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FaR-RMS: An Overarching Study for Children and Adults With Frontline and Relapsed RhabdoMyoSarcoma

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FaR-RMS is an over-arching study for children and adults with newly diagnosed and relapsed rhabdomyosarcoma (RMS).

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Study details:

FaR-RMS is an over-arching study for children and adults with newly diagnosed and relapsed rhabdomyosarcoma (RMS). It is a multi-arm, multi-stage format, involving several different trial questions. FaR-RMS is intended to be a rolling programme of research with new treatment arms being introduced dependant on emerging data and innovation.

This study has multiple aims. It aims to evaluate the impact of new agent regimens in both newly diagnosed and relapsed RMS; whether changing the duration of maintenance therapy affects outcome; and whether changes to dose, extent (in metastatic disease) and timing of radiotherapy improve outcome and quality of life. In addition the study will evaluate risk stratification through the use of PAX-FOXO1 fusion gene status instead of histological subtyping and explore the use of FDG PET-CT response assessment as a prognostic biomarker for outcome following induction chemotherapy.

Newly diagnosed patients should, where possible, be entered into the FaR-RMS study at the time of first diagnosis prior to receiving any chemotherapy. However, patients can enter at the point of radiotherapy or maintenance, and those with relapsed disease can enter the study even if not previously entered at initial diagnosis. Patients may be entered into more than one randomisation/registration, dependant on patient risk group and disease status.

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Eligibility criteria

Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.

Inclusion criteria

  • 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
  • Age >12 months and ≤25 years
  • No prior treatment for RMS other than surgery
  • Medically fit to receive treatment
  • Adequate hepatic function: Total bilirubin ≤ 1.5 times upper limit of normal (ULN) for age, unless the patient is known to have Gilbert's syndrome; ALT or AST < 2.5 X ULN for age
  • Absolute neutrophil count ≥1.0x 109/L
  • Platelets ≥ 80 x 109/L
  • Adequate renal function: estimated or measured creatinine clearance ≥60 ml/min/1.73 m2
  • Documented negative pregnancy test for female patients of childbearing potential
  • Patient agrees to use contraception during therapy and for 12 months after last trial treatment (females) or 6 months after last trial treatment (males), where patient is sexually active
  • Entered in to the FaR-RMS study at diagnosis
  • High Risk disease
  • Age ≥ 6 months
  • Available for randomisation ≤60 days after diagnostic biopsy/surgery
  • Medically fit to receive treatment
  • Adequate hepatic function: Total bilirubin ≤ 1.5 times upper limit of normal (ULN) for age, unless the patient is known to have Gilbert's syndrome
  • Absolute neutrophil count ≥1.0x 109/L (except in patients with documented bone marrow disease)
  • Platelets ≥ 80 x 109/L (except in patients with documented bone marrow disease)
  • Fractional Shortening ≥ 28%
  • Documented negative pregnancy test for female patients of childbearing potential
  • Patient agrees to use contraception during therapy and for 12 months after last trial treatment (females) or 6 months after last trial treatment (males), where patient is sexually active
  • Entered in to the FaR-RMS study at diagnosis
  • Very High Risk, High Risk and Standard Risk disease
  • ≥ 2 years of age
  • Receiving frontline induction treatment as part of the FaR-RMS trial or with a IVA/IVADo based chemotherapy regimen patients
  • Patient assessed as medically fit to receive the radiotherapy
  • Documented negative pregnancy test for female patients of childbearing potential
  • Patient agrees to use contraception during therapy and for 12 months after last trial treatment (females) or 6 months after last trial treatment (males), where patient is sexually active
  • Primary tumour deemed resectable (predicted R0/ R1 resection feasible) after 3 cycles of induction chemotherapy (6 cycles for metastatic disease)
  • Adjuvant radiotherapy required in addition to surgical resection (local decision).
  • Available for randomisation after cycle 3 and prior to the start of cycle 6 of induction chemotherapy for localised disease, or after cycle 6 and prior to the start of cycle 9 for metastatic disease
  • Primary tumour deemed resectable (predicted R0/R1 resection) after 3 cycles of induction chemotherapy (6 cycles for metastatic disease).
  • Adjuvant radiotherapy required in addition to surgical resection (local decision)
  • Higher Local Failure Risk (HLFR) based on presence of either of the following criteria: Unfavourable site, Age ≥ 18yrs
  • Available for randomisation after cycle 3 and prior to the start of cycle 6 of induction chemotherapy for localised disease, or after cycle 6 and prior to the start of cycle 9 for metastatic disease
  • Primary radiotherapy indicated (local decision)
  • Higher Local Failure Risk (HLFR) based on either of the following criteria: Unfavourable site, Age ≥ 18yrs
  • Available for randomisation after cycle 3 and prior to the start of cycle 6 of induction chemotherapy for localised disease, or after cycle 6 and prior to the start of cycle 9 for metastatic disease
  • Available for randomisation after cycle 6 and before the start of cycle 9 of induction chemotherapy.
  • Unfavourable metastatic disease, defined as Modified Oberlin Prognostic Score 2-4
  • Entered in to the FaR-RMS study (at diagnosis or at any subsequent time point)
  • Very High Risk disease
  • Received frontline induction chemotherapy as part of the FaR-RMS trial or with a IVA/IVADo based chemotherapy regimen
  • Completed 11 cycles of VnC maintenance treatment (either oral or IV regimens)
  • No evidence of progressive disease
  • Absence of severe vincristine neuropathy - i.e requiring discontinuation of vincristine treatment)
  • Medically fit to continue to receive treatment
  • Patient agrees to use contraception during therapy and for 12 months after last trial treatment (females) or 6 months after last trial treatment (males), where patient is sexually active
  • Entered in to the FaR-RMS study (at diagnosis or at any subsequent time point)
  • High Risk disease
  • Received frontline induction chemotherapy as part of the FaR-RMS trial or with a IVA based chemotherapy regimen.
  • Completed 5 cycles of VnC maintenance treatment
  • No evidence of progressive disease
  • Absence of severe vincristine neuropathy i.e. requiring discontinuation of vincristine treatment
  • Medically fit to continue to receive treatment
  • Entered in to the FaR-RMS study (at diagnosis or at any subsequent time point)
  • First or subsequent relapse of histologically verified RMS
  • Age ≥ 6 months
  • Measurable or evaluable disease
  • No cytotoxic chemotherapy or other investigational medicinal product (IMP) within previous three weeks: within two weeks for vinorelbine and cyclophosphamide maintenance chemotherapy
  • Medically fit to receive trial treatment
  • Documented negative pregnancy test for female patients of childbearing potential within 7 days of planned randomisation
  • Patient agrees to use contraception during therapy and for 12 months after last trial treatment (females) or 6 months after last trial treatment (males), where patient is sexually active

