Regorafenib in Patients With Refractory Primary Bone Tumors

Learn more about:
Related Clinical Trial
Regorafenib in Patients With Refractory Primary Bone Tumors A Randomised Placebo-Controlled Study of Use of GnRHa During Chemotherapy for Fertility Protection of Young Women With Cancer A Phase I Feasibility And Safety Study of Fluorescein-Specific (FITC-Ew) CAR T Cells In Combination With Parenterally Administered Folate-Fluorescein (UB-TT170) For Osteogenic Sarcoma Anti-GD2 CAR T Cells in Pediatric Patients Affected by High Risk and/or Relapsed/Refractory Neuroblastoma or Other GD2-positive Solid Tumors Thoracotomy Versus Thoracoscopic Management of Pulmonary Metastases in Patients With Osteosarcoma Predictive Models of Treatment Responses and Survival Outcomes in Patients With Osteosarcoma Combination of Pembrolizumab and Cabozantinib in Patients With Advanced Sarcomas Construction of Microfluidic Exosome Chip for Diagnosis of Lung Metastasis of Osteosarcoma A Study of Surufatinib in Combination With Gemcitabine in Pediatric, Adolescent, and Young Adult Patients With Recurrent or Refractory Solid Tumors The Prognostic Value of Serum Biomarkers in Osteosarcoma Early Ageing in AYA Cancer Patients Neoadjuvant Combination of Doxorubicin, Cisplatin and Methotrexate in Patients Aged 24-40 Years With Primary Bone Tumors Perioperative Use of Tranexamic (TXA) in Bone Tumor Surgery Reduce Blood Loss and Transfusion Requirements. Atezolizumab and Cabozantinib for the Treatment of Adolescents and Young Adults With Recurrent or Metastatic Osteosarcoma, TACOS Study Efficacy of Epidural Analgesia in Lower Extremity Osteosarcoma Treatment of Relapsed or Refractory Neuroblastoma and Osteosarcoma With Expanded Haploidentical NK Cells and Hu14.18-IL2 A Study to Evaluate the Safety and Efficacy of ALMB-0168 in Patients With Osteosarcoma CBL0137 for the Treatment of Relapsed or Refractory Solid Tumors, Including CNS Tumors and Lymphoma IBI188 Combination Therapy in Solid Tumors Clinical Study of CD276 Targeted Autologous Chimeric Antigen Receptor T Cell Infusion in Patients With CD276 Positive Advanced Solid Tumor A Study of ZN-c3 in Combination With Gemcitabine in Subjects With Osteosarcoma SARC038: Phase 2 Study of Regorafenib and Nivolumab in Osteosarcoma Efficacy of Exercise Program on Osteosarcoma Patients Metformin as Maintenance Therapy in Patients With Bone Sarcoma and High Risk of Relapse Testing the Combination of Two Immunotherapy Drugs (Magrolimab and Dinutuximab) in Patients With Relapsed or Refractory Neuroblastoma or Relapsed Osteosarcoma Dietary Habits, Metabolome, Immune Profile and Microbiota in Patients With Bone Sarcoma Efficacy of Regorafenib Combined With Best Supportive Care as Maintenance Treatment in High Grade Bone Sarcomas Patients Oleclumab and Durvalumab for the Treatment of Recurrent, Refractory, or Metastatic Sarcoma Cabozantinib With Topotecan-Cyclophosphamide An Analysis of the Efficacy of High Dose Isofosfamide Through Elastomer Study of Effectiveness of GEMDOX in Relapsed Osteosarcoma Trastuzumab Deruxtecan for the Treatment of HER2+ Newly Diagnosed or Recurrent Osteosarcoma Expanded Access Use of L-MTP-PE for the Treatment of Osteosarcoma Testing a New Immune Cell Therapy, GD2-Targeted Modified T-cells (GD2CART), in Children, Adolescents, and Young Adults With Relapsed/Refractory Osteosarcoma and Neuroblastoma, The GD2-CAR PERSIST Trial Evaluation of Myocardial Injury After Anthracycline Chemotherapy in Osteosarcoma Patients Using CMR Combination Immunotherapy Targeting Sarcomas Olaparib With Ceralasertib in Recurrent Osteosarcoma Alpha/Beta CD19+ Depleted Haploidentical Transplantation + Zometa for Pediatric Hematologic Malignancies and Solid Tumors Effects of Dexrazoxane Hydrochloride on Biomarkers Associated With Cardiomyopathy and Heart Failure After Cancer Treatment BLESSED: Expanded Access for DeltaRex-G for Advanced Pancreatic Cancer and Sarcoma Methionine PET/CT Studies In Patients With Cancer Nivolumab With or Without Ipilimumab in Treating Younger Patients With Recurrent or Refractory Solid Tumors or Sarcomas HIFU Hyperthermia With Liposomal Doxorubicin (DOXIL) for Relapsed or Refractory Pediatric and