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Clinico-biological Study/Characterization of Rhabdomyosarcoma in Adolescents and Young Adults, 15-25-year-old Patients (RMS-AJA-1701)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03462888
Recruitment Status : Recruiting
First Posted : March 13, 2018
Last Update Posted : July 5, 2019
Centre Leon Berard
Société Française de lutte contre les Cancers et les leucémies de l'Enfant et l'adolescent
French Sarcoma Group
Ligue contre le cancer, France
Information provided by (Responsible Party):
Centre Oscar Lambret

Brief Summary:

Rhabdomyosarcoma (RMS) stands for the most frequent soft tissue sarcoma in children and, adolescents and young adults (AYA, 15-25-year-old population), accounting for approximately half of the whole soft tissue sarcomas in these populations.. Conversely, RMS represents a very small proportion of the soft tissue sarcomas in adults (3%), that is less than 1% of all solid cancers of adults.

To date, previous studies undertaken among the paediatric population have pointed out several prognostic factors such as tumor localisation, tumor invasiveness at diagnosis, tumor size, histological subset, and treatment plans. Age at diagnosis remains an independent prognostic factor.

RMS management is consensual in Europe for paediatric population, essentially based on the protocol RMS 2005 within the framework of the European Paediatric Soft tissue sarcoma Study Group (EpSSG). Care in AYAs remain heterogeneous and are either achieved in paediatric department, according to EpSSG guidelines, or in oncology department, known as "adult unit", depending on ESMO (European Society for Medical Oncology), which are non-specific recommendations for the management of rhabdomyosarcoma.

No consensus has been published yet for RMS in AYA despite the growing interest in cancers in AYA population - topic.supported by the French National Cancer Institute (INCa) - and the increasing network between paediatricians and adult-oncologists. Thus management of RMS in AYA remains patchy/unequal depending on the type of care unit.

Herein, with the support of the Oscar Lambret Center, we aim at assessing and identifying clinico-biological prognostic factors of rhabdomyosarcoma in AYA. Eventually, we hope to offer a standardized treatment to this population. Data collected from medical file will be anonymised in a confidential database of which the recipient is the sponsor of the study.

The ancillary study will aim at characterizing the molecular profile of the difficult-to-classify RMS subtypes (fusiform or pleomorphic subsets) in molecular biology for ambiguous cases.

From a scientific point of view, this study aims at understanding the parameters that may influence the prognosis of RMS in AYAs by evaluating various clinical and biological factors.

Biologically, molecular profiling of RMS in AYA may improve the characterization of this tumour in this age group.

At the clinical level, the completeness of the data collected will lead to a better description of RMS in AYAs. We hope to harmonize their therapeutic management by providing therapeutic adjustments according to population subsets.

Finally, these results could also help to adapt the therapeutic management of AYAs within the framework of the European protocol that is currently under construction, and will involve both children and adults.

Condition or disease

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Study Type : Observational
Estimated Enrollment : 150 participants
Observational Model: Cohort
Time Perspective: Retrospective
Official Title: Clinico-biological Study/Characterization of Rhabdomyosarcoma in AYA (Adolescents and Young Adults, 15-25-year-old Patients)
Actual Study Start Date : February 2, 2018
Estimated Primary Completion Date : October 2020
Estimated Study Completion Date : October 2020

Resource links provided by the National Library of Medicine

Primary Outcome Measures :
  1. Progression-free survival [ Time Frame: 3 years after diagnosis ]
    To estimate progression-free survival in AYA patients with RMS, according to clinical factors known as prognostic in children, and of biological factors (MYOD1 mutation in non-alveolar tumor; FOXO1).

Secondary Outcome Measures :
  1. Clinical parameters of RMS in AYA [ Time Frame: 9 months ]
    Clinical data from the different databases will be merged using a standardized format in order to describe the Clinical parameters of RMS in AYA

  2. Biological parameters of RMS in AYA [ Time Frame: 9 months ]
    Description of the RMS in AYA using biological characteristics (FOXO1, MYOD1 - where appropriate -, CGH-array profile)

  3. therapeutic strategy [ Time Frame: 9 months ]
    Description of the therapeutic strategy according to the different type of patients (paediatric, AYA, or adult):

  4. Overall survival (OS) [ Time Frame: 3 years after diagnosis ]
    OS will be defined as the time from diagnosis to death or last contact with the patient.

Biospecimen Retention:   Samples With DNA
Tissue extracted at diagnosis from biopsy or from surgical resection of the primary tumor

Information from the National Library of Medicine

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Ages Eligible for Study:   15 Years to 25 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Sampling Method:   Non-Probability Sample
Study Population
  • Patients aged from 15 to 25 years old at diagnosis
  • Patients registered in EpSSG RMS 2005 trial, or RRePS / NerSarc /

Inclusion Criteria:

  • Patients aged from 15 to 25 years old at diagnosis
  • Patients registered in EpSSG RMS 2005 trial, or RRePS / NerSarc / Conticabase databases
  • Over the period from 2006 to 2014
  • Rhabdomyosarcoma histologically proven
  • Localized or metastatic
  • Histological review and molecular biology available
  • No previous treatment except surgery
  • No previous cancer
  • Absence of known serious chronic illness
  • Patient and/or parents information and non-opposition to data collection

Exclusion Criteria:

  • Age over 25 years, or under 15 years
  • Other histologies

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03462888

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Contact: Anne-Sophie DEFACHELLES, MD 0320295959
Contact: Laurène FENWARTH 0320295918

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BORDEAUX Bergonié Completed
Bordeaux, France
Bordeaux Chu Completed
Bordeaux, France
Brest Chu Completed
Brest, France
Centre Oscar Lambret/ CHRU de Lille Recruiting
Lille, France, 59020
Contact: Laurène FENWARTH    03 20 29 59 59   
Contact: Anne-Sophie DEFACHELLES, MD    03 20 29 59 59   
LYON- Bérard Recruiting
Lyon, France
Contact: Nadège CORRADINI, MD         
MARSEILLE La Timone Completed
Marseille, France
CHU de Nancy Recruiting
Nancy, France, 54035
Contact: Ludovic MANSUY, MD   
Contact: Pascal CHASTAGNER, MD   
NANCY Vautrin Recruiting
Nancy, France
Contact: Maria RIOS, MD         
Centre de Lacassagne à Completed
Nice, France
NICE CHU Recruiting
Nice, France
Contact: Maryline POIREE, MD         
Poitiers Chu Recruiting
Poitiers, France
Contact: Frédéric MILLOT MILLOT, MD         
Strasbourg Chru Completed
Strasbourg, France
Institut Claudius Regaud Completed
Toulouse, France, 31059
TOURS CHU-Bretonneau/Hôp G de Clocheville Completed
Tours, France
Villejuif Igr Recruiting
Villejuif, France
Contact: Laurène FENWARTH, MD         
Sponsors and Collaborators
Centre Oscar Lambret
Centre Leon Berard
Société Française de lutte contre les Cancers et les leucémies de l'Enfant et l'adolescent
French Sarcoma Group
Ligue contre le cancer, France
Additional Information:
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Responsible Party: Centre Oscar Lambret Identifier: NCT03462888    
Other Study ID Numbers: RMS-AJA-1701
First Posted: March 13, 2018    Key Record Dates
Last Update Posted: July 5, 2019
Last Verified: July 2019

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Centre Oscar Lambret:
Adolescents and young adults
prognostic factors
molecular profiling
Additional relevant MeSH terms:
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Neoplasms, Muscle Tissue
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type