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

January 17, 2018 updated by: Horizon Pharma USA, Inc.

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

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

27

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

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

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 years to 16 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Horizon Pharma USA, Inc.

Investigators

  • Principal Investigator: Gregory L Kearns, PharmD, Childrens Mercy Hospitals and Clinics, Kansas City, MO

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

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 (Estimate)

February 10, 2009

Study Record Updates

Last Update Posted (Actual)

January 19, 2018

Last Update Submitted That Met QC Criteria

January 17, 2018

Last Verified

January 1, 2018

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • Mpex-206

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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