Learn More About this Trial – From a Kid’s Point of View

Duchenne muscular dystrophy is called DMD for short and makes the muscles of young kids (mostly boys) weak. Scientists have made a drug that they think will help kids with DMD from getting weak muscles. This drug is called the study drug.

In order to see if this drug works, the scientists need to study the drug. When drugs are tested in people it’s called a clinical trial. This clinical trial will be testing this drug scientists are making in kids with DMD.

Do you want to be in this trial?

To be in the trial, you will first need to have some tests done. These will be done at your first visit to the doctor running the trial. These tests will help the doctor to understand if you are a good fit for this clinical trial.

You may be asked to read a document called the Assent Form. The Assent Form is a document that tells you, in an easy way, what the trial is about.

Click here to view the Assent Form for children ages 6 to 10

Click here to view the Assent Form for children ages 11 to 15

How long is this trial?

You will be in the trial for two years.

How often do I need to go to the doctor’s for trial visits?

After you start the trial, you will need to come back every month.

How is the study drug given to me?

The study drug is a fluid that is given to you through a needle that will be put in your arm. Before the needle is put in your arm, the nurse will put some medicine on your arm so you won’t be able to feel the needle. It will take 2 hours for the nurse to finish giving you the study drug. During that time the nurse will make sure you are comfortable, and you can usually watch a movie or listen to music.

The cartoons below are provided to help you understand some tests the doctors may do:

Please click on the pictures to learn more.