[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"X4 Pharmaceuticals\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":82},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,56],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":24,"conditions":25,"keywords":27,"overallStatus":43,"whyStopped":4,"lastUpdateSubmitDate":44,"lastUpdatePostDateStruct":45,"startDateStruct":48,"completionDateStruct":50,"leadSponsor":52,"locationsCount":55},"100520417","phase-3-a-study-of-mavorixafor-in-participants-with-congenital-and-acquired-primary-autoimmune-and-idiopathic-chronic-neutropenic-disorders-who-are-experiencing-recurrent-andor-serious-infections-100520417",false,"NCT06056297","A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and\u002For Serious Infections","A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and\u002For Serious Infections","Key Inclusion Criteria:\n\n* Diagnosis of congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorder ≥6 months prior to the screening visit that is not attributable to medications, active or recent infections or malignancy.\n* Congenital Neutropenia, including but not limited to these classifications:\n\n  1. Isolated with a permanent (non-cyclic) presentation, for example, elastase, neutrophil expressed (ELANE), colony stimulating factor 3 receptor (CSF3R), C-X-C chemokine receptor 2 (CXCR2), Wiskott-Aldrich syndrome (WAS)\n  2. Associated with extra-hematologic manifestations, for example, Barth syndrome, Cohen syndrome, glucose-6-phosphatase catalytic subunit 3 (G6PC3), Kostmann disease\n  3. Associated with metabolic disorders, for example, glycogen storage disease 1b (GSD1b)\n  4. Shwachman-Diamond syndrome\n* Acquired Primary Neutropenia\n\n  1. Chronic idiopathic neutropenia\n  2. Primary autoimmune neutropenia. Other chronic neutropenia (CN) disorders that may be eligible for enrollment can be clarified and approved upon discussion with study Medical Monitor.\n* Have an ANC \\\u003C1000 cells\u002FµL during screening (single ANC value from hematology) and confirmed trough mean ANC (mean value of multiple ANC measurements over 6 hours) at baseline visit, with no clinical evidence of systemic infection.\n* Prior history of recurrent and\u002For serious infections during the 12 months preceding the screening visit (that is, suffering sequelae of chronic neutropenia), as defined by having at least 2 infections in the last 12 months that meet the following criteria:\n* Infection requiring the use of antibiotics (intravenous \\[IV\\]\u002Foral); OR\n* Infection requiring a visit to healthcare facility (including but not limited to emergency room visit, urgent care facility, primary care physician's office, or in-patient hospitalization);\n\nAND for all potential participants:\n\n* Infections considered by the Investigator to be likely related to the potential participant's CN disorder.\n* Participants who are on G-CSF or other active background therapy must have been receiving these therapies during the previous 12 months while continuing to suffer from infections, be on a stable dose and dosing schedule for ≥4 weeks prior to screening visit and remain on this dose and dosing schedule throughout the study (unless ANC \\>10,000 cells\u002FµL for ≥4 weeks).\n* Participants must be willing to keep their G-CSF or other background therapy doses\u002Fregimens stable (other than for safety reasons) for the duration of the study.\n\nKey Exclusion Criteria:\n\n* A diagnosis of secondary neutropenia including those due to:\n\n  1. Hypersplenism\n  2. Infection\n  3. Malignancy\n  4. Autoimmune disease, for example, systemic lupus erythematosus, rheumatoid arthritis, inflammatory bowel disease, graft-versus-host disease, thyroid disease\n  5. Nutritional deficiency, for example, vitamin B12, folic acid, copper, caloric malnutrition\n  6. Drug-induced cause, for example, chemotherapy, clozapine, antiretrovirals, antibiotics, monoclonal antibodies.