Extrait

Background and study aims Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease (after a famous baseball player who suffered from it), is a serious condition which affects the nervous system. When a person is suffering from ALS, the nerve cells in the brain and spinal cord which control the movement of muscles (motor neurons) are gradually lost (neurodegeneration). The disease often begins with muscle twitching and weakness in the arms or legs, eventually leading to paralysis. ALS is ultimately fatal, as it affects the muscles involved with breathing. The diaphragm is a large dome-shaped muscle which separates the lungs from the digestive organs in the abdomen. It is one of the most important muscles involved in ventilation (breathing in and out), helping to inflate and deflate the lungs by moving down and up. When the diaphragm is weakened, a person is not able to get enough oxygen into their lungs (respiratory failure), which eventually leads to death. Non-invasive ventilation therapy (NIV) is the standard treatment given to respiratory failure patients with ALS. In this type of treatment, patients wear a mask over their nose and mouth which “pushes” air into their lungs, allowing them to get enough oxygen. Eventually however, this treatment stops being effective, as the ALS causes the diaphragm to become more and more weak. Diaphragm pacing (DP) is where a device (diaphragm pacemaker) is implanted into the diaphragm muscle to help support breathing. The diaphragm pacemaker sends pulses of electricity to the muscle, helping it to contract (causing air to be sucked into the lungs) and relax (pushing air out of the lungs). The pacemaker is attached to a small box which is easy to carry around, allowing patients more independence than when having NIV. The aim of this study is to find out whether giving DP and NIV together can help to improve quality of life for ALS patients with respiratory failure, compared to NIV alone. Who can participate? Adults suffering from familial or sporadic amyotrophic lateral sclerosis (ALS). What does the study involve? Participants are randomly allocated to one of two groups. Those in the first group (control group) continue with standard care, which involves them attending hospital in order to receive regular NIV. Those in the second group (intervention group) have an operation so that the diaphragm pacemaker can be placed in the diaphragm muscle. These participants are also given the standard treatment of NIV when needed. At 2, 3, 6, 9 and 12 months, participants in both groups complete a number of questionnaires to assess their quality of life. Participants in the intervention group also attend an additional follow-up appointment one week after their operation. A small group of participants from this group are also interviewed so that more information about how the DP has affected their lives. What are the possible benefits and risks of participating? Not provided at time of registration Where is the study run from? Seven specialist ALS and respiratory centres in the UK When is the study starting and how long is it expected to run for? March 2007 to September 2010 Who is funding the study? National Institute of Health Research (UK) Who is the main contact? Dr Christopher McDermott

Critère d'inclusion

• Motor Neurone Disease

Liens

• ictrp
• isrctn