Actively Recruiting

Phase 2
Age: 18Years +
All Genders
ID07301320

A Randomized, Double-Blind, Placebo-Controlled Trial to Assess the Efficacy, Safety, and Pharmacokinetics of Epetraborole in Adults With Mycobacterium Abscessus Lung Disease

Led by Kevin Winthrop · Updated on 2026-05-06

84

Participants Needed

1

Research Sites

21 weeks

Total Duration

On this page

Sponsors

K

Kevin Winthrop

Lead Sponsor

A

AN2 Therapeutics, Inc

Collaborating Sponsor

AI-Summary

What this Trial Is About

Researchers are evaluating epetraborole (EBO) monotherapy for adults with mild to moderate Mycobacterium abscessus complex (MABc) Lung Disease. This phase 2, double-blind, randomized, placebo-controlled trial aims to assess the efficacy, safety, and pharmacokinetics of two oral EBO doses compared to placebo. MABc lung disease is a chronic infection that requires complicated treatments, and EBO represents a potential new oral therapy option due to its ability to inhibit bacterial protein synthesis and its promising preclinical and pharmacokinetic data. Participants will be randomly assigned to one of four groups: 750 mg daily EBO, 500 mg daily EBO, or matching placebos for each dose. The treatment period lasts 84 days, during which patients take the assigned tablets daily. Some sites will collect pharmacokinetic samples to study drug levels. The study includes oversight by a Data Safety Monitoring Board and uses a comprehensive statistical analysis plan to evaluate results. Throughout the approximately six-month participation, patients will undergo clinical and microbiological assessments, including sputum cultures and symptom evaluations. Quality of life and symptom questionnaires will be completed, and safety will be monitored with clinical and laboratory tests. The primary outcomes include symptom improvement, sputum culture conversion, and quality of life changes by day 84. Secondary outcomes include additional symptom scales and microbiological measures.

CONDITIONS

Brief Title

Epetraborole in Patients With Mycobacterium Abscessus Lung Disease

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Male or female patients aged 18 years or older
  • Willing and able to provide written informed consent
  • Diagnosis of Mycobacterium abscessus lung disease confirmed by at least one MABc-positive respiratory specimen within 6 months prior to consent
  • Presence of at least one MABc-positive sputum sample at screening
  • At least two patient-reported clinical symptoms such as chest pain, chronic cough, coughing up blood, fatigue, fever, mucus production, night sweats, poor appetite, shortness of breath, or unintentional weight loss
  • Chest CT scan within 4 months prior to consent or during screening showing abnormalities consistent with MABc lung disease
  • Not expected to require antibiotic treatment for MABc lung disease within the next 6 months as judged by the investigator
  • Willing to comply with all study procedures
  • Use of effective birth control methods if applicable, including females of childbearing potential and sexually active males
  • Expected to survive with continued antimycobacterial therapy and supportive care through long-term follow-up
Not Eligible

You will not qualify if you...

  • Presence of cavitary lung disease with cavities larger than 2 cm
  • Diagnosis of cystic fibrosis or other inherited airway ciliary dysfunction disorders
  • Active allergic bronchopulmonary mycosis
  • Planned or anticipated lung surgery for MABc lung disease
  • Disseminated MABc infection or other non-pulmonary infections requiring antimicrobial therapy
  • Concurrent pulmonary infections requiring antimicrobials, including fungal, viral, or non-MABc bacterial infections
  • Use of brensocatib without stable dosing for at least 12 weeks
  • Active pulmonary malignancy or malignancy requiring chemotherapy or radiation within 1 year
  • Severe kidney impairment with creatinine clearance under 30 mL/min
  • Hemoglobin under 11.0 g/dL or recent blood donation or hemorrhage within 28 days
  • Pre-existing blood disorders affecting hemoglobin
  • Severe hemoptysis within 28 days
  • Severe liver impairment or signs of end-stage liver disease
  • Pregnancy or breastfeeding
  • Prolonged QT interval over 500 msec on ECG
  • Immunodeficiency conditions including lung transplantation, advanced HIV/AIDS, neutropenia, or immunosuppressive therapy
  • Planned new antimycobacterial therapy between screening and end of treatment
  • Participation in another investigational drug trial within 30 days prior to screening
  • Prior exposure to epetraborole
  • Hypersensitivity to epetraborole excipients
  • History of eosinophilic interstitial pneumonitis
  • Any condition interfering with safe study completion or compliance

AI-Screening

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Treatment

Duration - Up to 84 days

Participants receive daily oral doses of Epetraborole or placebo as part of a randomized treatment regimen for their lung disease.

Visits occur at baseline and periodically through Day 84

Trial Site Locations

Total: 1 location

1

Oregon Health & Science University

Portland, Oregon, United States, 97239

Actively Recruiting

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Research Team

F

Felicity Coulter, PhD

B

Brandy Peacock, DAOM, MAcOM

How is the study designed?

Study Type

INTERVENTIONAL

Masking

QUADRUPLE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

4

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