Oral Omadacycline Vs. Placebo in Adults with NTM Pulmonary Disease Caused by Mycobacterium Abscessus Complex (MABc)
- Conditions
- Mycobacterium Infections, NontuberculousNontuberculous Mycobacterial Pulmonary InfectionMycobacterium Abscessus InfectionNontuberculous Mycobacterial Lung Disease
- Interventions
- Drug: Placebo
- Registration Number
- NCT04922554
- Lead Sponsor
- Paratek Pharmaceuticals Inc
- Brief Summary
The purpose of this study is to evaluate the efficacy, safety and tolerability of oral omadacycline as compared to placebo in the treatment of adults with Nontuberculous Mycobacterial (NTM) pulmonary disease caused by Mycobacterium abscessus complex (MABc)
- Detailed Description
The total duration of subject participation in the study is approximately 5 months which includes a total duration of study treatment for approximately 3 months (84 days). Eligible participants will be randomized 1.5:1 to receive 3 months of treatment with either omadacycline or placebo (monotherapy). The study will use a double-dummy design in order to maintain the study blinding.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 66
- Has a diagnosis of Nontuberculous Mycobacterial pulmonary disease caused by MABc
- Has at least 2 of the following NTM-infection symptoms present at Screening and Baseline: chronic cough, coughing up blood (hemoptysis), wheezing, chest pain, frequent throat clearing, phlegm or sputum production, shortness of breath, fatigue, fever, night sweats, poor appetite, and/or weight loss.
- At least 1 positive pulmonary (sputum) culture for MABc in the 6 months prior to Screening and 1 positive culture at Screening
- Radiographic evidence of MABc infection via computed tomography (CT) scan of the chest within 3 months prior to Screening
- In the opinion of the investigator, guideline-directed antibiotic therapy for treatment of MABc will not be required within the next 3 months, and a delay, in order for the subject to participate in a placebo-controlled clinical trial, is considered reasonable and clinically acceptable
- Additional inclusion criteria as per protocol
Key
- Has received antibiotic treatment within 6 months prior to Screening for MABc or MAC
- Has received systemic or inhaled antibiotic therapy (other than chronic macrolide therapy) within 4 weeks prior to Screening
- Has any of the following medical conditions:
- Active pulmonary malignancy, or any type of malignancy requiring chemotherapy or radiation within 1 year prior to Screening
- Active allergic bronchopulmonary mycosis, or any other condition requiring chronic treatment with systemic corticosteroids within 90 days prior to Screening
- Radiologic evidence of cavitary disease
- Known active pulmonary tuberculosis
- Cystic fibrosis
- History of lung transplantation
- Another advanced lung disease with a known percent predicted forced expiratory volume in 1 second < 30%.
- Disseminated or extra-pulmonary NTM disease
- Has been previously treated with omadacycline
- Has a history of hypersensitivity or allergic reaction to tetracyclines
- Additional exclusion criteria as per protocol
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo PO Placebo Placebo tablets resembling omadacycline (x 2) administered once daily, q24h Omadacycline 300 mg PO Omadacycline Oral Tablet omadacycline 150 mg tablets (x 2) administered orally, once daily, q24h
- Primary Outcome Measures
Name Time Method Clinical Response on NTM Symptom Assessment Scale at Day 84 Day 1 to Day 84/EOT Improvement in severity of at least 50% of symptoms present at baseline
Reported adverse events (AEs) Day 1 to Day 84/EOT To assess reported adverse events
Clinically significant (CS), outside normal range laboratory tests Day 1 to Day 84/EOT To assess the incidents of CS abnormal hematology, biochemistry, coagulation and urinalysis assessments following 84 days of IP administration
Changes from baseline in laboratory tests Day 1 to Day 84/EOT To assess the incidents of abnormal hematology, biochemistry, coagulation and urinalysis assessments following 84 days of IP administration
Changes from baseline in vital signs Day 1 to Day 84/EOT To assess the incidents of abnormal heart rate and blood pressure assessments following 84 days of IP administration
Clinically significant (CS) vital signs Day 1 to Day 84/EOT To assess the incidents of CS heart rate and blood pressure following 84 days of IP administration
Changes from baseline in electrocardiogram (ECG) Day 1 to Day 84/EOT To assess the incidents of abnormal heart rate, cardiac rhythm, PR interval, RR interval, QRS interval, QT interval and QTc interval assessments following 84 days of IP administration
Clinically significant (CS) electrocardiogram (ECG) findings Day 1 to Day 84/EOT To assess the incidents of CS and QTc interval assessments following 84 days of IP administration
- Secondary Outcome Measures
Name Time Method Change from baseline in the total score of the Quality of Life - Bronchiectasis (QOL-B) questionnaire Day 1 to Day 84/EOT Change from baseline in global score and individual domain scores of the St. George Respiratory Questionnaire (SGRQ) Day 1 to Day 84/EOT Change from baseline in Patient-Reported Outcomes Measurement Information System Short Form v1.0 - Fatigue 7a Daily (PROMIS-7a) Day 1 to Day 84/EOT Change from baseline in Patient Clinical Impression of Severity (PGI-S) Day 1 to Day 84/EOT Change from baseline in Patient Clinical Impression of Change (PGI-C) Day 1 to Day 84/EOT Change from baseline in Clinical Global Impression - Severity of Illness (CGI-S) Day 1 to Day 84/EOT Time to first negative sputum culture Day 1 to Day 84/EOT Change from baseline in Clinical Global Impression - Improvement (CGI-I) Day 1 to Day 84/EOT Patients reporting no new symptoms with a severity worse than mild on the NTM Symptom Assessment Questionnaire Day 1 to Day 84/EOT Decrease in quantitative sputum culture at Day 84 Day 1 to Day 84/EOT Time to growth in liquid medium only Day 1 to Day 84/EOT
Trial Locations
- Locations (17)
Stanford University
🇺🇸Stanford, California, United States
Georgetown University Hospital
🇺🇸Washington, District of Columbia, United States
St. Francis Medical Institute
🇺🇸Clearwater, Florida, United States
University of Miami
🇺🇸Miami, Florida, United States
University of South Florida
🇺🇸Tampa, Florida, United States
Infectious Disease Consultants of the Treasure Coast
🇺🇸Vero Beach, Florida, United States
Emory University School of Medicine
🇺🇸Atlanta, Georgia, United States
Louisiana State University Medical Center Health Sciences Center-New Orleans Section of Pulmonary/Critical Care & Allergy/Immunology
🇺🇸New Orleans, Louisiana, United States
Johns Hopkins University
🇺🇸Baltimore, Maryland, United States
Dartmouth-Hitchcock Medical Center
🇺🇸Lebanon, New Hampshire, United States
Einstein/Montefiore Medical Center
🇺🇸Bronx, New York, United States
Northwell Health
🇺🇸New Hyde, New York, United States
University of North Carolina at Chapel Hill
🇺🇸Chapel Hill, North Carolina, United States
Oregon Health & Science University
🇺🇸Portland, Oregon, United States
Medical University of South Carolina (MUSC)
🇺🇸Charleston, South Carolina, United States
The University of Texas Health Science Center at Tyler
🇺🇸Tyler, Texas, United States
University of Wisconsin Hospitals and Clinics
🇺🇸Madison, Wisconsin, United States