Essai clinique REGO-INTER-EWING


This trial on Ewing sarcoma was co-funded by the Enfants sans Cancer 2021 race and was selected as part of our European Fight Kids Cancer grant call.




About Ewing Sarcoma

Ewing sarcoma is the second most common malignant bone tumor after osteosarcoma among adolescents and young adults. It affects approximately 80 to 100 new patients per year in France. The majority of cases (70%) occur in young people aged 5 to 25 years. However, these tumors can be diagnosed before 5 years of age and after 30 years.

Most Ewing sarcomas develop in bone, but 15% can develop independently of bone. Unfortunately, metastases are detected in one-third of patients at diagnosis. These metastases are found in 50% of cases only in the lungs, less frequently in other bones or bone marrow. Other factors influencing severity include tumor location, size, and response to treatment.


About Rego-Inter-Ewing 1

The efficacy of new treatments for Ewing sarcoma has been disappointing in recent decades, with no successful introduction of new drugs in first-line treatment thus far. Among the drugs tested, initial clinical data suggest that strategies using multi-targeted tyrosine kinase inhibitors (TKIs) with anti-angiogenic activities (limiting tumor blood vessel development) are among the most effective and could be beneficial. Protein kinases are small proteins essential for cell survival as they play roles in numerous cellular mechanisms (communication, energy, proliferation, etc.).

Several TKIs have been and are currently being tested as monotherapy in patients with relapsed or refractory Ewing sarcoma, with promising results in phase II trials. Among them, regorafenib has shown promising activity in relapsed Ewing sarcoma. However, regorafenib has never been combined with the intensive chemotherapy regimen VDC/IE (vincristine, doxorubicin, cyclophosphamide, ifosfamide, and etoposide). This combination therefore needs evaluation to avoid dose reduction of the current standard treatment and maintain its effectiveness.

Rego-Inter-Ewing 1 is a clinical trial designed to test the feasibility of regorafenib with conventional chemotherapy. It consists of a phase Ib trial recruiting only patients with multi-metastatic Ewing sarcoma (beyond lungs/pleura), representing the highest unmet medical need in childhood cancers (2-year event-free survival: 33%, similar to relapsed/refractory patients).


Summary of the REGO-INTER-EWING 1 Project

  • Lead Investigator: Dr. Pablo Berlanga
  • Program Duration: January 2022 – January 2025
  • Trial Opening: December 14, 2022
  • Expected Number of Patients: 24 (4 enrolled)
  • Countries Involved: France (5 centers), Netherlands (1 center), Australia (4 centers)
  • Presentation at Conferences: SIOPE 2023 (Vienna)
  • Fight Kids Cancer Funding: €784,001, including Imagine for Margo funding: €372,060


essai clinique sarcome d'ewing REGO-INTER-EWING


This project aims to advance treatment options for Ewing sarcoma through innovative combinations of regorafenib and conventional chemotherapy, addressing critical gaps in current therapeutic strategies for this challenging pediatric cancer.