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.
|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|
To be considered successful, phase 2 and 3 clinical trials have to show effectiveness in treating the neuromuscular condition. Each trial will select a primary endpoint, an aspect of the neuromuscular condition that can be measured and compared. For example, one endpoint might be peak expiratory flow, the amount of air you can breathe out in one go. This shows the strength of an individual’s breathing muscles. In order to be considered effective, the treatment being studied must improve the endpoints being studied when compared to no treatment or a placebo. Often trials will look at multiple endpoints.
Sometimes people appear to benefit from a treatment that doesn’t work, simply because they think and hope it will work. This is known as the placebo effect. Clinical trials often give some of the participants a placebo, a substance or treatment (often a sugar tablet or saline injection) that doesn’t do anything. This is to make sure that the real treatment works better than the placebo effect.
Our work so far has focused on Duchenne. Following our recent rebrand we will soon be adding research resources for other neuromuscular conditions too.
The majority of trials for new treatments for Duchenne are restricted to children. This is due to a number of factors, such as:
Adults may not be able to be involved in many trials, but increasingly trials on respiratory and cardiac treatments are focusing on adults. Some companies may also try to expand their trials to older age groups as they get into the later stages of development.
Regulators such as the MHRA will usually only allow treatments to be used on the same age group as has been involved in the clinical trial. This is because there is a lack of evidence that the treatment will also work for adults. That means many treatments being developed will not be available to adults unless an adult trial is conducted.
This is why it is important that adults can be involved in clinical trials. Pathfinders is currently working with Duchenne UK to encourage pharmaceutical companies to include adults in clinical trials.
Unfortunately some muscle cells cannot be replaced once they are lost. Most treatments therefore aim to prevent muscle loss before it occurs. There is currently no treatment at the clinical trial stage that could replace lost muscle. We continue to hope that this will be developed in future. In the near future, treatments may be made available which can slow down the progression of Duchenne and perhaps even restore some strength in the short term, but will not fully restore what has been lost.
You can search for information on all DMD clinical trials by visiting the DMD Hub website: Clinical Trial Finder.
You can also visit Clinicaltrials.gov run by the National Institute of Health in the USA, and search 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.
If there is a trial that you think might be suitable, contact the organisation running the trial, and find out which sites will be running the trial.
The mission of Duchenne UK’s DMD Hub is to ensure that all patients with DMD, including children and adults, have the opportunity to access clinical research opportunities.
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: