Theme: Clinical Trial: Trial Design
In this video, a mother of two sons with Duchenne Muscular Dystrophy describes how she made participating in a clinical trial more tolerable for her son by utilizing Child Life Specialists.
Theme: Clinical Trial: Trial Design
In this video, a mother of two sons with Duchenne Muscular Dystrophy offers guidance on deciding to participate in a clinical trial. She asks, “what are you looking for- curative treatments vrs mitigating symptoms?”; “What can your children stand – can they tolerate the tests required?”; “Can your family/marriage survive the lifestyle changes you may need to make to participate?”; “What if it is a trial with a placebo arm – can you handle your child getting the placebo?” She encourages asking lots of questions of the trial sponsor, doctors, and the disease community.
Theme: Clinical Trial: Trial Design
In this video, a mother of two sons with Duchenne Muscular Dystrophy talks about only one of her sons meeting the inclusion criteria and qualifying for the study as well as her naivety about how often timelines in studies change. She explains a double-blind placebo study and how in her son’s study, all the participants began receiving the drug once evidence proved safety and efficacy. She talks about the FDA process and the importance of knowing the sponsors plan for the period of time between study completion and approval of the treatment.
Theme: Clinical Trial: Trial Design
In this video, a mom discusses how participating in a clinical trial can be isolating. Not only do many trials contain restrictions about discussing your participation publicly but also because of some of the lifestyle changes that are necessary when participating.
Theme: Clinical Trial: Trial Design
This video is about Clinical Trials. A Study Coordinator explains that not all patients in a trial are treated at the same time; enrollment is staggered for a reason. She explains what the term cohort means and the process of dose escalation trials. She also talks about trials as a learning process.
Theme: Clinical Trial: Trial Design
In this video a patient advocate in biotechnology discusses a placebo arm in a clinical trial. She talks about questions to ask when considering a trial that includes a placebo arm including if the trial includes a rescue clause.
Theme: Clinical Trial: Trial Design
Theme: Clinical Trial: Trial Design
Theme: Clinical Trial: Trial Design
In this video a Principal Investigator explains the FDA’s definition of efficacy. He talks about the importance of endpoints in clinical trials as indicators on the road to efficacy. He describes the range of things that could be considered when determining endpoints.
Theme: Clinical Trial: Trial Design
Parents of two sons, one of whom has CLN2 (Batten disease) describe how his port goes into the ventricles in his brain and how the infusions are delivered. The side effects in his care are minimal which makes things easier. The port has to be replaced every couple of years.