What is a Clinical Trial?

Clinical trials are research studies that explore whether a medical strategy, treatment, or device is safe and effective for humans. Clinical trials produce the best data available for health care decision making.

The purpose of clinical trials is research, so the studies follow strict scientific standards. These standards protect patients and help produce reliable study results.

The treatment being investigated in a clinical trial can be a medicinal product, a procedure, a device or another type of therapeutic intervention. 

Clinical trials are an essential part of the process of evidenced based practice and can help guide treatment decisions for both health care professionals and patients.

Clinical trials are an important part of the pathway by which new medicinal products can obtain a licence from MHRA and become available for use as a new treatment in patients.

What are the stages of a clinical trial?

Phase

Purpose

Time

Preclinical

Assess safety and biological activity in the laboratory and in animal models.

3- 6 years

Phase 1

Determine which dose is safe and how often he treatment should be given via which route. Few particpants numbers

1-2 years

Phase 2

Evaluate effectiveness of the treatment with larger numbers of parctipants and access to side effects and tolerance.

2-4 years

Phase 3

Determine whether the new treatment works better than an alternative current standard treatment.

5-8 years

Post Clinical Trial review

Follow participants still taking the treatment to monitor side effects and

2-5 years

How do you find out about Duchenne clinical trial opportunities?

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.

Our mission through the DMD Hub is to ensure that all patients with DMD, including children and adults, have the opportunity to access clinical research opportunities.

Questions to ask before joining a clinical trial:

Each clinical trial is unique, with its own possible benefits and risks. Before you decide to take part in one, you may want answers to these questions. Some people take notes, record discussions, or bring a friend with them to help recall the answers and think of other questions:

  • 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?

  • Will the researchers work with my neuromuscular doctor? Who will be in charge of my care?

  • Who will I contact if I have problems, questions, or concerns?

  • What are my other options (standard treatments, other studies)? What are their pros and cons?

  • What were the results in earlier studies of this treatment? How likely are they to apply to me?

  • What kinds of treatments and tests would I need to have in this study? How often are they done?

  • How much time or travel is evolved?

  • How could the study treatment affect my daily life?

  • What side effects might I expect from the study treatment? Are there other risks? (Keep in mind that there can also be side effects from standard treatments and from the disease itself.)

  • How will we know or expect any indirection that the treatment is working?

  • How long will my son be in the research facility hospital for? Will there be overnight stays? 

  • Will I still be seeing my regular neuromuscular doctor? Who will be in charge of my care during the study?

  • Will my family have to pay for anything? Will travel and accommodation cost be paid up front or claimed back for? 

  • If I am harmed as a result of the research, what treatment will I be entitled to?

  • How long will I be in the study? How long will the study last?

  • Are there reasons I would be removed from the study? Are there reasons the study might be stopped early?

  • Is long-term follow-up care part of the study? What would it involve?

  • If the treatment is working for me, can I keep getting it even after the study ends?

  • Can I talk to other patients already taking part in the study?

  • Will I be able to find out about the results of the study?

  • How long do I have to make this decision?

  • Is there access to a phycologist to support the decision making process before and after consenting to this study?

  • Is there a play specialist available?

  • Can my son see the MRI scanner and any other assessments he may do during screening?

  • Is there a school teachers available to help my son with school work on long visits?

  • Is there a muscle biopsy in this study or the next phase of this study?

  • Is there any finical support for parents missing work or sibling care cost?

  • Is your child needle phobic?

  • Will my son require a central access like a port if his veins are unable to access?

  • Will we have access to my child’s results on the study?

You might find it helpful to include trusted friends and family members in your decision making process. They might ask questions you hadn’t thought of and can help you make sure that you’re making a decision that’s right for you. Every family is unique and will cope in different ways when participating in a clinical trial. Also, getting a second opinion from a doctor who’s not part of the study may help you decide if a certain study is the best one for you.​

Would you like someone to talk to?

Naomi Litchfield is our Director of Patient Engagement. Before joining Duchenne UK, she was a Senior Research Nurse running trials for Duchenne Muscular Dystrophy at Great Ormond Street Hospital. 

You can contact her between 9am and 5pm:  07398031662 or  02030967496
Alternatively please email: [email protected]