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Phase II Prospective Multicenter Study of High-Dose Treosulfan/Melphalan as Consolidation Treatment in Newly Diagnosed High-Risk and Very High-Risk Ewing Sarcoma
EU CTIS ID: 2024-518912-37-01
What this study is testing
To evaluate the safety and tolerability of using Treosulfan/Melphalan in Ewing Sarcoma within a multimodal treatment strategy To determine the 3y-EFS in high-risk and very high-risk Ewing Sarcoma patients treated with Treosulfan/Melphalan as part of a risk-adapted multimodal treatment strategy
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Primary diagnosed and histologically confirmed, localized or metastatic, Ewing sarcoma (ES) of bone or soft tissue, or ‘Ewing-like’ sarcoma but negative for EWSR1 gene rearrangement;
- Patients with disseminated ES are included if any of the following conditions are present: a) 0-5 bone lesions at age < 14 years; b) 0-1 bone lesion at age > 14 years; c) bone marrow (BM) involvement; d) Extraosseous metastases + BM involvement or + bone lesion(s) with the aforementioned age-cut-offs;
- Informed consent signed by the patient and/or by the parents/legal guardian;
- Age < 50 years. Paediatric patients (< 18 years old) must be treated in accredited centers for the treatment of paediatric cancer patients
- Registration to the protocol ≤ 45 days from diagnostic biopsy/surgery
- Karnofsky > 50% for participants > 16 years old or Lansky Play Score > 50% for paediatric participants ≤ 16 years old or ECOG ≤ 2 for adult participants;
You likely can't join if
- The presence of > 5 bone lesions at age < 14 years or > 1 bone lesion at age > 14 years. Bone lesions must be confirmed by CT scan, MRI, PET scan or, in doubtful cases, by biopsy;
- Presence of concomitant bone metastases + bone marrow involvement (at least one site positive for metastatic infiltration);
- Prior or concurrent chemotherapy or radiotherapy;
- Any other severe concomitant comorbiditiesthat, according to the opinion of the Investigator, makes the enrollment in the study inappropriate for the patient, in particular: o myocardial infarction/unstable angina/congestive heart failure within 6 months before enrollment, LVEF less than the lower limit of normal per local institutional standards, history of clinically significant atrial arrhythmia or any history of ventricular arrhythmia; o uncontrolled hypertension, according to age values (patients with hypertension should be under treatment on study entry to carry out blood pressure control); o cerebrovascular accident or transient ischemic attack within 6 months before enrollment, any history of peripheral arterial occlusive disease requiring revascularization, venous thromboembolism including deep venous thrombosis or pulmonary embolism within 6 months before enrolment; o uncontrolled metabolic alterations (such as hypertriglyceridemia); o severe active uncontrolled infection; o history of bleeding disorder; o history of acute pancreatitis (within 1 year before study entry) or history of chronic pancreatitis; o history of alcohol abuse;
- Second malignancy;
- Pregnant or breastfeeding women;
See the full eligibility criteria
Who can join
- Primary diagnosed and histologically confirmed, localized or metastatic, Ewing sarcoma (ES) of bone or soft tissue, or ‘Ewing-like’ sarcoma but negative for EWSR1 gene rearrangement;
- Patients with disseminated ES are included if any of the following conditions are present: a) 0-5 bone lesions at age < 14 years; b) 0-1 bone lesion at age > 14 years; c) bone marrow (BM) involvement; d) Extraosseous metastases + BM involvement or + bone lesion(s) with the aforementioned age-cut-offs;
- Informed consent signed by the patient and/or by the parents/legal guardian;
- Age < 50 years. Paediatric patients (< 18 years old) must be treated in accredited centers for the treatment of paediatric cancer patients
- Registration to the protocol ≤ 45 days from diagnostic biopsy/surgery
- Karnofsky > 50% for participants > 16 years old or Lansky Play Score > 50% for paediatric participants ≤ 16 years old or ECOG ≤ 2 for adult participants;
- Adequate bone marrow function, defined as: Peripheral absolute neutrophil count (ANC) > 0.75 ×10 9 /L • Haemoglobin > 8.0 g/dL (transfusion allowed) • Platelet count > 75 × 10 9 /L (transfusion allowed)
- Adequate organ function (serum creatinine < 1.5 x upper limit of normal (ULN), calculated creatinine clearance > 60 ml/min as determined by Cockcroft-Gault or according to age and sex as determined by Schwartz’s Formula, serum bilirubin ≤ 1.5 × ULN, except for Gilbert’s Syndrome, serum lipase and amylase ≤ 1.5 × ULN, ALT and AST≤ 2.5 × ULN or ≤ 5.0 × ULN for patients with hepatic metastases, normal ventricular ejection function as LVEF > 50% and SF > 28%);
- A negative pregnancy test before enrolment and once a month during therapy for female participants of potential childbearing; for patients sexually active, it is mandatory to use an effective contraception throughout the treatment and up to 6 months beyond its end. A woman is considered of childbearing potential, i.e. fertile, following menarche and until becoming post-menopausal unless permanently sterile. Permanent sterilisation methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy.
What rules you out
- The presence of > 5 bone lesions at age < 14 years or > 1 bone lesion at age > 14 years. Bone lesions must be confirmed by CT scan, MRI, PET scan or, in doubtful cases, by biopsy;
- Presence of concomitant bone metastases + bone marrow involvement (at least one site positive for metastatic infiltration);
- Prior or concurrent chemotherapy or radiotherapy;
- Any other severe concomitant comorbiditiesthat, according to the opinion of the Investigator, makes the enrollment in the study inappropriate for the patient, in particular: o myocardial infarction/unstable angina/congestive heart failure within 6 months before enrollment, LVEF less than the lower limit of normal per local institutional standards, history of clinically significant atrial arrhythmia or any history of ventricular arrhythmia; o uncontrolled hypertension, according to age values (patients with hypertension should be under treatment on study entry to carry out blood pressure control); o cerebrovascular accident or transient ischemic attack within 6 months before enrollment, any history of peripheral arterial occlusive disease requiring revascularization, venous thromboembolism including deep venous thrombosis or pulmonary embolism within 6 months before enrolment; o uncontrolled metabolic alterations (such as hypertriglyceridemia); o severe active uncontrolled infection; o history of bleeding disorder; o history of acute pancreatitis (within 1 year before study entry) or history of chronic pancreatitis; o history of alcohol abuse;
- Second malignancy;
- Pregnant or breastfeeding women;
- Neuropsychiatric, social, geographic or severe family problems that make it difficult for the patient to participate in the study.
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years, 18-64 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.