Clinical Trials

Clinical trials are a way of introducing new discoveries and new treatments into clinical practice in a safe and controlled way – so that we are continually gathering evidence for what really works – and what doesn’t.

It may surprise you to learn that while steroids have been the mainstay of treatment for DMD for many years, there has never been a properly designed and performed clinical trial to work out the best timing and dose.


The experience of improving treatment of childhood leukaemia provides an excellent example of how clinical trials improve survival.

The childhood leukaemia survival rate in the 1970’s was approx. 25%.

Since that time treatment for every patient has been provided as part of a clinical trial. This means that every time a new therapy or combination of cancer therapies is given to a patient, it is done so in an organised and uniform way for a series of patients, and the response and outcome is monitored.

Step by step – the best combinations of treatments are identified and continually improved upon.

In 2009 the survival rate for childhood leukemia was 92% – demonstrating the great success of this coordinated approach to introducing new therapies.

In order to attract more clinical trials to Australia, we need to build a coordinated clinical trial infrastructure.  Clinical trials also provide the opportunity to make the latest in therapies available to patients in Australia. Companies look at patient numbers, expertise and experience in  potential trial sites. The existing local experience and expertise provides an opportunity to train personnel to staff additional trial centres around Australia. This will simplify the onerous travelling requirements for many families.

The proposed model would involve representation from each state within the Australia/New Zealand Neuromuscular Network. As trial opportunities arise through TREAT-NMD, CINRG and other organisations, information regarding potential sites in Australia, patient cohorts and expertise will be readily available.

The establishment of a network would facilitate the training of evaluators in other centres within Australia to make clinical trials more accessible. Ultimately the expanding clinical trials program in Australia has provided families with hope for the future, and every child should have the opportunity to be cared for in a centre of best practice.

The establishment of an integrated network will:

  • increase the quality of clinical trials being conducted in Australia
  • increase the number of trials conducted
  • increase the number of patients routinely being offered clinical trial participation in a greater variety of clinical settings
  • increase the timeliness and efficiency of recruitment

The ANN aims to establish a database of active trials in Australia and New Zealand – in the meantime if you would like to know more about clinical trials please contact A/Prof Monique Ryan (Co-Chair, Clinical Trials Steering Group). For patients affected by Duchenne muscular dystrophy, registering their details in the Australian National Duchenne Muscular Dystrophy Registry (see below) will ensure that you and your patients will be notified as new trials come on line.