CompletedPhase 1

AMD 3100 for Treatment of Myelokathexis

United States6 participantsStarted 2010-10

Plain-language 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.

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Blood Neutrophil Counts.

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

Trial details

NCT IDNCT01058993

SponsorUniversity of Washington

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2011-03

Results submitted2011-08-03

View on ClinicalTrials.gov More Neutropenia trials