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

NCT06238856 | Completed Phase 1 FDA Drug

• 1 other identifier: TR02-101
• Study Type: interventional
• Target: 18
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

• Number of Participants who Experience a Treatment Emergent Adverse Event (TEAE)

  Safety and tolerability of three active doses of nebulized amikacin in a SLIT™ formulation.

  Up to Day 28

Secondary Outcomes (4)

• 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

Study Arms (3)

Cohort 1: Amikacin Dose 1 + Placebo

EXPERIMENTAL

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

Drug: SLIT™ Amikacin; Drug: Placebo

Cohort 2: Amikacin Dose 2 + Placebo

EXPERIMENTAL

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.

Drug: SLIT™ Amikacin; Drug: Placebo

Cohort 3: Amikacin Dose 3 + Placebo

EXPERIMENTAL

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.

Drug: SLIT™ Amikacin; Drug: Placebo

Interventions

SLIT™ Amikacin DRUG

Amikacin administered via the Pari LC STAR™ nebulizer.

Also known as: TR02

Cohort 1: Amikacin Dose 1 + Placebo; Cohort 2: Amikacin Dose 2 + Placebo; Cohort 3: Amikacin Dose 3 + Placebo

Placebo DRUG

Nebulized saline.

Cohort 1: Amikacin Dose 1 + Placebo; Cohort 2: Amikacin Dose 2 + Placebo; Cohort 3: Amikacin Dose 3 + Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Insmed Incorporated: lead

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 25, 2024
• First Posted: February 2, 2024
• Study Start: May 12, 2004
• Primary Completion: February 8, 2005
• Study Completion: February 8, 2005
• Last Updated: February 2, 2024

Record last verified: 2024-01

Data Sharing

• IPD Sharing: Will not share