Epetraborole in Patients With Mycobacterium Abscessus Lung Disease (NCT07301320) | Clinical Trial Compass
RecruitingPhase 2
Epetraborole in Patients With Mycobacterium Abscessus Lung Disease
United States84 participantsStarted 2026-03-30
Plain-language summary
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.
Who can participate
Age range18 Years
SexALL
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Inclusion criteria
✓. Male or female patients who are 18 years of age or older.
✓. Willing and able to provide written informed consent.
✓. Patients with MABc lung disease, meeting the following (a) Microbiological, (b) Clinical, (c) Radiographic
✓. Patients who, in the opinion of the investigator, will not require initiation of guideline-directed antibiotic therapy for treatment of MABc-LD within the next 6 months, and for whom a delay, in order for the subject to participate in a placebo-controlled clinical trial, is considered both reasonable and clinically acceptable.
✓. Patients who are willing to comply with all the study activities and procedures throughout the duration of the study.
✓. Patients must agree to use an effective method of birth control, if applicable, as follows: Females of childbearing potential (FOCPs; defined in Appendix 1, Contraception Requirements) must commit to either sexual abstinence or use of at least 2 medically accepted, effective methods of birth control (defined in Contraception Requirements) from Screening through the EOS Visit
✓. Males who are sexually active with a FOCP must agree to use an effective barrier method of contraception (defined in Contraception Requirements) from Screening through the EOS Visit
✓
What they're measuring
1
Clinical Response (NTM Symptom Evaluation Instrument) at Day 84
Timeframe: Day 1 to Day 84
2
Sputum culture conversion by Day 84
Timeframe: Day 1 to Day 84
3
Decrease in semi-quantitative sputum culture counts at Day 84
Timeframe: Day 1 to Day 84
4
Change from baseline in Quality of Life - Bronchiectasis (QOL-B) Respiratory Domain at Day 84
. Patients expected to survive with continued antimycobacterial therapy and appropriate supportive care from Screening through the LFU Visit, in the judgment of the Investigator.
Exclusion criteria
✕. Patients with a presence of any suspected or confirmed disease or condition at Screening or the time of randomization that, in the opinion of the Investigator, may confound the assessment of symptom-based clinical response, including, but not limited to, the following:
✕. Patients on brensocatib who have not been on a stable dose for at least 12 weeks.
✕. Patients with active pulmonary malignancy (primary or metastatic) or any malignancy that required or would require chemotherapy or radiation therapy within 1 year prior to randomization through the LFU Visit.
✕. Patients with creatinine clearance (CrCl) of \<30 mL/min, as estimated by the Cockcroft-Gault formula, at Screening: Estimated CrCl (mL/min) = (140 - Age \[years\]) × Actual Body Weight \[kg\] × \[0.85 if Female\]) / (72 × Serum Creatinine \[mg/dL\]).
✕. Patients with any of the following hematological findings:
✕. Hemoglobin \<11.0 g/dL or \<6.83 mmol/L.
✕. Donation of blood or plasma within 28 days prior to randomization; or symptomatic loss of blood or hemorrhage within 28 days prior to randomization.
✕. Pre-existing (inborn or acquired) disorder of hematopoiesis that could substantially worsen the effect of EBO on hemoglobin levels or potentially prevent hemoglobin recovery post cessation of treatment, for example, thalassemia, sickle cell disease, hemolytic anemia, Inherited bone marrow failure syndromes, myelodysplastic or myeloproliferative disorders, bone marrow transplantation