Oral Omadacycline vs. Placebo in Adults With NTM Pulmonary Disease Caused by Mycobacterium Abscessus Complex (MABc)

Paratek Pharmaceuticals Inc66 enrolled

Overview

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)

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Mycobacterium Infections, Nontuberculous Mycobacterium Abscessus Infection Nontuberculous Mycobacterial Lung Disease Nontuberculous Mycobacterial Pulmonary Infection

Interventions

Omadacycline Oral TabletDRUG

omadacycline 300 mg orally, once daily (150 mg tablets x 2)

PlaceboDRUG

placebo tablets resembling omadacycline orally, once daily (x 2 tablets)

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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

Locations (17)

Stanford University

Stanford, California, United States

Georgetown University Hospital

Washington D.C., District of Columbia, United States

Outcomes

Primary Outcomes

Percentage of Participants With Clinical Response on NTM Symptom Assessment Scale at Day 84

A clinical responder is defined as a participant who shows improvement in at least 50% of baseline symptoms on the NTM Symptom Assessment Questionnaire at the Day 84 Visit. This self-administered tool assesses 12 common NTM symptoms, each rated on a 4-point scale (absent, mild, moderate, severe), reflecting the participant's overall impression over the past week. The questionnaire is completed solely by the participant, without any interpretation from clinicians or site staff.

Time frame: Day 1 to Day 84

Percentage of Participants With Clinical Response on NTM Symptom Assessment Scale at Day 84 With no Deterioration in Severity of Symptoms That Were Present at Baseline.

A clinical responder is defined as a participant who shows improvement in at least 50% of baseline symptoms with no deterioration of symptoms present at baseline on the NTM Symptom Assessment Questionnaire at the Day 84 Visit. This self-administered tool assesses 12 common NTM symptoms, each rated on a 4-point scale (absent, mild, moderate, severe), reflecting the participant's overall impression over the past week. The questionnaire is completed solely by the participant, without any interpretation from clinicians or site staff.

Time frame: Day 1 to Day 84

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

A TEAE is an adverse event that occurred on or after first dose of test article and those existing AEs that worsened on or after first dose of test article. An SAE is an adverse event that results in death, is life-threatening, requires hospitalization or extends an existing one, causes significant or lasting disability/incapacity, or leads to a congenital anomaly or birth defect. Additionally, events that may not meet these criteria but are medically significant can also be classified as SAEs.

Time frame: From screening period (up to 8 weeks prior to randomization) through Day 114 (at any study timepoint)

Change From Baseline in Laboratory Test Parameters - Hepatic and Enzymatic Biomarkers

Data from ClinicalTrials.gov

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Secondary Outcomes

Change From Baseline in the Total Score of the Quality of Life - Bronchiectasis (QOL-B) Questionnaire - Emotional Functioning Domain

The QOL-B is a self-administered, patient-reported outcome measure assessing symptoms, functioning and health-related quality of life for participants with non-cystic fibrosis bronchiectasis using a series of 37 questions. The QOL-B includes domains for physical functioning, role functioning, vitality, emotional functioning, social functioning, treatment burden, health perceptions and respiratory symptoms. For each domain, scores will be standardized on a 0 to 100 scale and higher scores represent better outcomes.