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Phase 2 Haplotype Mismatched HSCT in Patients With Hematological Malignancies

Primary Purpose

Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Myelodysplasia

Status
Terminated
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Total Body Irradiation
Thiotepa
Fludarabine
Rabbit ATG
Palifermin
Sponsored by
Sherif S. Farag
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Acute Myeloid Leukemia

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Patients must have histologically documented AML, ALL, MDS, CML, Acute myeloid leukemia (AML) with one or more of the following criteria

    • CR 1 with poor risk features
    • CR 2, or higher order CR

  • Acute lymphoblastic leukemia (ALL) with one of the following criteria

    • CR 1 with poor risk features
    • CR 2, or higher order CR
  • Myelodysplasia, RAEB I
  • Donor has been identified
  • Age ≤ 65 years.
  • Performance Status 0-1.

Exclusion Criteria:

  • Patients relapsing <6 months after autologous SCT are not eligible.
  • Patients with active infections requiring oral or intravenous antibiotics are not eligible for enrollment until resolution of infection.
  • Non-pregnant and non-nursing

Sites / Locations

  • Indiana University Cancer Center

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Experimental

Arm Label

1

2

Arm Description

Total body Irradiation; Thiotepa; Fludarabine; Rabbit ATG;

Palifermin; Total Body Irradiation; Thiotepa; Fludarabine; Rabbit ATG

Outcomes

Primary Outcome Measures

Treatment-related Mortality (TRM) Rate at 6 Months After Transplantation
To determine if haplotype-mismatched HSCT is associated with a ≤40% treatment-related mortality (TRM) rate at 6 months after transplantation; a TRM ≥60% being considered unacceptable. The percent of patients with the exact 95% confidence interval who had treatment-related mortality within 6 months of their transplant is presented.

Secondary Outcome Measures

Regimen-related Toxicity
The number of unique patients who had adverse events that were possibly/probably/definitely related to treatment/regimen.
Time to Neutrophil Engraftment
Time to neutrophil engraftment will be analyzed by the Kaplan-Meier method. The time to engraftment of neutrophil is defined as the time from transplant until absolute neutrophil count (ANC) > 500 uL for 3 consecutive days. The median and 95% confidence intervals will be provided.
Time to Platelet Engraftment
Time to platelet engraftment will be analyzed by the Kaplan-Meier method. The time to engraftment of platelets is defined as the time from day 0 to the first of seven consecutive days after transplantation during which the platelet count is at least 20 x109/l without transfusion support. Only patients who achieved engraftment of platelets will be included in the analysis. The median and 95% confidence intervals will be provided.
Acute Graft vs. Host Disease (GvHD)
Number of unique patients who had acute Graft vs. Host Disease (GvHD) diagnosed while on the study.
Chronic Graft vs. Host Disease (GvHD)
Number of unique patients who had chronic Graft vs. Host Disease (GvHD) diagnosed while on the study.
Frequency of Infection
Number of unique patients with bacterial and/or viral infections reported.

Full Information

First Posted
January 4, 2008
Last Updated
March 12, 2018
Sponsor
Sherif S. Farag
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1. Study Identification

Unique Protocol Identification Number
NCT00593554
Brief Title
Phase 2 Haplotype Mismatched HSCT in Patients With Hematological Malignancies
Official Title
A Phase II Trial of Haplotype Mismatched Hematopoietic Stem Cell Transplantation Using Highly Purified CD34 Cells in Patients With Hematological Malignancies
Study Type
Interventional

2. Study Status

Record Verification Date
March 2018
Overall Recruitment Status
Terminated
Why Stopped
Slow accrual
Study Start Date
August 7, 2007 (Actual)
Primary Completion Date
August 27, 2016 (Actual)
Study Completion Date
July 28, 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Sherif S. Farag

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The purpose of this study is to determine if haplotype-mismatched HSCT is associated with an improvement in treatment-related mortality (TRM) rate at 6 months.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Myelodysplasia, Chronic Myeloid Leukemia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
9 (Actual)

