Study to Evaluate the Efficacy of Delpazolid as Add-on Therapy in Refractory Mycobacterium Abscessus Complex
Study Details
Study Description
Brief Summary
The purpose of this study is to evaluate the efficacy and safety of delpazolid add-on therapy in Patients with Refractory Mycobacterium abscessus Complex Pulmonary disease
Condition or Disease | Intervention/Treatment | Phase |
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Phase 2 |
Detailed Description
Delpazolid, which demonstrates effects similar to other oxazolidinone-class drugs and has confirmed good safety, aims to evaluate its efficacy in MABC-PD patients who are unresponsive to guideline-based treatments
Study Design
Arms and Interventions
Arm | Intervention/Treatment |
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Experimental: delpazolid In addition to background therapy for MABC, patients will be given orally three tablets (400 mg/tablet) of LCB01-0371 for 12 weeks. |
Drug: Delpazolid
Three tablets (400 mg/tablet) of delpazolid will be orally administered once daily for 12 weeks.
After 3 months (12 weeks) of administration, if the investigator determines that there are clinical benefits, an additional extended treatment of up to 9 months (40 weeks) may be given, amounting to 1 year of treatment in total.
Other Names:
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Outcome Measures
Primary Outcome Measures
- Change from baseline in the semi-quantitative scale (SQS) [12 weeks]
The SQS using liquid media and solid media will be scored a point of 1-7, with lower scores representing a reducing bacterial load.
Secondary Outcome Measures
- Change from baseline in SQS [4 weeks and 8 weeks]
The SQS using liquid media and solid media will be scored a point of 1-7, with lower scores representing a reducing bacterial load.
- Sputum culture conversion rate [12 weeks]
neg culture x3( sputum conversion)
- Time to culture conversion [12 weeks]
from the date of assignment to the first negative result.
- Time to positivity in the liquid culture automated system (MGIT) [12 weeks]
time to detection of positive in MGIT system
- Negative sputum culture rate at each time point after baseline [12 weeks]
negative culture results in MGIT and solid media
- Change from baseline in the inflammatory marker [12 weeks]
erythrocyte sedimentation rate [ESR]) at each time point
- Change from baseline in the CT score [12 weeks]
The CT scan score will be transformed onto a scale of 0-42, with lower scores representing improved lung presentations.
- Quality of Life Questionnaire-Bronchiectasis [12 weeks]
The QOL-B respiratory symptoms score was transformed onto a scale of 0-100, with higher scores representing a better quality of life.
- Six-minute walk test [12 weeks]
Six-minute walk test
Eligibility Criteria
Criteria
Inclusion Criteria:
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Pre-screening: Adults aged 19 years or above
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Pre-screening: Patients diagnosed with MABC (including subspecies abscessus, bolletii, and massiliense) pulmonary disease in radiologic and microbiologic evaluations
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LCB01-0371 MIC ≤ 4 μg/mL for MABC
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Patients who continue to show positivity for MABC even after treatments based on the guidelines of ATS/ERS/ESCMID/IDSA for at least 6 months prior to screening, and who meet all of the following criteria:
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Patients who have been confirmed positive at least once in the last sputum or bronchoscopy sample culture performed prior to screening
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Patients who have not achieved culture conversion (at least 3 consecutive negative mycobacteria cultures in the sputum or bronchoscopy sample collected at an interval of at least 4 weeks) within 6 months prior to screening
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Patients who can voluntarily expectorate sputum at screening
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Patients with a life expectancy of 12 weeks or more
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Patients with adequate organ function who meet the following criteria:
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Hemoglobin > 9.0 g/dL (without transfusion within 2 weeks prior to measurement)
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Absolute neutrophil count ≥ 1,500/µL (without administration of G-CSF within 2 weeks prior to measurement)
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Platelet ≥ 100,000/µL
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Total bilirubin ≤ 1.5 × upper limit of normal (ULN)
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Alanine aminotransferase/aspartate aminotransferase (ALT/AST) ≤ 2.5 × ULN
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Serum creatinine ≤ 1.5 × ULN or creatinine clearance >30 mL/min (calculated with the Cockcroft-Gault formula)
- Patients who voluntarily provided a written consent to participate in the clinical study
Exclusion Criteria:
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Patients who cannot swallow the study drug tablet due to dysphagia, nasogastric tube insertion, etc.
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Patients diagnosed with cystic fibrosis
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Patients who have received a lung transplant
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Patients with disseminated or extrapulmonary nontuberculous mycobacteria
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Patients with known active pulmonary tuberculosis
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Patients with atypical mycobacterial infections other than MABC
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Patients with an active pulmonary malignancy within 1 year prior to screening or Patients with other malignancies that require chemotherapy or radiotherapy
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Patients who were administered linezolid for more than 2 weeks to treat MABC
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Patients with known HIV positivity or a suspected infection thereof or Patients with a known active hepatitis B or C infection
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Patients who currently have a clinically significant cardiovascular disease
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Patients with severe cardiac failure (New York Heart Association [NYHA] class III/IV) that occurred within 24 weeks prior to screening
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Patients with pulmonary embolism or deep venous thrombosis that occurred within 24 weeks prior to screening
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Patients whose multidrug therapy for treatment of MABC was changed within 4 weeks prior to screening (Discontinuation, dose adjustment, change of administration route, etc., are allowed.)
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Patients for whom the administration of contraindicated concomitant drugs that correspond to the following cannot be discontinued during the clinical study or for whom their administration is necessary
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Administration of a new antibacterial agent for the prioritized treatment of atypical mycobacteria, especially MABC, other than background therapy
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Monoamine oxidase inhibitors
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Serotonin reuptake inhibitor or serotonin 5-HT1 receptor agonists
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Meperidine or buspirone
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Drugs that lower epilepsy threshold; tramadol, etc.
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Other investigational products: If there is a history of administration within 30 days prior to screening, it falls under the exclusion criteria.
- Pregnant or breastfeeding women and women of childbearing potential who do not agree to practice appropriate contraceptive methods*:
Contacts and Locations
Locations
No locations specified.Sponsors and Collaborators
- LegoChem Biosciences, Inc
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- LCB01-0371-2004