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Intrabone Infusion of Cord Blood Hemopoietic Stem Cells in Adult Patients With High Risk Haematological Malignancies.

Phase 2
18 Years
65 Years
Open (Enrolling)
Hematological Malignancies

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Trial Information

Intrabone Infusion of Cord Blood Hemopoietic Stem Cells in Adult Patients With High Risk Haematological Malignancies.

For many hematological malignancies, hemopoietic stem cell (HSC) transplant is the only
possible treatment. The source of HSC is often bone marrow (BM) or, in the past 10 years,
peripheral blood cell (PBSC) mobilized by granulocyte growth factor. Transplant needs a HLA
compatible (related or unrelated) donor. Around 10-30% of patients with indication for
allogeneic HSC transplant are not able to undergo the procedure because of the lack of a HLA
compatible donor. Cord blood (CB) cells represent another possible source, which needs a
lower degree of HLA compatibility, this type of transplant, however, offers a lower number
of HSC. For this reason, adult patients, until now, could not use this source, because of
the not suitable number of cell per kg, of recipient body weight. Recently, in experimental
animal models it was observed that intrabone HSC transplant allows, in the recipient,
engraftment of donor hemopoiesis by using a 1Log (10-1) lower number of cells compared to
the intravenous way (Yahata 2003, Castello 2004). Safety and feasibility of intrabone
infusion was verified by two clinical studies on humans: the first was conducted by Ringden
O. et al. in 18 patients without any evidence of collateral effects and with complete
engraftment of donor hemopoiesis with BM as a source of HSC (Hagglund 1998); the second one
was conducted by Frassoni et al. (Frassoni 2008) with CB as the source of HSC.

The aim of this study is to evaluate the intrabone infusion instead of the intravenous one,
for the HSC transplant from CB in patients with haematological malignancies when it is not
possible to find a HLA matched donor.

We will perform:

- evaluation of the engraftment kinetics;

- evaluation of the chimerism degree at 30, 60, 100 days, 6 months and 1 year after

- studies on immunological reconstitution and the role of the NK compartment.

Inclusion Criteria:

- Age between 18 and 65 years.

- Patients affected by hematological malignancies without a HLA identical sibling
donor or unrelated donor.

- Informed consent.

Exclusion Criteria:

- Patients with ECOG < 2.

- Patients with blood creatine > 2 mg/dl or with transaminase or cholestase index > 5
times compared to normality upper limits.

- Patients with Cardiac Fraction Ejection < 40%.

- Patients with DLCO < 60% or Diffusing Lung Capacity of carbon monoxide attesting a
severe pulmonary insufficiency.

- Patients with peripheral blast cell count over 10%.

- Second neoplasia diagnosed no more than 2 years before.

- Patients with active or suspected infection by fungi for which a therapeutic
treatment is ongoing.

- HIV positive patients.

- HCV-RNA and HBV-RNA positive patients (it is possible to enrol them after discussion
with the Principal Investigator).

- Pregnant or lactating women.

- Severe mental diseases.

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Proportion of transplanted patients with successful engraftment at day +42

Outcome Time Frame:

Within the first 42 days

Safety Issue:


Principal Investigator

Francesca Bonifazi, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

S. Orsola-Malpighi University Hospital


Italy: The Italian Medicines Agency

Study ID:




Start Date:

April 2009

Completion Date:

June 2013

Related Keywords:

  • Hematological Malignancies
  • Anti-thymocyte globulin Fresenius
  • Busulfan
  • Cord blood
  • Cyclophosphamide
  • Fludarabine
  • Human Leucocyte Antigen
  • Hematopoietic Stem Cell
  • Hematopoietic Stem Cell Transplant
  • Graft-versus-Host-Disease
  • Graft-versus-Host-Disease prophylaxis
  • Intrabone infusion
  • Thiotepa
  • Neoplasms
  • Hematologic Neoplasms