Young Adult Solid Tumors Safety and Efficacy Study of REOLYSIN® in the Treatment of Bone and Soft Tissue Sarcomas Metastatic to the Lung Sorafenib in Treating Patients With Soft Tissue Sarcomas (Extremity Sarcoma Closed to Entry as of 5/30/07) A Phase II of Nivolumab Plus Ipilimumab in Non-resectable Sarcoma and Endometrial Carcinoma Therapeutic Angiotensin-(1-7) in Treating Patients With Metastatic Sarcoma That Cannot Be Removed By Surgery Diffusion Weighted Magnetic Resonance in Imaging Younger Patients With Newly Diagnosed Bone or Soft Tissue Sarcomas Study to Find a Safe Dose and Show Early Clinical Activity of Weekly Nab-paclitaxel in Pediatric Patients With Recurrent/ Refractory Solid Tumors Eligibility Screening for a NCI Pediatric Oncology Branch Research Study A Pilot Study for the Treatment of Patients With Metastatic and High Risk Sarcomas and Primitive Neuroectodermal Tumors Dose Escalation Study of Sorafenib and Irinotecan Combination Therapy in Pediatric Patients With Solid Tumors Intravenous Palifosfamide-tris in Combination With Etoposide and Carboplatin in Patients With Malignancies Long-Term Follow-Up of Patients Who Have Participated in Children’s Oncology Group Studies Co-treatment With GnRH Analogs on the Ovarian Reserve in Young Women Treated With Alkylating Agents for Cancer Haploidentical Stem Cell Transplantation and NK Cell Therapy in Patients With High-risk Solid Tumors Acupuncture Point Stimulation for Treatment of Chemotherapy Nausea and Vomiting Study of AP23573/MK-8669 (Ridaforolimus), A Mammalian Target of Rapamycin (mTOR) Inhibitor, in Participants With Advanced Sarcoma (MK-8669-018 AM1)(COMPLETED) Phase 1 Study of MM-398 Plus Cyclophosphamide in Pediatric Solid Tumors NAC to Prevent Cisplatin-induced Hearing Loss Pain and Impact of Chronic Pain on Function After Total Knee Replacements Pretreatment Anti-Therapeutic Antibodies (PATA) in Patients Treated With hu14.18K322A Antibody ZD1839 and Oral Irinotecan in Treating Young Patients With Refractory Solid Tumors Informed Consent in Pediatric Cancer Trials Phase 2 STIR Trial: Haploidentical Transplant and Donor Natural Killer Cells for Solid Tumors Clinical Studies of Gemcitabine-Oxaliplatin HGS-ETR2 to Treat Children With Solid Tumors Clinical Orthopaedic Data Bank (Acute and Chronic) Safety and Efficacy Evaluation of 4th Generation Safety-engineered CAR T Cells Targeting Sarcomas Biological Characterisation of High Risk CHildhood Cancer in Children, Adolescents and Young Adults (MICCHADO) Study of Vinorelbine and Cyclofosfamide Among Patients With Refractory Tumours or in Relapse A Study of Pemetrexed in Children With Recurrent Cancer Study of CRX100 in Patients With Advanced Solid Tumors Pepinemab in Treating Younger Patients With Recurrent, Relapsed, or Refractory Solid Tumors Open-label Leucovorin Pharmacokinetic Study in Patients Receiving High Dose Methotrexate With or Without Voraxaze Pediatric PET/MR Image Registry Cryoablation, Radiofrequency Ablation, or Microwave Ablation in Treating Patients With Metastatic Sarcoma Stable on Chemotherapy Nab-Paclitaxel and Gemcitabine for Recurrent/Refractory Sarcoma Prehabilitation to Improve Cancer Surgery Outcomes in AYA Patients With Extremity Sarcomas Huntsman Biopsy Study A Phase II Study of Oral Cyclophosphamide and Sirolimus (OCR) in Advanced Sarcoma Assessment of Healing and Function After Reconstruction Surgery for Bone Sarcomas M6620 (VX-970) in Selected Solid Tumors SARC024: A Blanket Protocol to Study Oral Regorafenib in Patients With Selected Sarcoma Subtypes Eltrombopag for the Prevention of Chemotherapy Induced Thrombocytopenia LN-145 in Treating Patients With Relapsed or Refractory Ovarian Cancer, Osteosarcoma, or Other Bone and Soft Tissue Sarcomas Markers of Anthracycline-Related Cardiac Muscle Injury A Phase I Trial of T Cells Expressing an Anti-GD2 Chimeric Antigen Receptor in Children and Young Adults With GD2+ Solid Tumors A Dose Finding Study of CycloSam® Combined With External Beam Radiotherapy A Vaccine Study for High Risk Cancers A Clinical Research on the Relation of Blood Drug Concentration and Calcium Folinate Rescued in High-dose MTX Therapy Temsirolimus and Cixutumumab in