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.

What clinical studies are currently recruiting participants in Melbourne?
Study of early signs of breathing problems during sleep in DMD
Official title: Study of early signs of breathing problems during sleep in DMD
Location: Royal Children’s Hospital, Parkville
Description: The Departments of Neurology and Respiratory Medicine at the Royal Children’s Hospital (RCH) are conducting a world- first study that aims to find a test, which can be done during the day, to determine when an individual with DMD is likely to start having breathing problems while they sleep. This will help to detect and treat sleep-related breathing problems as soon as they develop. During the study changes in lung function will be monitored during the day and an annual sleep study will be done at the RCH.
Who can take part?:This study is for boys with Duchenne MD between the ages of 10 and 16. Boys that are already using BiPAP to treat diagnosed breathing problems during sleep cannot take part. To discuss involvement in this study please click on the link below for the contact details of the study coordinator.

Further information about the breathing problems during sleep in DMD study

Ataluren for DMD
Official title: Phase 3 Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy (ID number NCT01826487)
Location: The Royal Children’s Hospital, Parkville
Description: Ataluren (previously called PTC124) is an oral drug that targets a specific type of mistake in the genetic code, called a nonsense mutation. This is when the DNA code has a stop signal part way through the gene. Ataluren encourages the cell to ignore this stop signal and continue to read the full set of instructions contained within the gene. Ten to 15 percent of boys with Duchenne muscular dystrophy have this type of mutation in the dystrophin gene.  A previous phase 2b clinical trial of ataluren showed that it may be able to slow down the rate of decline in walking ability and this larger phase 3 trial which will be conducted at 53 different centres around the world aims to confirm these results prior to applying for the drug to be approved for sale. The trial participants will take the drug or placebo three times per day for 48 weeks and attend clinic every 8 weeks for assessment.
Who can take part?: Briefly, boys between the ages of 6 and 16 with DMD caused by a nonsense mutation who can walk at least 150 meters unassisted in the 6-minute walk test can take part. They should have started taking corticosteroids at least six months ago with no major change in dose in the past three months. Some other medications or medical conditions may prevent participation.

Further information about the Ataluren trial.

Can leg casts help with contractures in DMD?
Official title: A study of below knee serial casting for calf contracture in ambulant boys with Duchenne muscular dystrophy.
Location: The Royal Children’s Hospital, Parkville
Description: Boys with DMD often experience muscle tightness, particularly in their calf muscles. This in turn causes them to walk on their toes, which makes standing and walking balance more precarious. Stretching programs and night splinting address this muscle tightness but often these are not sufficient to overcome the muscle shortening. This project aims to assess the effectiveness of applying a series of casts to the lower legs to gradually improve the child’s range of motion. This trial will assess whether short periods of serial casting will maintain calf muscle length, improve gait, and maintain or improve motor function. The boy’s will be assessed before and after serial casting and then at 3 monthly intervals for a twelve month period.
Who can take part?: This trial is for boys over 4 years of age who can walk unassisted for at least 75 metres and are able to complete a 6 minute walk test. They must already have calf contractures and a physiotherapist will need to examine the strength and range of movement of the legs to decide if participation in the trial is possible.

Further information about the leg casting for DMD study

Trial of Zledronic acid to improve bone strength in DMD
Official title:
Clinical trial of zoledronic acid in children and adolescents with Duchenne muscular dystrophy
Location: The Royal Children’s Hospital, Parkville
Corticosteroids (such as prednisolone) have been shown to assist in keeping children with DMD more mobile for longer but one of the side effects of this medication is thinning of the bones and increased risk of bone fracture. Zledronic acid has been shown to increase bone strength in conditions such as osteoporosis and this phase 3 trial aims to test whether it could also be of benefit for DMD. All of the trial participants will also take calcium supplements and vitamin D. Half of the participants will receive five injections of Zledronic acid over an 18 months period.
Who can take part? This trial is for boys between 6 and 16 years with confirmed DMD who are taking corticosteroids (prednisolone or deflazacort). Some other medications or medical conditions may prevent participation.

Further information about the Zledronic acid trial.

Nutritional supplement for DMD
Official title:
Nutriceuticals in Duchenne muscular dystrophy
Location: The Royal Children’s Hospital, Parkville
Description: This project seeks to explore the effectiveness of nutritional supplements in maintaining or improving walking ability in boys with Duchenne muscular dystrophy. The boys will drink a high protein shake, and take chewable multivitamin and fish oil tablets. For half of the 12 month trial creatine monohydrate (CrM) and glutamine will be added to the shake and the boys will take a capsule containing beta-hydroxy-beta-methylbutyrate (HMB). Levels of activity will be monitored using a step monitor worn on the ankle for 5 days at five time points (total of 25 days). Distance walked in six minutes will be measured five times during the 12 month trial. Body composition will be measured using a DXA scan, blood samples will be taken 5 times and questionnaires will be completed.
Who can take part?
This trial includes boys between the ages of 5 and 13 with a confirmed diagnosis of DMD who can walk at least 75 meters unassisted during a 6 minute walk test. Some other medications, allergies or medical conditions may prevent participation.

Further information nutritional supplement study.

Infantile FSHD observational study
Official title:
A multicenter collaborative study on the clinical features, expression profiling, and quality of life of infantile onset facioscapulohumeral muscular dystrophy (ACH0311)
FSHD is usually an adult onset condition but rarely it presents in a severe form in infants. The purpose of this study is to better understand this form of FSHD, including the signs and symptoms and their impact on quality of life. The study also aims to establish a standardised way of assessing the condition that may be used in future treatment studies. Finally, this study will explore biological variations in individuals that may affect their clinical symptoms. This study will include one study visit, with the possibility of a second study visit within 30 days if all assessments could not be completed initially.
Who can take part?
Participants of any age are eligible as long they had an onset of FSHD symptoms prior to age 11. Participants must have genetic confirmation of their FSHD diagnosis.

