A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Prospective, Investigator-Initiated Trial to Assess the Efficacy, Safety, and Pharmacokinetics of Epetraborole in Patients With Mycobacterium Abscessus Lung Disease: REBOUND Study
Kevin Winthrop
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.
Description
Non-tuberculous mycobacterial lung disease (NTM-LD) is a chronic, debilitating disease that can cause significant morbidity and mortality, and reduces quality of life. The primary intervention to treat NTM-LD is antibiotic treatment to eliminate the causative pathogen and thereby prevent progression of NTM-associated lung destruction and respiratory compromise. Among NTM organisms causing pulmonary infection, Mycobacterium abscessus complex (MABc) LD requires particularly complicated, prolonged and onerous intravenous treatment regimens that are burdensome to patients. New therapeutic options,…
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Male or female patients who are 18 years of age or older. 2. Willing and able to provide written informed consent. 3. Patients with MABc lung disease, meeting the following (a) Microbiological, (b) Clinical, (c) Radiographic a. Microbiological criteria: i. Documentation of at least 1 Pre-Study MABc-positive respiratory specimen (sputum or deep bronchial specimen) collected per standard of care within 6 months prior to signing the consent form. ii. At least 1 Screening MABc-positive expectorated or induced sputum sample. b. Clinical criteria: At least 2 of the fo…
Interventions
- DrugEpetraborole
High-dose intervention (750mg daily)
- DrugEpetraborole
Low-dose intervention (500mg daily)
- DrugPlacebo
Placebo intervention (matching the high-dose experimental intervention)
- DrugPlacebo
Placebo intervention (matching the low-dose experimental intervention)
Location
- Oregon Health & Science UniversityPortland, Oregon