Rare Disease Group
To bring together clinicians, scientists and consumers for the purposes of:
- Undertaking clinical research into the causes of and treatment for MPGN and C3 glomerulopathies
- Promoting best clinical practice for patients with MPGN and C3 glomerulopathies
- Facilitating support for patients and families affected by MPGN and C3 glomerulopathies
Funder: Ra Pharma
Title of project: Complement biomarkers as predictors of response to anti-factor D therapy in C3 glomerulopathy
Principal Investigator(s): Claire Harris
Amount: ~ £192000
Using samples that were collected as part of the national study of MPGN and C3G
The National Study of MPGN Sponsor: The Newcastle Upon Tyne Hospitals NHS Foundation Trust Funder: Kids Kidney Research Chief Investigator: Dr Sally Johnson
Children or adults with MPGN, DDD or other diseases that look similar on a kidney biopsy (for example C3 glomerulopathy), can be entered into the study if their centre is participating (see below).
There are three main aims for the study. Firstly, to find out whether problems with the immune system are key in causing MPGN/DDD, secondly, to build a group of patients with these rare diseases in order to test new treatments and thirdly to look for signs that might predict a good or bad outcome.
The study is very simple. Participants’ clinical information is entered into the secure RaDaR database by the local team. In addition, a small amount of extra blood is taken every 6-12 months (with their usual blood tests) and sent to expert laboratories in the UK to look at aspects of the immune system. Finally, the original kidney biopsy is reviewed by a panel of pathologists (doctors who make diagnoses from tissue specimens), to see if there are any links between the biopsy features and the clinical course of the patient. It is hoped this will provide vital information to guide clinical trials of new treatments for MPGN and C3G.
Sites currently open to recruitment include: Newcastle Upon Tyne Hospitals NHS Trust, Guys and St Thomas’ NHS Trust, NHS Greater Glasgow and Clyde, Leeds Teaching Hospitals NHS Trust, Nottingham University Hospitals NHS Trust, Alder Hey Children’s NHS Foundation Trust, Central Manchester University, Southampton University Hospital, Birmingham Children’s Hospital, University Hospital Bristol NHS Trust, Cardiff and Vale NHS Trust, Great Ormond Street Hospital NHS Trust, Royal Free London NHS Foundation Trust, Royal Devon and Exeter NHS Foundation Trust.
A guide for RaDaR data entry for National Study of MPGN patients can be found here.
Patient and Family Conference 2013
The first UK patient and family day for people affected for people affected by MPGN, DDD, C3G and other related disorders was held in Newcastle on 22nd June 2013. There was a full programme of expert speakers in the field, along with an opportunity to ask questions and find support from others affected by these conditions.
The MPGN/DDD Support Group are a UK charity dedicated to providing information, understanding and support to patients and their families coping with these rare kidney diseases – MPGN Type 1, DDD and C3G.
- Professor Terry Cook, Professor of Renal Pathology, London
- Dr Daniel Gale, MRC Clinician Scientist/Nephrologist, London
- Ms Julie Gibbs, Patient Representative
- Dr Rodney Gilbert, Consultant Paediatric Nephrologist, Southampton
- Professor Tim Goodship, Professor of Renal Medicine, Newcastle
- Professor Lorraine Harper, Professor of Nephrology, Birmingham
- Professor Claire Harris, Professor of Immunology, Cardiff and Head of Complement at GlaxoSmithKline
- Dr Sally Johnson, Consultant Paediatric Nephrologist, Newcastle
- Dr David Kavanagh, Senior Lecturer in Nephrology, Newcastle
- Dr Roger Malcomson, Consultant Paediatric Pathologist, Leicester
- Dr Kevin Marchbank, Lecturer in Immunology, Newcastle
- Dr Stephen Marks, Consultant Paediatric Nephrologist, London
- Professor Matthew Pickering, Professor of Rheumatology, London
- Ms Sandra Richardson, Patient representative
- Professor Neil Sebire, Professor of Paediatric and Developmental Pathology, London
- Ms Sarah Whittall, Patient representative
- Dr Edwin Wong, Clinical Research Fellow, Newcastle (RDG Lead)
Sally Johnson serves on a scientific advisory board for a commercial study in a related kidney condition sponsored by Alexion Pharmaceuticals. All monies received go towards paediatric research activity at her host institution.
Claire Harris is Head of Complement in the ImmunoInflammation Unit at GlaxoSmithKline, UK.