Cystic FibrosisPhase 2NCT00457821
Safety Study of Ivacaftor in Subjects With Cystic Fibrosis
A Phase 2 study testing Ivacaftor 25 mg/75 mg, Ivacaftor 75 mg/150 mg and Ivacaftor 150 mg or 250 mg in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 15 sites in 3 countries.
Trial summary
The purpose of this study was to evaluate the safety and tolerability of ivacaftor in patients with cystic fibrosis (CF) who were aged 18 years or older and have a G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. 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?
- Ivacaftor 25 mg/75 mg, Ivacaftor 75 mg/150 mg and Ivacaftor 150 mg or 250 mg
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 39 participants
- Where
- 15 sitesin 3 countries
- Study dates
- May 2007to Aug 2008
- Phase
- Phase 2
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Weighing at least 40 kg
- Confirmed diagnosis of cystic fibrosis (CF) and G551D mutation in at least 1 allele
- Forced expiratory volume in 1 second (FEV1) of at least 40% of predicted normal for age, gender, and height
- Willing to remain on stable medication regimen for the duration of study participation
- No significant clinical laboratory abnormalities, not pregnant, and willing to use at least 2 highly effective birth control methods during Part 1 and 1 highly effective birth control method during Part 2 of the study
- No clinically significant abnormalities that would have interfered with the study assessments, as judged by the investigator
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
- Ongoing acute respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 14 days 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
- History of abnormal renal function (creatinine clearance < 50 mL/min using Cockcroft-Gault equation)
- History of solid organ or hematological transplantation
Show all 9Show fewer
- Pregnant or breast-feeding (for women)
- Ongoing participation in another therapeutic clinical trial, or prior participation in an investigational drug study without appropriate washout
- Concomitant use of any inhibitors or inducers of cytochrome P450 3A4 (CYP3A4)
Trial locations and contacts
15 sites in 3 countries.
15 sites in 3 countries
United States13 sites
The Children's Hospital
Aurora, Colorado, 80045
Johns Hopkins Hospital
Baltimore, Maryland, 21287
University of Alabama Hospital
Birmingham, Alabama, 35211
Pulmonary and Critical Care Medicine, Massachusetts General Hospital
Boston, Massachusetts, 02114
Children's Hospital of Boston
Boston, Massachusetts, 02215
Cystic Fibrosis Pulmonary Research and Treatment Center, University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599
Rainbow Babies and Children's Hospital
Cleveland, Ohio, 44106
Roy J. and Lucille A. Carver College of Medicine, The University of Iowa
Iowa City, Iowa, 52242-1083
Division of Pulmonary, Allergy and Critical Care Medicine, University of Minnesota
Minneapolis, Minnesota, 55455
Stanford University Medical Center
Palo Alto, California, 34304
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104-4399
University of Pittsburgh
Pittsburgh, Pennsylvania, 15213
Pulmonary Critical Care, University of Washington
Seattle, Washington, 98195
Canada1 site
Division of Gastroenterology, Hepatology and Nutrition, The Hospital for Sick Children
Toronto, Ontario, M5G 1X8
Germany1 site
CF Clinic, Pediatric Pulmonology and Neonatology, Medical School of Hannover
Straße, Hannover, D-30625
About this study
Official title: A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Study of VX-770 to Evaluate Safety, Pharmacokinetics, and Biomarkers of CFTR Activity in Cystic Fibrosis (CF) Subjects With Genotype G551D
The purpose of this study was to evaluate the safety and tolerability of ivacaftor in patients with cystic fibrosis (CF) who were aged 18 years or older and have a 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 double-blind, placebo-controlled, cross-over, multiple dose study of up to 28 days of dosing, in subjects with cystic fibrosis (CF) who have a G551D-CTFR gene mutation. Enrollment of 39 subjects occurred at 15 centers in the US, Canada, and Germany.
The study was conducted in 2 parts:
- Part 1 consisted of Group A and Group B. Subjects in Group A (10 subjects) were randomized to receive 25 mg of ivacaftor every 12 hours [q12h] (4 subjects), 75 mg of ivacaftor q12h (4 subjects), or placebo (2 subjects) for 14 days. Following a 7- to 28-day washout period, subjects who received active study drug crossed over to the alternate dose strength of ivacaftor for an additional 14 days. Placebo subjects continued to receive placebo for an additional 14 days. Subjects in Group B (10 subjects) were randomized to receive 75 mg of ivacaftor q12h (4 subjects), 150 mg of ivacaftor q12h (4 subjects), or placebo (2 subjects) for 14 days. Following a 7- to 28-day washout period, the subjects who received active study drug crossed over to the alternate dose strength of ivacaftor for an additional 14 days. Placebo subjects continued to receive placebo for an additional 14 days.
