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G-CSF PMRD: Granulocyte Colony Stimulating Factor (G-CSF) Stimulated Bone Marrow and In Vivo T-Cell Depletion in Patients With Hematologic Malignancies or Bone Marrow Failure Syndrome (G-CSF PMRD)

Primary Purpose

Hematologic Malignancies

Status
Terminated
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
Granulocyte Colony Stimulating Factor
Sponsored by
Emory University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hematologic Malignancies

Eligibility Criteria

undefined - 22 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Patients with hematologic malignancies or bone marrow failure syndrome who are candidates for allogeneic bone marrow transplantation are eligible for this study. Hematologic malignancies indicated for transplantation: Acute lymphoblastic leukemia (ALL) in first remission (with high risk feature), 2nd or greater remission. Acute myeloid leukemia (AML) in first remission (with high risk feature), 2nd or greater remission. Chronic myeloid leukemia (CML) in 2nd chronic phase or accelerated phase. Juvenile myelomonocytic leukemia (JMML). Myelodysplastic syndrome. Biphenotypic leukemia in first (with high risk feature), 2nd or greater remission. Induction failure leukemia. Refractory relapsed leukemia. Bone marrow failure syndrome. Severe aplastic anemia failed immunotherapy. Patients who do not have a 6 out of 6 matched related or unrelated donor or 4/6 and 5/6 matched cord blood will be eligible for this study. Partially mismatched related donor availability as defined by molecular typing with 3 to 5 HLA matches. Patients who are under 22 years of age. Exclusion Criteria: Patients will not be excluded based on sex, racial, or ethnic background. Patients will be excluded if they demonstrate significant functional deficits in major organs, which would obviously interfere with successful outcome following bone marrow transplant utilizing the following guidelines. Evidence of active, deep-seated, life-threatening infections for which there is no known effective therapy (certain fungal species, HIV, etc.). Patients who have been treated for infections must have appropriate responses as documented by 2 (two) consecutive negative cultures and/or stable radiographic examinations. Patients who have active central nervous system (CNS) leukemic disease. Patients will be excluded if they are women of childbearing potential who are currently pregnant (beta-HCG+) or who are not practicing adequate contraception. Patients who have had a previous hematopoietic stem cell transplant will be excluded. Donors will be excluded if they are sensitive to E. coli-derived protein.

Sites / Locations

  • Children's Healthcare of Atlanta/Emory University

Arms of the Study

Arm 1

Arm Type

Other

Arm Label

Granulocyte Colony Stimulating Factor (G-CSF) stimulation

Arm Description

Participants will receive bone marrow from donors who undergo Granulocyte Colony Stimulating Factor (G-CSF) stimulation prior to bone marrow collection.

Outcomes

Primary Outcome Measures

Engraftment Rate
The number of participants who received in vivo T-cell-depleted G-CSF stimulated bone marrow from partially mismatched related donor who reached engraftment by Day 45.
Incidence of Acute Graft-versus-host Disease
The number of participants diagnosed with new acute graft-versus-host disease (GVHD).
Incidence of Chronic Graft-versus-host Disease
The number of participants diagnosed with chronic graft-versus-host disease (GVHD).

Secondary Outcome Measures

Full Information

First Posted
September 27, 2005
Last Updated
August 10, 2017
Sponsor
Emory University
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1. Study Identification

Unique Protocol Identification Number
NCT00228813
Brief Title
G-CSF PMRD: Granulocyte Colony Stimulating Factor (G-CSF) Stimulated Bone Marrow and In Vivo T-Cell Depletion in Patients With Hematologic Malignancies or Bone Marrow Failure Syndrome
Acronym
G-CSF PMRD
Official Title
Feasibility Study of Using G-CSF Stimulated Bone Marrow and In Vivo T-Cell Depletion in Patients With Hematologic Malignancies or Bone Marrow Failure Syndrome With Partially Mismatched Related Donors
Study Type
Interventional

2. Study Status

Record Verification Date
August 2017
Overall Recruitment Status
Terminated
Why Stopped
lack of enrollment
Study Start Date
April 2004 (undefined)
Primary Completion Date
January 2015 (Actual)
Study Completion Date
January 2015 (Actual)