    • Exclusion criteria

  • Weight <10kg
  • Active > grade 2 diarrhoea
  • Prior allo- or autologous Stem Cell Transplant
  • Uncontrolled inter-current illness or active infection
  • Pre-existing medical condition precluding treatment
  • Urinary outflow obstruction that cannot be relieved prior to starting treatment
  • Active inflammation of the urinary bladder (cystitis)
  • Known hypersensitivity to any of the treatments or excipients
  • Second malignancy
  • Pregnant or breastfeeding women
  • Active > grade 2 diarrhoea
  • Prior allo- or autologous Stem Cell Transplant
  • Uncontrolled inter-current illness or active infection
  • Pre-existing medical condition precluding treatment
  • Urinary outflow obstruction that cannot be relieved prior to starting treatment
  • Active inflammation of the urinary bladder (cystitis)
  • Known hypersensitivity to any of the treatments or excipients
  • Second malignancy
  • Pregnant or breastfeeding women
  • Active > grade 2 diarrhoea
  • Prior allo- or autologous Stem Cell Transplant
  • Uncontrolled inter-current illness or active infection
  • Pre-existing medical condition precluding treatment
  • Urinary outflow obstruction that cannot be relieved prior to starting treatment
  • Active inflammation of the urinary bladder (cystitis)
  • Known hypersensitivity to any of the treatments or excipients
  • Second malignancy
  • Pregnant or breastfeeding women
  • Prior allo- or autologous Stem Cell Transplant
  • Uncontrolled intercurrent illness or active infection
  • Urinary outflow obstruction that cannot be relieved prior to starting treatment
  • Active inflammation of the urinary bladder (cystitis)
  • Second malignancy
  • Pregnant or breastfeeding women
  • Progression during frontline therapy without previous response (=Refractory to first line treatment)
  • Prior regorafenib or temozolomide
  • Active > grade 1 diarrhoea
  • ALT or AST >3.0 x upper limit normal (ULN)
  • Bilirubin, Total >1.5 x ULN; total bilirubin is allowed up to 3 x ULN if Gilbert's syndrome is documented
  • Patients with unstable angina or new onset angina (within 3 months of planned date of randomisation), recent myocardial infarction (within 6 months of randomisation) and those with cardiac failure New York Heart Association (NYHA) Classification 2 or higher Cardiac abnormalities such as congestive heart failure (Modified Ross Heart Failure Classification for Children = class 2) and cardiac arrhythmias requiring antiarrhythmic therapy (beta blockers or digoxin are permitted)
  • Uncontrolled hypertension > 95th centile for age and gender
  • Prior allo- or autologous Stem Cell Transplant
  • Uncontrolled inter-current illness or active infection
  • Pre-existing medical condition precluding treatment
  • Known hypersensitivity to any of the treatments or excipients
  • Second malignancy
  • Pregnant or breastfeeding women
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    Eligibility

