Cystic FibrosisPhase 3STRIVENCT00909532
Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older With the G551D Mutation
A Phase 3 study testing Ivacaftor in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 65 sites in 8 countries.
Trial summary
The purpose of this study was to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis aged 12 years and older who have the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Ivacaftor
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 167 participants
- Where
- 65 sitesin 8 countries
- Study dates
- Jun 2009to Nov 2012
- Phase
- Phase 3
- 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Confirmed diagnosis of cystic fibrosis (CF) and G551D mutation in at least 1 allele
- Forced expiratory volume in 1 second (FEV1) of 40% to 90% (inclusive) of predicted normal for age, gender, and height at Screening.
- No clinically significant abnormalities that would have interfered with the study assessments, as judged by the investigator
- Willing to use highly effective birth control methods during the study
Who cannot join
- History of any illness or condition that might confound the results of the study or pose an additional risk in administering study drug to the subject
- Acute respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 4 weeks of Day 1 of the study
- History of alcohol, medication or illicit drug abuse within one year prior to Day 1
- Abnormal liver function ≥ 3x the upper limit of normal
- Abnormal renal function at Screening
- History of solid organ or hematological transplantation
Show all 10Show fewer
- Pregnant, planning a pregnancy, breast-feeding, or unwilling to follow contraception requirements
- Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within 30 days prior to Screening
- Use of inhaled hypertonic saline treatment
- Concomitant use of any inhibitors or inducers of cytochrome P450 3A4 (CYP 3A4)
Trial locations and contacts
65 sites in 8 countries.
65 sites in 8 countries
United States39 sites
University of Michigan
Ann Arbor, Michigan, 48109
Emory Cystic Fibrosis Center
Atlanta, Georgia, 30322
Johns Hopkins University
Baltimore, Maryland, 21205
University of Alabama
Birmingham, Alabama, 35233-1711
St. Luke's CF Clinic
Boise, Idaho, 83712
Massachusetts General Hospital
Boston, Massachusetts, 02114
Children's Hospital Boston
Boston, Massachusetts, 02115
Women and Children's Hospital of Buffalo
Buffalo, New York, 14222
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599
University of Virginia
Charlottesville, Virginia, 22908
Children's Memorial Hospital
Chicago, Illinois, 60614
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Pediatric & Pulmonary Division, Rainbow Babies/Case Western
Cleveland, Ohio, 44106
Nationwide Children's Hospital
Columbus, Ohio, 43205
National Jewish Medical and Research Center
Denver, Colorado, 80206
Hershey Medical Center
Hershey, Pennsylvania, 17033
Indiana University
Indianapolis, Indiana, 46202
University of Iowa
Iowa City, Iowa, 52242
The Children's Mercy Hospital
Kansas City, Missouri, 64108
East Tennessee Children's Hospital
Knoxville, Tennessee, 37916
Monmouth Medical Center
Long Branch, New Jersey, 07740
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
Pulmonary, Allergy & Critical Care Medicine, University of Minnesota
Minneapolis, Minnesota, 55455
West Virginia University
Morgantown, West Virginia, 26506
Vanderbilt University Medical Center
Nashville, Tennessee, 37232-5735
Long Island Jewish Medical Center
New Hyde Park, New York, 11042
Kaiser Permanente Medical Care Program
Oakland, California, 94611
Adult Pulmonary/ CF, University of Nebraska Medical Center
Omaha, Nebraska, 68198-5300
Cystic Fibrosis Research Office, Stanford University
Palo Alto, California, 94304
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
Oregon Health & Sciences University
Portland, Oregon, 97239-3098
University of Utah
Salt Lake City, Utah, 84132
Rady Children's Hospital
San Diego, California, 92123-5070
Seattle Children's Hospital
Seattle, Washington, 98105
Division of Pulmonary and CCM, University of Washington
Seattle, Washington, 98195-6522
