Trial reportThorax2013
Phase II studies of nebulised Arikace in CF patients with Pseudomonas aeruginosa infection.
Trial report in Thorax, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 87 papers, 6 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Phase 2a Multidose Safety and Tolerability Study of Dose Escalation of Liposomal Amikacin for Inhalation (ARIKACE™) In Cystic Fibrosis Patients With Chronic Infections Due To Pseudomonas Aeruginosa.
Multidose Safety and Tolerability Study of Dose Escalation of Liposomal Amikacin for Inhalation (ARIKAYCE™) in Cystic Fibrosis Patients With Chronic Infections Due to Pseudomonas Aeruginosa
Who cites it
87 citing papers in PubMed, 6 syntheses or guidelines pooled it, 219 citations in OpenAlex.
- Inhaled anti-pseudomonal antibiotics for long-term therapy in cystic fibrosis.The Cochrane database of systematic reviews · 2022Pooled it
- Inhaled antibiotics for pulmonary exacerbations in cystic fibrosis.The Cochrane database of systematic reviews · 2022Pooled it
- Pooled it
- Inhaled antibiotics for pulmonary exacerbations in cystic fibrosis.The Cochrane database of systematic reviews · 2018Pooled it
- Antibiotic strategies for eradicating Pseudomonas aeruginosa in people with cystic fibrosis.The Cochrane database of systematic reviews · 2017Pooled it
- Guideline
- Long-term amikacin liposome inhalation suspension in cystic fibrosis patients with chronic P. aeruginosa infection.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2021Trial
- Amikacin liposome inhalation suspension for chronic Pseudomonas aeruginosa infection in cystic fibrosis.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2020Trial
- Randomized Trial of Liposomal Amikacin for Inhalation in Nontuberculous Mycobacterial Lung Disease.American journal of respiratory and critical care medicine · 2017Trial
- Nanomaterial Strategies for Pulmonary Delivery of Immunotherapeutics in Lung Cancer Treatment.Advanced healthcare materials · 2026Review
- Systematic Review of Antimicrobial Therapies and Clinical Outcomes inLife (Basel, Switzerland) · 2026Review
- Liposomal antimicrobials in the fight against bacterial and fungal pathogens: Clinical successes and development challenges.International journal of pharmaceutics: X · 2026Review
- Maximising opportunity for therapeutic success: sequential participation in cystic fibrosis nucleic acid-based therapy trials.The Lancet. Respiratory medicine · 2025Review
- Comparative efficacy and safety of inhaled antibiotics in managing chronicJournal of thoracic disease · 2025Article
- Nanotechnology-Based Therapeutics for Airway Inflammatory Diseases.Clinical reviews in allergy & immunology · 2025Review
- Pulmonary Delivery of Antibiotics to the Lungs: Current State and Future Prospects.Pharmaceutics · 2025Review
- Liposome-Based Nanoparticle Delivery Systems for Lung Diseases: Opportunities and Challenges.International journal of nanomedicine · 2025Review
- Research Progress on Liposome Pulmonary Delivery ofJournal of aerosol medicine and pulmonary drug delivery · 2024Review
- Synthesis and study of antibiofilm and antivirulence properties of flavonol analogues generated by palladium catalyzed ligand free Suzuki-Miyaura coupling againstRSC advances · 2024Article
- Pulmonary inhalation for disease treatment: Basic research and clinical translations.Materials today. Bio · 2024Review
27 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
17 authors at 13 institutions in 7 countries.
Funding
Abstract
rationaleArikace is a liposomal amikacin preparation for aerosol delivery with potent Pseudomonas aeruginosa killing and prolonged lung deposition.
objectivesTo examine the safety and efficacy of 28 days of once-daily Arikace in cystic fibrosis (CF) patients chronically infected with P aeruginosa.
methods105 subjects were evaluated in double-blind, placebo-controlled studies. Subjects were randomised to once-daily Arikace (70, 140, 280 and 560 mg; n=7, 5, 21 and 36 subjects) or placebo (n=36) for 28 days. Primary outcomes included safety and tolerability. Secondary outcomes included lung function (forced expiratory volume at one second (FEV1)), P aeruginosa density in sputum, and the Cystic Fibrosis Quality of Life Questionnaire-Revised (CFQ-R).
resultsThe adverse event profile was similar among Arikace and placebo subjects. The relative change in FEV1 was higher in the 560 mg dose group at day 28 (p=0.033) and at day 56 (28 days post-treatment, 0.093L±0.203 vs -0.032L±0.119; p=0.003) versus placebo. Sputum P aeruginosa density decreased >1 log in the 560 mg group versus placebo (days 14, 28 and 35; p=0.021). The Respiratory Domain of the CFQ-R increased by the Minimal Clinically Important Difference (MCID) in 67% of Arikace subjects (560 mg) versus 36% of placebo (p=0.006), and correlated with FEV1 improvements at days 14, 28 and 42 (p<0.05). An open-label extension (560 mg Arikace) for 28 days followed by 56 days off over six cycles confirmed durable improvements in lung function and sputum P aeruginosa density (n=49).
conclusionsOnce-daily Arikace demonstrated acute tolerability, safety, biologic activity and efficacy in patients with CF with P aeruginosa infection.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.