This is a multi-site study with plerixafor in pediatric cancer patients. The study will be conducted in 2 stages: * Stage 1 is a dose-escalation study. * Stage 2 is an open-label, randomized, comparative study using the appropriate dosing regimen identified in the Stage 1 dose-escalation study. All participating patients will receive a standard mobilization regimen as per study site practice guidelines (either chemotherapy plus once daily granulocyte-colony stimulating factor (G-CSF) or once daily G-CSF alone). The only change to the standard mobilization regimen is the addition of plerixafor treatment prior to apheresis for all patients in Stage 1 (dose escalation), and for those patients randomized to the plerixafor plus standard mobilization treatment arm in Stage 2 (randomized, comparative). Stage 1 will enroll at least 27 patients. Stage 2 will enroll at least 40 patients.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
46
160 μg/kg subcutaneous (SC) injection
240 μg/kg subcutaneous (SC) injection
320 μg/kg subcutaneous (SC) injection
Investigational Site Number 51
Ghent, Belgium
Investigational Site Number 81
Brno, Czechia
Investigational Site Number 82
Praha 5 - Motol, Czechia
Investigational Site Number 61
København Ø, Denmark
Investigational Site Number 42
Lyon, France
Investigational Site Number 43
Paris, France
Investigational Site Number 33
Frankfurt am Main, Germany
Investigational Site Number 34
Freiburg im Breisgau, Germany
Investigational Site Number 35
Hamburg, Germany
Investigational Site Number 31
Hanover, Germany
...and 17 more locations
Proportion of patients achieving at least a doubling of peripheral blood CD34+ count during Stage 2
Time frame: Up to 5 days
Number of days of apheresis required to reach ≥2 × 10^6 CD34+ cells/kg
During Stage 1 and Stage 2
Time frame: Up to 5 days
Yield of CD34+ cells for each apheresis
During Stage 1 and Stage 2
Time frame: Up to 5 days
Total CD34+ cell yield
During Stage 1 and Stage 2
Time frame: Up to 5 days
Percentage of patients proceeding to transplant
During Stage 1 and Stage 2
Time frame: Within 6 months of last apheresis
Percentage of patients successfully engrafting
During Stage 1 and Stage 2
Time frame: 3, 6, 12 and 24 months post-transplant
Percentage of patients with durable engraftment
During Stage 1 and Stage 2
Time frame: 3, 6, 12 and 24 months post-transplant
Summary of adverse events (AEs)
During Stage 1 and Stage 2
Time frame: Up to 24 months after last transplant or 24 months after last dose (for patients that do not transplant within 6 months of last apheresis)
Duration of hospitalizations (planned or unplanned)
During Stage 1 and Stage 2
Time frame: Throughout the duration of the study
Mobilization of tumor cells into peripheral blood
During Stage 1 and Stage 2
Time frame: Up to 5 days
Relapse rates
During Stage 1 and Stage 2
Time frame: 3, 6, 12 and 24 months post-transplant
Occurrence of secondary malignancies
During Stage 1 and Stage 2
Time frame: 3, 6, 12 and 24 months post-transplant
Incidence of primary and secondary graft failure
During Stage 1 and Stage 2
Time frame: 3, 6, 12 and 24 months post-transplant
Time to secondary graft failure
During Stage 1 and Stage 2
Time frame: Up to 24 months post-transplant
Survival rates
During Stage 1 and Stage 2
Time frame: 3, 6, 12 and 24 months post-transplant
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