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A Phase II Study Using the CliniMACS® Device for CD34+ Cell Selection and T Cell Depletion for Graft-versus-Host Disease Prophylaxis in Alternative Donor Stem Cell Transplant Recipients

Phase 2
30 Years
Open (Enrolling)
Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Chronic Myeloid Leukemia, Myelodysplastic Syndrome, Lymphomas, Bone Marrow Failure, Hemoglobinopathy, Immune Deficiency, Osteopetrosis

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Trial Information

A Phase II Study Using the CliniMACS® Device for CD34+ Cell Selection and T Cell Depletion for Graft-versus-Host Disease Prophylaxis in Alternative Donor Stem Cell Transplant Recipients

A major issue in alternative donor (mismatched related and unrelated donor transplantation
is the development of graft-versus-host disease (GVHD). Several clinical trials have shown
that the use of T-cell depleted peripheral blood stem cells (PBSC) reduces GVHD in
alternative donor transplants. The purpose of this study is to determine the ability of
CD34 positive selection and T cell depletion using the CliniMACS® Device as the only GVHD
prophylaxis to prevent severe acute GVHD in recipients of an alternative donor PBSC
transplant. Mismatched related donors will match at least 3 of 6 Human leukocyte
antigens(HLA)(haplocompatible) and unrelated donors will match at least 6 out of 8 HLA
antigens with the transplant recipient. The conditioning therapy including chemotherapy,
anti-thymocyte globulin (ATG), +/- total body irradiation (TBI) will be based on the
patient's diagnosis. The transplant recipient will be followed for 5 years after transplant
for GVHD, engraftment, post-transplant infections, disease relapse, and overall survival.
In addition, this study will serve as a platform for a companion study of therapy to
accelerate immune recovery after transplant.

Inclusion Criteria:

- Age < 30 years

- Patient must have a malignant or non-malignant disease that can benefit from
alternative stem cell transplantation. Examples include acute and chronic leukemias,
myelodysplastic syndrome, lymphoma, severe acquired and congenital cytopenias, white
and red blood cell abnormalities, and immunodeficiencies.

- Patients with acute lymphoblastic leukemia must be in morphological remission (< 5%
blasts) at the time of transplant. Patients with acute non-lymphocytic leukemia will
preferably be in morphologic remission but may be enrolled when aplastic after
chemotherapy or with < 20% blasts. Patients with lymphoma must be in complete or
close to complete remission (if residual adenopathy, PET scan must be negative or
only have slight uptake, eg. SUV < 2).

- Patients must lack a healthy HLA-identical related donor of at least one year of age.

- Patient must have a mismatched related or an unrelated donor who is:

1. Able to receive G-CSF and undergo apheresis either through placement of
catheters in antecubital veins or a temporary central venous catheter,

2. Healthy,

3. Willing,

4. d) For recipients of an unrelated donor transplant, recipient eligibility will
be restricted as follows if in the judgment of the recipients' transplant
physician, the recipient cannot receive a transplant with combined positive and
negative fractions as described in Section or an unmanipulated PBSC

5. Meets eligibility criteria for donors.

- If only one mismatched related relative is available, an acceptable unrelated donor
must be identified as a backup.

- Patient or authorized guardian must sign informed consent for this study.

Exclusion Criteria:

- Patient with an anticipated life expectancy of < 1 month

- Active infectious hepatitis or CMV infection

- HIV or HTLV-I/II infection

- Serious infection (bacterial, fungal, viral) within the last 4 weeks

- Cardiac ejection fraction < 45%; can be lower if patient is not in clinical cardiac
failure and a reduced intensity conditioning regimen is used.

- Creatinine clearance <60 ml/min/1.72 m2; can be lower if a reduced intensity
conditioning regimen is used.

- Pulmonary diffusion capacity (adjusted for Hgb), FEV1, or FVC <60% of predicted or O2
sat < 94% if unable to perform PFTs; can be lower if a reduced intensity conditioning
regimen is used.

- Serum ALT > 3 x upper limit of normal (can be up to 5 x upper limit of normal if a
reduced intensity conditioning regimen is used) or bilirubin > 2.

- Performance score (Lanksy/Karnofsky) < 50

- Any condition that compromises compliance with the procedures of this protocol, as
judged by the principal investigator.

Type of Study:


Study Design:

Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Determine rate of severe GVHD.

Outcome Time Frame:

Within 30 days after stem cell transplant

Safety Issue:


Principal Investigator

Andrew Gilman, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Department of Pediatrics, Levine Children's Hospital, Carolinas Healthcare System


United States: Food and Drug Administration

Study ID:

LCH BMT 09-01



Start Date:

August 2009

Completion Date:

June 2019

Related Keywords:

  • Acute Lymphoblastic Leukemia
  • Acute Myeloid Leukemia
  • Chronic Myeloid Leukemia
  • Myelodysplastic Syndrome
  • Lymphomas
  • Bone Marrow Failure
  • Hemoglobinopathy
  • Immune Deficiency
  • Osteopetrosis
  • T cell depleted
  • Matched unrelated donors
  • Haplocompatible donors
  • Graft vs Host Disease
  • Hemoglobinopathies
  • Leukemia
  • Leukemia, Lymphoid
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma
  • Leukemia, Myeloid, Acute
  • Leukemia, Myeloid
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive
  • Lymphoma
  • Myelodysplastic Syndromes
  • Preleukemia
  • Osteopetrosis
  • Pancytopenia



Levine Children's Hospital, Carolinas Medical CenterCharlotte, North Carolina  28204