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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01316276
• Date of registration: 14/03/2011
• Prospective Registration: Yes
• Primary sponsor: Insmed Incorporated
• Public title: Extension Study of Liposomal Amikacin for Inhalation in Cystic Fibrosis (CF) Patients With Chronic Pseudomonas Aeruginosa (Pa) Infection
• Scientific title: Long Term Safety and Tolerability Study of Open-Label Liposomal Amikacin for Inhalation (ARIKACE™) in Cystic Fibrosis Patients With Chronic Infection Due to Pseudomonas Aeruginosa
• Date of first enrolment: October 5, 2012
• Target sample size: 206
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01316276
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Austria, Belgium, Bulgaria, Canada, Denmark, France, Germany, Greece, Hungary, Ireland, Italy, Netherlands, Poland, Serbia, Slovakia, Spain, United Kingdom

Contacts

• Name: Gina Eagle, MD
• Affiliation: Insmed Incorporated

Key inclusion & exclusion criteria

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



Age minimum: 6 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: Liposomal amikacin for inhalation

Primary Outcome(s)

Acute Tolerability as Measured by Pulmonary Function Test (PFT) Changes Pre to Post Dose [Time Frame: Day 1, Day 84, Day 196, Day 281, Day 337, Day 449, Day 532 and Day 644]

Heart Rate: Change From Baseline From Day 672 [Time Frame: From Study Initiation up to Day 672]

Minimum Inhibitory Concentrations (MICs) for Pseudomonas Aeruginosa (Pa) and Burkholderia Species From Day 1 to Days 169, 337, 505 and 672 [Time Frame: Day 1, Day 169, Day 337, Day 505 and Day 672]

Diastolic BP: Change From Baseline at Day 672 [Time Frame: From Study Initiation up to Day 672]

Body Temperature: Change From Baseline at Day 672 [Time Frame: From Study Initiation up to Day 672]

Laboratory Abnormalities up to Day 672 [Time Frame: Baseline, Day 377 and Day 672]

Oxygen Saturation: Change From Baseline at Day 672 [Time Frame: From Study Initiation up to Day 672]

Systolic BP: Change From Baseline at Day 672 [Time Frame: From Study Initiation up to Day 672]

Treatment Emergent Adverse Events (TEAEs) up to Day 672 [Time Frame: From Study Initiation up to Day 672]

Change in Serum Creatinine Throughout the Study [Time Frame: Baseline, Day 337 and Day 672]

Respiratory Rate: Change From Baseline to Day 672 [Time Frame: From Study Initiation up to Day 672]

Evaluation of Audiology [Time Frame: Day 337 and Day 672]

Secondary Outcome(s)

Number of Participants Who Received Antipseudomonal Antibiotic Treatment for Protocol Defined Pulmonary Exacerbation [Time Frame: From Study Initiation up to Day 700]

Number of Subjects Initiating Treatment. [Time Frame: From Study Initiation up to Day 672]

Number of Subjects Experiencing a Protocol Defined Pulmonary Exacerbation [Time Frame: From Study Initiation up to Day 700]

Percent Change in FEV1 Throughout the Study [Time Frame: Baseline, Day 337 and Day 672]

Secondary ID(s)

TR02-110

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 26/07/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01316276