OsteosarcomaPhase 1/2RegboneNCT05395741
Regorafenib in Patients With Refractory Primary Bone Tumors
A Phase 1/2 study testing Regorafenib in people with Osteosarcoma and Ewing Sarcoma of Bone, run by Institute of Mother and Child, Warsaw, Poland at 2 sites in Poland.
Trial 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… Read more about this study
- Who is it for?
- Ages 9 to 21 years
- Conditions the study is for
- Osteosarcoma, Ewing Sarcoma of Bone
- What is being tested?
- Regorafenib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 2 sitesPoland
- Study dates
- 28 Apr 2022to 30 Jun 2026
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 9 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age >9 years ≤ 21 years.
- Histologically proven Ewing sarcoma or osteosarcoma.
- 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):
- progression on the I line or next, or
- relapse.
- Signing of informed consent for trial participation (including for Regorafenib treatment) according with current legal regulations.
Show all 9Show fewer
- Life expectancy of at least 12 weeks from the time informed consent was signed.
- Possibility of swallowing the tablet.
- 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.
Who cannot join
- Lack of inclusion criteria
- Previous treatment with Regorafenib.
- Pregnancy and breastfeeding.
- Hypersensitivity to the study drug or any of its ingredients.
- Simultaneous treatment with other drugs which might interact with Regorafenib.
- Persistent toxicity related to prior therapy, making it impossible to treat with Regorafenib.
Show all 11Show fewer
- Diagnosis of other malignancies before study inclusion.
- Patients with uncontrolled hypertension.
- Patients with diseases of the coagulation system.
- Patients with heart defects and / or cardiac arrhythmias requiring permanent treatment with antiarrhythmic drugs.
- 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.
Trial locations and contacts
2 sites in 1 country.
Poland2 sites
the Institute of Mother and Child
Warsaw, 01-211
Maria Sklodowska-Curie National Research Institute of Oncology
Warsaw, 02-781
About this study
Official title: Evaluation of the Efficacy and Safety of Regorafenib in Patients With Refractory Primary Bone Tumors
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.
Read the detailed description
The scope of the project is to cover the entire population of children, adolescents and young adults from the age of 9 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.
- Conditions
- Osteosarcoma, Ewing Sarcoma of Bone
- Being tested
- Regorafenib
- Keywords
- TKI, refractory bone tumors, osteosarcoma, Ewing sarcoma, targeted treatment, solid tumor
- Medical subject headings
- Osteosarcoma, Bone Neoplasms, Sarcoma, Ewing
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RegorafenibDrug
Also called Stivarga
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. 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.
| Study group | Role | What they receive |
|---|---|---|
| R1 - Regorafenib Arm | Experimental | 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. Receives: Drug: Regorafenib |
| R2 - Control Group | No intervention | 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
EFS - (Event-Free Survival).
To explore the efficacy in terms of EFS - (Event-Free Survival)
Measured: 1 year
Determining the dose of the test substance in patients between 9 and 21 years of age, at which exposure to the drug will be similar to that recommended for adults.
Safety will be assessed by the rate of participants presenting with Adverse Events stratified by grade, category, affected organ or system, as number of serious adverse events (SAEs)
Measured: 1 year
Assessment of safety in terms of AEs
Safety will be assessed by the rate of participants presenting with Adverse Events stratified by grade, category, affected organ or system, as number of adverse events (AEs), including adverse events of special interest
Measured: from date of randomization, until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 48 months.
Assessment of the safety of regorafenib
Safety will be assessed by analyzing recorded vital signs, laboratory test results, echocardiography, and ECG.
Measured: from date of randomization, until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 48 months.
Secondary outcomes (9)
PFS (Progression-Free Survival).
progression-free survival - will be measured from randomization to the detection of disease progression in imaging tests.
Measured: Safety analyzes are planned in accordance with the schedule of intermediate analyzes, at least every 12 months.
OS (Overall Survival).
overall survival - will be measured from randomization to death due to cancer.
Measured: Safety analyzes are planned in accordance with the schedule of intermediate analyzes, at least every 12 months.
ORR (Overall Response Rate).
the percentage of patients who achieved the response to treatment defined in the protocol.
Measured: Safety analyzes are planned in accordance with the schedule of intermediate analyzes, at least every 12 months.
Time to achieving sufficient drug concentration in serum.