    Age eligible for study : 0 and older

    Healthy volunteers accepted : No

    Gender eligible for study: All

    Things to know

    Study dates

    Study start: 2020-09-17

    Primary completion: 2030-06-01

    Study completion finish: 2030-06-01

    study type

    Study type

    TREATMENT

    phase

    Phase

      PHASE1

      PHASE2

    trial

    Trial ID

    NCT04625907

    Intervention or treatment

    DRUG: Irinotecan

    DRUG: Actinomycin D

    DRUG: Doxorubicin

    DRUG: Ifosfamide

    DRUG: Vincristine

    DRUG: Vinorelbine

    DRUG: Cyclophosphamide

    DRUG: Temozolomide

    RADIATION: radiotherapy

    DRUG: Regorafenib

    Conditions

    • • Rhabdomyosarcoma
    Image related to Rhabdomyosarcoma

    • Condition: Rhabdomyosarcoma

    • DRUG: Irinotecan and other drugs

    • Brisbane, Not Specified, Australia and more

    • Sponsor: University of Birmingham

    You may be eligible to participate in this trial based on your search. Check eligibility by answering some questions.

    Are you running this trial? If you're a clinic or sponsor, you can claim this study. Claim or learn more.

    Find a site

    Closest Location:

    Queensland Children's Hospital

    Research sites nearby

    Select from list below to view details:

    • Queensland Children's Hospital

      Brisbane, Not Specified, Australia

    • Monash Children's Hospital

      Clayton, Not Specified, Australia

    • Perth Children's Hospital

      Perth, Not Specified, Australia

    • Sydney Children's Hospital

      Sydney, Not Specified, Australia

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    Study Plan

    This section provides details of the study plan, including how the study is designed and what the study is measuring.

    How is the study designed?

