CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT01966367|
Recruitment Status : Recruiting
First Posted : October 21, 2013
Last Update Posted : July 17, 2017
|Condition or disease||Intervention/treatment||Phase|
|Bone Marrow Failure Syndrome Severe Aplastic Anemia Severe Congenital Neutropenia Amegakaryocytic Thrombocytopenia Diamond-Blackfan Anemia Schwachman Diamond Syndrome Primary Immunodeficiency Syndromes Acquired Immunodeficiency Syndromes Histiocytic Syndrome Familial Hemophagocytic Lymphocytosis Lymphohistiocytosis Macrophage Activation Syndrome Langerhans Cell Histiocytosis (LCH) Hemoglobinopathies Sickle Cell Disease Sickle Cell-beta-thalassemia||Biological: CD34 Stem Cell Selection Therapy||Early Phase 1|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||25 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||CD34+ Stem Cell Selection for Patients Receiving a Matched or Partially Matched Family or Unrelated Adult Donor Allogeneic Stem Cell Transplantation for Non-Malignant Disease|
|Study Start Date :||March 2013|
|Estimated Primary Completion Date :||December 2019|
|Estimated Study Completion Date :||December 2019|
Experimental: CliniMacs PLUS followed by chemotherapy
Patients will receive a pre-transplant conditioning regimen of Busulfan Fludarabine and Alemtuzumab. For patients with pre-transplant hepatic dysfunction, Melphalan will be substituted for the Busulfan. For patients receiving a second transplant or a "boost", pre-transplant conditioning based on the clinical condition of the patient will be determined by the Principal Investigator and the patient's bone marrow transplantation (BMT) physician. The donor peripheral blood stem cells will undergo CD34+ selection (Biological/Vaccine: CD34 Stem Cell Selection Therapy). The CliniMacs (PLUS) Reagent System will be used to remove T-cells from the peripheral blood stem cell transplant in order to decrease the risk of acute and chronic graft versus host disease (GVHD).
Biological: CD34 Stem Cell Selection Therapy
The CliniMacs (PLUS) Reagent System (Miltenyi CliniMacs CD34+ Cell Selection Device) will be used to remove T-cells from the peripheral blood stem cell transplant in order to decrease the risk of acute and chronic graft versus host disease (GVHD).
- Incidence of acute graft versus host disease (GVHD) [ Time Frame: 100 days ]Determine the incidence and severity of acute GVHD.
- Incidence of primary graft failure [ Time Frame: 1 year ]Quantify the incidence of primary and secondary graft failure.
- Survival Rate [ Time Frame: 5 years ]To assess event free survival and overall survival
- Time to neutrophil and platelet engraftment [ Time Frame: 1 year ]To determine the time to neutrophil and platelet engraftment
- Time to immune reconstitution [ Time Frame: 2 years ]To determine the time to immune reconstitution (including normalization of T, B and NK cell repertoire and Immunoglobulin G production)
- Incidence of infectious complications [ Time Frame: 2 years ]To establish the incidence of infectious complications including bacterial, viral, fungal and atypical mycobacterial and other infections
- Incidence of secondary graft failure [ Time Frame: 1 year ]Quantify the incidence of primary and secondary graft failure.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01966367
|Contact: Diane George, MDemail@example.com|
|Contact: Jean Sosna, RNfirstname.lastname@example.org|
|United States, New York|
|Morgan Stanley Children's Hospital, New York-Presbyterian, Columbia University||Recruiting|
|New York, New York, United States, 10032|
|Contact: Jean Sosna, RN email@example.com|
|Contact: Danielle Dietzen, CPNP 212-305-8443 firstname.lastname@example.org|
|Principal Investigator: Diane George, MD|
|Principal Investigator:||Diane George, MD||Columbia University|