Homework 3
Study planning
You are really excited that a collaborator has come up with a possible new Alzheimer’s disease treatment drug. The drug is thought of as reducing progression among those with mild cognitive impairment. A meeting with FDA officials stated the following. First, the primary outcome has to be the memory test CDR sum of boxes and, second, the drug has to show a significant decrease in progression over a one year follow up. Otherwise, all other study parameters are available for debate. There’s a secondary outcome in the volume of the hippocampus that would be beneficial to test after the primary outcome. All tests need to be multiplicity corrected.
You have no preliminary data.
Do the following:
- Propose a statistical analysis plan.
- Perform study planning for a clinical trial to test the primary and hopefully secondary outcome.
- Subjects enrollment and will cost $3k for enrollment and $2k per subsequent visit.
- Adding an MRI for the secondary outcome will cost an additional $5k per visit.
- Power for the primary hypothesis must be 90%
- Type 1 error rate (multiplicity adjusted) must be 5%
- Maximum spending for enrollment, visit and visit MRIs is hard capped at one million dollars.
- Investigators are interested in whether controlling for baseline age impacts power. All subjects will be over 65. However, little else is known about their age distribution. They plan on testing the treatment effect using an ANCOVA test of the treatment effect on the change in primary outcome adjusting for age. Give a recommendation as to whether this strategy should be employed.
Write up your results in a no more than 2 page technical paper.
This project will be used as the foundation for one of your in-class presentations.