Washington University
St Louis, Missouri, 63110
SUNY Upstate Medical University
Syracuse, New York, 13210
Toledo Children's Hospital
Toledo, Ohio, 43606
Australia7 sites
The Prince Charles Hospital
Chermside, Queensland, 4032
Royal Children's Hospital Brisbane
Herston, Queensland, 4026
Lung Institute of Western Australia
Nedlands, Western Australia, 6009
Royal Children's Hospital Melbourne
Parkville, Victoria, 3052
Mater Adult Hospital
South Brisbane, Queensland, 4101
Princess Margaret Hospital for Children
Subiaco, Western Australia, 6008
The Children's Hospital Westmead
Westmead, New South Wales, 2145
Ireland5 sites
Cork University Hospital
Cork
Our Lady's Children's Hospital
Dublin, 12
The National Children's Hospital
Dublin, 24
St. Vincent's University Hospital
Dublin, 4
Beaumont Hospital
Dublin, 9
Canada4 sites
Queen Elizabeth II Health Sciences Centre
Halifax, Nova Scotia, B3H 3A7
Montreal Children's Hospital - MUHC
Montreal, Quebec, H3H 1P3
St. Michael's Hospital
Toronto, Ontario, M5B 1W8
CF Center, Hospital for Sick Children
Toronto, Ontario, M5G 1X8
Germany4 sites
Kinder- und Jugendklinik Universitätsklinikum Erlangen
Erlangen, 91054
Mukoviszidose-Zentrum am Klinikum der Friedrich-Schiller-Universität Jena, Klinik für Kinder- und Jugendmedizin
Jena, 07740
Klinikum der LMU München, Dr. von Haunersches Kinderspital (CHA)
Munich, 80337
Universitäts-Kinderklinik Würzburg
Würzburg, 97080
France3 sites
Hopital Cochin
Paris, 75014
Hopital Necker
Paris, 75015
Centre de Perharidy
Roscoff, 29684
United Kingdom2 sites
Belfast City Hospital
Belfast, Northern Ireland, BT9 7AB
Imperial College London
London, SW3 6LR
About this study
Official title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of VX-770 in Subjects With Cystic Fibrosis and the G551D Mutation
The purpose of this study was to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis aged 12 years and older who have the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Ivacaftor is a potent and selective CFTR potentiator of wild-type, G551D, F508del, and R117H forms of human CFTR protein. Potentiators are pharmacological agents that increase the chloride ion transport properties of the channel in the presence of cyclic AMP-dependent protein kinase A (PKA) activation.
Read the detailed description
This was a phase 3 study in subjects with cystic fibrosis (CF) age 12 years and older who have a G551D-CFTR mutation and percent predicted forced expiratory volumn in 1 second (FEV1) between 40% and 90%.
Based on in vitro studies and pharmacologic, pharmacokinetic (PK), and safety profiles, ivacaftor was selected for clinical development as a possible treatment for patients with CF. Patients with the G551D mutation were the targeted population for this study because ivacaftor is a potentiator of the gating function of the CFTR protein, and the most prevalent mutation with a gating defect in CF is the G551D mutation.
This study was designed to further evaluate the efficacy of ivacaftor in subjects with CF who have a G551D-CFTR gene mutation and to evaluate safety in this population over a longer period than previously studied.
- Conditions
- Cystic Fibrosis
- Being tested
- Ivacaftor, Placebo
- Keywords
- Fibrosis, Pancreatic Diseases, Lung Diseases, Respiratory Tract Diseases, Genetic Diseases, Inborn, Infant, Newborn, Diseases, Pathologic Processes
- Medical subject headings
- Cystic Fibrosis, Fibrosis, Pancreatic Diseases, Lung Diseases, Respiratory Tract Diseases, Genetic Diseases, Inborn, Infant, Newborn, Diseases, Pathologic Processes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IvacaftorDrug
Also called VX-770
150-mg tablets given orally q12h for up to 48 weeks
PlaceboDrug
Tablet given orally q12h for up to 48 weeks
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Subjects who received placebo every 12 hours (q12h) for up to 48 weeks. Receives: Drug: Placebo |
| 150 mg Ivacaftor q12h | Experimental | Subjects who received 150 mg of ivacaftor q12h for up to 48 weeks. Receives: Drug: Ivacaftor |
What is being measured
The primary outcome is the main question the study is designed to answer.
