Study of Regorafenib with Chemotherapy for Newly Diagnosed Patients with Metastatic Ewing Sarcoma

1 1 1

What is this study about?

This clinical trial is focused on studying a type of cancer called Ewing sarcoma, which is a rare cancer that affects bones or the soft tissue around bones. The study is specifically for patients who have been newly diagnosed with Ewing sarcoma that has spread to other parts of the body, except for the lungs or the lining around the lungs. The treatment being tested in this study is a combination of a drug called regorafenib and standard chemotherapy. Regorafenib is a medication that is taken by mouth and is being tested in two forms: as granules and as film-coated tablets. The purpose of the study is to find the best dose of regorafenib when used with chemotherapy for treating this type of cancer.

Participants in the study will receive the combination of regorafenib and chemotherapy. The study will monitor how the body responds to the treatment and any side effects that may occur. The study will also look at how the cancer responds to the treatment, including whether it shrinks or stops growing. The study will last for several years, and participants will be closely monitored throughout the process to ensure their safety and to gather important information about the treatment’s effectiveness.

The study aims to improve the treatment options for patients with Ewing sarcoma by determining the most effective way to use regorafenib in combination with chemotherapy. This research could potentially lead to better outcomes for patients with this challenging form of cancer. Participants will be contributing to important research that may help future patients with Ewing sarcoma.

1 joining the study

Upon joining the study, you will be required to provide written informed consent. This is a document that confirms your understanding of the study and your agreement to participate.

You will need to confirm that you can swallow and retain oral medication, as the treatment involves taking medication by mouth.

2 initial assessments

Before starting the treatment, several health assessments will be conducted. These include blood tests to check your blood cell counts, liver function, kidney function, and heart function.

A pregnancy test will be conducted for female patients of childbearing potential.

Your blood pressure will be measured to ensure it is adequately controlled.

3 treatment cycle 1

The treatment involves taking a medication called regorafenib in combination with standard chemotherapy. Regorafenib is taken orally in the form of granules or film-coated tablets.

The first cycle of treatment lasts for 28 days. During this time, you will be monitored for any side effects or dose-limiting toxicities, which are side effects that may require adjusting the dose of the medication.

4 monitoring and adjustments

Throughout the treatment, regular monitoring will be conducted to assess your response to the medication and to check for any adverse effects.

If any severe side effects occur, the dosage of regorafenib or chemotherapy may be adjusted, or the treatment may be paused or stopped.

5 subsequent treatment cycles

After the first cycle, additional cycles of treatment will continue, with each cycle lasting 28 days.

The treatment will continue until the study’s end date or until it is determined that the treatment is no longer beneficial or safe for you.

6 end of study

The study is estimated to end by June 30, 2026. At the end of the study, a final assessment will be conducted to evaluate your overall health and response to the treatment.

You will be informed about the results of the study and any further steps that may be necessary for your health care.

Who Can Join the Study?

Must have a newly diagnosed condition called metastatic Ewing sarcoma, which means the cancer has spread to other parts of the body, but not just to the lungs or the lining around the lungs.
The type of cancer must be confirmed through specific tests, either as Ewing sarcoma of the bone or soft tissue, or a similar type that does not have a specific gene change called EWSR1 gene rearrangement.
Must be able to swallow and keep down oral medication.
Must provide written consent to participate, which can be given by the patient or their parent/legal guardian.
Must have metastatic disease, meaning the cancer has spread to other parts of the body.
Must be between the ages of 2 years and less than 50 years old.
Must be medically fit to receive standard treatment for Ewing sarcoma and a drug called regorafenib. This includes having:
- An Absolute Neutrophil Count (ANC) of at least 0.75×10⁹/L and platelets of at least 75×10⁹/L.
- Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels no more than 5 times the upper limit of normal (ULN).
- Bilirubin levels no more than 2 times the ULN.
- Creatinine levels less than 2 times the ULN or a creatinine clearance greater than 60 ml/min/1.73 m².
- International normalized ratio (INR) and Partial thromboplastin time (PTT) no more than 1.5 times the ULN.
Must have adequate heart function, shown by a left ventricular ejection fraction (LVEF) of at least 50% as measured by an ultrasound of the heart.
Must have controlled blood pressure, with or without medication. For those under 18, blood pressure should be below the 95th percentile for their sex, age, and height. For those over 18, blood pressure should be 150/90 mm Hg or lower.
Must not have received any prior treatment for Ewing sarcoma, except for surgery.
Female patients who can have children must have a negative pregnancy test within 7 days before joining the study.
Must agree to use highly effective birth control during the study and for 12 months after the last treatment for females, or 6 months for males, if applicable.

