VBP1

Summary

VBP1 (VHL binding protein 1, HGNC:12662) is a protein-coding gene on chromosome Xq28, encoding Prefoldin subunit 3 (P61758). Binds specifically to cytosolic chaperonin (c-CPN) and transfers target proteins to it. It is a selective cancer dependency (DepMap: 68.6% of cell lines).

The protein encoded by this gene interacts with the Von Hippel-Lindau protein to form an intracellular complex. The encoded protein functions as a chaperone protein, and may play a role in the transport of the Von Hippel-Lindau protein from the perinuclear granules to the nucleus or cytoplasm. Alternative splicing and the use of alternate transcription start sites results in multiple transcript variants encoding different protein isoforms.

Source: NCBI Gene 7411 — RefSeq curated summary.

At a glance

• Clinical variants (ClinVar): 43 total
• Cancer dependency (DepMap): dependent in 68.6% of screened cell lines
• MANE Select transcript: NM_003372

Identifiers

Gene identifiers

Field	Value
HGNC ID	HGNC:12662
Approved symbol	VBP1
Name	VHL binding protein 1
Location	Xq28
Locus type	gene with protein product
Status	Approved
Aliases	PFD3, PFDN3
Ensembl gene	ENSG00000155959
Ensembl biotype	protein_coding
OMIM	300133
Entrez	7411

Gene structure

Transcript identifiers

Ensembl transcripts: 9 — 8 protein_coding, 1 retained_intron

ENST00000286428, ENST00000460509, ENST00000535916, ENST00000625964, ENST00000910826, ENST00000926244, ENST00000926245, ENST00000926246, ENST00000971683

RefSeq mRNA: 4 — MANE Select: NM_003372 NM_001303543, NM_001303544, NM_001303545, NM_003372

CCDS: CCDS14765, CCDS78525

Canonical transcript exons

ENST00000286428 — 6 exons

Exon	Start	End
ENSE00001023641	155220183	155220307
ENSE00001023644	155227235	155227301
ENSE00001096478	155238772	155239841
ENSE00001096479	155216460	155216575
ENSE00003532837	155228384	155228482
ENSE00003545274	155236229	155236367

Expression profiles

Bgee: expression breadth ubiquitous, 300 present calls, max score 98.45.

FANTOM5 (CAGE): breadth ubiquitous, TPM avg 59.6194 / max 1285.2682, expressed in 1817 samples.

FANTOM5 promoters (2 alternative TSS)

Promoter ID	TPM avg	Samples expressed
198218	58.5054	1817
198217	1.1140	774

Top tissues by expression

300 total, by Bgee expression score (0-100, higher = more expressed):

Tissue	Anatomy ID	Expression score	Quality
biceps brachii	UBERON:0001507	98.45	gold quality
skeletal muscle tissue of rectus abdominis	UBERON:0004511	98.19	gold quality
diaphragm	UBERON:0001103	98.13	gold quality
vastus lateralis	UBERON:0001379	98.12	gold quality
ganglionic eminence	UBERON:0004023	98.10	gold quality
skeletal muscle tissue of biceps brachii	UBERON:0004502	98.10	gold quality
quadriceps femoris	UBERON:0001377	97.99	gold quality
left ventricle myocardium	UBERON:0006566	97.99	gold quality
ventricular zone	UBERON:0003053	97.81	gold quality
deltoid	UBERON:0001476	97.80	gold quality
heart right ventricle	UBERON:0002080	97.75	gold quality
myocardium	UBERON:0002349	97.61	gold quality
substantia nigra pars compacta	UBERON:0001965	97.59	gold quality
triceps brachii	UBERON:0001509	97.58	gold quality
postcentral gyrus	UBERON:0002581	97.58	gold quality
substantia nigra pars reticulata	UBERON:0001966	97.55	gold quality
orbitofrontal cortex	UBERON:0004167	97.38	gold quality
cranial nerve II	UBERON:0000941	97.34	gold quality
embryo	UBERON:0000922	97.29	gold quality
Brodmann (1909) area 46	UBERON:0006483	97.29	gold quality
pons	UBERON:0000988	97.23	gold quality
skeletal muscle tissue	UBERON:0001134	97.23	gold quality
corpus callosum	UBERON:0002336	97.11	gold quality
parietal lobe	UBERON:0001872	97.03	gold quality
cardiac muscle of right atrium	UBERON:0003379	96.98	gold quality
cortical plate	UBERON:0005343	96.96	gold quality
tibialis anterior	UBERON:0001385	96.95	gold quality
muscle tissue	UBERON:0002385	96.91	gold quality
lateral nuclear group of thalamus	UBERON:0002736	96.87	gold quality
trabecular bone tissue	UBERON:0002483	96.84	gold quality

Single-cell (SCXA)

Detected in 1 experiment(s), a significant marker in 1.

Experiment	Marker?	Max mean expression
E-ANND-3	no	0.00

Regulation

Is transcription factor: no

miRNA regulators (miRDB)

84 targeting VBP1, top 30 by miRDB confidence (max_score; target_count = how many genes the miRNA targets in total — lower means more specific):

miRNA	Max score	Avg score	miRNA target_count
HSA-MIR-5692A	100.00	74.40	6850
HSA-MIR-4262	100.00	73.26	3931
HSA-MIR-3662	99.99	73.82	5684
HSA-MIR-181A-5P	99.99	72.96	2995
HSA-MIR-181B-5P	99.99	72.97	2996
HSA-MIR-181C-5P	99.99	72.95	2996
HSA-MIR-181D-5P	99.99	73.04	2997
HSA-MIR-548C-3P	99.99	74.01	7587
HSA-MIR-4775	99.98	75.00	6394
HSA-MIR-568	99.98	69.86	2084
HSA-MIR-3148	99.97	75.06	6478
HSA-MIR-3658	99.96	73.87	4379
HSA-MIR-590-3P	99.96	74.34	6478
HSA-MIR-23A-3P	99.95	74.24	3163
HSA-MIR-23B-3P	99.95	74.24	3163
HSA-MIR-23C	99.95	73.92	3192
HSA-MIR-9718	99.94	68.91	918
HSA-MIR-539-5P	99.93	70.30	2855
HSA-MIR-627-3P	99.90	71.42	3316
HSA-MIR-7162-3P	99.89	68.16	1682
HSA-MIR-153-5P	99.89	73.86	6317
HSA-MIR-6124	99.87	69.78	3551
HSA-MIR-12119	99.87	68.35	1653
HSA-MIR-4503	99.85	71.45	1869
HSA-MIR-4307	99.82	70.45	3374
HSA-MIR-2052	99.79	69.37	2031
HSA-MIR-4802-3P	99.72	70.13	1273
HSA-MIR-1208	99.70	68.28	1533
HSA-MIR-518A-5P	99.70	69.01	2209
HSA-MIR-527	99.70	69.01	2209

Functional genomics

DepMap (CRISPR cell-line fitness): dependent in 68.6% of screened cell lines.

Literature-anchored findings (GeneRIF, showing 7)

• binds to HIF-1 alpha and regulates the function of HIF-1 alpha (PMID:12613020)
• VBP1 and cullin2/von Hippel-Lindau are required for proper HIV-1 expression at a step between integrase-dependent proviral integration into the host genome and transcription of viral genes (PMID:17698809)
• VBP1 acts together with p97 to regulate hMSH4 degradation. (PMID:23964080)
• Data show that von Hippel-Lindau-binding protein 1 (VBP1) enhances the stability of von Hippel-Lindau tumor suppressor protein (pVHL) and facilitates pVHL-mediated ubiquitination of hypoxia-inducible factor 1, alpha subunit (HIF-1alpha). (PMID:29121446)
• VBP1 modulates Wnt/beta-catenin signaling by mediating the stability of the transcription factors TCF/LEFs. (PMID:32989053)
• The prefoldin complex stabilizes the von Hippel-Lindau protein against aggregation and degradation. (PMID:33137104)
• VBP1 promotes tumor proliferation as a part of the hypoxia-related signature in esophageal squamous cell carcinoma. (PMID:38700744)

Cross-species orthologs

5 orthologs

Organism	Symbol	Gene ID
danio_rerio	vbp1	ENSDARG00000030241
mus_musculus	Vbp1	ENSMUSG00000031197
drosophila_melanogaster	CG15676	FBGN0034651
drosophila_melanogaster	mgr	FBGN0264694
caenorhabditis_elegans	pfd-3	WBGENE00006889

Protein

Protein identifiers

Prefoldin subunit 3 — P61758 (reviewed: P61758)

Alternative names: HIBBJ46, von Hippel-Lindau-binding protein 1

All UniProt accessions (1): P61758

UniProt curated annotations — full annotation on UniProt →

Function. Binds specifically to cytosolic chaperonin (c-CPN) and transfers target proteins to it. Binds to nascent polypeptide chain and promotes folding in an environment in which there are many competing pathways for nonnative proteins.

