AMD 3100 for Treatment of Myelokathexis

• Status: Completed
• Trial end date: 2011-04-01
• Target enrollment: 0
• Participant gender: All

Summary

This is an initial study to determine if CXCR4 inhibitor AMD 3100 or plerixafor may be a potential treatment for neutropenia due to CXCR4 mutations, the myelokathexis or WHIM (warts, hypogammaglobulinemia, immunodeficiency and myelokathexis) syndrome. This is the initial study of this concept and will involve up to 6 patients to receive increasing doses of plerixafor administered subcutaneously or on an alternate day basis. It is unknown if these patients will be highly sensitive to a blockade of CXCR4 activity and release more white blood cells than normal volunteers or cancer patients given the same dose of this drug. Therefore doses will begin at a level 12 fold less than currently used to mobilize stem cells and will be increased stepwise to achieve an acceptable circulating level of neutrophils.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University of Washington
• Treatments: JM 3100; Plerixafor

Criteria

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 per/deciliter,

• 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.