MDA’s Sponsorship of CINRG


 The Cooperative International Neuromuscular Research Group, (CINRG), was formed in 1999 as the clinical research arm of the Duchenne Muscular Dystrophy Research Center (DMDRC) and the Research Center for Genetic Medicine at the Children’s National Medical Center (CNMC).

The primary goal of the CINRG research program is to provide a continuum of well-designed clinical trials in a geographically dispersed network so that they are available to every child with Duchenne muscular dystrophy (DMD). Currently CINRG’s network expands across 23 clinical trial sites in 10 countries.

CINRG’s vision is to be pre-eminent in the advancement of knowledge and development of effective treatments for people with DMD.

The Department of Neurology at the Royal Children’s Hospital (RCH) has been an active CINRG clinical trial site for the last four years. The MDA has been instrumental in the establishment of the CINRG collaboration by funding the study coordinator position and research physiotherapists involved in strength testing for the CINRG studies. Without funding from MDA it would not have been possible for the RCH to establish a local CINRG Centre. We are extremely grateful to MDA for this support.

There are three currently active CINRG trials at RCH. They are:

  • Prednisone (high-dose vs daily) in Duchenne muscular dystrophy;
  • The UCD (University of California, Davis campus) Longitudinal study of the relationship between impairment, activity limitation, participation and quality of life in persons with confirmed Duchenne muscular dystrophy;  and
  • Clinical trial of coenzymeQ10 and prednisone in Duchenne muscular dystrophy

Prednisone (high-dose vs daily) in Duchenne muscular dystrophy study is in its final stages and close to completion. This study involved ambulant children aged 4-10 years with an established diagnosis of DMD. The aim of the study was to help determine whether a high-dose weekly course of prednisone (also known as prednisolone) therapy is safer than and at least as effective as daily therapy. Participants were placed into groups so that the effect of the drug could be evaluated by a randomized, double-blinded placebo controlled study. The participants were evaluated for muscle strength, timed function tests, functional grades for arms and legs, and pulmonary function tests at variable intervals for 12 months.

Longitudinal study of the relationship between impairment, activity limitation, participation and quality of life in persons with confirmed Duchenne muscular dystrophy, involves establishing the largest long-term assessment of people with DMD between the ages of 2 and 30 years.

This is a five year study collecting information from boys and men with DMD and their families. Information to be collected will include studies of patient’s physical abilities, medical problems and use of healthcare services. A second goal is to look for an association between modifying genes (which contain instructions for how the body works) and response to treatment of DMD.  Participants in this study will undergo strength and lung function testing at variable intervals during the 5 year study.

Clinical Trial of coenzymeQ10 and prednisone in Duchenne muscular dystrophy. This study is being initiated at RCH in mid-2007and is currently recruiting participants.
This study will help determine if CoQ10 and prednisone, alone and in combination, decrease the decline in cardiopulmonary and skeletal muscle function that occurs in the wheelchair phase of DMD. Participants in this study will be wheelchair confined males aged 10 to 18 years with DMD.

Participants are evaluated at variable intervals. Assessments will involve efficiency and safety assessments and monitoring for a 12 monthly period.

Involvement in CINRG and other trials offers families a chance to be at the cutting edge of research into DMD. We hope that effective new treatments for DMD will be identified through these studies. These studies have the potential to benefit all boys with DMD. The RCH commitment to CINRG and other research networks reflects our desire to bring the very best and most current medical treatment to all of our patients.

Please do not hesitate to contact Ms Daniella Villano, MDA – Neuromuscular Nurse Coordinator at RCH, with any questions regarding CINRG and our other research interests. Dani can be contacted at email: daniella.villano@rch.org.au


 MDA’s Synergy with CINRG


Until now, neuromuscular groups worldwide have had no co-ordinated network or unified framework for clinical trials research. That meant that any treatment trials in the neuromuscular disorders were usually small, and in many cases, not performed in the most cost effective manner.

DMD is common the world over, and millions of dollars have been raised to promote research in DMD. However, much of the research has focused on the basic biology of the disease – research that was done in laboratories, away from the children themselves. As the ultimate goal of medical research is to affect a change in the course of the disease, there was a need to improve the communication and knowledge base of both families and clinicians, with the end result being an improvement in the quality of life for patients and their families. Without a co-ordinated group of researchers, these goals would be difficult to achieve.

For these reasons, Eric Hoffman in the USA established the Cooperative International Neuromuscular Research Group (CINRG). CINRG’s vision is to be pre-eminent in the advancement of knowledge and development of effective treatments for people with Duchenne Muscular Dystrophy and related disorders. To achieve this, CINRG developed specialised equipment for muscle strength testing to be used in clinical trials and devised a structure where all participants were involved in sound scientific research in various centres around the world. Through the conduct of collaborative research and the use of state of the art technologies, it is likely that there will be the establishment of internationally recognized standards of care for people who suffer from neuromuscular diseases.

The ability of CINRG to involve a number of groups around the world in collaborative research will accelerate the introduction of treatments and innovations from the laboratory to the bedside more quickly.

The Department of Neurology at the Royal Children’s Hospital (RCH) was invited to be part of CINRG last year and it has now purchased the equipment necessary. The initial training schedule for the evaluators at RCH is going ahead in the next week. This association with CINRG will give patients in Australia early access to leading edge therapies, when and if they become available.

The MDA has been instrumental in the establishment of the CINRG collaboration by funding the study coordinator/evaluator position. Without funding from MDA, it would not be possible to establish a local CINRG Centre. We are extremely grateful to MDA for this support. This close association of CINRG and MDA, will give MDA an international profile as a supporter of the program and provide the opportunity for MDA to be more involved with other international lay organisations

It is hoped that the association with CINRG will attract increased community support for funding in Australia by raising the profile of neuromuscular disease within the community.

In the next few months, your child might be asked to participate in some training sessions with the equipment. Your child’s doctor will coordinate this with the evaluator at RCH.