Active not recruitingPHASE3INTERVENTIONAL

A Study to Compare Early Use of Vinorelbine and Maintenance Therapy for Patients With High Risk Rhabdomyosarcoma

This research is a 'Phase 3' study, which means it's one of the final steps before a new treatment might become widely available. It focuses on a type of cancer called rhabdomyosarcoma, especially when it's considered 'high-risk' – meaning it could grow quickly or return. The study compares the usual treatment plan with a new plan that includes an additional medicine called vinorelbine. We also want to see if continuing treatment with a 'maintenance' therapy for a period after the main treatment helps keep the cancer away for longer. All the medicines used are chemotherapy drugs, which work in different ways to fight cancer cells. The goal is to find better ways to treat this cancer and reduce the chance of it coming back.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Children's Oncology Group

Enrolment target

118

Start

14 Sep 2021

Estimated completion

30 Jun 2026

Alveolar Rhabdomyosarcoma Botryoid-Type Embryonal Rhabdomyosarcoma Embryonal Rhabdomyosarcoma Metastatic Embryonal Rhabdomyosarcoma Metastatic Rhabdomyosarcoma Solid Alveolar Rhabdomyosarcoma Spindle Cell Rhabdomyosarcoma Spindle Cell/Sclerosing Rhabdomyosarcoma

What is this study about?

This study is designed to find better ways to treat children and young people with a type of cancer called rhabdomyosarcoma. This cancer can sometimes be aggressive, meaning it grows quickly, or it might be likely to come back even after initial treatment. Because of this, it's called 'high-risk' rhabdomyosarcoma.

We're comparing two main approaches. One is the current standard treatment, which uses a combination of chemotherapy drugs. The other approach adds an extra drug called vinorelbine to this standard combination. Vinorelbine, like the other drugs used, is a chemotherapy medicine. These medicines work by targeting cancer cells and stopping them from growing and dividing, which can help get rid of the cancer. We want to see if adding vinorelbine makes the treatment more effective without causing too many extra side effects.

Another important part of this study is looking at 'maintenance therapy'. After the main course of treatment, some patients will receive a longer course of treatment with a combination of vinorelbine and cyclophosphamide. This 'maintenance' treatment is like a follow-up, aiming to keep the cancer away for a longer period and reduce the chances of it returning. By comparing these different treatment plans, we hope to understand which one gives patients the best chance of staying cancer-free, potentially for many years or even for life.

Key takeaways

This study evaluates new ways to treat specific types of rhabdomyosarcoma in children and young adults.
It compares standard chemotherapy with a modified approach that includes an extra drug called vinorelbine.
The study also looks at whether 'maintenance therapy' helps keep the cancer from returning.
You may receive different chemotherapy drugs depending on which treatment group you are in.
The goal is to improve long-term outcomes and survival for patients with this type of cancer.

Who may be eligible?

This study is for children and young people aged 50 or younger who have been newly diagnosed with certain types of rhabdomyosarcoma that are considered 'high-risk'. This includes specific kinds like Embryonal and Alveolar Rhabdomyosarcoma. Your doctors will check the exact type of cancer and its stage to see if it fits the study criteria.

To join, you must have generally good health otherwise, including your kidney function. This will be checked with blood tests. If you have cancer cells found in your bone marrow, your doctors will confirm whether this makes you suitable for the study or not, as only certain ways of finding these cells count for this study.

Your medical team will perform specific tests, such as looking at your tumour cells closely (including for something called FOXO1 fusion) to make sure your cancer matches the types being studied. They will explain all the details and help you understand if this trial is right for you.

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.

Are you 50 years old or younger?
Have you been newly diagnosed with a type of rhabdomyosarcoma that is considered 'high-risk'?
Do you have generally good health, including normal kidney function?
Has your doctor confirmed your specific cancer type fits the study criteria (e.g., Embryonal or Alveolar Rhabdomyosarcoma)?

Answer every question to see your result.

What does participation involve?

If you join this study, you'll be assigned to one of two treatment groups, much like flipping a coin, so it's random. Both groups will receive chemotherapy drugs, given through a drip (IV) into your vein, following a set schedule over several weeks. This will involve regular visits to the hospital for your treatment doses.

Throughout the study, you'll have various tests to monitor your health and how the treatment is working. These might include blood tests, scans like CT scans or MRI scans (to look at the inside of your body), bone scans (to check bones), and sometimes bone marrow samples.

After the main treatment, if you are in the group receiving 'maintenance therapy', you will continue with certain medications for about 24 weeks. Your doctors will keep a close eye on you, continuing to check your health and scan results. The total duration of active treatment and follow-up will depend on your specific treatment plan and how you respond.

Potential risks and benefits

Taking part in a clinical trial might offer the benefit of receiving a new, potentially more effective, treatment approach that isn't yet widely available. The goal is to improve the chances of getting rid of the cancer and preventing it from returning. However, like all medical treatments, there are potential risks, including side effects from the chemotherapy drugs. Your medical team will explain these in detail, as well as the types of tests you'll have, which can sometimes be uncomfortable. You always have the right to withdraw from the study at any time, for any reason, without it affecting your future medical care.

Locations (250)

Children's Hospital of Alabama
Verified postcode
Birmingham, United States
USA Health Strada Patient Care Center
Verified postcode
Mobile, United States
Providence Alaska Medical Center
Verified postcode
Anchorage, United States
Kingman Regional Medical Center
Verified postcode
Kingman, United States
Banner Children's at Desert
Verified postcode
Mesa, United States
Phoenix Childrens Hospital
Verified postcode
Phoenix, United States
Banner University Medical Center - Tucson
Verified postcode
Tucson, United States
Arkansas Children's Hospital
Verified postcode
Little Rock, United States
Kaiser Permanente-Anaheim
Verified postcode
Anaheim, United States
PCR Oncology
Verified postcode
Arroyo Grande, United States
Kaiser Permanente-Bellflower
Verified postcode
Bellflower, United States
Kaiser Permanente Downey Medical Center
Verified postcode
Downey, United States

Common questions

What is 'high-risk' rhabdomyosarcoma?

It means the cancer is more likely to come back or spread after treatment, so doctors are looking for more effective ways to treat it.

What is vinorelbine?

Vinorelbine is a chemotherapy drug that works by stopping cancer cells from growing and dividing.

What is 'maintenance therapy'?

It's a longer, ongoing treatment given after the main treatment to help keep the cancer from returning.

Will I get to choose my treatment group?

No, you will be randomly assigned to one of the two treatment groups by chance, like drawing lots.

What kind of tests will I have?

You might have blood tests, scans (like MRI or CT), and possibly bone marrow samples to check your health and how treatment is working.