CD34+ Enriched Transplants for Myelodysplastic Syndrome

Not yet recruiting at 1 trial location
GK
GK
Overseen ByGuenther Koehne, M.D., Ph.D.
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new method for preparing blood stem cell transplants for individuals with myelodysplastic syndrome (MDS), a condition where the bone marrow fails to produce enough healthy blood cells. The study employs a device called CliniMACS to remove specific immune cells (T-cells) from the donor's blood, aiming to reduce the risk of graft versus host disease (GVHD), a complication where the donor's cells attack the patient's body. The trial seeks participants with MDS who qualify for a blood stem cell transplant, particularly those requiring frequent blood transfusions or with therapy-related MDS.

As an unphased trial, this study provides a unique opportunity to contribute to innovative research that could enhance transplant outcomes for future patients.

What prior data suggests that the CliniMACS system is safe for processing blood stem cell transplants?

Research has shown that the CliniMACS system for stem cell transplants is generally well-tolerated. In studies, 89% of patients who received transplants processed with CliniMACS reached or exceeded the target dose of stem cells without major problems. This system reduces the risk of graft versus host disease (GVHD), a serious issue where the body attacks donor cells. By removing certain immune cells (T-cells) before the transplant, the risk of chronic GVHD is lowered while important immune functions remain intact. Overall, the CliniMACS system appears promising in making transplants safer for patients.12345

Why are researchers excited about this trial?

Unlike the standard treatments for myelodysplastic syndrome (MDS), which often involve medications like azacitidine or decitabine, the CD34+ enriched transplant approach uses a unique method of selecting and transplanting stem cells. This treatment is different because it employs the CliniMACS system to selectively enrich CD34+ cells and deplete T-cells, aiming to reduce the risk of graft-versus-host disease. Researchers are excited because this method could offer a more targeted and potentially safer transplant option, enhancing the effectiveness of stem cell transplants while minimizing complications.

What evidence suggests that the CliniMACS system is effective for reducing complications in myelodysplastic syndrome?

Research has shown that the CliniMACS system, used in this trial, can reduce the risk of graft versus host disease (GVHD) in patients receiving a blood stem cell transplant. This system removes certain cells, called T-cells, from the donor's blood, which usually cause GVHD. Studies have found that the CliniMACS system significantly lowers T-cell numbers, leading to fewer GVHD complications. In earlier research, almost 90% of patients received the right amount of important cells, known as CD34+ cells, essential for successful transplants, without increasing GVHD risk. These findings suggest that the CliniMACS system could make transplants safer for people with myelodysplastic syndrome (MDS).23678

Who Is on the Research Team?

GK

Guenther Koehne, M.D., Ph.D.

Principal Investigator

Baptist Health Herbert Wertheim Cancer Insitute

Are You a Good Fit for This Trial?

This trial is for people with certain types of myelodysplastic syndrome (MDS) who are healthy enough to undergo a stem cell transplant, have good heart, lung, liver, and kidney function, and do not have active infections or HIV. Pregnant or breastfeeding individuals cannot join.

Inclusion Criteria

Karnofsky (adult) Performance Status of at least 70%
Adequate organ function measured by:
Pulmonary: asymptomatic or if symptomatic, diffusion capacity of lung for carbon monoxide (DLCO) 50% of predicted (corrected for hemoglobin).
See 5 more

Exclusion Criteria

Pregnant or breast-feeding
Patient seropositive for human immunodeficiency virus (HIV)-I /II; human T-lymphotropic virus (HTLV)-I /II
Active viral, bacterial or fungal infection
See 1 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Cytoreduction Therapy

Participants receive cytoreduction therapy with busulfan, melphalan, and fludarabine to prepare for transplant

2 weeks
Daily visits for IV administration

Transplant

CD34+ selected, T-cell depleted, allogeneic PBSC transplant using CliniMACS system

1 day
1 visit (in-patient)

Follow-up

Participants are monitored for overall survival, disease-free survival, and incidence of GVHD

2 years

What Are the Treatments Tested in This Trial?

Interventions

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

Trial Overview

The study tests if using the CliniMACS device to remove immune T-cells from donor blood stem cells can reduce complications like graft-versus-host disease after transplants in MDS patients. Patients also receive chemotherapy drugs before transplant.

How Is the Trial Designed?

1

Treatment groups

Experimental Treatment

Group I: CD34+ Peripheral Blood Progenitor Cell (PBSC) Transplant with Busulfan/Melphalan/Fludarabine RegimenExperimental Treatment4 Interventions

Find a Clinic Near You

Who Is Running the Clinical Trial?

Guenther Koehne

Lead Sponsor

Trials
4
Recruited
160+

Baptist Health South Florida

Lead Sponsor

Trials
54
Recruited
8,100+

Citations

HLA-Compatible Related or Unrelated Donors With CD34+ ...

The purpose of this study is to find out the effects of using a system called CliniMACS to remove Tcells from blood stem cells. Removing T-cells may help ...

Characteristics of CliniMACS® System CD34-Enriched T Cell ...

The CliniMACS CD34 Reagent System for CD34-enrichment has been shown to be highly effective in reducing T cell content such that GVHD incidence is ...

NCT01119066

The purpose of this study is to find out the effects of using a system called CliniMACS to remove Tcells from blood stem cells. Removing T-cells may help…

Clinical data | Miltenyi Biotec | Lëtzebuerg

The CliniMACS® CD34 Reagent System was tested in a phase II single-arm multicenter study with a total of 37 patients in first morphologic complete remission ...

5.

pubmed.ncbi.nlm.nih.gov

pubmed.ncbi.nlm.nih.gov/21875505/

Characteristics of CliniMACS® System CD34-enriched T cell ...

All patients received the minimum CD34(+) cell dose, and 39 patients (89%) came within 10% or exceeded the target CD34(+) cell dose without exceeding the ...

Page 1 of 56 A Phase II Trial of Transplants from HLA- ...

regimen consisting of Busulfan, 0.8mg/kg/dose, every 6 hours x 10 doses, melphalan 70 mg/m2/day. T cell depleted CD34+ (CliniMACS) PBSC transplants to treat 10 ...

CLINIMACS® for Alpha/Beta T-Cell Depletion in Stem ...

The CD34 antibody is conjugated to an iron-containing particle that is only nanometers in size and safe for infusion. The enrichment of CD34+ ...

CliniMACS CD34 Reference List

Allogeneic CD34-selected stem cell boost as salvage treatment of life-threatening infection and severe cytopenias after CAR-T cell therapy.