Optimized Cord Blood Transplantation for the Treatment of Patients With High-risk Hematologic Malignancies Who Have Relapsed After First Allogeneic Stem Cell Transplantation

NCT ID: NCT06807606

Last Updated: 2025-12-04

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 35 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-03-03
• Study Completion Date: 2029-11-30

Brief Summary

The goal of this clinical research study is to learn if intermediate-intensity conditioning therapy followed by a cord blood transplant can help to control high-risk hematological malignancies in patients who need a second allogeneic stem cell transplantation.

Detailed Description

Primary Objective:

To evaluate 1-year overall survival (OS) following CBT with intermediate dose intensity conditioning for patients in need of a second allogeneic stem cell transplantation.

Secondary Objectives:

Speed and success of neutrophil and platelet engraftment. Incidences of graft failure.

• Incidence of day 100 grade II-IV and III-IV aGVHD and day 180 grades II-IV and III-IV aGVHD.

Incidence of 1-year, 2-year, and 3-year cGVHD.

Incidence of TRM (100 days, 6 months, 1 and 2 years).

The probabilities of relapse, OS, PFS, and GRFS at 1 year, 2, and 3 years as listed in the secondary endpoints in section 2.2.

Correlative laboratory studies investigating graft versus leukemia biology.

Conditions

Hematologic Malignancies

Study Design

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

Study Groups

Optimized CBT

Participants enrollment on trial will be determined by consultation with the physicians of the SCT Service.

Group Type: EXPERIMENTAL

Drugs Cyclophosphamide

Intervention Type: DRUG

Given as standard of care treatment through IV infusion

Fludarabine

Intervention Type: DRUG

Given as standard of care treatment through IV infusion

Thiotepa

Intervention Type: DRUG

Given as standard of care treatment through IV infusion

Tacrolimus

Intervention Type: DRUG

Given as standard of care treatment through IV infusion

Mycophenolate mofetil

Intervention Type: DRUG

Given as standard of care treatment through IV infusion

Interventions

Drugs Cyclophosphamide

Given as standard of care treatment through IV infusion

Intervention Type: DRUG

Fludarabine

Given as standard of care treatment through IV infusion

Intervention Type: DRUG

Thiotepa

Given as standard of care treatment through IV infusion

Intervention Type: DRUG

Tacrolimus

Given as standard of care treatment through IV infusion

Intervention Type: DRUG

Mycophenolate mofetil

Given as standard of care treatment through IV infusion

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Patient aged 0-60 y/o at the time of consent. Adult is defined as patients 18 years of age or older at the time of consent.

2. Patient must have relapsed >100 days since first transplant.

3. Diagnosis and Disease Status:

a. Acute myelogenous leukemia (AML): i. Patients in morphologic remission (<5% blasts) at the time of transplant, with or without persistent cytogenetic, flow cytometric, or molecular aberrations, or those with hypocellular marrows at time of transplant, are eligible. b. Acute lymphoblastic leukemia (ALL): i. Patients in morphologic remission with less than 5% blasts at time of transplant, with or without persistent cytogenetic, flow cytometric or molecular aberrations, or those or who have hypocellular bone marrows, are eligible. c. Other acute leukemias: i. Acute leukemias of ambiguous lineage or mixed phenotype in morphologic remission with less than 5% blasts at time of transplant, with or without persistent cytogenetic, flow cytometric or molecular aberrations, or those who have hypocellular bone marrows, are eligible. d. Myelodysplastic Syndromes (MDS) or CMML without myelofibrosis. i. Includes MDS with any IPSS risk category.

4. Prior treatment:

a. To be eligible for this study, patients need to have received one prior allogeneic stem cell transplantation.

5. Karnofsky score equal or greater than 70% for patients aged 16 years and older or Lansky score equal or greater than 70% for patients less than 16 years old (See Appendix B; inpatient Leukemia service transfers without discharge are acceptable provided patient has equivalent KPS as if were outpatient).

6. Renal and Liver function:

1. Calculated creatinine clearance > 50 ml/min.

2. Bilirubin < 2 mg/dL (unless benign congenital hyperbilirubinemia or hemolysis).

3. ALT < 5 x upper limit of normal (ULN).

7. Pulmonary function: corrected diffusion capacity of the lung for carbon monoxide (DLCO) > 60% predicted. This criteria is waived for patients who are developmentally unable to complete pulmonary function test.

8. Left ventricular ejection fraction (MOD-bp) > 50%.

9. Graft Criteria:

1. Two CB units will be selected according to the current MDACC CB unit selection algorithm.

2. High resolution 8-allele HLA typing and recipient HLA antibody profile will be performed.

3. Unit selection will occur based on HLA-match, total nucleated cell (TNC), and CD34+ cell dose adjusted per patient body weight.

4. The bank of origin will also be considered.

5. Donor-specific HLA antibodies, if present, will also be taken into consideration.

6. Each CB unit must be at least 3/8 HLA-matched to the patient considering high-resolution 8-allele HLA typing.

7. Each CB unit will be required to have a cryopreserved TNC dose of at least 1.5 x 107 TNC/ recipient body weight (TNC/ kg).

8. Each CB unit will be required to have a cryopreserved CD34+ cell dose of at least 1.0 x 105 CD34+ cells/ recipient body weight (CD34+ cells/kg).

9. A minimum of one unit will be reserved as a backup graft.

10. Each CB unit will be required to be cryopreserved in standard cryovolume. (24- 27 ml/s per unit) and be red blood cell depleted.

• Maximum Eligible Age: 60 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

M.D. Anderson Cancer Center

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Warren Fingrut, MD

Role: PRINCIPAL_INVESTIGATOR

M.D. Anderson Cancer Center

Locations

The University of Texas M. D. Anderson Cancer Center

Houston, Texas, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Warren Fingrut, MD

Role: CONTACT

wbfingrut@mdanderson.org

713-745-2214

Facility Contacts

Warren Fingrut, MD

Role: primary

wbfingrut@mdanderson.org

713-745-2214

Related Links

http://www.mdanderson.org

MD Anderson Cancer Center Website

Other Identifiers

NCI-2025-00794

Identifier Type: OTHER

Identifier Source: secondary_id

2024-1605

Identifier Type: -

Identifier Source: org_study_id