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

NCT ID: NCT00876031

Last Updated: 2022-11-08

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

195 participants

Study Classification

INTERVENTIONAL

Study Start Date

2009-07-01

Study Completion Date

2022-06-30

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.

Detailed Description

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Conditions

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Soft Tissue Sarcoma

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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O-TIE

oral maintenance therapy with trofosfamide, idarubicin, and etoposide

Group Type EXPERIMENTAL

trofosfamide, idarubicin, etoposide

Intervention Type DRUG

oral maintenance therapy for 6 months

control

Group Type NO_INTERVENTION

No interventions assigned to this group

Interventions

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trofosfamide, idarubicin, etoposide

oral maintenance therapy for 6 months

Intervention Type DRUG

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)
Minimum Eligible Age

6 Months

Maximum Eligible Age

21 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Cooperative Weichteilsarkom Study Group

OTHER

Sponsor Role collaborator

Deutsche Kinderkrebsstiftung

OTHER

Sponsor Role collaborator

German Society for Pediatric Oncology and Hematology GPOH gGmbH

OTHER

Sponsor Role collaborator

Gesellschaft fur Padiatrische Onkologie und Hamatologie - Austria

OTHER

Sponsor Role collaborator

The Swedish Childhood Solid Tumor Working Group

UNKNOWN

Sponsor Role collaborator

Polish Paediatric Solid Tumours Study Group

UNKNOWN

Sponsor Role collaborator

Swiss Pediatric Oncology Group

OTHER

Sponsor Role collaborator

University Hospital Tuebingen

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Ewa Koscielniak, MD

Role: PRINCIPAL_INVESTIGATOR

Olgahospital, CWS

Thomas Klingebiel, MD

Role: STUDY_CHAIR

Universitätsklinikum Frankfurt, CWS

Monika Sparber Sauer, MD

Role: STUDY_DIRECTOR

Olgahospital, CWS

Locations

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Krankenhaus der Stadt Dornbirn

