Brief Title
Umbilical Cord Blood Transplantation From Unrelated Donors
Official Title
Umbilical Cord Blood Transplantation From Unrelated Donors
Brief 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.
Detailed Description
This study is a single-center treatment protocol with four possible preparative regimens,
designed to validate the process of umbilical cord blood stem cell transplantation at our
institution. Enrolled patients will receive chemotherapy +/-total body radiation as a
pre-transplant conditioning regimen. Patients will then receive cord blood stem cells
followed by GvHD prophylaxis that will include Tacrolimus and Mycophenolate Mofetil, or
Cyclosporin A and Methylprednisolone. Multiple data points will be collected prior to,
during, and following transplantation to ensure safety of the process and to evaluate the
stated objectives.
Study Phase
Phase 1
Study Type
Interventional
Primary Outcome
Engraftment of ANC and Platelets
Secondary Outcome
Rate of non-engraftment and of secondary graft failure
Condition
Acute Leukemia
Intervention
Total Body Irradiation 1200 cGy
Study Arms / Comparison Groups
Full Intensity, TBI-based Conditioning
Description: 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
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Radiation
Estimated Enrollment
30
Start Date
June 2015
Completion Date
June 2026
Primary Completion Date
June 2025
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
Gender
All
Ages
2 Months - 75 Years
Accepts Healthy Volunteers
No
Contacts
Jane L Liesveld, MD, 585-275-4099, [email protected]
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT03016806
Organization ID
UBMT 15029
Responsible Party
Principal Investigator
Study Sponsor
University of Rochester
Study Sponsor
Jane L Liesveld, MD, Principal Investigator, Medical Director, Blood & Marrow Transplant Unit
Verification Date
June 2022