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A Pilot Trial of Unrelated Umbilical Cord Blood Transplantation Augmented With Ex Vivo CytokinePrimed ALDHbr Umbilical Cord Blood Cells

Phase 1
55 Years
Not Enrolling
MDS, Anemia, Aplastic, Inborn Errors of Metabolism, Congenital Marrow Failure, Congenital Immunodeficiency Syndrome

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Trial Information

A Pilot Trial of Unrelated Umbilical Cord Blood Transplantation Augmented With Ex Vivo CytokinePrimed ALDHbr Umbilical Cord Blood Cells

The main purpose of this study is to test whether transplantation of umbilical cord blood
cells can be safely supplemented with transfusion of a portion of these cells that have been
grown in a special system (designed to increase the number of cells transplanted) in the
laboratory prior to the transplant. This system is currently in the early phases of testing
in a FDA-IND-sponsored clinical trial. If the patient consents to participate in this
study, approximately 1/5th (20%) of the cord blood unit selected for the transplant will be
treated per protocol. The first 3 patients will receive ALDHbr sorted cells but not primed
in culture. This is to test the safety of the ALDHbr cells. The treated cells will be
given to the patient on the day of transplant approximately 4 hours after the standard or
conventional transplant which will be given from the 80% fraction of the cord blood unit. A
total of 26 evaluable patients are to be enrolled as outlined below (protocol has been
amended to allow this enrollment):

- 10 evaluable patients who received ALDHbr freshly sorted cells (20% portion)

- 10 evaluable patients who received ALDHbr sorted and cytokine primed cells (20%

- 3 evaluable patients who received a conventional cord blood unit and a cord blood unit
that has been ALDHbr sorted (sort of the UCB unit will be done on Day -1 due to the
time it takes to actually perform the sort)

- 3 evaluable patients who received a conventional cord blood unit and a cord blood unit
that has been ALDHbr sorted and cytokine primed (sort and priming will be done on day
-5 as described later in the protocol)

Inclusion Criteria:

-Hematologic Malignancy: High risk ALL in first complete remission ALL or ANLL in second
or subsequent remission ANLL in relapse MDS CML in any chronic phase or accelerated phase
Severe aplastic anemia refractory to medical therapy The subject is negative for CNS
disease at time of enrollment.

- Inborn errors of metabolism Hurler Syndrome (MPS I) Hurler-Scheie Syndrome Hunter
Syndrome (MPS II) Sanfilippo Syndrome (MPS III) Morquio Syndrome (MPS IV)
Maroteaux-Lamy Syndrome (MPS VI) Krabbe Disease (Globoid Leukodystrophy)
Metachromatic Leukodystrophy (MLD) Adrenoleukodystrophy(ALD and AMN) Sandhoff Disease
Tay Sachs Disease The subject does NOT have uncontrolled seizures, apnea, evidence of
aspiration pneumonia or evidence of brain stem involvement on MRI scans

- Congenital marrow failure Amegakaryocytic thrombocytopenia TAR Kostmann's Syndrome
Schwachman-Diamond Syndrome Blackfan-Diamond Anemia

- Congenital immunodeficiency syndromes requiring myeloablative therapy Wiscott

- SUBJECT'S DONOR Subject does NOT have a 6/6 or 5/6 antigen matched related bone
marrow donor. Suitably matched cord blood unit with adequate cell dose is available.
Unit must be in a dual compartment bag.


- <55 years of age at time of enrollment.

- Lansky score between 60% and 100%, or a Karnofsky score between 50% and 100%

- Adequate function of other organ systems

- Creatinine < 2.0 mg/dl and creatinine clearance > 50 cc/min/m2

- Hepatic transaminases (ALT/AST) < 4 x normal, bilirubin < 2.0 mg/dl

- Normal cardiac function by echocardiogram or radionuclide scan

- Pulmonary function tests demonstrating FVC, CVC, and FEV1 of >60% of predicted for
age. For adult patients DLCO > 60% of predicted. If patient cannot perform PFTs,
clearance by the pediatric or adult pulmonologist will be required

- No uncontrolled infections at the time of cytoreduction

- NOT pregnant or lactating (must have a current negative pregnancy test)

- HIV negative

- Subject is not concurrently involved in any other clinical trial that affects
engraftment or immune reconstitution (e.g. other hematopoietic growth factors).

- Subject does not have any co-morbid condition, which in the view of the Principal
Investigators, renders the patient at too high a risk from treatment complications
and regimen related morbidity/mortality.

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

To assess the safety of infusing ex vivo cytokine-primed ALDHbr cells isolated from banked unrelated umbilical cord blood (CB) into patients also receiving a standard unmanipulated unrelated CB transplant delivering >2.5x10e7 nucleated cells per kg.

Outcome Time Frame:


Safety Issue:


Principal Investigator

Joanne Kurtzberg, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Duke University


United States: Food and Drug Administration

Study ID:




Start Date:

October 2005

Completion Date:

July 2012

Related Keywords:

  • MDS
  • Anemia, Aplastic
  • Inborn Errors of Metabolism
  • Congenital Marrow Failure
  • Congenital Immunodeficiency Syndrome
  • ALDHbr
  • Umbilical Cord Blood
  • Transplantation
  • Hematological malignancy
  • MDS
  • severe aplastic anemia
  • inborn error of metabolism
  • congenital marrow failure or congenital immunodeficiency syndrome
  • Hurler's Disease
  • Tay Sach's
  • PMD
  • Hunter's Syndrome
  • Krabbe
  • MLD
  • ALD
  • ALL
  • AML
  • Anemia
  • Anemia, Aplastic
  • Immunologic Deficiency Syndromes
  • Metabolism, Inborn Errors



Duke University Medical Center Durham, North Carolina  27710