Pomalidomide in Children and young adults with brain tumors
Research type
Research Study
Full title
A phase 2 clinical study of Pomalidomide (CC-4047) monotherapy for children and young adults with recurrent or progressive primary brain tumours
IRAS ID
226326
Contact name
Lynley Marshall
Contact email
Sponsor organisation
Celgene Corporation
Eudract number
2016-002903-25
Duration of Study in the UK
5 years, 7 months, 5 days
Research summary
Central nervous system (CNS) tumours, including brain and spinal cord are the most common solid tumours in children and account for up to 25% of all childhood cancer cases (Fleming 2012). Overall survival is estimated to be over 70% at 5 years for the less aggressive tumour types. Outcomes for children with recurrent/progressive disease are much lower and once a child with a malignant brain tumour has suffered a recurrence, survival outcomes diminish considerably and in many cases are very poor indeed (Dolecek, 2012).
Pomalidomide destroys tumour cells by halting the development of new blood vessels and modifying the immune response. Pomalidomide was approved in 2013 for the treatment of patients with Multiple Myeloma who have received at least two prior therapies, and has been tested in phase 1 studies in adults with CNS tumours. In this study, Pomalidomide is given by mouth as capsules (pills) once daily, 21 days in a row, of each 28-day cycle. Patients will receive oral pomalidomide for up to 24 cycles or until documented Progressive Disease, withdrawal of consent or death.
This study will assess the efficacy, safety and tolerability of pomalidomide in children and young adults with recurrent or progressive brain tumours. Up to 80 patients will take part in this study which will consist of 3 phases: screening, treatment and follow-up.
The total duration on trial for the patient for treatment and follow up is around 45 months. Surviving patients will be in long term follow up for up to 5 years after they finish the trial. Study procedures include blood samples, MRI scans, physical examinations, and other study assessments. Participants on this study will be 1 - <21 years old.
This is an open-label, non-randomised study and Celgene Corporation is the sponsor.
REC name
London - Central Research Ethics Committee
REC reference
17/LO/1158
Date of REC Opinion
2 Aug 2017
REC opinion
Favourable Opinion