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New treatment options for rhabdomyosarcoma

Evolutionary Inspired Therapy for Newly Diagnosed, Metastatic, Fusion Positive Rhabdomyosarcoma

Phase 2 Interventional H. Lee Moffitt Cancer Center and Research Institute · NCT04388839

This study is testing four different chemotherapy options for newly diagnosed patients with a specific type of aggressive cancer called Fusion Positive rhabdomyosarcoma to see which works best.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment28 (estimated)
SexAll
SponsorH. Lee Moffitt Cancer Center and Research Institute Academic / other
Drugs / interventionschemotherapy, cyclophosphamide
Locations18 sites (Birmingham, Alabama and 17 other locations)
Trial IDNCT04388839 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates four different chemotherapy strategies for newly diagnosed patients with metastatic Fusion Positive rhabdomyosarcoma. Participants and their physicians will select from various treatment arms, including a first strike therapy, a maintenance therapy, an adaptively timed therapy, or conventional chemotherapy. The study aims to determine the effectiveness of these approaches in managing this aggressive form of cancer.

Who should consider this trial

Good fit: Ideal candidates are individuals with a new diagnosis of metastatic Fusion Positive rhabdomyosarcoma who have not received prior systemic chemotherapy.

Not a fit: Patients with non-metastatic rhabdomyosarcoma or those who have previously undergone systemic chemotherapy may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide more effective treatment options for patients with rhabdomyosarcoma, potentially improving survival rates.

How similar studies have performed: Other studies have explored various chemotherapy regimens for rhabdomyosarcoma, but this specific adaptive approach is relatively novel.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Participants must have a new histologic diagnosis of rhabdomyosarcoma
* Participants must have FISH, PCR or other molecular confirmation of PAX/FOXO1 fusion per institutional standards
* Participants must have sufficient tissue (up to 10 unstained FFPE) for correlative testing
* All participants must have distant metastatic disease; either biopsy positive or PET avid extranodal or distant nodal lesions determined by the investigator to be metastatic disease. Patients with a single distant metastatic site that has been excised prior to study entry are eligible
* No prior systemic chemotherapy
* Participants enrolled to Arm B, maintenance, must be able to take oral cyclophosphamide. Note: enteral administration of cyclophosphamide is allowable.
* Males and females of reproductive potential may not participate unless they have agreed to the use of, at minimum, two methods of contraception during and after treatment or abstinence.
* Women of childbearing potential should adhere to contraception for a period of 4 months after completion of systematic chemotherapy administration
* Men who are sexually active with women of child bearing potential should adhere to contraception for a period of 4 months after completion of systematic chemotherapy administration
* All patients and/or their parents or legal guardians must have the ability to understand and the willingness to sign a written informed consent or assent document.

Exclusion Criteria:

* Participants with regional lymph nodes as the only site of disease are not eligible. Distant nodal sites alone are eligible
* Participants who are receiving any other investigational agents for rhabdomyosarcoma are ineligible
* Participants must not be receiving any additional medicines being given for the specific purpose of treating cancer. Alternative medications including, but not limited to cannabis based products would not be a reason for exclusion
* Participants are ineligible if they have uncontrolled intercurrent illness including, but not limited to:

  * ongoing or active infection not expected to resolve with current antibiotic plan
  * cardiac arrhythmia
  * psychiatric illness/social situations that would limit compliance with study requirements
* Patients who are pregnant or breastfeeding are not eligible because there is no available information regarding human fetal or teratogenic toxicities. Females of childbearing potential must have a negative serum or urine pregnancy test within 24 hours of starting protocol therapy.
* Participants who are considered unable to comply with the safety monitoring requirements of the study are not eligible

Where this trial is running

Birmingham, Alabama and 17 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions RhabdomyosarcomaSoft Tissue CancerSkeletal Muscle Tissue CancerSarcoma
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.