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

Last updated: April 2, 2025
Sponsor: X4 Pharmaceuticals
Overall Status: Active - Recruiting

Phase

1

Condition

Liver Disease

Primary Biliary Cholangitis

Liver Failure

Treatment

Mavorixafor

Clinical Study ID

NCT06858696
X4P-001-003
  • Ages 18-75
  • All Genders
  • Accepts Healthy Volunteers

Study Summary

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.

Eligibility Criteria

Inclusion

Key Inclusion Criteria:

  • Body weight is more than 50.0 kilograms (kg) with body mass index (BMI) between 18.0and 40.0 kg/square meter (m^2) at the Screening Visit and at Day -1 Visit.

  • In good health, as determined by no clinically significant findings from medicalhistory, physical examination, 12-lead electrocardiogram (ECG), vital signsmeasurements, and clinical laboratory evaluations.

  • Current non-smoker or light smoker, that is, no more than 10 cigarettes or 10milligrams (mg) equivalent use of nicotine per day by e-vapor cigarette, pipe,cigar, chewing tobacco, nicotine patch, nicotine gum, and able and willing torefrain from smoking and tobacco use during the study.

Inclusion criteria applicable to participants with HI Only:

  • Aside from hepatic insufficiency, the participant is deemed by the Investigator tobe sufficiently healthy for study participation, based upon medical history,physical examination, vital signs, and screening laboratory evaluations.

  • Documented chronic stable liver disease according to CP classification withdiagnosis of HI due to parenchymal liver disease.

  • Currently on a stable medication regimen, defined as not starting new drug(s) orchanging drug dose(s) within 28 days of the mavorixafor administration (Day 1).

Exclusion

Key Exclusion Criteria:

  • Female participants/volunteers who are breastfeeding or female participants/volunteers with a positive pregnancy test at the Screening Visit or at Day -1.

  • History of allergy to mavorixafor excipients or drugs in a similar pharmacologicalclass with mavorixafor.

  • Has an active malignancy or history (≤ 5 years prior to enrollment) of solid,metastatic, or hematologic malignancy.

  • A known history of positive serology or viral load for human immunodeficiency virus (HIV) or a known history of acquired immunodeficiency syndrome.

  • Known active COVID-19 infection or a positive test within the local acceptedclinical and governmental guidelines for a communicable window.

  • Positive hepatitis B surface antigen (HbsAg) or hepatitis B core antibody (HbcAb).

  • Positive hepatitis C antibody test result at screening.

  • Have received mavorixafor previously.

  • Has used an investigational drug within 30 days (or 5 half-lives whichever islonger) before the first dose of mavorixafor.

Additional exclusion criteria applicable to Volunteers with Normal Hepatic Function Only:

  • History or evidence of liver disease such as alcoholic liver disease, autoimmunehepatitis, hepatitis B, hepatitis C, primary biliary cirrhosis, primary scleroticcholangitis, Wilson disease, iron overload, alpha-1-antitrypsin deficiency, druginduced liver injury, and/or hepatocellular carcinoma.

  • Significant history or clinical manifestation of any metabolic, allergic,dermatological, hepatic, renal, hematological, pulmonary, cardiovascular (includingany prior history of cardiomyopathy or cardiac failure), gastrointestinal,neurological, or psychiatric disorder.

  • Clinical laboratory test results must be strictly within the normal laboratoryreference ranges for liver function and hematology, and for other parameters, deemedas not clinically significant by the Investigator.

Additional exclusion criteria applicable to participants with HI Only:

  • Clinically significant abnormal laboratory values at screening or Day -1, in thejudgment of the Investigator.

  • History of liver transplant or currently in the top 5% of recipients on thetransplant list.

  • Evidence of hepatorenal syndrome or abnormal serum creatinine levels (above upperlimit for the local lab) and estimated glomerular filtration rate < 60 milliliters (mL)/minute (min) or abnormal sodium and potassium levels.

  • New medication or a change in dose for hepatic encephalopathy within the 3 monthsprior to admission to the clinical site, unless approved by the Investigator and thestudy Medical Monitor.

  • Concurrent conditions that could interfere with safety and/or tolerabilitymeasurements.

NOTE: Other protocol-defined inclusion and exclusion criteria may apply.

Study Design

Total Participants: 48
Treatment Group(s): 1
Primary Treatment: Mavorixafor
Phase: 1
Study Start date:
February 28, 2025
Estimated Completion Date:
April 30, 2026

Connect with a study center

  • Catalina Research Institute, LLC

    Montclair, California 91763
    United States

    Site Not Available

  • Catalina Research Institute, LLC

    Rialto, California 91763
    United States

    Active - Recruiting

  • Orlando Clinical Research Center

    Orlando, Florida 32809
    United States

    Site Not Available

  • Texas Liver Institute/Alamo Medical Research

    San Antonio, Texas 78215
    United States

    Active - Recruiting

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