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Clinical Trials in Australia / NCT05830084
Active, not recruiting Phase 1

Phase Ib / Regorafenib With Conventional Chemotherapy/Newly Diagnosed Patients/ Multimetastatic Ewing Sarcoma

NCT05830084 · tracked via the Priya Life Science Australia tracker
Phase
Phase 1
Started
2023-06-16
Last updated
2026-01-06

Condition(s) studied

Ewing Sarcoma

Investigational drug(s) / intervention(s)

regorafenib tablet →

regorafenib tablet: Regorafenib will be escalated/de-escalated, starting at DL0: * DL1: 82 mg/m\^2 once daily for 21 days/28 days (max 160mg) (100% of the RP2D) * DL0 (starting dose): 66 mg/m\^2 once daily for 21 days/28 days (max 120mg) (80% of the RP2D) * DL-1: 50 mg/m\^2 once daily for 21 days/28 days (max 80mg) (60% of the RP2D)

Study summary

New drug efficacy in ES has been disappointing in the last decades and no new drugs have been successfully introduced up to now in front line treatment. Among the tested drugs, early clinical data suggest that strategies using multi-targeted tyrosine kinase inhibitors (TKI) with anti-angiogenic activities are among the most efficient and may be beneficial in the treatment of patients with ES.

Several TKI have been and are currently being tested as single-agent in patients with relapsed/refractory ES with encouraging results in phase II trials. Regorafenib has shown promising activity in Ewing sarcoma relapse setting, Nevertheless, regorafenib has never been combined with the intensive chemotherapy VDC/IE schedule and therefore this combination needs to be evaluated in order to avoid dose reduction of the current standard treatment and hence its efficacy.

The current clinical trial has been therefore designed to test the feasibility of regorafenib with ES conventional chemotherapy. It consists of a phase Ib that will only recruit patients with multi-metastatic (other than lungs/pleura only) ES, that present the highest unmet medical need (2 year EFS: 33%, similar to patients with relapse/refractory ES).

Eligibility

Sex
ALL
Min age
2 Years
Max age
50 Years
Healthy volunteers
No
Inclusion Criteria: 1. Any histologically and genetically confirmed Ewing sarcoma of bone or soft tissue, or round cell sarcomas which are 'Ewing's-like' but negative for EWSR1 gene rearrangement 2. Metastatic disease 3. Age ≥2 years and \<50 years (from second birthday to 49 years 364 days) 4. Patient assessed as medically fit to receive the Ewing sarcoma standard multimodal treatment and regorafenib, including: * Absolute Neutrophil Count (ANC) ≥ 0.75x10\^9/L, platelets ≥ 75x10\^9/L. * Alanine aminotransferase (ALT), and aspartate aminotransferase (AST) ≤ 5×ULN * Bilirubin ≤ 2×ULN * Creatinine \< 2x ULN or creatinine clearance \>60 ml/min/1.73 m\^2 * International normalized ratio (INR)/ Partial thromboplastin time (PTT). INR and PTT ≤ 1.5 x ULN. INR \& PTT ≤ 1.5xULN 5. Adequate cardiac function as evidenced by left ventricular ejection fraction (LVEF) ≥50%) at baseline, as determined by echocardiography 6. Adequately controlled blood pressure (BP) with or without antihypertensive medications, defined as: a BP \<95th percentile for sex, age, and height at screening (as per National Heart Lung and Blood Institute \[NHLBI\] guidelines) and no change in antihypertensive medications within 1 week prior to Cycle 1 Day 1. Patients \>18 years old should have BP ≤ 150/90 mmHg. 7. No prior treatment for Ewing sarcoma other than surgery 8. Negative pregnancy test for female patients of childbearing potential within 7 days prior to study registration. 9. Patient agrees to use highly effective contraception during therapy and for 12 months after last trial treatment (females) or 6 months after last trial treatment (males), where applicable 10. Subject must be able to swallow and retain oral medication. 11. Written informed consent from the patient and/or the parent/legal guardian, according to local, regional or national regulation prior to any study specific procedures. 12. Patients must be affiliated to a social security system or beneficiary of the same, as per local regulatory requirements (France only) Exclusion Criteria: 1. Localized tumor or metastatic disease to lung/pleura only. 2. Contra-indication to the Ewing sarcoma standard multimodal treatment 3. Pregnant or breastfeeding women or intending to become pregnant during the study. 4. Follow-up not possible due to social, geographic or psychological reasons 5. Impairment of gastrointestinal (GI) function or GI disease that may significantly alter absorption of oral drugs 6. A clinically significant ECG abnormality, including a marked prolonged QTcF interval (eg, a repeated demonstration of a QTcF interval \>480 msec) Clinically significant, uncontrolled heart disease (including history of any cardiac arrhythmias, e.g., ventricular, supraventricular, nodal arrhythmias, or conduction abnormality, unstable angina, active coronary artery disease and myocardial infarction within 6 months before randomization.) Uncontrolled hypertension (systolic pressure \>150 mmHg or diastolic pressure \> 90 mmHg on repeated measurement) despite optimal medical management 7. Previous arterial or venous thromboembolisms Grade ≥ 3 per CTCAE v5.0 8. Hypersensitivity to any active substance or to any excipients 9. Radiographic evidence of encasement or invasion of a major blood vessel or of intratumoral cavitation 10. Major surgical procedure or significant traumatic injury within 28 days before starting study treatment 11. Non-healing wound, ulcer or bone fracture. 12. Uncontrolled systemic or local infection requiring systemic treatment 13. Any anticoagulant therapy (risk of hemorrhage with Regorafenib) 14. Interstitial lung disease with ongoing signs and symptoms. 15. Known prior history of HBV, HCV, HIV 16. Any other medical or other condition that, in the opinion of the investigator(s), would preclude the subject's participation in this clinical study

Primary outcome measure(s)

Trial sites (13)

FacilityCityRegionStatus
Queensland Children's Hospital Brisbane Australia
Monash Children's Hospital Clayton Australia
Royal Children's Hospital Parkville Australia
Perth Children's Hospital Perth Australia
Rigshospitalet Copenhagen Denmark
CHU Bordeaux Bordeaux France
Centre Oscar Lambret Lille France
centre Léon Bérard Lyon France
Institut Curie Paris France
Gustave Roussy Villejuif Île-de-France Region
Istituto Nazionale dei Tumori Milan Italy
Princess Máxima Center Utrecht Netherlands
Vall d'Hebron University Hospital Barcelona Spain

More Gustave Roussy, Cancer Campus, Grand Paris trials in Australia

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05830084 on ClinicalTrials.gov ↗ ← All trials in Australia