Should I Join a Trial?

Choosing whether to take part in a clinical trial is a personal decision. You should never feel pressured to take part.

Benefits of joining a clinical trial

Taking part in research can be very rewarding.

You may:

  • get access to treatments which would not be available to you otherwise
  • benefit from regular follow up meetings
  • understand DMD better
  • help advance medical research
  • help researchers develop new treatments for the benefit of future generations

Questions to consider

Each clinical trial is unique, with its own possible benefits and risks.

Before you decide to take part in one, you will meet with the doctors who are running it. They will tell you about the trial and answer your questions.

Some people take notes, record discussions, or bring a friend with them so they can remember the information they're given.

When you meet the doctors, you may want to ask them these questions:

TRIAL DESIGN

  • Why is this study being done?
  • What’s likely to happen if I decide to take part or decide not to take part in the study?
  • What were the results in earlier studies of this treatment? How likely are they to apply to my child or me?
  • What types of treatments and tests would my child or I need to have in this study? How often are they done?
  • How long will my child or I be in the research facility hospital for?  Will there be overnight stays?
  • How will we be able to tell if the treatment is working or not?
  • Will I be able to find out about the results of the study?
  • Is there a muscle biopsy in this study or the next phase of this study?
  • Will my child or I require a central access, like a port, if their/my veins are unable to be accessed?
  • Will we be able to see their/my results on the study?
  • Is long-term follow-up care part of the study? What would it involve?

HOW TRIALS ARE RUN

  • Will the researchers work with our neuromuscular doctor? Who will be in charge of our care?
  • How long will my child/I be in the study? How long will the study last?
  • Is there any financial support for parents or patients missing work or for the cost of childcare for siblings?
  • Is there access to a psychologist to support the decision-making process before and after consenting to this study?
  • Is there a school teacher available to help my child with school work on long visits?
  • If the treatment is working for my child or me, can they/I keep getting it even after the study ends?

SAFETY

  •  What side effects are possible from the trial treatment? Are there any other risks? (Keep in mind that there can also be side effects from standard treatments and from the disease itself.)
  • If my child or I are harmed because of the research, what treatment will they/I be entitled to?
  • What are the other options, including standard treatments and other studies? What are their benefits and risks?
  • Will my child/I still see their/my regular neuromuscular doctor? Who will be in charge of their/my care during the study?
  • Who will I contact if I have problems, questions, or concerns?
  • Are there any reasons my child/I could be removed from the study? Are there any reasons the study might be stopped early?

OTHER THINGS TO CONSIDER

  • How much time or travel is involved?
  • How could the study treatment affect my child's/my daily life?
  • Will my family have to pay for anything? Will travel and accommodation cost be paid upfront or claimed back for?
  • Is your child/you needle phobic?
  • How long do I have to make this decision?
  • Can I talk to other patients already taking part in the study?
  • Is there a play specialist available?
  • Can my child see the MRI scanner and any other assessments they may do during screening?

Please read About Clinical Trials to find out how to join a trial.

Am I eligible?

Anyone can potentially take part in a clinical trial, but suitability will depend on eligibility criteria for each specific trial. A screening will determine the suitability by checking health and medical history against the inclusion and exclusion criteria.
Eligibility criteria are usually based on characteristics such as:

Age
DMD
genotype/
mutation
Functional
abilities
Pre-existing
conditions

Being told that you are unable to take part in a trial because you are not eligible can be very dissapointing.

However, it is important to remember you may be eligible to participate in a different trial.

Clinical trials must have strict eligibility criteria in place to make sure the trial is conducted as safely and as accurately as possible.