Subunit / interactions. Heterohexamer of two PFD-alpha type and four PFD-beta type subunits. Binds to the C-terminal part of VHL.

Subcellular location. Cytoplasm. Nucleus.

Tissue specificity. Ubiquitous.

Similarity. Belongs to the prefoldin subunit alpha family.

Isoforms (2)

UniProt ID	Names	Canonical?
P61758-1	1	yes
P61758-2	2

RefSeq proteins (4): NP_001290472, NP_001290473, NP_001290474, NP_003363* (*=MANE)

Domains & families (InterPro)

ID	Name	Type
IPR004127	Prefoldin_subunit_alpha	Family
IPR009053	Prefoldin	Homologous_superfamily
IPR016655	PFD3	Family

Pfam: PF02996

UniProt features (6 total): modified residue 2, initiator methionine 1, chain 1, splice variant 1, sequence variant 1

Structure

Experimental structures (PDB)

6 structures.

PDB	Method	Resolution (Å)
7WU7	ELECTRON MICROSCOPY	3.85
6NR8	ELECTRON MICROSCOPY	7.8
6NRD	ELECTRON MICROSCOPY	8.2
6NRC	ELECTRON MICROSCOPY	8.3
6NR9	ELECTRON MICROSCOPY	8.5
6NRB	ELECTRON MICROSCOPY	8.7

Predicted structure (AlphaFold)

Model	pLDDT	Fraction very-high
AF-P61758-F1	87.09	0.70

Functional residue map

Curated UniProt residues grouped by drug-discovery relevance — catalytic, ligand-binding, modification, and mutation-validated positions. Source: UniProtKB sequence features.

Post-translational modifications (2): 2, 59

Function

Pathways and Gene Ontology

Reactome pathways

1 pathways

ID	Pathway
R-HSA-389957	Prefoldin mediated transfer of substrate to CCT/TriC

MSigDB gene sets: 175 (showing top): BORCZUK_MALIGNANT_MESOTHELIOMA_UP, MORF_BUB1, MODULE_151, CHIANG_LIVER_CANCER_SUBCLASS_UNANNOTATED_DN, MORF_HDAC1, MORF_UBE2N, MORF_HDAC2, MODULE_16, GOBP_NEGATIVE_REGULATION_OF_CELLULAR_COMPONENT_ORGANIZATION, PUJANA_CHEK2_PCC_NETWORK, GCM_PPP1CC, GOBP_PROTEIN_MATURATION, GNF2_ANP32B, MILI_PSEUDOPODIA_HAPTOTAXIS_UP, GOBP_PROTEIN_STABILIZATION

GO Biological Process (4): protein folding (GO:0006457), microtubule-based process (GO:0007017), tubulin complex assembly (GO:0007021), negative regulation of amyloid fibril formation (GO:1905907)

GO Molecular Function (4): amyloid-beta binding (GO:0001540), tubulin binding (GO:0015631), obsolete unfolded protein binding (GO:0051082), protein binding (GO:0005515)

GO Cellular Component (5): nucleus (GO:0005634), cytoplasm (GO:0005737), cytosol (GO:0005829), prefoldin complex (GO:0016272), protein-containing complex (GO:0032991)

Reactome top-level categories

Rollup of top-1 pathways:

Category	Pathways
Cooperation of Prefoldin and TriC/CCT in actin and tubulin folding	1

GO top-level categories

Rollup of top GO terms by namespace:

Category	Terms
cellular process	2
cellular anatomical structure	2
protein maturation	1
protein-containing complex assembly	1
negative regulation of protein metabolic process	1
negative regulation of supramolecular fiber organization	1
regulation of amyloid fibril formation	1
amyloid fibril formation	1
peptide binding	1
cytoskeletal protein binding	1
binding	1
intracellular membrane-bounded organelle	1
intracellular anatomical structure	1
cytoplasm	1
protein-containing complex	1
cellular_component	1

Protein interactions and networks

STRING

2303 interactions, top by confidence (×1000):

