Clinical trial NCT02212587: Tobramycin Inhalation Powder (TIP) in Cystic Fibrosis Patients Infected With Burkholderia Cepacia Complex
Official title: Pilot Study of the in Vivo Efficacy of Tobramycin Inhalation Powder (TIP) in Cystic Fibrosis Patients Infected With Burkholderia Cepacia Complex
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Cystic Fibrosis
Burkholderia Cepacia Infection
Keywords: inhaled antibiotics
Lead sponsor: Unity Health Toronto
Sponsor class: OTHER
First posted: 2014-08-08
Enrollment: 10
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The objective of this study is to determine if tobramycin inhalation powder (TIP) can reduce the amount of Burkholderia Cepacia Complex (BCC) species - type of bacteria, in the sputum of cystic fibrosis patient.


Eligibility criteria: Inclusion Criteria:

* Age 6 years or older
* Diagnosis of CF based on the following: sweat chloride\>60 mEq/L or genotype with 2 identifiable mutations consistent with CF; and one or more clinical features consistent with CF.
* Chronically infected with a Burkholderia cepacia complex species (\>50% of respiratory specimens positive in the 24 months prior to screening).
* Able to produce sputum (expectorated or induced).
* Able to reproducibly perform pulmonary function testing.
* Written informed consent provided.

Exclusion Criteria:

* Post lung transplantation.
* Pregnancy.
* Acute exacerbation requiring IV or oral antibiotics within 14 days
* Patients currently receiving inhaled tobramycin/TOBI
* A septic or clinically unstable patient, as determined by the investigator.

Primary outcomes:
measure: The Change in Sputum Density of BCC in Colony Forming Units (CFUs)/ml From Day 0 to Day 28 of TIP Treatment.
timeFrame: 0 to 28 days
Secondary outcomes:
measure: The Change in Pulmonary Function Tests, Including Forced Expiratory Volume in 1 Second (FEV1), Forced Vital Capacity (FVC) and Maximal Mid-expiratory Flow Rate (FEF25-75), Measured at Day 0 and Day 28 of TIP Treatment.
timeFrame: 0 to 28 days
Other outcomes:
measure: The Change in the Measurement of Markers of Pulmonary Inflammation (Neutrophil Counts, Neutrophil Elastase and IL-8 Levels in Sputum) Measured at Day 0 and Day 28 of TIP Treatment.
timeFrame: 0 to 28 days
measure: Trough and Peak Sputum Tobramycin Concentration Measured on Day 28 of TIP Treatment.
timeFrame: Day 28
measure: The Change in BCC Planktonic and Biofilm MICs to Tobramycin Measured on Day 0 and on Day 28 of TIP Treatment.
timeFrame: 0 to 28 days
measure: The Number of Participants With Adverse Events Measured at Day 14 and at Day 28 of TIP Treatment.
timeFrame: At Dat 0, Day 14, and Day 28
measure: The Change in the Measurement of Markers of Pulmonary Inflammation (Neutrophil Counts, Neutrophil Elastase and IL-8 Levels in Sputum) Measured at Day 0 and Day 28 of TIP Treatment.
timeFrame: 0 to 28 days
Locations:
city: Toronto
country: Canada
facility: St. Michael's Hospital
state: Ontario
city: Toronto
country: Canada
facility: Hospital for Sick Children
state: Ontario
Study officials:
affiliation: Hospital of Sick Children
name: Valerie Waters, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Waters V, Yau Y, Beaudoin T, Wettlaufer J, Tom SK, McDonald N, Rizvi L, Klingel M, Ratjen F, Tullis E. Pilot trial of tobramycin inhalation powder in cystic fibrosis patients with chronic Burkholderia cepacia complex infection. J Cyst Fibros. 2017 Jul;16(4):492-495. doi: 10.1016/j.jcf.2017.02.008. Epub 2017 Mar 3.