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HR-MB: An International High-Risk Medulloblastoma Clinical Trial

Fast facts

  • Official title: HR-MB: An international High-Risk Medulloblastoma trial
  • Lead researcher: Professor Simon Bailey
  • Where: Led from Newcastle University and the University of Birmingham Clinical Trials Unit
  • When: October 2018 – January 2031
  • Cost: The full cost is just over £2 million, which we fund 50/50 with Cancer Research UK
  • Research type: Paediatric, Medulloblastoma (High Grade), Clinical Trial

Professor Simon Bailey is conducting an international clinical trial to evaluate the advantages of different chemotherapy and radiotherapy strategies for high-risk medulloblastoma patients.

Medulloblastoma (MB) is the most common high-grade brain tumour in children. The medulloblastomas that are most likely to come back after treatment are known as high-risk medulloblastomas (HR-MBs). A number of factors can result in a MB being classified as high-risk i.e. if the cancer has spread to other parts of the brain, or if not all of the tumour was removed during surgery. Furthermore, HR-MB patients that are successfully treated are likely to be left with significant long term side effects including impact on IQ, memory and concentration. All of these significantly impact on the person’s quality of life.

This trial aims to compare existing, intense, treatment strategies for HR-MB and the resulting impact on survival and quality of life. Therapies that improve outcome (both survival and quality of life) will be prioritised for the future. Whereas, if an intensive therapy is found to offer no additional benefit, than less intensive therapies, it will be eliminated from future practice. Comprehensive analysis of the child’s tumour and blood samples will also be conducted. This will mean the relationship between the unique biology of each tumour and its response to treatment can be better understood.

How will the trial work?

The trial is designed to recruit up to 850 patients internationally, including 250 in the UK, over an eight year period.

Patients first receive surgery and chemotherapy according to the current best practice protocols. At this point they will enrol onto the trial and go through one or two further stages of treatment 1) randomisation 1 – where all patients will be randomly assigned treatments in Arm A, Arm B or Arm C (described below), 2) randomisation 2 – patients treated in Arms A and B will then be randomly assigned additional treatment from either Arm D or Arm E.

Patients who were previously randomised into Arm C (high-dose chemotherapy) will not take part in the second randomisation and will automatically go on to receive Temozolomide. This is because the researchers already know that the side effects of Arm C mean that Arm D would not be tolerated.

Randomisation 1

Arm A: Conventional radiotherapy – radiotherapy will be given once a day

Arm B: Hyperfractionated and Accelerated Radiotherapy (HART) – radiotherapy will be given twice a day

Arm C: High dose chemotherapy (with a drug called Thiotepa) followed by conventional once-a day) radiotherapy

Randomisation 2

Arm D: This arm will alternate between two cocktails of chemotherapy Regimen A (Cisplatin, Lomustine and Vincristine) and Regimen B (Cyclophosphamide and Vincristine);

Arm E: Temozolomide

During the study, the researchers will be closely monitoring overall survival, any side effects and quality of survival (as reported by the participants).

A vital part of the HR-MB trial is the quality of life and quality of survival assessment which was developed by Professor Kennedy – a researcher we’ve long supported and whose achievements in the quality of life field have been widely acknowledged as important progress.

What’s been achieved so far?

There are seven centres in the UK now open and actively recruiting children. These centres are: Birmingham Children’s Hospital, Christie Hospital Manchester, Royal Hospital for Children (Glasgow), Royal Manchester Children’s Hospital, Royal Victoria Hospital (Newcastle), Queen Elizabeth Hospital (Birmingham) and University College Hospital (UCLH, London).

At the time of their last report the researchers shared that, even after significant delays due to the COVID-19 pandemic, they have enrolled the first child onto the trial.

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