Umbilical Cord Blood Transplantation From Unrelated Donors

• Status: Recruiting
• Trial end date: 2026-06-01
• Target enrollment: 0
• Participant gender: All

Summary

This study is a single-center, treatment protocol with 4 possible preparative regimens, designed to validate the process of umbilical cord blood stem cell transplantation at our institution.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University of Rochester
• Treatments: Busulfan; Cyclophosphamide; Fludarabine; Fludarabine phosphate; Melphalan; Mesna

Criteria

Inclusion Criteria:

• Appropriate diagnosis: Patients must have a disease or syndrome amenable to therapy with hematopoietic stem cell transplantation. Diagnoses include, but are not limited to:

• Congenital and Other Non-malignant Disorders:

• Immunodeficiency disorders (e.g. Severe Combined Immunodeficiency, Wiskott-Aldrich Syndrome)

• Congenital hematopoietic stem cell defects (e.g. Chediak-Higashi Syndrome, Congenital Osteopetrosis, Osteogenesis Imperfecta)

• Metabolic disorders (e.g. Hurler's Syndrome)

• Severe aplastic anemia

• High-Risk Leukemia:

• Acute Myelogenous Leukemia

• Refractory to standard induction therapy (more than 1 cycle required to achieve remission)

• Recurrent (in CR ≥ 2)

• Treatment-related AML or MDS

• Evolved from myelodysplastic syndrome

• Presence of FLT3 abnormalities

• FAB M6 or M7

• Adverse cytogenetics

• Myelodysplastic Syndrome

• Acute Lymphoblastic Leukemia including T lymphoblastic leukemia:

• Refractory to standard induction therapy (time to CR >4 weeks)

• Recurrent (in CR ≥ 2)

• WBC count >30,000/mcL at diagnosis

• Age >30 at diagnosis

• Adverse cytogenetics, such as t(9:22), t(1:19), t(4:11), and other MLL rearrangements.

• Chronic Myelogenous Leukemia in accelerated phase or blast crisis

• Biphenotypic or undifferentiated leukemia

• Burkitt's leukemia or lymphoma

• Lymphoma:

• Large cell, Mantle cell, Hodgkin lymphoma refractory or recurrent, chemo-sensitive, and ineligible for an autologous stem cell transplant or previously treated with autologous SCT

• Marginal zone or follicular lymphoma that is progressive after at least two prior therapies

• Multiple Myeloma, recurrent following high-dose therapy and autologous SCT or ineligible for an autologous HSCT

• Solid tumors, with efficacy of allogeneic HSCT demonstrated for the specific disease and disease status

• Adequate organ function:

• Cardiac - LVEF >45%, or shortening fraction >25%, Absence of congestive heart failure or conduction disturbances with high risk for sudden death

• Pulmonary - DLCO (corrected for hemoglobin), FEV1 and FVC ≥ 50% predicted;

• Renal - serum Cr < 1.5 times the upper limit of normal for age or GFR ≥ 50 ml/min/1.73m2

• Hepatic - total bilirubin level < 2 times the upper limit of normal (except for patients with Gilbert's syndrome or hemolysis); if the primary disease process is causal, this criterion will be reconsidered. ALT, AST, and Alkaline phosphatase ≤ 5 times upper limit of normal.

• Performance Status Karnofsky or Lansky score ≥ 70%.

• Informed Consent must be obtained prior to initiating conditioning therapy.

• Receipt of viable cord blood product(s), single or dual, must be confirmed with the stem cell processing laboratory prior to initiating conditioning therapy.

Exclusion Criteria:

• Availability of 10/10 or 9/10 HLA-matched related or unrelated donor within a reasonable timeframe dictated by the clinical urgency of the transplant

• Autologous HSCT < 6 months prior to proposed UCB transplant

• Pregnant or breast feeding

• Current uncontrolled infection

• Evidence of HIV infection or positive HIV serology