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

NCT ID: NCT06858696

Last Updated: 2025-04-03

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE1
• Total Enrollment: 48 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-02-28
• Study Completion Date: 2026-04-30

Brief 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.

Conditions

Hepatic Insufficiency

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Group 1: Child-Pugh A

Participants with mild HI will receive a single dose of mavorixafor orally on an empty stomach, following a minimum 10-hour fasting period.

Group Type: EXPERIMENTAL

Mavorixafor

Intervention Type: DRUG

Mavorixafor will be administered per schedule specified in the arm description.

Group 2: Child-Pugh B

Participants with moderate HI will receive a single dose of mavorixafor orally on an empty stomach, following a minimum 10-hour fasting period.

Group Type: EXPERIMENTAL

Mavorixafor

Intervention Type: DRUG

Mavorixafor will be administered per schedule specified in the arm description.

Group 3: Child-Pugh C

Participants with severe HI will receive a single dose of mavorixafor orally on an empty stomach, following a minimum 10-hour fasting period.

Group Type: EXPERIMENTAL

Mavorixafor

Intervention Type: DRUG

Mavorixafor will be administered per schedule specified in the arm description.

Group 4: HVs

HVs matched with the mild HI participants will receive a single dose of mavorixafor orally on an empty stomach, following a minimum 10-hour fasting period.

Group Type: EXPERIMENTAL

Mavorixafor

Intervention Type: DRUG

Mavorixafor will be administered per schedule specified in the arm description.

Group 5: HVs

HVs matched with the moderate HI participants will receive a single dose of mavorixafor orally on an empty stomach, following a minimum 10-hour fasting period.

Group Type: EXPERIMENTAL

Mavorixafor

Intervention Type: DRUG

Mavorixafor will be administered per schedule specified in the arm description.

Group 6: HVs

HVs matched with the severe HI participants will receive a single dose of mavorixafor orally on an empty stomach, following a minimum 10-hour fasting period.

Group Type: EXPERIMENTAL

Mavorixafor

Intervention Type: DRUG

Mavorixafor will be administered per schedule specified in the arm description.

Interventions

Mavorixafor

Mavorixafor will be administered per schedule specified in the arm description.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Body weight is more than 50.0 kilograms (kg) with body mass index (BMI) between 18.0 and 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 medical history, physical examination, 12-lead electrocardiogram (ECG), vital signs measurements, and clinical laboratory evaluations.
• 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.

• 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.
• Documented chronic stable liver disease according to CP classification with diagnosis of HI due to parenchymal liver disease.
• 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).

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 pharmacological class 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 accepted clinical 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 is longer) before the first dose of mavorixafor.

• 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/or hepatocellular carcinoma.
• 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.
• 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.

• Clinically significant abnormal laboratory values at screening or Day -1, in the judgment of the Investigator.
• History of liver transplant or currently in the top 5% of recipients on the transplant list.
• Evidence of hepatorenal syndrome or abnormal serum creatinine levels (above upper limit 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 months prior to admission to the clinical site, unless approved by the Investigator and the study Medical Monitor.
• Concurrent conditions that could interfere with safety and/or tolerability measurements.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

X4 Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Chief Medical Officer

Role: STUDY_DIRECTOR

X4 Pharmaceuticals, Inc.

Locations

Catalina Research Institute, LLC

Montclair, California, United States

Site Status: NOT_YET_RECRUITING

Catalina Research Institute, LLC

Rialto, California, United States

Site Status: RECRUITING

Orlando Clinical Research Center

Orlando, Florida, United States

Site Status: NOT_YET_RECRUITING

Texas Liver Institute/Alamo Medical Research

San Antonio, Texas, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

X4 Pharmaceuticals, Inc.

Role: CONTACT

clinicaltrialinfo@x4pharma.com

857-529-5779

Other Identifiers

X4P-001-003

Identifier Type: -

Identifier Source: org_study_id