Clinical trial NCT02061800: CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant
Official title: CD34+ Stem Cell Selection for Patients Receiving a Matched or Partially Matched Family or Unrelated Adult Donor Allogeneic Stem Cell Transplant for Malignant Disease
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE1
PHASE2
Conditions: Chronic Myeloid Leukemia (CML)
Acute Myelogenous Leukemia (AML)
Myelodysplastic Syndrome (MDS)
Juvenile Myelomonocytic Leukemia (JMML)
Acute Lymphoblastic Leukemia (ALL)
Lymphoma (Hodgkin's and Non-Hodgkin's)
Keywords: Unrelated donor transplant
Allogenic Stem Cell Transplant
Adult Bone Marrow Transplant
Pediatric Bone Marrow Transplant
Related donor transplant
Haploidentical donor transplant
Peripheral blood stem cell transplantation
Non-malignant disease
Malignant disease
Bone marrow failure syndrome
Severe Aplastic Anemia
Severe Congenital Neutropenia
Amegakaryocytic Thrombocytopenia
Diamond-Blackfan Anemia
Schwachman Diamond Syndrome
Primary Immunodeficiency Syndrome
Acquired Immunodeficiency Syndrome
Histiocytic Syndrome
Familial Hemophagocytic Lymphocytosis
Lymphohistiocytosis
Macrophage Activation Syndrome
Langerhans Cell Histiocytosis (LCH)
Hemoglobinopathies
Reduced-Intensity Conditioning
Sickle Cell Disease
Sickle Cell-beta-thalassemia
Lead sponsor: Columbia University
Sponsor class: OTHER
First posted: 2014-02-13
Enrollment: 14
Sex: ALL
Maximum age (years): 22
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to learn more about the effects of (classification determinant) CD34+ stem cell selection on graft versus host disease (GVHD) in children, adolescents, and young adults. CD34+ stem cells are the cells that make all the types of blood cells in the body. GVHD is a condition that results from a reaction of transplanted donor T-lymphocytes (a kind of white blood cell) against the recipient's body and organs. Study subjects will be offered treatment involving the use of the CliniMACS® Reagent System (Miltenyi Biotec), a CD34+ selection device to remove T-cells from a peripheral blood stem cell transplant in order to decrease the risk of acute and chronic GVHD.

This study involves subjects who are diagnosed with a malignant disease, that has either failed standard therapy or is unlikely to be cured with standard non-transplant therapy, who will receive a peripheral blood stem cell transplant. A malignant disease includes the following: Chronic Myeloid Leukemia (CML) in chronic phase, accelerated phase or blast crisis; Acute Myelogenous Leukemia (AML); Myelodysplastic Syndrome (MDS); Juvenile Myelomonocytic Leukemia (JMML); Acute Lymphoblastic Leukemia (ALL); or Lymphoma (Hodgkin's and Non-Hodgkin's).

Detailed description: Graft versus host disease (GVHD) is one of the serious complications following allogeneic stem cell transplantation. The incidence and severity of GVHD increase with the degree of HLA incompatibility between the host and donor. The most reliable way to prevent acute and chronic GVHD is to remove T cells from the graft. However, the incidence of graft failure increases with the efficiency of T cell depletion and low T cell numbers are predictive of graft failure. Immunomagnetic selection of HLA-mismatched CD34+ progenitor cells has demonstrated high levels of T cell depletion and successful engraftment in adult and pediatric patients with the malignant and nonmalignant disease.

Eligibility criteria: Inclusion Criteria: General Eligibility (All Patients)

* Must be \< 22 years of age
* Diagnosed with a malignant disease
* Must be fully informed about their illness and the investigational nature of the study protocol (including foreseeable risks and possible side effects), and must sign an informed consent
* For unrelated donor: A human leukocyte antigen (HLA) 8/10, 9/10 or 10/10 matched unrelated adult donor (MUD) will be required for study entry
* For related donor: A 5/10, 6/10, 7/10, 8/10, 9/10 or 10/10 matched (or partially matched) family donor will be required for study entry
* Adequate renal function
* Adequate liver function
* Adequate cardiac function
* Adequate pulmonary function

Exclusion Criteria:

* Patients with documented uncontrolled infection at the time of study entry are not eligible
* Females who are pregnant or breast feeding at the time of study entry are not eligible

Primary outcomes:
measure: Incidence of acute GVHD
timeFrame: Up to 2 years post-transplant
Secondary outcomes:
measure: Time to neutrophil engraftment
timeFrame: Up to 1 year post-transplant
measure: Time to immune reconstitution
timeFrame: Up to 2 years post-transplant
measure: Incidence of infection complications including bacterial, viral, fungal and atypical mycobacterial and other infections
timeFrame: Up to 100 days post-transplant
measure: Time to platelet engraftment
timeFrame: Up to 1 year post-transplant
measure: Incidence of chronic GVHD
timeFrame: Up to 2 years post-transplant
measure: Severity of acute GVHD
timeFrame: Up to 2 years post-transplant
measure: Severity of chronic GVHD
timeFrame: Up to 2 years post-transplant
measure: Incidence of primary graft failure
timeFrame: 42 (or more) days post-transplant
measure: Incidence of secondary graft failure
timeFrame: 42 (or more) days post-transplant
Other outcomes:
Locations:
city: New York
country: United States
facility: Columbia University Irving Medical Center
state: New York
Study officials:
affiliation: Columbia University
name: Diane George, MD
role: PRINCIPAL_INVESTIGATOR
References: