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Disease

Rhabdomyosarcoma

Late-stage therapeutic development

Also known as rhabdomyosarcoma (disease), rhabdomyosarcoma, malignant.

19
Clinical trials
12
Associated genes
4
Related proteins

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Moderate
Key developments
  • 2 clinical trials expected to report results, the earliest in Q2 2030.
  • 1 industry development reported.
Upcoming Milestones
Clinical Milestones6View
Results posted 2
Results expected 2
Recently completed 2
Industry & Market1View
Activity timeline7

Clinical trials

12 sponsors · 0 new · 0 completed in the last 12 months (net +0)

The current development programme across all trial phases.

Clinical programme
19
All trials
6
Active
6
Late-stage
6
Completed
Late-stage studies

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

Phase 3 · Recruiting · Children's Oncology Group

A Randomized Phase 3 Study of Vincristine, Dactinomycin, Cyclophosphamide (VAC) Alternating With Vincristine and Irinotecan (VI) Versus VAC/VI Plus Temsirolimus (TORI, Torisel, NSC# 683864) in Patients With Intermediate Risk (IR) Rhabdomyosarcoma (RMS)

Phase 3 · Active, not recruiting · National Cancer Institute (NCI)

A Randomized Phase 3 Trial of Vinorelbine, Dactinomycin, and Cyclophosphamide (VINO-AC) Plus Maintenance Chemotherapy With Vinorelbine and Oral Cyclophosphamide (VINO-CPO) vs Vincristine, Dactinomycin and Cyclophosphamide (VAC) Plus VINO-CPO Maintenance in Patients With High Risk Rhabdomyosarcoma (HR-RMS)

Phase 3 · Active, not recruiting · Children's Oncology Group

Randomized Study of Vincristine, Dactinomycin and Cyclophosphamide (VAC) Versus VAC Alternating With Vincristine and Irinotecan (VI) for Patients With Intermediate-Risk Rhabdomyosarcoma (RMS)

Phase 3 · Completed · Children's Oncology Group

Vincristine, Dactinomycin, and Lower Doses of Cyclophosphamide With or Without Radiation Therapy for Patients With Newly Diagnosed Low-Risk Embryonal/Botryoid/Spindle Cell Rhabdomyosarcoma

Phase 3 · Completed · Children's Oncology Group

Rhabdomyosarcoma and Malignant Soft Tissue Tumours of Childhood

Phase 3 · Terminated · Gustave Roussy, Cancer Campus, Grand Paris
Recently completed

A Phase I/II Dose Escalation/Dose Expansion Study of Prexasertib in Combination With Irinotecan in Patients With Relapsed or RefractoryDesmoplastic Small Round Cell Tumor and Rhabdomyosarcoma

Phase 1/2 · Completed · Memorial Sloan Kettering Cancer Center

A Phase 2, Multicenter, Open-label Study to Assess Safety and Preliminary Activity of Eribulin Mesylate in Pediatric Subjects With Relapsed/Refractory Rhabdomyosarcoma (RMS), Non-rhabdomyosarcoma Soft Tissue Sarcoma (NRSTS) and Ewing Sarcoma (EWS)

Phase 2 · Completed · Eisai Inc.

A Phase II Study of Sorafenib With Chemotherapy, Radiation, and Surgery for High-Risk Soft Tissue Sarcomas

Phase 2 · Completed · OHSU Knight Cancer Institute

A Phase 2 Study of IMGN901 (Lorvotuzumab Mertansine; NSC#: 783609) in Children With Relapsed or Refractory Wilms Tumor, Rhabdomyosarcoma, Neuroblastoma, Pleuropulmonary Blastoma, Malignant Peripheral Nerve Sheath Tumor (MPNST) and Synovial Sarcoma

Phase 2 · Completed · Children's Oncology Group

Randomized Study of Vincristine, Dactinomycin and Cyclophosphamide (VAC) Versus VAC Alternating With Vincristine and Irinotecan (VI) for Patients With Intermediate-Risk Rhabdomyosarcoma (RMS)

Phase 3 · Completed · Children's Oncology Group

Vincristine, Dactinomycin, and Lower Doses of Cyclophosphamide With or Without Radiation Therapy for Patients With Newly Diagnosed Low-Risk Embryonal/Botryoid/Spindle Cell Rhabdomyosarcoma

Phase 3 · Completed · Children's Oncology Group

Associated genes

12 matches

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

FGFR40.60SLC67A10.45MYOD10.44FOXO10.42PAX70.38EWSR10.38FLI10.37SS180.37SSX10.37SSX20.37CLIP40.26MYOG0.25

Disease biology

4 matches

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

Fibroblast growth factor receptor 4via FGFR40.60Forkhead box protein O1via FOXO10.42RNA-binding protein EWSvia EWSR10.38Friend leukemia integration 1 transcription factorvia FLI10.37

Reference

Authoritative identity, definition & identifiers.

Defined in MeSH

A malignant solid tumor arising from mesenchymal tissues which normally differentiate to form striated muscle. It can occur in a wide variety of sites. It is divided into four distinct types: pleomorphic, predominantly in male adults; alveolar (RHABDOMYOSARCOMA, ALVEOLAR), mainly in adolescents and young adults; embryonal (RHABDOMYOSARCOMA, EMBRYONAL), predominantly in infants and children; and botryoidal, also in young children. It is one of the most frequently occurring soft tissue sarcomas and the most common in children under 15. (From Dorland, 27th ed; Holland et al., Cancer Medicine, 3d ed, p2186; DeVita Jr et al., Cancer: Principles & Practice of Oncology, 3d ed, pp1647-9)

Synonyms

rhabdomyosarcoma (disease), rhabdomyosarcoma, malignant

References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.