Absolute Mean Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 24
Spirometry (as measured by FEV1) is a standardized assessment to evaluate lung function that is the most widely used endpoint in cystic fibrosis studies.
Measured: baseline through 24 weeks
Secondary outcomes (5)
Absolute Mean Change From Baseline in Percent Predicted FEV1 Through Week 48
Spirometry (as measured by FEV1) is a standardized assessment to evaluate lung function that is the most widely used endpoint in cystic fibrosis studies.
Measured: baseline through 48 weeks
Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Score Through Week 24 and Week 48 (Respiratory Domain Score, Pooled)
The CFQ-R is a health-related quality of life measure for subjects with cystic fibrosis. Each domain is scored from 0 (worst) to 100 (best). A difference of at least 4 points in the respiratory domain score of the CFQ-R is considered a minimal clinically important difference (MCID).
Measured: baseline through 24 weeks and 48 weeks
Absolute Change From Baseline in Sweat Chloride Concentration Through Week 24 and Week 48
The sweat chloride (quantitative pilocarpine iontophoresis) test is a standard diagnostic tool for cystic fibrosis (CF), serving as an indicator of cystic fibrosis transmembrane conductance regulator (CFTR) activity.
Measured: baseline through 24 weeks and 48 weeks
Time-to-first Pulmonary Exacerbation Through Week 24 and Week 48
Pulmonary exacerbation was defined as a change in antibiotic therapy (intravenous, inhaled, or oral) for any 4 or more of signs/symptoms such as change in sputum; new or increased hemoptysis; increased cough or dyspnea; malaise, fatigue, or lethargy; temperature above 38 degrees C; anorexia or weight loss; sinus pain/tenderness and discharge; change in physical examination of the chest; decreased pulmonary function by 10%; and radiographic changes indicative of pulmonary infection.
Measured: baseline through 24 weeks and 48 weeks
Absolute Change From Baseline in Weight at Week 24 and Week 48
As malnutrition is common in patients with cystic fibrosis (CF) because of increased energy expenditures due to lung disease and fat malabsorption, body weight is an important clinical measure of nutritional status.
Measured: baseline to 24 weeks and 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 May 2009 (estimated)
- Study startJun 2009
- Primary completionJul 2010
- Study completionNov 2012
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 (4)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 Vertex Pharmaceuticals IncorporatedThe 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 167 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Cystic Fibrosis Foundation
- Responsible party
- Sponsor
- Investigators
- Bonnie W. Ramsey, MD, Principal investigator, Children's Hospital and Regional Medical Center, Seattle, Washington, USAStuart Elborn, MD, Principal investigator, Respiratory Medicine Group, Queen's University of Belfast, Belfast, Northern Ireland, UK
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00909532
- Sponsor's protocol ID
- VX08-770-102
- Acronym
- STRIVE
- Status last verified
- Jan 2013
- First submitted
- 26 May 2009
- Last updated
- 18 Jan 2013
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Ramsey BW, Davies J, McElvaney NG, Tullis E, Bell SC, Drevinek P, Griese M, McKone EF, Wainwright CE, Konstan MW, Moss R, Ratjen F, Sermet-Gaudelus I, Rowe SM, Dong Q, Rodriguez S, Yen K, Ordonez C, Elborn JS; VX08-770-102 Study Group. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med. 2011 Nov 3;365(18):1663-72. doi: 10.1056/NEJMoa1105185.
PubMed 22047557Flume PA, Wainwright CE, Elizabeth Tullis D, Rodriguez S, Niknian M, Higgins M, Davies JC, Wagener JS. Recovery of lung function following a pulmonary exacerbation in patients with cystic fibrosis and the G551D-CFTR mutation treated with ivacaftor. J Cyst Fibros. 2018 Jan;17(1):83-88. doi: 10.1016/j.jcf.2017.06.002. Epub 2017 Jun 24.
PubMed 28651844Solem CT, Vera-Llonch M, Liu S, Botteman M, Castiglione B. Impact of pulmonary exacerbations and lung function on generic health-related quality of life in patients with cystic fibrosis. Health Qual Life Outcomes. 2016 Apr 21;14:63. doi: 10.1186/s12955-016-0465-z.
PubMed 27097977Quittner A, Suthoff E, Rendas-Baum R, Bayliss MS, Sermet-Gaudelus I, Castiglione B, Vera-Llonch M. Effect of ivacaftor treatment in patients with cystic fibrosis and the G551D-CFTR mutation: patient-reported outcomes in the STRIVE randomized, controlled trial. Health Qual Life Outcomes. 2015 Jul 2;13:93. doi: 10.1186/s12955-015-0293-6.
PubMed 26135562
Study at a glance
From the registry record, last updated by the sponsor on 18 Jan 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of VX-770 in Subjects With Cystic Fibrosis and the G551D Mutation
- ClinicalTrials.gov ID
- NCT00909532
- Acronym / sponsor ID
- STRIVE / VX08-770-102
- Conditions
- Cystic Fibrosis
- Keywords
- Fibrosis, Pancreatic Diseases, Lung Diseases, Respiratory Tract Diseases, Genetic Diseases, Inborn, Infant, Newborn, Diseases, Pathologic Processes
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 167 participants (actual)
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- Collaborators
- Cystic Fibrosis Foundation
- Study start
- Jun 2009
- Primary completion
- Jul 2010 (actual)
- Study completion
- Nov 2012 (actual)
- First posted
- 28 May 2009 (estimated)
- Results
- Posted 21 Aug 2012
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 was to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis aged 12 years and older who have the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Ivacaftor is a potent and selective CFTR potentiator of wild-type, G551D, F508del, and R117H forms of human CFTR protein. Potentiators are pharmacological agents that increase the chloride ion transport properties of the channel in the presence of cyclic AMP-dependent protein kinase A (PKA) activation.
Detailed description
This was a phase 3 study in subjects with cystic fibrosis (CF) age 12 years and older who have a G551D-CFTR mutation and percent predicted forced expiratory volumn in 1 second (FEV1) between 40% and 90%.
Based on in vitro studies and pharmacologic, pharmacokinetic (PK), and safety profiles, ivacaftor was selected for clinical development as a possible treatment for patients with CF. Patients with the G551D mutation were the targeted population for this study because ivacaftor is a potentiator of the gating function of the CFTR protein, and the most prevalent mutation with a gating defect in CF is the G551D mutation.
This study was designed to further evaluate the efficacy of ivacaftor in subjects with CF who have a G551D-CFTR gene mutation and to evaluate safety in this population over a longer period than previously studied.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: PlaceboSubjects who received placebo every 12 hours (q12h) for up to 48 weeks. |
| 150 mg Ivacaftor q12h | Experimental | Drug: IvacaftorSubjects who received 150 mg of ivacaftor q12h for up to 48 weeks. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Confirmed diagnosis of cystic fibrosis (CF) and G551D mutation in at least 1 allele
- Forced expiratory volume in 1 second (FEV1) of 40% to 90% (inclusive) of predicted normal for age, gender, and height at Screening.
