NCT04625907

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

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 1,672 (estimated)
• Sponsor: University of Birmingham · Academic / Other
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

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

Detailed description

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.

Conditions

• Rhabdomyosarcoma

Interventions

Type	Name	Description
DRUG	Irinotecan	antineoplastic enzyme inhibitor
DRUG	Actinomycin D	Antineoplastic agent that is a polypeptide antibiotic
DRUG	Doxorubicin	An anthracycline topoisomerase inhibitor isolated from streptpmyces peucetius var. casesius
DRUG	Ifosfamide	chemotherapeutic agent chemically related to the nitrogen mustards and is a synthetic analog of cyclophosphamide
DRUG	Vincristine	anti neoplastic vinca alkaloid agent
DRUG	Vinorelbine	vinca alkaloid with a role as an antineoplastic agent
DRUG	Cyclophosphamide	Precursor of an alkylating nitrogen mustard antineoplastic and immunosuppressive agent
DRUG	Temozolomide	oral antineoplastic alkylating agent
RADIATION	radiotherapy	Ionising radiation
DRUG	Regorafenib	Oral 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).

Timeline

• Start date: 2020-09-17
• Primary completion: 2030-06-01
• Completion: 2030-06-01
• First posted: 2020-11-12
• Last updated: 2024-05-23

Locations

128 sites across 20 countries: Australia, Austria, Belgium, Czechia, Denmark, France, Greece, Ireland, Israel, Italy, Netherlands, New Zealand, Norway, Portugal, Slovakia, Slovenia, Spain, Sweden, Switzerland, United Kingdom