A Pilot Study of AMD3100 as a Sensitizing Agent in Myeloablative Allogeneic Blood and Marrow Transplantation for Chemotherapy Resistant Pediatric Acute Leukemia
The first six patients will receive three daily doses of AMD3100 (240 mcg/kg via IV). If it
appears that three doses do not significantly increase the side effects of transplant
conditioning, the investigators will give a second group of six patients five daily doses.
AMD3100 is given in combination with a standard pre-transplant conditioning regimen (total
body irradiation, etoposide and cyclophosphamide.) AMD3100 causes healthy bone marrow cells
to be released from the bone marrow into the blood so that they can be collected in patients
who will have peripheral (blood stream) blood stem cell transplants. AMD3100 also pushes out
leukemia cells from the bone marrow. Research in animals and in test tubes shows that the
bone marrow partially protects leukemia cells from chemotherapy and radiation. AMD3100 could
make leukemia treatments better by pushing out the leukemia cells from the bone marrow and
making them more sensitive to treatment. Clinical trials combining AMD3100 with normal doses
of chemotherapy are being done for relapsed acute leukemia. Researchers hope AMD3100 can be
given with conditioning regimen safely without causing more side effects. Up to 12
participants will be enrolled and estimated accrual duration is 2 years.
Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Incidence of grade 3 and 4 regimen-related toxicity assessed per Bearman scale at day 42. Patients in dose level 1 get 3 days of AMD3100. If 2 cases of grade 4 toxicity or 3 cases of grade 3-4 toxicity occur, dose level 1 and study will be closed. Otherwise, after 6 patients have been assessed at day 42, the dose will be escalated to 5 days of AMD3100 (dose level 2). If 2 cases of grade 4 toxicity or 3 cases of grade 3-4 toxicity occur, the study will be closed. Otherwise, after 6 patients have been enrolled at this level the study will be closed to enrollment.
day 42 after bone marrow transplant
Kuang-Yueh Chiang, MD, PhD
United States: Food and Drug Administration
CHOA AMD3100 Pilot
|Children's Healthcare of Atlanta||Atlanta, Georgia 30342|