kidney Wilms tumor

disease
On this page

Also known as adult nephroblastomaadult renal Wilms' tumourchildhood renal Wilms tumorchildhood renal Wilms tumourchildhood renal Wilms' cancerembryonal nephromanephroblastomanephroblastoma, malignantnonanaplastic renal Wilm's tumornonanaplastic renal Wilm's tumourrenal embryonic tumorrenal embryonic tumourrenal Wilms tumorrenal Wilms tumourrenal Wilms' tumorWilms tumorWilms tumor of the kidneyWilms tumourWilms tumour of the kidney

Summary

kidney Wilms tumor (MONDO:0019004) is a cancer (an umbrella term covering 8 Mondo subtypes) with 3 cohort genes (1 CIViC-evidence somatic driver) and 90 clinical trials. Top therapeutic interventions include dactinomycin, larotrectinib, and cabozantinib.

At a glance

  • Classification: Cancer
  • Prevalence: 1-9 / 100 000 (Europe) [Orphanet-validated]
  • Umbrella term: 8 Mondo subtypes
  • Cohort genes: 3
  • Phenotypes (HPO): 27
  • Clinical trials: 90

Clinical features

Epidemiology

Prevalence records

27 prevalence record(s), Orphanet, top 20 (validated / broadest geography first):

TypeClassValueGeographyValidation
Annual incidence1-9 / 1 000 0000.14EuropeValidated
Lifetime Prevalence1-9 / 100 0003.65EuropeValidated
Point prevalence1-9 / 100 000EuropeValidated
Prevalence at birth1-5 / 10 00010EuropeValidated
Annual incidence1-9 / 1 000 0000.144United KingdomValidated
Annual incidence<1 / 1 000 0000.072BulgariaValidated
Annual incidence<1 / 1 000 0000.006Czech RepublicValidated
Annual incidence<1 / 1 000 0000.089GermanyValidated
Annual incidence<1 / 1 000 0000.043IcelandValidated
Annual incidence<1 / 1 000 0000.065LatviaValidated
Annual incidence<1 / 1 000 0000.066PortugalValidated
Annual incidence1-9 / 1 000 0000.121AustriaValidated
Annual incidence1-9 / 1 000 0000.195BelgiumValidated
Annual incidence1-9 / 1 000 0000.155CroatiaValidated
Annual incidence1-9 / 1 000 0000.139EstoniaValidated
Annual incidence1-9 / 1 000 0000.158FinlandValidated
Annual incidence1-9 / 1 000 0000.17IrelandValidated
Annual incidence1-9 / 1 000 0000.111ItalyValidated
Annual incidence1-9 / 1 000 0000.12LithuaniaValidated
Annual incidence1-9 / 1 000 0000.157MaltaValidated

Signs & symptoms

Clinical features (HPO)

27 HPO clinical features (Orphanet curated; top 27 by frequency):

HPO IDTermFrequency
HP:0002664NeoplasmVery frequent (80-99%)
HP:0002667NephroblastomaVery frequent (80-99%)
HP:0031500Abdominal massVery frequent (80-99%)
HP:0002027Abdominal painFrequent (30-79%)
HP:0005580Duplication of renal pelvisFrequent (30-79%)
HP:0012587Macroscopic hematuriaFrequent (30-79%)
HP:0000028CryptorchidismOccasional (5-29%)
HP:0000047HypospadiasOccasional (5-29%)
HP:0000085Horseshoe kidneyOccasional (5-29%)
HP:0000086Ectopic kidneyOccasional (5-29%)
HP:0000526AniridiaOccasional (5-29%)
HP:0000822HypertensionOccasional (5-29%)
HP:0001528HemihypertrophyOccasional (5-29%)
HP:0001824Weight lossOccasional (5-29%)
HP:0001901PolycythemiaOccasional (5-29%)
HP:0001903AnemiaOccasional (5-29%)
HP:0001945FeverOccasional (5-29%)
HP:0002716LymphadenopathyOccasional (5-29%)
HP:0002896Neoplasm of the liverOccasional (5-29%)
HP:0002907Microscopic hematuriaOccasional (5-29%)
HP:0003072HypercalcemiaOccasional (5-29%)
HP:0008330Reduced von Willebrand factor activityOccasional (5-29%)
HP:0012871VaricoceleOccasional (5-29%)
HP:0031105Abnormal uterus morphologyOccasional (5-29%)
HP:0033834MalaiseOccasional (5-29%)
HP:0100526Neoplasm of the lungOccasional (5-29%)
HP:0002094DyspneaVery rare (<1-4%)

