A Study of Mavorixafor in Participants With Congenital Neutropenia and Chronic Idiopathic Neutropenia Disorders
- Conditions
- Neutropenia
- Registration Number
- NCT04154488
- Lead Sponsor
- X4 Pharmaceuticals
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Active, not recruiting
- Sex
- All
- Target Recruitment
- 43
Key Inclusion Criteria:<br><br>For all participants (Parts 1 and 2):<br><br> - Sign the informed consent form (ICF) and be willing and able to comply with the<br> protocol.<br><br> - Weigh =15 kg<br><br> - Agree to use a highly effective form of contraception if sexually active.<br><br> - Participants may be eligible for the study whether they are on or off<br> granulocyte-colony stimulating factor (G-CSF) treatment.<br><br> - Note: Participants who are on G-CSF must be on a stable dose for =14 days prior<br> to the Baseline visit and should not have an ANC =10,000 cells/µL.<br><br> - Note: Participants who are not on G-CSF must be off for =14 days prior to the<br> Baseline visit and have an ANC =1000 cells/µL at the Screening visit.<br><br> - Note: Participants with Shwachman-Diamond syndrome, Cohensyndrome, and warts,<br> hypogammaglobulinemia, infections and myelokathexis syndrome are eligible.<br> Other types of chronic neutropenic disorders may also be eligible for<br> enrollment upon discussion and approval with Sponsor and Study Medical Monitor.<br><br> - Have been diagnosed with chronic neutropenia for =6 months prior to the Screening<br> visit that is not attributable to medications, active or recent (=3 months)<br> infections, or malignant cause.<br><br>Part 2 only:<br><br> - Participants enrolled in the study before implementation of Protocol Version 8.0<br> must have completed Part 1 and exhibited a positive response to treatment.<br><br> - Participant has a history of symptomatic chronic neutropenia confirmed by the<br> Investigator.<br><br>Key Exclusion Criteria (Parts 1 and 2):<br><br> - Known systemic hypersensitivity to the mavorixafor drug substance or its inactive<br> ingredients.<br><br> - Is pregnant, breastfeeding, or plans to become pregnant over the next 8 months.<br><br> - Known history of a positive serology or viral load for human immunodeficiency virus<br> (HIV) or a known history of acquired immune deficiency syndrome.<br><br> - Known active SARS-CoV-2 virus (COVID-19) infection or a positive test within the<br> local accepted clinical and governmental guidelines for a communicable window.<br><br> - At the Screening Visit, has laboratory test results meeting one or more of the<br> following criteria:<br><br> - Positive hepatitis C virus (HCV) antibodies with confirmation by<br> HCV-ribonucleic acid polymerase chain reaction reflex testing.<br><br> - Positive hepatitis B surface antigen (hBsAg) or hepatitis B core antibody<br> (hBcAb).<br><br> - Note: If a participant tests negative for hBsAg but positive for hBcAb, the<br> participant would be considered eligible if the participant tests positive for<br> antibody to hBsAg reflex testing.<br><br> - At the Screening visit, has laboratory test results meeting one or more of the<br> following criteria:<br><br> - Hemoglobin <9.0 grams/deciliter (g/dL)<br><br> - Platelets <30,000/µL<br><br> - Estimated glomerular filtration rate (eGFR) =60 mL/minute/1.73 meter (m)^2, as<br> estimated by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI)<br> equation<br><br> - Serum aspartate transaminase >2.5 x upper limit of normal (ULN)<br><br> - Serum alanine transaminase >2.5 x ULN<br><br> - Total bilirubin >1.5 x ULN (unless due to Gilbert's syndrome, in which case<br> total bilirubin greater than or equal to (=) 3.0 x ULN and direct bilirubin<br> >1.5 x ULN)<br><br> - =14 days before Day 1, received any of the following treatments:<br><br> - Systemic glucocorticoids (>5 mg prednisone equivalent per day).<br><br> - Medication prohibited based on cytochrome P450 (CYP)2D6 potential for<br> interaction.<br><br> - Is taking a medication which is a strong inhibitor of CYP3A4, unless the<br> Investigator and Medical Monitor conclude that the medication must be used during<br> the study and is permissible with modification of mavorixafor dose.<br><br> - Has an infection requiring use of systemic antibiotics =4 weeks before the Baseline<br> visit.<br><br> - Has a medical or personal condition that may potentially compromise the safety or<br> compliance of the participant, or may preclude the participant's successful<br> completion of the clinical study or that in the opinion of the Investigator or the<br> Sponsor could interfere with the objectives of the study.<br><br> - Has had major surgery =4 weeks before the Baseline visit.<br><br> - Inability to ingest mavorixafor capsules.<br><br> - Has an active malignancy or history of (=5 years prior to enrollment) in the study<br> of solid, metastatic, or hematologic malignancy. Exception: basal cell carcinoma in<br> situ of the skin that has been adequately treated.<br><br> - Diagnosed or has suspected congenital long QT syndrome. Any history of clinically<br> significant ventricular arrhythmias (such as ventricular tachycardia, ventricular<br> fibrillation, or torsades de pointes); any history of arrhythmia will be discussed<br> with the sponsor's medical monitor before participant's entry into the study.<br><br> - Prolonged corrected QT interval using Fridericia's formula at the Screening visit<br> electrocardiogram (ECG) (>450 milliseconds [ms])
Not provided
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Number of Participants With Treatment-Emergent Adverse Events (TEAEs) After a Single Dose of Mavorixafor;Number of Participants With Treatment-Emergent Adverse Events (TEAEs) After Multiple Doses of Mavorixafor;Change From Baseline in Absolute Neutrophil Count (ANC) to 8 hours Post-dose On Day 1;Change From Baseline in ANC to Month 6
- Secondary Outcome Measures
Name Time Method Serum Concentration of Mavorixafor in Relation to ANC and Area Under the Curve (AUC) for ANC (AUCANC);Serum Concentrations of Mavorixafor;Change from Baseline in Absolute Lymphocyte Count (ALC);Change from Baseline in Total White Blood Cells (WBC);Change from Baseline in Absolute Monocyte (AMC);AUC of ANC (AUCANC);AUC of ALC (AUCALC);AUC of AMC (AUCAMC);AUC of WBC (AUCWBC)