Who Cannot Join the Study?

Patients who have been diagnosed with Ewing sarcoma that has spread to areas other than the lungs or the lining around the lungs (pleura) cannot participate.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Institut Gustave Roussy	Villejuif	France

Other Sites

Site Name	City	Country
Hospital General Universitario Gregorio Maranon	Madrid	Spain
Prinses Maxima Centrum voor Kinderoncologie B.V.	Utrecht	The Netherlands
Roskilde University	Roskilde	Denmark
Fondazione I.R.C.C.S. Istituto Neurologico Besta	Milan	Italy
CHU Bordeauxt	Bordeaux	France
Apfamsx Oyqnjtmcvtq Uvrzmgcazlqsu Cwxwgcttlyou Dpbob Sbxtjo E Dvhgw Sclylpr Db Tdvdkn	Turin	Italy
Cwkkmg Ldab Bspocz	Lyon	France
Floyiwmbj Ppbt La Iebyjcsisgkqa Bxrudfeag Dzv Hlotcbdc Uuvxxzmmvelfs Ln Pxf	Madrid	Spain
Hqzhebon Vfxj dwimjwrc	Barcelona	Spain
Cqvzkn Oopmx Lqlnanw	Lille	France
Inainepb Cmvgx	Paris	France

Trial status

Country	Status	Recruitment Start
Denmark	Not recruiting	30.06.2023
France	Not recruiting	30.06.2023
Italy	Recruiting	30.06.2023
Spain	Not recruiting	30.06.2023
The Netherlands	Not recruiting	30.06.2023

Trial locations

Investigated drugs:

Regorafenib

Regorafenib is a medication used in this trial to help treat Ewing sarcoma, a type of cancer. It works by blocking certain proteins that cancer cells need to grow and spread. In this study, regorafenib is being tested in combination with other chemotherapy drugs to see if it can improve treatment outcomes for patients with this type of cancer.

VDC/IE is a combination of chemotherapy drugs used as a standard treatment for Ewing sarcoma. VDC stands for vincristine, doxorubicin, and cyclophosphamide, while IE stands for ifosfamide and etoposide. These drugs work together to kill cancer cells or stop them from growing. In this trial, VDC/IE is used alongside regorafenib to see if the combination is more effective in treating patients with newly diagnosed metastatic Ewing sarcoma.

Investigated diseases:

Ewing Sarcoma – Ewing sarcoma is a rare type of cancer that occurs in bones or in the soft tissue around the bones. It primarily affects children and young adults. The disease often begins in the long bones of the arms and legs, pelvis, or chest. It can spread to other parts of the body, including bone marrow and other organs. The progression of Ewing sarcoma involves the growth of a tumor, which can cause pain and swelling in the affected area. As the disease advances, it may lead to additional symptoms such as fever, fatigue, and weight loss.

Trial ID:

2023-503322-39-00

Protocol code:

CSET2022/3545

Trial Phase:

Human Pharmacology (Phase I) – Other

Other Trials to Consider

#1 Clinical Trials Search in Europe

Study of Regorafenib with Chemotherapy for Newly Diagnosed Patients with Metastatic Ewing Sarcoma

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?