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CompletedNCT02212587Updated Nov 4, 2020Results posted

Tobramycin Inhalation Powder (TIP) in Cystic Fibrosis Patients Infected With Burkholderia Cepacia Complex

A Phase 1 interventional study of TOBI in Cystic Fibrosis and Burkholderia Cepacia Infection, sponsored by Unity Health Toronto. Completed at 2 sites in Canada. Open to participants aged 6 Years and older. Per ClinicalTrials.gov, last updated 2020-11-04.

Sponsored by Unity Health Toronto · Phase 1, Interventional, and Treatment

Phase
Phase 1
Study type
Interventional
Enrollment
10
Allocation
Not applicable
Ages
6 Years and older
Sex
All
01

Study 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.

02

Conditions studied

  • Cystic Fibrosis
  • Burkholderia Cepacia Infection

Keywords

  • inhaled antibiotics
03

In context

Cystic Fibrosis

1,581 studies on the registry are indexed under Cystic Fibrosis; 190 are open to participants now.

This study's enrollment of 10 is below the median of 36 across 1,034 interventional studies indexed under Cystic Fibrosis.

Browse Cystic Fibrosis studies →

Lead sponsor

Unity Health Toronto is the lead sponsor of 434 studies on the registry; 76 are open to participants now.

Of its 5 completed or terminated interventional studies of FDA-regulated products, 1 (20%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
6 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

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

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.
05

Study design

Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
10 participants (actual)

Study arms

  • Experimental
    TOBI Podhaler

    Drug: TOBI

Interventions

  • DrugTOBI

    New inhalation devices such as the podhaler can administer tobramycin inhalation powder TIP/TOBI and achieve very high sputum drug levels. TOBI will be administered using the Podhaler

06

What researchers measure

Primary outcomes

  1. The Change in Sputum Density of BCC in Colony Forming Units (CFUs)/ml From Day 0 to Day 28 of TIP Treatment.

    The change in sputum density of BCC in colony forming units (CFUs)/ml from day 0 to day 28 of TIP treatment. Sputum density was calculated by doing serial dilutions of sputum on agar plates and counting colony forming units expressed per ml.

    Time frame: 0 to 28 days

Secondary outcomes

  1. 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.

    The main lung function measure was relative change in FEV1 from day 0 to Day 28 reported as %.

    Time frame: 0 to 28 days

Other outcomes

  1. 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.

    We measured both neutrophil elastase sputum for participants.

    Time frame: 0 to 28 days

  2. Trough and Peak Sputum Tobramycin Concentration Measured on Day 28 of TIP Treatment.

    We did trough (0 hour) and peak (1 hour post dose) TIP sputum tobramycin concentrations and reported it for participants.

    Time frame: Day 28

  3. The Change in BCC Planktonic and Biofilm MICs to Tobramycin Measured on Day 0 and on Day 28 of TIP Treatment.

    For all participants, the minimum inhibitory concentration of tobramycin for planktonic and biofilm grown BCC was measured in the lab on the isolate collected before and after TIP treatment.

    Time frame: 0 to 28 days

  4. The Number of Participants With Adverse Events Measured at Day 14 and at Day 28 of TIP Treatment.

    The number of participants with adverse events was reported for all participants in the trial.

    Time frame: At Dat 0, Day 14, and Day 28

  5. 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.

    We measured both IL-8 sputum for participants.

    Time frame: 0 to 28 days

07

Results

Posted Nov 4, 2020

Participant flow

Participant flow — Overall Study
MilestoneTOBI Podhaler
Started10
Completed9
Not completed1

Outcome measures

PrimaryThe Change in Sputum Density of BCC in Colony Forming Units (CFUs)/ml From Day 0 to Day 28 of TIP Treatment.

The change in sputum density of BCC in colony forming units (CFUs)/ml from day 0 to day 28 of TIP treatment. Sputum density was calculated by doing serial dilutions of sputum on agar plates and counting colony forming units expressed per ml.

Time frame:
0 to 28 days
Reported as:
Mean · CFU/ml log10
The Change in Sputum Density of BCC in Colony Forming Units (CFUs)/ml From Day 0 to Day 28 of TIP Treatment.
CFU/ml log10TOBI Podhaler
The Change in Sputum Density of BCC in Colony Forming Units (CFUs)/ml From Day 0 to Day 28 of TIP Treatment.-1.4 (-3.9 to 0.8)
SecondaryThe 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.

The main lung function measure was relative change in FEV1 from day 0 to Day 28 reported as %.

Time frame:
0 to 28 days
Reported as:
Mean · relative change in FEV1 expressed as %
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.
relative change in FEV1 expressed as %TOBI Podhaler
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.4.6 (-6.6 to 28.1)
Other pre-specifiedThe 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.

We measured both neutrophil elastase sputum for participants.

Time frame:
0 to 28 days
Reported as:
Mean · NE = ug/ml
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.
NE = ug/mlTOBI Podhaler
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.-51.9 (-188.4 to 47.3)
Other pre-specifiedTrough and Peak Sputum Tobramycin Concentration Measured on Day 28 of TIP Treatment.

We did trough (0 hour) and peak (1 hour post dose) TIP sputum tobramycin concentrations and reported it for participants.

Time frame:
Day 28
Reported as:
Median · ug/ml
Trough and Peak Sputum Tobramycin Concentration Measured on Day 28 of TIP Treatment.
ug/mlTOBI Podhaler
Trough (0 hr)194 (11 to 2100)
Peak (1 hr post dose)1025 (306 to 4700)
Other pre-specifiedThe Change in BCC Planktonic and Biofilm MICs to Tobramycin Measured on Day 0 and on Day 28 of TIP Treatment.

For all participants, the minimum inhibitory concentration of tobramycin for planktonic and biofilm grown BCC was measured in the lab on the isolate collected before and after TIP treatment.

Time frame:
0 to 28 days
Reported as:
Mean · ug/ml (MIC50)
The Change in BCC Planktonic and Biofilm MICs to Tobramycin Measured on Day 0 and on Day 28 of TIP Treatment.
ug/ml (MIC50)TOBI Podhaler
Day 0200 (10 to 1600)
Day 28400 (10 to 1600)
Other pre-specifiedThe Number of Participants With Adverse Events Measured at Day 14 and at Day 28 of TIP Treatment.

The number of participants with adverse events was reported for all participants in the trial.

Time frame:
At Dat 0, Day 14, and Day 28
Reported as:
Count of participants · Participants
The Number of Participants With Adverse Events Measured at Day 14 and at Day 28 of TIP Treatment.
ParticipantsTOBI Podhaler
The Number of Participants With Adverse Events Measured at Day 14 and at Day 28 of TIP Treatment.6
Other pre-specifiedThe 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.

We measured both IL-8 sputum for participants.

Time frame:
0 to 28 days
Reported as:
Mean · IL-8 = ng/ml
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.
IL-8 = ng/mlTOBI Podhaler
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.-127 (-298.62 to 101.8)

Adverse events

Collected over 1 month. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
TOBI Podhaler—0/10 (0%)0/10 (0%)

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)TOBI Podhaler
<=18 years4
Between 18 and 65 years6
>=65 years0
Sex: Female, Male
Sex: Female, Male(Participants)TOBI Podhaler
Female2
Male8
Region of Enrollment
Region of Enrollment(participants)TOBI Podhaler
Canada10
Burkholderia cenocepacia IIIA, n (%)
Burkholderia cenocepacia IIIA, n (%)(Participants)TOBI Podhaler
Count of participants4
Burkholderia cenocepacia IIIB, n (%)
Burkholderia cenocepacia IIIB, n (%)(Participants)TOBI Podhaler
Count of participants3
Burkholderia multivorans, n (%)
Burkholderia multivorans, n (%)(Participants)TOBI Podhaler
Count of participants3
Sputum density log10 CFU/ml, median (range)
Sputum density log10 CFU/ml, median (range)(log10 CFU/ml)TOBI Podhaler
Median7.9 (3.1 to 8.8)
08

Study locations

2 sites
  • St. Michael's Hospital
    Toronto, Ontario M5B 1W8, Canada
  • Hospital for Sick Children
    Toronto, Ontario M5G 1X8, Canada
09

References and documents

Publications

  • 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. PubMed 28262569 ↗
10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Nov 4, 2020, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT02212587
Lead sponsor
Unity Health Toronto
Responsible party
Sponsor
First posted
Aug 8, 2014
Start date
Sep 2014
Primary completion
May 2016
Completion
May 2016
Results posted
Nov 4, 2020
Last update
Nov 4, 2020

Study contacts

Valerie Waters, MD
principal investigator · Hospital of Sick Children

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

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