Treating Patients With Locally Advanced, Metastatic, or Recurrent Soft Tissue Sarcoma or Bone Sarcoma Study of the Safety and Efficacy of Humanized 3F8 Bispecific Antibody (Hu3F8-BsAb) in Patients With Relapsed/Refractory Neuroblastoma, Osteosarcoma and Other Solid Tumor Cancers A Phase II Study Evaluating Efficacy and Safety of Regorafenib in Patients With Metastatic Bone Sarcomas Comparison of MAPI+Camrelizumab Versus API+Apatinib Versus MAPI in Patients With a Poor Response to Preoperative Chemotherapy for Newly Diagnosed High-grade Osteosarcoma Metronomic Chemotherapy in Patients With Advanced Solid Tumor With Bone Metastasis and Advanced Pretreated Osteosarcoma Cancer Genome Study Using Samples From Patients Treated on Clinical Trial SHR1020-SHR-1210-II-OS Accuracy of Deep-learning Algorithm for Detection and Risk Stratification of Lung Nodules Feasibility Study of Interval Compressed Regimen Using Four-drugs for Osteosarcoma Denosumab in Treating Patients With Recurrent or Refractory Osteosarcoma Biomarker Expression in Tissue Samples From Patients With Bone Sarcomas Mifamurtide (L-MTP-PE) for High-Risk Osteosarcoma Will Glucarpidase After Methotrexate Treatment for Bone Sarcoma Lead to Fewer Side Effects and Reduce Chemotherapy Delays? A Study to Determine the Activity of Robatumumab (SCH 717454) in Participants With Relapsed Osteosarcoma or Ewing’s Sarcoma (MK-7454-002/P04720) Losartan + Sunitinib in Treatment of Osteosarcoma Dinutuximab in Combination With Sargramostim in Treating Patients With Recurrent Osteosarcoma Biomarkers in Predicting Response to Chemotherapy in Samples From Young Patients With Osteosarcoma Pulmonary Resectable Metastases of Osteosarcoma With Apatinib and CHemotherapy Study of Pazopanib in the Treatment of Osteosarcoma Metastatic to the Lung A Phase I Trial Of The Humanized Anti-GD2 Antibody In Children And Adolescents With Neuroblastoma, Osteosarcoma, Ewing Sarcoma and Melanoma A Phase I Study of OncoLAR® (Registered Trademark) (NSC 685403) With/Without Tamoxifen in Patients With Osteosarcoma Cabozantinib S-malate in Treating Patients With Relapsed Osteosarcoma or Ewing Sarcoma Efficacy and Safety of Regorafenib as Maintenance Therapy After First-line Treatment in Patients With Bone Sarcomas Monocyte Phenotypic and Functional Differences Outpatient Administration of High Dose Methotrexate (HD MTX) in Patients With Osteosarcoma DNA Biomarkers in Tissue Samples From Patients With Osteosarcoma Doxorubicin With Cisplatin, High-Dose Methotrexate, and Additional Risk-Adapted Outpatient Chemotherapy Humanized Monoclonal Antibody 3F8 (Hu3F8) With Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in the Treatment of Recurrent Osteosarcoma ABCB1/P-glycoprotein Expression Influence on Non-metastatic Osteosarcoma of the Extremities Biospecimen Analysis in Determining Effects of Chemotherapy on Fertility in Osteosarcoma Survivors Gemcitabine, Docetaxel, and Hydroxychloroquine in Treating Participants With Recurrent or Refractory Osteosarcoma High Dose Methotrexate With Leucovorin Rescue With or Without Glucarpidase in Osteosarcoma Preventing Nephrotoxicity and Ototoxicity From Osteosarcoma Therapy Genetic Characterisation of High-grade Paediatric Osteosarcomas Quantitative Imaging Biomarkers of Treatment Response in Osteosarcoma and Ewing Sarcoma Eribulin Mesylate in Treating Patients With Recurrent or Refractory Osteosarcoma A Phase I Clinical Trial of Neoadjuvant Chemotherapy With/Without SPIONs/SMF for Patients With Osteosarcoma Glembatumumab Vedotin in Treating Patients With Recurrent or Refractory Osteosarcoma Phase II Study of Intravenous Rexin-G in Osteosarcoma Apatinib for Advanced Osteosarcoma After Failure of Standard Multimodal Therapy Phase II Trial for the Treatment of Relapsed Osteosarcoma 18F-FTC-146 PET/CT in Newly-Diagnosed Osteosarcoma Genetic Biomarkers in Tissue Samples From Patients With Osteosarcoma Adjuvant High-Dose Thiotepa and Stem Cell Rescue Associated With Conventional Chemotherapy in Relapsed Osteosarcoma Clinical Trial of Lithium Carbonate Combined With Neo-adjuvant Chemotherapy to Treat Osteosarcoma Comparison of High-Dose Methotrexate (HDM) Plus Doxorubicin to HDM Plus Etoposide-Ifosfamide in Osteosarcoma Children Chemotherapy for Patients With Osteosarcoma A Clinical Study of PD-L1 Antibody ZKAB001(Drug Code) in Limited Stage of High-grade Osteosarcoma Investigation of [6R] 5,10-methylenetetrahydrofolate as Rescue Therapy for Osteosarcoma Patients Treated With HDMTX. Mifamurtide Combined With Post-operative Chemotherapy for Newly Diagnosed High Risk Osteosarcoma Patients Patient-derived Xenograft (PDX) Modeling to Test Drug Response for High-grade Osteosarcoma Chemotherapy Resistance in Osteogenic Sarcoma and Other Solid Tumors Pilot Study of Curcumin Formulation and Ashwagandha Extract in Advanced Osteosarcoma Phase II Open Label, Non-randomized Study of Sorafenib and Everolimus in Relapsed and Non-resectable Osteosarcoma A Study of Bevacizumab in Combination With Chemotherapy for Treatment of Osteosarcoma Osteosarcoma Study #2: A Randomized Trial of Pre-Surgical Chemotherapy vs. Immediate Surgery and Adjuvant Chemotherapy in the Treatment of Non-Metastatic Osteosarcoma. A Pediatric Oncology Group Phase III Study Study of Blood Samples From Patients With Osteosarcoma Glucarpidase After High-Dose Methotrexate in Patients With Osteosarcoma Nivolumab or Nivolumab and Azacitidine in Patients With Recurrent, Resectable Osteosarcoma Circulating Exosome RNA in Lung Metastases of Primary High-Grade Osteosarcoma A Study of Pre-Operative Treatment of Newly-Diagnosed, Surgically-Resectable Osteosarcoma With Doxorubicin, Ifosfamide, Etoposide, and Cisplatin With Early Metabolic Assessment of Response Phase II Study of Chemotherapy and Pamidronate for the Treatment of Newly Diagnosed Osteosarcoma Evaluation of Zoledronic Acid as a Single Agent or as an Adjuvant to Chemotherapy in High Grade Osteosarcoma ICONIC: Improving Outcomes Through Collaboration in OsteosarComa A Study of Endostar Combined With Chemotherapy for Treatment of Osteosarcoma Sorafenib in Relapsed High Grade Osteosarcoma European Study in Bone Sarcoma Patients Over 40 Years A Retrospective Non-intervention Study to Characterize FOlate Rescue Treatment in Osteosarcoma Patients Treated With HDMTX Osteosarcoma and Ewing Sarcoma Treatment Response Assessment With Functional MRI Imaging in Children and Young Adults A Placebo-Controlled Study of Saracatinib (AZD0530) in Patients With Recurrent Osteosarcoma Localized to the Lung Osteosarcoma1999-A Study Of Intensive Chemotherapy for Osteosarcoma Recombinant Interferon Alpha and Etoposide in Relapsed Osteosarcoma Osteosarcoma in Patients Older Than 40 Years. Prognostic Factors and Survival Proteomics Research of Osteosarcoma Collecting and Storing Samples of Blood and Tumor Tissue From Patients With Osteosarcoma Development of Indices Predicting Response to Pre-operative Chemotherapy in Osteosarcoma Patients Therapy Trial to Determine the Safety and Efficacy of Heavy Ion Radiotherapy in Patients With Osteosarcoma A Eurosarc Study of Mifamurtide in Advanced Osteosarcoma (MEMOS) A Study to Compare the Efficacy and Safety of Ifosfamide and Etoposide With or Without Lenvatinib in Children, Adolescents and Young Adults With Relapsed and Refractory Osteosarcoma Diffusion Study on Patients With Osteosarcoma Phase II Clinical Trial Scheme of Ganoderma Lucidum Spore Powder for Postoperative Chemotherapy of Osteosarcoma ABCB1/P-glycoprotein Expression as Biologic Stratification Factor for Patients With Non Metastatic Osteosarcoma Inheritance of Osteosarcoma & Paget’s Disease Through Chromosome 18: Surveillance Study of Patients With Newly Diagnosed Osteosarcoma A Study of Pembrolizumab in Patients With Relapsed Or Metastatic Osteosarcoma Not Eligible for Curative Surgery A Phase II Trial of Avelumab in Patients With Recurrent or Progressive Osteosarcoma Clinical Trial Evaluating Metronomic Chemotherapy in Patients With Metastatic Osteosarcoma Trial With Gemcitabine and Rapamycin in Second Line of Metastatic Osteosarcoma Biology of Osteosarcoma (BOOST) Registry and Biobank The Impact of Cancer on the Physical and Psychosocial Well-being Among Childhood Osteosarcoma Survivors The Osteosarcoma Biobank in Peking University People’s Hospital Differentiation of Bone Sarcomas and Osteomyelitis With Ferumoxytol-Enhanced MRI

