Clinical trials: Your questions answered

Promising new treatments for many muscle and nerve conditions have been developed in recent years, promising enough in fact, for pharmaceutical companies and other funding bodies to invest in expensive clinical trials. On average only about one fifth of treatments that go to clinical trial are proven to be safe and effective, so the more trials that start, the closer we will be to finding treatments. Here we answer some of the questions you might have about clinical trials.

What is a clinical trial?
A clinical trial is a carefully controlled study designed to examine the safety and/or effectiveness of drugs, devices, treatments or preventive measures in humans. Clinical trials follow strict guidelines to ensure that the testing is completed as quickly and safely as possible and accurately answers the questions being asked.

What are the phases of a clinical trial?
Phase 1 is usually quite small and almost always designed purely to assess the safety of the new treatment and how well it’s tolerated. Often phase I studies recruit healthy volunteers to take part rather than patients. Approximately 70 percent of new medical treatments pass Phase 1 testing stage.

Phase 2 tests the effectiveness of a treatment on a larger number of patients. Participants are typically divided into groups and the benefit of the drug compared to a placebo. Usually the patients don’t know whether they have been given the real drug or the placebo. The trial is then known as a ‘blinded study’. One-third of drugs that enter clinical testing successfully complete phase II and progress to larger-scale phase III studies. Phase 2 trials are sometimes divided into phase 2a and phase 2b.

Phase 2a is specifically designed to determine the best dose of the drug.

Phase 2b is specifically designed to study how well the drug works at the dose determined in the phase 2a study.

Phase 3 involves extensive testing to assess safety, efficacy and dosage levels in a large group of patients. This step can take two to five years. About 80 percent of drugs that enter Phase 3 will successfully complete this stage.

Phase 4 evaluates the long term risks and benefits of the drug once it’s available on the market.

Why participate in a clinical trial?
One common reason is to benefit from new research developments before they become more widely available. You should keep in mind that although the start of a clinical trial is a very promising sign, it isn’t a guarantee for a treatment and during the trial you may receive a placebo rather than the new drug or treatment. However, you might still get some satisfaction in knowing that you have helped moved the drug development to the next stage even if you haven’t directly benefited.

Another advantage is that people taking part in clinical trials are followed up even more carefully than usual, even after the trial has finished. This close attention could result in better management of the condition.

What are the risks of taking part in a trial?
Procedures could be painful, for example injections and biopsies and, of course, there is always the risk of an unwanted or unexpected negative reaction to the treatment. Trials also often involve multiple and frequent visits to hospital. This is obviously not always easy or practical. Participants in a trial should keep in mind that the treatment they receive might not provide any direct benefit for them. Therefore, it is very important to discuss what is involved in detail with the trial nurse or doctor before giving consent to take part.

Who can participate in a clinical trial?
All clinical trials have guidelines about who can take part. The factors that allow someone to participate in a clinical trial are called ‘inclusion criteria’ and those that disallow someone from participating are called ‘exclusion criteria’. These criteria are based on such factors as age, gender, the type and stage of a disease, previous treatment history, and other medical conditions.

Inclusion and exclusion criteria are not used to reject people personally, instead they:

  • Keep the participants safe, for example another underlying condition could make participation in the trial dangerous
  • Increase the reliability of the results and therefore get the treatment to market as quickly as possible so that the wider population can benefit. If the trial participants are very different at the beginning of a trial it is difficult for the researchers to interpret the results because they don’t know if the reason one patient responded to treatment and another didn’t is due to the drug or differences in their condition to begin with. This is especially helpful in the early phases of a clinical trial when there are few participants

In most circumstances, people who wish to participate in a clinical trial will find it easier if they live relatively near the team of people who are conducting the research, because they need to be monitored frequently. The clinical trial organisers will usually reimburse travel costs (within reason).

What do I do if I want to participate in a clinical trial?
Join patient registries if they are available for your condition. Patient registries are databases that contain information about patients with a particular condition. The registries are then used by clinical trial organisers to contact suitable trial participants and invite them to take part. Find out more on the MDA website. Talk to your doctor, he or she might know of trials that may be of interest to you. Finally, you can also directly contact the centre involved in the clinical study. They will get in touch with your local doctor whose involvement is essential.

Where can I find out about trials for my condition?
Two useful websites are:

However the information provided in these trial summaries is not always easy to understand so you may wish to discuss them with a health professional.

The Muscular Dystrophy Campaign in the UK has translated many trial summaries into lay language

The Australasian Neuromuscular Network also has a list of research projects calling for patient participation. These are mostly laboratory based projects which are studying the DNA of patients with certain symptoms who do not have a genetic diagnosis.

What are observational studies?
Often when searching databases such as those above, you will come across ‘observational studies’. No new treatments or drugs are given in these studies, instead a group of patients is observed to learn more about the condition. This can help with planning the provision of services for patients. Observational studies can also uncover groups of patients that are doing better or worse than others and try to figure out why. For example are those patients who are receiving more physiotherapy able to walk further? This could provide evidence to get this service provided more widely. Or is there a genetic factor that makes the condition worse for some people? Is there anything that can be done for these patients?

Importantly, observational studies are an essential step to put the infrastructure in place for future clinical trials of new treatments. Often observational studies are geared towards finding out the best way to measure the progress of a condition, which could then be used in future trials to measure if a treatment is working. Data gathered during observational trials also helps trial organisers to design many other aspects of the trial protocol.

What is the MDA doing to support clinical trials?
MDA is keen to ensure that the MD community of Victoria doesn’t miss out on clinical trials of new potential treatments. Since 2007 MDA has provided funding to the Cooperative International Neuromuscular Research Group (CINRG) Centre at the Royal Children’s Hospital. CINRG is an international network of 20 clinical trial sites in 10 countries. Membership of this network allows patients in Melbourne to join large international studies that are coordinated by CINRG. More information about CINRG.

Importantly, the CINRG funding allowed the employment of an MDA Neuromuscular Coordinator (Daniella Villano). Besides her role running the clinic at the Royal Children’s Hospital, she coordinates the enrolment of patients into the clinical trials currently underway at the hospital. Without Dani these clinical trials would not be possible.