Paediatric Liver Transplant study with Everolimus

• Research type

  Research Study

• Full title

  A 24-month, multi-center,single arm, prospective study to evaluate renal function, efficacy, safety and tolerability of everolimus in combination with reduced exposure cyclosporine or tacrolimus in paediatric liver transplant recipients.

• IRAS ID

  107636

• Contact name

  Raj Hanvesakul

• Sponsor organisation

  Novartis Pharma AG

• Eudract number

  2011-003069-14

• ISRCTN Number

  N/A

• Research summary

  Liver transplantation is the treatment of choice for children with liver failure. While the short-term success rates are high, the need for lifelong anti-rejection medication brings a burden of side-effects that, over time, can damage the kidney and predispose to infections and cancers. There are a limited number of anti-rejection drugs and few have been systematically studied in the setting of liver transplantation in children. The purpose of this research is to measure the effectiveness and safety of everolimus in recently transplanted children in order to gain formal approval for this indication from the European Medicines Agency (EMA) and other Regulatory Authorities. As such, this study forms part of a Paediatric Investigational Plan agreed with the EMA. This is a 2 year, single arm, prospective study conducted at approximately 30 sites worldwide (3 in UK). The objective of the study is to demonstrate that everolimus can be used to part-replace and minimise exposure to other anti-rejection drugs (tacrolimus or ciclosporin) that are known to cause kidney injury. Approximately 125 paediatric patients (>1 month <18 years) who have undergone liver transplantation and for whom consent has been given, will be screened for the study. Of these, an expected 75 eligible subjects will enter the study at 1-6 months after their transplant and will receive treatment with everolimus in combination with steroids and reduced doses of tacrolimus or ciclosporin. Once started on study treatment, subjects will visit on 8 more occasions over the 2 year period for clinical and laboratory assessments. In addition to routine health checks and blood tests appropriate to liver transplant patients, the study will focus on the evolution of kidney function, growth/development of the child and surveillance monitoring for viral infections.

• REC name

  North East - Tyne & Wear South Research Ethics Committee

• REC reference

  12/NE/0235

• Date of REC Opinion

  25 Jul 2012

• REC opinion

  Further Information Favourable Opinion