Clinical trial NCT04845893: Observational Study on Skeletal Ewing's Sarcoma
Official title: Observational Study on Treatment of Skeletal Ewing Sarcoma at Diagnosis
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Ewing Sarcoma of Bone
Keywords: Ewing Sarcoma
Lead sponsor: Italian Sarcoma Group
Sponsor class: NETWORK
First posted: 2021-04-15
Enrollment: 100
Sex: ALL
Minimum age (years): 0
Maximum age (years): 49
Standard ages: CHILD
ADULT
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with Ewing's sarcoma of the bone or with Ewing-like sarcoma (round cell sarcoma with fusion of the EWSR1 gene with others not of the Ewings' Tumor Sarcoma family, sarcoma with Capicua Transcriptional Repressor (CIC) rearrangement or sarcoma with BCL6 corepressor (BCOR) rearrangement

Brief summary: Observational prospective trial aimed to collect the collect demographic, clinical, surgical, pathological and molecular characteristics and treatment from patients affected by skeletal Ewing Sarcoma

Detailed description: The Ewing sarcoma treatment is based on chemotherapy, surgery and radiotherapy. Chemotherapy, performed with a combination of several drugs, is given as primary treatment before surgery.

Surgical intervention must be performed in all cases in which it is possible to obtain an excision with free margins and with acceptable functional deficits. However, when, after surgery in case of incomplete resection, post-operative radiotherapy is used, in order to reduce the risk of local recurrence as much as possible.

Subsequently in the local treatment, a phase of maintenance chemotherapy is foreseen, the intensity of which is modulated according to the response to induction therapy.

The treatments are carried out with different programs for patients with localized disease or metastatic disease and derive from clinical trials carried out in the last 30 years.

These results are the result of national and international prospective trials which, precisely due to the rarity of the disease, took many years to execute, and it is significant to know that randomized studies were in very limited numbers, as almost all the studies were represented by studies of uncontrolled phase II Improving treatments requires the expansion of knowledge on the biological behavior of this tumor and the acquisition of as much information as possible deriving from clinical experience.

For this reason, pending the definition of a new prospective randomized trial, which will take time (approximately two years) to be made operational, it is considered useful from a scientific point of view to prospectively collect data relating to new cases of Ewing's sarcoma 'bone.

Eligibility criteria: Inclusion Criteria:

1. Diagnosis of Ewing's sarcoma of the bone
2. Diagnosis of Ewing-like sarcoma (round cell sarcoma with fusion of the EWSR1 gene with others not of the ETS family, sarcoma with CIC rearrangement or sarcoma with BCOR rearrangement)
3. Diagnosis of extraosseous Ewing's sarcoma for patients
4. Age at diagnosis \<50 years
5. Assessment of the extent of the disease at onset in accordance with the European Society of Medical Oncology guidelines
6. Patients or parents oe guardians of minors who have given their written informed consent to participate in the study

Exclusion Criteria:

1. Presence of comorbid factors who can compromise the compliance of the treatment plan or the evaluation of the outcomes, including but not limited to organ pathologies that contraindicate the use of chemotherapy and psychological or social conditions that do not allow for adequate compliance treatment or adequate follow-up

Primary outcomes:
measure: 5 years Event Free Survival (EFS)
timeFrame: 5 years
Secondary outcomes:
measure: Overall Survival (OS)
timeFrame: at 5 years
measure: Adverse events related to the treatments in patients with age >21 years at the time of diagnosis
timeFrame: Every 3 weeks (Week 3, week 6, week 9, week 12, ...) up to 30 months
measure: To assess baseline clinical and disease-specific factors with possible impact on survival analyses in the subpopulation with Ewing-like sarcoma
timeFrame: Baseline
Other outcomes:
Locations:
city: Rozzano
country: Italy
facility: Istituto Clinico Humanitas
state: MI
city: Aviano
country: Italy
facility: Centro di Riferimento Oncologico - Unit of Medical Oncology
state: Pordenone
city: Candiolo
country: Italy
facility: I.R.C.C. - Unit of Medical Oncology
state: Torino
city: Bari
country: Italy
facility: Azienda ospedaliero universitaria consorziale policlinico - bari
city: Bologna
country: Italy
facility: A.O. Universitaria Policlinico S. Orsola Malpighi di Bologna
city: Bologna
country: Italy
facility: Istituto ortopedico Rizzoli
city: Firenze
country: Italy
facility: A.O. Universitaria Meyer
city: Genova
country: Italy
facility: Istituto Giannina Gaslini
city: Milano
country: Italy
facility: FONDAZIONE IRCCS Istituto Nazionale dei Tumori
city: Milano
country: Italy
facility: FONDAZIONE IRCCS Istituto Nazionale dei Tumori
city: Napoli
country: Italy
facility: Azienda Ospedaliera Santobono Pausilipon
city: Padova
country: Italy
facility: Azienda Ospedaliera di Padova
city: Palermo
country: Italy
facility: ARNAS P. O. "Civico e Benfratelli"
city: Parma
country: Italy
facility: Azienda Ospedaliera Universitaria di Parma
city: Pisa
country: Italy
facility: Azienda Ospedaliero-Universitaria Pisana
city: Roma
country: Italy
facility: Ospedale Pediatrico Bambin Gesu'
city: Rome
country: Italy
facility: Istituto Regina Elena - IFO
city: Torino
country: Italy
facility: Ospedale Infantile Regina Margherita - Unit of Paediatric Oncoematology
city: Trieste
country: Italy
facility: IRCCS materno infantile Burlo Garofolo
Study officials:
References:
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citation: Casali PG, Bielack S, Abecassis N, Aro HT, Bauer S, Biagini R, Bonvalot S, Boukovinas I, Bovee JVMG, Brennan B, Brodowicz T, Broto JM, Brugieres L, Buonadonna A, De Alava E, Dei Tos AP, Del Muro XG, Dileo P, Dhooge C, Eriksson M, Fagioli F, Fedenko A, Ferraresi V, Ferrari A, Ferrari S, Frezza AM, Gaspar N, Gasperoni S, Gelderblom H, Gil T, Grignani G, Gronchi A, Haas RL, Hassan B, Hecker-Nolting S, Hohenberger P, Issels R, Joensuu H, Jones RL, Judson I, Jutte P, Kaal S, Kager L, Kasper B, Kopeckova K, Krakorova DA, Ladenstein R, Le Cesne A, Lugowska I, Merimsky O, Montemurro M, Morland B, Pantaleo MA, Piana R, Picci P, Piperno-Neumann S, Pousa AL, Reichardt P, Robinson MH, Rutkowski P, Safwat AA, Schoffski P, Sleijfer S, Stacchiotti S, Strauss SJ, Sundby Hall K, Unk M, Van Coevorden F, van der Graaf WTA, Whelan J, Wardelmann E, Zaikova O, Blay JY; ESMO Guidelines Committee, PaedCan and ERN EURACAN. Bone sarcomas: ESMO-PaedCan-EURACAN Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2018 Oct 1;29(Suppl 4):iv79-iv95. doi: 10.1093/annonc/mdy310. No abstract available.
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