Identifiers

Disease identifiers

FieldValue
Canonical namekidney Wilms tumor
Mondo IDMONDO:0019004
Orphanet654
DOIDDOID:2154, DOID:5176
NCITC40407
SNOMED CT302849000
GARD0007892
MedDRA10029145
NORD1855
Is cancer (heuristic)yes

Also known as: adult nephroblastoma · adult renal Wilms’ tumour · childhood renal Wilms tumor · childhood renal Wilms tumour · childhood renal Wilms’ cancer · embryonal nephroma · kidney Wilms tumor · nephroblastoma · nephroblastoma, malignant · nonanaplastic renal Wilm’s tumor · nonanaplastic renal Wilm’s tumour · renal embryonic tumor · renal embryonic tumour · renal Wilms tumor · renal Wilms tumour · renal Wilms’ tumor · Wilms tumor · Wilms tumor of the kidney · Wilms tumour · Wilms tumour of the kidney (+5 more)

Data availability: 2 GenCC gene-disease records · 59 cell lines · 8 intOGen driver records.

Disease family

An umbrella term covering 8 Mondo subtypes.

Classification path: disease › human disease › disease by etiologic mechanism › cancer or benign tumorneoplastic disease or syndromeneoplasmcancermalignant urinary system neoplasmkidney cancerkidney Wilms tumor

Related subtypes (7): kidney sarcoma, mesoblastic nephroma, renal carcinoma, Graham-Boyle-Troxell syndrome, malignant mixed epithelial stromal tumor of the kidney, childhood malignant kidney neoplasm, malignant renal pelvis neoplasm

Subtypes (8): nonanaplastic kidney Wilms tumor, metachronous kidney Wilms’ tumor, mixed cell type kidney Wilms’ tumor, blastema predominant kidney Wilms tumor, epithelial predominant Wilms’ tumor, stromal predominant kidney Wilms tumor, adult kidney Wilms tumor, childhood kidney Wilms tumor

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

Mendelian disease overlap and somatic drivers

GenCC: 12 · Orphanet: 23 · OMIM-shared: 0 · Dual-evidence (GWAS+Mendelian): 0

Somatic driver evidence (intOGen + CIViC, cohort fanout)

GeneintOGen roleCancer typesCIViC
TP53LoFACC,ALL,AML,ANGS,ANSC,BCC,BL,BLADDER,BLCA,BRCA,CCRCC,CEAD,CESC,CHOL,CHRCC,CLLSLL,COAD,COADREAD,CSCC,DLBCLNOS,EGC,ES,ESCA,ESCC,GB,GBC,GBM,GIST,HCC,HGGNOS,HNSC,LGGNOS,LIPO,LMS,LNM,LUAD,LUSC,MBL,MEL,MLYM,MT,NBL,NETNOS,NHL,NPC,NSCLC,OS,OVT,PAAD,PANCREAS,PAST,PCM,PLMESO,PRAD,PRCC,PROSTATE,RCC,READ,SACA,SARCNOS,SCLC,SIC,SKCM,SKIN,SOFT_TISSUE,STAD,STOMACH,THYM,UCEC,UCS,UTUC,VULVA,WDTC,WTCIViC #45

GenCC gene–disease validity (cohort genes)

the Disease column is the GenCC-asserted condition — a cohort gene’s strongest validity may be for a related predisposition syndrome.

