This is a Phase 2 study with primary objective of looking whether YPT-01 phage therapy reduces sputum bacterial load in cystic fibrosis subjects with Pseudomonas aeruginosa. In addition, study evaluates the safety profile of phage therapy in this patient population.
This is a prospective, randomized, placebo-controlled, double-blinded, single-site study of Yale Phage Therapy (YPT) 01 in cystic fibrosis subjects with chronic Pseudomonas aeruginosa airway infections. The study has 2 parallel arms of phage therapy and placebo, with all study materials GMP-manufactured. The purpose of this study is to demonstrate efficacy and safety of inhaled (nebulized) phage therapy YPT-01. Clinically stable subjects who have confirmed diagnosis of CF with PsA in sputum cultures on at least two occasions within past year, and in sputum at screening visit, will be recruited into this study. An open-label extension is available for subjects in the placebo group to receive YPT-01 following completion of blinded portion of the study. Nov 2022 study ended enrollment after 8 subjects. The Double-Blind Randomized portion of the study was closed and the Open-Label Extension was opened.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
8
Participants will be randomized to receive the standard dose of phage therapy YPT-01.
Participants will be randomized to receive the placebo.
Yale New Haven Hospital
New Haven, Connecticut, United States
Change in Sputum Bacterial Culture
Change in sputum bacterial culture titers of Pseudomonas as measured by colony forming units/mL at day 14
Time frame: Day 14
Change in Lung Function
Change in lung function \[percent predicted forced expiratory volume in 1 second (FEV1pp)\] in subjects randomized to phage therapy and placebo from screening to day 14, 21, 28, and 56
Time frame: Screening, day 14, day 21, day 28, and day 56
Difference in the Rate of Pulmonary Exacerbations
Compare the rates of pulmonary exacerbations between subjects randomized to phage therapy versus placebo during the first 56 days of the study
Time frame: Baseline, day 56
Difference in the Rate of Hospitalization
Compare the rates of hospitalizations between subjects randomized to phage therapy versus placebo during the first 56 days of the study
Time frame: Baseline, day 56
Difference in the Rate of Acute Antibiotic Use
Compare the rates of acute antibiotic use between subjects randomized to phage therapy versus placebo during the first 56 days of the study
Time frame: Baseline, day 56
Patient's Quality of Life
Changes in subject-reported quality of life, using the Cystic Fibrosis Questionnaire Revised (CFQ-R) Teen/Adult, from baseline to Day 56. This survey consists of 50 questions that relate to a subjects clinical condition and mental health. Scores for each domain range from 0 to 100, with higher scores indicating a higher patient-reported quality of life with regard to the domain being evaluated.
Time frame: Baseline, day 56
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