Clinical Trial Details
— Status: Active, not recruiting
Administrative data
NCT number |
NCT01966367 |
Other study ID # |
AAAL0156 |
Secondary ID |
|
Status |
Active, not recruiting |
Phase |
Phase 1/Phase 2
|
First received |
|
Last updated |
|
Start date |
March 2013 |
Est. completion date |
February 2030 |
Study information
Verified date |
June 2024 |
Source |
Columbia University |
Contact |
n/a |
Is FDA regulated |
No |
Health authority |
|
Study type |
Interventional
|
Clinical Trial Summary
This study's goal is to determine the frequency and severity of acute graft versus host
disease, to evaluate incidence of primary and secondary graft rejection, to assess event free
survival and overall survival, to determine the time to neutrophil and platelet engraftment,
to determine the time to immune reconstitution (including normalization of T, B and natural
killer (NK) cell repertoire and Immunoglobulin G production), and to establish the incidence
of infectious complications including bacterial, viral, fungal and atypical mycobacterial and
other infections following CD34+ selection in children, adolescents and young adults
receiving an allogeneic peripheral blood stem cell transplant from a family member or
unrelated adult donor for a non-malignant disease.
Description:
Graft-versus-host disease (GVHD) is a condition that results from a reaction of transplanted
donor T-lymphocytes against the body and organs of the patient receiving the transplanted
cells. There are two forms: acute (early) and chronic (late). Acute GVHD may produce skin
rashes, liver disease, diarrhea, and an increased risk of infection. Chronic GVHD can appear
in patients without prior acute GVHD. Chronic GVHD may also produce skin rashes, liver
disease, diarrhea and an increased risk of infection. GVHD can make patients very sick, and
have GVHD can make it more likely that patients will not survive their transplant. In this
study, the investigators are offering to treat the donor peripheral blood stem cells in the
hope that it will make it less likely for the patient who receives them from having GVHD.
Patients on this study are being offered an experimental treatment involving the use of the
CliniMACS® Reagent System (Miltenyi Biotec, Germany), a CD34+ selection device to remove
T-cells from the peripheral blood stem cell transplant in order to decrease the risk of acute
and chronic GVHD. CD34+ stem cells are selected from the donor's peripheral blood stem cells.
In doing this, T-cells are also removed. T-cells are the cells which are responsible for
graft versus host disease (GVHD). This study is a clinical trial for patients diagnosed with
a non-malignant disease who will receive a peripheral blood stem cell transplant. Patients
with the following types of non-malignant diseases can participate in this study: Bone marrow
failure syndromes (including Severe Aplastic Anemia, Severe Congenital Neutropenia,
Amegakaryocytic Thrombocytopenia (Kostmann's Syndrome), Diamond-Blackfan Anemia, Schwachman
Diamond Syndrome, Primary Immunodeficiency Syndromes, Acquired Immunodeficiency Syndromes,
and Histiocytic Disorders) and Hemoglobinopathies (including Sickle Cell Anemia and
Sickle/Beta Thalassemia). Patients on this study will be given standard transplant therapy
with either high doses of chemotherapy drugs or lower doses of chemotherapy drugs, depending
on their disease. Diseases within each disease group will receive chemotherapy that is
standard for that condition.
Some patients on this study will receive an allogeneic stem cell transplant (AlloSCT) from a
matched related donor. If a patient does not have a matched related donor, a bone marrow
search will be done at all of the bone marrow banks in the world. The patient will then go on
to receive an AlloSCT from either a partially matched family member or an unrelated adult
stem cell transplant donor. The transplanted cells will allow all the normal parts of the
patient's blood system to recover. The experimental portion of this treatment involves the
use of a Miltenyi CliniMACS CD34+ selection device to remove T-cells from the peripheral
blood stem cell transplant in order to decrease the risk of acute and chronic GVHD. CD34+
stem cell selection AlloSCT has been studied in adults with the malignant and non-malignant
disease with successful engraftment and has shown some improvement in GVHD. It is unknown if
CD34+ stem cell selection will work to prevent severe GVHD in children and adolescents.