GeneClassificationInheritanceDiseaseRecords
TRIM28DefinitiveAutosomal dominantchildhood kidney Wilms tumor2
TRIP13SupportiveAutosomal dominantkidney Wilms tumor10

Orphanet rare-disease linkage (cohort genes)

GeneOrphanet IDRare disease
TP53Orphanet:1333Familial pancreatic carcinoma
TP53Orphanet:145Hereditary breast and/or ovarian cancer syndrome
TP53Orphanet:1501Adrenocortical carcinoma
TP53Orphanet:210159Adult hepatocellular carcinoma
TP53Orphanet:251576Gliosarcoma
TP53Orphanet:251579Giant cell glioblastoma
TP53Orphanet:251899Choroid plexus carcinoma
TP53Orphanet:2807Papilloma of choroid plexus
TP53Orphanet:293199Pleomorphic rhabdomyosarcoma
TP53Orphanet:3318Essential thrombocythemia
TP53Orphanet:524Li-Fraumeni syndrome
TP53Orphanet:52688Myelodysplastic syndrome
TP53Orphanet:585909B-lymphoblastic leukemia/lymphoma with t(9;22)(q34.1;q11.2)
TP53Orphanet:667662Breast implant-associated anaplastic large cell lymphoma
TP53Orphanet:668Osteosarcoma
TP53Orphanet:67038B-cell chronic lymphocytic leukemia
TP53Orphanet:70573Small cell lung cancer
TP53Orphanet:96253Cushing disease
TP53Orphanet:99756Alveolar rhabdomyosarcoma
TP53Orphanet:99757Embryonal rhabdomyosarcoma
TRIP13Orphanet:1052Mosaic variegated aneuploidy syndrome
TRIP13Orphanet:654Nephroblastoma
TRIM28Orphanet:654Nephroblastoma

Cohort genes → proteins

3 cohort genes, 3 distinct canonical proteins.

Evidence partition

SubsetGenes
civic_only1
multi_evidence2

Cohort genes (full)

SymbolHGNCEnsemblUniProtNameEvidence
TP53HGNC:11998ENSG00000141510P04637Cellular tumor antigen p53civic_evidence
TRIP13HGNC:12307ENSG00000071539Q15645Pachytene checkpoint protein 2 homologgencc
TRIM28HGNC:16384ENSG00000130726Q13263Transcription intermediary factor 1-betagencc

Cohort function summary

Lead sentence per gene, UniProt-curated.

SymbolProtein nameFunction (lead sentence)
TP53Cellular tumor antigen p53Multifunctional transcription factor that induces cell cycle arrest, DNA repair or apoptosis upon binding to its target DNA sequence.
TRIP13Pachytene checkpoint protein 2 homologPlays a key role in chromosome recombination and chromosome structure development during meiosis.
TRIM28Transcription intermediary factor 1-betaNuclear corepressor for KRAB domain-containing zinc finger proteins (KRAB-ZFPs).

Protein-family classification

Druggable: 0 · Difficult: 2 · Unknown: 1 · Druggable fraction: 0.0

Family distribution

Cohort families vs a genome-wide background (hypergeometric, BH-FDR; fold = observed/expected). Counts kept; sorted by enrichment, so the catch-all Other/Unknown bucket no longer leads.

FamilyGenesFoldFDR
Transcription factor25.5×0.081
Other/Unknown10.6×0.914

Per-gene assignment

SymbolFamilyDruggable?ECInterPro (top 3)
TP53Transcription factornop53_tumour_suppressor, p53-like_TF_DNA-bd_sf, p53_tetrameristn
TRIP13Other/UnknownnoClpA/B, AAA+_ATPase, ATPase_AAA_core
TRIM28Transcription factornoZnf_B-box, Bromodomain, Znf_RING

Expression context

Cohort genes with no expression data: 0.

3 cohort genes are a single-cell marker in ≥1 SCXA experiment.

