A Phase 2 interventional study of Busulfan and Melphalan in Myelodysplastic Syndromes, Graft Vs Host Disease and Graft-versus-host-disease, sponsored by Guenther Koehne. Suspended at 1 site in United States. Open to participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2026-03-03.
Sponsored by Guenther Koehne · Phase 2, Interventional, and Treatment
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 a blood malignancy (cancer) or blood disorder.
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.
2,124 studies on the registry are indexed under Myelodysplastic Syndromes; 320 are open to participants now.
This study's planned enrollment of 50 is above the median of 39 across 1,740 interventional studies indexed under Myelodysplastic Syndromes.
Browse Myelodysplastic Syndromes studies →Guenther Koehne is the lead sponsor of 3 studies on the registry; 1 is open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
1. Cytoreduction therapy: 1. 0.8 mg/kg q6h x 12 doses busulfan via IV injection 2. 70 mg/m\^2/day x 2 days melphalan via IV infusion over 30 minutes 3. 25 mg/m\^2/day x 5 days fludarabine vis IV infusion over 30 minutes 2. CD34+ selected, T-cell depleted, allogeneic PBSC transplant using CliniMACS system to select CD34+ cells
Drug: Busulfan · Drug: Melphalan · Drug: Fludarabine · Device: CliniMACS CD34+ enriched, T-cell depleted peripheral blood stem cell (PBSC)
0.8 mg/kg q6h x 12 doses via IV injection on Days -9, -8, and -7 prior to transplant
Also known as: Busulfex
70 mg/m\^2/day x 2 days via IV infusion over 30 minutes on Days -6 and -5 prior to transplant
Also known as: Alkeran
25 mg/m\^2/days x 5 days via IV infusion over 30 minutes on Days -6, -5, -4, -3, and -2 prior to transplant
Also known as: Fludara
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.
Change in incidence of graft vs. host disease (GVHD)
Incidence of acute and chronic GVHD
Time frame: Weekly until 3 months, monthly until 6 months, 12 months, 24 months
Change in severity of acute graft vs. host disease (GVHD)
Severity of acute GvHD as graded using the International Bone Marrow Transplant Registry Severity Index, which includes assessment of skin, liver, and gut, grading each's severity from 0 to 4 (higher numbers reflecting the more severe disease). The overall assessment is based on the involvement and severity of each of the areas. A = Stage 1 skin involvement, no liver or gut involvement; B = Stage 2 skin involvement, Stage 1 to 2 gut or liver involvement; C = Stage 3 skin, liver or gut involvement; D = Stage 4 skin, liver, or gut involvement.
Time frame: Weekly until 3 months, monthly until 6 months, 12 months, 24 months
Change in severity of chronic graft vs. host disease (GVHD)
Severity of chronic GvHD as graded using the NIH scoring system, which includes assessment of skin, mouth, eyes, GI tract, liver, lungs, joint/fascia, and genital tract and grades the severity of affected organs from 0 to 3 (higher scores reflecting the more severe disease). The overall assessment is based on the number of organs/sites with clinically significant functional impairment (i.e., score 2-3). No GVHD = no organs/sites with significant functional impairment; Mild GVHD = involves two or fewer organs/sites with significant functional impairment; Moderate GVHD = involves three or more organs/sites with significant functional impairment -OR- involves one or zero organs/sites with NO significant functional impairment; Severe GVHD = Major disability caused by chronic GVHD.
Time frame: Weekly until 3 months, monthly until 6 months, 12 months, 24 months
Change in incidence of relapse-free mortality (transplant-related mortality)
Incidence of relapse-free mortality, defined as mortality related to the transplant rather than the disease
Time frame: 6 months, 12 months, 24 months
Change in overall survival
Incidence of overall survival, defined as time from transplant to death or last follow-up.
Time frame: 6 months, 12 months, 24 months
Change in disease-free survival
Incidence of disease-free survival, defined as the minimum time interval of relapse/recurrence, to death or to the last follow-up, from the time of transplant.
Time frame: 6 months, 12 months, 24 months
Proportion of patients receiving optimal vs. suboptimal CD34+/CD3+ PBSC doses
Proportion of patients receiving optimal CD34+ (\>5 x 10\^6/kg) and CD3+ (\< 5 x10\^4/kg) cell doses versus the proportion recurring suboptimal doses (\<3 x 10\^6/kg) CD34+ cells; and the proportion of patients receiving CD3+ T-cell doses (\>5 x 10\^4/kg)
Time frame: Day 0
Plan to share: No
This study is suspended, as verified in Mar 2026. You cannot join it, but the record below documents what was studied.
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Guenther Koehne