What is Mozobil studying for WHIMS?

Mozobil is being studied to see if it can safely increase white blood cell counts in people with WHIMS, a rare condition causing low white blood cells and frequent infections. This research also looks at whether Mozobil can reduce related problems like warts and HPV lesions. The study is sponsored by the National Institute of Allergy and Infectious Diseases (NIAID).

Who may be eligible for this WHIMS clinical trial?

Adults aged 18 to 75 years diagnosed with WHIMS and a history of severe infections may be eligible. Participants must not be pregnant or breastfeeding, must agree to pause certain white blood cell medications during the study, and have a personal physician. Please review eligibility criteria for more details.

Is there any cost to participate in this WHIMS clinical trial?

Study-related care, including the study drug and all trial visits, is provided at no cost. Personal travel and other expenses may vary and are not guaranteed to be covered.

Actively 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

Participants Needed

Research Sites

N/A

Total Duration

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

Research Team

Elena J Cho

David H McDermott, M.D.

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