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

University of Minnesota (UMN)

Status

Enrolling

Conditions

Myelofibrosis

Acute Myeloid Leukemia (AML)

Small Lymphocytic Lymphoma

Chronic Myelogenous Leukemia

Lymphoplasmacytic Lymphoma

Mantle-Cell Lymphoma

Non-Hodgkin Lymphoma

Burkitt's Lymphoma

Plasma Cell Leukemia

Multiple Myeloma

Lymphoblastic Lymphoma

Myelodysplasia

Follicular Lymphoma

Chronic Lymphocytic Leukemia

Diffuse Large B Cell Lymphoma

Marginal Zone B-Cell Lymphoma

Acute Lymphocytic Leukemia (ALL)

Prolymphocytic Leukemia

Treatments

Drug: Cyclophosphamide

Radiation: Total Body Irradiation

Biological: Umbilical cord blood

Drug: Mycophenylate mofetil

Drug: Fludarabine

Drug: Cyclosporine A

Study type

Interventional

Funder types

Other

Identifiers

NCT01962636

2013OC013

Details and patient eligibility

About

This is a treatment guideline for an unrelated umbilical cord blood transplant (UCBT) using a myeloablative preparative regimen for the treatment of hematological diseases, including, but not limited to acute leukemias. The myeloablative preparative regimen will consist of cyclophosphamide (CY), fludarabine (FLU) and fractionated total body irradiation (TBI).

Full description

This is a study to collect routine clinical data from UCBT using unrelated single or double UCB units as an alternative, non-HLA-matched stem cell source for patients with hematological diseases.

data collection from transplant preparative therapy consisting of treatments with chemotherapeutic regimens and total body irradiation.
data collection from umbilical cord blood selection and infusion.
data collection from standard supportive disease and transplant related care.

Pre- and post-transplant medication, UCB selection and infusion, supportive care, and follow-up will be according to the current University of Minnesota BMT guidelines.

An average of 18 patients are expected to be treated on this protocol per year.

Enrollment

200 estimated patients

Sex

All

Ages

Under 55 years old

Volunteers

No Healthy Volunteers

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

Trial design

Primary purpose

Treatment

Allocation

N/A

Interventional model

Single Group Assignment

Masking

None (Open label)

200 participants in 1 patient group

Umbilical Cord Blood Transplant

Experimental group

Description: