Clinical Trial: CD34+ Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation for Non-Malignant Disease

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

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

Brief 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.

Detailed Summary:

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 having 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 to have GVHD.

Patients on this study are being offered an experimental treatment involving 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 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 (Kostman'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
Sponsor: Diane George, MD

Current Primary Outcome: Incidence of acute graft versus host disease (GVHD) [ Time Frame: 100 days ]

Determine the incidence and severity of acute GVHD.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • 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.


Original Secondary Outcome: Same as current

Information By: Columbia University

Dates:
Date Received: October 17, 2013
Date Started: March 2013
Date Completion: December 2019
Last Updated: March 10, 2016
Last Verified: March 2016