    Participant Group/ArmIntervention/Treatment
    EXPERIMENTAL: Phase 1b Dose finding: VHR induction - IRIVA
    • Irinotecan: an i.v. infusion over 1 hour on days 8,9,10,11 and 12 . For the phase 1b registration, starting dose of 20 mg/m2.
    • Ifosfamide: 3g/m2 as an i.v. infusion over 3 hours on days 1 and 2 Vincristine: 1.5 mg/m2 as an As per local practice: recommended as a short infusion (maximum dose 2mg). Administered days 1,8,15 on cycles 1-2 and on days 1 and 8 on cycles 3-9.
    • Actinomycin: 1.5 mg/m2 as an i.v. bolus injection (maximum dose 2mg) on day 1.
    DRUG: Irinotecan
    • antineoplastic enzyme inhibitor
    ACTIVE_COMPARATOR: CT1A: VHR induction - IVADO
    • Ifosfamide: 3g/m2 as an i.v. infusion over 3 hours on days 1 and 2 Vincristine: 1.5 mg/m2 As per local practice: recommended as a short infusion (maximum dose 2mg). Administered days 1,8,15 on cycles 1-2 and on day 1 on cycles 3-9.
    • Actinomycin: 1.5 mg/m2 as an i.v. bolus injection (maximum dose 2mg) on day 1. Doxorubicin: 30 mg/m2 as an i.v infusion over 1 hour on days 1 and 2 on cycles 1-4
    DRUG: Actinomycin D
    • Antineoplastic agent that is a polypeptide antibiotic
    EXPERIMENTAL: CT1A: VHR Induction IRIVA
    • Irinotecan: an i.v. infusion over 1 hour on days 8,9,10,11 and 12 . Phase 2 recommended dose as determined by IRIVA dose finding arm Ifosfamide: 3g/m2 as an i.v. infusion over 3 hours on days 1 and 2 Vincristine: 1.5 mg/m2 As per local practice: recommended as a short infusion (maximum dose 2mg). Administered days 1,8,15 on cycles 1-2 and on days 1 and 8 on cycles 3-9.
    • Actinomycin: 1.5 mg/m2 as an i.v. bolus injection (maximum dose 2mg) on day 1.
    DRUG: Irinotecan
    • antineoplastic enzyme inhibitor
    ACTIVE_COMPARATOR: CT1B: HR Induction IVA
    • Ifosfamide: 3g/m2 as an i.v. infusion over 3 hours on days 1 and 2 Vincristine: 1.5 mg/m2 As per local practice: recommended as a short infusion (maximum dose 2mg). Administered days 1,8,15 on cycles 1-2 and on day 1 on cycles 3-9.
    • Actinomycin: 1.5 mg/m2 as an i.v. bolus injection (maximum dose 2mg) on day 1.
    DRUG: Actinomycin D
    • Antineoplastic agent that is a polypeptide antibiotic
    EXPERIMENTAL: CT1B: HR Induction IRIVA
    • Irinotecan: an i.v. infusion over 1 hour on days 8,9,10,11 and 12 . Phase 2 recommended dose as determined by IRIVA dose finding arm Ifosfamide: 3g/m2 as an i.v. infusion over 3 hours on days 1 and 2 Vincristine: 1.5 mg/m2 As per local practice: recommended as a short infusion (maximum dose 2mg). Administered days 1,8,15 on cycles 1-2 and on days 1 and 8 on cycles 3-9.
    • Actinomycin: 1.5 mg/m2 as an i.v. bolus injection (maximum dose 2mg) on day 1.
    DRUG: Irinotecan
    • antineoplastic enzyme inhibitor
    EXPERIMENTAL: RT1A: Preoperative Radiotherapy
    • To be given either 41.4 Gy or 50.4 Gy prior to surgery
    RADIATION: radiotherapy
    • Ionising radiation
    ACTIVE_COMPARATOR: RT1A: Post operative radiotherapy
    • To be given either 41.4 Gy or 50.4 Gy following surgery
    RADIATION: radiotherapy
    • Ionising radiation
    EXPERIMENTAL: RT1B: Radiotherapy for resectable disease: dose escalated
    • To receive 50.4 Gy
    RADIATION: radiotherapy
    • Ionising radiation
    ACTIVE_COMPARATOR: RT1B: Radiotherapy for resectable disease: standard dose
    • To receive 41.4 Gy
    RADIATION: radiotherapy
    • Ionising radiation
    EXPERIMENTAL: RT1C: Radiotherapy for unresectable disease: dose escalated
    • To receive 59.4 Gy
    RADIATION: radiotherapy
    • Ionising radiation
    ACTIVE_COMPARATOR: RT1C: Radiotherapy for unresectable disease: standard dose
    • To receive 50.4 Gy
    RADIATION: radiotherapy
    • Ionising radiation
    EXPERIMENTAL: RT2: Radiotherapy to primary tumour and involved lymph nodes
    • Radiotherapy to the primary tumour and involved regional lymph nodes only
    RADIATION: radiotherapy
    • Ionising radiation
    EXPERIMENTAL: RT2: Radiotherapy to all metastatic sites
    • Radiotherapy given to all metastatic sites
    RADIATION: radiotherapy
    • Ionising radiation
    EXPERIMENTAL: CT2A: VHR Maintenance - VC
    • Vinorelbine: 25 mg/m2 i.v. or 60 mg/m2 orally on days 1,8 and 15 Cyclophosphamide 25 mg/m2 orally daily for 28 days
    DRUG: Vinorelbine
    • vinca alkaloid with a role as an antineoplastic agent
    NO_INTERVENTION: CT2A: Maintenance -Stop treatment
    • To stop treatment at the point of randomisation
    Not specified
    EXPERIMENTAL: CT2B: HR Maintenance - VC
    • Vinorelbine: 25 mg/m2 i.v. on days 1,8 and 15 Cyclophosphamide 25 mg/m2 orally daily for 28 days
    DRUG: Vinorelbine
    • vinca alkaloid with a role as an antineoplastic agent
    NO_INTERVENTION: CT2B: HR Maintenance - Stop Treatment
    • To stop treatment at the point of randomisation
    Not specified
    ACTIVE_COMPARATOR: CT3: Relpased Chemotherapy - VIRT
    • Vincristine: 1.5 mg/m2 As per local practice: recommended as a short infusion (maximum dose 2mg) on days 1 and 8 Irinotecan: 50 mg/m2 as an i.v. infusion over 1 hour on days 1-5 Temozolomide: 125 mg/m2 (Escalate to 150mg/m2/day in Cycle 2 if no toxicity \> grade 3) as an oral tablets prior to vincristine and irinotecan on days 1-5
    DRUG: Irinotecan
    • antineoplastic enzyme inhibitor
    EXPERIMENTAL: CT3: Relapsed Chemotherapy - VIRR
    • Vincristine: 1.5 mg/m2 As per local practice: recommended as a short infusion (maximum dose 2mg) on days 1 and 8 Irinotecan: 50 mg/m2 as an i.v. infusion over 1 hour on days 1-5 Regorafenib: Children between 6 and 24 months = 65 mg/m2, children less than 12 and/or less than 40kg dose = 82 mg/m2 Maximum 120 mg, Fixed dose of 120 mg for patients over 12 years of age AND ≥ 40 kg, as an oral tablets on days 8 to 21.
    DRUG: Irinotecan
    • antineoplastic enzyme inhibitor

