Using umbilical cord blood for treating blood cancers

Umbilical Cord Blood Transplantation Using a Myeloablative Preparative Regimen for the Treatment of Hematological Diseases

This study is testing whether using umbilical cord blood can safely treat blood cancers like acute leukemia in patients.

Quick facts

• Phase: NA
• Study type: Interventional
• Enrollment: 200 (estimated)
• Ages: N/A to 55 Years
• Sex: All
• Sponsor: Masonic Cancer Center, University of Minnesota (other)
• Drugs / interventions: imatinib, chemotherapy, Radiation, cyclophosphamide, fludarabine
• Locations: 1 site (Minneapolis, Minnesota)
• Trial ID: NCT01962636 on ClinicalTrials.gov

What this trial studies

This study focuses on the use of unrelated umbilical cord blood transplantation (UCBT) as a treatment for various hematological diseases, including acute leukemias. It employs a myeloablative preparative regimen consisting of cyclophosphamide, fludarabine, and total body irradiation. The study aims to collect clinical data on the effectiveness and safety of this treatment approach, including pre- and post-transplant care and UCB selection. An average of 18 patients will be treated annually under this protocol at the University of Minnesota Masonic Cancer Center.

Who should consider this trial

Good fit: Ideal candidates include patients with high-risk acute myeloid leukemia or acute lymphocytic leukemia who are in complete remission but have specific high-risk features.

Not a fit: Patients with low-risk hematological diseases or those not in complete remission may not benefit from this study.

Why it matters

Potential benefit: If successful, this approach could provide a new treatment option for patients with high-risk blood cancers who lack suitable stem cell donors.

How similar studies have performed: Other studies have shown promise in using umbilical cord blood for transplantation, indicating potential success for this approach.

Eligibility criteria

Inclusion Criteria:

* Eligible Disease Status

  * Acute Myeloid Leukemia (AML): high risk CR1 (as evidenced by preceding MDS, high risk cytogenetics, ≥ 2 cycles to obtain CR, erythroblastic or megakaryocytic leukemia; CR2+. All patients must be in CR as defined by hematological recovery, AND \<5% blasts by light microscopy within the bone marrow with a cellularity of ≥15%.
  * Very high risk pediatric patients with AML: Patients \<21 years, however, are eligible with (M2 marrow) with \< 25% blasts in marrow after having failed one or more cycles of chemotherapy.
  * Acute Lymphocytic Leukemia (ALL): high risk CR1 as defined by cytogenetics (such as t(9;22), t (1:19), t(4;11), other MLL rearrangements, hypodiploidy, or IKZF1 abnormalities), DNA index \< 0.81, \> 1 cycle to obtain CR or presence minimal residual disease (MRD). Patients in CR2+ are eligible. All patients must be in CR as defined by hematological recovery, AND \<5% blasts by light microscopy within the bone marrow with a cellularity of ≥15%.
  * Very high risk pediatric patients with ALL: patients \<21 years are also considered high risk CR1 if they had M2 or M3 marrow at day 42 from the initiation of induction or M3 marrow at the end of induction. They are eligible once they achieved a complete remission.
  * Chronic Myelogenous Leukemia excluding refractory blast crisis: To be eligible in first chronic phase (CP1) patient must have failed or be intolerant to imatinib mesylate.
  * Plasma Cell Leukemia after initial therapy, who achieved at least a partial remission
  * Advanced Myelofibrosis
  * Myelodysplasia (MDS) IPSS INT-2 or High Risk (i.e. RAEB, RAEBt) or Refractory Anemia with severe pancytopenia or high risk cytogenetics: Blasts must be \< 10% by a representative bone marrow aspirate morphology.
  * Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CLL/SLL), Marginal Zone B-Cell Lymphoma or Follicular Lymphoma are eligible if there was disease progression/relapse within 12 of achieving a partial or complete remission. Patients who had remissions lasting \> 12 months, are eligible after at least two prior therapies. Patients with bulky disease (nodal mass greater than 5 cm) should be considered for de-bulking chemotherapy before transplant.
  * Lymphoplasmacytic Lymphoma, Mantle-Cell Lymphoma, Prolymphocytic Leukemia are eligible after initial therapy in CR1+ or PR1+.
  * Large Cell NHL \> CR2/\> PR2: Patients in CR2/PR2 with initial short remission (\<6 months) are eligible.
  * Lymphoblastic Lymphoma, Burkitt's Lymphoma, and other high-grade NHL after initial therapy if stage III/IV in CR1/PR1 or after progression if stage I/II \< 1 year.
  * Multiple Myeloma beyond PR2: Patients with chromosome 13 abnormalities, first response lasting less than 6 months, or β-2 microglobulin \> 3 mg/L, may be considered for this protocol after initial therapy.
  * Myeloproliferative Syndromes
* Availability of suitable UCB unit(s)
* 0 to 55 years
* Voluntary written consent (adult or parental/guardian)

Exclusion Criteria:

* previous irradiation that precludes the safe administration of TBI - Radiation Oncology will evaluate all patients who have had previous radiation therapy
* chemotherapy refractory large cell and high grade NHL (ie progressive disease after \> 2 salvage regimens)
* if ≤ 18 years old, prior myeloablative transplant within the last 6 months. If \>18 years old prior myeloablative allotransplant or autologous transplant
* extensive prior therapy including \> 12 months alkylator therapy or \> 6 months alkylator therapy with extensive radiation
* pregnant or breastfeeding
* HIV positive

Where this trial is running

Minneapolis, Minnesota

• University of Minnesota Masonic Cancer Center — Minneapolis, Minnesota, United States (RECRUITING)

Study contacts

• Principal investigator: Claudio Brunstein, MD — University of Minnesota
• Study coordinator: Claudio Brunstein, MD

Conditions: Acute Myeloid Leukemia, Acute Lymphocytic Leukemia, Chronic Myelogenous Leukemia, Plasma Cell Leukemia, Myelofibrosis, Myelodysplasia, Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma