drugset / Trial / NCT00876031
Trial for Localised High-risk Rhabdomyosarcoma and Rhabdomyosarcoma-like Soft Tissue Sarcoma
Phase 3
Completed
195 enrolled
University Hospital Tuebingen
Cooperative Weichteilsarkom Study Group · collabDeutsche Kinderkrebsstiftung · collabGerman Society for Pediatric Oncology and Hematology GPOH gGmbH · collabGesellschaft fur Padiatrische Onkologie und Hamatologie - Austria · collabPolish Paediatric Solid Tumours Study Group · collabSwiss Pediatric Oncology Group · collabThe Swedish Childhood Solid Tumor Working Group · collab
RandomizedParallel-groupOpen-labelTreatment
Summary
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.
Timeline
- Start
- 2009-07-01
- Primary completion
- 2022-06-30
- Completion
- 2022-06-30
Outcome
Missed primary endpoint
paper HR 1.62, 95% CI 0.98-2.69, p = 0.06 PMID 39687431 ↗
paper The addition of 25 weeks of oral maintenance therapy with trofosfamide, etoposide and idarubicin after standard therapy does not improve EFS and OS PMID 39687431 ↗
Publications
- Background Dantonello TM, Int-Veen C, Harms D, Leuschner I, Schmidt BF, Herbst M, Juergens H, Scheel-Walter HG, Bielack SS, Klingebiel T, Dickerhoff R, Kirsch S, Brecht I, Schmelzle R, Greulich M, Gadner H, Greiner J, Marky I, Treuner J, Koscielniak E. Cooperative trial CWS-91 for localized soft tissue sarcoma in children, adolescents, and young adults. J Clin Oncol. 2009 Mar 20;27(9):1446-55. doi: 10.1200/JCO.2007.15.0466. Epub 2009 Feb 17.
- Background Mattke AC, Bailey EJ, Schuck A, Dantonello T, Leuschner I, Klingebiel T, Treuner J, Koscielniak E. Does the time-point of relapse influence outcome in pediatric rhabdomyosarcomas? Pediatr Blood Cancer. 2009 Jul;52(7):772-6. doi: 10.1002/pbc.21906.
- Background Dantonello TM, Int-Veen C, Winkler P, Leuschner I, Schuck A, Schmidt BF, Lochbuehler H, Kirsch S, Hallmen E, Veit-Friedrich I, Bielack SS, Niggli F, Kazanowska B, Ladenstein R, Wiebe T, Klingebiel T, Treuner J, Koscielniak E. Initial patient characteristics can predict pattern and risk of relapse in localized rhabdomyosarcoma. J Clin Oncol. 2008 Jan 20;26(3):406-13. doi: 10.1200/JCO.2007.12.2382.
- Background Weihkopf T, Blettner M, Dantonello T, Jung I, Klingebiel T, Koscielniak E, Luckel M, Spix C, Kaatsch P. Incidence and time trends of soft tissue sarcomas in German children 1985-2004 - a report from the population-based German Childhood Cancer Registry. Eur J Cancer. 2008 Feb;44(3):432-40. doi: 10.1016/j.ejca.2007.11.013.
- Background Koscielniak E, Morgan M, Treuner J. Soft tissue sarcoma in children: prognosis and management. Paediatr Drugs. 2002;4(1):21-8. doi: 10.2165/00128072-200204010-00003.
- Background Dantonello TM, Winkler P, Boelling T, Friedel G, Schmid I, Mattke AC, Ljungman G, Bielack SS, Klingebiel T, Koscielniak E; CWS Study Group. Embryonal rhabdomyosarcoma with metastases confined to the lungs: report from the CWS Study Group. Pediatr Blood Cancer. 2011 May;56(5):725-32. doi: 10.1002/pbc.22862. Epub 2010 Nov 8.
- Background Siepermann M, Koscielniak E, Dantonello T, Klee D, Boos J, Krefeld B, Borkhardt A, Hoehn T, Asea A, Wessalowski R. Oral low-dose chemotherapy: successful treatment of an alveolar rhabdomyosarcoma during pregnancy. Pediatr Blood Cancer. 2012 Jan;58(1):104-6. doi: 10.1002/pbc.22934. Epub 2011 Jan 16.
- Results Klingebiel T, Boos J, Beske F, Hallmen E, Int-Veen C, Dantonello T, Treuner J, Gadner H, Marky I, Kazanowska B, Koscielniak E. Treatment of children with metastatic soft tissue sarcoma with oral maintenance compared to high dose chemotherapy: report of the HD CWS-96 trial. Pediatr Blood Cancer. 2008 Apr;50(4):739-45. doi: 10.1002/pbc.21494.
- Koscielniak E, Ljungman G, Kazanowska B, Niggli F, Sparber-Sauer M, Handgretinger R, Zimmermann M, Boos J, Blank B, Hallmen E, Teichert von Luttichau I, Schmid I, Frohlich B, Muller HL, Behnisch W, Ladenstein R, Scheer M, Vokuhl C, von Kalle T, Blattmann C, Bielack S, Klingebiel T. Maintenance therapy with trofosfamide, idarubicin and etoposide in patients with rhabdomyosarcoma and other high-risk soft tissue sarcomas (CWS-2007-HR): a multicentre, open-label, randomised controlled phase 3 trial. EClinicalMedicine. 2024 Nov 29;78:102957. doi: 10.1016/j.eclinm.2024.102957. eCollection 2024 Dec.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Etoposide | Small molecule | 25 mg/m2 | Oral |
| Subject | Idarubicin | Small molecule | 5 mg/m2 | Oral |
| Subject | Trofosfamide | Small molecule | 75 mg/m2 | Oral |