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Combined Haploidentical-Cord Blood Transplantation for Adults and Children

Primary Purpose

Leukemia, Myelodysplastic Syndrome, Multiple Myeloma

Status
Completed
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
Fludarabine-Melphalan & Rabbit antithymocyte globulin (r-ATG)
Stem Cell Transplant
Stem Cells Collections
Fludarabine, Thiotepa, Antithymocyte globulin (ATG), and Total Body Irradiation (TBI)
Fludarabine, Busulfan, and ATG
Sponsored by
University of Chicago
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Leukemia focused on measuring Appropriate candidate for transplantation, An HLA-identical related or unrelated donor cannot be identified within an appropriate time frame.

Eligibility Criteria

1 Year - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Patients will be eligible for this study if they have any one of the diseases that are known to be cured after allogeneic stem cell transplantation.

  1. Relapsed or refractory acute leukemia (myeloid or lymphoid)
  2. Acute leukemia in first remission at high-risk for recurrence
  3. Chronic myelogenous leukemia in accelerated phase or blast-crisis
  4. Chronic myelogenous leukemia in chronic phase
  5. Recurrent or refractory malignant lymphoma or Hodgkin lymphoma
  6. Chronic lymphocytic leukemia, relapsed or with poor prognostic features
  7. Multiple myeloma
  8. Myelodysplastic syndrome
  9. Chronic myeloproliferative disease
  10. Hemoglobinopathies
  11. Aplastic anemia

Exclusion Criteria:

  1. Zubrod performance status > 2
  2. Life expectancy is severely limited by concomitant illness
  3. Patients with severely decreased LVEF or impaired pulmonary function tests(PFT's)
  4. Estimated Creatinine Clearance <50 ml/min
  5. Serum bilirubin> 2.0 mg/dl or SGPT >3 x upper limit of normal
  6. Evidence of chronic active hepatitis or cirrhosis
  7. HIV-positive
  8. Patient is pregnant
  9. Patient or guardian not able to sign informed consent

Sites / Locations

  • The University of Chicago

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Experimental

Arm Label

Good Risks patients

High Risk Patients eligible for radiation

High Risk Patients not eligible for radiation

Arm Description

For patients transplanted in remission.

Outcomes

Primary Outcome Measures

Percentage of Participants With Neutrophil Engraftment
Cumulative incidence of graft failure (neutrophil) by day 28 was reported. Patients who did not have neutrophil engraftment before death was considered as a competing risk. Failure to engraft was defined as lack of evidence of hematopoietic recovery (ANC <500/mm3 and platelet count < 20,000/mm3) by day +35, confirmed by a biopsy revealing a marrow cellularity < 5%. Graft failure was also defined as initial myeloid engraftment by day +35, documented to be of donor origin, followed by a drop in the ANC to < 500/mm3 for more than three days, independent of any myelosuppressive drugs, severe GVHD, CMV, or other infection.

Secondary Outcome Measures

Percentage of Participants With Incidence of Acute (Grade II-IV) and Chronic Graft-vs-host Disease(GVHD)
Acute GVHD is defined by the Przepiorka criteria, which stages the degree of organ involvement in the skin, liver, and gastrointestinal (GI) tract, based on severity, with Stage 1+ being least severe and stage 4+ being the most severe. Grading of acute GVHD is as follows: Grade II (skin involvement stages 1+ to 3+, liver 1+, GI tract 1+), Grade III (skin involvement stages 2+ to 3+, liver 1+, GI tract 2+ to 4+), Grade IV (skin involvement stages 4+, Liver 4+). Chronic GVHD is assessed by NIH consensus development project on criteria for clinical trials in chronic graft-versus-host disease: I. Diagnosis and staging working group report. Biol Blood Marrow Transplant. 2005; 11:945-56., for grading criteria. (See Citation: Filipovich AH et al) The incidence of patients with acute GVHD (Grade II-IV) was determined at 180 days. The incidence of Chronic GVHD by 2 years was reported
Overall Survival- Percentage of Participants Who Survived at 2 Years and 5 Years
We reported overall survival at 2 years and 5 years after transplant

Full Information

First Posted
July 20, 2009
Last Updated
January 8, 2021
Sponsor
University of Chicago
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1. Study Identification

Unique Protocol Identification Number
NCT00943800
Brief Title
Combined Haploidentical-Cord Blood Transplantation for Adults and Children
Official Title
Combined Haploidentical-Cord Blood Transplantation for Adults and Children
Study Type
Interventional

2. Study Status

Record Verification Date
January 2021
Overall Recruitment Status
Completed
Study Start Date
October 9, 2006 (Actual)
Primary Completion Date
September 2018 (Actual)
Study Completion Date
September 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University of Chicago

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The primary objective is to assess the rate of engraftment with combined haploidentical-cord blood transplantation. The secondary objective is to evaluate the incidence and severity of acute and chronic graft-versus-host disease (GVHD).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Leukemia, Myelodysplastic Syndrome, Multiple Myeloma, Lymphoma
Keywords
Appropriate candidate for transplantation, An HLA-identical related or unrelated donor cannot be identified within an appropriate time frame.