Concentration parameters will come directly from the concentration values measured in PK samples.
Measured: Safety analyzes are planned in accordance with the schedule of intermediate analyzes, at least every 12 months.
Maximum serum concentration in steady state Cmaxs.
Concentration parameters will come directly from the concentration values measured in PK samples.
Measured: Safety analyzes are planned in accordance with the schedule of intermediate analyzes, at least every 12 months.
Minimum serum concentration in steady state Cminss.
Concentration parameters will come directly from the concentration values measured in PK samples.
Measured: Safety analyzes are planned in accordance with the schedule of intermediate analyzes, at least every 12 months.
Random serum concentration in steady state Css.
Concentration parameters will come directly from the concentration values measured in PK samples.
Measured: Safety analyzes are planned in accordance with the schedule of intermediate analyzes, at least every 12 months.
Drug exposure Ctau.
by monitoring the patient's clinical and molecular status
Measured: Safety analyzes are planned in accordance with the schedule of intermediate analyzes, at least every 12 months.
Time to achieving steady state drug concentration in serum.
by monitoring the patient's clinical and molecular status
Measured: Safety analyzes are planned in accordance with the schedule of intermediate analyzes, at least every 12 months.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 May 2022
- Study start28 Apr 2022
- Primary completion12 Sep 2025
- Study completion30 Jun 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (9)Papers the sponsor has linked to this study.
- Other trials for OsteosarcomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Institute of Mother and Child, Warsaw, Poland
- Sponsor type
- University, hospital or other
- Collaborators
- Maria Sklodowska-Curie National Research Institute of Oncology
- Responsible party
- Principal investigator, Anna Raciborska, Institute of Mother and Child, Warsaw, Poland
- Investigators
- Anna Raciborska, Prof., Principal investigator, the Institue of Mother and Child
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05395741
- Sponsor's protocol ID
- Regbone
- Acronym
- Regbone
- Status last verified
- Oct 2026
- First submitted
- 5 May 2022
- Last updated
- 7 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Agulnik 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.
PubMed 29931504Allard 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.
PubMed 28494338Berry 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.
PubMed 28295221Bruix 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.
PubMed 27932229Cardoso 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.
PubMed 29544202Davis KL, Fox E, Merchant MS, Reid JM, Kudgus RA, Liu X, Minard CG, Voss S, Berg SL, Weigel BJ, Mackall CL. Nivolumab in children and young adults with relapsed or refractory solid tumours or lymphoma (ADVL1412): a multicentre, open-label, single-arm, phase 1-2 trial. Lancet Oncol. 2020 Apr;21(4):541-550. doi: 10.1016/S1470-2045(20)30023-1. Epub 2020 Mar 17.
PubMed 32192573Davis LE, Bolejack V, Ryan CW, Ganjoo KN, Loggers ET, Chawla S, Agulnik M, Livingston MB, Reed D, Keedy V, Rushing D, Okuno S, Reinke DK, Riedel RF, Attia S, Mascarenhas L, Maki RG. Randomized Double-Blind Phase II Study of Regorafenib in Patients With Metastatic Osteosarcoma. J Clin Oncol. 2019 Jun 1;37(16):1424-1431. doi: 10.1200/JCO.18.02374. Epub 2019 Apr 23.
PubMed 31013172Dirksen U, Brennan B, Le Deley MC, Cozic N, van den Berg H, Bhadri V, Brichard B, Claude L, Craft A, Amler S, Gaspar N, Gelderblom H, Goldsby R, Gorlick R, Grier HE, Guinbretiere JM, Hauser P, Hjorth L, Janeway K, Juergens H, Judson I, Krailo M, Kruseova J, Kuehne T, Ladenstein R, Lervat C, Lessnick SL, Lewis I, Linassier C, Marec-Berard P, Marina N, Morland B, Pacquement H, Paulussen M, Randall RL, Ranft A, Le Teuff G, Wheatley K, Whelan J, Womer R, Oberlin O, Hawkins DS; Euro-E.W.I.N.G. 99 and Ewing 2008 Investigators. High-Dose Chemotherapy Compared With Standard Chemotherapy and Lung Radiation in Ewing Sarcoma With Pulmonary Metastases: Results of the European Ewing Tumour Working Initiative of National Groups, 99 Trial and EWING 2008. J Clin Oncol. 2019 Dec 1;37(34):3192-3202. doi: 10.1200/JCO.19.00915. Epub 2019 Sep 25.
PubMed 31553693Duffaud F, Mir O, Boudou-Rouquette P, Piperno-Neumann S, Penel N, Bompas E, Delcambre C, Kalbacher E, Italiano A, Collard O, Chevreau C, Saada E, Isambert N, Delaye J, Schiffler C, Bouvier C, Vidal V, Chabaud S, Blay JY; French Sarcoma Group. Efficacy and safety of regorafenib in adult patients with metastatic osteosarcoma: a non-comparative, randomised, double-blind, placebo-controlled, phase 2 study. Lancet Oncol. 2019 Jan;20(1):120-133. doi: 10.1016/S1470-2045(18)30742-3. Epub 2018 Nov 23.
PubMed 30477937
Study at a glance
From the registry record, last updated by the sponsor on 7 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of the Efficacy and Safety of Regorafenib in Patients With Refractory Primary Bone Tumors
- ClinicalTrials.gov ID
- NCT05395741
- Acronym / sponsor ID
- Regbone / Regbone
- Conditions
- Osteosarcoma, Ewing Sarcoma of Bone
- Keywords
- TKI, refractory bone tumors, osteosarcoma, Ewing sarcoma, targeted treatment, solid tumor
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Institute of Mother and Child, Warsaw, PolandUniversity, hospital or other
- Collaborators
- Maria Sklodowska-Curie National Research Institute of Oncology
- Study start
- 28 Apr 2022 (actual)
- Primary completion
- 12 Sep 2025 (actual)
- Study completion
- 30 Jun 2026 (actual)
- First posted
- 27 May 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 9 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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| R1 - Regorafenib Arm | Experimental | Drug: RegorafenibR1 - 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. |
| R2 - Control Group | No intervention | –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. |
Who may qualify
- Ages
- 9 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (9 listed)
- Age >9 years ≤ 21 years.
- Histologically proven Ewing sarcoma or osteosarcoma.
- 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):
- Signing of informed consent for trial participation (including for Regorafenib treatment) according with current legal regulations.
- Life expectancy of at least 12 weeks from the time informed consent was signed.
Key exclusion criteria (11 listed)
- Lack of inclusion criteria
- Previous treatment with Regorafenib.
- Pregnancy and breastfeeding.
- Hypersensitivity to the study drug or any of its ingredients.
- Simultaneous treatment with other drugs which might interact with Regorafenib.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RegorafenibAlso: StivargaPatients 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. 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. | Drug | R1 - Regorafenib Arm |
Primary outcome
EFS - (Event-Free Survival).
Time frame: 1 year
To explore the efficacy in terms of EFS - (Event-Free Survival)
Determining the dose of the test substance in patients between 9 and 21 years of age, at which exposure to the drug will be similar to that recommended for adults.
Time frame: 1 year
Safety will be assessed by the rate of participants presenting with Adverse Events stratified by grade, category, affected organ or system, as number of serious adverse events (SAEs)
Assessment of safety in terms of AEs
Time frame: from date of randomization, until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 48 months.
Safety will be assessed by the rate of participants presenting with Adverse Events stratified by grade, category, affected organ or system, as number of adverse events (AEs), including adverse events of special interest
Assessment of the safety of regorafenib
Time frame: from date of randomization, until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 48 months.
Safety will be assessed by analyzing recorded vital signs, laboratory test results, echocardiography, and ECG.
9 secondary outcomes
PFS (Progression-Free Survival).
Time frame: Safety analyzes are planned in accordance with the schedule of intermediate analyzes, at least every 12 months.
OS (Overall Survival).
Time frame: Safety analyzes are planned in accordance with the schedule of intermediate analyzes, at least every 12 months.
ORR (Overall Response Rate).
Time frame: Safety analyzes are planned in accordance with the schedule of intermediate analyzes, at least every 12 months.
Time to achieving sufficient drug concentration in serum.
Time frame: Safety analyzes are planned in accordance with the schedule of intermediate analyzes, at least every 12 months.
Maximum serum concentration in steady state Cmaxs.
Time frame: Safety analyzes are planned in accordance with the schedule of intermediate analyzes, at least every 12 months.
Minimum serum concentration in steady state Cminss.
