Osteosarcoma Clinical Trial
— RegboneOfficial 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.
Status | Recruiting |
Enrollment | 30 |
Est. completion date | December 31, 2025 |
Est. primary completion date | September 12, 2025 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 9 Years to 21 Years |
Eligibility | 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. |
Country | Name | City | State |
---|---|---|---|
Poland | Maria Sklodowska-Curie National Research Institute of Oncology | Warsaw | |
Poland | the Institute of Mother and Child | Warsaw |
Lead Sponsor | Collaborator |
---|---|
Institute of Mother and Child, Warsaw, Poland | Maria Sklodowska-Curie National Research Institute of Oncology |
Poland,
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. — View Citation
Allard 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. — View Citation
Berry 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- — View Citation
Bruix 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 — View Citation
Cardoso 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, pazopa — View Citation
Davis 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, pha — View Citation
Davis 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 Me — View Citation
Dirksen 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 — View Citation
Duffaud 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 — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | EFS - (Event-Free Survival). | To explore the efficacy in terms of EFS - (Event-Free Survival) | 1 year | |
Primary | 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) | 1 year | |
Primary | 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 | 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. | |
Primary | Assessment of the safety of regorafenib | Safety will be assessed by analyzing recorded vital signs, laboratory test results, echocardiography, and ECG. | 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 | PFS (Progression-Free Survival). | progression-free survival - will be measured from randomization to the detection of disease progression in imaging tests. | Safety analyzes are planned in accordance with the schedule of intermediate analyzes, at least every 12 months. | |
Secondary | OS (Overall Survival). | overall survival - will be measured from randomization to death due to cancer. | Safety analyzes are planned in accordance with the schedule of intermediate analyzes, at least every 12 months. | |
Secondary | ORR (Overall Response Rate). | the percentage of patients who achieved the response to treatment defined in the protocol. | Safety analyzes are planned in accordance with the schedule of intermediate analyzes, at least every 12 months. | |
Secondary | Time to achieving sufficient drug concentration in serum. | Concentration parameters will come directly from the concentration values measured in PK samples. | Safety analyzes are planned in accordance with the schedule of intermediate analyzes, at least every 12 months. | |
Secondary | Maximum serum concentration in steady state Cmaxs. | Concentration parameters will come directly from the concentration values measured in PK samples. | Safety analyzes are planned in accordance with the schedule of intermediate analyzes, at least every 12 months. | |
Secondary | Minimum serum concentration in steady state Cminss. | Concentration parameters will come directly from the concentration values measured in PK samples. | Safety analyzes are planned in accordance with the schedule of intermediate analyzes, at least every 12 months. | |
Secondary | Random serum concentration in steady state Css. | Concentration parameters will come directly from the concentration values measured in PK samples. | Safety analyzes are planned in accordance with the schedule of intermediate analyzes, at least every 12 months. | |
Secondary | Drug exposure Ctau. | by monitoring the patient's clinical and molecular status | Safety analyzes are planned in accordance with the schedule of intermediate analyzes, at least every 12 months. | |
Secondary | Time to achieving steady state drug concentration in serum. | by monitoring the patient's clinical and molecular status | Safety analyzes are planned in accordance with the schedule of intermediate analyzes, at least every 12 months. |
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