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A Phase II Trial Evaluating the Safety and Efficacy of Plerixafor and Sargramostim (GM-CSF) for the Mobilization of Peripheral Blood Stem Cells (PBSC) From Normal, HLA-Matched Allogeneic Sibling Donors


Phase 2
18 Years
65 Years
Open (Enrolling)
Both
Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Lymphoma, Non-Hodgkin, Hodgkin Disease, Leukemia, Lymphocytic, Chronic, B-Cell, Multiple Myeloma

Thank you

Trial Information

A Phase II Trial Evaluating the Safety and Efficacy of Plerixafor and Sargramostim (GM-CSF) for the Mobilization of Peripheral Blood Stem Cells (PBSC) From Normal, HLA-Matched Allogeneic Sibling Donors


The main purpose of this study is to gather information about the combination the drugs
plerixafor with sargramostim in donors of blood-forming cells (stem cells). Stem cells can
be taken from the bone marrow of the pelvic bones or from the blood following treatment with
drugs called growth factors; sargramostim is such a drug. Once stem cells leave the bone
marrow and circulate in the blood, they are called peripheral blood stem cells (PBSCs).
These cells can be collected through a routine procedure called apheresis, which involves
placing two IVs into the arm which are connected to an apheresis machine; the machine then
takes blood from the body, removes the stem cells, and returns the blood to the body.

Normally, a growth factor called filgrastim is given to donors in order to collect the stem
cells used for transplantation. However, when stem cells collected using filgrastim are
transplanted in patients, a possible unpredictable complication is graft versus host
disease. It's thought that using a different growth factor such as sargramostim might
reduce the occurrences of graft versus host disease in patients. However, sargramostim
alone does not provide as many stem cells for transplantation as other growth factors.
Plerixafor is another drug that can increase the number of PBSCs in a donor, but like with
sargramostim, plerixafor alone does not always provide enough stem cells. This is why
sargramostim and plerixafor are being combined in this study: the investigators believe that
the two drugs together will provide enough stem cells for transplantation and may also
reduce the risk of graft versus host disease.


Inclusion Criteria:



Donor Eligibility

- Donor is 18 to 65 years of age inclusive.

- If female and of child-bearing age, donor must be non-pregnant, not breastfeeding,
and agree to use adequate contraception.

- Donor is a 6/6 HLA-matched sibling willing to donate PBSC for transplant.

- Donor has adequate cardiac function with no history of congestive heart failure and
no history of atrial fibrillation or ventricular tachyarrhythmia.

- Donor has adequate renal function as defined by a calculated serum creatinine
clearance of ≥56 ml/min for females and ≥64 ml/min for males.

- Donor has adequate hepatic function as defined by a total bilirubin <2x normal or
absence of hepatic fibrosis/cirrhosis.

- Donor has adequate neurologic function as defined by NO evidence of a severe central
or peripheral neurologic abnormality. No history of cerebrovascular accident or
seizure disorder requiring anticonvulsant medication.

- Donor must be HIV-1&2 antibody and HTLV-I&II antibody sero-negative, by FDA licensed
test.

- Donor must have an ECOG performance status of 0 or 1.

- Donor must demonstrate ability to be compliant with study regimen.

- Donor must not have an active infection at the time of study entry.

- Donor does not have active alcohol or substance abuse within 6 months of study entry.

- Donor is not currently enrolled on another investigational agent study.

- Donor does not have any medical condition, which, in the opinion of the clinical
investigator, would interfere with his/her evaluation.

- Ability of the donor to understand and the willingness to sign a written informed
consent document.

Recipient Eligibility

- Recipient must have available the successful collection of a GM-CSF + plerixafor
mobilized product. When an adequate collection cannot be obtained, G-CSF will be
used and some recipients may need to receive a combined product of mobilized cells
with plerixafor + GM-CSF and G-CSF mobilized cells. Recipients who receive less than
2.0 X 106 CD34+ cells/kg/actual recipient weight after six days of GM-CSF and two
days of IV plerixafor will not be considered "eligible" but followed per protocol for
safety purposes only.

- Recipient is 18 to 65 years of age inclusive.

- Recipient is willing and has a 6/6 HLA-matched sibling willing to donate PBSC for
transplant.

- Recipient must provide signed informed consent.

- If female and of child-bearing age, recipient must be non-pregnant, not
breastfeeding, and using adequate contraception.

