Using Mozobil to treat WHIMS syndrome
A Phase I Study of MozobilTM in the Treatment of Patients With WHIMS
This study is testing if the drug Mozobil can help adults with WHIMS syndrome by improving their white blood cell counts and reducing their risk of severe infections.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 20 (estimated) |
| Ages | 18 Years to 75 Years |
| Sex | All |
| Sponsor | National Institutes of Health Clinical Center (CC) NIH |
| Locations | 1 site (Bethesda, Maryland) |
| Trial ID | NCT00967785 on ClinicalTrials.gov |
What this trial studies
This clinical trial investigates the use of Mozobil (plerixafor) to treat patients diagnosed with WHIMS syndrome, a rare genetic disorder characterized by severe neutropenia and increased susceptibility to infections. The study aims to assess the safety and efficacy of Mozobil in partially blocking the CXCR4 receptor, which is responsible for the abnormal retention of neutrophils in the bone marrow. Participants will be monitored for changes in white blood cell counts and overall health outcomes. The trial includes adults aged 18 to 75 who have documented severe infections related to WHIMS.
Who should consider this trial
Good fit: Ideal candidates for this study are adults aged 18 to 75 with a clinical diagnosis of WHIMS and a history of severe infections.
Not a fit: Patients who are not diagnosed with WHIMS or those who are pregnant or breastfeeding will not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve immune function and reduce the frequency of infections in patients with WHIMS syndrome.
How similar studies have performed: While the use of Mozobil has been established in other contexts, this specific application for WHIMS syndrome is novel and has not been extensively tested in prior studies.
Eligibility criteria
Show full inclusion / exclusion criteria
* 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
Where this trial is running
Bethesda, Maryland
- National Institutes of Health Clinical Center — Bethesda, Maryland, United States (Recruiting)
Study contacts
- Principal investigator: David H McDermott, M.D. — National Institute of Allergy and Infectious Diseases (NIAID)
- Study coordinator: Elena J Cho
- Email: elena.cho@nih.gov
- Phone: (301) 761-7280
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.