- Part 2 consisted of Group C; these subjects did not participate in Part 1. Subjects were randomized to receive 150 mg of ivacaftor q12h (7 subjects), 250 mg of ivacaftor q12h (7 subjects), or placebo (4 subjects) for a total of 28 days. Ivacaftor doses studied in Part 2 were selected following an interim pharmacokinetic/pharmacodynamic (PK/PD) and statistical analyses of data from Part 1. The 2 doses selected for Part 2 were anticipated to enable better definition of the optimal therapeutic dose.
- Conditions
- Cystic Fibrosis
- Being tested
- Ivacaftor 25 mg/75 mg, Ivacaftor 75 mg/150 mg, Ivacaftor 150 mg or 250 mg, Placebo
- Keywords
- G551D mutation, Fibrosis, Pancreatic Diseases, Digestive System Diseases, Lung Diseases, Respiratory Tract Diseases, Genetic Diseases, Inborn, Infant, Newborn, Diseases, Pathologic Processes
- Medical subject headings
- Cystic Fibrosis, Fibrosis, Pancreatic Diseases, Digestive System Diseases, Lung Diseases, Respiratory Tract Diseases, Genetic Diseases, Inborn, Infant, Newborn, Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ivacaftor 25 mg/75 mgDrug
Also called VX-770
25 mg or 75 mg q12h for a total of 28 days (Part 1)
Ivacaftor 75 mg/150 mgDrug
Also called VX-770
75 mg or 150 mg q12h for a total of 28 days (Part 1)
Ivacaftor 150 mg or 250 mgDrug
Also called VX-770
150 mg or 250 mg of ivacaftor q12h for 28 days (Part 2)
PlaceboDrug
Given q12h for 28 days each in Part 1 and Part 2 of the study
| Study group | Role | What they receive |
|---|---|---|
| Ivacaftor Group A | Experimental | Subjects in Part 1 who first received 25 mg or 75 mg of ivacaftor every 12 hours (q12h) for 14 days, then crossed over to receive the alternate dose for another 14 days. Receives: Drug: Ivacaftor 25 mg/75 mg |
| Ivacaftor Group B | Experimental | Subjects in Part 1 who first received 75 mg or 150 mg of ivacaftor q12h for 14 days then crossed over to receive the alternate dose for another 14 days. Receives: Drug: Ivacaftor 75 mg/150 mg |
| Ivacaftor Group C | Experimental | Subjects in Part 2 who received 150 mg or 250 mg of ivacaftor q12h for 28 days. Receives: Drug: Ivacaftor 150 mg or 250 mg |
| Placebo | Placebo comparator | Subjects who received placebo in Part 1 and subjects who received placebo in Part 2. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Subjects With Adverse Events (Combined Part 1 and Part 2)
Adverse event data were collected up to the follow-up visit (5 to 9 days after last dose of study drug). Serious adverse events that were ongoing at the follow-up visit were followed until the event resolved, returned to baseline, or was determined to be a stable or chronic condition.
Measured: Baseline to Follow-up
Number of Adverse Events (Combined Part 1 and Part 2)
Adverse event data were collected up to the follow-up visit (5 to 9 days after last dose of study drug). Serious adverse events that were ongoing at the follow-up visit were followed until the event resolved, returned to baseline, or was determined to be a stable or chronic condition.
Measured: Baseline to Follow-up
Secondary outcomes (4)
Change From Baseline in Nasal Potential Difference (Combined Part 1 and Part 2)
The transepithelial nasal potential difference (NPD) is a direct measure of transepithelial ion transport. NPD under conditions of zero chloride concentration perfusion solution in the presence of isoproterenol was of primary interest.
Measured: 14 days and 28 days
Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second [FEV1] (Combined Part 1 and Part 2)
Spirometry is a standardized assessment to evaluate lung function that is the most widely used endpoint in cystic fibrosis studies.
Relative change reflects the percent change from the baseline values [100% * (X-Y)/Y], where X and Y are post-baseline and baseline values, respectively.
