CompletedPhase 2

Study of Dose Escalation of Liposomal Amikacin for Inhalation (ARIKAYCE™) - Extension Phase

Belgium, Hungary, North Macedonia49 participantsStarted 2009-01-08

Plain-language summary

A major factor in the respiratory health of cystic fibrosis (CF) patients is acquisition of chronic Pseudomonas (P.) aeruginosa infections. The infection rate with P. aeruginosa increases with age and by age 18 years, 80% of patients with CF in the U.S. are infected. Liposomal amikacin for inhalation (LAI; Arikayce™) is a sterile aqueous liposomal suspension consisting of amikacin sulfate encapsulated in liposomes. This formulation of amikacin maximizes the achievable dose and delivery to the lungs of infected patients when delivered via a nebulizer. Because liposome particles are small enough to penetrate and diffuse through sputum into the bacterial biofilm, they deposit drug close to the bacterial colonies (Meers, et al., 2008) (Clancy, et al., 2013), thus improving the bioavailability of amikacin at the infection site. The clinically achievable doses of amikacin in the LAI formulation can effectively increase the half-life of the drug in the lungs, and decrease the potential for systemic toxicity. LAI offers several advantages over current therapies in treating patients with CF with chronic infection caused by P. aeruginosa.

Who can participate

Age range6 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Written informed consent obtained from the patient or designated legal guardian prior to the performance of any study related procedures.
✓. Male or female study subjects ≥ 6 years of age or older.
✓. Confirmed diagnosis of CF defined as a positive sweat chloride \> 60 milliequivalents (mEq)/liter (by pilocarpine iontophoresis) and/or a genotype with 2 identifiable mutations consistent with CF accompanied by one or more clinical features of the CF phenotype.
✓. History of chronic infection with P. aeruginosa (defined as 3 documented positive cultures in the prior 2 years of which at least one was obtained in the 3 months prior to randomization). The cultures could be obtained from the following respiratory secretions: sputum, throat swabs, nasopharyngeal swabs, or broncho-alveolar lavage fluid specimens.
✓. Study subjects must produce a screening specimen (expectorated or induced sputum, throat swabs, nasopharyngeal swabs, or broncho-alveolar lavage fluid) that is positive for growth of P. aeruginosa.
✓. FEV1 ≥ 40% of predicted at Screening.
✓. SaO2 ≥ 90% at Screening while breathing room air.
✓. Ability to comply with study medication use, study visits, and study procedures as judged by the investigator.

What they're measuring

Adverse Event Profile of 560 mg Once Daily Dose of Arikayce™ Administered for Six Cycles Over Eighteen Months.

Timeframe: 18 Months

Trial details

NCT IDNCT03905642

SponsorInsmed Incorporated

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2010-11-02

Results submitted2019-04-24

View on ClinicalTrials.gov More Cystic Fibrosis trials

Plain-language summary

Who can participate

Age range6 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Written informed consent obtained from the patient or designated legal guardian prior to the performance of any study related procedures.
✓. Male or female study subjects ≥ 6 years of age or older.
✓. Confirmed diagnosis of CF defined as a positive sweat chloride \> 60 milliequivalents (mEq)/liter (by pilocarpine iontophoresis) and/or a genotype with 2 identifiable mutations consistent with CF accompanied by one or more clinical features of the CF phenotype.
✓. History of chronic infection with P. aeruginosa (defined as 3 documented positive cultures in the prior 2 years of which at least one was obtained in the 3 months prior to randomization). The cultures could be obtained from the following respiratory secretions: sputum, throat swabs, nasopharyngeal swabs, or broncho-alveolar lavage fluid specimens.
✓. Study subjects must produce a screening specimen (expectorated or induced sputum, throat swabs, nasopharyngeal swabs, or broncho-alveolar lavage fluid) that is positive for growth of P. aeruginosa.
✓. FEV1 ≥ 40% of predicted at Screening.
✓. SaO2 ≥ 90% at Screening while breathing room air.
✓. Ability to comply with study medication use, study visits, and study procedures as judged by the investigator.

Study of Dose Escalation of Liposomal Amikacin for Inhalation (ARIKAYCE™) - Extension Phase

Plain-language summary

Who can participate

Inclusion criteria

What they're measuring

Trial details

Study of Dose Escalation of Liposomal Amikacin for Inhalation (ARIKAYCE™) - Extension Phase

Plain-language summary

Who can participate

Inclusion criteria

What they're measuring

Trial details

Exclusion criteria