What Is A Trial?

In order for a potential treatment to become an actual medicine that your doctor can prescribe, it has to go through a series of tests known as clinical trials. A clinical trial is a type of medical research that compares one treatment with another, or with a placebo. It may involve patients or healthy people, or both. A trial finds out if treatments are safe, if they have any side effects and are better than what’s available at the moment. There are four possible stages of a trial, before the treatment can be approved as a drug.

What are the stages of a clinical trial?

Phase One Trial

Phase one trials test the basic safety of a new drug or treatment. A small number of people, usually healthy volunteers, and in the case of DMD research, adults, are given the medicine. Researchers test for side effects and work out what the right dose might be for treatment. This will be the first time the medicine has been tested on humans.

Phase Two Trial

Phase two trials test the new medicine on a larger group of people who are ill, this time this will usually be boys with Duchenne.

Phase Three Trial

If a drug passes phases one and two they can then be tested on a larger group. This will test the medicine existing treatment or a placebo to see if it's better and if it has important side effects. Phase three trials often last a year or more.

Phase Four Trial

At the end of the trial process results are sent, in the UK, to the Medicines and Healthcare Products Regulatory Agency (MHRA). The MHRA decides whether to allow the company making the medicine to market it. A similar process occurs in the USA, where the Food & Drug Administration (FDA) decides which potential medicines are approved.

How Do You Get Onto A Trial?

Your consultant may be able to advise you on upcoming trials. If not, you can search to see if there is a trial appropriate for your child. For this you will need to know as much as possible about how Duchenne affects them: For instance, what is their mutation? Some trials target a particular mutation (ie the nonsense gene, a gene duplication or a mutation of particular exon) others are for anyone with Duchenne, regardless of their mutation. 

Trials also have what’s known as “inclusion” and “exclusion” criteria. This means that there are certain things your child has to be able to do, or certain drugs they can or can’t be on, before they can be considered eligible for a trial. For instance, some trials require children to have been on a stable dose of steroids for 6 months at least. Other trials coming through, require steroid naive children, which means children can only take part if they have not yet started on steroids.

You can search for information on all clinical trials by visiting Clinicaltrials.gov. run by the National Institute of Health in the USA,  and searching for ‘Duchenne Muscular Dystrophy’.

The World Health Organisation's International Clinical Trials Registry Platform (ICTRP), collects information from different registers to provide a central database of clinical trials.

The UK Clinical Trials Gateway is also helpful.

Once you have found a trial that you think might be suitable, contact the organisation running the trial, and find out which sites will be running the trial.

Advantages Of Being In A Clinical Trial

Your child has the chance of benefiting from a new treatment

The doctors involved will monitor your son’s basic condition more closely

Your child is helping advance treatments which could help others.

Disadvantages Of Being In A Clinical Trial

The drug your child is testing might turn out not to work.

There may be side effects.

You and your child will have to attend multiple clinic visits.