Protein A	Protein B	Partner UniProt	Score
VBP1	PFDN1	O60925	993
VBP1	PFDN5	Q99471	988
VBP1	PFDN4	Q9NQP4	980
VBP1	PFDN2	Q9UHV9	928
VBP1	PFDN6	O15212	866
VBP1	MSH4	O15457	827
VBP1	VHL	P40337	809
VBP1	CUL2	Q13617	780
VBP1	TCP1	P17987	602
VBP1	UXT	Q9UBK9	558
VBP1	FUNDC2	Q9BWH2	505
VBP1	TCHP	Q9BT92	503
VBP1	ANP32A	P39687	495
VBP1	CMC4	P56277	479
VBP1	URI1	O94763	476

IntAct

221 interactions, top by confidence:

A	B	Type	Score
VBP1	PFDN4	psi-mi:“MI:0915”(physical association)	0.870
PFDN4	VBP1	psi-mi:“MI:0915”(physical association)	0.870
VBP1	PFDN2	psi-mi:“MI:0915”(physical association)	0.870
VBP1	UBL7	psi-mi:“MI:0915”(physical association)	0.830
UBL7	VBP1	psi-mi:“MI:0915”(physical association)	0.830
VBP1	PFDN5	psi-mi:“MI:0915”(physical association)	0.740
PFDN4	PFDN6	psi-mi:“MI:0914”(association)	0.730
CFTR	ESYT2	psi-mi:“MI:2364”(proximity)	0.710
VBP1	PFDN6	psi-mi:“MI:0914”(association)	0.640
MAPK7	PFDN6	psi-mi:“MI:0914”(association)	0.640
RCCD1	SPAG9	psi-mi:“MI:0914”(association)	0.640
CCT2	PPP6C	psi-mi:“MI:0914”(association)	0.640
PFDN1	PFDN6	psi-mi:“MI:0914”(association)	0.640
DCAF7	PFDN6	psi-mi:“MI:0914”(association)	0.570
PNMA1	VBP1	psi-mi:“MI:0915”(physical association)	0.560

BioGRID (320): VBP1 (Two-hybrid), PNMA1 (Two-hybrid), UBL7 (Two-hybrid), VBP1 (Affinity Capture-MS), VBP1 (Affinity Capture-MS), CBS (Co-fractionation), FLNA (Co-fractionation), TXNDC12 (Co-fractionation), VBP1 (Co-fractionation), VBP1 (Co-fractionation), VBP1 (Co-fractionation), VBP1 (Co-fractionation), VBP1 (Co-fractionation), VBP1 (Co-fractionation), VBP1 (Co-fractionation)

ESM2 similar proteins: A0A1D5P556, A1A5P5, A7RX34, A7T0W1, A8WVJ9, A8XPL7, O18054, O94307, P40005, P46988, P48363, P57741, P59048, P61758, P61759, Q09305, Q0VIA1, Q10143, Q148L7, Q17827, Q21993, Q2KI89, Q2TBX2, Q3ZCF2, Q4R6W4, Q54JS0, Q54LS2, Q54ND3, Q5R629, Q5R6P5, Q5RCG9, Q5RFA9, Q61SU8, Q642A0, Q6CKH5, Q6DJ25, Q6GKV1, Q6IP67, Q8SYD0, Q9BTT4

Diamond homologs: B0ZT47, O18054, P48363, P57741, P61758, P61759, Q10143, Q2TBX2, Q54LS2, Q5RCG9, Q9VGP6

SIGNOR signaling

1 interactions.

A	Effect	B	Mechanism
VBP1	“form complex”	“Prefoldin co-chaperone”	binding

Enriched among interaction partners

Reactome pathways and GO biological processes over-represented among this gene’s 116 IntAct physical interaction partners (hypergeometric vs the genome-wide background, BH-FDR, gene-set size 15–500, ranked by fold). A functional readout of the neighbourhood — distinct from this gene’s own memberships above, and biased toward well-studied / hub proteins, so read it as themes rather than proof.