Breadth distribution (Bgee present_calls)

BucketGenes
narrow (1-5 tissues)0
moderate (6-20)0
broad (>20)3
unknown0

Top tissues across cohort

TissueCohort genes
ventricular zone2
ganglionic eminence1
tendon of biceps brachii1
bronchial epithelial cell1
bronchus1
epithelium of bronchus1
left testis1
right testis1

Per-gene tissue summary (top 30)

SymbolBgee breadthFANTOM5 breadthSCXATop tissues
TP53223ubiquitousmarkerventricular zone, ganglionic eminence, tendon of biceps brachii
TRIP13212ubiquitousmarkerbronchial epithelial cell, epithelium of bronchus, bronchus
TRIM28145ubiquitousmarkerleft testis, right testis, ventricular zone

Protein interactions among cohort

Intra-cohort edges: 0.

Hub genes (top 10 by interactor count)

SymbolInteractor count
TP5322,736
TRIM288,167
TRIP134,676

Structural data

PDB: 3 · AlphaFold-only: 0 · No structure: 0

Cohort genes with PDB structures (top 30)

SymbolUniProtPDB entries
TP53P04637313
TRIM28Q1326310
TRIP13Q156456

Function

Pathway analysis

Distinct Reactome pathways touched by cohort: 62. Enrichment computed across 3 evidence-associated genes (2 with Reactome annotation).

Pathways by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 2 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

PathwayCohort genesFoldFDRSample cohort genes
Loss of function of TP53 in cancer due to loss of tetramerization ability15710.0×0.011TP53
Regulation of TP53 Expression12855.0×0.011TP53
Transcriptional activation of cell cycle inhibitor p2111427.5×0.011TP53
Activation of NOXA and translocation to mitochondria1951.7×0.011TP53
RUNX3 regulates CDKN1A transcription1815.7×0.011TP53
PI5P Regulates TP53 Acetylation1634.4×0.011TP53
Activation of PUMA and translocation to mitochondria1571.0×0.011TP53
TP53 Regulates Transcription of Caspase Activators and Caspases1475.8×0.011TP53
TP53 Regulates Transcription of Death Receptors and Ligands1475.8×0.011TP53
Urea cycle1439.2×0.011TP53
Regulation of TP53 Activity through Association with Co-factors1407.9×0.011TP53
TP53 regulates transcription of several additional cell death genes whose specific roles in p53-dependent apoptosis remain uncertain1380.7×0.011TP53
Stabilization of p531380.7×0.011TP53
TP53 Regulates Transcription of Genes Involved in G1 Cell Cycle Arrest1356.9×0.011TP53
Formation of Senescence-Associated Heterochromatin Foci (SAHF)1335.9×0.011TP53
Zygotic genome activation (ZGA)1335.9×0.011TP53
Regulation of NF-kappa B signaling1317.2×0.011TP53
TP53 Regulates Transcription of Genes Involved in G2 Cell Cycle Arrest1300.5×0.011TP53
SUMOylation of transcription factors1285.5×0.011TP53
TP53 Regulates Transcription of Genes Involved in Cytochrome C Release1271.9×0.011TP53
Regulation of TP53 Activity through Methylation1271.9×0.011TP53
TP53 regulates transcription of additional cell cycle genes whose exact role in the p53 pathway remain uncertain1259.6×0.011TP53
Regulation of TP53 Activity through Acetylation1228.4×0.012TP53
Pyroptosis1211.5×0.012TP53
Regulation of endogenous retroelements1184.2×0.013TRIM28
Oncogene Induced Senescence1167.9×0.014TP53
HCMV Infection1163.1×0.014TRIM28
Association of TriC/CCT with target proteins during biosynthesis1146.4×0.015TP53
Regulation of TP53 Degradation1146.4×0.015TP53
Ovarian tumor domain proteases1139.3×0.015TP53

GO biological processes by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 3 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