8. Arms, Groups, and Interventions

Arm Title
1
Arm Type
Active Comparator
Arm Description
Total body Irradiation; Thiotepa; Fludarabine; Rabbit ATG;
Arm Title
2
Arm Type
Experimental
Arm Description
Palifermin; Total Body Irradiation; Thiotepa; Fludarabine; Rabbit ATG
Intervention Type
Radiation
Intervention Name(s)
Total Body Irradiation
Intervention Description
8 Gy on Day -9
Intervention Type
Drug
Intervention Name(s)
Thiotepa
Intervention Description
5 mg/kg/d on Day -8 to -7
Intervention Type
Drug
Intervention Name(s)
Fludarabine
Intervention Description
40 mg/m2/d on Day -6 to -3
Intervention Type
Biological
Intervention Name(s)
Rabbit ATG
Other Intervention Name(s)
Antithymocyte globulin, Thymoglobulin
Intervention Description
2.5 mg/kg/d on Day -5 to -2
Intervention Type
Drug
Intervention Name(s)
Palifermin
Other Intervention Name(s)
Recombinant human keratinocyte growth factor, Kepivance
Intervention Description
60 ug/kg (actual body weight) on Day -9 to -7 and Day 0 to +2
Primary Outcome Measure Information:
Title
Treatment-related Mortality (TRM) Rate at 6 Months After Transplantation
Description
To determine if haplotype-mismatched HSCT is associated with a ≤40% treatment-related mortality (TRM) rate at 6 months after transplantation; a TRM ≥60% being considered unacceptable. The percent of patients with the exact 95% confidence interval who had treatment-related mortality within 6 months of their transplant is presented.
Time Frame
thru 6 months after transplant
Secondary Outcome Measure Information:
Title
Regimen-related Toxicity
Description
The number of unique patients who had adverse events that were possibly/probably/definitely related to treatment/regimen.
Time Frame
Up to 1 year
Title
Time to Neutrophil Engraftment
Description
Time to neutrophil engraftment will be analyzed by the Kaplan-Meier method. The time to engraftment of neutrophil is defined as the time from transplant until absolute neutrophil count (ANC) > 500 uL for 3 consecutive days. The median and 95% confidence intervals will be provided.
Time Frame
Transplant (Day 0) up to 1 year
Title
Time to Platelet Engraftment
Description
Time to platelet engraftment will be analyzed by the Kaplan-Meier method. The time to engraftment of platelets is defined as the time from day 0 to the first of seven consecutive days after transplantation during which the platelet count is at least 20 x109/l without transfusion support. Only patients who achieved engraftment of platelets will be included in the analysis. The median and 95% confidence intervals will be provided.
Time Frame
Transplant (Day 0) up to 1 year
Title
Acute Graft vs. Host Disease (GvHD)
Description
Number of unique patients who had acute Graft vs. Host Disease (GvHD) diagnosed while on the study.
Time Frame
Up to 1 year
Title
Chronic Graft vs. Host Disease (GvHD)
Description
Number of unique patients who had chronic Graft vs. Host Disease (GvHD) diagnosed while on the study.
Time Frame
Up to 1 year
Title
Frequency of Infection
Description
Number of unique patients with bacterial and/or viral infections reported.
Time Frame
Day 0 through 1 year post transplantation

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Patients must have histologically documented AML, ALL, MDS, CML, Acute myeloid leukemia (AML) with one or more of the following criteria CR 1 with poor risk features CR 2, or higher order CR Acute lymphoblastic leukemia (ALL) with one of the following criteria CR 1 with poor risk features CR 2, or higher order CR Myelodysplasia, RAEB I Donor has been identified Age ≤ 65 years. Performance Status 0-1. Exclusion Criteria: Patients relapsing <6 months after autologous SCT are not eligible. Patients with active infections requiring oral or intravenous antibiotics are not eligible for enrollment until resolution of infection. Non-pregnant and non-nursing
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Sherif Farag, MD/PhD
Organizational Affiliation
Indiana University School of Medicine
Official's Role
Principal Investigator
Facility Information:
Facility Name
Indiana University Cancer Center
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States

12. IPD Sharing Statement

Learn more about this trial

Phase 2 Haplotype Mismatched HSCT in Patients With Hematological Malignancies

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