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PNH Registry

The PNH Registry is an international research database in Leeds which collects anonymised data about PNH patients. It is non –interventional study and participation does not affect a patient’s normal medical care. The registry collects data about natural history, management of patients with PNH and safety data on those treated with eculizumab. Participation is voluntary and all patients with a PNH clone are eligible to participate as long as they give informed written consent. Patients are asked to complete a health assessment questionnaire when they join the registry and then again at six month intervals at outpatient appointments.

The information collected by the registry hopes to improve understanding of the disease and should help to improve treatments in the future.

Other Research and Clinical Trials

This video is a simple explanation about what research is and the roles patients can play in it:

There is a registry and results database of worldwide clinical trials with human participants (whether publicly or privately funded) which can be found at www.clinicaltrials.gov.

Here is an EU datebase of clinical trial Clinical Study Reports and/or summary data: http://www.clinicaltrialsregister.eu

"Bio-similars" (biological medicines developed as 'copies' of existing medicines) are being developed based on understandings made possible through the development and use of eculizumab (soliris) in PNH over the past decade. This will likely see several new medicines come to market in the medium term.

There are multiple pharmarceutical companies which are in the process of starting clinical trials to the treatment of PNH. Most of these compounds have undergone testing with animal and healthy volunteers and are ready to be used in clinical trials. Some of the companies who have recently presented data at the Amercian Society of Haematology in October 2014 are: Alnylam, Ra Pharma, Achillion, Apellis, Chugai, Volution and Alexion. Plans for clinical trials at the NHS National Centres for these new compounds are currently being drafted and participation in these trials (both Phase 2 and 3) can be discussed with your PNH clinician.

There is a Boston based Pharmaceutical company headed by a scientist who has previously developed three successful orphan medicines. This company has been developing a compliment-inhibitor as a treatment for PNH over that past two and a half years and early signs are that it could prove to be an effective medicine. This medicine is not biological which provides several advantages, including that it can be self-administered, but more importantly the costs of development have been well below those for eculizumab - which will mean it will cost less once it is on the market.

Research at the PNH National Service Centres

Multiple research projects into PNH and bone marrow failure syndromes are taking place (as at 2015) at the PNH NHS National Service centres in Leeds and London.

  • The National Service centres are in the process of developing eculizumab assay and free C5 levels and samples are currently being shipped to the USA for interpretation.
  • The Leeds National Service centre is developing specialised tests to detect mutations in the causative gene for PNH, i.e. PIG-A, using next generation sequencing.
  • A culture system for PNH stem cells has been established in vitro (outside the body, in the lab) which has shown that the T lymphocytes are of great importance. This project is being taken forward and further research is being conducted to establish this link.
  • Despite being treated with eculizumab, some PNH patients continue to require blood transfusions and it is well established that the breakdown of red blood cells happens in the liver and spleen. Further research is being planned and newer drugs blocking the complement protein called C3 would be a major step forward. These drugs are available and research is being conducted to see if these drugs are effective and safe. 




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