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A Study of Mavorixafor in Participants With Severe Congenital Neutropenia and Chronic Neutropenia Disorders

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04154488
Recruitment Status : Not yet recruiting
First Posted : November 6, 2019
Last Update Posted : June 11, 2020
Sponsor:
Information provided by (Responsible Party):
X4 Pharmaceuticals

Brief Summary:
This Phase 1b study will determine the safety and tolerability of mavorixafor in participants with severe chronic idiopathic neutropenia (CIN) and selected congenital neutropenia disorders. The anticipated enrollment is up to 45 participants.

Condition or disease Intervention/treatment Phase
Neutropenia Drug: Mavorixafor Phase 1

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 45 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1B, Open-Label, Multicenter Study of Mavorixafor in Patients With Severe Congenital Neutropenia and Chronic Neutropenia Disorders
Estimated Study Start Date : August 15, 2020
Estimated Primary Completion Date : October 15, 2021
Estimated Study Completion Date : December 15, 2021


Arm Intervention/treatment
Experimental: Mavorixafor 400 mg
Participants will receive mavorixafor 400 milligrams (mg) (4 capsules of 100 mg each) orally once daily (QD) in the morning for 14 days.
Drug: Mavorixafor
Mavorixafor capsules will be administered per dose and schedule specified in the arm.
Other Name: X4P-001




Primary Outcome Measures :
  1. Number of Participants With Treatment-Emergent Adverse Events (TEAEs) [ Time Frame: Baseline (Day -1) up to end of study (Day 44) ]

Secondary Outcome Measures :
  1. Absolute Neutrophil Count (ANC) of Mavorixafor in Participants With Severe CIN in Combination With Steady-State Granulocyte-Colony Stimulating Factor (G-CSF) [ Time Frame: Time 0 (pre-dose, up to 15 minutes prior), 30, 60, and 90 minutes (each ± 5 minutes) and 2, 3, 4, and 6 hours (each ± 15 minutes) post-dose at Day 1, 8, and 14 ]
  2. Area Under the Curve for ANC (AUCANC) of Mavorixafor in Participants With Severe CIN in Combination With Steady-State G-CSF [ Time Frame: Time 0 (pre-dose, up to 15 minutes prior), 30, 60, and 90 minutes (each ± 5 minutes) and 2, 3, 4, and 6 hours (each ± 15 minutes) post-dose at Day 1, 8, and 14 ]
  3. Serum Concentration of Mavorixafor in Relation to ANC and AUCANC in Participants With Severe CIN [ Time Frame: Time 0 (pre-dose, up to 15 minutes prior), 30, 60, and 90 minutes (each ± 5 minutes) and 2, 3, 4, and 6 hours (each ± 15 minutes) post-dose at Day 1, 8, and 14 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • For all participants:

    • Sign the informed consent form (ICF) and be willing and able to comply with the protocol.
    • Agree to use contraception as follows:

      i) For women of childbearing potential (WOCBP): agree to use highly effective contraceptive methods from screening through the study and for at least 4 weeks after the last dose of study drug.

ii) For male participants: agree to use a condom with any WOCBP sexual partner from screening through the study and for at least 4 weeks after the last dose of study drug.

  • Participants with severe chronic idiopathic neutropenia (CIN):

    • Be at least 18 years of age.
    • Have a history of ANC less than (<) 500 cells/microliter (μL), lasting for more than 3 months at any time since diagnosis.
    • Have been diagnosed with severe CIN more than 12 months ago that is not attributable to medications, infectious, genetic, inflammatory, autoimmune, or malignant causes.
    • Be currently treated with a prophylactic steady-state G-CSF regimen for greater than (>) 15 days before receiving the first dose of study drug.
    • Have normal cytogenetics on the most recent bone marrow biopsy/aspirate, if performed.
    • Have no associated thrombocytopenia nor anemia before G-CSF therapy initiation.
  • Participants with selected congenital neutropenia conditions including GSD1b (GSD1b; SLC37A4), G6PC3 deficiency (G6PC3), or GATA2 deficiency (GATA2):

    • Be at least 12 years of age.
    • May be currently receiving steady-state G-CSF dosing or not have been on G-CSF for >15 days.
    • Must have documentation of the mutational status before enrolling in the study.

