Clinical trial · Interventional
Regorafenib in Patients With Refractory Primary Bone Tumors
Evaluation of the Efficacy and Safety of Regorafenib in Patients With Refractory Primary Bone Tumors
NCT05395741CI-TRIAL-00123324RegbonecompletedPhase 1 / Phase 2ClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
The aim of the project is to improve treatment outcomes in patients with primary malignant bone tumors, refractory to standard therapy, by increasing the availability of advanced therapy, as well as to develop treatment options using advanced molecular diagnostics for patients who have not responded to the standard therapeutic regimen, and to introduce modern diagnostics for risk stratification and for the use in molecularly targeted therapies.
Conditions
Conditions (2)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Ewing Sarcoma of Bone | Bone Ewing Sarcoma | ALIAS | 0.90 |
| Osteosarcoma | Osteosarcoma | ONTOLOGY_EXACT | 0.90 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Regorafenib | Drug | Regorafenib | ALIAS |
Design
Arms and outcomes
Arms (2)
- type
- EXPERIMENTAL
- label
- R1 - Regorafenib Arm
- description
- R1 - the experimental group. Standard oncological treatment will be started. Additionally, patients will receive regorafenib orally at doses adjusted for age, body surface area and pharmacokinetics. Treatment with regorafenib will be continued for up to 1 year or until disease progression, patient death, unacceptable toxicity, or study closure. Pharmacokinetics and safety profile of the investigational product (IP) will be determined throughout the course therapy.
- interventionNames
- Drug: Regorafenib
- type
- NO_INTERVENTION
- label
- R2 - Control Group
- description
- R2 - the control group - will receive only standard treatment. In the event of progression or relapse, patients in the control group will have the option to receive the IP along with the standard treatment of the next line.
Primary outcomes (4)
- measure
- EFS - (Event-Free Survival).
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 9 Years
- Maximum age
- 21 Years
Show eligibility criteria text
Inclusion Criteria: 1. Age \>9 years ≤ 21 years. 2. Histologically proven Ewing sarcoma or osteosarcoma. 3. Failure of the treatment identified no earlier than 30 days prior to study treatment initiation (at least one of below needs to apply in order for this requirement to be satisfied): 1. progression on the I line or next, or 2. relapse. 4. Signing of informed consent for trial participation (including for Regorafenib treatment) according with current legal regulations. 5. Life expectancy of at least 12 weeks from the time informed consent was signed. 6. Possibility of swallowing the tablet. 7. Consent to the use of effective contraception throughout the period of the study and a minimum of 2 year after discontinuation of study treatment in patients at puberty and sexual maturity. Exclusion Criteria: 1. Lack of inclusion criteria 2. Previous treatment with Regorafenib. 3. Pregnancy and breastfeeding. 4. Hypersensitivity to the study drug or any of its ingredients. 5. Simultaneous treatment with other drugs which might interact with Regorafenib. 6. Persistent toxicity related to prior therapy, making it impossible to treat with Regorafenib. 7. Diagnosis of other malignancies before study inclusion. 8. Patients with uncontrolled hypertension. 9. Patients with diseases of the coagulation system. 10. Patients with heart defects and / or cardiac arrhythmias requiring permanent treatment with antiarrhythmic drugs. 11. Other acute or persistent disorders, behaviors or abnormal laboratory test results, which might increase the risk related to the participation in this clinical trial or to taking the study drug, or which might influence the interpretation of the study results, or which, in the investigator's opinion, disqualify a patient from participating in the trial.
References
Publications (9)
- RESULTAgulnik M, Attia S. Growing Role of Regorafenib in the Treatment of Patients with Sarcoma. Target Oncol. 2018 Aug;13(4):417-422. doi: 10.1007/s11523-018-0575-0. PMID 29931504
- RESULTAllard M, Khoudour N, Rousseau B, Joly C, Costentin C, Blanchet B, Tournigand C, Hulin A. Simultaneous analysis of regorafenib and sorafenib and three of their metabolites in human plasma using LC-MS/MS. J Pharm Biomed Anal. 2017 Aug 5;142:42-48. doi: 10.1016/j.jpba.2017.04.053. Epub 2017 May 1. PMID 28494338
- RESULTBerry V, Basson L, Bogart E, Mir O, Blay JY, Italiano A, Bertucci F, Chevreau C, Clisant-Delaine S, Liegl-Antzager B, Tresch-Bruneel E, Wallet J, Taieb S, Decoupigny E, Le Cesne A, Brodowicz T, Penel N. REGOSARC: Regorafenib versus placebo in doxorubicin-refractory soft-tissue sarcoma-A quality-adjusted time without symptoms of progression or toxicity analysis. Cancer. 2017 Jun 15;123(12):2294-2302. doi: 10.1002/cncr.30661. Epub 2017 Mar 10. PMID 28295221
- RESULTBruix J, Qin S, Merle P, Granito A, Huang YH, Bodoky G, Pracht M, Yokosuka O, Rosmorduc O, Breder V, Gerolami R, Masi G, Ross PJ, Song T, Bronowicki JP, Ollivier-Hourmand I, Kudo M, Cheng AL, Llovet JM, Finn RS, LeBerre MA, Baumhauer A, Meinhardt G, Han G; RESORCE Investigators. Regorafenib for patients with hepatocellular carcinoma who progressed on sorafenib treatment (RESORCE): a randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2017 Jan 7;389(10064):56-66. doi: 10.1016/S0140-6736(16)32453-9. Epub 2016 Dec 6. PMID 27932229
- RESULTCardoso E, Mercier T, Wagner AD, Homicsko K, Michielin O, Ellefsen-Lavoie K, Cagnon L, Diezi M, Buclin T, Widmer N, Csajka C, Decosterd L. Quantification of the next-generation oral anti-tumor drugs dabrafenib, trametinib, vemurafenib, cobimetinib, pazopanib, regorafenib and two metabolites in human plasma by liquid chromatography-tandem mass spectrometry. J Chromatogr B Analyt Technol Biomed Life Sci. 2018 Apr 15;1083:124-136. doi: 10.1016/j.jchromb.2018.02.008. Epub 2018 Feb 8. PMID 29544202