This is a multi-center, randomized phase III study to evaluate the clinical effectiveness of AeroVanc in persistent methicillin-resistant Staphylococcus aureus (MRSA) infection in patients with cystic fibrosis (CF).
This is a phase III, randomized, multicenter, double-blind, placebo-controlled, parallel-group study to examine the safety and efficacy of AeroVanc in the treatment of persistent MRSA lung infection in patients diagnosed with CF. After the Screening period to confirm study eligibility, participants are randomly assigned in a blinded fashion to receive either AeroVanc 30 mg twice daily (BID), or placebo BID (1:1 active to placebo) by inhalation for 24 weeks or 3 dosing cycles (Period 1). Upon completion of Period 1, participants receive open-label AeroVanc 30 mg BID for an additional 24 weeks or 3 dosing cycles (Period 2), to evaluate long-term safety of AeroVanc. A dosing cycle is defined as 28 days of treatment followed by 28 days of observation. Participants on a 28-day cyclical on/off anti-Pseudomonal antibiotic regimen enter the Screening period at a time such that the Baseline visit coincide with the end of their anti-Pseudomonas antibiotic cycle. Study drug is thereby administered during the off-cycle, and participants can then resume anti-Pseudomonal therapy during the 28-day observation period. Participants continuing alternating anti-Pseudomonal therapy can continue their treatment during the study drug administration, and observation period. The primary and secondary analyses are conducted in participants ≤21 years old. Subjects \>21 years old are analyzed separately as supportive analyses.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
188
100 participants are to be treated with double-blind vancomycin inhalation powder (75 subjects ≤21 years old, 25 subjects \>21 years old) for 24 weeks during Period 1.
100 participants are to be treated with double-blind placebo (75 subjects ≤21 years old, 25 subjects \>21 years old) for 24 weeks during Period 1.
In the 24-week Period 2, all participants are to be treated with open-label vancomycin inhalation powder.
Absolute Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) Percent Predicted
The mean absolute change from baseline in FEV1 percent predicted was analyzed sequentially at Week 4 (end of Cycle 1), Week 12 (end of Cycle 2) and at Week 20 (end of Cycle 3).
Time frame: Baseline and Week 4, 12 and 20
Frequency of Pulmonary Exacerbations
The number of pulmonary exacerbations during Period 1 adjusted for the length of follow-up.
Time frame: Week 20
Time to First Pulmonary Exacerbation
Time to first pulmonary exacerbation requiring use of another antibiotic medication (oral, IV, and/or inhaled). The Outcome Measure Data presented are the median percentiles and 95% confidence intervals from Kaplan-Meier estimates.
Time frame: Week 20
Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Scores
The CFQ-R was administered every two weeks using a hand-held e-Diary. CFQ-R scores range between 0 and 100, where higher scores indicate a better outcome. The CFQ-R measures functioning in a variety of domains, including Physical Functioning, Vitality, Health Perceptions, Respiratory Symptoms, Treatment Burden, Role Functioning, Emotional Functioning and Social Functioning. The Outcome Measure Data presented are the Respiratory Symptoms Scores.
Time frame: Baseline and Week 4, 12, and 20
Change From Baseline in Cystic Fibrosis Respiratory Symptom Diary-Chronic Respiratory Symptom Score (CFRSD-CRISS) Scores
The CFRSD-CRISS was administered every two weeks using a hand-held e-Diary. Scores range between 0 and 100, where higher scores indicate a worse outcome.
Time frame: Baseline and Week 4, 12 and 20
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Pulmonary Associates of Mobile
Mobile, Alabama, United States
Phoenix Children's Hospital
Phoenix, Arizona, United States
University of Arkansas for Medical Sciences
Little Rock, Arkansas, United States
Miller Childrens Hospital MemorialCare Health System Pediatric Pulmonology
Long Beach, California, United States
Children's Hospital Los Angeles
Los Angeles, California, United States
University of Southern California Keck Medical Center of USC
Los Angeles, California, United States
UC Davis Medical Center
Sacramento, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
National Jewish Health Adult Cystic Fibrosis Center
Denver, Colorado, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
...and 61 more locations
Relative Change in FEV1 Percent Predicted
The mean relative change from Baseline in FEV1 percent predicted
Time frame: Baseline and Week 4, 12 and 20
Number of Successful Response Cycles
The number of successful response cycles a participant achieves over Period 1. A response in a cycle is defined by at least a 5 % relative improvement in FEV1 percent predicted at the end of each the respective cycle.
Time frame: Week 20
Area Under the FEV1-time Profile
The mean treatment difference in FEV1 across all post-baseline visits
Time frame: Week 20