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New treatment option for Leukopenia
Official title A Phase I Study of Mozobil in the Treatment of Patients With WHIMS
ClinicalTrials.gov ID: NCT00967785
What this study is testing
What is Mozobil (TM)?
Mozobil (TM) is an investigational medicine, given as an once-daily injection under the skin, being studied as a potential treatment for leukopenia.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- Background: WHIMS (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis Syndrome) is caused by various genetic changes that increase the activity of the chemokine receptor, CXCR4. Excessive function of this receptor causes mature neutrophils (part of the white blood cells) to be retained within the bone marrow rather than being released to the blood and is one of the causes of severe inherited neutropenia (low white blood counts).
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 75
You may be able to join if
- All of the following 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
You likely can't join if
- 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
See the full eligibility criteria
- All of the following 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
- people 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...
- Condoms, male or female, with or without a spermicide
- Diaphragm or cervical cap with spermicide
- Intrauterine device
- Contraceptive pills or patch, Norplant, Depo-Provera or other FDA-approved contraceptive method
- Male partner has previously undergone a vasectomy for which there is documentation of aspermatogenic sterility
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Bethesda, Maryland, United States
Compensation & support
Compensation mentioned.
ClinicalTrials.gov doesn't provide a reliable structured field for payment or travel support - confirm details with the study team.
Questions & answers
Do participants get paid in this trial?
This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team - confirm the details with them.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 75 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Bethesda, Maryland, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.