Could a stem cell drug tame rare immune disease?
NCT ID NCT00967785
First seen Jun 27, 2026 · Last updated Sep 10, 2026 · Updated 15 times
Summary
This early-phase study tests whether Mozobil, a drug already used to boost stem cells for transplants, can safely raise white blood cell counts in people with WHIMS. WHIMS is a rare genetic condition that traps infection-fighting cells in the bone marrow, leading to frequent infections. Up to 20 adults aged 18-75 will receive Mozobil injections, with some continuing treatment for up to 5 years, to see if it reduces infections and warts.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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14 people
The number who actually took part.
- Started
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Jan 2010
- Finished
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Aug 2026
- Lead sponsor
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A government research agency
The lead sponsor is the US National Institutes of Health.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 to 75 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
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Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
* INCLUSION CRITERIA: All of the following inclusion criteria must be met for a subject to be enrolled in this study: * Clinical diagnosis of WHIMS and documented severe infection * Must be greater than or equal to 18 and less than or equal to 75 years of age * Willingness to interrupt medications to raise the white count (WBC) such as G-CSF or GM-CSF for at least 2 days before and while on the study drug * Must not be pregnant or breastfeeding * Must have a personal physician * Must be willing to provide blood, plasma, serum, and DNA samples for storage * Subjects must agree not to become pregnant or to impregnate a female. If of childbearing potential, must agree to consistently use two types of contraception throughout study participation. Acceptable forms of contraception include the following: 1. Condoms, male or female, with or without a spermicide 2. Diaphragm or cervical cap with spermicide 3. Intrauterine device 4. Contraceptive pills or patch, Norplant, Depo-Provera or other FDA-approved contraceptive method 5. Male partner has previously undergone a vasectomy for which there is documentation of aspermatogenic sterility EXCLUSION CRITERIA: If any of the following exclusion criteria are met, a subject will not be enrolled in this study: * Absence of a diagnosis of WHIMS * Patient is less than 18 years old * Absence of a documented history of severe infection * Neutropenia due to maturation defects in the myeloid lineage or that the PI feels is unlikely to benefit from this medication * Pregnant women or breastfeeding * History of serious cardiac arrhythmia or cardiac defects that make such more likely * Renal failure (calculated creatinine clearance \[CrCl\] \<15 mL/min or requiring dialysis) * Signs or symptoms of active microbial infection at the time of study entry. * Any condition that, in the investigator s opinion, places the patient at undue risk by participating in the study * Unwillingness to undergo testing or procedures associated with this protocol
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
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Other studies related to the condition(s) this trial covers.
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