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Understanding Clinical Trials in Duchenne

Hope is critical to survival. Hope helps us get through those times of darkness with the thought that there is something new on the horizon, a potential new destination. Hope enables us to focus on meeting challenges even in the midst of adversity. Hope allows us to see that there might be another path to achieve our goal and it allows us to look for alternative ways to solve our problems.

In Duchenne, while there are many oppressive obstacles, we celebrate the hope that clinical trials provide to our community. We wanted to share some of this hope with you by highlighting the current clinical trial landscape and show you resources available to you to explore clinical trials on your own.

The Duchenne clinical trial landscape


Let’s start by taking a peek at the general clinical trial landscape. Did you know that there are over 50 clinical trials going on in the US focused on Duchenne and Becker muscular dystrophy?

While having so many clinical trials is exciting it can also be overwhelming when you are trying to make a decision about if you want to be in a study and if so, which study is the best fit for you and your family.

New to learning about clinical trials?


If you are new to learning about clinical trials, we have some resources to help get you started. Our Exploring Clinical Trials section on PPMD's DuchenneConnect Registry is a good way to begin (under the Clinical Trials tab on the top menu bar).


Watch our short and Introductory Video (above) that highlights what others have said are important things to know when considering being in a clinical trial. The next two sections What is a Clinical Trial? and Types and Phases of Clinical Trials are designed to take a deeper dive into what a clinical trial is and define some of the key terms involved. 


Don’t miss our Clinical Trial Video Library! Here we have short videos devoted to topics such as evaluating clinical trials, learning about potential benefits and risks of being in a trial, managing expectations in a trial and more! These short videos are composed of interviews with families and professionals who have all been involved in clinical trials so listening to their stories will hopefully help you in your decision.

 

Tools to help in your decision


Once you have a general understanding of clinical trials you still need to decide IF you even want to think about participating in a clinical trial.

To help with this decision, download our interactive Clinical Trial Decision Guide (pictured below). There is a version for adults thinking about clinical trials and one for children. In addition, we have questions that you will want to make sure you ask your study team when you are thinking about specific clinical trials.

Finding the right clinical trial for you


Once you come to the decision that you would like to be in a trial, the decision around if a specific trial is a good fit for your family can be daunting. The chart below illustrates the various types and categories of clinical trials and research ongoing in Duchenne.

To find out more about a specific clinical trial, there are several different resources. First, you can download a copy of the 2017 PPMD Connect Conference Program Book which contains our Research FAQ’s on clinical trials that are ongoing, trials currently ongoing but not recruiting and pre-clinical work.

Presentations on many of these research studies were recorded at our 2017 Connect Conference and can be viewed online:

And finally another tool we have is through a partner, Antidote, which offers an additional way to search through clinical trials.

And don’t forget we are your partner in this!


PPMD and our DuchenneConnect Registry team are here for you and want to make sure you have all the information and support to navigate the clinical trial landscape.

If you have questions about clinical trials, please reach out to us and we will help you find the answers. Email coordinator@duchenneconnect.org or call 888-520-8675.  

We also encourage you to join DuchenneConnect as another tool in your belt. Many trial sponsors recruit through DuchenneConnect, so if you are registered in DuchenneConnect you will receive email notifications when you or your child appear to match criteria for a trial. We also send frequent research updated through newsletters and social media.
 

Register on DuchenneConnect or update your account today!

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