Exclusion Criteria:

  • Known systemic hypersensitivity to the mavorixafor drug substance or its inactive ingredients.
  • Diagnosis of cyclical neutropenia.
  • Is pregnant or nursing.
  • Known history of a positive serology or viral load for human immunodeficiency virus (HIV) or a known history of acquired immune deficiency syndrome.
  • At screening, has laboratory test results meeting one or more of the following criteria:

    • Positive hepatitis C virus (HCV) antibodies with confirmation by HCV-ribonucleic acid polymerase chain reaction reflex testing.
    • Positive hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb).

Note: If a participant tests negative for HBsAg but positive for HBcAb, the participant would be considered eligible if the participant tests positive for antibody to HBsAg reflex testing.

  • At screening, has laboratory test results meeting one or more of the following criteria:

    • Hemoglobin <10.0 grams/deciliter (g/dL)
    • Platelet count <175,000 cells/μL
    • Mild/moderate renal impairment 30 to 60 glomerular filtration rate
    • Serum aspartate transaminase >2.5 * upper limit of normal (ULN)
    • Serum alanine transaminase >2.5 * ULN
    • Total bilirubin >1.5 * ULN (unless due to Gilbert's syndrome, in which case total bilirubin greater than or equal to (≥) 3.0 * ULN and direct bilirubin >1.5 * ULN)
  • Within 2 weeks before Day 1, received any of the following treatments:

    • Glucocorticoids (>5 mg prednisone equivalent per day)
    • Medication prohibited based on cytochrome P450 (CYP) and/or P-glycoprotein (P-gp) interaction.
  • At the planned initiation of study drug, has an uncontrolled infection requiring chronic or prophylactic use of antibiotics (systemic or inhaled) within 4 weeks before Day 1.
  • Has any other medical or personal condition that, in the opinion of the Investigator, may potentially compromise the safety or compliance of the participant, or may preclude the participant's successful completion of the clinical study.
  • Inability to ingest study drug as presented.
  • Has a history of any hematologic malignancy.
  • Diagnosed or suspected congenital long QT syndrome. Any history of clinically significant ventricular arrhythmias (such as ventricular tachycardia, ventricular fibrillation, or torsades de pointes); any history of arrhythmia will be discussed with the sponsor's medical monitor before participant's entry into the study.
  • Prolonged corrected QT interval (QTc) using Fridericia's formula (QTcF) on pre-entry electrocardiogram (ECG) (≥450 milliseconds [ms]).
  • Has an associated diagnosis of rheumatoid arthritis, systemic lupus erythematosus, or inflammatory bowel disease (IBD).
  • Has been treated with G-CSF >8 micrograms/kilogram/day (μg/kg/day) for more than 1 consecutive month within the past 2 years and/or has been on G-CSF therapy >10 years regardless of the dose.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04154488


Contacts
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Contact: Patient Affairs and Advocacy 857-529-5779 patientinfo@x4pharma.com
Contact: Patient Affairs and Advocacy patientinfo@x4pharma.com

Locations
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United States, Washington
University of Washington
Seattle, Washington, United States, 98195
Contact: David C. Dale         
Sponsors and Collaborators
X4 Pharmaceuticals
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Responsible Party: X4 Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04154488    
Other Study ID Numbers: X4P-001-104
First Posted: November 6, 2019    Key Record Dates
Last Update Posted: June 11, 2020
Last Verified: June 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Neutropenia
Agranulocytosis
Leukopenia
Leukocyte Disorders
Hematologic Diseases