GO termCohort genesFoldFDRSample cohort genes
negative regulation of helicase activity15617.3×0.005TP53
cellular response to actinomycin D15617.3×0.005TP53
regulation of intrinsic apoptotic signaling pathway by p53 class mediator15617.3×0.005TP53
negative regulation of G1 to G0 transition15617.3×0.005TP53
positive regulation of mitochondrial membrane permeability12808.7×0.005TP53
oligodendrocyte apoptotic process12808.7×0.005TP53
negative regulation of glucose catabolic process to lactate via pyruvate12808.7×0.005TP53
negative regulation of pentose-phosphate shunt12808.7×0.005TP53
obsolete homolactic fermentation11872.4×0.005TP53
meiotic recombination checkpoint signaling11872.4×0.005TRIP13
signal transduction by p53 class mediator11872.4×0.005TP53
negative regulation of miRNA processing11872.4×0.005TP53
intrinsic apoptotic signaling pathway in response to hypoxia11872.4×0.005TP53
regulation of fibroblast apoptotic process11872.4×0.005TP53
T cell proliferation involved in immune response11404.3×0.005TP53
positive regulation of programmed necrotic cell death11404.3×0.005TP53
oxidative stress-induced premature senescence11404.3×0.005TP53
B cell lineage commitment11123.5×0.005TP53
T cell lineage commitment11123.5×0.005TP53
mRNA transcription11123.5×0.005TP53
positive regulation of RNA polymerase II transcription preinitiation complex assembly11123.5×0.005TP53
convergent extension involved in axis elongation11123.5×0.005TRIM28
embryonic placenta morphogenesis11123.5×0.005TRIM28
positive regulation of thymocyte apoptotic process11123.5×0.005TP53
cellular response to UV-C11123.5×0.005TP53
double-strand break repair2135.4×0.005TP53, TRIP13
regulation of mitochondrial membrane permeability involved in apoptotic process1936.2×0.006TP53
viral process1802.5×0.006TP53
mitochondrial DNA repair1802.5×0.006TP53
regulation of cell cycle G2/M phase transition1802.5×0.006TP53

Therapeutics

Drug target analysis

Approved (phase 4): 1 · Phase ≥3: 1 · Phased (≥1): 1 · Undrugged: 2

Druggability breadth: 3 of 3 evidence-associated genes (100%) have a ChEMBL target (buckets above are over the deeply-mined display cohort).

Genes with an approved drug

The molecule shown is one approved compound that hits the gene — not necessarily a drug of choice or one indicated for this disease.

SymbolExample approved molecule
TP53NITROFURANTOIN

Top cohort targets by molecule count

SymbolMoleculesMax phase
TP531964
TRIP1300
TRIM2800

Drugs targeting cohort genes (top 30)

MoleculeMax phaseTargets in cohort
NITROFURANTOIN4TP53
DIOSMIN4TP53
VERTEPORFIN4TP53
CANDESARTAN CILEXETIL4TP53
DIENESTROL4TP53
CLOTRIMAZOLE4TP53
COLCHICINE4TP53
NABUMETONE4TP53
SALMETEROL XINAFOATE4TP53
AMIODARONE HYDROCHLORIDE4TP53
FURAZOLIDONE4TP53
AMOXAPINE4TP53
RALOXIFENE HYDROCHLORIDE4TP53
NICARDIPINE HYDROCHLORIDE4TP53
SULCONAZOLE NITRATE4TP53
PYRITHIONE ZINC4TP53
LACTIC ACID4TP53
OXYMETHOLONE4TP53
CHLOROXINE4TP53
PROPIOLACTONE4TP53
CLOMIPRAMINE HYDROCHLORIDE4TP53
PHENYL AMINOSALICYLATE4TP53
THIORIDAZINE HYDROCHLORIDE4TP53
AMITRIPTYLINE HYDROCHLORIDE4TP53
ETHOPROPAZINE HYDROCHLORIDE4TP53
MECHLORETHAMINE HYDROCHLORIDE4TP53
ECONAZOLE NITRATE4TP53
TRIFLUPROMAZINE HYDROCHLORIDE4TP53
PROCHLORPERAZINE EDISYLATE4TP53
DEQUALINIUM CHLORIDE4TP53

Bioactivity and enzyme data

Enzyme cohort genes (≥1 EC): 0.

Cohort genes with ChEMBL bioactivity (full, sorted by assay count)

SymbolAssaysType breakdown
TP53869Binding:775, ADMET:83, Functional:10, Toxicity:1
TRIM2819Binding:19
TRIP139Binding:9

Cohort genes with high screening signal

≥100 ChEMBL assays — a studied-ness signal; see Therapeutics for approved-drug status.

SymbolChEMBL assays
TP53869

Pharmacogenomics

Cohort genes with a PharmGKB record: 3; with CPIC/DPWG dosing guidelines: 0.

No cohort gene has a CPIC/DPWG genotype-guided dosing guideline (PharmGKB).

Drug repurposing candidates

30 approved/phased drugs hit cohort targets but don’t yet appear in disease-level clinical trials. Target-inhibition rationale is strongest for cancer driver genes; a bioactivity hit is a screening signal, not a treatment claim.

CompoundMax phaseCohort target (bioactivity)
NITROFURANTOIN4TP53
DIOSMIN4TP53
VERTEPORFIN4TP53
CANDESARTAN CILEXETIL4TP53
DIENESTROL4TP53
CLOTRIMAZOLE4TP53
COLCHICINE4TP53
NABUMETONE4TP53
SALMETEROL XINAFOATE4TP53
AMIODARONE HYDROCHLORIDE4TP53
FURAZOLIDONE4TP53
AMOXAPINE4TP53
RALOXIFENE HYDROCHLORIDE4TP53
NICARDIPINE HYDROCHLORIDE4TP53
SULCONAZOLE NITRATE4TP53
PYRITHIONE ZINC4TP53
LACTIC ACID4TP53
OXYMETHOLONE4TP53
CHLOROXINE4TP53
PROPIOLACTONE4TP53
CLOMIPRAMINE HYDROCHLORIDE4TP53
PHENYL AMINOSALICYLATE4TP53
THIORIDAZINE HYDROCHLORIDE4TP53
AMITRIPTYLINE HYDROCHLORIDE4TP53
ETHOPROPAZINE HYDROCHLORIDE4TP53
MECHLORETHAMINE HYDROCHLORIDE4TP53
ECONAZOLE NITRATE4TP53
TRIFLUPROMAZINE HYDROCHLORIDE4TP53
PROCHLORPERAZINE EDISYLATE4TP53
DEQUALINIUM CHLORIDE4TP53

Druggability pyramid

Cohort genes binned by druggability tier (high → low):

TierDefinitionGenesSymbols
AApproved (phase 4 drug)1TP53
BPhased (≥1) drug, not yet approved0
CDruggable family + PDB, no drug0
DDruggable family + AlphaFold only, no drug0
EDifficult family or no structure, no drug2TRIP13, TRIM28

Undrugged target profiles

2 cohort genes are undrugged. Ranked by ‘starting-point quality’ (assay depth + drugged-partner adjacency).

SymbolChEMBL assaysDrugged partners (top 3)
TRIP139
TRIM2819

Clinical trials & evidence

Clinical trials

Clinical trials: 90.

Phase distribution (across all retrieved trials)

PhaseTrials
PHASE228
Not specified28
PHASE125
PHASE1/PHASE24
PHASE33
PHASE41
EARLY_PHASE11

