Clinical Trial Video Library
Sometimes the easiest way to understand how something works, especially a process as complicated as clinical trials, is to hear the experiences of experts and participants who have been on the journey. PPMD and The Duchenne Registry have compiled a series of videos to answer some of the questions you may have about the clinical trial process, as well as provide insight into what you or your child can expect.
If you have additional questions or concerns, please do not hesitate to reach out to one of our genetic counselors at The Duchenne Registry.
Please note that these videos were made before DuchenneConnect was renamed The Duchenne Registry.
Introduction
This first video provides you with basic information about clinical trials.
How do I get the information I need to evaluate a clinical trial?
Families discuss the strategies they have used and found effective as well as what types of questions to ask.
Why do we need a placebo arm for some trials and what if I don’t want to be on it?
This video explores the use of placebo arms for some clinical trials and what this means for people in clinical trials.
What are the benefits that I get for enrolling in a clinical trial?
Clinical trials are experiments that have their own risks and benefits specific to each trial. While benefits are not guaranteed in clinical trials, listen to these families and health care professionals discuss what they see as potential benefits to being in a clinical trial.
What are the risks of a clinical trial and how can I evaluate that for my child?
Clinical trials are experiments and each has their own benefits and risks. Each person and their family have to weigh the potential risks against the benefits. Families and experts discuss their experience around risks in clinical trials.
Managing Expectations: I know that it is a clinical trial but what is the possibility of getting a clinical benefit?
Managing expectations around what the potential is of receiving a benefit during a trial is important. While we all hope each trial succeeds, clinical trials are experiments and no benefit is guaranteed.
What are the inclusion/exclusion criteria and why are they so focused on ambulatory boys?
Listen as families and experts discuss inclusion/exclusion criteria and the importance as well as the hope that more people can be included in future clinical trials.
With so many choices of clinical trials available, how do I make the right decision?
With so many choices for clinical trials available, making the right decision is critical. Listen to families and experts weigh in on this process and ways to navigate the decision process.
How do the trial logistics fit into my family’s decision to enroll?
Trial logistics such as travel, how much time a trial will take, what tests are involved and how the trial will impact other family members are important to consider when deciding on whether to be in a clinical trial. Listen as families and experts discuss their experiences with trials and things to consider.
What are Natural History trials and what do we learn from them?
Learn more from families and professionals as they describe these trials, their experiences and the importance of natural history studies.
What does it mean when a clinical trial is halted or fails?
While this is a difficult process, listen to families and experts weigh in on their experience. Failures are hard but they do enable us to ask questions and learn from the experience.
What will the role of combination therapy be for the treatment of Duchenne?
Listen to this short video to hear parents and professionals discuss what this means for Duchenne.
Why is it important to have genetic testing done?
This short video reviews the reasons those with Duchenne and Becker should know their genetic mutation.
How can I get help with genetic testing?
Decode Duchenne is a free genetic testing program for patients with Duchenne and Becker muscular dystrophy. Learn more on our website.
Why is it important to be in The Duchenne Registry?
The Duchenne Registry (formerly DuchenneConnect) is an international registry for individuals with Duchenne and Becker muscular dystrophy, as well as female carriers.
