Cystic Fibrosis
Conditions
Brief summary
The primary purpose of this study is to evaluate the safety and tolerability of three active doses of nebulized amikacin in a SLIT™ formulation.
Interventions
DRUGSLIT™ Amikacin
Amikacin administered via the Pari LC STAR™ nebulizer.
DRUGPlacebo
Nebulized saline.
Sponsors
Insmed Incorporated
Study design
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Study participants must produce sputum that is positive for Pseudomonas aeruginosa. * Confirmed diagnosis of CF (positive sweat chloride \>60 milliequivalents (mEq)/liter (by pilocarpine iontophoresis) and/or a genotype with two identifiable mutations consistent with CF accompanied by one or more clinical features with the CF phenotype. * Forced expiratory volume (FEV1) ≥40% predicted at Screening as calculated by the Knudsen reference equations. * Clinically stable with no evidence of current pulmonary exacerbation.
Exclusion criteria
* History of lung transplantation. * Use of intravenous antibiotics or oral quinolones within 14 days of Screening. * Use of low dose oral antibiotics (e.g. tetracycline, sulfa) for acne or other conditions within 30 days of Screening. * Use of systemic corticosteroids (≥20 milligrams \[mg\] of prednisone per day) within 30 days of Screening. * Initiation of TOBI® (tobramycin), high dose ibuprofen, recombinant human DNase (rhDNase), or macrolide antibiotics within 60 days of Screening. * History of sputum or throat swab culture yielding Burkholderia cepacia complex within 2 years of Screening or growth of Burkholderia cepacia complex from the sputum or throat swab culture obtained at Screening. * History of biliary cirrhosis, portal hypertension, or splenomegaly or splenomegaly on physical exam at Screening or enrollment. * History of daily continuous oxygen supplementation or requirement for more than 2 liter per minute (L/min) at night. Note: Other inclusion/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants who Experience a Treatment Emergent Adverse Event (TEAE) | Up to Day 28 | Safety and tolerability of three active doses of nebulized amikacin in a SLIT™ formulation. |
Secondary
| Measure | Time frame |
|---|---|
| Area Under the Concentration-time Curve (AUC) of SLIT™ Amikacin in Serum | Pre-dose and at multiple time points post-dose up to Day 3 |
| Percent Dose of SLIT™ Amikacin in Urine | At multiple time points post-dose up to Day 3 |
| AUC of SLIT™ Amikacin in Sputum | Pre-dose and at multiple time points post-dose on Days 1, 2, 3, 8, 14, and 28 |
| Change From Baseline in Sputum Density of Pseudomonas Aeruginosa | Baseline up to Day 28 |
Outcome results
None listed