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Granulocyte Colony-stimulating Factor (G-CSF) Plus or Minus AMD3100 for Engraftment Post Allogeneic Transplant

Phase 1
Completed
Conditions
Stem Cell Transplant Patients
Interventions
Procedure: Stem Cell Infusion
Procedure: Leukapheresis
Registration Number
NCT01026987
Lead Sponsor
Washington University School of Medicine
Brief Summary

Patients who have not had adequate blood count recovery post related or unrelated stem cell transplant will be given a "boost" of T-cell depleted, enriched stem cells to hopefully improve their blood counts.

Detailed Description

Patients who have not had adequate blood count recovery post related or unrelated stem cell transplant will be given a "boost" of T-cell depleted, enriched stem cells to hopefully improve their blood counts.

The unrelated donors will receive G-CSF prior to pheresis (collection of the stem cells) to boost the number of CD34+ cells. The related donors will receive G-CSF and AMD3100 prior to pheresis to boost the number of CD34+ cells. Once the CD34+ cells are collected they will be T-cell depleted using a cell separation device called the CliniMACS systems. The CliniMACS system will select the CD34+ cell and remove the T-cells. By removing the T-cells we can minimize the risk of Graft Versus Host Disease (GVHD). The enriched CD34+ cells will be given to them to hopefully give them a "boost" of cells that can permanently produce new blood cells to improve their risk of infection and bleeding.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
29
Inclusion Criteria

Recipient

  • Must be age ≥ 18
  • Must have ≥90 % donor cells in the unfractionated peripheral blood based on either XY FISH or standard STR.
  • More than 60 days post allogeneic stem cell transplantation.
  • Must meet one of the following criteria:
  • platelets < 20,000 or
  • ANC<500 or
  • transfusion dependent for at least one cell line and /or
  • on growth factor support (G-CSF) without adequate response for 30 days and
  • no reversible etiology found after an allogeneic stem cell transplantation
  • Patient has an ECOG performance status of 0-2.
  • The original stem cell donor must be available, willing, and medically able to undergo Mobilization and a maximum of 2 apheresis procedures
  • Each patient (recipient) or legal guardian and donor must be willing to participate as a research subject and must sign an informed consent form.

Unrelated Donors

  • NMDP guidelines for eligibility will be followed using G-CSF alone mobilization.

Related donors

  • Must be ≥18 yrs old and ≤ 75 years old.
  • Donor must be sero-negative for HIV-1&2 antibody and HTLV-I&II antibody, by FDA licensed test.
  • Donor must have adequate renal function as defined by serum creatinine ≤ 1.5X institution ULN and AST and ALT ≤ 3X ULN and total bilirubin less than 2 mg/dl.
  • Donor must be agreeable to mobilization and the second donation of PBMC.
  • Women of child bearing potential should be willing to avoid becoming pregnant while receiving treatment with plerixafor.
  • Donor must have adequate peripheral venous catheter access for leukapheresis or must agree to placement of a central catheter.
Exclusion Criteria

Recipient

  • Patients with confirmed relapse of their original disease
  • Participation in other clinical trials that involve investigational drugs or devices except with permission from the Principal Investigator and Sponsor.
  • Patients with documented active viral, bacterial or fungal infections.
  • Documented allergy to murine proteins or iron dextran.
  • Pregnancy
  • Patients with immune mediated graft dysfunction.

Donor

  • Evidence of active infection at the time of study entry.
  • Medical or physical reason which makes the donor unlikely to tolerate or cooperate with growth factor therapy and leukapheresis
  • Factors which place the donor at increased risk for complications from leukapheresis or G-CSF therapy(e.g., autoimmune disease, multiple sclerosis, sickle cell trait, coronary artery disease).
  • Pregnancy (positive serum or urine beta-HCG) or breastfeeding. Women of childbearing age must avoid becoming pregnant while on the study.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Related Donors: G-CSF & AMD3100AMD3100G-CSF 10 ug/kg SC daily for 5 days. AMD3100 320 mcg/kg IV over 30 min on Day 5. Leukapheresis on Day 5.
Related Donors: G-CSF & AMD3100LeukapheresisG-CSF 10 ug/kg SC daily for 5 days. AMD3100 320 mcg/kg IV over 30 min on Day 5. Leukapheresis on Day 5.
RecipientStem Cell InfusionStem Cell Infusion on Day 0
Related Donors: G-CSF & AMD3100G-CSFG-CSF 10 ug/kg SC daily for 5 days. AMD3100 320 mcg/kg IV over 30 min on Day 5. Leukapheresis on Day 5.
Primary Outcome Measures
NameTimeMethod
Time to neutrophil engraftment100 days post CD34+ selected, T-Cell depleted transplant

For recipients with ANC \< 500 or growth factor support dependent at study entry, Time to neutrophil improvement is measured from the date of CD34+ selected, T-Cell depleted infusion to the first of 3 consecutive measurements of neutrophil count \> 500/μl without growth factor support for \>7 days prior.

RBC transfusion engraftment - independence from RBCs without growth factors.

Time to platelet engraftment100 days post CD34+ selected, T-Cell depleted transplant

For recipients with platelets \< 20,000 or platelet transfusion dependent at study entry, Time to platelet improvement is measured from the date of CD34+ selected, T-Cell depleted infusion to the of 3 consecutive measurements of platelet count ≥ 20,000/ul without platelet transfusion support for 7 days.

Time to red blood cell (RBC) improvement100 days post CD34+ selected, T-Cell depleted transplant

For recipients who are RBC transfusion dependent at study entry, Time to RBC improvement is measured from the date of CD34+ selected, T-Cell depleted infusion to the first date of hemoglobin \>9.0g/dL without \> 1 RBC transfusion during the previous 56 days.

Secondary Outcome Measures
NameTimeMethod
Overall survival (recipients)1 year from date of transplant

Overall survival is the time from the date of CD34+ selected, T-Cell depleted infusion to death.

Incidence and severity of acute Graft vs Host Disease (GVHD)2 years post-transplant

Incidence and severity of chronic GVHD will be assessed based on the Seattle criteria

Toxicities associated with CD34+ cell infusion (recipients)30 days post-transplant

NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 will be utilized for all toxicity reporting.

To assess the feasibility of collecting adequate donor CD34+ enriched T-cell depleted peripheral blood stem cells using G-CSF+ plerixafor from related donors and G-CSF alone from unrelated donors.Day 0 (transplant day)
Disease-free survival1 year from date of transplant

Disease-Free survival is the time from the date of CD34+ selected, T-Cell depleted infusion to disease relapse or death.

Rate of transplant-related mortality (TRM)100 days post-transplant
Toxicities associated with the CD34+ collection (donors)30 days post mobilization

NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 will be utilized for all toxicity reporting.

Phenotypically and functionally characterize donor CD34+ and donor T-cells mobilized by G-CSF from unrelated donors and mobilized with G-CSF + plerixafor from related donors.Day of mobilization (Day 0)

Trial Locations

Locations (1)

Washington University School of Medicine

🇺🇸

St. Louis, Missouri, United States

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