Dornbirn, , Austria

Site Status

Universitätsklinik für Kinder- und Jugendheilkunde

Graz, , Austria

Site Status

Universitätsklinik für Kinder- und Jugendheilkunde

Innsbruck, , Austria

Site Status

Landeskrankenhaus

Klagenfurt, , Austria

Site Status

Landeskrankenhaus

Leoben, , Austria

Site Status

Landesfrauen - und Kinderklinik Linz

Linz, , Austria

Site Status

St. Johanns Spital / Landeskrankenhaus

Salzburg, , Austria

Site Status

St. Anna Kinderspital

Vienna, , Austria

Site Status

Universitätsklinikum

Aachen, , Germany

Site Status

Klinikum Augsburg

Augsburg, , Germany

Site Status

Charité

Berlin, , Germany

Site Status

Helios Klinikum Berlin-Buch

Berlin, , Germany

Site Status

Krankenhaus Bielefeld

Bielefeld, , Germany

Site Status

Universitätskinderklinik

Bonn, , Germany

Site Status

Klinikum Braunschweig

Braunschweig, , Germany

Site Status

Prof. Hess Kinderklinik

Bremen, , Germany

Site Status

Klinikum Chemnitz

Chemnitz, , Germany

Site Status

Kliniken der Stadt Köln

Cologne, , Germany

Site Status

Universitätsklinikum

Cologne, , Germany

Site Status

Vestische Kinderklinik

Datteln, , Germany

Site Status

Westfälisches Kinderzentrum, Klinikum Dortmund

Dortmund, , Germany

Site Status

Universitätsklinikum

Dresden, , Germany

Site Status

Universitätskinderklinik

Düsseldorf, , Germany

Site Status

Helios Klinikum

Erfurt, , Germany

Site Status

Universitätsklinikum

Erlangen, , Germany

Site Status

Universitätsklinikum

Essen, , Germany

Site Status

Universitätsklinikum

Frankfurt, , Germany

Site Status

Universitätsklinikum

Freiburg im Breisgau, , Germany

Site Status

Universitätsklinikum

Giessen, , Germany

Site Status

Universitätsklinikum

Göttingen, , Germany

Site Status

Universitätsklinikum

Greifswald, , Germany

Site Status

Universitätsklinikum

Halle, , Germany

Site Status

Universitätsklinikum

Hamburg, , Germany

Site Status

Medizinische Hochschule

Hanover, , Germany

Site Status

Universitätsklinikum

Heidelberg, , Germany

Site Status

Gemeinschaftskrankenhaus Herdecke

Herdecke, , Germany

Site Status

Universitätsklinikum

Homburg, , Germany

Site Status

Universitätsklinikum

Jena, , Germany

Site Status

Städtisches Klinikum Karlsruhe

Karlsruhe, , Germany

Site Status

Klinikum Kassel

Kassel, , Germany

Site Status

Universitätsklinikum

Kiel, , Germany

Site Status

Gemeinschaftsklinikum Mayen-Koblenz

Koblenz, , Germany

Site Status

Universitätsklinikum

Leipzig, , Germany

Site Status

Universitätsklinikum

Lübeck, , Germany

Site Status

Universitätsklinikum

Magdeburg, , Germany

Site Status

Universitätsklinikum

Mainz, , Germany

Site Status

Universitätsklinikum

Mannheim, , Germany

Site Status

Klinikum Minden

Minden, , Germany

Site Status

Dr. von Haunersches Kinderspital

München, , Germany

Site Status

Krankenhaus München Schwabing

München, , Germany

Site Status

Universitätsklinikum

Münster, , Germany

Site Status

Cnopf'sche Kinderklinik

Nuremberg, , Germany

Site Status

Klinikum Oldenburg

Oldenburg, , Germany

Site Status

Universitätsklinikum

Regensburg, , Germany

Site Status

Universitätsklinikum

Rostock, , Germany

Site Status

Asklepios Kinderklinik

Sankt Augustin, , Germany

Site Status

Helios Kliniken Schwerin

Schwerin, , Germany

Site Status

Olgahospital

Stuttgart, , Germany

Site Status

Mutterhaus der Borromäerinnen

Trier, , Germany

Site Status

Universitätsklinikum

Tübingen, , Germany

Site Status

Universitätsklinikum

Ulm, , Germany

Site Status

Universitätsklinikum

Würzburg, , Germany

Site Status

Medical University

Bialystok, , Poland

Site Status

Collegium Medicum

Bydgoszcz, , Poland

Site Status

Medical Academy

Gdansk, , Poland

Site Status

Silesian Medical Academy

Katowice, , Poland

Site Status

Polish-American Institute of Paediatrics Jagiellonian University Medical College

Krakow, , Poland

Site Status

Medical Academy

Lodz, , Poland

Site Status

Medical Academy

Lublin, , Poland

Site Status

Pian Medical University

Szczecin, , Poland

Site Status

Department of Surgical Oncology for Children and Youth

Warsaw, , Poland

Site Status

Medical University

Wroclaw, , Poland

Site Status

Sahlgrenska University Hospital, The Queen Siliva Childrens Hospital

Gothenburg, , Sweden

Site Status

University Hospital, Dept. of Pediatrics

Linköping, , Sweden

Site Status

University Hospital, Dept. of Pediatric Hematology and Oncology

Lund, , Sweden

Site Status

Karolinska University Hospital, Astrid Lindgrens Children's Hospital

Stockholm, , Sweden

Site Status

Norrland's University Hospital, Dept. of Pediatrics

Umeå, , Sweden

Site Status

Children's University Hospital

Uppsala, , Sweden

Site Status

Kantonsspital

Aarau, , Switzerland

Site Status

Universitätskinderklinik beider Basel

Basel, , Switzerland

Site Status

Ospedale San Giovanni

Bellinzona, , Switzerland

Site Status

Inselspital University hospital

Bern, , Switzerland

Site Status

Hopital Cantonal de Geneve

Geneva, , Switzerland

Site Status

CHUV

Lausanne, , Switzerland

Site Status

Kinderspital

Lucerne, , Switzerland

Site Status

Ostschweizer Kinderspital

Sankt Gallen, , Switzerland

Site Status

University Children's Hospital

Zurich, , Switzerland

Site Status

Countries

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Austria Germany Poland Sweden Switzerland

References

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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.

Reference Type BACKGROUND
PMID: 19224858 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 19165889 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 18202417 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 18077150 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 11817983 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 21370403 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 22076833 (View on PubMed)

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.

Reference Type RESULT
PMID: 18286501 (View on PubMed)

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.

Reference Type DERIVED
PMID: 39687431 (View on PubMed)

Related Links

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Other Identifiers

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4033024 (BfArM)

Identifier Type: OTHER

Identifier Source: secondary_id

293/2007AMG1 (Ethikkommission)

Identifier Type: OTHER

Identifier Source: secondary_id

2007-001478-10

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

A2007/14 (Kinderkrebsstiftung)

Identifier Type: OTHER

Identifier Source: secondary_id

498 (Krebsstudienregister)

Identifier Type: REGISTRY

Identifier Source: secondary_id

CWS-2007-HR

Identifier Type: -

Identifier Source: org_study_id

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