\n* A diagnosis of any of the following:\n\n  1. Aplastic anemia\n  2. Warts, hypogammaglobulinemia, infections, and myelokathexis (WHIM) syndrome\n  3. Certain CNs, including but not limited to these classifications are excluded:\n\n     1. Isolated with a cyclic presentation, for example, elastase, neutrophil expressed (ELANE)\n     2. Associated with immune dysregulation, for example, common variable immunodeficiency (CVID), autoimmune lymphoproliferative syndrome (ALPS), familial hemophagocytic lymphohistiocytosis, Chédiak-Higashi syndrome, GATA-binding protein 2 (GATA2) deficiency syndrome\n     3. Associated with bone marrow failure, for example, Fanconi anemia, Diamond-Blackfan anemia\n  4. Neutropenia associated with a Duffy-null phenotype (formerly known as benign ethnic neutropenia). However, a participant with an autosomal dominant pathogenic variant in a gene associated with CN on a Duffy-null background may be eligible for inclusion\n* A medical or personal condition that may potentially compromise the safety of the participant, may preclude the participant's successful completion of the clinical study, or could, in the opinion of the Investigator or the Medical Monitor, interfere with the objectives of the study.\n* Received more than 1 dose of mavorixafor in the past.\n* Received C-X-C chemokine receptor 4 (CXCR4) antagonist (other than mavorixafor) in the past 6 months.\n* Participants taking pegylated-G-CSF unless they have a diagnosis of congenital neutropenia confirmed at screening.\n* Participant is currently taking or has taken other investigational drug \\\u003C30 days prior to the screening visit or 5 half-lives, whichever is longer.\n\nNote: Other protocol-defined inclusion and exclusion criteria may apply.","ALL","12 Years",{"count":19,"type":20},176,"ESTIMATED","INTERVENTIONAL",[23],"PHASE3","The purpose of this study is to demonstrate the efficacy and evaluate the safety and tolerability of mavorixafor in participants with congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorders who are experiencing recurrent and\u002For serious infections as assessed by demonstrating its clinical benefit and increasing levels of circulating neutrophils.",[26],"Neutropenia",[28,29,30,31,32,33,34,35,36,37,38,39,40,41,42],"Mavorixafor","Chronic neutropenia","Chronic idiopathic neutropenia","Severe Congenital Neutropenia (SCN)","C-X-C chemokine receptor 4 (CXCR4)","Congenital and acquired neutropenia","Autoimmune disease","Cohen syndrome","Barth syndrome","ELANE","G6PC3","GSD1b","Kostmann disease","Shwachman-Diamond syndrome","Autoimmune neutropenia (AIN)","RECRUITING","2026-06-04",{"date":46,"type":47},"2026-06-05","ACTUAL",{"date":49,"type":47},"2024-06-06",{"date":51,"type":20},"2027-11",{"name":53,"class":54},"X4 Pharmaceuticals","INDUSTRY",114,{"id":57,"slug":58,"hasResults":11,"nctId":59,"briefTitle":60,"officialTitle":61,"acronym":4,"eligibilityCriteria":62,"healthyVolunteers":63,"sex":16,"minAge":64,"maxAge":65,"enrollmentInfo":66,"targetDuration":4,"studyType":21,"phases":68,"briefSummary":70,"conditions":71,"keywords":4,"overallStatus":43,"whyStopped":4,"lastUpdateSubmitDate":73,"lastUpdatePostDateStruct":74,"startDateStruct":76,"completionDateStruct":78,"leadSponsor":80,"locationsCount":81},"100582083","phase-1-a-study-to-investigate-pharmacokinetics-pk-and-safety-of-a-single-dose-of-mavorixafor-in-participants-with-hepatic-impairment-hi-compared-to-matched-healthy-volunteers-with-normal-hepatic-function-100582083","NCT06858696","A Study to Investigate Pharmacokinetics (PK) and Safety of a Single Dose of Mavorixafor in Participants With Hepatic Impairment (HI) Compared to Matched Healthy Volunteers With Normal Hepatic Function","An Open-label, Non-randomized Study to Evaluate the Pharmacokinetics (PK), Safety and Tolerability of a Single Dose of Mavorixafor in Participants With Hepatic Impairment (HI) Compared to Matched Healthy Volunteers With Normal Hepatic Function","Key Inclusion Criteria:\n\n* Body weight is more than 50.0 kilograms (kg) with body mass index (BMI) between 18.0 and 40.0 kg\u002Fsquare meter (m\\^2) at the Screening Visit and at Day -1 Visit.\n* In good health, as determined by no clinically significant findings from medical history, physical examination, 12-lead electrocardiogram (ECG), vital signs measurements, and clinical laboratory evaluations.\n* Current non-smoker or light smoker, that is, no more than 10 cigarettes or 10 milligrams (mg) equivalent use of nicotine per day by e-vapor cigarette, pipe, cigar, chewing tobacco, nicotine patch, nicotine gum, and able and willing to refrain from smoking and tobacco use during the study.\n\nInclusion criteria applicable to participants with HI Only:\n\n* Aside from hepatic insufficiency, the participant is deemed by the Investigator to be sufficiently healthy for study participation, based upon medical history, physical examination, vital signs, and screening laboratory evaluations.\n* Documented chronic stable liver disease according to CP classification with diagnosis of HI due to parenchymal liver disease.\n* Currently on a stable medication regimen, defined as not starting new drug(s) or changing drug dose(s) within 28 days of the mavorixafor administration (Day 1).\n\nKey Exclusion Criteria:\n\n* Female participants\u002Fvolunteers who are breastfeeding or female participants\u002F volunteers with a positive pregnancy test at the Screening Visit or at Day -1.\n* History of allergy to mavorixafor excipients or drugs in a similar pharmacological class with mavorixafor.\n* Has an active malignancy or history (≤ 5 years prior to enrollment) of solid, metastatic, or hematologic malignancy.\n* A known history of positive serology or viral load for human immunodeficiency virus (HIV) or a known history of acquired immunodeficiency syndrome.\n* Known active COVID-19 infection or a positive test within the local accepted clinical and governmental guidelines for a communicable window.\n* Positive hepatitis B surface antigen (HbsAg) or hepatitis B core antibody (HbcAb).\n* Positive hepatitis C antibody test result at screening.\n* Have received mavorixafor previously.\n* Has used an investigational drug within 30 days (or 5 half-lives whichever is longer) before the first dose of mavorixafor.\n\nAdditional exclusion criteria applicable to Volunteers with Normal Hepatic Function Only:\n\n* History or evidence of liver disease such as alcoholic liver disease, autoimmune hepatitis, hepatitis B, hepatitis C, primary biliary cirrhosis, primary sclerotic cholangitis, Wilson disease, iron overload, alpha-1-antitrypsin deficiency, drug induced liver injury, and\u002For hepatocellular carcinoma.\n* Significant history or clinical manifestation of any metabolic, allergic, dermatological, hepatic, renal, hematological, pulmonary, cardiovascular (including any prior history of cardiomyopathy or cardiac failure), gastrointestinal, neurological, or psychiatric disorder.\n* Clinical laboratory test results must be strictly within the normal laboratory reference ranges for liver function and hematology, and for other parameters, deemed as not clinically significant by the Investigator.\n\nAdditional exclusion criteria applicable to participants with HI Only:\n\n* Clinically significant abnormal laboratory values at screening or Day -1, in the judgment of the Investigator.\n* History of liver transplant or currently in the top 5% of recipients on the transplant list.\n* Evidence of hepatorenal syndrome or abnormal serum creatinine levels (above upper limit for the local lab) and estimated glomerular filtration rate \\\u003C 60 milliliters (mL)\u002Fminute (min) or abnormal sodium and potassium levels.\n* New medication or a change in dose for hepatic encephalopathy within the 3 months prior to admission to the clinical site, unless approved by the Investigator and the study Medical Monitor.\n* Concurrent conditions that could interfere with safety and\u002For tolerability measurements.\n\nNOTE: Other protocol-defined inclusion and exclusion criteria may apply.",true,"18 Years","75 Years",{"count":67,"type":20},48,[69],"PHASE1","The purpose of this study is to measure the effect of HI on the PK, safety, and tolerability of a single dose of mavorixafor compared to matched healthy volunteers (HVs) with normal hepatic function.",[72],"Hepatic Insufficiency","2025-04-02",{"date":75,"type":47},"2025-04-03",{"date":77,"type":47},"2025-02-28",{"date":79,"type":20},"2026-04",{"name":53,"class":54},4,""]