Plerixafor Versus G-CSF in the Treatment of People With WHIM Syndrome (NCT02231879) | Clinical Trial Compass
CompletedPhase 2/3
Plerixafor Versus G-CSF in the Treatment of People With WHIM Syndrome
United States20 participantsStarted 2014-10-14
Plain-language summary
Background:
\- WHIMS (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis Syndrome) is a rare disease. It can cause cancers, infections, and warts. Researchers want to see if a drug called plerixafor can treat WHIMS.
Objective:
\- To compare plerixafor versus granulocyte colony stimulating factor (G-CSF) for preventing infections in people with WHIMS.
Eligibility:
\- People ages 10-75 with WHIMS who have a CXCR4 gene mutation.
Design:
* Participants will be screened with a medical history, physical exam, and blood and urine tests. They may have heart and spleen tests and body scans. They may have samples of skin or warts taken. Researchers may take photographs of warts.
* Participants will start twice daily self-injections of G-CSF. Their doctors will decide the dosage.
* Initial Period (4-12 weeks)
* Participants will:
* continue the injections and their usual antibiotics and/or immunoglobulin
* have blood drawn
* keep a daily health diary
* Participants will visit the clinic for 2 days without injections.
* Adjustment Period 1 (8 weeks):
* Participants will:
* continue twice daily injections from home
* continue the daily health diary
* have blood tests every 2 weeks.
* Treatment Year 1:
* Participants will
* receive either plerixafor or G-CSF injections twice daily
* continue the health diary
* have blood tests every 2 months
* visit the clinic about every 4 months
* At the end of year 1, participants will visit the clinic for an evaluation. They will switch to the other study drug. They will have an 8-week adjustment and 1-year treatment period.
* At the end of year 2, participants will visit the clinic to complete their injections and go back to their previous G-CSF regimen. Participants will continue their daily health diary and have blood tests for 5-6 months.
Who can participate
Age range
10 Years – 75 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 greater than or equal to 10 and less than or equal to 75 years.
. Heterozygous mutation in the C-tail of CXCR4 in addition to a clinical diagnosis of WHIMS.
. Documented neutropenia with a baseline ANC below 1500 cells/microL of blood.
. History of severe and/or recurrent infections.
. Willingness to interrupt G-CSF medication, 2 days prior to study drug injection.
. Must have a local medical provider for medical management.
. Must be willing to provide blood, plasma, serum, and DNA samples for storage.
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
1
Severity of Infection
Timeframe: Scores from 12 months treatment on each study drug
2
Difference in Total Infection Severity Score
Timeframe: Scores from 12 months treatment on each study drug
Trial details
NCT IDNCT02231879
SponsorNational Institute of Allergy and Infectious Diseases (NIAID)
. All study subjects must agree not to become pregnant or impregnate a female. Women of childbearing potential must agree to take appropriate steps to avoid becoming pregnant for the duration of the study. Participants in whom pregnancy is biologically possible must use at least 2 study approved methods of contraception, one of which must be a barrier method, and must continue contraception until 5 months after stopping the study drug:
Exclusion criteria
. Neutropenia due to maturation defects in the myeloid lineage or a type of neutropenia, which in the investigator s opinion, is unlikely to improve from the medication administered in this study.
. Pregnant or breast-feeding women.
. Known hypersensitivity to plerixafor, G-CSF, or any components of the products.
. Predisposition to or history of life-threatening cardiac arrhythmia.
. Requiring dialysis or having markedly impaired renal function with a Creatinine Clearance (CrCl) \<15 mL/min.
. Condition that in the investigator s opinion places a subject at undue risk by participating in the study.