Clinical trial NCT06238856: Single Dose Escalation Study of TR02 (Sustained Lipid Inhalation Technology [SLIT™] Amikacin) in Participants With Cystic Fibrosis (CF) Having Chronic Infections of Pseudomonas Aeruginosa
Official title: Safety and Tolerability Study of Single Dose Escalations of TR02 (SLIT™ Amikacin) by Inhalation in Cystic Fibrosis Study Patients With Chronic Infections of Pseudomonas Aeruginosa
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Cystic Fibrosis
Lead sponsor: Insmed Incorporated
Sponsor class: INDUSTRY
First posted: 2024-02-02
Enrollment: 18
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Number of Participants who Experience a Treatment Emergent Adverse Event (TEAE)
timeFrame: Up to Day 28
Secondary outcomes:
measure: Area Under the Concentration-time Curve (AUC) of SLIT™ Amikacin in Serum
timeFrame: Pre-dose and at multiple time points post-dose up to Day 3
measure: Percent Dose of SLIT™ Amikacin in Urine
timeFrame: At multiple time points post-dose up to Day 3
measure: AUC of SLIT™ Amikacin in Sputum
timeFrame: Pre-dose and at multiple time points post-dose on Days 1, 2, 3, 8, 14, and 28
measure: Change From Baseline in Sputum Density of Pseudomonas Aeruginosa
timeFrame: Baseline up to Day 28
Other outcomes:
Locations:
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