Single Dose Escalation Study of TR02 (Sustained Lipid Inhalation Technology [SLIT™] Amikacin) in Participants With Cystic Fibrosis (CF) Having Chronic Infections of Pseudomonas Aeruginosa

Insmed

Status and phase: Completed; Phase 1

Conditions: Cystic Fibrosis

Treatments: Drug: Placebo; Drug: SLIT™ Amikacin

Study type: Interventional

Funder types: Industry

Identifiers

NCT06238856

TR02-101

Details and patient eligibility

About

The primary purpose of this study is to evaluate the safety and tolerability of three active doses of nebulized amikacin in a SLIT™ formulation.

Enrollment: 18 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: Quadruple Blind

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18 participants in 3 patient groups

Cohort 1: Amikacin Dose 1 + Placebo

Experimental group

Description:

Participants will receive a single dose of SLIT™ amikacin at Dose 1 or matching placebo by inhalation on Day 0.

Treatment:

Drug: SLIT™ Amikacin

Drug: Placebo

Cohort 2: Amikacin Dose 2 + Placebo

Experimental group

Description:

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.

Treatment:

Drug: SLIT™ Amikacin

Drug: Placebo

Cohort 3: Amikacin Dose 3 + Placebo

Experimental group

Description:

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.

Treatment:

Drug: SLIT™ Amikacin

Drug: Placebo