Study on Treosulfan and Melphalan for High-Risk Ewing Sarcoma Patients

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What is this study about?

This clinical trial is focused on studying a type of cancer called Ewing Sarcoma, which affects bones or soft tissues. The study is investigating the use of two medications, Treosulfan and Melphalan, as a treatment for patients who have been newly diagnosed with high-risk or very high-risk Ewing Sarcoma. Treosulfan is a medication that helps destroy cancer cells, and Melphalan is a type of chemotherapy that works by stopping the growth of cancer cells. The purpose of the study is to evaluate the safety and effectiveness of these medications when used together as part of a comprehensive treatment plan.

Participants in the study will receive the medications through an intravenous infusion, which means the drugs are given directly into a vein. The study will follow a specific schedule where patients will receive high doses of Treosulfan and Melphalan over a set period. The treatment is designed to be part of a larger strategy that includes other therapies to manage Ewing Sarcoma. The study aims to see how well patients tolerate the treatment and to measure how long they remain free from cancer progression or relapse.

Throughout the study, researchers will monitor participants for any side effects and track their overall health. The study will also look at the long-term survival of patients over a period of three years. By participating in this study, researchers hope to gather important information that could improve treatment options for people with high-risk Ewing Sarcoma in the future.

1 initial assessment

Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes reviewing medical history and conducting necessary tests to ensure the patient meets the study criteria.

2 treatment initiation

The treatment phase begins with the administration of medications. The primary drugs used are treosulfan and melphalan, which are given through an intravenous infusion. This means the medication is delivered directly into the bloodstream through a vein.

The dosage and frequency of these medications are determined by the study protocol and the patient’s specific condition. The treatment is part of a multimodal strategy aimed at managing Ewing sarcoma.

3 monitoring and follow-up

Throughout the treatment, regular monitoring is conducted to assess the patient’s response to the medication and to identify any side effects. This includes routine blood tests and imaging studies.

The study aims to evaluate the safety and tolerability of the treatment, as well as its effectiveness in preventing disease progression.

4 completion of treatment

After completing the treatment regimen, a final assessment is conducted to evaluate the overall response to the therapy. This includes a comprehensive review of the patient’s health status and any changes in the condition of the disease.

5 long-term follow-up

The study includes a long-term follow-up period to monitor the patient’s health and any potential late effects of the treatment. This involves periodic check-ups and assessments over a specified timeframe, which may extend up to 36 months from the start of the treatment.

Who Can Join the Study?

Must have a primary diagnosis of Ewing Sarcoma or a similar type of sarcoma that does not have a specific gene change called EWSR1 gene rearrangement.
If the cancer has spread, patients can participate if they have:
- 0-5 bone lesions (areas where cancer has spread to the bone) if they are younger than 14 years old.
- 0-1 bone lesion if they are older than 14 years old.
- Cancer in the bone marrow (the soft tissue inside bones).
- Cancer that has spread outside the bones along with cancer in the bone marrow or bone lesions, following the age guidelines above.
Must have signed an informed consent form, which is a document that explains the study and confirms that the patient agrees to participate. If the patient is a child, a parent or legal guardian must sign.
Must be younger than 50 years old. Children under 18 must be treated in specialized centers for pediatric cancer.
Must register for the study within 45 days after the initial biopsy or surgery to diagnose the cancer.
Must have a performance status score indicating they are able to carry out daily activities:
- For those older than 16, a Karnofsky score greater than 50%.
- For those 16 or younger, a Lansky Play Score greater than 50%.
- For adults, an ECOG score of 2 or less.
Must have adequate bone marrow function, which means:
- A certain level of white blood cells called neutrophils greater than 0.75 x 10⁹/L.
- A platelet count greater than 75 x 10⁹/L. For those with widespread disease or extensive bone marrow involvement, a platelet count greater than 80 x 10⁹/L is required.
- Hemoglobin level greater than 8.0 g/dL, with transfusions allowed if needed.
Must have adequate organ function, which includes:
- Serum creatinine (a kidney function marker) less than 1.5 times the upper normal limit.
- Creatinine clearance (a measure of kidney function) greater than 60 ml/min.
- Serum bilirubin (a liver function marker) less than or equal to 1.5 times the upper normal limit, except for those with Gilbert’s Syndrome.
- Serum lipase and amylase (pancreas function markers) less than or equal to 1.5 times the upper normal limit.
- ALT and AST (liver enzymes) less than or equal to 2.5 times the upper normal limit, or 5.0 times for those with liver metastases.
- Normal heart function, with a left ventricular ejection fraction (LVEF) greater than 40% and a shortening fraction (SF) greater than 28%.
Female participants who can have children must have a negative pregnancy test before joining the study and monthly during treatment. They must use effective birth control during treatment and for 6 months after it ends. A woman is considered able to have children from the start of menstruation until menopause unless she has had surgery to remove reproductive organs.

Who Cannot Join the Study?

Patients who have had another type of cancer in the past, except for Ewing Sarcoma.
Patients who have an active infection that is not controlled with treatment.
Patients who are pregnant or breastfeeding.
Patients who have a known allergy to the study drugs, Treosulfan or Melphalan.
Patients who have severe heart, lung, liver, or kidney problems.
Patients who have received another experimental drug within the last 4 weeks.
Patients who have a condition that the study doctors believe would make it unsafe for them to participate.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Azienda Ospedaliero Universitaria Consorziale Policlinico di Bari	Bari	Italy
Fondazione Policlinico Universitario Agostino Gemelli IRCCS	Rome	Italy

Other Sites

Site Name	City	Country
IRCCS Istituto Nazionale Tumori Fondazione Pascale	Naples	Italy
Istituto Tumori Bari Giovanni Paolo II	Bari	Italy
Azienda Ospedaliera Santobono Pausilipon	Naples	Italy
Azienda Ospedaliero Universitaria Ospedali Riuniti Umberto I G M Lancisi G Salesi	Ancona	Italy
Universita’ Degli Studi Di Verona	Verona	Italy
Universita’ Di Pisa	Pisa	Italy
Universita’ Degli Studi Di Modena E Reggio Emilia	Modena	Italy

Trial status

Country	Status	Recruitment Start
Italy	Not yet recruiting	14.06.2023

Trial locations

Investigated drugs:

Treosulfan is a medication used in this clinical trial to help treat Ewing Sarcoma, a type of cancer. It is part of a treatment plan aimed at reducing the risk of cancer coming back after initial treatment. Treosulfan works by interfering with the growth of cancer cells, which can help to slow down or stop the progression of the disease.

Melphalan is another medication used in this trial alongside Treosulfan. It is also used to treat Ewing Sarcoma by targeting and destroying cancer cells. Melphalan is part of a comprehensive treatment strategy designed to improve the chances of recovery and prevent the cancer from returning. It works by damaging the DNA of cancer cells, which can lead to their death and help control the spread of the disease.

Investigated diseases:

Ewing's sarcoma

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. As it progresses, it can cause pain, swelling, and sometimes fractures in the affected area. The cancer cells can spread to other parts of the body, such as the lungs or other bones. The progression of Ewing Sarcoma can vary, with some cases advancing more rapidly than others.

Trial ID:

2024-518912-37-01

Protocol code:

INES

Trial Phase:

Therapeutic exploratory (Phase II)

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Study on Treosulfan and Melphalan for High-Risk Ewing Sarcoma Patients

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