Childhood botryoid rhabdomyosarcoma

Summary

Childhood botryoid rhabdomyosarcoma (MONDO:0003992) is a disease and 3 clinical trials. Top therapeutic interventions include cyclophosphamide anhydrous, dactinomycin, and irinotecan hydrochloride. A subtype of botryoid rhabdomyosarcoma — broader associated-gene and molecular evidence is on the parent page (see Disease family below).

At a glance

• Clinical trials: 3

Clinical features

No curated clinical features (Orphanet) for this disease.

Identifiers

Disease identifiers

Field	Value
Canonical name	childhood botryoid rhabdomyosarcoma
Mondo ID	MONDO:0003992
DOID	DOID:6786
NCIT	C35574
UMLS	C1332944
MedGen	232054
GARD	0023765
Is cancer (heuristic)	no

Also known as: botryoid rhabdomyosarcoma of childhood · botryoid-type embryonal rhabdomyosarcoma · childhood botryoid-type embryonal rhabdomyosarcoma · childhood sarcoma Botryoides · paediatric botryoid rhabdomyosarcoma · pediatric botryoid rhabdomyosarcoma

Disease family

This is a subtype of botryoid rhabdomyosarcoma. Genetic, therapeutic, and trial evidence is largely curated at the broader-term level — see the parent page for the associated-gene cohort and molecular evidence.

Classification path: disease by etiologic mechanism › cancer or benign tumor › neoplastic disease or syndrome › neoplasm › cancer › sarcoma › soft tissue sarcoma › rhabdomyosarcoma › embryonal rhabdomyosarcoma › botryoid rhabdomyosarcoma › childhood botryoid rhabdomyosarcoma

Related subtypes (2): botryoid-type embryonal rhabdomyosarcoma of the vagina, adult botryoid rhabdomyosarcoma

Subtypes (2): childhood vagina botryoid rhabdomyosarcoma, vulvar childhood botryoid-type embryonal rhabdomyosarcoma

Genetics & variants

GWAS landscape

No GWAS associations recorded — common-variant (GWAS) studies don’t cover this disease (typical for Mendelian / rare diseases). See the curated gene cohort and Mendelian overlap below.

Variant details and genetic-evidence tiers

No tiered GWAS variants or ClinVar records for this disease.

Genes & proteins

No associated-gene cohort resolved for this disease. Atlas builds the molecular and therapeutic sections — associated genes, protein families, druggability, pathways, interactions, and drug associations — by aggregating over a disease’s associated genes (resolved via GWAS / GenCC / ClinVar / CIViC), and none resolved here. This is expected for antibody-mediated, autoimmune, or otherwise non-gene-defined conditions; the curated evidence for this disease is its clinical features, GWAS susceptibility, and clinical trials (above).

Function

No pathway enrichment — requires an associated-gene cohort.

Therapeutics

No druggable-target or therapeutic data for this disease’s cohort.

Clinical trials & evidence

Clinical trials

Clinical trials: 3.

Phase distribution (across all retrieved trials)

Phase	Trials
PHASE3	3

Top trials by phase / activity

NCT	Phase	Status	Title
NCT02567435	PHASE3	ACTIVE_NOT_RECRUITING	Combination Chemotherapy With or Without Temsirolimus in Treating Patients With Intermediate Risk Rhabdomyosarcoma
NCT04994132	PHASE3	ACTIVE_NOT_RECRUITING	A Study to Compare Early Use of Vinorelbine and Maintenance Therapy for Patients With High Risk Rhabdomyosarcoma
NCT00354835	PHASE3	COMPLETED	Combination Chemotherapy and Radiation Therapy in Treating Patients With Newly Diagnosed Rhabdomyosarcoma

Drugs tested across these trials (top 30)

Molecule	Max phase	Trials referencing
CYCLOPHOSPHAMIDE ANHYDROUS	4	3
DACTINOMYCIN	4	3
IRINOTECAN HYDROCHLORIDE	4	2
VINORELBINE	4	2
TEMSIROLIMUS	4	1
CHEMBL4748391	0	3
CHEMBL541887	0	2