3. Sponsor/Collaborators

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

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The purposes of this study are: To examine the engraftment rate in patients receiving in vivo T-cell-depleted G-CSF stimulated bone marrow from partially mismatched related donors. To evaluate the incidence and severity of acute and chronic graft-versus-host disease in patients receiving in vivo T-cell-depleted G-CSF stimulated bone marrow from partially mismatched related donors.
Detailed Description
This study is a single-arm, non-randomized feasibility study. Patients meeting the criteria for this study will be entered sequentially until completion or closure of the study. Early stopping rules will be employed to ascertain whether an unacceptable rate of toxicity (non-engraftment, and/or acute GVHD) occurs. Patients will be prepared for transplant through the administration of the following conditioning regimen based on their primary disease: Total body irradiation (1400 rads in 8 fractionated doses) and high dose chemotherapy, including cytosine arabinoside, etoposide, and cyclophosphamide. Patients with bone marrow failure syndrome will not receive etoposide in the conditioning regimen. Post transplant immunosuppression prophylaxis against acute GVHD will include sequential administration of cyclosporine, methotrexate, basiliximab and mycophenolate. The donor will receive 3 daily G-CSF injections prior to marrow harvest starting on day -3. The injections may be initiated by the donor's primary physician prior to donor's arrival, or by the BMT service at Children's Healthcare of Atlanta. Patients will receive daily GM-CSF injections (250 mcg/m2) starting from day +7 post transplant until absolute neutrophil count (ANC) is greater than 2,000/µL for three days.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hematologic Malignancies

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
8 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Granulocyte Colony Stimulating Factor (G-CSF) stimulation
Arm Type
Other
Arm Description
Participants will receive bone marrow from donors who undergo Granulocyte Colony Stimulating Factor (G-CSF) stimulation prior to bone marrow collection.
Intervention Type
Drug
Intervention Name(s)
Granulocyte Colony Stimulating Factor
Intervention Description
FILGRASTIM: G-CSF (NEUPOGEN®) is administered as a short IV infusion over 30 minutes or subcutaneously. It is given beginning on day -3 for 3 days to the donor prior to the bone marrow harvest. Drug Information: FILGRASTIM: G-CSF (Neupogen®) Formulation: G-CSF is available as a preservative-free solution for injection in 1.0 ml and 1.6 ml vials containing 300 mcg/ml. Administration: G-CSF 5 mcg/kg/d will be given subcutaneously or as a short I.V. infusion over 30 minutes. Recombinant GM-CSF at the dose of 250 mcg/m2 will be given intravenously from day +7 to help white counts recovery. The drug will be diluted in NS at a concentration of at least 10 mcg/ml. Drug Information: Sargramostim (Leukine) Formulation: 250 mcg, 500 mcg lyophlized powder for injection
Primary Outcome Measure Information:
Title
Engraftment Rate
Description
The number of participants who received in vivo T-cell-depleted G-CSF stimulated bone marrow from partially mismatched related donor who reached engraftment by Day 45.
Time Frame
Day 45
Title
Incidence of Acute Graft-versus-host Disease
Description
The number of participants diagnosed with new acute graft-versus-host disease (GVHD).
Time Frame
Day 100
Title
Incidence of Chronic Graft-versus-host Disease
Description
The number of participants diagnosed with chronic graft-versus-host disease (GVHD).
Time Frame
Duration of Study (Up to two years)

10. Eligibility

Sex
All
Maximum Age & Unit of Time
22 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Patients with hematologic malignancies or bone marrow failure syndrome who are candidates for allogeneic bone marrow transplantation are eligible for this study. Hematologic malignancies indicated for transplantation: Acute lymphoblastic leukemia (ALL) in first remission (with high risk feature), 2nd or greater remission. Acute myeloid leukemia (AML) in first remission (with high risk feature), 2nd or greater remission. Chronic myeloid leukemia (CML) in 2nd chronic phase or accelerated phase. Juvenile myelomonocytic leukemia (JMML). Myelodysplastic syndrome. Biphenotypic leukemia in first (with high risk feature), 2nd or greater remission. Induction failure leukemia. Refractory relapsed leukemia. Bone marrow failure syndrome. Severe aplastic anemia failed immunotherapy. Patients who do not have a 6 out of 6 matched related or unrelated donor or 4/6 and 5/6 matched cord blood will be eligible for this study. Partially mismatched related donor availability as defined by molecular typing with 3 to 5 HLA matches. Patients who are under 22 years of age. Exclusion Criteria: Patients will not be excluded based on sex, racial, or ethnic background. Patients will be excluded if they demonstrate significant functional deficits in major organs, which would obviously interfere with successful outcome following bone marrow transplant utilizing the following guidelines. Evidence of active, deep-seated, life-threatening infections for which there is no known effective therapy (certain fungal species, HIV, etc.). Patients who have been treated for infections must have appropriate responses as documented by 2 (two) consecutive negative cultures and/or stable radiographic examinations. Patients who have active central nervous system (CNS) leukemic disease. Patients will be excluded if they are women of childbearing potential who are currently pregnant (beta-HCG+) or who are not practicing adequate contraception. Patients who have had a previous hematopoietic stem cell transplant will be excluded. Donors will be excluded if they are sensitive to E. coli-derived protein.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Kuang-Yueh Chiang, M.D.
Organizational Affiliation
Children's Healthcare of Atlanta/Emory University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Children's Healthcare of Atlanta/Emory University
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States

12. IPD Sharing Statement

Learn more about this trial

G-CSF PMRD: Granulocyte Colony Stimulating Factor (G-CSF) Stimulated Bone Marrow and In Vivo T-Cell Depletion in Patients With Hematologic Malignancies or Bone Marrow Failure Syndrome

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