Reactome pathways:

Pathway	Partners	Fold	FDR
Prefoldin mediated transfer of substrate to CCT/TriC	10	54.7×	5e-13
Activation of AMPK downstream of NMDARs	9	47.6×	3e-11
Formation of tubulin folding intermediates by CCT/TriC	8	47.0×	4e-10
Cooperation of Prefoldin and TriC/CCT in actin and tubulin folding	8	45.3×	5e-10
Microtubule-dependent trafficking of connexons from Golgi to the plasma membrane	6	45.3×	8e-08
Transport of connexons to the plasma membrane	6	45.3×	8e-08
Gap junction trafficking and regulation	6	39.6×	2e-07
Gap junction trafficking	6	39.6×	2e-07

GO biological processes:

GO term	Partners	Fold	FDR
mitotic spindle organization	6	15.2×	6e-04
microtubule cytoskeleton organization	10	11.3×	6e-06
mitotic cell cycle	7	8.8×	2e-03

Disease & clinical

Clinical variants and AI predictions

ClinVar

43 variants total. Per-class counts are floors (≥ shown; pagination cap):

Classification	Count (floor)
Pathogenic	0
Likely pathogenic	0
Uncertain significance	6
Likely benign	0
Benign	1

Top pathogenic / likely-pathogenic (0)

SpliceAI

1106 predictions. Top by Δscore:

Variant	Effect	Δscore
X:155216576:G:GG	donor_gain	1.0000
X:155220182:GGAA:G	acceptor_gain	1.0000
X:155220304:GAAG:G	donor_gain	1.0000
X:155220305:AAGG:A	donor_loss	1.0000
X:155220306:AG:A	donor_gain	1.0000
X:155220306:AGG:A	donor_loss	1.0000
X:155220307:GG:G	donor_gain	1.0000
X:155220307:GGT:G	donor_loss	1.0000
X:155220308:G:GG	donor_gain	1.0000
X:155220308:GTAA:G	donor_loss	1.0000
X:155227226:T:TA	acceptor_gain	1.0000
X:155227230:CACA:C	acceptor_loss	1.0000
X:155227232:CAG:C	acceptor_loss	1.0000
X:155227233:A:AG	acceptor_gain	1.0000
X:155227233:A:T	acceptor_loss	1.0000
X:155227233:AG:A	acceptor_gain	1.0000
X:155227234:G:GA	acceptor_gain	1.0000
X:155227234:GG:G	acceptor_gain	1.0000
X:155227234:GGC:G	acceptor_gain	1.0000
X:155227234:GGCT:G	acceptor_gain	1.0000
X:155227234:GGCTA:G	acceptor_gain	1.0000
X:155227302:G:GG	donor_gain	1.0000
X:155236223:CTTCA:C	acceptor_loss	1.0000
X:155236224:TTCA:T	acceptor_loss	1.0000
X:155236225:TCA:T	acceptor_loss	1.0000
X:155236226:CA:C	acceptor_loss	1.0000
X:155236227:A:AG	acceptor_gain	1.0000
X:155236228:G:C	acceptor_loss	1.0000
X:155236228:G:GG	acceptor_gain	1.0000
X:155236228:GGCT:G	acceptor_gain	1.0000

AlphaMissense

1309 scored. Top likely-pathogenic:

Variant	Protein change	am_pathogenicity
X:155216570:T:C	F30L	1.000
X:155216572:T:A	F30L	1.000
X:155216572:T:G	F30L	1.000
X:155220289:T:C	L67P	1.000
X:155228472:T:C	L125P	1.000
X:155228474:T:A	W126R	1.000
X:155228474:T:C	W126R	1.000
X:155228478:T:C	L127S	1.000
X:155228480:G:A	G128R	1.000
X:155228480:G:C	G128R	1.000
X:155228480:G:T	G128W	1.000
X:155228481:G:A	G128E	1.000
X:155228481:G:T	G128V	1.000
X:155236242:T:A	L133H	1.000
X:155236242:T:C	L133P	1.000
X:155236295:G:C	A151P	1.000
X:155236296:C:A	A151D	1.000
X:155236338:T:C	L165P	1.000
X:155236341:G:C	R166P	1.000
X:155236347:A:C	Q168P	1.000
X:155238777:G:C	A177P	1.000
X:155238778:C:A	A177D	1.000
X:155238781:G:C	R178T	1.000
X:155238781:G:T	R178M	1.000
X:155238782:G:C	R178S	1.000
X:155238782:G:T	R178S	1.000
X:155238786:T:G	Y180D	1.000
X:155238791:T:A	N181K	1.000
X:155238791:T:G	N181K	1.000
X:155216556:T:C	I25T	0.999

dbSNP variants (sampled 300 via entrez): RS1000251998 (X:155218009 A>T), RS1000557317 (X:155237998 T>C), RS1000583890 (X:155206688 C>G,T), RS1000702418 (X:155217700 G>A,C), RS1000821034 (X:155228429 C>A,G), RS1000961085 (X:155227965 A>T), RS1001224265 (X:155233129 A>G), RS1001275065 (X:155233795 A>T), RS1001482109 (X:155223962 C>G,T), RS1001621806 (X:155223351 C>A,T), RS1001923243 (X:155216226 C>G,T), RS1002221356 (X:155225556 T>C), RS1002224996 (X:155235440 T>C), RS1002277449 (X:155235911 C>G), RS1002383160 (X:155215266 G>A,T)