Top trials by phase / activity

NCTPhaseStatusTitle
NCT00336531PHASE4COMPLETEDEfficacy of Prophylactic Itraconazole in High-Dose Chemotherapy and Autologous Hematopoietic Stem Cell Transplantation
NCT00352534PHASE3ACTIVE_NOT_RECRUITINGVincristine, Dactinomycin, and Doxorubicin With or Without Radiation Therapy or Observation Only in Treating Younger Patients Who Are Undergoing Surgery for Newly Diagnosed Stage I, Stage II, or Stage III Wilms’ Tumor
NCT00379340PHASE3ACTIVE_NOT_RECRUITINGCombination Chemotherapy With or Without Radiation Therapy in Treating Young Patients With Newly Diagnosed Stage III or Stage IV Wilms’ Tumor
NCT00945009PHASE3ACTIVE_NOT_RECRUITINGCombination Chemotherapy and Surgery in Treating Young Patients With Wilms Tumor
NCT02867592PHASE2ACTIVE_NOT_RECRUITINGCabozantinib-S-Malate in Treating Younger Patients With Recurrent, Refractory, or Newly Diagnosed Sarcomas, Wilms Tumor, or Other Rare Tumors
NCT03155620PHASE2ACTIVE_NOT_RECRUITINGTargeted Therapy Directed by Genetic Testing in Treating Pediatric Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphomas, or Histiocytic Disorders (The Pediatric MATCH Screening Trial)
NCT03210714PHASE2ACTIVE_NOT_RECRUITINGErdafitinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With FGFR Mutations (A Pediatric MATCH Treatment Trial)
NCT03213652PHASE2ACTIVE_NOT_RECRUITINGEnsartinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With ALK or ROS1 Genomic Alterations (A Pediatric MATCH Treatment Trial)
NCT03213704PHASE2ACTIVE_NOT_RECRUITINGLarotrectinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With NTRK Fusions (A Pediatric MATCH Treatment Trial)
NCT03698994PHASE2ACTIVE_NOT_RECRUITINGUlixertinib in Treating Patients With Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With MAPK Pathway Mutations (A Pediatric MATCH Treatment Trial)
NCT04195555PHASE2ACTIVE_NOT_RECRUITINGIvosidenib in Treating Patients With Advanced Solid Tumors, Lymphoma, or Histiocytic Disorders With IDH1 Mutations (A Pediatric MATCH Treatment Trial)
NCT04284774PHASE2ACTIVE_NOT_RECRUITINGTipifarnib for the Treatment of Advanced Solid Tumors, Lymphoma, or Histiocytic Disorders With HRAS Gene Alterations, a Pediatric MATCH Treatment Trial
NCT04320888PHASE2ACTIVE_NOT_RECRUITINGSelpercatinib for the Treatment of Advanced Solid Tumors, Lymphomas, or Histiocytic Disorders With Activating RET Gene Alterations, a Pediatric MATCH Treatment Trial
NCT04322318PHASE2RECRUITINGA Study of Combination Chemotherapy for Patients With Newly Diagnosed DAWT and Relapsed FHWT
NCT04851119PHASE1/PHASE2RECRUITINGTegavivint for the Treatment of Recurrent or Refractory Solid Tumors, Including Lymphomas and Desmoid Tumors
NCT04901702PHASE1/PHASE2RECRUITINGStudy of Onivyde With Talazoparib or Temozolomide in Children With Recurrent Solid Tumors and Ewing Sarcoma
NCT04968990PHASE2RECRUITINGTreatment of Newly Diagnosed Patient’s With Wilm’s Tumor Requiring Abdominal Radiation Delivered With Proton Beam Irradiation
NCT05384821PHASE1/PHASE2RECRUITINGMetronomic Chemotherapy in Wilms Tumor (MetroWilms-1906)
NCT05985161PHASE2RECRUITINGA Study of Selinexor in People With Wilms Tumors and Other Solid Tumors
NCT00001509PHASE2COMPLETEDA Phase II Trial of All-Trans-Retinoic Acid in Combination With Interferon-Alpha 2a in Children With Recurrent Neuroblastoma or Wilms’ Tumor
