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

NCT06858696 | Recruiting Phase 1 FDA Drug

• 1 other identifier: X4P-001-003
• Study Type: interventional
• Target: 48
• Locations: 1 country
• Sites: 4

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

Outcome Measures

Primary Outcomes (2)

• Maximum Observed Plasma Concentration (Cmax) of Mavorixafor

  Predose up to 192 hours postdose (Day 1 up to Day 9)

• Area Under the Serum Concentration Curve From Time Zero to the Last Measurable Concentration (AUC0-last) of Mavorixafor

  Predose up to 192 hours postdose (Day 1 up to Day 9)

Secondary Outcomes (2)

• Number of Participants With Treatment-emergent Adverse Events (TEAEs)

  Day 1 up to Day 15

• Time to Reach Cmax (Tmax) of Mavorixafor

  Predose up to 192 hours postdose (Day 1 up to Day 9)

Study Arms (6)

Group 1: Child-Pugh A

EXPERIMENTAL

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

Drug: Mavorixafor

Group 2: Child-Pugh B

EXPERIMENTAL

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

Drug: Mavorixafor

Group 3: Child-Pugh C

EXPERIMENTAL

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

Drug: Mavorixafor

Group 4: HVs

EXPERIMENTAL

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.

Drug: Mavorixafor

Group 5: HVs

EXPERIMENTAL

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.

Drug: Mavorixafor

Group 6: HVs

EXPERIMENTAL

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.

Drug: Mavorixafor

Interventions

Mavorixafor DRUG

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

Group 1: Child-Pugh A; Group 2: Child-Pugh B; Group 3: Child-Pugh C; Group 4: HVs; Group 5: HVs; Group 6: HVs

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• X4 Pharmaceuticals: lead

Study Sites (4)

Catalina Research Institute, LLC

Montclair, California, 91763, United States

NOT YET RECRUITING

Catalina Research Institute, LLC

Rialto, California, 91763, United States

RECRUITING

Orlando Clinical Research Center

Orlando, Florida, 32809, United States

NOT YET RECRUITING

Texas Liver Institute/Alamo Medical Research

San Antonio, Texas, 78215, United States

RECRUITING

MeSH Terms

Conditions

Hepatic Insufficiency

Interventions

mavorixafor

Condition Hierarchy (Ancestors)

Liver Diseases; Digestive System Diseases

Study Officials

• Chief Medical Officer

  X4 Pharmaceuticals, Inc.

  STUDY DIRECTOR

Central Study Contacts

X4 Pharmaceuticals, Inc.

CONTACT

857-529-5779; clinicaltrialinfo@x4pharma.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 27, 2025
• First Posted: March 5, 2025
• Study Start: February 28, 2025
• Primary Completion: March 1, 2026
• Study Completion: April 1, 2026
• Last Updated: April 3, 2025

Record last verified: 2025-04

Locations

US (4)