Trial for Localised High-risk Rhabdomyosarcoma and Rhabdomyosarcoma-like Soft Tissue Sarcoma

Study Overview
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Brief Summary
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The purpose of this study is to investigate whether the addition of oral maintenance chemotherapy with O-TIE (Etoposide, Idarubicin, Trofosfamide) for 6 months improves the event free survival (EFS) in patients with localised high-risk RMS and RMS-like Soft Tissue Sarcoma.

Official Title
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A Randomised Phase-III Trial of the Cooperative Weichteilsarkom Study Group (CWS) for Localised High-risk Rhabdomyosarcoma and Localised Rhabdomyosarcoma-like Soft Tissue Sarcoma in Children, Adolescents, and Young Adults

Conditions
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Soft Tissue Sarcoma

Intervention / Treatment
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* Drug: trofosfamide, idarubicin, etoposide


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: written informed consent for registration, randomisation, data collection/transfer, and tumour material asservation available pathologically (including molecular pathology) proven diagnosis of rhabdomyosarcoma (RMS) or RMS-like soft tissue sarcoma (STS) and tumour material available for pathology review Rhabdomyosarcoma of the High Risk Group, i.e.: RME, N0, M0, IRS II&III, >5 cm or >10 years in EXT, HN-PM, OTH, UG-BP RME, N1, M0, any IRS-group, any size or age RMA, NO, M0, any IRS-group, any size or age (exception: paratesticular RMA are not eligible) or Rhabdomyosarcoma of the Very High Risk Group, i.e.: RMA, N1, MO, IRS II&III, any size or age or localised high-risk RMS-like Soft Tissue Sarcoma, i.e.: EES, pPNET, UDS: any N, M0, any IRS-group, any size or age SySa, any N, M0, any size or age (exception: SySa IRSI&II, not T2b, N0, M0 are not eligible) no pre-existing illness preventing treatment (esp. those listed in the medicinal product information, e.g. cardiac, hepatic, metabolic, or renal dysfunction; hypersensitivity) no previous malignant tumours available for long term follow up through the treating centre in remission (according to the CWS-2007-HR definition (see X7.1.8X)) at the time of randomisation after standard multimodal therapy e.g. according to the CWS-guidance Exclusion Criteria: pregnant or lactating women other medical condition precluding treatment with protocol therapy (e.g. HIV, psychiatric disorder, etc.) for sexually active females and males in Arm B (O-TIE treatment): refusal to use effective contraception (e.g. oral, IUD)

Ages Eligible for Study
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Minimum Age: 6 Months
Maximum Age: 21 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: O-TIE<br>oral maintenance therapy with trofosfamide, idarubicin, and etoposide | Drug: trofosfamide, idarubicin, etoposide<br>* oral maintenance therapy for 6 months<br>|
| No Intervention: control<br> | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| event free survival (EFS) |  | 3 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall survival |  | 5 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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pediatric soft tissue sarcoma, rhabdomyosarcoma, extraosseous Ewing family tumours, synovial sarcoma, undifferentiated sarcoma