A Phase 1b/2a, Multi-Center, Double-Blind, Randomized, Placebo-Controlled, Single and Multiple Ascending Dose Study to Evaluate the Safety and Tolerability of AP-PA02 Multi-Phage Therapeutic Candidate for Inhalation in Subjects With Cystic Fibrosis and Chronic Pulmonary Pseudomonas Aeruginosa (Pa) Infection

AP-PA02-101

Phase 1b/2a, double-blind, randomized, placebo-controlled, single and multiple ascending dose study to evaluate the safety, tolerability and phage recovery profile of AP-PA02 multi-bacteriophage therapeutic candidate administered by inhalation in subjects with cystic fibrosis and chronic pulmonary Pseudomonas aeruginosa (PA) infection.

2020-12-22

2022-12-14

2022-12-14

Completed

Early phase I

29

Key Inclusion Criteria: * ≥ 18 years old * Body mass index (BMI) of ≥ 18 kg/m2 * Documented diagnosis of CF * Evidence of chronic pulmonary Pseudomonas aeruginosa infection * Willing to undergo sputum induction procedures at designated study visits, and willing to provide expectorated sputum samples at all other timepoints (for subjects who are able to expectorate) * For SAD: FEV1 ≥ 60% of predicted normal \[per Global Lung Function Initiative (GLI) standards\] at Screening * For MAD: FEV1 ≥ 40% of predicted normal \[per Global Lung Function Initiative (GLI) standards\] at Screening * Adequate renal function Key Exclusion Criteria: * Recent significant weight loss * Abnormal vital signs at Screening * History of prolonged QT syndrome * Use of supplemental oxygen during the day at rest * Abnormal liver function tests greater than 3X the upper limit of normal (ULN) * Recent oral or IV antibiotics received for acute pulmonary exacerbation. Inhaled antibiotic use for chronic suppression of P. aeruginosa is acceptable. * Recent clinically significant infection requiring systemic antimicrobial therapy * Currently receiving anti-pseudomonal antibiotic treatment for acute sinusitis. * Currently receiving systemic corticosteroids * Currently receiving treatment for active infection with nontuberculous mycobacteria (NTM), Staphylococcus aureus, or Burkholderia cepacia complex lung infection * Currently receiving treatment for aspergillosis or ABPA (allergic bronchopulmonary aspergillosis) * Initiation of a CFTR potentiator/corrector therapy, such as Trikafta®, less than 90 days prior to Screening * Acquired or primary immunodeficiency syndromes * Active pulmonary malignancy (primary or metastatic) * History of lung transplantation * Recent hemoptysis * Female pregnant or breastfeeding * Heavy smoker

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2024-01-31