- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03016806
Umbilical Cord Blood Transplantation From Unrelated Donors
July 17, 2023 updated by: Jane Liesveld, University of Rochester
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.
Study Overview
Status
Recruiting
Conditions
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 Type
Interventional
Enrollment (Estimated)
30
Phase
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Jane L Liesveld, MD
- Phone Number: 585-275-4099
- Email: jane_liesveld@urmc.rochester.edu
Study Contact Backup
- Name: Michael W Becker, MD
- Phone Number: 585-275-4099
- Email: michael_becker@urmc.rochester.edu
Study Locations
-
-
New York
-
Rochester, New York, United States, 14642
- Recruiting
- Wilmot Cancer Institute
-
Contact:
- Jane L Liesveld, MD
- Phone Number: 585-275-4099
- Email: jane_liesveld@urmc.rochester.edu
-
Contact:
- Michael W Becker, MD
- Phone Number: 585-275-4099
- Email: michael_becker@urmc.rochester.edu
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
2 months to 75 years (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Description
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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Non-Randomized
- Interventional Model: Parallel Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Other: 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 in 8 fractions
Other Names:
50 mg/kg or 60 mg/kg
Other Names:
50 mg/kg or 60 mg/kg plus 10% loading dose
Other Names:
Intravenous infusion of cord blood stem cells
|
Other: 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
|
50 mg/kg or 60 mg/kg
Other Names:
50 mg/kg or 60 mg/kg plus 10% loading dose
Other Names:
Intravenous infusion of cord blood stem cells
0.8 mg/kg x 16 doses
Other Names:
|
Other: 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
|
Intravenous infusion of cord blood stem cells
30 mg/m2/day x 5 or 40 mg/m2/day x 5
Other Names:
140 mg/m2
Other Names:
|
Other: 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
|
50 mg/kg or 60 mg/kg
Other Names:
50 mg/kg or 60 mg/kg plus 10% loading dose
Other Names:
30 mg/m2/day x 5 or 40 mg/m2/day x 5
Other Names:
Total Body Irradiation 200 cGy in one fraction
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Engraftment of ANC and Platelets
Time Frame: 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.]
|
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 Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Rate of non-engraftment and of secondary graft failure
Time Frame: 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.
|
The percentage of patients who fail to initially engraft ANC will be tabulated as well as the percentage of patients who have primary engraftment of ANC but whose graft fails as evidenced by pancytopenia, failure of bone marrow function and loss of donor chimerism following initial engraftment of ANC.
|
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
Time Frame: At 30 days and 100 days after transplant from the date of transplant until the date of documented acute GvHD.
|
Routine physical exams, liver function tests, and clinical history of diarrhea and upper GI symptoms will be used to assess the presence of, the maximum severity of and the date of onset of Acute GvHD based on the criteria published by Przepioka et al., Bone Marrow Transplant 1995; 15(6):825-8 as used by the Center for International Blood & Marrow Transplant Research.
The percentage of patients developing symptoms of acute graft-versus-host disease will be tabulated.
|
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
Time Frame: 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.
|
Assess the presence of and the maximum severity of and the date of onset of chronic GvHD based on Sullivan KM, Blood 1981;57-267 as used by the Cneter for International Blood & Marrow Transplant Research.
|
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
Time Frame: 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.
|
Document and update the length of time a subject survives without recurrence of the disease for which they were transplanted at 30 days, 100 days, 6 months and yearly following the infusion of cord blood stem cells for as long as the subjects survive and remain disease-free.
|
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.
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Principal Investigator: Jane L Liesveld, MD, Medical Director, Blood & Marrow Transplant Unit
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
June 1, 2015
Primary Completion (Estimated)
June 1, 2025
Study Completion (Estimated)
June 1, 2026
Study Registration Dates
First Submitted
December 26, 2016
First Submitted That Met QC Criteria
January 7, 2017
First Posted (Estimated)
January 11, 2017
Study Record Updates
Last Update Posted (Actual)
July 19, 2023
Last Update Submitted That Met QC Criteria
July 17, 2023
Last Verified
July 1, 2023
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Cardiovascular Diseases
- Vascular Diseases
- Immune System Diseases
- Neoplasms by Histologic Type
- Neoplasms
- Lymphoproliferative Disorders
- Immunoproliferative Disorders
- Bone Marrow Diseases
- Hemorrhagic Disorders
- Hemostatic Disorders
- Paraproteinemias
- Blood Protein Disorders
- Anemia
- Neoplasms, Plasma Cell
- Bone Marrow Failure Disorders
- Myelodysplastic Syndromes
- Multiple Myeloma
- Leukemia
- Hematologic Diseases
- Immunologic Deficiency Syndromes
- Anemia, Aplastic
- Physiological Effects of Drugs
- Molecular Mechanisms of Pharmacological Action
- Antirheumatic Agents
- Antineoplastic Agents
- Immunosuppressive Agents
- Immunologic Factors
- Protective Agents
- Antineoplastic Agents, Alkylating
- Alkylating Agents
- Myeloablative Agonists
- Cyclophosphamide
- Melphalan
- Fludarabine
- Busulfan
- Mesna
Other Study ID Numbers
- UBMT 15029
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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