NCT01315691

Study to Evaluate Arikace™ in CF Patients With Chronic Infection Due to Pseudomonas Aeruginosa

Withdrawn Phase 3 Last updated 31 July 2018

What this trial tests

Phase 3 trial testing Liposomal amikacin for inhalation in Cystic Fibrosis. Withdrawn.

Quick facts

Lead sponsor	Insmed Incorporated
Phase	Phase 3
Status	Withdrawn
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	quadruple
Primary purpose	treatment

Drugs / interventions tested

• Liposomal amikacin for inhalation — full drug profile →
• Placebo for liposomal amikacin for inhalation — full drug profile →

Conditions studied

• Cystic Fibrosis — all drugs for Cystic Fibrosis →

Sponsor

Insmed Incorporated — full company profile →

Who can join

6 and older, any sex, with Cystic Fibrosis. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

A major factor in the respiratory health of Cystic Fibrosis (CF) subjects is the prevalence of chronic Pseudomonas aeruginosa infections. The Pseudomonas aeruginosa infection rate in CF patients increases with age and by age 18 years approximately 85% of CF patients in the US are infected. Liposomal amikacin for inhalation (Arikace™) was developed as a possible treatment for chronic infection due to Pseudomonas aeruginosa in CF patients.

The purpose of this double-blind, placebo controlled study is to determine whether Arikace™ is effective in treating chronic lung infections caused by Pseudomonas aeruginosa in Cystic Fibrosis subjects. The study will enroll approximately 300 subjects in clinics in the US, Canada, Europe, Australia and New Zealand. Subjects will be randomized to 590 mg Arikace™ or placebo and will receive treatment for 28 days followed by a 56 day safety follow-up period. The subjects will be required to visit the clinic 8 times (including the Screening visit) over a period of approximately 3 months. No overnight stays at the clinic will be required. At the completion of the TR02-109 protocol, subjects who have consented and meet study safety criteria may enroll in the long-term, open-label, multi-cycle extension study of 590 mg of Arikace™ (under a separate protocol TR02-110).

Publications & conference data

7 peer-reviewed publications reference this trial (live from Europe PMC):

1. Nanomaterial-based therapeutics for antibiotic-resistant bacterial infections.
   Makabenta JMV, Nabawy A, Li CH, Schmidt-Malan S, et al · · 2021 · cited 751× · PMID 32814862 · DOI 10.1038/s41579-020-0420-1
2. Nano-Strategies to Fight Multidrug Resistant Bacteria-"A Battle of the Titans".
   Baptista PV, McCusker MP, Carvalho A, Ferreira DA, et al · · 2018 · cited 389× · PMID 30013539 · DOI 10.3389/fmicb.2018.01441
3. Therapeutic Strategies against Biofilm Infections.
   Mishra S, Gupta A, Upadhye V, Singh SC, et al · · 2023 · cited 67× · PMID 36676121 · DOI 10.3390/life13010172
4. Aerosol pulmonary immune engineering.
   Sudduth ER, Trautmann-Rodriguez M, Gill N, Bomb K, et al · · 2023 · cited 18× · PMID 37100206 · DOI 10.1016/j.addr.2023.114831
5. Inhaled anti-pseudomonal antibiotics for long-term therapy in cystic fibrosis.
   Smith S, Rowbotham NJ. · · 2022 · cited 18× · PMID 36373968 · DOI 10.1002/14651858.cd001021.pub4
6. Formulation and clinical translation of inhalable nanomedicines for the treatment and prevention of pulmonary infectious diseases.
   Ahmed R, Tewes F, Aucamp M, Dube A. · · 2025 · cited 6× · PMID 40301249 · DOI 10.1007/s13346-025-01861-5
7. The Multifaceted Mechanistic Actions of Antimicrobial Nanoformulations: Overcoming Resistance and Enhancing Efficacy.
   Gudepu R, Kyatham R, Ediga ND, Penta G, et al · · 2026 · PMID 42076075 · DOI 10.3390/pharmaceutics18040423