- Recipient must have one of the following diagnoses:

- Acute myelogenous leukemia (AML) in 1st or subsequent remission or in relapse,

- Acute lymphoblastic leukemia (ALL) in 1st or subsequent remission or in relapse,

- Myelodysplastic syndrome either intermediate 1 or 2, or high risk by the
International Prognostic Scoring System,

- Chronic myelogenous leukemia (CML) in accelerated or second chronic phase,

- Non-Hodgkin's lymphoma (NHL) or Hodgkin's disease (HD) in 2nd or greater complete
remission, partial remission, or refractory relapse,

- Chronic lymphocytic leukemia (CLL), Rai Stage 2-4, failing at least 2 prior regimens,
OR

- Multiple myeloma (MM), Stage 2-3.

- Myeloproliferative disorder or neoplasm

- Recipient must have adequate cardiac function with a left ventricular ejection
fraction ≥ 40%.

- Recipient must have adequate pulmonary function defined as NO severe or symptomatic
restrictive or obstructive lung disease, and formal pulmonary function testing
showing an FEV1 ≥50% of predicted and a DLCO ≥40% of predicted, corrected for
hemoglobin.

- Recipient must have adequate renal function as defined by a serum creatinine
clearance of ≥75% of normal (Cockcroft-Gault equation).

- Recipient must have adequate hepatic function as defined by a total bilirubin <2x
normal or absence of hepatic fibrosis/cirrhosis.

- Recipient must have adequate neurologic function as defined by NO evidence of a
severe central or peripheral neurologic abnormality. Patients with a history of
previous CNS tumor involvement are eligible provided they are without symptoms or
signs and the CNS is now free of disease on lumbar puncture and CT scan of the brain.

- Recipient must have no evidence of active infection at the time of the transplant
preparative regimen or at time of transplantation.

- Recipient must be HIV-1&2 antibody and HTLV-I & II antibody sero-negative, by FDA
licensed test.

- Recipient has an ECOG performance status of 0 or 1.

- Recipient must demonstrate ability to be compliant with medical regimen.

- Recipient must not have active alcohol or substance abuse within 6 months of study
entry.

- Recipient must not be enrolled on another investigational agent concurrently.

- Recipient must not have any medical condition, which, in the opinion of the clinical
investigator, would interfere with the evaluation of the patient.

- Recipient must have a life expectancy of greater than 4 weeks.

- Both men and women and members of all races and ethnic groups are eligible for this
trial.

Exclusion Criteria:

Donor Exclusion Criteria in addition to that stated above

- Donor may not be receiving any other investigational agents.

- Donor may not have a history of allergic reactions attributed to compounds of similar
chemical or biologic composition to plerixafor or GM-CSF, or known hypersensitivity
to yeast-derived products or any component of the product.

Type of Study:

Interventional

Study Design:

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

Outcome Measure:

Reduce the number of donors requiring a second collection

Outcome Description:

The primary endpoint is to reduce the number of donors treated with GM-CSF who require a second collection to obtain a minimum CD34/Kg (2 x 106) necessary for allogeneic stem cell transplantation when compared to historic controls mobilized with GM-CSF or plerixafor alone. A reduction in failed first leukapheresis from 40% to less than 10% as seen with G-CSF alone would be considered clinically meaningful.

Outcome Time Frame:

5 days donor and 30 days recipient

Safety Issue:

No

Principal Investigator

Mark Schroeder, M.D.

Investigator Role:

Principal Investigator

Investigator Affiliation:

Washington University School of Medicine

Authority:

United States: Institutional Review Board

Study ID:

10-1154 / 201108083

NCT ID:

NCT01158118

Start Date:

November 2010

Completion Date:

April 2013

Related Keywords:

  • Leukemia, Myeloid, Acute
  • Myelodysplastic Syndromes
  • Lymphoma, Non-Hodgkin
  • Hodgkin Disease
  • Leukemia, Lymphocytic, Chronic, B-Cell
  • Multiple Myeloma
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive
  • Hodgkin Disease
  • Leukemia
  • Leukemia, Lymphocytic, Chronic, B-Cell
  • Leukemia, Lymphoid
  • Leukemia, Myeloid, Acute
  • Leukemia, Myeloid
  • Lymphoma
  • Lymphoma, Non-Hodgkin
  • Multiple Myeloma
  • Neoplasms, Plasma Cell
  • Myelodysplastic Syndromes
  • Preleukemia

Name

Location

Washington University School of MedicineSaint Louis, Missouri  63110