Cystic FibrosisPhase 1NCT02212587
Tobramycin Inhalation Powder (TIP) in Cystic Fibrosis Patients Infected With Burkholderia Cepacia Complex
A Phase 1 study testing TOBI in people with Cystic Fibrosis and Burkholderia Cepacia Infection, run by Unity Health Toronto at 2 sites in Canada.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Cystic Fibrosis, Burkholderia Cepacia Infection
- What is being tested?
- TOBI
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 2 sitesCanada
- Study dates
- Sep 2014to May 2016
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
2 sites in 1 country.
Canada2 sites
St. Michael's Hospital
Toronto, Ontario, M5B 1W8
Hospital for Sick Children
Toronto, Ontario, M5G 1X8
About this study
Official title: Pilot Study of the in Vivo Efficacy of Tobramycin Inhalation Powder (TIP) in Cystic Fibrosis Patients Infected With Burkholderia Cepacia Complex
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.
- Conditions
- Cystic Fibrosis, Burkholderia Cepacia Infection
- Being tested
- TOBI
- Keywords
- inhaled antibiotics
- Medical subject headings
- Cystic Fibrosis, Burkholderia Infections
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TOBIDrug
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
| Study group | Role | What they receive |
|---|---|---|
| TOBI Podhaler | Experimental | Not described Receives: Drug: TOBI |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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 %.
Measured: 0 to 28 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Aug 2014 (estimated)
- Study startSep 2014
- Primary completionMay 2016
- Study completionMay 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Unity Health TorontoThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Unity Health Toronto
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Valerie Waters, MD, Principal investigator, Hospital of Sick Children
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02212587
- Sponsor's protocol ID
- TIPBCC002
- Other IDs
- 165476
- Status last verified
- Oct 2020
- First submitted
- 6 Aug 2014
- Last updated
- 4 Nov 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
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
Study at a glance
From the registry record, last updated by the sponsor on 4 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study of the in Vivo Efficacy of Tobramycin Inhalation Powder (TIP) in Cystic Fibrosis Patients Infected With Burkholderia Cepacia Complex
- ClinicalTrials.gov ID
- NCT02212587
- Acronym / sponsor ID
- TIPBCC002
- Conditions
- Cystic Fibrosis, Burkholderia Cepacia Infection
- Keywords
- inhaled antibiotics
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Unity Health TorontoUniversity, hospital or other
- Study start
- Sep 2014
- Primary completion
- May 2016 (actual)
- Study completion
- May 2016 (actual)
- First posted
- 8 Aug 2014 (estimated)
- Results
- Posted 4 Nov 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| TOBI Podhaler | Experimental | Drug: TOBI |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TOBINew 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 | Drug | TOBI Podhaler |
Primary outcome
The Change in Sputum Density of BCC in Colony Forming Units (CFUs)/ml From Day 0 to Day 28 of TIP Treatment.
Time frame: 0 to 28 days
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.
1 secondary outcome
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.
Time frame: 0 to 28 days
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Nov 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 28262569Waters 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Valerie Waters, MD principal investigator
Hospital of Sick Children
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing TOBI in people with Cystic Fibrosis and Burkholderia Cepacia Infection, run by Unity Health Toronto at 2 sites in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Nov 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 4 Nov 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The Change in Sputum Density of BCC in Colony Forming Units (CFUs)/ml From Day 0 to Day 28 of TIP Treatment. (time frame: 0 to 28 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02212587, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Nov 2020.