Continuous Infusion of the CXCR4 Antagonist Plerixafor for WHIM Syndrome.
David H McDermott, Shamik Majumdar, Daniel Velez...
https://pubmed.ncbi.nlm.nih.gov/40330596Actively Recruiting
Phase 1
Phase 2
Age: 18Years - 75Years
All Genders
ID00967785
A Phase I Study of MozobilTM in the Treatment of Patients With WHIMS
Led by National Institute of Allergy and Infectious Diseases (NIAID) · Updated on 2026-05-22
20
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying a rare genetic condition called WHIMS, which causes severe neutropenia, a low white blood cell count that makes it harder for the body to fight infections. WHIMS leads to serious infections and complications like warts and cancers related to human papillomavirus (HPV). This research aims to evaluate the safety and effectiveness of a drug called Mozobil in treating neutropenia in people with WHIMS, exploring doses within the approved range. Participants will receive Mozobil through twice daily subcutaneous injections or continuous infusion using a pump. The study includes a dose escalation phase where doses increase over five days until white blood cell counts improve or the maximum dose is reached. After this, patients may enter a long-term phase receiving Mozobil once or twice daily for up to five years. Treatment will be paused for two days before starting Mozobil if participants are on certain white blood cell stimulating medications. During the study, participants will undergo medical history reviews, physical exams, lung and heart function tests, and blood and urine sampling to monitor safety and effectiveness. Researchers will track white blood cell levels, infection rates, and HPV-related skin lesions. Participants will also provide samples for further analysis and adhere to contraception requirements if applicable. The study may last up to seven years, with ongoing safety and response monitoring throughout.
CONDITIONS
Brief Title
A Phase I Study of Mozobil in the Treatment of Patients With WHIMS
Who Can Participate
Age: 18Years - 75Years
All Genders
Eligibility Criteria
You may qualify if you...
- Clinical diagnosis of WHIMS and documented severe infection
- Age between 18 and 75 years
- Willing to stop medications like G-CSF or GM-CSF for at least 2 days before and during the study
- Not pregnant or breastfeeding
- Have a personal physician
- Willing to provide blood, plasma, serum, and DNA samples for storage
- Agree to use two forms of contraception if of childbearing potential
You will not qualify if you...
- No diagnosis of WHIMS
- Under 18 years old
- No documented history of severe infection
- Neutropenia due to other maturation defects unlikely to benefit from study drug
- Pregnant or breastfeeding
- History of serious cardiac arrhythmia or defects
- Renal failure with creatinine clearance under 15 mL/min or dialysis
- Signs or symptoms of active infection at study entry
- Any condition posing undue risk as judged by investigator
- Unwillingness to undergo study testing or procedures
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
1
2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Treatment
Duration - Up to 7 years
Participants receive Mozobil via twice daily subcutaneous injections or continuous subcutaneous infusion to treat WHIMS and monitor safety and blood cell counts.
Regular visits for monitoring during treatment
Trial Site Locations
Total: 1 location
1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
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Research Team
E
Elena J Cho
D
David H McDermott, M.D.
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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Published Research Related To This Trial
A WHIM satisfactorily addressed.
Hal E Broxmeyer
https://pubmed.ncbi.nlm.nih.gov/24723677A phase 1 clinical trial of long-term, low-dose treatment of WHIM syndrome with the CXCR4 antagonist plerixafor.
David H McDermott, Qian Liu, Daniel Velez...
https://pubmed.ncbi.nlm.nih.gov/24523241The CXCR4 antagonist plerixafor corrects panleukopenia in patients with WHIM syndrome.
David H McDermott, Qian Liu, Jean Ulrick...
https://pubmed.ncbi.nlm.nih.gov/21890643