Clinical trials

Research into Duchenne muscular dystrophy has gathered pace over the last few years, with Action Duchenne supporting and funding a number of clinical trials. More can be found out about research in research basics.

There are a number of new investigational compounds and drugs entering the late stage of the clinical trial process, with the recent approval of translarna to treat Duchenne caused by nonsense mutations (around 13-15% of cases).

Clinical trials are an important part of medical research comparing the effects of a potential treatment with another treatment or placebo (drug with no effect). Clinical trials can either involve patients or healthy volunteers.

Find out more about  the recommendations and funding priorities of Action Duchenne for potential drug therapies in our Research strategy.

Your child can be signed up to the DMD registry to ensure they can be put forward for any suitable and relevant clinical trials.

Read the ‘Overview of clinical trial process’ presentation from the Action Duchenne International Conference.

Participants who partake in a clinical trial are allocated to a treatment group or placebo group (also known as a control group). Sometimes there can be more than two groups, with different groups given different drugs or doses of drugs compared to placebo. There are a number of stages in the clinical trial process.

Randomisation of participants in a clinical trial is important. Randomisation ensures differences observed between each group are due to the treatment being studied and not differences between participants.

Bias must be avoided during a clinical trial. Clinical trial studies are double-blinded, ensuring the assessor of the potential treatment and participant do not know which group they are in.

The clinical trial process can take up to 10 years before potential regulatory approval, however this time scale can be variable, particularly with rare disorders.

This process is vital to determine if potential treatments are well tolerated, safe and to highlight any side effects in patient groups or healthy individuals before drugs can be approved.

The European medical agency (EMA) is responsible for regulatory approval of new drugs within the European union (EU). As a result of the UK leaving the EU this is subject to change.

The medicines and healthcare products regulatory agency (MHRA) is responsible for enforcing European drug approval regulations within the UK, making sure drugs are safe and effective.

After a drug has been approved the national institute of clinical excellence (NICE) decides whether a drug should be made widely available on the NHS.

Read the ‘Access to medicines’ presentation from the Action Duchenne International Conference.

Endpoints in clinical trials are used to determine an appropriate goal of a clinical trial, for example increased survival or decreased disease severity.

Biomarkers allow for the identification of disease, this could be a small molecule or gene produced as a result of a disease such as Duchenne. Biomarkers are used to determine clinical trial endpoints.

There are three types of biomarkers:

Diagnostic biomarkers (telling you something is wrong).

Prognostic biomarkers (predicting disease course and/or severity).

Therapeutic monitoring biomarkers (can predict whether a therapy will work or is working).

Clinical trials aid in the discovery and development of new drugs to treat diseases such as Duchenne, allowing doctors and scientists to understand more about the condition and how to treat it.

It is important to note that not every clinical trial is successful, some potential treatments may not be better than current treatments. All research is valuable, aiding in the discovery of better treatments now and in the future.

Call us 020 7250 8240 to discuss your clinical trial options.

In order for new drugs to be approved there are a number of stages in the clinical trial process, this can take time.

Animal studies

• Animal models are used to conduct initial studies into the use of a potential treatment before studies are conducted in patient volunteers.
• Animal studies identify the effectiveness, tolerability and highlight any side effects of potential treatments to determine whether this can be transferred into patient volunteers.

Phase 1

• First trials conducted in human volunteers.
• Small number of volunteers recruited to partake in phase 1 trials.
• Correct dose of drug determined with tolerance, effectiveness and any potential side effects observed.
• Dose of drug increased until minor side effects observed, used to determine correct dosage.

Phase 2

• Increased number of volunteers partake in study with effects of potential drug treatment tested in the short-term.

Phase 3

• Drug must have passed phases 1 and 2 to progress into phase 3.
• Drug tested in increased number of volunteers.
• Drug compared to current treatments or placebo (drug with no effect).
• Side effects and drug effectiveness continually monitored.

Phase 4

• Drug tolerance, safety and side effects studied over a longer period of time in patient volunteers of study, with the risks and benefits of the drug being compared.
• Drug may be granted regulatory approval during or after phase 4.

Figure 1 – There are various phases to clinical trials to ensure new drug candidates and potential treatments are effective, well tolerated and safe for use in patient populations. If potential treatments are successful and pass through all phases they may reach the regulatory process and eventually be granted regulatory approval.