Skip to Main Content

ARST2032: Very Low-Risk and Low-Risk Rhabdomyosarcoma Study

About this study

Rhabdomyosarcoma (RMS) is a type of cancerous tumor found in soft tissue, such as muscles. Cancers can happen when there is a change in the genes (DNA) of a person’s cells. A patient’s prognosis can vary, depending on what kind of changes (mutations) they have. Some mutations increase a person’s risk for cancer.

Past clinical trials of patients with low-risk rhabdomyosarcoma (LR-RMS) showed that 90% of them survived for 4 years without a significant health event. But recently, scientists found new mutations for RMS. Patients with these mutations have a greater risk of a poor outcome. These new mutations are known as MYOD1 and TP53. Because they are newly discovered, researchers did not look for them in past RMS studies.

In this study, we will reclassify RMS patients and look for MYOD1 or TP53 mutations in tumor tissue samples. We will find out who has these changes and who does not.

Based on lab results, we will put RMS patients without these mutations into 2 groups:

  • Very low-risk (VLR)-RMS patients. These patients will be treated for 24 weeks with VA (vincristine and dactinomycin) chemotherapy. Because they are lower risk, they will not be treated as long, and will not get the chemotherapy drug cyclophosphamide, as used in other trials.
  • Low-risk (LR)-RMS patients. These patients will be treated with 12 weeks of VAC (vincristine, dactinomycin, and cyclophosphamide) chemotherapy, followed by 12 weeks of VA chemotherapy. We want to see how well these patients do.

For patients who do have the MYOD1 or TP53 mutations, and have VLR or LR disease, we will treat them with VAC therapy for 42 weeks to see if they would benefit from getting therapy longer than normal to fight their disease.

Eligibility overview

To take part in this study, you must:

  • Be already enrolled in the APEC14B1 clinical trial
  • Consent to DNA testing before being enrolled and treated on the ARST2032 trial
  • Up to 21 years old
  • Have newly diagnosed embryonal rhabdomyosarcoma, spindle cell/sclerosing RMS, or FOXO1 fusion negative alveolar rhabdomyosarcoma (ARMS)

The above information is intended to provide only a basic description about a research protocol that may be currently active at St. Jude. The details made available here may not be the most up-to-date information on protocols used by St. Jude. To receive full details about a protocol and its status and or use at St. Jude, a physician must contact St. Jude directly.


Full title:

A Prospective Phase 3 Study of Patients with Newly Diagnosed Very Low-Risk and Low-Risk Fusion Negative Rhabdomyosarcoma

Study goal:

Compare the effects of giving less chemotherapy drugs to people with VLR-RMS, who do not have the MYOD1 or TP53 mutation, to find out which is better; to find out how well people with LR-RMS, without MYOD1 or TP53 mutations, respond to standard chemotherapy, and to see the effects of giving chemotherapy drugs for a longer amount of time. We want to see if patients with RMS and the MYOD1 or TP53 mutation benefit from longer therapy than the shorter treatment we give to VLR or LR patients.




Up to 21 years old

Clinical trials categories:

Childhood Cancer Solid Tumors Rhabdomyosarcoma

For physicians and researchers

Patients accepted to St. Jude must be referred by a physician or other qualified medical professional. Learn how St. Jude can partner with you to care for your patient.


Learn more