Single Dose Escalation Study of TR02 (Sustained Lipid Inhalation Technology [SLIT™] Amikacin) in Participants With Cystic Fibrosis (CF) Having Chronic Infections of Pseudomonas Aeruginosa
- Registration Number
- NCT06238856
- Lead Sponsor
- Insmed Incorporated
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 18
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/exclusion criteria may apply.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Cohort 1: Amikacin Dose 1 + Placebo Placebo Participants will receive a single dose of SLIT™ amikacin at Dose 1 or matching placebo by inhalation on Day 0. Cohort 2: Amikacin Dose 2 + Placebo Placebo Participants will receive a single dose of SLIT™ amikacin at Dose 2 or matching placebo by inhalation on Day 0 upon initiation of Cohort 2. Cohort 3: Amikacin Dose 3 + Placebo Placebo Participants will receive a single dose of SLIT™ amikacin at Dose 3 or matching placebo by inhalation on Day 0 upon initiation of Cohort 3. Cohort 3: Amikacin Dose 3 + Placebo SLIT™ Amikacin Participants will receive a single dose of SLIT™ amikacin at Dose 3 or matching placebo by inhalation on Day 0 upon initiation of Cohort 3. Cohort 1: Amikacin Dose 1 + Placebo SLIT™ Amikacin Participants will receive a single dose of SLIT™ amikacin at Dose 1 or matching placebo by inhalation on Day 0. Cohort 2: Amikacin Dose 2 + Placebo SLIT™ Amikacin Participants will receive a single dose of SLIT™ amikacin at Dose 2 or matching placebo by inhalation on Day 0 upon initiation of Cohort 2.
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method 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 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