CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant | Aplastic Anemia and MDS International Foundation

Clinical Trial: NCT02061800

CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant
For more details on this clinical trial, including contact information, please see this trial’s listing on clinicaltrials.gov:
Purpose: 

CD34+ stem cells (the cells that make all the types of blood cells in the body) are selected (removed) from the donor's peripheral blood stem cells. In doing this, T-cells (a type of blood cell) are also removed. T-cells are the cells which are responsible for severe acute and chronic graft versus host disease (GVHD). GVHD is a condition that results from a reaction of the transplanted donor T-lymphocytes (a kind of white blood cell) against the body and organs. 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 also appear in subjects without prior acute GVHD. Chronic GVHD may also produce skin rashes, liver disease, diarrhea and an increased risk of infection. Chronic GVHD may be mild and respond to agents which suppress the immune system, or it could be very severe. It may also last for over a year.

Once this CD34 selection process is complete, the CD34+ stem cell AlloSCT is given to the recipient without most of the T-cells to see if this therapy will lessen the incidence and seriousness of graft versus host disease (GVHD). CD34+ stem cell selection AlloSCT has been studied in adults with malignant and non-malignant disease with successful engraftment and has shown some improvement in GVHD. We do not know if CD34+ stem cell selection will work to prevent severe GVHD in children, adolescents and young adults.

Subjects are being offered this 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. There will be about 25 subjects participating in this study at Columbia University Medical Center. The purpose of this study is to learn more about the effects of CD34+ stem cell selection on GVHD in children, adolescents and young adults.

The goals of this study are:

  • To determine how often acute GVHD occurs and how severe the acute GVHD is in the children, adolescents and young adults receiving CD34+ selection of their peripheral blood stem cell transplant from a family member or adult unrelated donor.
  • To determine how often primary graft failure and secondary graft failure occurs in the children, adolescents and young adults receiving a peripheral blood stem cell transplant from a family member or adult unrelated donor.
  • To see how quickly neutrophils (infection fighting white blood cells) will recover following CD34+ selection of their peripheral blood stem cell transplant from a family member or adult unrelated donor.
  • To see if the time for the immune system (the body's defense system) to recover will benefit from receiving CD34+ selection of their peripheral blood stem cell transplant from a family member or adult unrelated donor.
  • To see how often bacterial, viral, fungal and atypical mycobacteria (TB-like) infections will occur in the children, adolescents and young adults receiving CD34+ selection of their peripheral blood stem cell transplant from a family member or adult unrelated donor.

Study specific requirements occur up to 2 years post-transplant, although the study team will collect data on the progress of subjects yearly until termination of the study to observe if subjects are responding to therapy and have no worsening GVHD.

Status: 
Recruiting
Study Date: 
Tue, 02/11/2014 to Fri, 12/01/2017
Bone Marrow Disease(s): 
aplastic anemia
myelodysplastic syndromes (MDS)
Associated Drug(s): 
Intervention: 
Device: CliniMACS CD34+ Reagent System The CliniMACS® Reagent System (Miltenyi Biotec, Bergisch Gladbach, Germany), is a semi-automated immunomagnetic cell selection medical device that is used in vitro to select and enrich specific cell populations in a closed, sterile environment. The system is comprised of a computer controlled medical device containing a permanent magnet, a closed-system sterile tubing set containing columns coated with a ferromagnetic matrix, and a magnetic cell specific labeling reagent. Drug: Thiotepa Thiotepa should be diluted in NS (1-5 mg/ml) and infused over 2 hrs on Days -5, -4. IV fluids should be at maintenance rate (1500 ml/m2). It is recommended that total parental nutrition not be used during Thiotepa administration as amino acid infusions may interfere with Thiotepa metabolism. Other Name: Thioplex Drug: Cyclophosphamide Cyclophosphamide (Cytoxan) should be infused over one hour. The drug can be diluted in D5W, NS, or other solutions (250cc) to a maximum concentration of 20 mg/mL. Other Names: Cytoxan CTX Drug: Alemtuzumab Each dose of alemtuzumab is to be diluted in D5W or NS (maximum concentration: 0.3 mg/mL) for IV infusion over two hours. Other Name: Campath Drug: Tacrolimus Tacrolimus dosing will be 0.03mg/kg/24 hours as continuous IV infusion or 0.12 mg/kg/day po divided Q8-12 hr Other Names: Prograf FK506 Drug: Melphalan Melphalan 45mg/m2 (1.5 mg/kg IV for children <1 year of age or <10 kg) diluted in 0.9% NS to a concentration of 0.1- 0.45mg/ml, given IV over 30 minutes. Other Name: Alkeran Drug: Busulfan (Busulfex) will be given IV in 0.9% sodium chloride or D5W to a final solution for infusion equal to 10 times the volume of diluent to Busulfex (to a concentration >0.5 mg/mL), through a central venous access device over 2 hours. Other Name: Busulfex Drug: Fludarabine Fludarabine will be given IV in 50-100 ml of D5W or 0.9% sodium chloride, over 30 minutes. Other Name: Fludara Drug: Methylprednisolone Methylprednisolone will be give IV slow infusion over 15-30 minutes. Other Name: Solu-Medrol