AeroVanc in the Treatment of Methicillin-resistant Staphylococcus Aureus Infection in Patients With Cystic Fibrosis
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|ClinicalTrials.gov Identifier: NCT03181932|
Recruitment Status : Completed
First Posted : June 9, 2017
Last Update Posted : August 9, 2021
|Condition or disease||Intervention/treatment||Phase|
|MRSA Cystic Fibrosis||Drug: Vancomycin inhalation powder Drug: Placebo inhalation powder||Phase 3|
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 (Clinical Trial)|
|Actual Enrollment :||188 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||A Phase III, Randomized, Double-blind, Placebo-controlled Study of AeroVanc for the Treatment of Persistent Methicillin-resistant Staphylococcus Aureus Lung Infection in Cystic Fibrosis Patients|
|Actual Study Start Date :||September 20, 2017|
|Actual Primary Completion Date :||July 28, 2020|
|Actual Study Completion Date :||January 12, 2021|
Experimental: Double-blind vancomycin inhalation powder
Vancomycin inhalation powder 30 mg is administered twice daily (BID) during the 24-week double-blind period (Period 1) by inhalation during three dosing cycles, each cycle being 28 days of treatment followed by 28 days of observation.
Drug: Vancomycin inhalation powder
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.
Other Name: AeroVanc
Placebo Comparator: Double-blind placebo inhalation powder
Matching placebo is administered BID during the 24-week double-blind period (Period 1) by inhalation during three dosing cycles, each cycle being 28 days of treatment followed by 28 days of observation.
Drug: Placebo inhalation powder
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.
Experimental: Open-label vancomycin inhalation powder
In the 24-week Period 2, all participants receive AeroVanc 30 mg BID by inhalation during three dosing cycles, each cycle being 28 days of treatment followed by 28 days of observation.
Drug: Vancomycin inhalation powder
In the 24-week Period 2, all participants are to be treated with open-label vancomycin inhalation powder.
Other Name: AeroVanc
- Absolute change from Baseline in forced expiratory volume in 1 second (FEV1) percent predicted [ Time Frame: Week 4, 12 and 20 ]The mean absolute change from baseline in FEV1 percent predicted; analyzed sequentially at Week 4 (end of Cycle 1), Week 12 (end of Cycle 2), and at Week 20 (end of Cycle 3).
- Frequency of pulmonary exacerbations [ Time Frame: Week 20 ]The number of pulmonary exacerbations during Period 1 adjusted for the length of follow-up
- Time to first pulmonary exacerbation [ Time Frame: Week 4, 12 and 20 ]Time to first pulmonary exacerbation requiring use of another antibiotic medication (oral, IV, and/or inhaled)
- Cystic Fibrosis Questionnaire-Revised (CFQ-R) scores [ Time Frame: Week 4, 12, and 20 ]Change from Baseline in the CFQ-R respiratory domain. Scores range between 0 and 100, where higher scores indicate a better outcome.
- Cystic Fibrosis Respiratory Symptom Diary-Chronic Respiratory Symptom Score (CFRSD-CRISS) scores [ Time Frame: Week 4, 12 and 20 ]Change from Baseline in the CFRSD-CRISS scores. Scores range between 0 and 100, where higher scores indicate a worse outcome.
- Relative change in FEV1 percent predicted [ Time Frame: Week 4, 12 and 20 ]The mean relative change from Baseline in FEV1 percent predicted
- Number of successful response cycles [ Time Frame: Week 4, 12 and 20 ]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.
- Area under the FEV1-time profile [ Time Frame: Week 20 ]The mean treatment difference in FEV1 across all post-baseline visits
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03181932
|Principal Investigator:||Patrick Flume, MD||Medical University of South Carolina|