Trial Design
Clinical Trials: Inclusion and Exclusion criteria defined.
In this video, a Principal Investigator explains inclusion criteria and exclusion criteria. He explains who determines the criteria, defining both the role of the investigator and the sponsor. He acknowledges that sometimes individuals/families feel frustration with criteria and left out when they do not qualify for a trial.
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Clinical Trials: Inclusion and Exclusion criteria defined.
Clinical Trials: Limited slots - first come, first served and screened re criteria.
A Canavan mom on learning gene therapy was on the horizon and getting information from the company. “Information is being shared the way it needs to be shared.”
Balancing hope and realistic expectations in clinical trials.
Community input is valued but every trial design is unique
Clinical Trials: Efficacy and End Points defined. End points are indicators on the road to efficacy.