About this trial
FaR-RMS is an over-arching study for children and adults with newly diagnosed and relapsed rhabdomyosarcoma (RMS)
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Histologically confirmed diagnosis of RMS (except pleomorphic RMS)
Written informed consent from the patient and/or the parent/legal guardian
Entered in to the FaR-RMS study at diagnosis
Very High Risk disease
Disqualifiers
None
Trial design
Parallel
Treatments tested in this trial
Irinotecan
Drugantineoplastic enzyme inhibitor
Actinomycin D
DrugAntineoplastic agent that is a polypeptide antibiotic
Doxorubicin
DrugAn anthracycline topoisomerase inhibitor isolated from streptpmyces peucetius var. casesius
Ifosfamide
Drugchemotherapeutic agent chemically related to the nitrogen mustards and is a synthetic analog of cyclophosphamide
Vincristine
Druganti neoplastic vinca alkaloid agent
Vinorelbine
Drugvinca alkaloid with a role as an antineoplastic agent
Cyclophosphamide
DrugPrecursor of an alkylating nitrogen mustard antineoplastic and immunosuppressive agent
Temozolomide
Drugoral antineoplastic alkylating agent
radiotherapy
RadiationIonising radiation
Regorafenib
DrugOral multi-kinase inhibitor that targets a broad range of angiogenic, stromal and oncogenic kinases, including vascular endothelial growth factor receptors (VEFGR) 1, 2 and 3, tyrosine kinase with immunoglobulin and epidermal growth factor homology domain 2 (TIE2), platelet-derived growth factor receptor (PDGFR), fibroblast growth factor receptors (FGFR), c-KIT, RET, RAF-1 and BRAF (wild-type and V600E mutant).
Treatment groups
19
Treatment groupsSee each treatment group below.
Trial outcomes
Primary outcomes
Event Free Survival (RT2)
Failure events are: * Relapse or progression of existing disease, or occurrence of disease at new sites, * Death from any cause without disease progression, * Second malignant neoplasm
Event Free Survival (CT1A)
Failure events are: * Relapse or progression of existing disease, or occurrence of disease at new sites, * Death from any cause without disease progression, * Second malignant neoplasm
Event Free Survival (CT1B)
Failure events are: * Relapse or progression of existing disease, or occurrence of disease at new sites, * Death from any cause without disease progression, * Second malignant neoplasm
Event Free Survival (CT2A)
Failure events are: * Relapse or progression of existing disease, or occurrence of disease at new sites, * Death from any cause without disease progression, * Second malignant neoplasm
Secondary outcomes
Recommended Phase II Dose (Phase 1b)
Based on tolerability, where tolerability is evaluated through the occurrence of dose limiting toxicity (DLT).
Maximum Tolerated Dose (Phase 1b)
Dose level at which no or one participant experiences a DLT when at least two of three to six participants experience a DLT at the next highest dose.
Toxicity (All chemotherapy randomisations)
Categorised and graded using Common Terminology Criteria for Adverse Events
Dose Limiting Toxicity (Phase 1b)
Diarrhoea: Grade 3 for \>3 days despite loperamide therapy Diarrhoea: Grade 4 despite loperamide therapy. Enterocolitis: Grade 3 or above Ileus: Grade 3 or above for more than 3 days Oral mucositis: Grade 3 above for \>3 days despite optimal supportive care Persistent neutropenia or thrombocytopenia leading to delay of start of next course by \>7 days; i.e. starting \> day 28 Any grade 3 or 4 toxicity resulting in discontinuation of the new combination Any grade 5 toxicity
Sponsors and contacts
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