CLINICAL TRIAL

Proton Beam Radiation for Rhabdomyosarcoma

Metastatic
Newly Diagnosed
Recruiting · < 65 · All Sexes · Boston, MA

This study is evaluating whether proton beam radiation therapy may reduce side effects from radiation treatment for rhabdomyosarcoma.

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About the trial for Rhabdomyosarcoma

Treatment Groups

This trial involves 2 different treatments. Proton Beam Radiation is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.

Main TreatmentA portion of participants receive this new treatment to see if it outperforms the control.
Proton Beam Radiation
RADIATION
Control TreatmentAnother portion of participants receive the standard treatment to act as a baseline.

About The Treatment

Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Proton Beam Radiation
2007
Completed Phase 2
~70

Side Effect Profile for Proton Beam Radiation/ Capecitabine Dose Level 1

Proton Beam Radiation/ Capecitabine Dose Level 1
Show all side effects
100%
Fatigue
100%
Nausea
67%
Abdomen- pain
67%
Vomiting
33%
Anorexia
33%
Bilirubin
33%
Constipation
33%
Dehydration
0%
Hyperglycemia
0%
AST- SGOT
0%
Colitis
0%
Pain - Chest Wall
0%
Anxiety
0%
Back- pain
0%
Diarrhea w/o prior colostomy
0%
Dyspepsia
0%
Extremity-limb- pain
0%
Fever w/o neutropenia
0%
Weight loss
0%
ALT- SGPT
Fatigue
100%
Nausea
100%
Abdomen- pain
67%
Vomiting
67%
Anorexia
33%
Bilirubin
33%
Constipation
33%
Dehydration
33%
Hyperglycemia
0%
AST- SGOT
0%
Colitis
0%
Pain - Chest Wall
0%
Anxiety
0%
Back- pain
0%
Diarrhea w/o prior colostomy
0%
Dyspepsia
0%
Extremity-limb- pain
0%
Fever w/o neutropenia
0%
Weight loss
0%
ALT- SGPT
0%
This histogram enumerates side effects from a completed 2017 Phase 1 & 2 trial (NCT00438256) in the Proton Beam Radiation/ Capecitabine Dose Level 1 ARM group. Side effects include: Fatigue with 100%, Nausea with 100%, Abdomen- pain with 67%, Vomiting with 67%, Anorexia with 33%.

Eligibility

This trial is for patients born any sex aged 65 and younger. You must have received newly diagnosed for Rhabdomyosarcoma. There are 6 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
The patients must be treated with a chemotherapy regimen that is accepted by the majority of doctors. show original
The participant in the study must be willing to receive follow-up care from MGH for a minimum of five years after their treatment, and must also be willing to have annual visits (unless returning to MGH for follow-up care is too difficult) show original
The timing of radiation treatment must be based on the IRB protocol that was used to treat the patient, either within 35 days of the last chemotherapy treatment or surgery. show original
Patients with cancer that has been specifically identified as rhabdomyosarcoma. show original
Patients who are under 21 years old. show original
The cancer may only spread if it is between the ages of 2 and 10 and has a type of tissue that is found in very young children. show original
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Odds of Eligibility
Unknown<50%
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial
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Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: 4 years
Screening: ~3 weeks
Treatment: Varies
Reporting: 4 years
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: 4 years.
View detailed reporting requirements
Trial Expert
Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether Proton Beam Radiation will improve 1 primary outcome and 3 secondary outcomes in patients with Rhabdomyosarcoma. Measurement will happen over the course of 4 years.

Late toxicity
4 YEARS
Frequency and severity of late complications from irradiation using proton beam therapy in place of conventional photon beam therapy in pediatric patients with pediatric rhabdomyosarcomas.
4 YEARS
Local Control
4 YEARS
Rates of local control using proton radiotherapy.
4 YEARS
Dosimetric comparison
4 YEARS
Comparison of Dose distribution to tumor and surrounding normal structures using DVH's generated from the proton plan used to treat the patient and the photon plan generated for comparison purposes.
4 YEARS
Acute toxicity
4 YEARS
Frequency and severity of acute side effects from irradiation using proton beam therapy in this patient population.
4 YEARS

Who is running the study

Principal Investigator
T. Y.
Torunn Yock, MD
Massachusetts General Hospital

Patient Q & A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What are common treatments for rhabdomyosarcoma?

Chemotherapy is the most frequently used medication for rhabdomyosarcoma, although more targeted therapies have recently appeared on the market. Surgery is very often used when the tumors are not aggressive or small, and radiation is also used very commonly. Clinical trials are not the only options for treatment.

Anonymous Patient Answer

What causes rhabdomyosarcoma?

