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

Sponsor

Horizon Pharma USA, Inc. (Industry)

Overall Status

Completed

CT.gov ID

NCT00840333

Collaborator

(none)

Enrollment

Locations

Arms

Duration (Months)

4.5

Patients Per Site

0.6

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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.

Condition or Disease	Intervention/Treatment	Phase
Cystic Fibrosis	Drug: MP-376 (Levofloxacin solution for Inhalation)	Phase 1

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 Design

Study Type:

Interventional

Actual Enrollment :

27 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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 Start Date :

Apr 1, 2009

Actual Primary Completion Date :

Dec 1, 2009

Actual Study Completion Date :

Dec 1, 2009

Arms and Interventions

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

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

Outcome Measures

Primary Outcome Measures

Safety and Tolerability of MP-376 administered for 14 days to CF patients ages 6-16 [21 days]

Secondary Outcome Measures

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]

Eligibility Criteria

Criteria

Ages Eligible for Study:

6 Years to 16 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	City	State	Country
1	Mobile	Alabama	United States
2	San Diego	California	United States
3	Orlando	Florida	United States
4	Louisville	Kentucky	United States
5	Kansas City	Missouri	United States
6	Akron	Ohio	United States

Sponsors and Collaborators

Horizon Pharma USA, Inc.

Investigators

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

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Horizon Pharma USA, Inc.

ClinicalTrials.gov Identifier:

NCT00840333

Other Study ID Numbers:

Mpex-206

First Posted:

Feb 10, 2009

Last Update Posted:

Jan 19, 2018

Last Verified:

Jan 1, 2018

Additional relevant MeSH terms:

Study Results

No Results Posted as of Jan 19, 2018