

HLA-mismatched GPBMCs are infused following chemotherapy.

Cohort 1

Allogeneic Stem Cell Transplantation PROCEDURE

Patients receive conditioning including but not limited to fludarabine, cyclophosphamide, antithymocyte globulin (ATG), and total body irradiation (TBI). HLA-matched sibling/haploidentical/unrelated GPBMCs are infused at day 0. Post-transplant cyclophosphamide, tacrolimus/cyclosporin, and mycophenolate mofetil are administered as graft-versus-host disease prophylaxis.

Cohort 1

Eligibility Criteria

• Age: 15 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Age \>=15 years, male or female, non-limited by race or ethnicity.
• Confirmed diagnosis of R/R leukemia and risk stratification according to the World Health Organization (WHO) 5th edition classification, based on histopathology and cytogenetics.
• Adequate hepatic function including alanine transaminase (ALT) and aspartate aminotransferase (AST )\<= 3 × upper limit of normal(ULN), and total bilirubin \<= 1.5 × ULN.
• Adequate renal function including serum creatinine \<= 2 × ULN or CrCl\>= 40mL/min.
• LVEF measured by echocardiogram is within the normal range (LVEF \> 50%).
• The subject must have one HLA mismatched donor who is \>= 18 years old to provide GPBMCs for the first-step infusion. If this donor is not qualified as an allo-HSCT donor, the subject must also have another donor who is \>= 18 years old and qualified as allo-HSCT donor (i.e., matched sibling, 9-10/10 loci matched unrelated, or haploidentical). In addition, the donor voluntarily donates hematopoietic stem cells and signs the consent form. Each subject (or his/her legal representatives) must sign the Informed Consent Form (ICF), indicating that he/she understands the purpose and procedures of research, and is willing to participate in research.

You may not qualify if:

• Uncontrolled infection or hemorrhage.
• Cardiovascular disease with clinical significance, such as uncontrolled or highly symptomatic cardiac arrhythmias, congestive heart failure, or myocardial infarction within 6 months prior to screening, or New York Heart Association (NYHA) function class 3 (moderate) or class 4 (severe) heart disease.
• Uncontrolled autoimmune disease or requiring immunosuppression treatment.
• History of severe blood infusion reaction.
• Nursing women, women of childbearing potential with positive urine pregnancy test, or women of childbearing potential who are not willing to maintain adequate contraception.
• Psychiatric disorder or cognitive impairment that in the researcher's judgment would make the subject not likely to adhere to the protocol requirements.
• Major surgery within 4 weeks prior to enrollment.
• Life-threatening illness other than leukemia or uncontrolled intercurrent illness.

Sponsors & Collaborators

• Beijing 302 Hospital: lead

Study Sites (1)

Department of Hematology, the Fifth Medical Center of Chinese PLA General Hospital

Beijing, 100071, China

RECRUITING

MeSH Terms

Conditions

Leukemia

Interventions

Drug Therapy

Condition Hierarchy (Ancestors)

Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Therapeutics

Study Officials

• Bo Cai, MD

  Department of Hematology, the Fifth Medical Center of Chinese PLA General Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Bo Cai, MD

CONTACT

+861066947168; caibo2008@163.com

Yangyang Lei, MD

CONTACT

+861066947180; 942056176@qq.com

Study Design

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

Study Record Dates

• First Submitted: December 9, 2025
• First Posted: December 22, 2025
• Study Start: December 1, 2025
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): December 1, 2030
• Last Updated: December 22, 2025

Record last verified: 2025-12

Locations

CN (1)