Actively Recruiting
Epetraborole in Patients With Mycobacterium Abscessus Lung Disease
Led by Kevin Winthrop · Updated on 2026-05-06
84
Participants Needed
1
Research Sites
109 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
This double-blind, randomized, placebo-controlled, parallel-group, multicenter, prospective, investigator-initiated trial will evaluate epetraborole (EBO) monotherapy in the treatment of adults with Mycobacterium abscessus complex (MABc) Lung Disease (LD) of mild to moderate severity. For this study, two EBO oral dose regimens will be studied in patients with MABc-LD, each compared to a placebo group (ie, 4 treatment groups): 500 mg daily and 750 mg daily. Detailed inclusion and exclusion criteria attempt to identify only those patients who have acceptable risks based upon the EBO preclinical findings, phase 1, phase 2, and Phase 3 experience; standard-of-care procedures; and the specified procedures of the study. Following receipt of informed consent, and a Screening period, eligible patients will be randomized to one of the 4 treatment groups to receive active or matched placebo EBO tablets for 84 days. Patients will be assessed for clinical and microbiological evidence of efficacy. At selected investigative sites, patients will undergo sparse PK sampling. Safety and tolerability will be determined by standard clinical and laboratory assessment, with oversight by a qualified and appropriately constituted Data Safety Monitoring Board (DSMB). Data collected during the study will be analyzed per a comprehensive Statistical Analysis Plan (SAP). The study will be registered on clinicaltrials.gov. The total duration of patient participation is approximately 6 months.
CONDITIONS
Official Title
Epetraborole in Patients With Mycobacterium Abscessus Lung Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female patients 18 years of age or older
- Able and willing to provide written informed consent
- Diagnosis of MABc lung disease confirmed by at least one positive respiratory specimen within 6 months prior to consent and one positive sputum sample at screening
- Presence of at least two clinical symptoms such as chest pain, chronic cough, coughing up blood, fatigue, fever, sputum production, night sweats, poor appetite, shortness of breath, or unintentional weight loss
- Chest CT scan within 4 months before consent or during screening showing abnormalities consistent with MABc lung disease
- Investigator judgment that no need for antibiotic therapy for MABc lung disease within the next 6 months and that delay is reasonable
- Willingness to comply with all study activities and procedures for the study duration
- Females of childbearing potential must agree to use two effective birth control methods or sexual abstinence from screening through study end
- Males sexually active with females of childbearing potential must use effective barrier contraception from screening through study end
- Expected to survive with continued antimycobacterial therapy and supportive care through follow-up visit as judged by investigator
You will not qualify if you...
- Presence of diseases or conditions that may affect symptom assessment, including cavitary lung disease with cavities larger than 2 cm
- Cystic fibrosis or 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
- Concomitant pulmonary infections requiring antimicrobial therapy (fungal, viral, non-MABc mycobacteria, or other bacteria) unless resolved prior to randomization
- Unstable use of brensocatib (less than 12 weeks at stable dose)
- Active pulmonary malignancy or malignancy requiring chemotherapy or radiation within 1 year prior to randomization
- Creatinine clearance below 30 mL/min at screening
- Hemoglobin below 11.0 g/dL or recent blood loss or donation within 28 days prior to randomization
- Pre-existing blood disorders affecting hemoglobin recovery
- Severe hemoptysis (>100 mL in 24 hours) within 28 days prior to randomization
- Severe liver impairment or signs of end-stage liver disease
- Pregnancy or breastfeeding
- QTcF interval over 500 msec at screening ECG
- Immunodeficiency or immunocompromised conditions increasing risk of opportunistic infections
- Planned new non-study antimycobacterial therapy between screening and end of treatment
- Participation in another investigational trial within 30 days prior to screening
- Prior exposure to epetraborole
- Any condition interfering with safe study completion or adherence
- Known hypersensitivity to epetraborole excipients
- History of eosinophilic interstitial pneumonitis
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Oregon Health & Science University
Portland, Oregon, United States, 97239
Actively Recruiting
Research Team
F
Felicity Coulter, PhD
CONTACT
B
Brandy Peacock, DAOM, MAcOM
CONTACT
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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