Safety and efficacy of LNP023 in C3 glomerulopathy patients

• Research type

  Research Study

• Full title

  An open-label, non-randomised study on efficacy, pharmacokinetics, pharmacodynamics, safety and tolerability of LNP023 in two patient populations with C3 glomerulopathy

• IRAS ID

  254062

• Contact name

  Andrea Biodani

• Contact email

  andrea.biodani@novartis.com

• Sponsor organisation

  Novartis Pharma AG

• Eudract number

  2017-000889-29

• Duration of Study in the UK

  1 years, 6 months, 8 days

• Research summary

  Research Summary
  C3 glomerulopathy (C3G) is a rare inflammatory kidney disease caused by abnormalities in the “alternative pathway of complement” - part of our immune system which provides protection against infections.

  Most patients with C3G have significant disease, with approximately 50% developing end stage renal disease within 10 years. Even after kidney transplantation, the disease often recurs. Currently, there are no approved drugs available to patients to treat C3G.

  LNP023 is a medicine that blocks key steps in the alternative complement pathway, which may improve C3G.

  The purpose of this study is to find out if LNP023 is safe and has beneficial effects in people who have C3G. The study will include patients who have not received a kidney transplant with reduced C3 blood levels and patients who have received a kidney transplant and have C3G recurrence.
  
  The main objective of this research for non-transplanted patients is to show reduction in urine protein; for transplanted patients the main objective is to evaluate the reduction of C3 deposit in renal biopsies. The study will also provide information on the appropriate dose of LNP023 to be used in future studies in similar patients.

  Patients will be treated with LNP023, a tablet which they will take twice per day for 13 weeks. During the study, patients will be visit their doctor in hospital between 13-20 times, depending on whether they enter the dose-tapering period or the proposed extension study. This includes three overnight stays, and three long visits, lasting approximately 10 hours each. All other visits will last about 1-2 hours. During the visits, patients will undergo assessments to monitor their condition. In total, each patient’s participation will last 9 months.

  The study will last for approximately two years with 4 patients enrolled across 2 sites in the UK.

  Summary of Results
  This will be finalised by next week

• REC name

  North East - Tyne & Wear South Research Ethics Committee

• REC reference

  18/NE/0360

• Date of REC Opinion

  13 Dec 2018

• REC opinion

  Further Information Favourable Opinion