This workshop is intended for patients and caregivers, along with advocates and providers, to better understand what to expect when participating in a clinical trial for gene therapy. View timestamps below.
Session 1: The Process from Setting up a Clinical Trial to Being a Participant
0:00 Intro
3:10 Current Trends in Clinical Trials—Kimberly Goodspeed, MD
18:44 How a Clinical Trial Is Setup and Run—Megan Waldrop, MD
32:27 Complexities of Eligibility: Why do we have to exclude patients?—Heather Lau, MD
52:45 What to Expect Before and During a Clinical Trial—Nicolas Abreu, MD
1:15:00 What to Expect After a Clinical Trial—Perry Shieh, MD, PhD, FAAN
1:39:48 With AAV and understanding that you only have one chance to be dosed, how do you feel about putting patients on studies that are first in human dose escalation, where you know the first dose is most likely to be the least efficacious and how do your patients feel about that?
1:47:16 In what ways for the clinical trial design process have you engaged stakeholders to help you co-create it so the end outcome is getting to things like travel reimbursement or a unique ways or enrolling the study?
1:55:30 What are you guys doing in your practice or research to help navigate some of the cultural barriers or you know as we try to broaden our scope of getting more people into clinical trials, maybe some of the misnomers of information out there, and have you guys seen a setback with the challenges of having the pandemic and a lot of misinformation out there?
1:59:00 What are some of the challenges experienced with long term follow-up with trials, circumstances where patients have been lost to follow-up and proactive strategies on how to avoid or handle that?
Session 2: Risks, Challenges, and Hopes
2:34:06 Caregiver Insights in Clinical Trial Participation—Emily McGinnis
2:44:01 Weighing the Risks for Participation in a Gene Therapy Clinical Trial—Diana Bharucha
3:02:32 Participant Challenges—Pat Furlong
3:12:35 Making the Decision—Julia Vitarello
3:30:49 How can patient advocacy groups support families who have participated in gene therapy clinical trials? Especially since we are thinking about that natural history is changing, our medicines are getting better but might still not be perfect, identifying those new issues that can creep up and how can advocacy groups play a role in that?
3:43:57 A question that often comes up is talking about gene therapies inclusive of RNA based therapies (CAR T) as a cure, and we will sometimes says it’s a 4 letter word that we don’t use, sometimes you say it and think maybe it could be but I think often we're thinking about what are the other treatments going to be come or need to be added on, and not to think about that in terms of a clinical trial but I am curious about the panels thoughts and how you navigate those discussions with your community partners in your groups of "is it a cure is it not a cure"? Is it a therapy, how do we talk about that?
