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A Phase I Study of the CXCR-4 Inhibitor AMD3100 for the Treatment of Neutropenia Due to Mutations of CXCR-4, the Myelokathexis Syndrome

Phase 1
18 Years
Not Enrolling

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Trial Information

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.

Inclusion Criteria:

- age over 18 years, WBC (white blood count) less than 3.0 x 10^9 per Liter,

- Absolute neutrophil count less than 2.0 x 10^9 per Liter,

- platelets greater than 100 x 10^6 per Liter, creatinine less than 2.0/milligrams

- Creatinine clearance > 60 ml/min calculated,

- Aspartate Aminotransferase-GOT (SGOT), Alanin Aminotransferase-GPT (SGPT), bilirubin
< 2.5 upper limit of normal,

- Eastern Cooperative Oncology Group (ECOG) status 0 or 1,

- mutation identified and confirmed in CXCR4,

- on no granulocyte-colony stimulating factor (G-CSF), granulocyte-macrophage-colony
stimulating factor (GM-CSF) within 3 weeks of the study drug

- patient signs consent, accepts contraception

Exclusion Criteria:

- greater than 18 years of age,

- sensitivity to plerixafor,

- pregnant,

- prisoner,

- decisionally impaired,

- judged unlikely to comply,

- illness that may interfere with interpretation of results,

- leukemia,

- malignancy,

- active infection requiring antibiotics within one week of study drug administration,

- history of cardiac conduction or electrocardiogram (EKG) abnormality,

- previous experimental therapy within one week.

Type of Study:


Study Design:

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

Outcome Measure:

Blood Neutrophil Counts.

Outcome Description:

Effectiveness of drug based on increases of blood neutrophil counts to greater than 2.0 x 10^9 per liter

Outcome Time Frame:

up to 14 days, depending on when subject reached peak response, i.e., the highest count after the stimulus (plerixafor)

Safety Issue:


Principal Investigator

David C Dale, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

University of Washington


United States: Food and Drug Administration

Study ID:




Start Date:

October 2010

Completion Date:

April 2011

Related Keywords:

  • Neutropenia
  • neutropenia
  • myelokathexis
  • WHIM syndrome
  • AMD 3100
  • plerixafor
  • Myelokathexis syndrome
  • Neutropenia due to mutations of CXCR-4
  • Neutropenia



University of Washington Medical Center Seattle, Washington  98195-6043