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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

This study is currently recruiting participants. (see Contacts and Locations)
Verified June 2014 by Emory University
Sponsor:
Information provided by (Responsible Party):
Kuang-Yueh Chiang, Emory University
ClinicalTrials.gov Identifier:
NCT00228813
First received: September 27, 2005
Last updated: June 24, 2014
Last verified: June 2014
  Purpose

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.

Condition Intervention
Hematologic Malignancies
Drug: Granulocyte Colony Stimulating Factor

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Emory University:

Primary Outcome Measures:
  • To examine the engraftment rate in patients receiving in vivo T-cell-depleted G-CSF stimulated bone marrow from partially mismatched related donor. [ Time Frame: End of study ] [ Designated as safety issue: No ]
  • 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 donor. [ Time Frame: end of study ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 10
Study Start Date: April 2004
Estimated Study Completion Date: April 2018
Estimated Primary Completion Date: April 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
single arm Drug: Granulocyte Colony Stimulating Factor

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


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.
  Eligibility

Ages Eligible for Study:   up to 22 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00228813

Contacts
Contact: Sindy Moon 404-785-1441 sindy.moon@choa.org
Contact: Jaclyn Smith, MBA 404-785-0692 jaclyn.smith@choa.org

Locations
United States, Georgia
Children's Healthcare of Atlanta/Emory University Recruiting
Atlanta, Georgia, United States, 30322
Contact: Sindy Moon    404-785-1441    sindy.moon@choa.org   
Principal Investigator: Kuang-Yueh Chiang, M.D.         
Sponsors and Collaborators
Emory University
Investigators
Principal Investigator: Kuang-Yueh Chiang, M.D. Children's Healthcare of Atlanta/Emory University
  More Information

No publications provided

Responsible Party: Kuang-Yueh Chiang, Associate Professor, Emory University
ClinicalTrials.gov Identifier: NCT00228813     History of Changes
Other Study ID Numbers: 0159-2004
Study First Received: September 27, 2005
Last Updated: June 24, 2014
Health Authority: United States: Institutional Review Board

Additional relevant MeSH terms:
Hemoglobinuria, Paroxysmal
Neoplasms
Pancytopenia
Anemia
Anemia, Hemolytic
Bone Marrow Diseases
Hematologic Diseases
Myelodysplastic Syndromes
Lenograstim
Adjuvants, Immunologic
Immunologic Factors
Pharmacologic Actions
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on November 27, 2014