Disease associations

OMIM: gene MIM:300133 | disease phenotypes:

GenCC curated gene-disease

Mondo (1): ependymoma (MONDO:0016698)

Orphanet (1): Ependymoma (Orphanet:251636)

HPO phenotypes

0 total (0 of 0 shown, HPO-id order):

GWAS associations

0 associations (top):

MeSH disease descriptors (1)

Descriptor	Name	Tree numbers
D004806	Ependymoma	C04.557.465.625.600.380.290; C04.557.470.670.380.290; C04.557.580.625.600.380.290

Drugs & pharmacology

Drug and pharmacology data

Is drug target: no

PharmGKB: 1 entry (VIP=true, CPIC=false)

CTD chemical–gene interactions

30 total (human), top 30 by PubMed support.

Chemical	Actions (top 5)	PubMed papers
bisphenol A	decreases expression, increases expression	3
sodium arsenite	increases expression, decreases expression, increases abundance	3
Valproic Acid	affects expression, decreases methylation	2
dicrotophos	decreases expression	1
potassium chromate(VI)	decreases expression	1
nickel sulfate	decreases expression	1
di-n-butylphosphoric acid	affects expression	1
4-(5-benzo(1,3)dioxol-5-yl-4-pyridin-2-yl-1H-imidazol-2-yl)benzamide	affects cotreatment, increases expression	1
bisphenol S	increases expression	1
jinfukang	decreases expression	1
LDN 193189	affects cotreatment, increases expression	1
bisphenol AF	increases expression	1
Resveratrol	affects cotreatment, increases expression	1
Air Pollutants	decreases expression, increases abundance	1
Arsenic	increases abundance, increases expression	1
Benzo(a)pyrene	increases methylation, affects methylation	1
Cadmium	increases abundance, increases expression	1
Doxorubicin	increases expression	1
Enzyme Inhibitors	decreases activity, increases O-linked glycosylation	1
Estradiol	decreases expression	1
Homocysteine	affects cotreatment, affects expression	1
Ivermectin	decreases expression	1
Methionine	affects cotreatment, affects expression	1
Plant Extracts	affects cotreatment, increases expression	1
Rotenone	increases expression	1
Tobacco Smoke Pollution	affects expression	1
Aflatoxin M1	decreases expression	1
Cadmium Chloride	increases abundance, increases expression	1
Particulate Matter	decreases expression, increases abundance	1
Soot	increases expression	1

Cellosaurus cell lines

1 cell lines: 1 cancer cell line

First 10 cell lines (id-ordered, not curated):

Cellosaurus	Name	Category	Sex
CVCL_E0SQ	Ubigene HeLa VBP1 KO	Cancer cell line	Female

Clinical trials (associated diseases)

95 trials via MONDO — disease-level, not drug-specific.