    What is the study measuring?

    Primary outcome

    Primary Outcome MeasurePrimary Outcome DescriptionPrimary Outcome Time Frame
    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 neoplasmFrom randomisation to first failure event, timeframe 36 months
    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 neoplasmFrom randomisation to first failure event, timeframe 36 months
    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 neoplasmFrom randomisation to first failure event, timeframe 36 months
    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 neoplasmFrom randomisation to first failure event, timeframe 36 months
    Event Free Survival (CT2B)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 neoplasmTime from randomisation to first failure event, timeframe 36 months
    Event Free Survival (CT3)To determine whether new systemic therapy regimens improve event free survival in relapsed RMS compared to standard therapy (VIRT) (CT3): Initial new systemic therapy combination to be tested: o Regorafenib (R) added to vincristine and irinotecan (VIR) (VIRR)Patients will be followed up for a minimum of 6 years from trial entry (or 5 years from end of relapsed trial treatment, whichever comes later). Patients will be followed up for progression and death until the end of trial definition has been met.
    Local Failure Free Survival (RT1A and RT1B)A local failure event is relapse or progression of tumour at the primary site at any time even if there has been a prior /concurrent, regional or distant failureTime from randomisation to first local failure event, timeframe 36 months
    Local Failure Free Survival (RT1C)A local failure event is relapse or progression of tumour at the primary site at any time even if there has been a prior /concurrent, regional or distant failureTime from randomisation to first local failure event, timeframe 36 months

