A Phase I Study of the CXCR-4 Inhibitor AMD3100 for the Treatment of Neutropenia Due to Mutations of CXCR-4, the Myelokathexis Syndrome
This is an open label, single Center, phase I study to examine the hematological effects,
pharmacokinetics and safety of plerixafor in patients with myelokathexis attributable to
mutations of CXCR4, utilizing serial, escalating doses of plerixafor administered on days 1,
3, 5, 8, and 10. Five intrapatient escalating dose levels, 20 micrograms per kilogram
(mcg/kg), 40 micrograms/kilogram(mcg/kg), 80 micrograms/kilogram(mcg/kg), and 240
micrograms/kilogram (mcg/kg)will be examined. The subjects will be patients at the
University of Washington General Clinical Research Center for up to 10 days; the study
requires subject be available for up to 14 days. Patients will be monitored for
hematological effects of plerixafor and observed for adverse effects. If a normal blood
neutrophil count is achieved and maintained for at least 24 hours prior to the highest dose,
we will stop at that level.
Interventional
Endpoint Classification: Pharmacodynamics Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Blood Neutrophil Counts.
Effectiveness of drug based on increases of blood neutrophil counts to greater than 2.0 x 10^9 per liter
up to 14 days, depending on when subject reached peak response, i.e., the highest count after the stimulus (plerixafor)
Yes
David C Dale, MD
Principal Investigator
University of Washington
United States: Food and Drug Administration
35419-D
NCT01058993
October 2010
April 2011
Name | Location |
---|---|
University of Washington Medical Center | Seattle, Washington 98195-6043 |