What side effects are associated with this type of intervention?

Common treatments for Mycobacterium Avium Complex (MAC) Lung Disease include macrolides (clarithromycin, azithromycin), ethambutol, and rifamycins (rifampin). These treatments can cause gastrointestinal disturbances, hepatotoxicity, and visual disturbances. Clarithromycin and azithromycin may lead to gastrointestinal upset and liver toxicity, ethambutol can cause optic neuritis, and rifampin is associated with hepatotoxicity and drug interactions. Epetraborole, a newer agent targeting leucyl-tRNA synthetase, may have a different side effect profile, but similar drugs often cause gastrointestinal issues and potential hepatotoxicity.

What prior FDA approvals exist?

FDA-approved treatments for Mycobacterium Avium Complex (MAC) lung disease typically include a combination of antibiotics such as clarithromycin, ethambutol, and rifampin. These drugs work by inhibiting protein synthesis, cell wall synthesis, and RNA synthesis, respectively. Amikacin, an aminoglycoside, is also used in severe cases. These treatments differ from Epetraborole, which targets leucyl-tRNA synthetase to inhibit bacterial protein synthesis. While Epetraborole's mechanism is unique, it is being studied for its potential to treat MAC lung disease, especially in cases that are refractory to existing treatments.

What other types of research exist?

Promising novel alternatives to Epetraborole for MAC Lung Disease include the combination of rifabutin, clarithromycin, and ethambutol, which has shown effectiveness in animal models. Additionally, the use of inhaled aztreonam lysine for chronic Pseudomonas aeruginosa infection in cystic fibrosis patients may also be considered, given its efficacy and favorable delivery method.

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Epetraborole for Mycobacterium Avium Complex Lung Disease

Q: What is the mechanism of action of this treatment?

The most common treatments for Mycobacterium Avium Complex (MAC) Lung Disease include macrolides (azithromycin, clarithromycin), which inhibit bacterial protein synthesis by binding to the 50S ribosomal subunit, rifamycins (rifampin, rifabutin), which inhibit bacterial RNA synthesis by targeting RNA polymerase, and ethambutol, which disrupts cell wall formation by inhibiting arabinosyl transferases. These mechanisms are crucial for MAC Lung Disease patients as they target essential bacterial processes, reducing bacterial load and improving clinical outcomes. Epetraborole, a newer treatment under study, inhibits bacterial protein synthesis by targeting leucyl-tRNA synthetase, offering a novel mechanism that may be effective against treatment-refractory MAC infections, potentially addressing issues of drug resistance and treatment failure.

Phase 2 & 3

Waitlist Available

Research Sponsored by AN2 Therapeutics, Inc

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Cough with sputum production

Cough without sputum

Must not have

Patients with creatinine clearance (CrCl) of ≤30 mL/min, as estimated by the Cockcroft Gault formula, at Screening

Patients with hemoglobin <10.0 g/dL or <6.2 mmol/L at Screening; donation of blood or plasma within 28 days prior to randomization; or symptomatic loss of blood or hemorrhage within 28 days prior to randomization

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline to month 16

Summary

This trial is testing a new drug called epetraborole, given with standard treatments, in adults with hard-to-treat MAC lung disease. The goal is to see if epetraborole can help clear the lung infection when other treatments have failed.

Who is the study for?

Adults with treatment-refractory MAC lung disease, who have persistent symptoms like coughing and fatigue despite previous treatments. They must show signs of the disease on a CT scan and have positive sputum samples for MAC. Participants should be able to survive with ongoing care, agree to birth control use, and commit to study requirements.

What is being tested?

The trial is testing Epetraborole added to an optimized background regimen (OBR) against a placebo plus OBR in patients whose MAC lung disease hasn't improved with standard treatment. It's a critical Phase 2/3 study where participants are randomly assigned to either the drug or placebo group.

What are the potential side effects?

While specific side effects of Epetraborole aren't listed here, common ones for new drugs can include nausea, diarrhea, liver issues like raised enzyme levels, potential heart rhythm problems (QT interval changes), allergic reactions or skin rashes.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have a cough that produces phlegm.

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I have a dry cough.

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I have chest congestion.

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I have been coughing up blood.

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I experience night sweats or unusual sweating.

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My MAC lung disease has not improved with treatment.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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My kidney function, measured by creatinine clearance, is 30 mL/min or less.

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My hemoglobin is above 10.0 g/dL, and I haven't donated blood or had significant blood loss recently.

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I have not had severe coughing up of blood recently.

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I do not have severe liver problems, such as very high liver enzyme levels or signs of advanced liver disease.

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I have a weak immune system that could lead to lung infections.

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I have never taken epetraborole before.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline to month 16

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline to month 16

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to month 16 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Phase 2: Adverse Event Profile of 500 mg Once Daily Dose of Epetraborole

Phase 2: Assessment of novel Patient Reported Outcome instrument psychometric properties

Phase 2: Percentage of Participants Achieving Clinical Response

+1 more

Secondary study objectives

14. Phase 2: Area Under the Plasma Concentration-Time Curve from Time Point 0 Hours Until 24 hours [AUC(0-24)] post dose

Phase 2: Change from Baseline in NTM Symptoms Module PRO

Phase 2: Change from Baseline in QOL-B Respiratory Domain PRO

+20 more

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: epetraborole + OBRExperimental Treatment1 Intervention

epetraborole + Optimized Background Regimen

Group II: placebo + OBRPlacebo Group1 Intervention

Placebo + Optimized Background Regimen

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Epetraborole

2022

Completed Phase 1

~120

0 / 12Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Mycobacterium Avium Complex (MAC) Lung Disease include macrolides (azithromycin, clarithromycin), which inhibit bacterial protein synthesis by binding to the 50S ribosomal subunit, rifamycins (rifampin, rifabutin), which inhibit bacterial RNA synthesis by targeting RNA polymerase, and ethambutol, which disrupts cell wall formation by inhibiting arabinosyl transferases. These mechanisms are crucial for MAC Lung Disease patients as they target essential bacterial processes, reducing bacterial load and improving clinical outcomes. Epetraborole, a newer treatment under study, inhibits bacterial protein synthesis by targeting leucyl-tRNA synthetase, offering a novel mechanism that may be effective against treatment-refractory MAC infections, potentially addressing issues of drug resistance and treatment failure.