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Appointment dossier — Ovarian Rhabdomyosarcoma

Bring this to your appointment. It summarizes what published studies report — it is not medical advice and does not say anything works. Decisions are yours and your care team’s.

Compounds studied in Ovarian Rhabdomyosarcoma

“Positive” means a study reported a positive result — most are early lab/animal work that may not translate to people.

Open recruiting trials (18)

Most-relevant first: trials that name Ovarian Rhabdomyosarcoma, then broader trials you may still qualify for. Eligibility is decided by each trial's team — bring these NCT numbers to your appointment.

Questions to ask your oncologist

  1. Has my tumor been tested for Desmin / myogenin / MyoD1, and would the result open up targeted treatments or trials?
  2. Has my tumor been tested for PAX3–FOXO1 / PAX7–FOXO1, and would the result open up targeted treatments or trials?
  3. Has my tumor been tested for DICER1, and would the result open up targeted treatments or trials?
  4. Of the open trials I found (for example NCT04657068), am I eligible for any — here or at a larger cancer center?
  5. Which RMS subtype do I have (embryonal, alveolar, spindle/sclerosing, pleomorphic) and how does it change treatment?
  6. Is fertility-sparing surgery possible and safe in my case?
  7. Which chemo backbone is recommended for my age and subtype?
  8. Do I need radiation for margin-positive or nodal disease?
  9. Are there trials matched to my tumor’s drivers (e.g., PAX–FOXO1, PI3K/AKT/mTOR, MSI)?
  10. Can we send tissue for RNA fusion panel and DNA NGS now, and again at progression to capture targets (e.g., TFCP2, FGFR4, ALK)?
  11. Do we have enough banked tumor (FFPE + fresh-frozen) and a recent biopsy to qualify for molecular trials?
  12. What is the plan if there’s inadequate response after 2–3 cycles—what are our pre-agreed switch criteria?
  13. Am I a candidate for neoadjuvant therapy to improve the chance of an R0 resection?
  14. If disease is limited, could metastasectomy, ablation, or SBRT consolidate a systemic response?
  15. Would anthracycline cardioprotection (dexrazoxane) help preserve intensity without compromising efficacy?
  16. Should we involve a sarcoma specialty center or enroll via a national consortium (SARC, EORTC, NCI)?
  17. Are there basket trials for my biomarkers (FGFR4, ALK, NTRK, BET/BRD4, YAP/TEAD, MDM2)?
  18. Could we combine targeted agents with RT or chemo on a protocol to blunt resistance feedback (e.g., PI3K/mTOR + MEK)?
  19. What’s our VTE prevention plan and symptom-triggered pathway for urgent evaluation?
  20. How will we monitor for cardiac, renal, and neurotoxicity, and what dose-modification rules protect outcomes?
  21. Do I qualify for compassionate use / expanded access if a matched trial isn’t available?
  22. Is there a role for ctDNA as an adjunct to imaging for trend-tracking in my case?