[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"leukopenia\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:leukopenia":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,49,74],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":26,"conditions":27,"keywords":33,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":37,"lastUpdatePostDateStruct":38,"startDateStruct":41,"completionDateStruct":43,"leadSponsor":45,"locationsCount":48},"100130517","phase-1-a-phase-i-study-of-mozobil-in-the-treatment-of-patients-with-whims-100130517",false,"NCT00967785","A Phase I Study of Mozobil in the Treatment of Patients With WHIMS","A Phase I Study of MozobilTM in the Treatment of Patients With WHIMS","* INCLUSION CRITERIA:\n\nAll of the following inclusion criteria must be met for a subject to be enrolled in this study:\n\n* Clinical diagnosis of WHIMS and documented severe infection\n* Must be greater than or equal to 18 and less than or equal to 75 years of age\n* 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\n* Must not be pregnant or breastfeeding\n* Must have a personal physician\n* Must be willing to provide blood, plasma, serum, and DNA samples for storage\n* 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:\n\n  1. Condoms, male or female, with or without a spermicide\n  2. Diaphragm or cervical cap with spermicide\n  3. Intrauterine device\n  4. Contraceptive pills or patch, Norplant, Depo-Provera or other FDA-approved contraceptive method\n  5. Male partner has previously undergone a vasectomy for which there is documentation of aspermatogenic sterility\n\nEXCLUSION CRITERIA:\n\nIf any of the following exclusion criteria are met, a subject will not be enrolled in this study:\n\n* Absence of a diagnosis of WHIMS\n* Patient is less than 18 years old\n* Absence of a documented history of severe infection\n* Neutropenia due to maturation defects in the myeloid lineage or that the PI feels is unlikely to benefit from this medication\n* Pregnant women or breastfeeding\n* History of serious cardiac arrhythmia or cardiac defects that make such more likely\n* Renal failure (calculated creatinine clearance \\[CrCl\\] \\\u003C15 mL\u002Fmin or requiring dialysis)\n* Signs or symptoms of active microbial infection at the time of study entry.\n* Any condition that, in the investigator s opinion, places the patient at undue risk by participating in the study\n* Unwillingness to undergo testing or procedures associated with this protocol","ALL","18 Years","75 Years",{"count":20,"type":21},20,"ESTIMATED","INTERVENTIONAL",[24,25],"PHASE1","PHASE2","Background:\n\n* 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). 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.\n* Current treatment for WHIMS consists of regular injections of a white blood cell growth stimulating medication called granulocyte colony stimulating factor (G-CSF), and supplemental immunoglobulin (antibody). 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).\n* 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.\n\nObjectives:\n\n* To evaluate whether Mozobil is safe and effective to treat neutropenia (low white blood cell count) in patients with WHIMS.\n* To determine an appropriate treatment dose of Mozobil, within currently approved dosage levels.\n\nEligibility:\n\n\\- Individuals between 18 and 75 years of age who have been diagnosed with WHIMS and have a history of severe infections.\n\nDesign:\n\n* Potential participants will undergo a screening 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.\n* Patients who are being treated with G-CSF will stop injections for 2 days before being admitted to the National Institutes of Health (NIH) Clinical Center.\n* Patients may participate in a Dose Escalation study and 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.\n* Patients may also participate in a long-term Chronic Dosing study and receive Mozobil once or twice a day for up to a maximum of 60 months.",[28,29,30,31,32],"Leukopenia","Neutropenia","Infections","Warts","Myelokathexis",[29,34,32,31,35],"Hypogammaglobulinemia","Immunodeficiency","RECRUITING","2026-06-27",{"date":39,"type":40},"2026-06-30","ACTUAL",{"date":42,"type":40},"2010-01-06",{"date":44,"type":21},"2026-12-31",{"name":46,"class":47},"National Institute of Allergy and Infectious Diseases (NIAID)","NIH",1,{"id":50,"slug":51,"hasResults":11,"nctId":52,"briefTitle":53,"officialTitle":54,"acronym":4,"eligibilityCriteria":55,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":56,"targetDuration":4,"studyType":22,"phases":58,"briefSummary":59,"conditions":60,"keywords":4,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":64,"lastUpdatePostDateStruct":65,"startDateStruct":67,"completionDateStruct":69,"leadSponsor":71,"locationsCount":48},"100576699","phase-2-luspatercept-for-clonal-cytopenias-of-uncertain-significance-100576699","NCT06788691","Luspatercept for Clonal Cytopenias of Uncertain Significance","Efficacy of Luspatercept In Clonal Cytopenias of Uncertain Significance","Inclusion Criteria:\n\n* Male or female ≥ 18 years of age.\n* Documentation of a CCUS diagnosis.