CD34+ Enriched Transplants to Treat Myelodysplastic Syndrome

Trial Title: CD34+ Enriched Transplants to Treat Myelodysplastic Syndrome

NCT ID: NCT05617625

Condition: Myelodysplastic Syndromes
Graft Vs Host Disease
Graft-versus-host-disease

Conditions: Official terms:
Preleukemia
Myelodysplastic Syndromes
Graft vs Host Disease
Syndrome
Fludarabine
Melphalan
Busulfan

Conditions: Keywords:
stem cell transplant
allogeneic transplant

Study type: Interventional

Study phase: Phase 2

Overall status: Suspended

Study design:

Allocation: N/A

Intervention model: Single Group Assignment

Primary purpose: Treatment

Masking: None (Open Label)

Intervention:

Intervention type: Drug
Intervention name: Busulfan
Description: 0.8 mg/kg q6h x 12 doses via IV injection on Days -9, -8, and -7 prior to transplant
Arm group label: CD34+ Peripheral Blood Progenitor Cell (PBSC) Transplant with Busulfan/Melphalan/Fludarabine Regimen

Other name: Busulfex

Intervention type: Drug
Intervention name: Melphalan
Description: 70 mg/m^2/day x 2 days via IV infusion over 30 minutes on Days -6 and -5 prior to transplant
Arm group label: CD34+ Peripheral Blood Progenitor Cell (PBSC) Transplant with Busulfan/Melphalan/Fludarabine Regimen

Other name: Alkeran

Intervention type: Drug
Intervention name: Fludarabine
Description: 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
Arm group label: CD34+ Peripheral Blood Progenitor Cell (PBSC) Transplant with Busulfan/Melphalan/Fludarabine Regimen

Other name: Fludara

Intervention type: Device
Intervention name: CliniMACS CD34+ enriched, T-cell depleted peripheral blood stem cell (PBSC)
Description: 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.
Arm group label: CD34+ Peripheral Blood Progenitor Cell (PBSC) Transplant with Busulfan/Melphalan/Fludarabine Regimen

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 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.

Criteria for eligibility:
Criteria:
Inclusion Criteria: - Myelodysplastic syndrome (MDS): Refractory anemia/refractory anemia with ring sideroblasts/refractory cytopenia with multilineage anemia (RA/RARS/RCMA) with high-risk cytogenetic features or transfusion dependence, as well as Refractory Anemia with Excess Blasts Type 1 and 2 (RAEB-1 and RAEB-2) - Karnofsky (adult) Performance Status of at least 70% - Adequate organ function measured by: - Cardiac: asymptomatic or if symptomatic then left ventricular ejection fraction (LVEF) at rest must be 50% and must improve with exercise. - Hepatic: < 3x upper limit of normal (ULN) aspartate aminotransferase (AST) and: 1.5 total serum bilirubin, unless there is congenital benign hyperbilirubinemia or if the hyperbilirubinemia is directly caused by the disease in which the patient is receiving a transplant [e.g., acute myeloid leukemia (AML) Chloroma obstructing the biliary tree]. Patients with higher bilirubin levels due to causes other than active liver disease is also eligible with Pl approval e.g., patients with paroxysmal nocturnal hemoglobinuria (PNH), Gilbert's disease or other hemolytic disorders. - Renal: serum creatinine: <1.2 mg/dL (normal range 0.7-1.3) or if serum creatinine is outside the normal range, then creatinine clearance (CrCl) > 5940 mL/min (measured or calculated/estimated). - Pulmonary: asymptomatic or if symptomatic, diffusion capacity of lung for carbon monoxide (DLCO) 50% of predicted (corrected for hemoglobin). - Willing to participate and must sign an informed consent form Exclusion Criteria: - Pregnant or breast-feeding - Active viral, bacterial or fungal infection - Patient seropositive for human immunodeficiency virus (HIV)-I /II; human T-lymphotropic virus (HTLV)-I /II - Presence of leukemia in the central nervous system (CNS)

Gender: All

Minimum age: 18 Years

Maximum age: 75 Years

Healthy volunteers: No

Locations:

Facility:
Name: Miami Cancer Institute

Address:
City: Miami
Zip: 33176
Country: United States

Start date: October 2024

Completion date: October 2031

Lead sponsor:
Agency: Guenther Koehne
Agency class: Other

Source: Baptist Health South Florida

Record processing date: ClinicalTrials.gov processed this data on November 12, 2024

Source: ClinicalTrials.gov page: https://clinicaltrials.gov/ct2/show/NCT05617625