"Safety, Tolerability and Pharmacokinetics of MP-376 Administered for 14 Days to Stable Pediatric (CF) Patients"

A Phase 1B, Multi-Center, Open Label Study to Evaluate the Safety, Tolerability and Pharmacokinetics of MP-376 Inhalation Solution Given Daily for 14 Days to Stable Pediatric Cystic Fibrosis Patients.

Patients with cystic fibrosis (CF) suffer from chronic infections of the lower respiratory tract that can be caused by one or multiple bacteria, including Pseudomonas aeruginosa, which has been particularly problematic to eradicate and been implicated as the major cause of morbidity and mortality in CF patients. Aerosol delivery of antibiotics directly to the lung increases the local concentrations of antibiotic at the site of infection resulting in improved antimicrobial effects compared to systemic administration. Bacterial resistance to current aerosol antibiotic treatments indicate a need for improved therapies to treat CF patients with pulmonary infections caused by multi-drug resistant Pseudomonas aeruginosa and other bacteria. High concentrations of MP-376 delivered directly to the lung are projected to have antimicrobial effects on even the most resistant organisms. MP-376 is a novel formulation of the fluoroquinolone levofloxacin that has been optimized for aerosol delivery using the PARI electronic eFlow® nebulizer. Preclinical and early clinical studies in adults show that aerosol doses of MP-376 appear to be safe and well tolerated, and exert an antimicrobial effect when administered once or twice daily. High concentrations of levofloxacin in the lung delivered using MP-376 are expected to be active against CF pathogens such as P. aeruginosa and S. aureus, including those resistant to aminoglycosides (such as TOBI®) and other inhaled antimicrobial agents. Inhaled MP-376 can be delivered rapidly and efficiently using the PARI eFlow® nebulizer system. This Phase 1 study is being performed to obtain safety, tolerability and PK data in children ages 6-16 in order to determine if MP-376 is safe, prior to enrolling children of these ages in the planned pivotal Phase 3 studies.

Study Overview

• Status: Completed
• Conditions: Cystic Fibrosis
• Intervention / Treatment: Drug: MP-376 (Levofloxacin solution for Inhalation)

Detailed Description

A Phase 1B, Multi-Center, Open Label Study to Evaluate the Safety, Tolerability and Pharmacokinetics of MP-376 Inhalation Solution given Daily for 14 Days to Stable Pediatric Cystic Fibrosis Patients.

Study acquired from Horizon in 2024.

• Study Type: Interventional
• Enrollment (Actual): 27
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Alabama
    • Mobile, Alabama, United States
  • California
    • San Diego, California, United States
  • Florida
    • Orlando, Florida, United States
  • Kentucky
    • Louisville, Kentucky, United States
  • Missouri
    • Kansas City, Missouri, United States
  • Ohio
    • Akron, Ohio, United States

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years to 16 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria (selected):

• 6 to 16 years of age (inclusive) at Visit 1
• Weight is greater than or equal to 14 kilograms (kg)
• Confirmed Diagnosis of Cystic Fibrosis
• Patients are able to elicit an FEV1 >/= 25% of predicted value (Wang criteria)
• Clinically stable with no changes in health status within the last 14 days
• Able to reproducibly undergo spirometry testing

Exclusion Criteria (selected):

• Use of any nebulized or systemic antibiotics within 7 days prior to baseline
• History of intolerance or hypersensitivity to fluoroquinolones or intolerance with aerosol medications including bronchodilators
• CrCl < 50mL/min/1.73m2, AST, ALT or total bilirubin >/= 3 x ULN at Screening or evidence of severe liver disease

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 1; CF PATIENTS 6-11 YEARS OF AGE	Drug: MP-376 (Levofloxacin solution for Inhalation); DOSE BASED ON PATIENTS WEIGHT
Experimental: 2; CF PATIENTS 12-16 YEARS OF AGE	Drug: MP-376 (Levofloxacin solution for Inhalation); DOSE BASED ON PATIENTS WEIGHT

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Safety and Tolerability of MP-376 administered for 14 days to CF patients ages 6-16	21 days

Secondary Outcome Measures

Outcome Measure	Time Frame
Serum PK Profile of MP-376 administered for 14 days to CF patients ages 6-16	21 days
Sputum PK Profile of MP-376 administered for 14 days to CF patients ages 6-16	21 days
Evaluate changes in FEV1 and FVC from baseline to end of treatment	21 Days

Collaborators and Investigators

• Sponsor: Amgen
• Investigators: Study Director: MD, Amgen

Study record dates

Study Major Dates

• Study Start: April 1, 2009
• Primary Completion (Actual): December 1, 2009
• Study Completion (Actual): December 1, 2009

Study Registration Dates

• First Submitted: February 5, 2009
• First Submitted That Met QC Criteria: February 6, 2009
• First Posted (Estimated): February 10, 2009

Study Record Updates

• Last Update Posted (Actual): December 4, 2024
• Last Update Submitted That Met QC Criteria: December 2, 2024
• Last Verified: December 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Genetic Diseases, Inborn; Respiratory Tract Diseases; Digestive System Diseases; Lung Diseases; Infant, Newborn, Diseases; Pancreatic Diseases; Fibrosis; Cystic Fibrosis; Anti-Bacterial Agents; Anti-Infective Agents; Antineoplastic Agents; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Cytochrome P-450 Enzyme Inhibitors; Topoisomerase Inhibitors; Topoisomerase II Inhibitors; Cytochrome P-450 CYP1A2 Inhibitors; Anti-Infective Agents, Urinary; Levofloxacin
• Other Study ID Numbers: Mpex-206