Brief Title

Regorafenib in Patients With Refractory Primary Bone Tumors

Official Title

Evaluation of the Efficacy and Safety of Regorafenib in Patients With Refractory Primary Bone Tumors

Brief Summary

      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.

Detailed Description

      The scope of the project is to cover the entire population of children, adolescents and young
      adults from the age of 2 to the age of 21, who progressed to first-line treatment or who
      presented with a recurrence of Ewing's sarcoma or osteosarcoma. Despite escalating doses of
      chemotherapy and radiotherapy, aggressive surgical procedures in patients with dissemination
      disease and negative prognostic factors, no improvement in treatment outcomes has been
      achieved for over 30 years. For this reason, other therapeutic options are being
      investigated. There have been no significant responses to immunotherapy. Although, the
      inclusion of tyrosine kinase inhibitors (TKIs) appears to be promising.

      The identification of new mutations in bone tumors has led to a better insight into the
      molecular basis of these tumors, which has resulted in a more significant role of genetic
      research in everyday practice. Although traditional histopathological examinations are
      currently the basis for the diagnosis of bone tumors, the developing techniques of molecular
      biology make it possible, in many cases, to refine the diagnosis and, in the near future,
      will become the basis for the classification of these neoplasms. Moreover, these technics are
      expected to enable the qualification of patients to modern molecularly targeted therapies.

      Based on the above data, the objectives of the project are as follows: 1. to estimate the
      nature and frequency of mutations in the tumor tissue, 2. to compare molecular test results
      with clinical data (which will allow for the initial assessment of the impact of the mutation
      status on the clinical condition, course of treatment and prognosis), 3. to include targeted
      treatment - broad spectrum tyrosine kinase inhibitor - regorafenib in standard therapy.

Study Phase

Phase 1/Phase 2

Study Type


Primary Outcome

EFS - (Event-Free Survival).

Secondary Outcome

 PFS (Progression-Free Survival).





Study Arms / Comparison Groups

 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.


* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status


Estimated Enrollment


Start Date

April 28, 2022

Completion Date

December 31, 2025

Primary Completion Date

September 12, 2025

Eligibility Criteria

        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

               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

          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.




9 Years - 21 Years

Accepts Healthy Volunteers



, , 

Location Countries


Location Countries


Administrative Informations



Organization ID


Responsible Party

Principal Investigator

Study Sponsor

Institute of Mother and Child, Warsaw, Poland


 Maria Sklodowska-Curie National Research Institute of Oncology

Study Sponsor

, , 

Verification Date

May 2022