A Phase I Study of Mozobil in the Treatment of Patients With WHIMS

Launched by NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES (NIAID) · Aug 27, 2009

Keywords

ClinConnect Summary

This clinical trial is studying a medication called Mozobil to see if it can help patients with WHIMS, a rare genetic condition that leads to very low white blood cell counts (neutropenia) and makes it hard for the body to fight infections. Currently, patients typically receive injections of a different medication to boost their white blood cells, but this can be costly and has side effects. Mozobil may provide a more targeted and better-tolerated treatment option.

To participate in this trial, individuals must be between 18 and 75 years old, have a confirmed diagnosis of WHIMS, and a history of severe infections. Participants will undergo various health screenings and may receive increasing doses of Mozobil over five days. They will then continue with the treatment for up to five years. Throughout the study, patients will have regular check-ups to monitor their health and response to the medication. This trial is currently recruiting participants and aims to find a safe and effective dose for those with WHIMS.

Gender

ALL

Eligibility 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