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

Cystic Fibrosis Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT06238856
• Other study ID #: TR02-101
• Status: Completed
• Phase: Phase 1
• Start date: May 12, 2004
• Est. completion date: February 8, 2005

Study information

• Verified date: January 2024
• Source: Insmed Incorporated
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 18
• Est. completion date: February 8, 2005
• Est. primary completion date: February 8, 2005
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis
• Persistent Infection

Intervention

Drug:
• SLIT™ Amikacin
Amikacin administered via the Pari LC STAR™ nebulizer.
• Placebo
Nebulized saline.

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Insmed Incorporated

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	Percent Dose of SLIT™ Amikacin in Urine		At multiple time points post-dose up to Day 3
Secondary	AUC of SLIT™ Amikacin in Sputum		Pre-dose and at multiple time points post-dose on Days 1, 2, 3, 8, 14, and 28
Secondary	Change From Baseline in Sputum Density of Pseudomonas Aeruginosa		Baseline up to Day 28