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AuthorisedTherapeutic exploratory (Phase II)Interventional

Phase II Prospective Multicenter Study of High-Dose Treosulfan/Melphalan as Consolidation Treatment in Newly Diagnosed High-Risk and Very High-Risk Ewing Sarcoma.

This study is for adults with a type of bone cancer called Ewing Sarcoma, specifically those with high-risk disease. It's testing a stronger treatment plan that uses a combination of chemotherapy drugs, including Treosulfan and Melphalan. The main goals are to understand how safe this treatment is, such as any side effects people experience, and how well it keeps the cancer from returning over 36 months. Researchers also want to see if this treatment helps people live longer. By participating, patients could help doctors learn more about improving treatment options for Ewing Sarcoma, with the hope of finding more effective ways to fight this challenging cancer.

At a glance

Status
Authorised
Phase
Therapeutic exploratory (Phase II)
Sponsor
Fondazione Santobono Pausilipon Onlus
Enrolment target
60
Start
09 Dec 2024

What is this study about?

This study is focused on a type of cancer called Ewing Sarcoma, which often affects children and young adults. Specifically, it's for adults whose cancer is considered 'high-risk' or 'very high-risk.' This means their cancer might be more aggressive or harder to treat, and there's a higher chance it could return after initial treatments.

The main idea behind this research is to see if a stronger combination of chemotherapy drugs, including Treosulfan and Melphalan, can help keep the cancer from coming back. Think of it like a 'consolidation treatment' – it's an extra, more intensive step after the first set of treatments, aimed at getting rid of any remaining cancer cells that might be lurking. The study is particularly interested in understanding if this more powerful treatment is manageable for patients and how well it works in preventing the cancer from returning.

Researchers will be carefully tracking two main things. First, they want to know about any side effects patients experience – how often they happen, how long they last, and how severe they are. This helps them understand the safety of the treatment. Second, they will look at 'event-free survival,' which essentially means how long people live without the cancer returning, getting worse, or developing new cancers, and without dying from treatment complications. They will also look at overall survival, which means how long people live after starting the treatment. The hope is that this new approach could offer a better chance for long-term health for those with high-risk Ewing Sarcoma.

Key takeaways

  • Tests a stronger chemotherapy combination for high-risk Ewing Sarcoma.
  • Aims to prevent cancer from coming back.
  • Will monitor safety (side effects) and effectiveness (cancer not returning, longer life).
  • For adults aged 18 and over with high-risk Ewing Sarcoma.
  • Involves treatments and 3 years of follow-up.
  • Could help improve future treatments for this cancer.

Who may be eligible?

This study is for adults, meaning you need to be 18 years old or older to take part. There is no upper age limit, so older adults are also welcome to consider joining.

It's open to both men and women, so your sex doesn't affect whether you can participate. The most important thing is that you have been recently diagnosed with high-risk or very high-risk Ewing Sarcoma.

Before you can join, a medical team will review your specific health situation and cancer details to make sure the study treatment is suitable and safe for you. They will check other things like your overall health and any existing medical conditions.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

  1. Are you 18 years old or older?
  2. Have you been diagnosed with high-risk or very high-risk Ewing Sarcoma?
  3. Are you able to travel to a study centre if required?
  4. Are you generally in good enough health to have intensive treatment (your doctor will check this)?
Answer every question to see your result.

What does participation involve?

If you decide to join this study, you will receive a combination of chemotherapy drugs, including Treosulfan and Melphalan, as part of your treatment plan. Other chemotherapy drugs like Vincristine, Doxorubicin, Cyclophosphamide, Ifosfamide, and Etoposide may also be part of your overall care, depending on your individual treatment pathway.

The study involves regular check-ups and assessments to monitor your health, how you're responding to treatment, and any side effects you might experience. These could include blood tests, scans, and discussions about how you're feeling. The medical team will provide detailed schedules for your treatments and appointments.

After your main course of treatment, there will be follow-up appointments over a period of 36 months (three years). These follow-ups are important to track your long-term health, check for any cancer recurrence, and understand the overall success of the treatment. The total duration of active treatment and follow-up will be several years.

Potential risks and benefits

Taking part in this study could offer potential benefits, such as receiving a novel and potentially more effective treatment for your high-risk Ewing Sarcoma, which may improve your chances of preventing the cancer from returning. However, like all medical treatments, there are potential risks, mainly from the side effects of strong chemotherapy drugs. These side effects can range from mild (like nausea or fatigue) to severe (potentially affecting blood counts, organs, or increasing infection risk). The study team will closely monitor you and manage any side effects. You are free to withdraw from the study at any time, for any reason, without affecting your standard medical care.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.
  • Unverified
    Italy

Common questions

What is Ewing Sarcoma?

Ewing Sarcoma is a rare type of cancer that often starts in the bones, but can also be found in soft tissues.

What does 'high-risk' mean?

'High-risk' generally means the cancer is more likely to spread or come back, making it harder to treat effectively with standard methods.

What are Treosulfan and Melphalan?

These are chemotherapy drugs that work by killing cancer cells. They are being used together in this study as a stronger treatment.

Will I get standard treatment if I don't join the study?

Yes, if you choose not to join the study, you will still receive the best available standard care for your condition.

How long will the study last?

The study involves treatment and then follow-up for 36 months (three years) to see how you are doing in the long term.

How to find out more

Always speak to your GP or specialist before deciding to take part in a study.

Discussion

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