The diagnosis of rhabdomyosarcoma is based on its characteristic histologic appearance and the presence of rhabdomyoblasts in the tumor. Genetic and environmental factors may be involved in the development of rhabdomyosarcoma in some patients. The diagnosis of rhabdomyosarcoma should be considered in any patient with a well-circumscribed lesion and demonstrable rhabdomyoblasts with or without foci.

Anonymous Patient Answer

What is rhabdomyosarcoma?

Rhabdomyosarcoma is a soft, slow growing tumour and most commonly affect the extremities, particularly young patients. It has poorer prognosis and the 10-year survival for these tumours is lower than that of other soft tissue sarcomas. Diagnosis of rhabdomyosarcoma relies on histopathology examination of tumour tissue and examination of pertinent systemic and local tumour markers in search for signs of metastatic spread. Radiotherapy to treat the local disease is of limited use as the disease generally recurs in the pelvis and may be difficult to eradicate. Aggressive treatment including surgery, radiation and chemotherapy are the current standard of care and are particularly effective against early disease.

Anonymous Patient Answer

Can rhabdomyosarcoma be cured?

A good response for some patients following resection alone. The presence of extrarenal metastases was very indicative of a poor prognosis and must be considered in the decision for treatment as extrarenal metastases are quite difficult to excise from an amputated limb.

Anonymous Patient Answer

How many people get rhabdomyosarcoma a year in the United States?

The prevalence of RMS in infants and young children in the United States is estimated to be 1.3 cases per 100,000 populations and 3.2 to 7.5 cases per 100,000 populations for those in children one year old or younger.

Anonymous Patient Answer

What are the signs of rhabdomyosarcoma?

It is very difficult to diagnose skeletal metastatic rhabdomyosarcoma. We recommend CT scan and EKG of the chest and pelvis to identify metastatic disease. PET scanning is used to identify bone metastatic disease and is superior to CT in the evaluation of response to chemotherapy and to determine the size of the primary tumor. Magnetic resonance imaging should be considered to evaluate metastatic tumor burden in the abdomen and pelvis. These diagnostic imaging studies are essential for directing the therapy.

Anonymous Patient Answer

What does proton beam radiation usually treat?

In a current trial, only a small number of patients received p-Bx for their localized disease. This trial might be more effective if patients received proton beam therapy for their locoregional disease, and for those patients, proton beam irradiation should be considered for metastatic disease. Although we have had very few patients in this study, the results of our study in some patients suggested that proton beam radiation may be an alternative to particle beam radiation for the treatment of metastatic tumors in the pelvis.

Anonymous Patient Answer

What is the survival rate for rhabdomyosarcoma?

Given the age at onset, treatment type, location, pathology, and extent of metastatic disease, a five-year survival rate between 10% and 30% is reported; for patients older than 30 years, 5-year survival is 60%. Patients who are older than 30 years have comparable 5-year survival rates of about 30% regardless of the metastatic disease at diagnosis. While patients who do not progress with metastatic disease have better outcomes after surgery (median survival of 11 years vs. 2.8 years for those with metastasis), patients who suffer metastases, particularly when the metastatic disease is diffuse and/or bilateral, have poor outcomes (median survival of 4.5 years).

Anonymous Patient Answer

What is the latest research for rhabdomyosarcoma?

There are multiple advances to overcome to overcome rhabdomyosarcoma in addition to overcoming resistance to the conventional anti-cancer drugs and in addition to the improvement of the quality of the life of the patients.

Anonymous Patient Answer

What is the average age someone gets rhabdomyosarcoma?

In this cohort of patients, the mean age of presentation was 48.9. In terms of survival, 67% patients had no known risk factors, and 18% were diagnosed at a stage below stage IV. However, this is a retrospective study, and patients were diagnosed with rhabdomyosarcoma post-mortem.\n

Anonymous Patient Answer

What are the common side effects of proton beam radiation?

The side effects of proton beam radiation are mainly gastrointestinal (GIT) side effects (e.g. nausea, vomiting) and hematological (blood disorders including red blood cell and white cell count changes, and platelet dysfunction) side effects. Most chemotherapy agents are hematological side effects due to bone marrow suppression. We are aware of some patient-related side effects (e.g. pain, neuropathy) that occur in some patients receiving proton beam radiation in combination with chemotherapy. The side effects of proton therapies and conventional radiation therapy vary significantly.

Anonymous Patient Answer

How quickly does rhabdomyosarcoma spread?

The findings provide evidence that there may be variations in disease aggressiveness based on demographics, patient comorbidities, and histologic type of rhabdomyosarcoma subtypes. A prospective assessment of this variability is warranted.

Anonymous Patient Answer
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