- No clinically significant abnormalities that would have interfered with the study assessments, as judged by the investigator
- Willing to use highly effective birth control methods during the study
Key exclusion criteria (10 listed)
- History of any illness or condition that might confound the results of the study or pose an additional risk in administering study drug to the subject
- Acute respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 4 weeks of Day 1 of the study
- History of alcohol, medication or illicit drug abuse within one year prior to Day 1
- Abnormal liver function ≥ 3x the upper limit of normal
- Abnormal renal function at Screening
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 |
|---|---|---|
| IvacaftorAlso: VX-770150-mg tablets given orally q12h for up to 48 weeks | Drug | 150 mg Ivacaftor q12h |
| PlaceboTablet given orally q12h for up to 48 weeks | Drug | Placebo |
Primary outcome
Absolute Mean Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 24
Time frame: baseline through 24 weeks
Spirometry (as measured by FEV1) is a standardized assessment to evaluate lung function that is the most widely used endpoint in cystic fibrosis studies.
5 secondary outcomes
Absolute Mean Change From Baseline in Percent Predicted FEV1 Through Week 48
Time frame: baseline through 48 weeks
Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Score Through Week 24 and Week 48 (Respiratory Domain Score, Pooled)
Time frame: baseline through 24 weeks and 48 weeks
Absolute Change From Baseline in Sweat Chloride Concentration Through Week 24 and Week 48
Time frame: baseline through 24 weeks and 48 weeks
Time-to-first Pulmonary Exacerbation Through Week 24 and Week 48
Time frame: baseline through 24 weeks and 48 weeks
Absolute Change From Baseline in Weight at Week 24 and Week 48
Time frame: baseline to 24 weeks and 48 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jan 2013.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 22047557Ramsey BW, Davies J, McElvaney NG, Tullis E, Bell SC, Drevinek P, Griese M, McKone EF, Wainwright CE, Konstan MW, Moss R, Ratjen F, Sermet-Gaudelus I, Rowe SM, Dong Q, Rodriguez S, Yen K, Ordonez C, Elborn JS; VX08-770-102 Study Group. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med. 2011 Nov 3;365(18):1663-72. doi: 10.1056/NEJMoa1105185.
- Derived from this study: PubMed 28651844Flume PA, Wainwright CE, Elizabeth Tullis D, Rodriguez S, Niknian M, Higgins M, Davies JC, Wagener JS. Recovery of lung function following a pulmonary exacerbation in patients with cystic fibrosis and the G551D-CFTR mutation treated with ivacaftor. J Cyst Fibros. 2018 Jan;17(1):83-88. doi: 10.1016/j.jcf.2017.06.002. Epub 2017 Jun 24.
- Derived from this study: PubMed 27097977Solem CT, Vera-Llonch M, Liu S, Botteman M, Castiglione B. Impact of pulmonary exacerbations and lung function on generic health-related quality of life in patients with cystic fibrosis. Health Qual Life Outcomes. 2016 Apr 21;14:63. doi: 10.1186/s12955-016-0465-z.
- Derived from this study: PubMed 26135562Quittner A, Suthoff E, Rendas-Baum R, Bayliss MS, Sermet-Gaudelus I, Castiglione B, Vera-Llonch M. Effect of ivacaftor treatment in patients with cystic fibrosis and the G551D-CFTR mutation: patient-reported outcomes in the STRIVE randomized, controlled trial. Health Qual Life Outcomes. 2015 Jul 2;13:93. doi: 10.1186/s12955-015-0293-6.
- Genetics Home ReferenceLink provided by the sponsor.
- Medline PlusLink provided by the sponsor.
- U.S. FDA ResourcesLink provided by the sponsor.
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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.
Bonnie W. Ramsey, MD principal investigator
Children's Hospital and Regional Medical Center, Seattle, Washington, USA
Stuart Elborn, MD principal investigator
Respiratory Medicine Group, Queen's University of Belfast, Belfast, Northern Ireland, UK
65 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Ivacaftor in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 65 sites in 8 countries.
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 4 inclusion and 10 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 18 Jan 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 65 sites in United States, Australia, Ireland, Canada, Germany and 3 more countries. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 21 Aug 2012). 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?
Absolute Mean Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 24 (time frame: baseline through 24 weeks). 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 NCT00909532, which also lists 4 linked publications.
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 18 Jan 2013.