Measured: 14 days and 28 days
Change From Baseline in the Cystic Fibrosis Questionnaire-Revised (CFQ-R) Score (Part 2 Only)(Respiratory Domain Score)
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: 14 days and 28 days
Change From Baseline in Maximum Sweat Chloride Concentration (Combined Part 1 and Part 2)
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: 14 days and 28 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Apr 2007 (estimated)
- Study startMay 2007
- Primary completionAug 2008
- Study completionAug 2008
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 (2)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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 39 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Cystic Fibrosis Foundation
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, Study director, Vertex Pharmaceuticals Incorporated
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00457821
- Sponsor's protocol ID
- VX06-770-101
- Status last verified
- Oct 2012
- First submitted
- 5 Apr 2007
- Last updated
- 5 Oct 2012
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Accurso FJ, Rowe SM, Clancy JP, Boyle MP, Dunitz JM, Durie PR, Sagel SD, Hornick DB, Konstan MW, Donaldson SH, Moss RB, Pilewski JM, Rubenstein RC, Uluer AZ, Aitken ML, Freedman SD, Rose LM, Mayer-Hamblett N, Dong Q, Zha J, Stone AJ, Olson ER, Ordonez CL, Campbell PW, Ashlock MA, Ramsey BW. Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation. N Engl J Med. 2010 Nov 18;363(21):1991-2003. doi: 10.1056/NEJMoa0909825.
PubMed 21083385Accurso FJ, Van Goor F, Zha J, Stone AJ, Dong Q, Ordonez CL, Rowe SM, Clancy JP, Konstan MW, Hoch HE, Heltshe SL, Ramsey BW, Campbell PW, Ashlock MA. Sweat chloride as a biomarker of CFTR activity: proof of concept and ivacaftor clinical trial data. J Cyst Fibros. 2014 Mar;13(2):139-47. doi: 10.1016/j.jcf.2013.09.007.
PubMed 24660233
Study at a glance
From the registry record, last updated by the sponsor on 5 Oct 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Study of VX-770 to Evaluate Safety, Pharmacokinetics, and Biomarkers of CFTR Activity in Cystic Fibrosis (CF) Subjects With Genotype G551D
- ClinicalTrials.gov ID
- NCT00457821
- Acronym / sponsor ID
- VX06-770-101
- Conditions
- Cystic Fibrosis
- Keywords
- G551D mutation, Fibrosis, Pancreatic Diseases, Digestive System Diseases, Lung Diseases, Respiratory Tract Diseases, Genetic Diseases, Inborn, Infant, Newborn, Diseases
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 39 participants (actual)
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- Collaborators
- Cystic Fibrosis Foundation
- Study start
- May 2007
- Primary completion
- Aug 2008 (actual)
- Study completion
- Aug 2008 (actual)
- First posted
- 9 Apr 2007 (estimated)
- Results
- Posted 5 Oct 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 safety and tolerability of ivacaftor in patients with cystic fibrosis (CF) who were aged 18 years or older and have a 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 double-blind, placebo-controlled, cross-over, multiple dose study of up to 28 days of dosing, in subjects with cystic fibrosis (CF) who have a G551D-CTFR gene mutation. Enrollment of 39 subjects occurred at 15 centers in the US, Canada, and Germany.
The study was conducted in 2 parts:
- Part 1 consisted of Group A and Group B. Subjects in Group A (10 subjects) were randomized to receive 25 mg of ivacaftor every 12 hours [q12h] (4 subjects), 75 mg of ivacaftor q12h (4 subjects), or placebo (2 subjects) for 14 days. Following a 7- to 28-day washout period, subjects who received active study drug crossed over to the alternate dose strength of ivacaftor for an additional 14 days. Placebo subjects continued to receive placebo for an additional 14 days. Subjects in Group B (10 subjects) were randomized to receive 75 mg of ivacaftor q12h (4 subjects), 150 mg of ivacaftor q12h (4 subjects), or placebo (2 subjects) for 14 days. Following a 7- to 28-day washout period, the subjects who received active study drug crossed over to the alternate dose strength of ivacaftor for an additional 14 days. Placebo subjects continued to receive placebo for an additional 14 days.
