A Phase II Trial of CD34+ Enriched Transplants From HLA-Compatible Related or Unrelated Donors for Treatment of Patients With Myelodysplastic Syndrome

Summary

This study will evaluate whether processing blood stem cell transplants using an investigational device (the CliniMACS system) results in fewer complications for patients who undergo transplant to treat myelodysplastic syndrome (MDS). The CliniMACS system will be used to remove immune T-cells from the transplant donor's blood. Immune T-cells contribute to graft versus host disease (GVHD) - a serious complication that can happen after transplant. GVHD occurs when a patient's immune system attacks the donor's cells. The study aims to reduce the number of the donor immune T-cells thereby preventing or reducing the severity of GVHD.

Eligibility

Age range

18–72 years

Sex

All

Healthy volunteers

No

Interventions

• Drug
  Busulfan

  0.8 mg/kg q6h × 12 doses via IV injection on Days -9, -8, and -7 prior to transplant

• Drug
  Melphalan

  70 mg/m²/day × 2 days (50 mg/m²/day is patients ≥65 years with a comorbidity score of ≥3) via IV infusion over 30 minutes on Days -6 and -5 prior to transplant

• Drug
  Fludarabine

  25 mg/m²/days × 5 days via IV infusion over 30 minutes on Days -6, -5, -4, -3, and -2 prior to transplant

• Device
  CliniMACS CD34+ enriched, T-cell depleted peripheral blood stem cell (PBSC)

  CliniMACS system will be used to derive CD34+ enriched, T-cell depleted (T-cells limited to 1.0 x 10\^5 CD3+ cells/kg) PBSC for transplant, which will occur on Day 0. PBSC (5 x 10\^6 CD34+ cells/kg) are suspended in a volume of approximately 20-50 mL and delivered via IV infusion over 15 minutes.

Location