Further information about the infantile FSHD study.

DMD observational study
Official title:
Longitudinal Study of the Relationship Between Impairment, Activity Limitation, Participation and Quality of Life in Persons with Confirmed Duchenne Muscular Dystrophy (UCD0305)
The purpose of this study is to continue a world-wide, long-term study of people with Duchenne muscular dystrophy (DMD) that has been following over 300 families at universities and clinics around the world since 2005. This study, will take a detailed look at people’s physical abilities across all ages, medical problems they experience, and how they use healthcare services. It will also look at how families of people with DMD interact with their communities and at their quality of life. One of the first uses of this study will be to see how long-term steroid therapy affects daily activities of study volunteers who use the drug. A small blood sample at the first study visit will also be taken for analysis.  This will be used to look at how small changes in genetic makeup can affect how DMD progresses, and how an individual may respond to steroids. The researchers will also study whether certain proteins are present in the blood of people with DMD and not in healthy controls. These proteins are called ‘biomarkers’ and they might be used to tell researchers and doctors more about DMD and how it progresses.
Who can take part? Boys with DMD between ages 4-7 years.  The study will also recruit healthy boys and men aged 6 to 30 years old without Duchenne muscular dystrophy to complete strength and function evaluations.

Further information about the DMD observational study

The Triple F Study
Official title: Footwear, fatigue and falls in paediatric neuromuscular disease.
Description: The Children’s Neurosciences Centre at The Royal Children’s Hospital, are studying how different types of shoes affect the walking and the incidence of falls in children and adolescents who have Duchenne or Becker muscular dystrophy (DMD/BMD) or Charcot Marie Tooth disease (CMT).They are also interested in looking at whether walking for six minutes causes physical fatigue and changes in the way the children and adolescents walk. Two, one and a half to two hour assessments, 12 months apart will be done. You will be asked to bring in your best fitting “optimal” footwear and your less than ideal “suboptimal” footwear. How you walk in your optimal footwear will be compared to your suboptimal footwear and bare feet. After walking for six minutes in your optimal footwear you will be asked how tired your body feels. A six month falls diary will also be kept.
Who can take part? The study is seeking children and adolescents aged 4 to 18 years who are able to walk at least 75 metres and are diagnosed with Duchenne or Becker muscular dystrophy (DMD/BMD) or Charcot Marie Tooth disease (CMT). If you are interested, please contact Rachel Kennedy: (03) 9345 4287;

Bimagrumab/BYM338 for inclusion body myositis (IBM)
Official title: Randomized, Double-blind, Placebo-controlled, Dose-finding Study to Evaluate Efficacy, Safety, Tolerability of i.v. BYM338 at 52 Weeks on Physical Function, Muscle Strength, and Mobility in Sporadic Inclusion Body Myositis Patients
Description: Drug company Novartis is testing an investigational drug called BYM338 (also known as bimagrumab) for IBM. BYM338 is an antibody that blocks the action of proteins naturally in the body called ‘myostatin’ and ‘activin’ which inhibit muscle growth. This could result in increased muscle growth and strength. This phase 3 trial will be carried out at approximately 35 study locations worldwide. In Australia the study locations are in Melbourne (Caulfield), Sydney and Perth. Participants will be randomly divided into 4 groups to receive one of three dose levels of BYM338 or placebo by injection into a vein (intravenous infusion) and its effect on physical function, muscle strength, and mobility will be measured.
Who can take part? This trial is for men and women between 36 and 85 years of age diagnosed with sporadic inclusion body myositis. They must be able to walk (assistive aids are allowed, including intermittent use of wheelchair). Patients with other medical conditions and taking certain medication may not be able to take part (follow the link below for more details).

Further information about the BYM338 trial

Fingolimod for Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIPD)
Official title: Evaluate Efficacy and Safety of Fingolimod 0.5 mg Orally Once Daily Versus Placebo in Chronic Inflammatory Demyelinating Polyradiculoneuropathy Patients
Description: In this phase 3 trial pharmaceutical company Novartis is testing Fingolimod – a drug that acts on the immune system – in people with CIPD. The drug has already been approved for treating multiple sclerosis. The study centres are located in Fitzroy and Parkville.
Who can take part? This trial is for men and women aged 18 to 75 years with a diagnosis of CIPD. Other inclusion and exclusion criteria apply, follow the link below for details and discuss with your doctor.

Further information about the Fingolimod trial

Immunoglobulin treatment for Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
Official title: Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) and Treatment With Subcutaneous Immunoglobulin (IgPro20)
Description: Intravenous immunoglobulin (IVIG) is a recommended treatment option for CIDP and this study plans to test a new immunoglobulin product which rather than being given intravenously (a needle inserted into a vein) is injected under the surface of the skin. It may, therefore, offer another treatment option for CIDP, possibly allowing patients to administer the medication at home, or wherever and whenever it suits them. The study centre is located in Fitzroy.
Who can take part? This study is for men and women over 18 years of age with a diagnosis of CIPD who have had repeated treatment with IVIG (≥ 4 infusions) within the last 9 months prior to enrolment. Some other medical conditions may exclude participation in the study; follow the link below for details.

Further information about the immunoglobulin for CIPD study

If you have any questions about clinical trials please contact us:

Phone +61 3 9320 9555
This page was last updated on 25 March 2014