A Phase I Study of Mozobil in the Treatment of Patients With WHIMS
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|ClinicalTrials.gov Identifier: NCT00967785|
Recruitment Status : Recruiting
First Posted : August 28, 2009
Last Update Posted : June 13, 2018
- WHIMS (warts, hypogammaglobulinemia, infection, and myelokathexis syndrome) is caused by various genetic changes that increase the activity of the CXCR4 gene. Excessive function of this gene causes mature neutrophils (part of white blood cells) to be retained within the bone marrow rather than being released to the general blood circulation, and is one of the causes of severe inherited neutropenia (low white blood counts). In neutropenia, the body is less able to fight off infection. Patients with WHIMS usually are at risk for skin, soft tissue, sinus, and lung infections, which can result in loss of hearing, teeth, and lung function.
- Current treatment for WHIMS consists of regular injections of a white blood cell growth stimulating medication called granulocyte colony stimulating factor (G-CSF), and monthly infusions of intravenous immunoglobulin (IVIG). These therapies are expensive, nonspecific, have significant side effects and toxicities, and do not fully correct all problems, especially warts and cancers related to human papillomavirus (HPV).
- A drug called Mozobil has been approved for use in combination with G-CSF to increase the number of stem cells that can be collected prior to bone marrow transplantation. Mozobil may offer a specific and well-tolerated new treatment for WHIMS and other syndromes characterized by neutropenia.
- To evaluate whether Mozobil is safe and effective to treat neutropenia (low white blood cell count) in patients with WHIMS.
- To determine an appropriate treatment dose of Mozobil, within currently approved dosage levels.
- Individuals between 18 and 75 years of age who have been diagnosed with WHIMS and have a history of severe infections.
- Potential participants will undergo a screening study, with a medical history, physical examination, questionnaire, heart and lung function scans, and blood and urine samples. Tests will also be done for hepatitis B and C virus, and human immunodeficiency virus (HIV) that causes acquired immunodeficiency syndrome (AIDS), as well as to check neutrophil function.
- Patients who are being treated with G-CSF will stop injections for 5 days before being admitted to the National Institutes of Health (NIH) Clinical Center. While off the medication, patients will keep a diary to report information about their general well-being while off the medications and will bring it to NIH when admitted.
- Patients will be admitted to the NIH Clinical Center inpatient until 2 days before starting the treatment and will stay for approximately 10 days. Before the treatment, patients will have blood samples taken regularly.
- Patients will receive increasing doses of Mozobil over 5 days of treatment until their white blood cell count improves sufficiently or the maximum approved dose is reached. Blood samples will be taken regularly throughout the treatment process. Patients will then receive an additional dose of Mozobil at the maximum approved dose or the dose sufficient to cause improvement, before restarting the G-CSF injections.
- Between 4 and 6 weeks after the treatment phase, patients will return to the NIH for an outpatient study completion and evaluation visit.
|Condition or disease||Intervention/treatment||Phase|
|WHIMS Neutrophil Disorder Myelokathexis Hypogammaglobulinemia||Drug: Mozobil (TM)||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||20 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase I Study of Mozobil (TM) in the Treatment of Patients With WHIMS|
|Study Start Date :||August 4, 2009|
|Estimated Primary Completion Date :||September 30, 2021|
|Estimated Study Completion Date :||December 31, 2021|
- Determine whether or not Mozobil (TM) is safe in study population.
- Whether or not treatment results in increased ANC.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00967785
|Contact: Elena J Cho||(301) firstname.lastname@example.org|
|Contact: David H McDermott, M.D.||(301) email@example.com|
|United States, Maryland|
|National Institutes of Health Clinical Center, 9000 Rockville Pike||Recruiting|
|Bethesda, Maryland, United States, 20892|
|Contact: For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR) 800-411-1222 ext TTY8664111010 firstname.lastname@example.org|
|Principal Investigator:||David H McDermott, M.D.||National Institute of Allergy and Infectious Diseases (NIAID)|