RITPBC Study

• Research type

  Research Study

• Full title

  Rituximab for the Treatment of Fatigue in Primary Biliary Cirrhosis

• IRAS ID

  90909

• Contact name

  David Jones

• Eudract number

  2012-000145-12

• ISRCTN Number

  N/A

• Clinicaltrials.gov Identifier

  N/A

• Research summary

  Primary Biliary Cirrhosis (PBC) is a liver disease that predominantly affects females, can present for the first time at any age and which develops over many years. It is caused by the immune system attacking the body??s own tissues. People with PBC frequently experience profound fatigue or tiredness which they liken to their ??batteries running down? and although People'still want to undertake normal activities they simply lack the energy to be able to do them. This reduces quality of life, makes it difficult for people to work, and can end up with them becoming isolated in the community. At present we have no treatment for fatigue in PBC. Finding a treatment for fatigue in PBC is one of the highest research priorities identified by patient groups. We have shown that PBC patients with fatigue have an abnormality in the way they generate energy within their muscles. This appears to be associated with the presence of an antibody in the blood which is directed against an important protein which normal cells in the body use to generate energy. In recent years new drug treatments have been developed which allow us to safely suppress the part of the immune system which produces antibodies of the type that seem to cause energy production problems in PBC. As yet, however, the extent to which these medicines can improve fatigue through removal of antibodies in PBC has not been tested. The aim of this study is to undertake a clinical trial to examine the effects of this treatment (Rituximab) on severe fatigue in PBC to help us understand whether this will be a potentially useful treatment. This information will tell us how energy generation changes in patients with PBC with and without the treatment and will also help us to develop new treatments for fatigue in other diseases. The study has the potential to improve the quality of life of many patients with PBC, for whom there is currently no hope of improvement. We will perform a randomized controlled trial of Rituximab therapy in PBC compared to placebo with the primary end point of fatigue severity. The study will be performed in a specialised Clinical Research Facility at the Royal Victoria Infirmary, Newcastle. We have, for many years, worked closely with PBC patient groups to focus on the problems that are important to our patients. This study reflects these close links and is fully supported by LiverNorth, a liver disease charity and patient support group.

• REC name

  North East - Newcastle & North Tyneside 1 Research Ethics Committee

• REC reference

  12/NE/0095

• Date of REC Opinion

  16 May 2012

• REC opinion

  Further Information Favourable Opinion