CED of Carboplatin & Valproate into pons for children with DIPG 001
Research type
Research Study
Full title
A safety run-in, phase 2 trial of Convection Enhanced Delivery (CED) of Carboplatin and Sodium Valproate into the pons for the treatment of Diffuse Intrinsic Pontine Glioma (DIPG) after radiotherapy in children aged 3-17 years. A combined trial of: Safety and functionality of the Renishaw® Drug Delivery System (IMD). Safety and efficacy of CED of Carboplatin and sodium valproate (IMP).
IRAS ID
257211
Contact name
Steven Gill
Contact email
Sponsor organisation
Xpead Limited
Eudract number
2018-005031-24
Clinicaltrials.gov Identifier
U1111-1226-8937, WHO Universal Trial Number
Duration of Study in the UK
3 years, 0 months, 4 days
Research summary
Diffuse intrinsic pontine glioma (DIPG) is an almost universally fatal and disabling brainstem tumour of children. Because of its diffuse characteristics and anatomical location its surgical removal is not possible and its growth causes early death by damaging the brain centres controlling breathing and cardiac function. The only therapy in use is radiotherapy, which is at best temporarily palliative. Consequently there is an urgent need to explore other therapies. Historically cancer drug trials in this disease have been totally ineffective. We believe the blood brain barrier blocks their access to the tumour. We have developed a device to deliver drugs directly to the brain tumour comprising of four micro-catheters attached to skull-anchored externalised port that emerges through the skin behind the ear. This permits attachment of 4 syringe drivers to deliver drugs under steady pressure when children are awake called convection enhanced drug delivery (CED). We will use carboplatin and sodium valproate, both established drugs with demonstrated synergy in their anti-tumour action, delivered directly to the tumour by CED. The trial is assessing the safety and feasibility of the device and the drug infusions as well as measuring efficacy of the treatment by measuring the progression free survival. The trial is eligible for children between the ages of 3 to 17 years old and will be carried out at The Harley Street Children's Hospital in London. Preliminary experience has identified tumour responses with this approach. Children have remained stable and well for prolonged periods in therapy. If this trial confirms the safety and feasibility of the technique it will open the door to explore the possibility of other drugs given by this technique.
REC name
East of England - Cambridge Central Research Ethics Committee
REC reference
19/EE/0324
Date of REC Opinion
13 Feb 2020
REC opinion
Further Information Favourable Opinion