Chemotherapy for the Treatment of Patients With Newly Diagnosed Very Low-Risk and Low Risk Fusion Negative Rhabdomyosarcoma
This study is investigating new ways to treat children and young adults (up to 21 years old) with a type of soft tissue cancer called rhabdomyosarcoma (RMS). We know that RMS can affect different parts of the body. The main goals are to check if a shorter, less intense chemotherapy (24 weeks of vincristine and dactinomycin) works just as well for patients with very low-risk RMS, keeping in mind their quality of life. For patients with low-risk RMS, the study wants to confirm how effective a slightly longer, standard chemotherapy is. Additionally, it aims to understand how to best tailor treatments for patients whose cancer has specific genetic changes (mutations) to improve their chances of a good outcome. This is a Phase 3 trial, meaning it's comparing new approaches to existing treatments.
At a glance
What is this study about?
This study is looking at how to give the best treatment to children and young people (up to 21 years old) who have just been diagnosed with a type of cancer called rhabdomyosarcoma (RMS). RMS is a rare cancer that starts in soft tissues like muscles. The researchers want to find out if they can make treatments better by making them less harsh for some, and more targeted for others.
For those with very low-risk RMS, the study is testing if a shorter course of chemotherapy (24 weeks of two drugs: vincristine and dactinomycin) works as well as current treatments. The idea is to reduce the side effects and impact on their lives without making the treatment less effective. For patients with a low-risk RMS, the study will continue to use a standard chemotherapy plan to confirm its effectiveness, making sure that the new way of categorizing patients leads to the right treatments.
Finally, the study is also investigating if patients whose RMS has specific genetic changes (called MYOD1 or TP53 mutations) can benefit from a more intensive treatment. Researchers will look at blood and tissue samples to understand more about these cancers and how best to treat them. Overall, the aim is to improve the chances of recovery for all young people with RMS, while reducing the impact of treatment where possible.
Key takeaways
- This study is for children and young adults (up to age 21) with newly diagnosed rhabdomyosarcoma.
- It aims to find safer or more effective chemotherapy treatments.
- Treatment plans differ based on whether the cancer is very low-risk, low-risk, or has specific genetic changes.
- You'll have regular medical tests and check-ups during the study.
- Your doctors will closely monitor your health and treatment response.
Who may be eligible?
This study is for children and young adults up to 21 years old who have been newly diagnosed with certain types of rhabdomyosarcoma. These include embryonal rhabdomyosarcoma, spindle cell/sclerosing rhabdomyosarcoma, or a specific type of alveolar rhabdomyosarcoma (called FOXO1 fusion negative). Before joining this study, all patients must first enroll in another related study (called APEC14B1).
Your cancer will be carefully checked to see if it's considered 'very low-risk' or 'low-risk'. This helps decide which treatment plan you'll follow. For example, if you have very low-risk cancer that is Stage 1 and Clinical Group I, you might be in one group. If your cancer is low-risk, such as Stage 1 and Clinical Group II, or in some cases involving the eye, you might be in another group. Sometimes, a small sample of lymph nodes (small glands that are part of your immune system) may need to be taken, especially for certain tumour locations, to help understand the cancer better.
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 21 years old or younger?
- Have you been newly diagnosed with embryonal, spindle cell/sclerosing, or FOXO1 negative alveolar rhabdomyosarcoma?
- Are you willing to enroll in a related study (APEC14B1) first?
- Are you able to have the necessary scans and tests?
- Are you healthy enough to receive chemotherapy?
What does participation involve?
If you join this study, your journey will involve receiving chemotherapy. The type and length of chemotherapy will depend on the specific characteristics of your rhabdomyosarcoma. For some, this might be 24 weeks of vincristine and dactinomycin. Other patients might receive 12 weeks of vincristine, dactinomycin, and cyclophosphamide, followed by another 12 weeks of vincristine and dactinomycin. If your cancer has certain genetic changes, your treatment might be adjusted to be more intense. Throughout the study, you'll have various tests like biopsies, bone scans, CT scans, and MRI scans to monitor your condition and treatment response. You'll have regular hospital visits to receive medication and have check-ups. The total duration of active treatment will be 24 weeks for most, but follow-up appointments would continue after that.
Potential risks and benefits
Locations (180)
- Children's Hospital of AlabamaVerified postcodeBirmingham, United States· Recruiting
- USA Health Strada Patient Care CenterVerified postcodeMobile, United States· Recruiting
- Phoenix Childrens HospitalVerified postcodePhoenix, United States· Recruiting
- Arkansas Children's HospitalVerified postcodeLittle Rock, United States· Recruiting
- Kaiser Permanente Downey Medical CenterVerified postcodeDowney, United States· Recruiting
- Loma Linda University Medical CenterVerified postcodeLoma Linda, United States· Recruiting
- Miller Children's and Women's Hospital Long BeachVerified postcodeLong Beach, United States· Recruiting
- Children's Hospital Los AngelesVerified postcodeLos Angeles, United States· Recruiting
- Cedars-Sinai Medical CenterVerified postcodeLos Angeles, United States· Recruiting
- Mattel Children's Hospital UCLAVerified postcodeLos Angeles, United States· Recruiting
- Valley Children's HospitalVerified postcodeMadera, United States· Recruiting
- UCSF Benioff Children's Hospital OaklandVerified postcodeOakland, United States· Recruiting
Common questions
What is rhabdomyosarcoma?
It's a rare type of cancer that starts in soft tissues, often in places like muscles. This study focuses on treating it in children and young adults.
What does a 'Phase 3' trial mean?
A Phase 3 trial compares a new treatment or approach with standard treatments to see which is better or if the new one is at least as good but with fewer side effects.
What are 'genetic mutations' in cancer?
These are changes in the DNA of cancer cells. In this study, doctors are looking at specific changes (MYOD1 or TP53) to see if they can guide treatment decisions.
Will my treatment be different if I have "very low-risk" cancer?
Yes, the study is testing if a shorter, less intense chemotherapy course (24 weeks) can be just as effective for very low-risk cases, to reduce side effects.
What are the chemotherapy drugs used?
The main drugs in this study are vincristine, dactinomycin, and sometimes cyclophosphamide. Your doctor will explain how these drugs work and potential side effects.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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