Phase 3
N=19
Plerixafor Versus G-CSF in the Treatment of People With WHIM Syndrome
Myelokathexis · Infections · Neutropenia · Warts · Hypogammaglobulinemia
Bottom Line
View on ClinicalTrials.gov: NCT02231879 ↗Enrolled (actual)
19
Serious AEs
21.1%
Results posted
Apr 2023
Primary outcome: Primary: Severity of Infection — 12.5; 9 score on a scale (with no maximum)
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Plerixafor (Drug); G-CSF (Drug)
- Age
- Pediatric, Adult, Older Adult · 10+ yrs
- Sex
- All
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- Primary completion
- Oct 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Severity of Infection |
12.5; 9 | — |
| PRIMARY Difference in Total Infection Severity Score |
10.6; 9.33 | 0.65 |
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.
Eligibility Criteria
- INCLUSION CRITERIA:
Subjects are eligible to enter the study if they meet all of the following 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.
- 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:
- Male or female condoms with a spermicide,
- Diaphragm or cervical cap with spermicide,
- Intrauterine device,
- Contraceptive pills or patch, Norplant, Depo-Provera or other FDA-approved contraceptive,
- Male partner with vasectomy and documented aspermatogenic sterility.
- Willingness to comply with the study medications, visits, and procedures, as deemed necessary by the principal investigator (PI).
EXCLUSION CRITERIA
If any of the following exclusion criteria are met, a subject will not be enrolled in this study:
- 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.
Data sourced from ClinicalTrials.gov (NCT02231879). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.