Umbilical Cord Blood Transplantation From Unrelated Donors

Launched by UNIVERSITY OF ROCHESTER · Jan 7, 2017

Keywords

ClinConnect Summary

This clinical trial is studying the use of umbilical cord blood stem cell transplantation as a treatment for various serious conditions such as acute leukemia, immune deficiency disorders, and certain blood disorders. The goal is to improve the way this type of transplant is done and to see how well it works at our center. The trial is currently looking for participants, including patients aged 60 and older, who have specific diseases that can be treated with stem cell transplants, such as severe forms of leukemia or congenital disorders.

To be eligible, patients must have a suitable diagnosis and meet certain health criteria, like having good heart and lung function. Those who join the trial can expect to receive one of four different treatment preparations before the transplant. It’s important to know that this study is not for everyone; for example, patients who have a close match with another donor or who have had a recent stem cell transplant may not qualify. Participants will be closely monitored throughout the process to ensure their safety and well-being.

Gender

ALL

Eligibility 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