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.|
|ClinicalTrials.gov Identifier: NCT03462888|
Recruitment Status : Recruiting
First Posted : March 13, 2018
Last Update Posted : July 5, 2019
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|
|Study Type :||Observational|
|Estimated Enrollment :||150 participants|
|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|
- 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).
- 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
- 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)
- therapeutic strategy [ Time Frame: 9 months ]Description of the therapeutic strategy according to the different type of patients (paediatric, AYA, or adult):
- 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
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 ClinicalTrials.gov identifier (NCT number): NCT03462888
|Contact: Anne-Sophie DEFACHELLES, MD||0320295959||as-Defachelles@o-lambret.fr|
|Contact: Laurène FENWARTHemail@example.com|
|Centre Oscar Lambret/ CHRU de Lille||Recruiting|
|Lille, France, 59020|
|Contact: Laurène FENWARTH 03 20 29 59 59 firstname.lastname@example.org|
|Contact: Anne-Sophie DEFACHELLES, MD 03 20 29 59 59 as-Defachelles@o-lambret.fr|
|Contact: Nadège CORRADINI, MD|
|MARSEILLE La Timone||Completed|
|CHU de Nancy||Recruiting|
|Nancy, France, 54035|
|Contact: Ludovic MANSUY, MD 03.83.15.46.31 email@example.com|
|Contact: Pascal CHASTAGNER, MD 03.83.15.46.31 firstname.lastname@example.org|
|Contact: Maria RIOS, MD|
|Centre de Lacassagne à||Completed|
|Contact: Maryline POIREE, MD|
|Contact: Frédéric MILLOT MILLOT, MD|
|Institut Claudius Regaud||Completed|
|Toulouse, France, 31059|
|TOURS CHU-Bretonneau/Hôp G de Clocheville||Completed|
|Contact: Laurène FENWARTH, MD|