A Study of Bedaquiline Administered as Part of a Treatment Regimen With Clarithromycin and Ethambutol in Adult Patients With Treatment-refractory Mycobacterium Avium Complex-lung Disease (MAC-LD)

Janssen Pharmaceutical K.K.129 enrolled

Overview

The purpose of the study is to evaluate the efficacy of bedaquiline (BDQ) compared with rifamycin when administered as part of a treatment regimen with clarithromycin (CAM) and ethambutol (EB) in adult participants with treatment-refractory Mycobacterium avium complex-lung disease (MAC-LD) at Week 24 for microbiological assessment in mycobacteria growth indicator tube (MGIT).

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

129

Conditions

Treatment-refractory Mycobacterium Avium Complex-lung Disease (MAC-LD)

Interventions

Bedaquiline

Eligibility

Sex: ALLMin age: 20 YearsMax age: 79 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Has body weight greater than or equal to (\>=) 40 kilograms (kg) at screening and on Day 1 * Has radiological evidence consistent with nontuberculous mycobacterial lung disease (NTM-LD) based on a chest Computed Tomography (CT) scan taken within 6 months prior to screening or at screening * Has at least 2 positive sputum cultures of Mycobacterium avium complex (MAC) (sputum cultures to be taken at least 4 weeks apart): one obtained within 12 months prior to screening, which was documented while being treated for Mycobacterium avium complex lung disease (MAC-LD) for a total of at least 6 months; and one at screening (by central microbiology laboratory) * Received at least 6 months of consecutive MAC-LD treatment (at least 2 antibiotics for MAC, including a macrolide), that is either ongoing or has stopped within 12 months prior to screening * No presence of cognitive dysfunction that would impact the completion of the patient reported outcome (PRO) assessments Exclusion Criteria: * Had previous exposure to bedaquiline (BDQ) * Has active Tuberculosis (TB) disease * Has cystic fibrosis, medically unstable respiratory disease (for example, chronic obstructive pulmonary disease, bronchiectasis, asthma) * Has one or more cavities \>=2 centimeter (cm) in diameter on a chest CT scan taken within 6 months prior to screening or at screening * Treatment already includes an injectable/inhaled aminoglycoside within 3 months prior to screening or the investigator deems the participant to be a candidate for an injectable/inhaled aminoglycoside during screening period or at Day 1

Locations (55)

Fukuoka University Chikushi Hospital

Chikushino-shi, Japan

St. Luke's International Hospital

Chūōku, Japan

Fukui Prefectural Hospital

Fukui-shi, Japan

National Hospital Organization Ibarakihigashi

Funaishikawa, Japan

Gifu Prefectural General Medical Center

Gifu, Japan

Hamamatsu Rosai Hospital

Outcomes

Primary Outcomes

Number of Participants With Sputum Culture Conversion in Mycobacteria Growth Indicator Tube (MGIT) at Week 24

Number of participants with sputum culture conversion in MGIT at Week 24 was reported. Sputum culture conversion was defined as 3 consecutive negative sputum cultures taken at least 25 days apart.

Time frame: At Week 24

Secondary Outcomes

Number of Participants With Sputum Culture Conversion in 7H11 Agar Media at Week 24

Number of participants with sputum culture conversion in 7H11 agar media at Week 24 was reported. Sputum culture conversion was defined as 3 consecutive negative sputum cultures taken at least 25 days apart based on actual collection dates.

Time frame: At week 24

Change From Baseline in Patient Reported Health Status on Total Score of St. George's Respiratory Questionnaire (SGRQ) at Week 24

SGRQ is a 50-item questionnaire with 76 weighted responses. It provides a total score and three component scores: Symptoms (distress caused by respiratory symptoms), Activity (physical activities that cause or are limited by breathlessness), and Impacts (social and psychological effects of the disease). The composite total score is derived from the 3 domain scores. Each domain score and the total score has a range of 0 to 100, with 0 indicating the best possible quality of life. An increase in score indicates worsening health.

Time frame: Baseline (Day 1), Week 24

Percentage of Participants With Sputum Culture Conversion in MGIT at Week 48

Time frame: At Week 48

Percentage of Participants With Sputum Culture Conversion in 7H10 or 7H11 Agar Media at Week 48

Time frame: At Week 48

Percentage of Participants With Sputum Culture Negativity in MGIT

Time frame: From Week 2 to Week 60

Percentage of Participants With Sputum Culture Negativity in 7H10 or 7H11 Agar Media

Time frame: From Week 2 to Week 60

Time to Sputum Culture Conversion in MGIT up to Week 48

Time frame: From baseline (Day 1) up to Week 48

Time to Positivity in MGIT up to Week 48

Time frame: From baseline (Day 1) up to Week 48

Change From Baseline in Patient-Reported Health Status on Total Score of SGRQ at Weeks 48 and 60

Time frame: Baseline (Day 1), Week 48 and Week 60

Change From Baseline in Lung Function Parameters at Week 24

Change from baseline in lung function parameters (Forced expiratory volume in one second, Forced vital capacity, Inspiratory capacity, Functional residual capacity, Total lung capacity) at Week 24 was reported.

Time frame: Baseline (Day 1), Week 24

Change From Baseline in Lung Function Parameters at Weeks 48 and 60

Time frame: Baseline (Day 1), Week 48 and Week 60

Percentage of Participants Who Underwent a Change in Their Mycobacterium Avium Complex-lung Disease (MAC-LD) Treatment Regimen by Week 24

Percentage of participants who underwent a change in their Mycobacterium avium Complex-lung disease (MAC-LD) treatment regimen by Week 24 was reported.

Time frame: Baseline (Day 1) up to Week 24

Percentage of Participants Who Underwent a Change in Their Mycobacterium Avium Complex-lung Disease (MAC-LD) Treatment Regimen by Week 48 and Week 60

Time frame: Baseline (Day 1), Week 48 and Week 60

Percentage of Participants With Sputum Culture Conversion in MGIT at Week 60

Time frame: At Week 60

Percentage of Participants With Sputum Culture Conversion in 7H10 or 7H11 Agar Media at Week 60

Time frame: At Week 60

Number of Participants With Treatment-emergent Adverse Events (TEAEs)