CD34+ Enriched Transplants to Treat Myelodysplastic Syndrome

March 2, 2026 updated by: Guenther Koehne

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

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.

Study Overview

Status

Suspended

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

50

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Florida
      • Miami, Florida, United States, 33176
        • Miami Cancer Institute

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 75 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: CD34+ Peripheral Blood Progenitor Cell (PBSC) Transplant with Busulfan/Melphalan/Fludarabine Regimen

  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
0.8 mg/kg q6h x 12 doses via IV injection on Days -9, -8, and -7 prior to transplant
Other Names:
  • Busulfex
Drug: Melphalan
70 mg/m^2/day x 2 days via IV infusion over 30 minutes on Days -6 and -5 prior to transplant
Other Names:
  • Alkeran
Drug: Fludarabine
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
Other Names:
  • Fludara
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in incidence of graft vs. host disease (GVHD)
Time Frame: Weekly until 3 months, monthly until 6 months, 12 months, 24 months
Incidence of acute and chronic GVHD
Weekly until 3 months, monthly until 6 months, 12 months, 24 months
Change in severity of acute graft vs. host disease (GVHD)
Time Frame: Weekly until 3 months, monthly until 6 months, 12 months, 24 months
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.
Weekly until 3 months, monthly until 6 months, 12 months, 24 months
Change in severity of chronic graft vs. host disease (GVHD)
Time Frame: Weekly until 3 months, monthly until 6 months, 12 months, 24 months
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.
Weekly until 3 months, monthly until 6 months, 12 months, 24 months
Change in incidence of relapse-free mortality (transplant-related mortality)
Time Frame: 6 months, 12 months, 24 months
Incidence of relapse-free mortality, defined as mortality related to the transplant rather than the disease
6 months, 12 months, 24 months
Change in overall survival
Time Frame: 6 months, 12 months, 24 months
Incidence of overall survival, defined as time from transplant to death or last follow-up.
6 months, 12 months, 24 months
Change in disease-free survival
Time Frame: 6 months, 12 months, 24 months
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.
6 months, 12 months, 24 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Proportion of patients receiving optimal vs. suboptimal CD34+/CD3+ PBSC doses
Time Frame: Day 0
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)
Day 0

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Guenther Koehne

Investigators

  • Principal Investigator: Guenther Koehne, M.D., Miami Cancer Institute/Baptist Health South Florida

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

June 1, 2026

Primary Completion (Estimated)

June 1, 2033

Study Completion (Estimated)

June 1, 2033

Study Registration Dates

First Submitted

November 8, 2022

First Submitted That Met QC Criteria

November 8, 2022

First Posted (Actual)

November 15, 2022

Study Record Updates

Last Update Posted (Actual)

March 3, 2026

Last Update Submitted That Met QC Criteria

March 2, 2026

Last Verified

March 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2021-KOE-002

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

Yes

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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