Time frame: Safety analyzes are planned in accordance with the schedule of intermediate analyzes, at least every 12 months.
Random serum concentration in steady state Css.
Time frame: Safety analyzes are planned in accordance with the schedule of intermediate analyzes, at least every 12 months.
Drug exposure Ctau.
Time frame: Safety analyzes are planned in accordance with the schedule of intermediate analyzes, at least every 12 months.
Time to achieving steady state drug concentration in serum.
Time frame: Safety analyzes are planned in accordance with the schedule of intermediate analyzes, at least every 12 months.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Oct 2026.
- Results publication: PubMed 29931504Agulnik 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.
- Results publication: PubMed 28494338Allard 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.
- Results publication: PubMed 28295221Berry 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.
- Results publication: PubMed 27932229Bruix 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.
- Results publication: PubMed 29544202Cardoso 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.
- Results publication: PubMed 32192573Davis KL, Fox E, Merchant MS, Reid JM, Kudgus RA, Liu X, Minard CG, Voss S, Berg SL, Weigel BJ, Mackall CL. Nivolumab in children and young adults with relapsed or refractory solid tumours or lymphoma (ADVL1412): a multicentre, open-label, single-arm, phase 1-2 trial. Lancet Oncol. 2020 Apr;21(4):541-550. doi: 10.1016/S1470-2045(20)30023-1. Epub 2020 Mar 17.
- Results publication: PubMed 31013172Davis LE, Bolejack V, Ryan CW, Ganjoo KN, Loggers ET, Chawla S, Agulnik M, Livingston MB, Reed D, Keedy V, Rushing D, Okuno S, Reinke DK, Riedel RF, Attia S, Mascarenhas L, Maki RG. Randomized Double-Blind Phase II Study of Regorafenib in Patients With Metastatic Osteosarcoma. J Clin Oncol. 2019 Jun 1;37(16):1424-1431. doi: 10.1200/JCO.18.02374. Epub 2019 Apr 23.
- Results publication: PubMed 31553693Dirksen U, Brennan B, Le Deley MC, Cozic N, van den Berg H, Bhadri V, Brichard B, Claude L, Craft A, Amler S, Gaspar N, Gelderblom H, Goldsby R, Gorlick R, Grier HE, Guinbretiere JM, Hauser P, Hjorth L, Janeway K, Juergens H, Judson I, Krailo M, Kruseova J, Kuehne T, Ladenstein R, Lervat C, Lessnick SL, Lewis I, Linassier C, Marec-Berard P, Marina N, Morland B, Pacquement H, Paulussen M, Randall RL, Ranft A, Le Teuff G, Wheatley K, Whelan J, Womer R, Oberlin O, Hawkins DS; Euro-E.W.I.N.G. 99 and Ewing 2008 Investigators. High-Dose Chemotherapy Compared With Standard Chemotherapy and Lung Radiation in Ewing Sarcoma With Pulmonary Metastases: Results of the European Ewing Tumour Working Initiative of National Groups, 99 Trial and EWING 2008. J Clin Oncol. 2019 Dec 1;37(34):3192-3202. doi: 10.1200/JCO.19.00915. Epub 2019 Sep 25.
- Results publication: PubMed 30477937Duffaud F, Mir O, Boudou-Rouquette P, Piperno-Neumann S, Penel N, Bompas E, Delcambre C, Kalbacher E, Italiano A, Collard O, Chevreau C, Saada E, Isambert N, Delaye J, Schiffler C, Bouvier C, Vidal V, Chabaud S, Blay JY; French Sarcoma Group. Efficacy and safety of regorafenib in adult patients with metastatic osteosarcoma: a non-comparative, randomised, double-blind, placebo-controlled, phase 2 study. Lancet Oncol. 2019 Jan;20(1):120-133. doi: 10.1016/S1470-2045(18)30742-3. Epub 2018 Nov 23.
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Refractory Malignant Solid Neoplasm, Osteosarcoma
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Anna Raciborska, Prof. principal investigator
the Institue of Mother and Child
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Regorafenib in people with Osteosarcoma and Ewing Sarcoma of Bone, run by Institute of Mother and Child, Warsaw, Poland at 2 sites in Poland.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 7 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Poland. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
EFS - (Event-Free Survival). (time frame: 1 year). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05395741, which also lists 9 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Oct 2026.