NCT00038207PHASE2COMPLETEDLiposomal Vincristine for Pediatric and Adolescent Patients With Relapsed Malignancies
NCT00141765PHASE2COMPLETEDStudy of High-Dose Chemotherapy With Bone Marrow or Stem Cell Transplant for Rare Poor-Prognosis Cancers
NCT00187031PHASE2COMPLETEDA Phase II Study of Topotecan in Children With Recurrent Wilms Tumor
NCT00335556PHASE2COMPLETEDCombination Chemotherapy, Radiation Therapy, and/or Surgery in Treating Patients With High-Risk Kidney Tumors
NCT01095926PHASE2COMPLETEDPharmacokinetic Study of Doxorubicin in Children With Cancer
NCT02452554PHASE2COMPLETEDLorvotuzumab Mertansine in Treating Younger Patients With Relapsed or Refractory Wilms Tumor, Rhabdomyosarcoma, Neuroblastoma, Pleuropulmonary Blastoma, Malignant Peripheral Nerve Sheath Tumor, or Synovial Sarcoma
NCT02581384PHASE1/PHASE2TERMINATEDStereotactic Body Radiotherapy (SBRT) for Pulmonary Metastases in Ewing Sarcoma, Rhabdomyosarcoma, and Wilms Tumors
NCT02624388PHASE2TERMINATEDStudy of Genistein in Pediatric Oncology Patients (UVA-Gen001)
NCT02689336PHASE2WITHDRAWNErlotinib in Combination With Temozolomide in Treating Relapsed/Recurrent/Refractory Pediatric Solid Tumors
NCT03213665PHASE2COMPLETEDTazemetostat in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With EZH2, SMARCB1, or SMARCA4 Gene Mutations (A Pediatric MATCH Treatment Trial)
NCT03213678PHASE2COMPLETEDSamotolisib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With TSC or PI3K/MTOR Mutations (A Pediatric MATCH Treatment Trial)
NCT03220035PHASE2COMPLETEDVemurafenib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With BRAF V600 Mutations (A Pediatric MATCH Treatment Trial)
NCT03233204PHASE2COMPLETEDOlaparib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With Defects in DNA Damage Repair Genes (A Pediatric MATCH Treatment Trial)
NCT03526250PHASE2COMPLETEDPalbociclib in Treating Patients With Relapsed or Refractory Rb Positive Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With Activating Alterations in Cell Cycle Genes (A Pediatric MATCH Treatment Trial)
NCT04791228PHASE2WITHDRAWNA Pilot Study of Thermodox and MR-HIFU for Treatment of Relapsed Solid Tumors
NCT05302921PHASE2COMPLETEDNeoadjuvant Dual Checkpoint Inhibition and Cryoablation in Relapsed/Refractory Pediatric Solid Tumors
NCT03618381PHASE1ACTIVE_NOT_RECRUITINGEGFR806 CAR T Cell Immunotherapy for Recurrent/Refractory Solid Tumors in Children and Young Adults
NCT04308330PHASE1RECRUITINGVorinostat in Combination With Chemotherapy in Relapsed/Refractory Solid Tumors and CNS Malignancies
NCT04377932PHASE1ACTIVE_NOT_RECRUITINGInterleukin-15 Armored Glypican 3-specific Chimeric Antigen Receptor Expressed in T Cells for Pediatric Solid Tumors
NCT04483778PHASE1ACTIVE_NOT_RECRUITINGB7H3 CAR T Cell Immunotherapy for Recurrent/Refractory Solid Tumors in Children and Young Adults

Drugs tested across these trials (top 30)

MoleculeMax phaseTrials referencing
DACTINOMYCIN44
LAROTRECTINIB44
CABOZANTINIB43
ENSARTINIB42
ERDAFITINIB42
IVOSIDENIB42
SELPERCATINIB42
SELUMETINIB42
TAZEMETOSTAT42
VEMURAFENIB42
ITRACONAZOLE41
PEGFILGRASTIM41
SELINEXOR41
TALAZOPARIB41
TOPOTECAN41
TIPIFARNIB32
BECOTATUG VEDOTIN31
DOCIPARSTAT SODIUM31
GALINPEPIMUT-S31
TARIQUIDAR31
SAMOTOLISIB22
ULIXERTINIB22
ENOBLITUZUMAB21
GENISTEIN21
LORVOTUZUMAB MERTANSINE21
TEGAVIVINT21
CHEMBL474839104
CHEMBL341555302
CHEMBL420955502
CHEMBL539843102