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)
Brief Title
Oral Omadacycline Vs. Placebo in Adults with 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.
Categories
Completion Date
Completion Date Type
Actual
Conditions
Mycobacterium Infections, Nontuberculous
Mycobacterium Abscessus Infection
Nontuberculous Mycobacterial Lung Disease
Nontuberculous Mycobacterial Pulmonary Infection
Eligibility Criteria
Key Inclusion Criteria:
* 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 Exclusion Criteria:
* 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
* 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 Exclusion Criteria:
* 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
Inclusion Criteria
Inclusion Criteria:
* 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
* 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
Gender
All
Gender Based
false
Keywords
Nontuberculous Mycobacteria (NTM)
Mycobacterium abscessus complex (MABc)
NTM pulmonary disease
NTM lung disease
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Actual
Last Update Submit Date
Minimum Age
18 Years
NCT Id
NCT04922554
Org Class
Industry
Org Full Name
Paratek Pharmaceuticals Inc
Org Study Id
PTK0796-NTM-20203
Overall Status
Completed
Phases
Phase 2
Primary Completion Date
Primary Completion Date Type
Actual
Official Title
A Ph. 2, Double-Blind, Randomized, Parallel-Group, Placebo-Controlled, Multi-Center Study to Evaluate the Efficacy, Safety, & Tolerability of Oral Omadacycline in Adults with NTM Pulmonary Disease Caused by Mycobacterium Abscessus Complex
Primary Outcomes
Outcome Description
Improvement in severity of at least 50% of symptoms present at baseline
Outcome Measure
Clinical Response on NTM Symptom Assessment Scale at Day 84
Outcome Time Frame
Day 1 to Day 84/EOT
Outcome Description
To assess reported adverse events
Outcome Measure
Reported adverse events (AEs)
Outcome Time Frame
Day 1 to Day 84/EOT
Outcome Description
To assess the incidents of abnormal hematology, biochemistry, coagulation and urinalysis assessments following 84 days of IP administration
Outcome Measure
Changes from baseline in laboratory tests
Outcome Time Frame
Day 1 to Day 84/EOT
Outcome Description
To assess the incidents of CS abnormal hematology, biochemistry, coagulation and urinalysis assessments following 84 days of IP administration
Outcome Measure
Clinically significant (CS), outside normal range laboratory tests
Outcome Time Frame
Day 1 to Day 84/EOT
Outcome Description
To assess the incidents of abnormal heart rate and blood pressure assessments following 84 days of IP administration
Outcome Measure
Changes from baseline in vital signs
Outcome Time Frame
Day 1 to Day 84/EOT
Outcome Description
To assess the incidents of CS heart rate and blood pressure following 84 days of IP administration
Outcome Measure
Clinically significant (CS) vital signs
Outcome Time Frame
Day 1 to Day 84/EOT
Outcome Description
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
Outcome Measure
Changes from baseline in electrocardiogram (ECG)
Outcome Time Frame
Day 1 to Day 84/EOT
Outcome Description
To assess the incidents of CS and QTc interval assessments following 84 days of IP administration
Outcome Measure
Clinically significant (CS) electrocardiogram (ECG) findings
Outcome Time Frame
Day 1 to Day 84/EOT
Secondary Outcomes
Outcome Time Frame
Day 1 to Day 84/EOT
Outcome Measure
Change from baseline in the total score of the Quality of Life - Bronchiectasis (QOL-B) questionnaire
Outcome Time Frame
Day 1 to Day 84/EOT
Outcome Measure
Change from baseline in global score and individual domain scores of the St. George Respiratory Questionnaire (SGRQ)
Outcome Time Frame
Day 1 to Day 84/EOT
Outcome Measure
Change from baseline in Patient-Reported Outcomes Measurement Information System Short Form v1.0 - Fatigue 7a Daily (PROMIS-7a)
Outcome Time Frame
Day 1 to Day 84/EOT
Outcome Measure
Change from baseline in Patient Clinical Impression of Severity (PGI-S)
Outcome Time Frame
Day 1 to Day 84/EOT
Outcome Measure
Change from baseline in Patient Clinical Impression of Change (PGI-C)
Outcome Time Frame
Day 1 to Day 84/EOT
Outcome Measure
Change from baseline in Clinical Global Impression - Severity of Illness (CGI-S)
Outcome Time Frame
Day 1 to Day 84/EOT
Outcome Measure
Change from baseline in Clinical Global Impression - Improvement (CGI-I)
Outcome Time Frame
Day 1 to Day 84/EOT
Outcome Measure
Patients reporting no new symptoms with a severity worse than mild on the NTM Symptom Assessment Questionnaire
Outcome Time Frame
Day 1 to Day 84/EOT
Outcome Measure
Decrease in quantitative sputum culture at Day 84
Outcome Time Frame
Day 1 to Day 84/EOT
Outcome Measure
Time to growth in liquid medium only
Outcome Time Frame
Day 1 to Day 84/EOT
Outcome Measure
Time to first negative sputum culture
Start Date
Start Date Type
Actual
Status Verified Date
First Post Date
First Post Date Type
Actual
First Submit Date
First Submit QC Date
Std Ages
Adult
Older Adult
Maximum Age Number (converted to Years and rounded down)
999
Minimum Age Number (converted to Years and rounded down)
18
Investigators
Investigator Type
Principal Investigator
Investigator Name
Eric Meyerowitz
Investigator Email
emeyerowit@montefiore.org
Investigator Phone
631-338-4592