\n\n  * Clonal cytopenia of undetermined significance (CCUS) is defined as clonal hematopoiesis of indeterminate potential (CHIP) detected in the presence of one or more persistent cytopenias that are otherwise unexplained by hematologic or non-hematologic conditions and that do not meet diagnostic criteria for defined myeloid neoplasms. Cytopenia definitions for diagnosis of CCUS include Hb \\\u003C13 g\u002FdL in males and \\\u003C12 g\u002FdL in females for anemia, absolute neutrophil count \\\u003C1.8 ×109\u002FL for leukopenia, and platelets \\\u003C150 × 109\u002FL for thrombocytopenia.\n  * Patients should harbor somatic mutations of myeloid malignancy-associated genes detected in the blood or bone marrow at a variant allele fraction (VAF) of ≥ 2% (≥4% for X-linked gene mutations in males\n* Clinically significant cytopenias demonstrated in two separate lab draws and defined as cytopenia in any one of the following:\n\n  * Anemia: Transfusion dependent (LTD or HTD for Hb \\\u003C 9 g\u002FdL based on IWG 2018 criteria). Exception for higher threshold up to 10g\u002FdL for documented moderate or severe angina pectoris, cardiac or pulmonary insufficiency, or ischemic neurologic diseases (per IWG 2018 consensus recommendation).\n  * Anemia NTD: symptomatic NTD CCUS with Hb \\\u003C10 g\u002Fdl, symptomatic defined as moderate or worse on ≥ 1 Patient Global Impression of Severity (PGI-S) item (fatigue, shortness of breath, weakness, or dizziness)\n  * Thrombocytopenia: platelet count less than 30,000 \u002FmicroL or \\\u003C 50,000\u002FmicroL with documented bleeding events or high risk for bleeding, for example on blood thinners or drugs that inhibit platelet function for other comorbidities.\n  * Neutropenia: Neutropenia below 750\u002Fmicrol are included in the study. For subjects with neutropenia between 750-1000\u002Fmicrol, subjects should have neutropenia AND a history of serious infection(s).\n* Eastern Cooperative Oncology Group (ECOG) performance status 0, 1, or 2\n* Adequate organ function as defined by:\n\n  * Direct bilirubin \\\u003C 3 x ULN. Indirect hyperbilirubinemia from hemolysis or Gilberts disease are not considered as impaired.\n  * Estimated Creatinine clearance or GFR \\>30 ml\u002Fmin by institutional standards (for example MDRD or Cockcroft Gault or measured by 24 hour urine clearance).\n  * ALT and AST \\\u003C 3 x ULN\n* Females of childbearing potential (FCBP), defined as a sexually mature woman who: 1) has achieved menarche at some point, 2) not undergone a hysterectomy or bilateral oophorectomy or 3) has not been naturally postmenopausal (amenorrhea following cancer therapy or amenorrhea due to other medical reasons does not rule out childbearing potential) for at least 24 consecutive months (i.e., has had menses at any time in the preceding 24 consecutive months), must:\n\n  * Have two negative pregnancy tests (serum or urine) as verified by the investigator prior to starting study therapy (unless the screening pregnancy test was done within 72 hours of W1D1). She must agree to ongoing pregnancy testing during the course of the study, and after end of study treatment.\n  * Either commit to true abstinence1 from heterosexual contact (which must be reviewed on a monthly basis and source documented) or agree to use, and be able to comply with, highly effective contraception2 without interruption, 5 weeks prior to starting investigational product, during the study therapy (including dose interruptions), and for 12 weeks after discontinuation of study therapy.\n\nMale subjects must:\n\n\\- Practice true abstinence1(which must be reviewed prior to each IP administration or on a monthly basis \\[e.g., in the event of dose delays\\]) or agree to use a condom (latex or non-latex, but not made out of natural \\[animal\\] membrane) during sexual contact with a pregnant female or a female of childbearing potential while participating in the study, during dose interruptions and for at least 12 weeks following investigational product discontinuation, even if he has undergone a successful vasectomy.\n\nContraception\n\n* True abstinence is acceptable when this is in line with the preferred and usual lifestyle of the subject. \\[Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception\\].\n* Highly effective contraception is defined in this protocol as the following (information will also appear in the ICF): Hormonal contraception (for example, birth control pills, injection, implant, transdermal patch, vaginal ring); intrauterine device (IUD); tubal ligation (tying your tubes); or a partner with a successful vasectomy.\n\nExclusion Criteria:\n\n* Concurrent malignancy requiring active concurrent systemic chemotherapy. Hormonal therapy for malignancy and targeted radiation is allowed. If patients after enrollment, have a clinical need for chemotherapy after achieving response on treatment, subjects deriving clinical benefit can be continued on study after discussion with study PI.\n* Diagnosis of MDS, AML, MPN or any other myeloid malignancy in the patient's lifetime\n* Active uncontrolled infection that in the investigators opinion will affect study procedures and\u002For results\n* Active uncontrolled hypertension not responding to blood pressure lowering medications which in the investigator's opinion will be harmful for the patient.\n* Use of ESA or growth factors within four weeks prior to the start of the study\n* Known risk factors for thromboembolism (splenectomy, concomitant use of hormone replacement therapy or recent uncontrolled pulmonary embolism or DVT in the last 6 months). Subjects adequately controlled on anticoagulation are permitted.