ong Term Safety and Tolerability Study of Open-Label Liposomal Amikacin for Inhalation (Arikace*) in Cystic Fibrosis Patients with Chronic Infection due to Pseudomonas aeruginosa

Phase 3

Completed

• Conditions: cystic fibrosis; 10038686

• Registration Number: NL-OMON39469
• Lead Sponsor: Chiltern International Ltd

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 6 May 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 3

Inclusion Criteria

1) Written informed consent or assent obtained from the subject, parent or legal guardian to participate in the open label multi-cycle study, TR02-110
2) Subject has completed study TR02-108 or TR02-109, and has been compliant with the study protocol
3) Negative pregnancy result on pregnancy test performed within 2 weeks prior to Day 1 and use of reliable methods of contraception (e.g., abstinence, hormonal or barrier methods, partner sterilization, or IUD) throughout the study duration in women of child-bearing potential. Women not of childbearing potential are defined as prepubescent, postmenopausal (i.e., amenorrhea for at least 1 year), or surgically or naturally sterile

Exclusion Criteria

1) Subject met any of the listed criteria for study drug discontinuation (safety reasons or non-compliance) in protocol TR02-108 or TR02-109
2) Elevated AST, ALT or GGT >= 3× the upper limit of normal (ULN) within 4 weeks prior to Day 1
3) Absolute neutrophil count < 1000 within 4 weeks prior to Day 1
4) Serum creatinine > 2× ULN within 4 weeks prior to Day 1
5) Psychotic, addictive or other disorder limiting the ability to provide informed consent or to comply with study requirements.
6) History of alcohol, medication, or illicit drug abuse within the 6 months prior to consent
7) Smoking tobacco or any substance within 6 months prior to consent or anticipated inability to refrain from smoking throughout the study
8) Any condition which in the opinion of the investigator interferes with the subject*s ability to safely complete the study or adhere to the study requirements

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Primary endpoints<br /><br>• Adverse events (AE) as a result of treatment up to Day 672<br /><br>• Adverse events leading to study medicine being permanently discontinued<br /><br>• Severe adverse events (SAEs) up to Day 672<br /><br>• Laboratory abnormalities up to Day 672<br /><br>• Acute tolerability as measured by PFT changes pre to post dose<br /><br>• Vital signs and oxygen saturation (changes from pre- to post dose) and change<br /><br>from baseline<br /><br>• Shift in MICs for Pa and Burkholderia sp from Day 1 to Days 169, 337, 505 and<br /><br>672<br /><br>• Proportion of subjects with emergent pathogens<br /><br>• Evaluation of audiology<br /><br>• Change in serum creatinine throughout the entire study<br /><br>• Concomitant medication including antibiotics, anti-viral agents, antifungal<br /><br>agents and other inhalation medicines, such as bronchodilators, DNase, inhaled<br /><br>steroids </p><br>