Umbilical Cord Blood Transplantation From Unrelated Donors

NCT03016806 | Recruiting DMC

• 1 other identifier: UBMT 15029
• Study Type: observational
• Target: 30
• Locations: 1 country
• Sites: 1

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).

Conditions

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

Outcome Measures

Primary Outcomes (1)

• Engraftment of ANC and Platelets

  The date of engraftment of ANC is the first of 3 consecutive days of ANC of 500 or higher based on daily CBC and Differential Counts. The date of engraftment of platelets is the first of three consecutive days of platelet counts of 20,000 or higher in the absence of platelet transfusions for a t least 7 days prior.

  42 days following the infusion of stem cells for ANC [If engraftment of ANC does not occur within 42 days, a subsequent transplant will be performed if a donor is available.]

Secondary Outcomes (4)

• Rate of non-engraftment and of secondary graft failure

  At 30 days, 100 days, 6 months and yearly from the date of transplant until the date of documented graft failure or the subject's death up to 120 months.

• Incidence of acute graft-versus-host disease

  At 30 days and 100 days after transplant from the date of transplant until the date of documented acute GvHD.

• Incidence of chronic graft-versus-host disease

  At 100 days, 6 months and yearly after transplant from the date of transplant until the date of documented graft failure or the subject's death up to 120 months.

• Disease-free survival

  At 30 days, 100 days, 6 months and yearly after transplant from the date of transplant until the date of documented graft failure or the subject's death up to 120 months.

Study Arms (4)

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

Radiation: Total Body Irradiation 1200 cGy; Drug: Cyclophosphamide; Drug: Mesna; Procedure: Cord Blood Infusion

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

Drug: Cyclophosphamide; Drug: Mesna; Procedure: Cord Blood Infusion; Drug: Busulfan

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

Procedure: Cord Blood Infusion; Drug: Fludarabine; Drug: Melphalan

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

Radiation: Total Body Irradiation 200 cGy; Drug: Cyclophosphamide; Drug: Mesna; Drug: Fludarabine

Interventions

Total Body Irradiation 1200 cGy RADIATION

Total Body Irradiation 1200 cGy in 8 fractions

Also known as: TBI 1200 cGy

Full Intensity, TBI-based Conditioning

Total Body Irradiation 200 cGy RADIATION

Total Body Irradiation 200 cGy in one fraction

Also known as: TBI 200 cGy

Non-Myeloablative Conditioning

Cyclophosphamide DRUG

50 mg/kg or 60 mg/kg

Also known as: Cy

Full Intensity, Chemo-based Conditioning; Full Intensity, TBI-based Conditioning; Non-Myeloablative Conditioning

Mesna DRUG

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

Also known as: Pre-Mesna

Full Intensity, Chemo-based Conditioning; Full Intensity, TBI-based Conditioning; Non-Myeloablative Conditioning

Cord Blood Infusion PROCEDURE

Intravenous infusion of cord blood stem cells

Full Intensity, Chemo-based Conditioning; Full Intensity, TBI-based Conditioning; Reduced Intensity Chemotherapy

Busulfan DRUG

0.8 mg/kg x 16 doses

Also known as: Bu

Full Intensity, Chemo-based Conditioning

Fludarabine DRUG

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

Also known as: Flu

Non-Myeloablative Conditioning; Reduced Intensity Chemotherapy

Melphalan DRUG

140 mg/m2

Also known as: Mel

Reduced Intensity Chemotherapy

Eligibility Criteria

• Age: 2 Months - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• University of Rochester: lead

Study Sites (1)

Wilmot Cancer Institute

Rochester, New York, 14642, United States

RECRUITING

MeSH Terms

Conditions

Immunologic Deficiency Syndromes; Metabolic Diseases; Anemia, Aplastic; Myelodysplastic Syndromes; Lymphoma; Multiple Myeloma

Interventions

Cyclophosphamide; Mesna; Busulfan; fludarabine; Influenza Vaccines; Melphalan; Honey

Condition Hierarchy (Ancestors)

Immune System Diseases; Nutritional and Metabolic Diseases; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Neoplasms, Plasma Cell; Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Paraproteinemias; Blood Protein Disorders; Hemorrhagic Disorders

Intervention Hierarchy (Ancestors)

Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds; Alkanesulfonates; Alkanesulfonic Acids; Alkanes; Hydrocarbons, Acyclic; Sulfhydryl Compounds; Sulfur Compounds; Sulfonic Acids; Sulfur Acids; Butylene Glycols; Glycols; Alcohols; Mesylates; Viral Vaccines; Vaccines; Biological Products; Complex Mixtures; Phenylalanine; Amino Acids, Aromatic; Amino Acids, Cyclic; Amino Acids; Amino Acids, Peptides, and Proteins; Food; Diet, Food, and Nutrition; Physiological Phenomena; Food and Beverages

Study Officials

• Omar Aljitawi, MD

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

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Omar Aljitawi, MD

CONTACT

585-275-4099; omar_aljitawi@urmc.rochester.edu

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor - Department of Medicine, Hematology/Oncology (SMD)

Study Record Dates

• First Submitted: December 26, 2016
• First Posted: January 11, 2017
• Study Start: June 1, 2015
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): June 1, 2027
• Last Updated: October 10, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)