and mitigate the symptoms if it does happen. you have two outcomes to test, frequency and severity. Each one of them demand a trial of its own.
This is a preventive procedure, therefore, at the initial stage, i would just test 'frequency" and forget about "severity" because it is too important to be a secondary. The reason is becuase the measurement of "frequency" is less prone to bias as compared with "severity". YOu have better chance to succeed in getting a grant and eventually the trial. This will be a very sound study. If it goes well, do another trial for "severity". To any of these studies, you can add a QoL as secondary.
This is important also because it determines how you select your patients. You would focus more on baseline frequency of attacks rather than "severity". Inclusion criterir will be different. your score may not be relavant now unless it serves as a diagnosing tool.
In clinical trial, don't try to over-achieve. The simple the better. One question, one trial. you can add secondaries, but your design of study should not be influenced by the addition of a secondary outcome. you should do another trial, if you think you have answers to a secondary question. Ask yourself, if I remove the secondary, will my design still be the same?
Then get a statistician to help with: statistical design (sample size, randomization ect), data collection, qualification, and analysis (statistical model), ect.
[quote]and mitigate the symptoms if it does happen. you have two outcomes to test, frequency and severity. Each one of them demand a trial of its own.
This is a preventive procedure, therefore, at the initial stage, i would just test 'frequency" and forget about "severity" because it is too important to be a secondary. The reason is becuase the measurement of "frequency" is less prone to bias as compared with "severity". YOu have better chance to succeed in getting a grant and eventually the trial. This will be a very sound study. If it goes well, do another trial for "severity". To any of these studies, you can add a QoL as secondary.
This is important also because it determines how you select your patients. You would focus more on baseline frequency of attacks rather than "severity". Inclusion criterir will be different. your score may not be relavant now unless it serves as a diagnosing tool.
Don't try to over-achieve. The simple the better. One question, one trial. you can add secondaries, but your design of study should not be influenced by the addition of a secondary outcome. Ask yourself, if I remove the secondary, will my design still be the same?
Then get a statistician to help with: statistical design (sample size, randomization ect), data collection, qualification, and analysis (statistical model), ect.
carefully think about your inclusion and exclusion criteria. it is another key to success.
I don't understand this:
the control will be simply not treated. Non-stressful and no medication and it has a long lasting effect.
REgarding this:
Phase III has to be a placebo design?
No. it has be RCT and preferably double blinded. The control can be a gold standard or placebo. Helsinky declaration stated that a gold standard is preferred, placebo is acceptable if justified. There are some flaws in this statement. The FDA abandoned this and stated that placebo is equally acceptable. Placebo is the standard for new drug testing now days, which makes sense. This is not relevant to you. Phased studies are most applicable for innovative interventions when its safety is unknown and/or cost prohibitive. This is not relevant to you.
hope this helps.