Mavorixafor for Liver Failure

Recruiting at 3 trial locations

Overseen By X4 Pharmaceuticals, Inc.

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: X4 Pharmaceuticals

Must not be taking: Investigational drugs

Disqualifiers: Pregnancy, Active malignancy, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: Single dose

48 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test how a new drug, mavorixafor, affects people with liver failure. Researchers seek to understand the drug's behavior in the body and assess its safety and tolerability. Participants will include individuals with mild to severe liver problems and healthy individuals for comparison. Ideal candidates have stable liver disease, have not recently changed their medications, and are in generally good health aside from their liver condition. As a Phase 1 trial, participants will be among the first to receive this new treatment, aiding researchers in understanding its effects in people.

Will I have to stop taking my current medications?

The trial requires that participants with liver issues be on a stable medication regimen, meaning you should not start new medications or change doses within 28 days before taking mavorixafor. If you're already on stable medications, you likely won't need to stop them.

Is there any evidence suggesting that mavorixafor is likely to be safe for humans?

Research has shown that mavorixafor has undergone safety testing in humans. The FDA first approved it for treating warts, indicating it passed safety checks for that use. However, its safety in individuals with liver problems remains under investigation.

Mavorixafor is processed by the liver, so liver issues could affect how the body handles the drug. The exact effects on individuals with severe liver problems are not fully known, but the ongoing study aims to learn more about this.

As this study is in its early stages, it primarily focuses on how well individuals can tolerate mavorixafor and any side effects they might experience. The treatment is not yet proven safe for liver issues, but the study will help gather important information. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Mavorixafor is unique because, unlike traditional treatments for liver failure that focus on managing symptoms or supporting liver function, it is designed to target the underlying mechanisms of liver impairment. Researchers are excited because Mavorixafor acts on the CXCR4 receptor, which plays a crucial role in immune cell trafficking and inflammation. This novel mechanism could potentially reduce liver damage and improve liver function more effectively than current options. Additionally, its oral administration after a short fasting period offers a convenient and non-invasive alternative for patients.

What evidence suggests that mavorixafor might be an effective treatment for liver failure?

Studies have shown that the liver mainly processes mavorixafor. As a result, individuals with liver problems might experience higher levels of the drug in their bodies. Previous research in animals did not indicate that mavorixafor caused additional liver damage or inflammation, suggesting it might be safe for those with liver issues. However, limited information exists on its effects in people with liver failure. This trial will assess the safety and tolerability of mavorixafor in participants with varying degrees of liver impairment, including mild, moderate, and severe cases, as well as in healthy volunteers matched to these groups. ^{1 3 5 6 7}

Who Is on the Research Team?

Chief Medical Officer

Principal Investigator

X4 Pharmaceuticals, Inc.

Are You a Good Fit for This Trial?

This trial is for adults with liver impairment and healthy volunteers matched by age, weight, and gender. Participants must not have other significant medical conditions or be taking conflicting medications that could affect the study results.

Inclusion Criteria

I smoke less than 10 cigarettes a day or use an equivalent amount of nicotine and can stop during the study.

I have a long-term liver condition with a specific diagnosis related to liver tissue damage.

I weigh over 50 kg and my BMI is between 18.0 and 40.0.

Exclusion Criteria

Positive hepatitis C antibody test result at screening

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

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

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single dose of mavorixafor orally on an empty stomach after a minimum 10-hour fasting period

Single dose

Follow-up

Participants are monitored for safety and effectiveness after receiving the single dose

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Mavorixafor

Trial Overview The study measures how a single dose of Mavorixafor is processed in the body (pharmacokinetics) and its safety in people with different levels of liver function compared to those with normal liver health.

How Is the Trial Designed?

Group I: Group 6: HVs Experimental Treatment 1 Intervention

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 II: Group 5: HVs Experimental Treatment 1 Intervention

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 III: Group 4: HVs Experimental Treatment 1 Intervention

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 IV: Group 3: Child-Pugh C Experimental Treatment 1 Intervention

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

Group V: Group 2: Child-Pugh B Experimental Treatment 1 Intervention

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

Group VI: Group 1: Child-Pugh A Experimental Treatment 1 Intervention

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

Who Is Running the Clinical Trial?

X4 Pharmaceuticals

Lead Sponsor

Trials

11

Recruited

400+

Citations

1. centerwatch.com centerwatch.com/clinical-trials/listings/NCT06858696/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?state=TX&city=San%20Anatanio

A Study to Investigate Pharmacokinetics (PK) and Safety of ... 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 ...

2. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/nda/2024/218709Orig1s000IntegratedR.pdf

218709Orig1s000 INTEGRATED REVIEW - accessdata.fda.gov Mavorixafor is primary metabolized by the liver and hepatic impairment can be expected to result in increased mavorixafor exposures. Data from a dedicated ...

3. withpower.com withpower.com/trial/phase-1-liver-failure-1-2025-d0ee3

Mavorixafor for Liver Failure 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 ...

4. clinconnect.io clinconnect.io/trials/NCT06858696

A Study to Investigate Pharmacokinetics (PK) and Safety This clinical trial is investigating how a single dose of a medication called mavorixafor affects people with liver issues (hepat hepatic insufficiency)

5. cdn.clinicaltrials.gov cdn.clinicaltrials.gov/large-docs/27/NCT03005327/Prot_000.pdf

This study will be conducted according to the protocol and ... Overall, there was no meaningful elevation of circulating hepatic enzymes reflective of liver injury or active inflammatory response in these animals.

6. go.drugbank.com go.drugbank.com/drugs/DB05501

Mavorixafor: Uses, Interactions, Mechanism of Action Mavorixafor is a CXC chemokine receptor 4 (CXCR4) antagonist. 6 It was first approved by the FDA on April 30, 2024, for the treatment of warts, ...

7. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/label/2024/218709s000lbl.pdf

XOLREMDI™ (mavorixafor) - accessdata.fda.gov The pharmacokinetics of mavorixafor have not been studied in subjects with moderate to severe hepatic impairment [see Clinical Pharmacology (12.3)]. 11 ...