CompletedPhase 3

Extension Study of Liposomal Amikacin for Inhalation in Cystic Fibrosis (CF) Patients With Chronic Pseudomonas Aeruginosa (Pa) Infection

Austria, Belgium, Bulgaria206 participantsStarted 2012-10-05

Plain-language summary

The purpose of this study is to evaluate the long term safety and tolerability of Liposomal Amikacin for Inhalation (LAI) 590 mg once daily (QD) in Cystic Fibrosis patients with chronic infection due to pseudomonas aeruginosa. This long-term, open-label, multi-cycle extension study enrolled subjects who had successfully completed study TR02-108, were compliant with the study protocol, and did not meet any of the listed study discontinuation criteria. The safety and tolerability of LAI were evaluated for up to approximately 2 years.

Who can participate

Age range

6 Years

Sex

ALL

See this in plain English?

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

Key Inclusion Criteria: * Written informed consent or assent * Subject has completed study TR02-108, and has been compliant with the study protocol * Women of childbearing potential must agree to use reliable methods of contraception for the duration of the study Key Exclusion Criteria: * Subject met any of the listed criteria for study drug discontinuation in protocol TR02-108. * Abnormal laboratory assessments including LFT (≥ 3× upper limit of normal \[ULN\]), serum creatinine (\> 2× ULN) and absolute neutrophil count \[ANC\] (\< 1000). * Psychotic, addictive or other disorder limiting the ability to provide informed consent or to comply with study requirements. * History of alcohol, medication or illicit drug abuse within the 6 months prior to consent. * Smoking tobacco or any substance within 6 months prior to consent or anticipated inability to refrain from smoking throughout the study

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Treatment Emergent Adverse Events (TEAEs) up to Day 672

Timeframe: From Study Initiation up to Day 672

Laboratory Abnormalities up to Day 672

Timeframe: Baseline, Day 377 and Day 672

Acute Tolerability as Measured by Pulmonary Function Test (PFT) Changes Pre to Post Dose

Timeframe: Day 1, Day 84, Day 196, Day 281, Day 337, Day 449, Day 532 and Day 644

Respiratory Rate: Change From Baseline to Day 672

Timeframe: From Study Initiation up to Day 672

Heart Rate: Change From Baseline From Day 672

Timeframe: From Study Initiation up to Day 672

Systolic BP: Change From Baseline at Day 672

Timeframe: From Study Initiation up to Day 672

Diastolic BP: Change From Baseline at Day 672

Timeframe: From Study Initiation up to Day 672

Trial details

NCT IDNCT01316276

SponsorInsmed Incorporated

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2015-07-16

Results submitted2019-04-03

View on ClinicalTrials.gov More Cystic Fibrosis trials

Plain-language summary

Who can participate

Age range

6 Years

Sex

ALL

See this in plain English?

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

What they're measuring

Treatment Emergent Adverse Events (TEAEs) up to Day 672

Timeframe: From Study Initiation up to Day 672

Laboratory Abnormalities up to Day 672

Timeframe: Baseline, Day 377 and Day 672

Acute Tolerability as Measured by Pulmonary Function Test (PFT) Changes Pre to Post Dose

Timeframe: Day 1, Day 84, Day 196, Day 281, Day 337, Day 449, Day 532 and Day 644

Respiratory Rate: Change From Baseline to Day 672

Timeframe: From Study Initiation up to Day 672

Heart Rate: Change From Baseline From Day 672

Timeframe: From Study Initiation up to Day 672

Systolic BP: Change From Baseline at Day 672

Timeframe: From Study Initiation up to Day 672

Diastolic BP: Change From Baseline at Day 672

Timeframe: From Study Initiation up to Day 672