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
87 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Good Risks patients
Arm Type
Experimental
Arm Description
For patients transplanted in remission.
Arm Title
High Risk Patients eligible for radiation
Arm Type
Experimental
Arm Title
High Risk Patients not eligible for radiation
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Fludarabine-Melphalan & Rabbit antithymocyte globulin (r-ATG)
Intervention Description
Fludarabine is given through the vein daily for 5 days. Melphalan is given through the vein daily for 2 days. ATG is given every day in the vein for four days.
Intervention Type
Procedure
Intervention Name(s)
Stem Cell Transplant
Intervention Description
Infusion of haploidentical donor, umbilical cord blood
Intervention Type
Procedure
Intervention Name(s)
Stem Cells Collections
Intervention Description
Haploidentical cells will be T-cell depleted using the Miltenyi Clinimax device.
Intervention Type
Drug
Intervention Name(s)
Fludarabine, Thiotepa, Antithymocyte globulin (ATG), and Total Body Irradiation (TBI)
Intervention Description
Fludarabine is given through the vein daily for 5 days. Thiotepa is given through the vein daily for 2 days. ATG is given through the vein every other day for 4 days. TBI is given twice a day for 3 days.
Intervention Type
Drug
Intervention Name(s)
Fludarabine, Busulfan, and ATG
Intervention Description
Fludarabine is given through the vein daily for 5 days. Busulfan is given through the vein daily for 4 days. ATG is given through the vein every other day for 4 days.
Primary Outcome Measure Information:
Title
Percentage of Participants With Neutrophil Engraftment
Description
Cumulative incidence of graft failure (neutrophil) by day 28 was reported. Patients who did not have neutrophil engraftment before death was considered as a competing risk. Failure to engraft was defined as lack of evidence of hematopoietic recovery (ANC <500/mm3 and platelet count < 20,000/mm3) by day +35, confirmed by a biopsy revealing a marrow cellularity < 5%. Graft failure was also defined as initial myeloid engraftment by day +35, documented to be of donor origin, followed by a drop in the ANC to < 500/mm3 for more than three days, independent of any myelosuppressive drugs, severe GVHD, CMV, or other infection.
Time Frame
Transplant (Day 0) through Day +28
Secondary Outcome Measure Information:
Title
Percentage of Participants With Incidence of Acute (Grade II-IV) and Chronic Graft-vs-host Disease(GVHD)
Description
Acute GVHD is defined by the Przepiorka criteria, which stages the degree of organ involvement in the skin, liver, and gastrointestinal (GI) tract, based on severity, with Stage 1+ being least severe and stage 4+ being the most severe. Grading of acute GVHD is as follows: Grade II (skin involvement stages 1+ to 3+, liver 1+, GI tract 1+), Grade III (skin involvement stages 2+ to 3+, liver 1+, GI tract 2+ to 4+), Grade IV (skin involvement stages 4+, Liver 4+). Chronic GVHD is assessed by NIH consensus development project on criteria for clinical trials in chronic graft-versus-host disease: I. Diagnosis and staging working group report. Biol Blood Marrow Transplant. 2005; 11:945-56., for grading criteria. (See Citation: Filipovich AH et al) The incidence of patients with acute GVHD (Grade II-IV) was determined at 180 days. The incidence of Chronic GVHD by 2 years was reported
Time Frame
Up to 2 years
Title
Overall Survival- Percentage of Participants Who Survived at 2 Years and 5 Years
Description
We reported overall survival at 2 years and 5 years after transplant
Time Frame
up to 5 years

10. Eligibility

Sex
All
Minimum Age & Unit of Time
1 Year
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Patients will be eligible for this study if they have any one of the diseases that are known to be cured after allogeneic stem cell transplantation. Relapsed or refractory acute leukemia (myeloid or lymphoid) Acute leukemia in first remission at high-risk for recurrence Chronic myelogenous leukemia in accelerated phase or blast-crisis Chronic myelogenous leukemia in chronic phase Recurrent or refractory malignant lymphoma or Hodgkin lymphoma Chronic lymphocytic leukemia, relapsed or with poor prognostic features Multiple myeloma Myelodysplastic syndrome Chronic myeloproliferative disease Hemoglobinopathies Aplastic anemia Exclusion Criteria: Zubrod performance status > 2 Life expectancy is severely limited by concomitant illness Patients with severely decreased LVEF or impaired pulmonary function tests(PFT's) Estimated Creatinine Clearance <50 ml/min Serum bilirubin> 2.0 mg/dl or SGPT >3 x upper limit of normal Evidence of chronic active hepatitis or cirrhosis HIV-positive Patient is pregnant Patient or guardian not able to sign informed consent
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Hongtao Liu, M.D.
Organizational Affiliation
University of Chicago
Official's Role
Principal Investigator
Facility Information:
Facility Name
The University of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60637
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
21976674
Citation
Liu H, Rich ES, Godley L, Odenike O, Joseph L, Marino S, Kline J, Nguyen V, Cunningham J, Larson RA, del Cerro P, Schroeder L, Pape L, Stock W, Wickrema A, Artz AS, van Besien K. Reduced-intensity conditioning with combined haploidentical and cord blood transplantation results in rapid engraftment, low GVHD, and durable remissions. Blood. 2011 Dec 8;118(24):6438-45. doi: 10.1182/blood-2011-08-372508. Epub 2011 Oct 5.
Results Reference
result
PubMed Identifier
26869630
Citation
van Besien K, Hari P, Zhang MJ, Liu HT, Stock W, Godley L, Odenike O, Larson R, Bishop M, Wickrema A, Gergis U, Mayer S, Shore T, Tsai S, Rhodes J, Cushing MM, Korman S, Artz A. Reduced intensity haplo plus single cord transplant compared to double cord transplant: improved engraftment and graft-versus-host disease-free, relapse-free survival. Haematologica. 2016 May;101(5):634-43. doi: 10.3324/haematol.2015.138594. Epub 2016 Feb 11.
Results Reference
derived

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Combined Haploidentical-Cord Blood Transplantation for Adults and Children

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