Umbilical Cord Blood Transplantation From Unrelated Donors

NCT ID: NCT03016806

Last Updated: 2025-10-10

Basic Information

• Recruitment Status: RECRUITING
• Total Enrollment: 30 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2015-06-30
• Study Completion Date: 2027-06-30

Brief Summary

This study is being done to determine how long it takes for the engraftment (recovery of blood cell counts) of umbilical cord stem cells and also how often engraftment of umbilical cord stem cells transplanted from an unrelated donor fails. Another purpose will be to document the rate of disease-free survival and the rate of relapse (a return of your disease or syndrome) as well as the incidence and severity of graft versus host disease (GvHD) following cord blood stem cell transplantation. GvHD is a complication of stem cell transplants in which white blood cells from the transplanted tissue (graft) attack the transplant recipient's body (host).

Detailed Description

PRIMARY OBJECTIVES:

I. Determine the kinetics of engraftment of umbilical cord blood (UCB) following transplantation into unrelated individuals.

II. Determine the incidence of non-engraftment and secondary graft failure when unrelated donor UCB cells are administered to patients receiving myeloablative, reduced intensity cytoreductive, or non-ablative conditioning regimens together with immunosuppressive therapy.

III. Determine the incidence and severity of graft-vs-host disease (GVHD) for patients receiving unrelated donor UCB grafts.

IV. Document the overall survival, disease-free-survival, and rates of relapse for UCB transplant recipients.

OUTLINE: This is an observational study.

Patients undergo a conditioning regimen per standard of care at the discretion of the treating provider, followed by a planned UCB transplantation on study. Patients also have their medical records reviewed for engraftment data on study.

Conditions

Acute Leukemia; Immune Deficiency Disorder; Congenital Hematological Disorder; Metabolism Disorder; Aplastic Anemia; Myelodysplastic Syndromes; Chronic Leukemia; Lymphoma; Multiple Myeloma; Solid Tumor

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

Full Intensity, TBI-based Conditioning

Full Intensity TBI-based Conditioning Total Body Irradiation 1200 cGy in fractions of 150 cGy days -8 or -7 to -4 Cyclophosphamide 60 mg/kg/day x 2 doses days -3 and -2 Mesna 60 mg/kg/day with 20% loading dose with first Cyclophosphamide followed by continuous infusion over 24 hours x 2 doses \[to be completed 24 hours after final Cyclophosphamide dose\] followed by Cord Blood Infusion Other names: TBI/Cy

Total Body Irradiation 1200 cGy

Intervention Type: RADIATION

Total Body Irradiation 1200 cGy in 8 fractions

Cyclophosphamide

Intervention Type: DRUG

50 mg/kg or 60 mg/kg

Mesna

Intervention Type: DRUG

50 mg/kg or 60 mg/kg plus 10% loading dose

Cord Blood Infusion

Intervention Type: PROCEDURE

Intravenous infusion of cord blood stem cells

Full Intensity, Chemo-based Conditioning

Full Intensity, Chemotherapy Conditioning Busulfan days -7 to -4 Recipients \<5 years - 1 mg/kg/dose x 16 doses every 6 hours Recipients \>/= 5 years - 0.8 mg/kg/dose x 16 doses every 6 hours Cyclophosphamide 60 mg/kg/day x 2 doses days -3 and -2 Mesna 60 mg/kg/day with 20% loading dose with first Cyclophosphamide followed by continuous infusion over 24 hours x 2 doses \[to be completed 24 hours after final Cyclophosphamide dose\] followed by Cord Blood Infusion Other names: Bu/Cy

Cyclophosphamide

Intervention Type: DRUG

50 mg/kg or 60 mg/kg

Mesna

Intervention Type: DRUG

50 mg/kg or 60 mg/kg plus 10% loading dose

Cord Blood Infusion

Intervention Type: PROCEDURE

Intravenous infusion of cord blood stem cells

Busulfan

Intervention Type: DRUG

0.8 mg/kg x 16 doses

Reduced Intensity Chemotherapy

Reduced Intensity Chemotherapy Fludarabine 30 mg/m2/day x 5 doses days -6 to -2 Melphalan 140 mg/m2/day x 1 dose day -2 Cord Blood Infusion Other names: Flu/Mel

Cord Blood Infusion

Intervention Type: PROCEDURE

Intravenous infusion of cord blood stem cells

Fludarabine

Intervention Type: DRUG

30 mg/m2/day x 5 or 40 mg/m2/day x 5

Melphalan

Intervention Type: DRUG

140 mg/m2

Non-Myeloablative Conditioning

Fludarabine 40 mg/m2/day x 5 doses days -6 to -2 Cyclophosphamide 50 mg/kg/day x 1 dose day -6 Mesna 50 mg/kg/day with 20% loading dose with Cyclophosphamide dose followed by continuous infusion over 24 hours x 1 dose \[to be completed 24 hours after Cyclophosphamide dose\] Total Body Irradiation 200 cGy in a single fraction day -1 Cord Blood Infusion Other names: Flu/Cy/TBI

Total Body Irradiation 200 cGy

Intervention Type: RADIATION

Total Body Irradiation 200 cGy in one fraction

Cyclophosphamide

Intervention Type: DRUG

50 mg/kg or 60 mg/kg

Mesna

Intervention Type: DRUG

50 mg/kg or 60 mg/kg plus 10% loading dose

Fludarabine

Intervention Type: DRUG

30 mg/m2/day x 5 or 40 mg/m2/day x 5

Interventions

Total Body Irradiation 1200 cGy

Total Body Irradiation 1200 cGy in 8 fractions

Intervention Type: RADIATION

Total Body Irradiation 200 cGy

Total Body Irradiation 200 cGy in one fraction

Intervention Type: RADIATION

Cyclophosphamide

50 mg/kg or 60 mg/kg

Intervention Type: DRUG

Mesna

50 mg/kg or 60 mg/kg plus 10% loading dose

Intervention Type: DRUG

Cord Blood Infusion

Intravenous infusion of cord blood stem cells

Intervention Type: PROCEDURE

Busulfan

0.8 mg/kg x 16 doses

Intervention Type: DRUG

Fludarabine

30 mg/m2/day x 5 or 40 mg/m2/day x 5

Intervention Type: DRUG

Melphalan

140 mg/m2

Intervention Type: DRUG

Other Intervention Names

TBI 1200 cGy; TBI 200 cGy; Cy; Pre-Mesna; Bu; Flu; Mel

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

• Minimum Eligible Age: 2 Months
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University of Rochester

OTHER

Sponsor Role: lead

Responsible Party

Omar Aljitawi

Professor - Department of Medicine, Hematology/Oncology (SMD)

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Omar Aljitawi, MD

Role: PRINCIPAL_INVESTIGATOR

Professor - Department of Medicine, Hematology/Oncology (SMD)

Locations

Wilmot Cancer Institute

Rochester, New York, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Omar Aljitawi, MD

Role: CONTACT

omar_aljitawi@urmc.rochester.edu

585-275-4099

Facility Contacts

Omar Aljitawi, MD

Role: primary

omar_aljitawi@urmc.rochester.edu

585-275-4099

Other Identifiers

UBMT 15029

Identifier Type: -

Identifier Source: org_study_id