Brief Summary
FaR-RMS is an over-arching study for children and adults with newly diagnosed and relapsed rhabdomyosarcoma (RMS).
Intervention / Treatment
- Drug: Irinotecan
- Drug: Actinomycin D
- Drug: Doxorubicin
- Drug: Ifosfamide
- Drug: Vincristine
- Drug: Vinorelbine
- Drug: Temozolomide
- Radiation: radiotherapy
- Drug: Regorafenib
Inclusion Criteria for study entry – Mandatory at first point of study entry
- Histologically confirmed diagnosisthe process of identifying a disease based on signs and symptoms, patient history and medical test results of RMS (except pleomorphicmany forms; cells that have different size, shape etc. RMS)
- Written informed consenta process in which a patient receives detailed information about a procedure or treatment, including its potential risks, benefits, and alternatives. The patient then has the opportunity to understand the information and voluntarily agree to the procedure or treatment. from the patient and/or the parent/legal guardian
Phase 1b Dosethe amount of medication taken Finding – IRIVA Inclusion
- Entered in to the FaR-RMS study at diagnosis
- Very High Riskthe possibility that something bad will happen disease
- Age >12 months and ≤25 years
- No prior treatment for RMS other than surgerytreatment involving removal of cancerous tissue and/or tumours and a margin of healthy tissue around it to reduce recurrence
- 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
- Plateletssmall disc-shaped blood cells that clump together to form clots to stop bleeding ≥ 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
- Written informed consent from the patient and/or the parent/legal guardian