Appointment dossier — 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 Rhabdomyosarcoma
- Cisplatin — Human evidence · 2 positive · PMID 37660086, 23151423
- Topotecan — Human evidence · 1 positive · PMID 23151423
- Vincristine — Review evidence · 2 positive · PMID 18004572, 11558020, 41474586
- Actinomycin D — Review evidence · 1 positive · PMID 11558020, 41474586
- Cyclophosphamide — Review evidence · 1 positive · PMID 18004572, 41474586
- Doxorubicin — Review evidence · 1 positive · PMID 18004572
- Etoposide — Review evidence · 1 positive · PMID 37660086
- Genistein — Review evidence · 1 positive · PMID 42133114
- Ifosfamide — Review evidence · 1 positive · PMID 11558020
- Pembrolizumab — Animal evidence · 1 positive · PMID 37469254
“Positive” means a study reported a positive result — most are early lab/animal work that may not translate to people.
Open recruiting trials (18)
- NCT07466316 · Phase 3 — A Study Comparing Higher Dose Chemotherapy Over a Shorter Amount of Time to Lower Dose Chemotherapy Plus Maintenance Over a Longer Amount of Time in Patients With Newly Diagnosed Intermediate-Risk Rhabdomyosarcoma (IR RMS) (United States)
- NCT06816771 · Phase 2 — Evaluation of the Efficacy and Safety of Pazopanib in Combination with TGI/CIV for Recurrent or Refractory Rhabdomyosarcoma in Children or Adolescents (China)
- NCT06541262 · Phase 1 / Phase 2 — Silmitasertib (CX-4945) in Combination With Chemotherapy for Relapsed Refractory Solid Tumors (United States)
- NCT06094101 · Phase 1 / Phase 2 — Personalized Vaccination in Fusion+ Sarcoma Patients (PerVision) (Germany)
- NCT05457829 · Phase 2 — Doxorubicin Hydrochloride Liposome Combined With Irinotecan (AI Regimen) Versus VIT Regimen in the Treatment of Relapsed and Refractory Pediatric Rhabdomyosarcoma: a Prospective, Open-label, Randomized Controlled, Multicenter, Phase II Clinical Study (China)
- NCT06721689 · Phase 1 / Phase 2 — PEEL-224, Vincristine and Temozolomide in Pediatric Solid Tumors (United States)
- NCT06625190 · Phase 1 / Phase 2 — Alpha/Beta T and B Cell Depletion With Zoledronic Acid for Solid Tumors (United States)
- NCT06684327 · Phase 2 — Multi-cohort, Single-arm Phase II Study of Albumin-paclitaxel, Ifosfamide, and Cisplatin in the Treatment of Rare Advanced Tumors (China)
- NCT07173972 · Phase 2 — Dose-escalated, Hypofractionated, Definitive Proton Radiotherapy for Patients With Inoperable Soft Tissue Sarcoma. (Norway)
- NCT04796012 · Phase 1 / Phase 2 — VITAS: Atezolizumab in Combination With Chemotherapy for Pediatric Relapsed/Refractory Solid Tumors (United States)
- NCT06942416 · Phase 2 — Iparomlimab and Tuvonralimab With Chemoradiation for the Treatment of Locally Recurrent and Oligometastatic Cervical Cancer (China)
- NCT06289062 · Phase 2 — Neoadjuvant Chemotherapy Plus Camrelizumab for FIGO Stage IB1 Cervical Cancer (China)
- NCT06998394 · Phase 2 — A Single-Arm, Phase II Clinical Trail of Cadonilimab Combined With Chemoradiotherapy in Oligometastatic Stage IVB Cervical Cancer (China)
- NCT02013336 · Phase 1 — Phase 1 Dose-escalating Study of MM-398 (Irinotecan Sucrosofate Liposome Injection) Plus Intravenous Cyclophosphamide in Recurrent or Refractory Pediatric Solid Tumors (United States)
- NCT04715191 · Phase 1 — Interleukin-15 and -21 Armored Glypican-3-specific Chimeric Antigen Receptor Expressed in T Cells for Pediatric Solid Tumors (United States)
- NCT02508038 · Phase 1 — Alpha/Beta CD19+ Depleted Haploidentical Transplantation + Zometa for Pediatric Hematologic Malignancies and Solid Tumors (United States)
- NCT05857969 · Phase 1 — Ex Vivo Drug Sensitivity Testing and Multi-Omics Profiling (United States)
- NCT07148050 · Phase 1 — Immunotherapy for Solid Tumor Malignancies in Pediatrics Using Interleukin-15 and -21 Armored Glypican-3-specific Chimeric Antigen Receptor T Cells (United States)
Most-relevant first: trials that name Rhabdomyosarcoma, then broader trials you may still qualify for. 48 recruiting trials name this cancer on ClinicalTrials.gov. Eligibility is decided by each trial's team — bring these NCT numbers to your appointment.
Financial help to look into
- PAN Foundation — Copay assistance funds by diagnosis (funds open and close as money allows). https://www.panfoundation.org/
- HealthWell Foundation — Copay and premium assistance funds by disease. https://www.healthwellfoundation.org/
- CancerCare — financial assistance — Limited grants plus free financial counseling. https://www.cancercare.org/financial
- Family Reach — Help with everyday living costs (rent, transport, food) during treatment. https://familyreach.org/
- NeedyMeds — Searchable directory of drug patient-assistance and discount programs. https://www.needymeds.org/
For each medicine above, search manufacturer and nonprofit programs at medicineassistancetool.org.
Questions to ask your oncologist
- I've read that Cisplatin has been studied in people for Rhabdomyosarcoma — what's the evidence, and is it an option or available in a trial for me?
- I've read that Topotecan has been studied in people for Rhabdomyosarcoma — what's the evidence, and is it an option or available in a trial for me?
- Of the open trials I found (for example NCT07466316), am I eligible for any — here or at a larger cancer center?
- What is my exact diagnosis — the type, subtype, stage, and grade?
- Has my tumor had molecular or genomic testing (e.g. next-generation sequencing), and what did it find?
- Should I have inherited (germline) genetic testing, and could it affect my treatment or my family?
- What is the goal of treatment for me — cure, long-term control, or comfort?
- What are all of my standard treatment options, and what does each one involve?
- What is the realistic benefit of each option, in actual numbers?
- What are the most common and the most serious side effects, and how are they managed?
- How will we know if treatment is working, and how often will I be scanned or tested?
- If the first treatment doesn't work, what are the next options?
- Are there gentler options if I want to prioritize quality of life?
- Am I eligible for any clinical trials — here or at a larger/academic cancer center?
- Is my case reviewed by a multidisciplinary tumor board?
- Would a second opinion at a center that treats my cancer often be worthwhile?
- Could any of my prescriptions, over-the-counter drugs, or supplements interfere with treatment?
- Which symptoms are emergencies, and who do I call after hours?
- Should I see palliative or supportive care alongside my treatment?
- How will treatment affect my daily life, work, and (if it matters to me) fertility?
- What can I safely do myself — diet and activity — and is anything I'm taking risky?
- What will treatment cost, and is financial assistance available?