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