\n* Pregnant or nursing women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using basic methods of contraception during dosing of study treatment and for up to 130 days after last dose of study drug. Basic contraception methods are defined in Section 4.4.\n\n  * Women are considered post-menopausal and not of childbearing potential if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (i.e. age appropriate, history of vasomotor symptoms) or have had surgical bilateral oophorectomy (with or without hysterectomy), total hysterectomy, or bilateral tubal ligation at least six weeks prior to first dose of study drug. In the case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment is she considered not of childbearing potential. If local regulations deviate from the contraception methods listed above to prevent pregnancy, local regulations apply and will be described in the Informed Consent Form (ICF).",{"count":57,"type":21},50,[25],"The purpose of this clinical trial is to test how well the drug luspatercept works in improving low blood cell counts in people with clonal cytopenias of uncertain significance (CCUS). The main questions the study seeks to answer include:\n\n* How many patients experience improvements in their low blood counts (red cells, platelets, or white cells) within 24 weeks, based on specific criteria for blood conditions like myelodysplastic syndromes (MDS)?\n* How long these improvements last before the condition worsens or changes.\n* The percentage of participants showing improvements at 12, 24, and 48 weeks.\n* How long it takes for the condition to progress to more severe diseases like myeloid disorders.\n* How long red blood cell responses last and how quickly these responses are seen.\n* The average change in hemoglobin levels over 24 weeks.\n* How many patients need blood transfusions during the study and how soon transfusions are required.\n* Changes in participants' well-being and energy levels based on a standardized questionnaire.\n* Monitoring for any side effects, including progression to MDS or leukemia, heart-related issues, or sudden increases in hemoglobin.\n\nParticipants will:\n\n* Receive luspatercept as an injection every three weeks.\n* Visit the clinic every three weeks for treatment and monitoring.",[61,62,28,63,29],"CCUS Clonal Cytopenia of Undetermined Significance","Anemia","Thrombocytopenia","2026-03-20",{"date":66,"type":40},"2026-03-24",{"date":68,"type":40},"2025-03-25",{"date":70,"type":21},"2028-02",{"name":72,"class":73},"Weill Medical College of Cornell University","OTHER",{"id":75,"slug":76,"hasResults":11,"nctId":77,"briefTitle":78,"officialTitle":79,"acronym":80,"eligibilityCriteria":81,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":82,"targetDuration":4,"studyType":84,"phases":4,"briefSummary":85,"conditions":86,"keywords":92,"overallStatus":103,"whyStopped":4,"lastUpdateSubmitDate":104,"lastUpdatePostDateStruct":105,"startDateStruct":107,"completionDateStruct":109,"leadSponsor":111,"locationsCount":4},"100601248","hematological-disorders-in-ehpvo-patients-100601248","NCT07108023","Hematological Disorders in EHPVO Patients","A Prospective Study of the Spectrum of Haematological Disorders in Patients With Extrahepatic Portal Vein Obstruction","EHPVO-HEM","Inclusion Criteria:\n\n* Age 18 years or older . Diagnosis of extra hepatic portal vein obstruction based on imaging ( Doppler, CT , MRI ) preserved liver function . Available complete medical records including CBC , LFTs and coagulation profile .\n\nExclusion Criteria:\n\n* Patients with cirrhosis or intrahepatic portal hypertension • Incomplete or missing medical records • Patients with known hematologic malignancies or undergoing chemotherapy",{"count":83,"type":21},115,"OBSERVATIONAL","This study focuses on patients who have a condition called extrahepatic portal vein obstruction (EHPVO), where a blood clot blocks the portal vein outside the liver. This blockage can cause problems like an enlarged spleen, bleeding from swollen veins in the digestive system, and low blood cell counts. Many of these patients may have hidden blood disorders that increase the risk of clotting, such as myeloproliferative neoplasms (MPNs), antiphospholipid syndrome (APS), or paroxysmal nocturnal hemoglobinuria (PNH). This study will collect and analyze blood test results-such as complete blood count (CBC), liver function tests (LFTs), and clotting tests-from patients with EHPVO. The aim is to find patterns that may suggest an underlying blood disorder, even if the patient doesn't show obvious symptoms.By understanding these patterns early, doctors may be able to diagnose and treat the root causes of clotting in these patients more accurately, helping prevent complications and improve outcomes.",[87,88,89,90,91,63,62,28],"Extrahepatic Portal Vein Obstruction (EHPVO)","Thrombophilia","Myeloproliferative Neoplasms (MPN)","Antiphospholipid Syndrome (APS)","Paroxysmal Nocturnal Hemoglobinuria (PNH)",[93,94,95,96,97,98,99,100,101,102],"Portal vein thrombosis","EHPVO","Hypersplenism","Hematological disorders","Coagulation profile","Liver function tests","CBC","JAK2 mutation","Thrombosis in MPN","Cross-sectional study","NOT_YET_RECRUITING","2025-07-31",{"date":106,"type":40},"2025-08-06",{"date":108,"type":21},"2025-08",{"date":110,"type":21},"2025-12",{"name":112,"class":73},"Rahab Nady"]