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CD34+ Enriched Transplants to Treat Myelodysplastic Syndrome

ConditionsMyelodysplastic SyndromesGraft Vs Host DiseaseGraft-versus-host-disease
Trial statusNot yet recruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age18-72
SponsorGuenther Koehne

About this trial

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 criteria

This trial does not accept healthy volunteers

Qualifiers

MDS patients who qualify for transplant, for which CD34+ selected, T-cell-depleted allogeneic hematopoietic stem cell transplantation is indicated

Patients with high-risk features with a score equal to or higher than 4.5 using the IPSS-R.

Patients with therapy-related MDS and patients with transfusion-dependent MDS are high-risk patient cohorts that are eligible independent of Revised International Prognostic Scoring System (IPSS-R) scoring.

Patient's age is from ≥18 to ≤72 years old.

Disqualifiers

Individuals who are pregnant or breastfeeding

Uncontrolled bacterial, viral, or fungal infections; or known human immunodeficiency virus (HIV), Hepatitis B, or Hepatitis C infection.

Patients cured of Hepatitis C for ≥3 years prior to enrollment are permitted

Patients with detectable hepatitis B surface antigen (HBsAg) will be excluded. Patients with detectable hepatitis B surface antigen (HBsAg) are excluded. Patients with viral hepatitis B core antibody (HBcAb) positivity, but absence of HBsAg, are eligible if hepatitis B virus (HBV) DNA is undetectable and if they are willing to be on hepatitis B antiviral prophylaxis and undergo monitoring for HBV reactivation (every 12 weeks while on immune suppression) per institutional protocols. Patients will be referred promptly to hepatology specialists with the first detectable viral load.

Trial design

Design model

Single group

Treatments tested in this trial

  • Busulfan

    Drug

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

  • Melphalan

    Drug

    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

  • Fludarabine

    Drug

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

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

    Device

    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.

Treatment groups

62 Participants
are divided into 1 treatment group
Group A: CD34+ Peripheral Blood Progenitor Cell (PBSC) Transplant with Busulfan/Melphalan/Fludarabine RegimenExperimental treatment 4 interventions

Trial outcomes

Primary outcomes

1

Overall survival (OS)

OS is defined as time from transplant to death due to any cause

Time frame
2 years

Secondary outcomes

1

Disease-free survival (DFS)

DFS is defined as the time from transplant to relapse, progression, death due to any cause, whichever occurs first.

Time frame
2 years
2

Transplant-related mortality (TRM)

Incidence of TRM defined as mortality related to the transplant rather than the disease

Time frame
2 years
3

Acute graft vs. host disease (GVHD)

Incidence 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
2 years
4

Chronic graft vs. host disease (GVHD)

Incidence 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
2 years

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Guenther Koehne

Lead sponsor

Baptist Health South Florida

Sponsor institution

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: