A Phase 1/2 interventional study of Regorafenib in Osteosarcoma and Ewing Sarcoma of Bone, sponsored by Institute of Mother and Child, Warsaw, Poland. Completed at 2 sites in Poland. Open to participants aged 9 Years to 21 Years. Per ClinicalTrials.gov, last updated 2026-10-07.
Sponsored by Institute of Mother and Child, Warsaw, Poland · Phase 1/2, Interventional, and Treatment
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.
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.
437 studies on the registry are indexed under Osteosarcoma; 116 are open to participants now.
This study's enrollment of 30 is below the median of 42 across 325 interventional studies indexed under Osteosarcoma.
Browse Osteosarcoma studies →Institute of Mother and Child, Warsaw, Poland is the lead sponsor of 8 studies on the registry; 4 are open to participants now.
Counted across the registry records on this site, refreshed daily.
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):
Exclusion Criteria:
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.
Drug: Regorafenib
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.
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.
Also known as: Stivarga
EFS - (Event-Free Survival).
To explore the efficacy in terms of EFS - (Event-Free Survival)
Time frame: 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)
Time frame: 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
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.
Assessment of the safety of regorafenib
Safety will be assessed by analyzing recorded vital signs, laboratory test results, echocardiography, and ECG.
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.
PFS (Progression-Free Survival).
progression-free survival - will be measured from randomization to the detection of disease progression in imaging tests.
Time frame: 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.
Time frame: 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.
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.
Concentration parameters will come directly from the concentration values measured in PK samples.
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.
Concentration parameters will come directly from the concentration values measured in PK samples.
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.
Concentration parameters will come directly from the concentration values measured in PK samples.
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.
Concentration parameters will come directly from the concentration values measured in PK samples.
Time frame: 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
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.
by monitoring the patient's clinical and molecular status
Time frame: Safety analyzes are planned in accordance with the schedule of intermediate analyzes, at least every 12 months.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is completed, as verified in Oct 2026. You cannot join it, but the record below documents what was studied.
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Institute of Mother and Child, Warsaw, Poland