Regorafenib in Patients With Refractory Primary Bone Tumors

• Status: Recruiting
• Trial end date: 2025-12-31
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Institute of Mother and Child, Warsaw, Poland
• Collaborator: Maria Sklodowska-Curie National Research Institute of Oncology

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 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.