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A Pilot Study To Design a Clinical Trial That Will Compare the Ability of Plerixafor Alone Versus Plerixafor Plus G-CSF To Generate a Bone Marrow Versus Blood Transplant Product In Normal Healthy Adults

Phase 2
18 Years
30 Years
Not Enrolling
Malignant Lymphoma, Stem Cell Type

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Trial Information

A Pilot Study To Design a Clinical Trial That Will Compare the Ability of Plerixafor Alone Versus Plerixafor Plus G-CSF To Generate a Bone Marrow Versus Blood Transplant Product In Normal Healthy Adults


Stem cell transplantation (SCT) involves transfusing stem cells collected from 3 potential
sources: bone marrow, peripheral blood, or umbilical cord. There are 2 major types of
transplantation. Autologous SCT involves transplanting the patients own stem cells, while
allogeneic transplantation involves transplanting stem cells from someone else. Whatever the
source, the transplanted stem cell product is called the graft. Currently, the most common
source is peripheral blood, However, there is considerably debate as to the optimal stem
cell source because different sources have been associated with different transplant
outcomes. One key outcome is graft versus host disease (GVHD). This is the most common
complication of SCT and contributes significantly to transplant morbidity and mortality, as
well as relapse.

Stem cells normally populate the bone marrow. Drugs are used to encourage stem cells to go
into the peripheral blood, a process call mobilization. In the peripheral blood stem cells
can be collected and subsequently transplanted. In Canada, the only drug used to mobilize
stem cells from donors is filgrastim, a form of granulocyte colony stimulating factor
(G-CSF). Although well tolerated, there are some shortcomings to this agent and there is
clearly room to improve in terms of the donor experience and providing the optimal stem cell

A soon to be completed study conducted by the Canadian Blood and Bone Marrow Transplant
Group (CBMTG), called CBMTG 0601, is evaluating the best stem cell source. The study is
aimed to compare the outcomes between allogeneic transplant patients who receive either
G-CSF stimulated bone marrow or peripheral blood. This will be a seminal study that should
provide valuable information as to the optimal stem cell source in allogeneic SCT. Early
results suggest that the investigators may be able to predict important SCT outcomes, such
as transplant related mortality, relapse or graft versus host disease, by looking at cell
populations in the graft.

Plerixafor is a new mobilization agent that is approved for use in combination with neupogen
in myeloma and relapsed lymphoma who are undergoing an autologous SCT. Following numerous
publications on the safety and dosing of plerixafor, there were 2 pivotal high quality
studies performed in this patient population. The results of this study lead to FDA
approval, and Health Canada approval is close. Plerixafor may be a clinically superior
mobilizing agent to Neupogen. More importantly though, it has a formidable safety profile
and arguably superior in this respect. There has also been a case report and a trial
demonstrating both the safety and efficacy of plerixafor in healthy donors as well. Finally,
preclinical work suggests that plerixafor mobilization may yield a superior graft. Taken
together, this work supports the notion that plerixafor may be a superior mobilization agent
over neupogen.


Considering the shortcomings of neupogen and the demonstrated potential of plerixafor in
trials with autologous SCT patients, it would be logical to further investigate the efficacy
of plerixafor in normal healthy donors for allogeneic SCT. The investigators can confidently
state that plerixafor is safe. Furthermore, plerixafor can mobilize myeloma and lymphoma
patients who have failed neupogen mobilization. However, although suggested in preclinical
data, there is no firm evidence to suggest that plerixafor mobilization provides a superior
stem cell graft. The investigators will administer either plerixafor or plerixafor and
neupogen then compare the cell populations in both peripheral blood and bone marrow..


The use of SCT has steadily increased in the last 20 years. This has especially increased
since using neupogen to mobilize stem cells thereby making transplants easier. Now that
there is a new agent available that is likely safer and perhaps more efficacious, its use
may improve both donor experience and patient outcomes. If plerixafor grafts consist of cell
populations that are associated with superior transplant outcomes as determined in CBMTG
0601, then it will likely be used in an upcoming larger clinical trial comparing neupogen
and plerixafor in normal healthy donors.


Our primary objective is to determine the best timing for harvesting stem cells from normal
donors given plerixafor with or without neupogen. A secondary objective is to evaluate and
compare the change over time after plerixafor with or without neupogen administration on the
population of cells in the graft predicted to give superior transplant outcomes. The other
secondary objective is to evaluate and compare the change over time after plerixafor with or
without neupogen administration on the concentration of cells in the PB and BM of normal
donors predicted to give GVHD or relapse. The investigators hypothesise that plerixafor with
neupogen will provide a graft with increased cell populations associated with superior SCT

The inclusion and exclusion criteria are designed to reflect those that are used in
practice to choose appropriate normal healthy donors for allogeneic stem cell

Inclusion Criteria:

- Male or female between the ages of 18 and 30

- Unable or unwilling to give written informed consent

- No history of cardiac, pulmonary, liver or renal disease

- Normal CBC, creatinine, liver enzymes, bilirubin, INR and PTT

Exclusion Criteria:

- Allergy to G or to E.coli-derived agents

- Allergy to "caine" type anesthetics

- Pregnancy or breast feeding

- BMI greater than 25 to avoid difficulty with the number of bone marrows performed

- Skin conditions, autoimmune disease, sickle cell disease or splenomegaly to avoid
rare side effects of G-CSF

- Any subject, who in the opinion of the investigator, should not participate in this

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

The frequency of CD34+ and CD34+CD38- cells at different time points as compared to baseline.

Outcome Description:

The frequency of CD34+ and CD34+CD38- cells in a graft has been show to be an excellent measure of hematopoietic engrafting potential.

Outcome Time Frame:

Day -1, 0, +1

Safety Issue:


Principal Investigator

Stephen Couban

Investigator Role:

Principal Investigator

Investigator Affiliation:



Canada: Health Canada

Study ID:




Start Date:

September 2011

Completion Date:

December 2011

Related Keywords:

  • Malignant Lymphoma, Stem Cell Type
  • Hematopoietic Stem Cell Transplantation
  • Plerixafor
  • Healthy Donors
  • Stem Cell Graft
  • Lymphoma