Cystic FibrosisPhase 2NCT00794586
Study Evaluating Fosfomycin/Tobramycin for Inhalation in Cystic Fibrosis Patients With Pseudomonas Aeruginosa Lung Infection
A Phase 2 study testing FTI, AZLI in people with Cystic Fibrosis, run by Gilead Sciences at 36 sites in United States.
Trial summary
The purpose of this study is to evaluate the safety and efficacy of 2 dose combinations of fosfomycin/tobramycin for inhalation (FTI), following a 28-day course of Aztreonam for Inhalation (AZLI) in patients with cystic fibrosis and Pseudomonas aeruginosa lung infection. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- FTI, AZLI
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 120 participants
- Where
- 36 sitesUnited States
- Study dates
- Nov 2008to Mar 2010
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Males or females aged 18 years and older
- Patients with CF as diagnosed by one of the following:
- Documented sweat chloride greater than or equal to 60 mEq/L by quantitative pilocarpine iontophoresis test, OR
- Documented sweat sodium greater than or equal to 60 mmol/L, OR
- Abnormal nasal potential difference, OR
- Two well-characterized genetic mutations in the CF transmembrane conductance regulator (CFTR) gene, AND
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- Accompanying symptoms characteristic of CF
- Documented presence of PA in an expectorated sputum or throat swab culture at Visit 1 OR documented PA in 2 expectorated sputum or throat swab cultures within 12 months prior to Visit 1; one of the previous PA-positive cultures must be within 3 months prior to Visit 1
- Patients must be able to provide written informed consent prior to any study related procedures
- FEV1 greater than or equal to 25% and less than or equal to 75% predicted at Visit 1
- Ability to perform reproducible pulmonary function tests
- Chest radiograph at Visit 1 without significant acute findings (e.g., infiltrates [lobar or diffuse interstitial], pleural effusion, pneumothorax); or chest radiograph, CT, or MRI obtained within the 90 days prior to Visit 1 without acute findings or significant intercurrent illness; chronic, stable findings (e.g., chronic scarring or atelectasis) are allowed.
Who cannot join
- Current use of oral corticosteroids in doses exceeding the equivalent of 10 mg prednisone a day or 20 mg prednisone every other day
- History of sputum or throat swab culture yielding B. cepacia in the previous 2 years
- Current requirement for daily continuous oxygen supplementation or requirement for more than 2 L/minute at night
- Administration of any investigational drug or device within 28 days of Visit 1 or within 6 half-lives of the investigational drug (whichever is longer)
- Known local or systemic hypersensitivity to monobactam antibiotics
- Known allergies/intolerance to tobramycin or other aminoglycosides
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- Known allergies/intolerance to fosfomycin
- Inability to tolerate inhalation of a short acting beta2 agonist
- Changes in or initiation of chronic azithromycin treatment within 14 days prior to Visit 1
- Administration of antipseudomonal antibiotics by inhalation, IV, or oral routes within the 14 days prior to Visit 1
- Changes in antimicrobial, bronchodilator (BD), corticosteroid, hypertonic saline, or dornase alfa medications within 7 days prior to Visit 1
- Changes in physiotherapy technique or schedule within 7 days prior to Visit 1
- History of lung transplantation
- Abnormal renal or hepatic function or serum chemistry at Visit 1, defined as:
- AST, ALT > 3 times upper limit of normal range (ULN)
- Creatinine > 1.5 times ULN
- Positive pregnancy test at Visit 1; all women of childbearing potential will be tested
- Female patients of childbearing potential who are lactating or are not (in the opinion of the investigator) practicing an acceptable method of birth control; female patients who utilize hormonal contraceptives as a birth control method must have used the same method for at least 3 months before study dosing
- Any serious or active medical or psychiatric illness, which in the opinion of the investigator, would interfere with patient treatment, assessment, or compliance with the protocol
Trial locations and contacts
36 sites in 1 country.
36 sites in 1 country
United States36 sites
Study site
Albany, New York, 12208
Study site
Anchorage, Alaska, 99508
Study site
Ann Arbor, Michigan, 48109
Study site
Boston, Massachusetts, 02114
Study site
Boston, Massachusetts, 02115
Study site
Buffalo, New York, 14222
Study site
Chapel Hill, North Carolina, 27599
Study site
Charleston, South Carolina, 29425
Study site
Chicago, Illinois, 60614
Study site
Cincinnati, Ohio, 45229
Study site
Cleveland, Ohio, 44106
Study site
Columbus, Ohio, 43205
Study site
Denver, Colorado, 80206
Study site
Detroit, Michigan, 48201
Study site
Glenview, Illinois, 60025
Study site
Hartford, Connecticut, 06102
Study site
Hershey, Pennsylvania, 17033
Study site
Houston, Texas, 77030
Study site
Las Vegas, Nevada, 89107
Study site
Little Rock, Arkansas, 72205
Study site
New Haven, Connecticut, 06520
Study site
New Hyde Park, New York, 11040
Study site
New York, New York, 10032
Study site
Oklahoma City, Oklahoma, 73112
Study site
Orlando, Florida, 32803
Study site
Palo Alto, California, 94304
Study site
Philadelphia, Pennsylvania, 19102
Study site
Phoenix, Arizona, 85016
Study site
Portsmouth, Virginia, 23708
Study site
Richmond, Virginia, 23298
Study site
San Antonio, Texas, 78212
Study site
Syracuse, New York, 13210
Study site
Tacoma, Washington, 98405
Study site
Tampa, Florida, 33606
Study site
Tucson, Arizona, 85724
Study site
Washington D.C., District of Columbia, 20010
About this study
Official title: A Phase 2, Double-Blind, Multicenter, Randomized, Placebo-Controlled Trial Evaluating Fosfomycin/Tobramycin for Inhalation in Patients With Cystic Fibrosis and Pseudomonas Aeruginosa
The purpose of this study is to evaluate the safety and efficacy of 2 dose combinations of fosfomycin/tobramycin for inhalation (FTI), following a 28-day course of Aztreonam for Inhalation (AZLI) in patients with cystic fibrosis and Pseudomonas aeruginosa lung infection.
Read the detailed description
Gilead is developing a broad spectrum combination antibiotic (FTI) consisting of fosfomycin (an antibiotic with activity against gram-positive and gram-negative bacteria) and tobramycin (an aminoglycoside antibiotic with potent gram-negative activity) for treatment of patients with CF. FTI offers a potential option for treatment of CF lung infections. It is important to note that the concentration of tobramycin in FTI is lower than that of the approved dose of inhaled tobramycin alone, thereby demonstrating the potential of FTI to minimize long-term toxicity from repeated exposure to aminoglycosides like tobramycin. This study will evaluate the safety and efficacy of 2 dose combinations of fosfomycin/tobramycin for inhalation (FTI), following a 28-day course of Aztreonam for Inhalation (AZLI) in patients with cystic fibrosis and Pseudomonas aeruginosa lung infection.
- Conditions
- Cystic Fibrosis
- Being tested
- FTI, AZLI, Placebo, AZLI
- Keywords
- cystic fibrosis, CF, inhaled antibiotic, antipseudomonal antibiotic, tobramycin, fosfomycin, lung infection, pseudomonal infection, FTI
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FTI, AZLIDrug
Evaluation of 2 dosage combinations of fosfomycin and tobramycin inhaled twice daily for 28 days following 28 days of inhaled AZLI (Aztreonam Lysine for Inhalation)
Placebo, AZLIDrug
Volume-matched placebo inhaled twice daily for 28 days following 28 days of AZLI (Aztreonam Lysine for Inhalation).
| Study group | Role | What they receive |
|---|---|---|
| FTI 80mg/20mg BID | Experimental | Fosfomycin/Tobramycin combination 80mg/20 mg inhaled twice daily Receives: Drug: FTI, AZLI |
| FTI 160mg/40mg BID | Experimental | Fosfomycin/Tobramycin combination 160 mg/40 mg inhaled twice daily Receives: Drug: FTI, AZLI |
| Placebo A BID | Placebo comparator | Placebo A inhaled twice daily Receives: Drug: Placebo, AZLI |
| Placebo B BID | Placebo comparator | Placebo B inhaled twice daily Receives: Drug: Placebo, AZLI |
What is being measured
The primary outcome is the main question the study is designed to answer.
Relative change in lung function from baseline at Day 28.
Measured: 28 Days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Nov 2008 (estimated)
- Study startNov 2008
- Primary completionFeb 2010
- Study completionMar 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Gilead SciencesThe 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 120 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Gilead Sciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Bruce Trapnell, MD, Principal investigator, Children's Hospital Medical Center, Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00794586
- Sponsor's protocol ID
- GS-US-207-0103
- Status last verified
- Dec 2013
- First submitted
- 18 Nov 2008
- Last updated
- 27 Dec 2013
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 27 Dec 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Double-Blind, Multicenter, Randomized, Placebo-Controlled Trial Evaluating Fosfomycin/Tobramycin for Inhalation in Patients With Cystic Fibrosis and Pseudomonas Aeruginosa
- ClinicalTrials.gov ID
- NCT00794586
- Acronym / sponsor ID
- GS-US-207-0103
- Conditions
- Cystic Fibrosis
- Keywords
- cystic fibrosis, CF, inhaled antibiotic, antipseudomonal antibiotic, tobramycin, fosfomycin, lung infection, pseudomonal infection
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 120 participants (estimated)
- Lead sponsor
- Gilead SciencesIndustry (pharma, biotech, devices)
- Study start
- Nov 2008
- Primary completion
- Feb 2010 (actual)
- Study completion
- Mar 2010 (actual)
- First posted
- 20 Nov 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to evaluate the safety and efficacy of 2 dose combinations of fosfomycin/tobramycin for inhalation (FTI), following a 28-day course of Aztreonam for Inhalation (AZLI) in patients with cystic fibrosis and Pseudomonas aeruginosa lung infection.
Detailed description
Gilead is developing a broad spectrum combination antibiotic (FTI) consisting of fosfomycin (an antibiotic with activity against gram-positive and gram-negative bacteria) and tobramycin (an aminoglycoside antibiotic with potent gram-negative activity) for treatment of patients with CF. FTI offers a potential option for treatment of CF lung infections. It is important to note that the concentration of tobramycin in FTI is lower than that of the approved dose of inhaled tobramycin alone, thereby demonstrating the potential of FTI to minimize long-term toxicity from repeated exposure to aminoglycosides like tobramycin. This study will evaluate the safety and efficacy of 2 dose combinations of fosfomycin/tobramycin for inhalation (FTI), following a 28-day course of Aztreonam for Inhalation (AZLI) in patients with cystic fibrosis and Pseudomonas aeruginosa lung infection.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| FTI 80mg/20mg BID | Experimental | Drug: FTI, AZLIFosfomycin/Tobramycin combination 80mg/20 mg inhaled twice daily |
| FTI 160mg/40mg BID | Experimental | Drug: FTI, AZLIFosfomycin/Tobramycin combination 160 mg/40 mg inhaled twice daily |
| Placebo A BID | Placebo comparator | Drug: Placebo, AZLIPlacebo A inhaled twice daily |
| Placebo B BID | Placebo comparator | Drug: Placebo, AZLIPlacebo B inhaled twice daily |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Males or females aged 18 years and older
- Patients with CF as diagnosed by one of the following:
- Documented presence of PA in an expectorated sputum or throat swab culture at Visit 1 OR documented PA in 2 expectorated sputum or throat swab cultures within 12 months prior to Visit 1; one of the previous PA-positive cultures must be within 3 months prior to Visit 1
- Patients must be able to provide written informed consent prior to any study related procedures
- FEV1 greater than or equal to 25% and less than or equal to 75% predicted at Visit 1
Key exclusion criteria (19 listed)
- Current use of oral corticosteroids in doses exceeding the equivalent of 10 mg prednisone a day or 20 mg prednisone every other day
- History of sputum or throat swab culture yielding B. cepacia in the previous 2 years
- Current requirement for daily continuous oxygen supplementation or requirement for more than 2 L/minute at night
- Administration of any investigational drug or device within 28 days of Visit 1 or within 6 half-lives of the investigational drug (whichever is longer)
- Known local or systemic hypersensitivity to monobactam antibiotics
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 |
|---|---|---|
| FTI, AZLIEvaluation of 2 dosage combinations of fosfomycin and tobramycin inhaled twice daily for 28 days following 28 days of inhaled AZLI (Aztreonam Lysine for Inhalation) | Drug | FTI 160mg/40mg BID, FTI 80mg/20mg BID |
| Placebo, AZLIVolume-matched placebo inhaled twice daily for 28 days following 28 days of AZLI (Aztreonam Lysine for Inhalation). | Drug | Placebo A BID, Placebo B BID |
Primary outcome
Relative change in lung function from baseline at Day 28.
Time frame: 28 Days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Dec 2013.
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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.
Bruce Trapnell, MD principal investigator
Children's Hospital Medical Center, Cincinnati
36 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing FTI, AZLI in people with Cystic Fibrosis, run by Gilead Sciences at 36 sites in United States.
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 12 inclusion and 19 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 27 Dec 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 36 sites in United States. 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. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Relative change in lung function from baseline at Day 28. (time frame: 28 Days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 27 Dec 2013.