ESMART
Research type
Research Study
Full title
European Proof-of-Concept Therapeutic Stratification Trial of Molecular Anomalies in Relapsed or Refractory Tumours
IRAS ID
228992
Contact name
Lynley Marshall
Contact email
Sponsor organisation
Gustave Roussy
Eudract number
2016-000133-40
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
MX2009, CRCTU CAS ; ITCC-057, ITCC
Duration of Study in the UK
3 years, 3 months, 30 days
Research summary
Despite the good overall survival in children and adolescents with cancer, outcomes remain poor for high risk groups and those that do not respond to frontline therapies. Better treatment options which work in novel ways are required. A number of drugs that target the specific underlying pathways which allow individual cancers to grow have been developed in adult oncology and have potential to be effective in paediatric tumours. ESMART is a European trial which will offer children and adolescents with different types of relapsed or refractory cancer who have exhausted standard treatment access to several new drugs/drug combinations as part of a single umbrella study with multiple arms. A unique feature of entry to the trial is the requirement for results from extensive molecular analysis from a biopsy at the time of relpase (biopsy and analysis not part of ESMART) so that the treatment arm offered is the ‘best fit’ in targeting the biology of each patient’s cancer. This will be the first study in the UK to routinely offer this advanced tumour profiling in children and adolescents. The hope is that this approach will increase the chances of the drug working and expand our knowledge of what molecular and genetic features predict therapeutic response. The trial aims to first identify the safety and correct dose of these treatments in children using knowledge from adult trials and where there seems to be evidence of anti-tumour activity to enrol more patients to assess if these drugs are effective. An efficient study design has been adopted to accelerate these drug development objectives and with the option to add treatment arms as new drugs become available. The following drugs are currently being tested in this trial: Ribocliclib, Topotecan, Temozolomide, AZD1775, Carboplatin, Olaparib, Irinotecan, Nivolumab, Enasidenib, Lirilumab.
REC name
London - Riverside Research Ethics Committee
REC reference
19/LO/0456
Date of REC Opinion
12 Apr 2019
REC opinion
Favourable Opinion