SIOP-HRMB
Research type
Research Study
Full title
An International Prospective Trial on High-Risk Medulloblastoma in Patients Older than 3 Years
IRAS ID
256748
Contact name
Simon Bailey
Contact email
Sponsor organisation
University of Birmingham
Eudract number
2018-004250-17
Duration of Study in the UK
12 years, 6 months, 1 days
Research summary
SIOP-HRMB is an international phase III clinical trial in children, young people and adults with high risk medulloblastoma (HR-MB). The prognosis for patients with HR-MB is poor, with only 60% of patients cured. Those that are cured suffer significant long-term side effects mainly associated with radiotherapy, which can severely affect the quality of life for these patients. This trial aims to improve survival for this group of patients, while limiting the impact of side effects.
The trial consists of two randomised questions. All patients will be treated with two courses of induction chemotherapy. The next phase of treatment (radiotherapy) will be randomised. Three treatment strategies will be compared: Conventional radiotherapy; hyperfractionated-accelerated radiotherapy (HART); and high-dose chemotherapy followed by conventional radiotherapy. HART is similar to conventional radiotherapy but doses are delivered twice a day over a shorter treatment time, albeit with a slightly higher overall dose. Patients randomised to the high-dose chemotherapy arm will still be treated with conventional radiotherapy, but will also be treated with a chemotherapy drug called thiotepa. These patients will have stem cells collected from their blood during induction chemotherapy. After the high-dose chemotherapy, these cells will be reinfused, allowing the bone marrow to repopulate more rapidly using the patient’s previously collected healthy bone marrow stem cells. This part of the trial aims to intensify treatment, without increasing radiotherapy-associated side-effects.
The second randomisation will compare two different maintenance chemotherapy regimens. Patients will either be treated with the current standard four-drug regimen, which is delivered as 8 cycles consisting of cisplatin, vincristine, lomustine alternating with cyclophosphamide and vincristine; or with single agent temozolomide, which is given orally for 6 cycles. The standard regimen is associated with significant toxicities, and has not been evaluated as part of a randomised controlled trial. There is evidence to suggest that temozolomide might be as or more effective as the standard chemotherapy but with less toxicity.REC name
London - Central Research Ethics Committee
REC reference
19/LO/1336
Date of REC Opinion
11 Oct 2019
REC opinion
Further Information Favourable Opinion