Unrelated Umbilical Cord Blood Transplantation Augmented With ALDHbr Umbilical Cord Blood Cells - Full Text View

Sponsors and Collaborators:	Duke University Aldagen
Information provided by:	Duke University
ClinicalTrials.gov Identifier:	NCT00692926

Purpose

The main purpose of this investigational (not approved by the FDA) Phase I research is to test whether transplantation of umbilical cord blood cells can be safely supplemented with a transfusion of a portion of these cells that have been sorted (collected from a special machine called a cell sorter) and then either infused a few hours after the standard transplant or for some patients grown in a special system in the laboratory prior to the transplant, designed to increase the number of stem cells transplanted. This system is currently in the early phases of testing.

Condition	Intervention	Phase
MDS Anemia, Aplastic Inborn Errors of Metabolism Congenital Marrow Failure Congenital Immunodeficiency Syndrome	Biological: ALDHbr Umbilical Cord Blood Cells	Phase I

Genetics Home Reference related topics: mucopolysaccharidosis type II

MedlinePlus related topics: Anemia

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Single Group Assignment, Safety Study
Official Title:	A Pilot Trial of Unrelated Umbilical Cord Blood Transplantation Augmented With Ex Vivo CytokinePrimed ALDHbr Umbilical Cord Blood Cells

Further study details as provided by Duke University:

Primary Outcome Measures:

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. [ Time Frame: Prospective ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

To describe the biological effect of ex vivo cytokine-primed ALDHbr cells in these patients on neutrophil and platelet engraftment and immune reconstitution. [ Time Frame: prospective ] [ Designated as safety issue: No ]
To describe the clinical outcomes in these patients: - incidence of infections -Non relapse mortality -incidence of acute and chronic graft-vs-host disease -overall survival at 180 days [ Time Frame: prospective ] [ Designated as safety issue: No ]

Estimated Enrollment:	30
Study Start Date:	October 2005
Estimated Study Completion Date:	December 2009
Estimated Primary Completion Date:	December 2009 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
20% primed UCB 20% of UCB is ALDHbr sorted and primed and give on transplant day after conventional graft	Biological: ALDHbr Umbilical Cord Blood Cells ALDHbr sorted Umbilical Cord Blood Cells
20% un-primed 20% of UCB is ALDHbr freshly sorted and give on transplant day 4-8 hrs after conventional graft	Biological: ALDHbr Umbilical Cord Blood Cells ALDHbr sorted Umbilical Cord Blood Cells
Double- 1 unit primed patient receives 1 conventional UCB unit and 1 unit that has been ALDHbr sorted and primed	Biological: ALDHbr Umbilical Cord Blood Cells ALDHbr sorted Umbilical Cord Blood Cells
Double- 1 unit unprimed Patient receives 1 UCB unit and a second UCB unit that has been freshly ALDHbr sorted	Biological: ALDHbr Umbilical Cord Blood Cells ALDHbr sorted Umbilical Cord Blood Cells

Detailed Description:

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% portion)
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)

Eligibility

Ages Eligible for Study:	up to 55 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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 Aldrich Syndrome LAD CGD FEL/HLH CVID/CID
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.

PERFORMANCE STATUS and ORGAN FUNCTION

<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.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00692926

Contacts

Contact: Joanne Kurtzberg, MD	919-668-1100	kurtz001@mc.duke.edu
Contact: Jennifer H. Baker, RN	919-668-1100	baker133@mc.duke.edu

Locations

United States, North Carolina
Duke University Medical Center	Recruiting
Durham, North Carolina, United States, 27705

Sponsors and Collaborators

Duke University

Aldagen

Investigators

Principal Investigator:

Joanne Kurtzberg, MD

Duke University

More Information

Responsible Party:	Aldagen ( Becky Durham )
Study ID Numbers:	7177, BBIND 12289
Study First Received:	June 3, 2008
Last Updated:	November 6, 2008
ClinicalTrials.gov Identifier:	NCT00692926
Health Authority:	United States: Food and Drug Administration

Keywords provided by Duke University:

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

Study placed in the following topic categories:

Hunter-McAlpine syndrome
Mucopolysaccharidosis II
Metabolic Diseases
Hematologic Diseases
Anemia
Immunologic Deficiency Syndromes
Metabolism, Inborn Errors

Genetic Diseases, Inborn
Anemia, Aplastic
Hunter syndrome
Metabolic disorder
Bone Marrow Diseases
Aplastic anemia
Hurler syndrome

Additional relevant MeSH terms:

Pathologic Processes
Disease
Immune System Diseases
Syndrome

ClinicalTrials.gov processed this record on January 16, 2009