- Part 2 consisted of Group C; these subjects did not participate in Part 1. Subjects were randomized to receive 150 mg of ivacaftor q12h (7 subjects), 250 mg of ivacaftor q12h (7 subjects), or placebo (4 subjects) for a total of 28 days. Ivacaftor doses studied in Part 2 were selected following an interim pharmacokinetic/pharmacodynamic (PK/PD) and statistical analyses of data from Part 1. The 2 doses selected for Part 2 were anticipated to enable better definition of the optimal therapeutic dose.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Ivacaftor Group A | Experimental | Drug: Ivacaftor 25 mg/75 mgSubjects in Part 1 who first received 25 mg or 75 mg of ivacaftor every 12 hours (q12h) for 14 days, then crossed over to receive the alternate dose for another 14 days. |
| Ivacaftor Group B | Experimental | Drug: Ivacaftor 75 mg/150 mgSubjects in Part 1 who first received 75 mg or 150 mg of ivacaftor q12h for 14 days then crossed over to receive the alternate dose for another 14 days. |
| Ivacaftor Group C | Experimental | Drug: Ivacaftor 150 mg or 250 mgSubjects in Part 2 who received 150 mg or 250 mg of ivacaftor q12h for 28 days. |
| Placebo | Placebo comparator | Drug: PlaceboSubjects who received placebo in Part 1 and subjects who received placebo in Part 2. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Weighing at least 40 kg
- Confirmed diagnosis of cystic fibrosis (CF) and G551D mutation in at least 1 allele
- Forced expiratory volume in 1 second (FEV1) of at least 40% of predicted normal for age, gender, and height
- Willing to remain on stable medication regimen for the duration of study participation
- No significant clinical laboratory abnormalities, not pregnant, and willing to use at least 2 highly effective birth control methods during Part 1 and 1 highly effective birth control method during Part 2 of the study
Key exclusion criteria (9 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
- Ongoing acute respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 14 days 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
- History of abnormal renal function (creatinine clearance < 50 mL/min using Cockcroft-Gault equation)
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 |
|---|---|---|
| Ivacaftor 25 mg/75 mgAlso: VX-77025 mg or 75 mg q12h for a total of 28 days (Part 1) | Drug | Ivacaftor Group A |
| Ivacaftor 75 mg/150 mgAlso: VX-77075 mg or 150 mg q12h for a total of 28 days (Part 1) | Drug | Ivacaftor Group B |
| Ivacaftor 150 mg or 250 mgAlso: VX-770150 mg or 250 mg of ivacaftor q12h for 28 days (Part 2) | Drug | Ivacaftor Group C |
| PlaceboGiven q12h for 28 days each in Part 1 and Part 2 of the study | Drug | Placebo |
Primary outcome
Number of Subjects With Adverse Events (Combined Part 1 and Part 2)
Time frame: Baseline to Follow-up
Adverse event data were collected up to the follow-up visit (5 to 9 days after last dose of study drug). Serious adverse events that were ongoing at the follow-up visit were followed until the event resolved, returned to baseline, or was determined to be a stable or chronic condition.
Number of Adverse Events (Combined Part 1 and Part 2)
Time frame: Baseline to Follow-up
Adverse event data were collected up to the follow-up visit (5 to 9 days after last dose of study drug). Serious adverse events that were ongoing at the follow-up visit were followed until the event resolved, returned to baseline, or was determined to be a stable or chronic condition.
4 secondary outcomes
Change From Baseline in Nasal Potential Difference (Combined Part 1 and Part 2)
Time frame: 14 days and 28 days
Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second [FEV1] (Combined Part 1 and Part 2)
Time frame: 14 days and 28 days
Change From Baseline in the Cystic Fibrosis Questionnaire-Revised (CFQ-R) Score (Part 2 Only)(Respiratory Domain Score)
Time frame: 14 days and 28 days
Change From Baseline in Maximum Sweat Chloride Concentration (Combined Part 1 and Part 2)
Time frame: 14 days and 28 days
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Oct 2012.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 21083385Accurso FJ, Rowe SM, Clancy JP, Boyle MP, Dunitz JM, Durie PR, Sagel SD, Hornick DB, Konstan MW, Donaldson SH, Moss RB, Pilewski JM, Rubenstein RC, Uluer AZ, Aitken ML, Freedman SD, Rose LM, Mayer-Hamblett N, Dong Q, Zha J, Stone AJ, Olson ER, Ordonez CL, Campbell PW, Ashlock MA, Ramsey BW. Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation. N Engl J Med. 2010 Nov 18;363(21):1991-2003. doi: 10.1056/NEJMoa0909825.
- Derived from this study: PubMed 24660233Accurso FJ, Van Goor F, Zha J, Stone AJ, Dong Q, Ordonez CL, Rowe SM, Clancy JP, Konstan MW, Hoch HE, Heltshe SL, Ramsey BW, Campbell PW, Ashlock MA. Sweat chloride as a biomarker of CFTR activity: proof of concept and ivacaftor clinical trial data. J Cyst Fibros. 2014 Mar;13(2):139-47. doi: 10.1016/j.jcf.2013.09.007.
- 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.
Medical Monitor study director
Vertex Pharmaceuticals Incorporated
15 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Ivacaftor 25 mg/75 mg, Ivacaftor 75 mg/150 mg and Ivacaftor 150 mg or 250 mg in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 15 sites in 3 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 6 inclusion and 9 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 5 Oct 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in United States, Canada, Germany. 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?
Yes. Results are posted in the registry (first posted 5 Oct 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?
Number of Subjects With Adverse Events (Combined Part 1 and Part 2) (time frame: Baseline to Follow-up). The study lists 2 primary outcomes in all. 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 NCT00457821, which also lists 2 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 5 Oct 2012.