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](https://i.ytimg.com/vi/BMNWxn-FV5E/maxresdefault.jpg)
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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![](https://i.ytimg.com/vi/BMNWxn-FV5E/hqdefault.jpg)
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
![](https://i.ytimg.com/vi/rMI48kZ7BxI/hqdefault.jpg)
Clinical Trials: Inclusion and Exclusion Criteria; Age and Ability of the child are often key factors
![](https://i.ytimg.com/vi/q8RezIMjWlM/hqdefault.jpg)
Clinical Trials: Phase 1 defined – focusing on safety, with less certainty about benefit. Phase 1 trials for rare disease are designed for some efficacy
![](https://i.ytimg.com/vi/93KVJ9TUyJg/hqdefault.jpg)
Clinical Trials: Inclusion and Exclusion criteria defined.
![](https://i.ytimg.com/vi/VP_yLrNsc3c/hqdefault.jpg)
Clinical Trials: The Role of the Principal Investigator, Sponsor, IRB and FDA.
![](https://i.ytimg.com/vi/37CwmjDZn-0/hqdefault.jpg)
Clinical Trials: Informed Consent defined. Parents and providers together making the best decision based on the limited information available
![](https://i.ytimg.com/vi/Cw9F4YlF1Ak/hqdefault.jpg)
Clinical Trials: Limited slots - first come, first served and screened re criteria.
![](https://i.ytimg.com/vi/yJuyRAq86QU/hqdefault.jpg)
Clinical Trials are designed to get to FDA approval ASAP
![](https://i.ytimg.com/vi/JqIgpQwEH6c/hqdefault.jpg)
Clinical Trials: Efficacy and End Points defined. End points are indicators on the road to efficacy.
![](https://i.ytimg.com/vi/Z6kOnHMTMHs/hqdefault.jpg)
Clinical Trials: With Gene Therapy, it is ONE and DONE
![](https://i.ytimg.com/vi/EAYMdH1uzGM/hqdefault.jpg)
Clinical Trials: Dose escalation, Phase by Phase
![](https://i.ytimg.com/vi/_iLEnoiyyVo/hqdefault.jpg)
Compassionate use is no substitute for the trial pathway
![](https://i.ytimg.com/vi/R-ICb57p6Vs/hqdefault.jpg)
Clinical Trials: Defining Compassionate Use, Expanded Access
![](https://i.ytimg.com/vi/eMzTuty9quY/hqdefault.jpg)
Clinical Trials: Timing is everything. Participate early or wait for Phase 3?
Made possible by a grant from:![Sponsor Logo](https://api.courageousparentsnetwork.org/app/uploads/2020/02/SANOFI_Genzyme_RVB.jpg)
![Sponsor Logo](https://api.courageousparentsnetwork.org/app/uploads/2020/02/SANOFI_Genzyme_RVB.jpg)