Terence Flotte, MD
Dean of the School of Medicine, UMass Medical School
Pediatrician and Gene Therapy Investigator
Clinical Trials: In the early phase, it’s about both gaining knowledge and trying to help. To figure it out, we need some families to step up and go first
In this video a Principal Investigator explains that clinical trials are about both research and therapy; their goal is to gain knowledge and help the participants. Trials are a process and at each step, researchers learn things. He stresses research needs families to step up and agree to be in the early phases; those families are trailblazers. He acknowledges that when trial outcomes are not met, participants often feel like the trial is a failure but stresses that at each step scientific knowledge is gained.
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Clinical Trials: In the early phase, it’s about both gaining knowledge and trying to help. To figure it out, we need some families to step up and go first
Clinical Trials: Inclusion and Exclusion Criteria; Age and Ability of the child are often key factors
Clinical Trials: Phase 1 defined – focusing on safety, with less certainty about benefit. Phase 1 trials for rare disease are designed for some efficacy
Clinical Trials: Inclusion and Exclusion criteria defined.
Clinical Trials: The Role of the Principal Investigator, Sponsor, IRB and FDA.
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