    Secondary outcome

    Secondary Outcome MeasureSecondary Outcome DescriptionSecondary Outcome Time Frame
    Recommended Phase II Dose (Phase 1b)Based on tolerability, where tolerability is evaluated through the occurrence of dose limiting toxicity (DLT).From first patient first visit in dose finding study until appropriate dose level found, estimated 9 months
    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.From first patient first visit in dose finding study until appropriate dose level
    Toxicity (All chemotherapy randomisations)Categorised and graded using Common Terminology Criteria for Adverse EventsFrom date of protocol defined treatment until 30 days after the administration of the last treatment
    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 toxicityFrom commencement of treatment until 21 days after the start of cycle 2 (each cycle is 21 days)
    Response (Phase 1b, CT1A, CT1B)defined as complete (CR) or partial response (PR) and is clinically defined. Patients who are not assessable for response - e.g. because of early stopping of treatment or death - will be assumed to be non-responders.Response assessed after course 3 (63 days) and 6 (126 days)
    Tolerability (CT3)To determine the tolerability of the regimens.From registration/randomisation until death/study endpoint
    Overall Survival (CT1A)Death from any causeFrom randomisation to death from any cause, assessed for 36 months
    Overall Survival (CT1B)Death from any causeFrom randomisation to death from any cause, assessed for 36 months
    Overall Survival (CT2A)Death from any causeFrom randomisation to death from any cause, assessed for 36 months
    Overall Survival (CT2B)Death from any causeFrom randomisation to death from any cause, assessed for 36 months
    Overall Survival (RT1A and RT1B)Death from any causeFrom randomisation to death from any cause, assessed for 36 months
    Overall Survival (RT1C)Death from any causeFrom RT1C randomisation to death from any cause, assessed for 36 months
    Overall Survival (RT2)Death from any causeFrom RT2 randomisation to death from any cause, as assessed for 36 months
    Overall Survival (CT3)To evaluate the anti-tumour activity and effect on overall survival of VIRR when compared to standard therapyPatients will be followed up for a minimum of 6 years from trial entry (or 5 years from end of relapsed trial treatment, whichever comes later). Patients will be followed up for progression and death until the end of trial definition has been met.
    Overall Survival (all patients)Death from any causeFrom randomisation/registration to death from any cause, assessed for 36 months
    Acute wound complications and post-operative complications (RT1A and RT1B)specific grade 3 and above complications according to CTCAE v 4 and Clavien Dindo scale. Specific wound complications within the same time frame will also be collectedWithin 120 days from surgery
    Acute post-radiotherapy complications (All radiotherapy randomisations)any grade 3 and above event according to CTCAE v 4Within 120 days from start of radiotherapy
    Late complications (RT1A, RT1B. RT1C)specific grade 3 and above events according to CTCAE and Clavien-Dindo scaleAfter 120 days from last local therapy
    Loco-regional failure-free survival (All radiotherapy randomisations)A local failure event is relapse or progression of tumour at the primary site at any time even if there has been a prior concurrent local, regional or distant failure. A regional event is relapse or progression of tumour at regional lymph nodes at any time even if there has been a prior distant failure.From randomisation to first local and/or regional failure event, assessed for 36 months
    Health related quality of life (RT1A and RT2) self-reported questionnaire completed by patientwill be assessed using Pediatric quality of life questionnaire (PedsQL) for the paediatric population (under 18 years). The minimum score is 0 where quality of life is completely unaffected by the intervention to 4 where quality of life is severely affected.4 timepoints: 1) 1 day of start of radiotherapy, 2) at completion of radiotherapy, average 5 weeks after start of radiotherapy, 3) 3 months and 4) 24 months following radiotherapy
    Health related quality of life (RT1A and RT2) self-reported questionnaire completed by the patientwill be assessed using European Organisation for Research and Treatment of Cancer, Quality of Life Questionnaire 30 (EORTC QLQ-C30) for patients 18 years of age and over. The minimum score is 0 where quality of life is completely unaffected by the intervention to 4 where quality of life is severely affected.4 timepoints: 1) 1 day of start of radiotherapy, 2) at completion of radiotherapy, average 5 weeks after start of radiotherapy, 3) 3 months and 4) 24 months following radiotherapy
    Health related quality of life (CT3) self-reported questionnaire completed by the patientwill be assessed using Pediatric quality of life questionnaire (PedsQL) for the paediatric population (under 18 years). The minimum score is 0 where quality of life is completely unaffected by the intervention to 4 where quality of life is severely affected.3 timepoints: Each Cycle is 28 days. Timepoint 1: Day 0 of cycle 1 (prior to starting treatment), Timepoint 2 day 0 cycle 3, Timepoint 3) day 0 cycle 5
    Health related quality of life (CT3) self-reported questionnaire completed by the patientwill be assessed using European Organisation for Research and Treatment of Cancer, Quality of Life Questionnaire 30 (EORTC QLQ-C30) for patients 18 years of age and over. The minimum score is 0 where quality of life is completely unaffected by the intervention to 4 where quality of life is severely affected.3 timepoints: Each Cycle is 28 days. Timepoint 1: Day 0 of cycle 1 (prior to starting treatment), Timepoint 2 day 0 cycle 3, Timepoint 3) day 0 cycle 5
    Acceptability and Palatability of Regorafenib (CT3)"Acceptability and Palatability Questionnaire" To evaluate the acceptability and palatability of regorafenib formulations1 timepoint: Day 8 of cycle 1 (Each Cycle is 28 days)
    PET Response (if participating in PET Sub-study)assessed by PERCIST criteria and visual 'Deauville like' criteriaAfter three cycles of chemotherapy (each cycle is 21 days)
    Event Free Survival (all patients)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 neoplasmFrom date of randomisation/registration to death from any cause, assessed for 36 months
    Event Free Survival (if participating in PET Sub-study)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 neoplasmFrom date of randomisation/registration to death from any cause, assessed for 36 months
    Local Failure Free Survival (if participating in PET Sub-study)A local failure event is relapse or progression of tumour at the primary site at any time even if there has been a prior /concurrent, regional or distant failureFrom date of randomisation/registration to first local failure event, assessed for 36 months

    Frequently Asked Questions

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    References

    Clinical Trials Gov: FaR-RMS: An Overarching Study for Children and Adults With Frontline and Relapsed RhabdoMyoSarcoma

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