Trial	Phase	Status	Title
NCT00517959	PHASE3	UNKNOWN	SCRT Versus Conventional RT in Children and Young Adults With Low Grade and Benign Brain Tumors
NCT01096368	PHASE3	COMPLETED	Maintenance Chemotherapy or Observation Following Induction Chemotherapy and Radiation Therapy in Treating Patients With Newly Diagnosed Ependymoma
NCT00003479	PHASE2	TERMINATED	Antineoplaston Therapy in Treating Patients With Ependymoma
NCT00520936	PHASE2	COMPLETED	A Study of Pemetrexed in Children With Recurrent Cancer
NCT00840047	PHASE2	ACTIVE_NOT_RECRUITING	Methionine PET/CT Studies In Patients With Cancer
NCT01088035	PHASE2	TERMINATED	Carboplatin as a Radiosensitizer in Treating Childhood Ependymoma
NCT01247922	PHASE2	TERMINATED	Single-agent Erlotinib in Patients Previously Treated With Oral Etoposide in Protocol OSI-774-205
NCT01288235	PHASE2	COMPLETED	Proton Radiotherapy for Pediatric Brain Tumors Requiring Partial Brain Irradiation
NCT01295944	PHASE2	COMPLETED	Carboplatin and Bevacizumab for Recurrent Ependymoma
NCT01356290	PHASE2	RECRUITING	Antiangiogenic Therapy for Children With Recurrent Medulloblastoma, Ependymoma, ATRT and Rare CNS Tumors
NCT01836549	PHASE2	TERMINATED	Imetelstat Sodium in Treating Younger Patients With Recurrent or Refractory Brain Tumors
NCT02125786	PHASE2	ACTIVE_NOT_RECRUITING	A Trial of Surgery and Fractionated Re-Irradiation for Recurrent Ependymoma
NCT02689336	PHASE2	WITHDRAWN	Erlotinib in Combination With Temozolomide in Treating Relapsed/Recurrent/Refractory Pediatric Solid Tumors
NCT03095248	PHASE2	TERMINATED	Trial of Selumetinib in Patients With Neurofibromatosis Type II Related Tumors
NCT03155620	PHASE2	ACTIVE_NOT_RECRUITING	Targeted 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)
NCT03173950	PHASE2	COMPLETED	Immune Checkpoint Inhibitor Nivolumab in People With Recurrent Select Rare CNS Cancers
NCT03194906	PHASE2	COMPLETED	Memantine for Prevention of Cognitive Late Effects in Pediatric Patients Receiving Cranial Radiation Therapy for Localized Brain Tumors
NCT03210714	PHASE2	ACTIVE_NOT_RECRUITING	Erdafitinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With FGFR Mutations (A Pediatric MATCH Treatment Trial)
NCT03213652	PHASE2	ACTIVE_NOT_RECRUITING	Ensartinib 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)
NCT03213665	PHASE2	COMPLETED	Tazemetostat 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)
NCT03213678	PHASE2	COMPLETED	Samotolisib 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)
NCT03213704	PHASE2	ACTIVE_NOT_RECRUITING	Larotrectinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With NTRK Fusions (A Pediatric MATCH Treatment Trial)
NCT03220035	PHASE2	COMPLETED	Vemurafenib 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)
NCT03233204	PHASE2	COMPLETED	Olaparib 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)
NCT03526250	PHASE2	COMPLETED	Palbociclib 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)
NCT03698994	PHASE2	ACTIVE_NOT_RECRUITING	Ulixertinib in Treating Patients With Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With MAPK Pathway Mutations (A Pediatric MATCH Treatment Trial)
NCT03727841	PHASE2	TERMINATED	Marizomib for Recurrent Low-Grade and Anaplastic Supratentorial, Infratentorial, and Spinal Cord Ependymoma
NCT04049669	PHASE2	ACTIVE_NOT_RECRUITING	Pediatric Trial of Indoximod With Chemotherapy and Radiation for Relapsed Brain Tumors or Newly Diagnosed DIPG
NCT04195555	PHASE2	ACTIVE_NOT_RECRUITING	Ivosidenib in Treating Patients With Advanced Solid Tumors, Lymphoma, or Histiocytic Disorders With IDH1 Mutations (A Pediatric MATCH Treatment Trial)
NCT04284774	PHASE2	ACTIVE_NOT_RECRUITING	Tipifarnib for the Treatment of Advanced Solid Tumors, Lymphoma, or Histiocytic Disorders With HRAS Gene Alterations, a Pediatric MATCH Treatment Trial
NCT04320888	PHASE2	ACTIVE_NOT_RECRUITING	Selpercatinib for the Treatment of Advanced Solid Tumors, Lymphomas, or Histiocytic Disorders With Activating RET Gene Alterations, a Pediatric MATCH Treatment Trial
NCT04374305	PHASE2	RECRUITING	Innovative Trial for Understanding the Impact of Targeted Therapies in NF2-Related Schwannomatosis (INTUITT-NF2)
NCT04743661	PHASE2	ACTIVE_NOT_RECRUITING	131I-Omburtamab, in Recurrent Medulloblastoma and Ependymoma
NCT06804655	PHASE2	NOT_YET_RECRUITING	Pharmacoscopy for Patients With Refractory Primary Brain Tumors
NCT07424092	PHASE2	RECRUITING	Intratumoral DNX-2401 for High Grade Pediatric Brain Tumors
NCT00634231	PHASE1	COMPLETED	A Phase I Study of AdV-tk + Prodrug Therapy in Combination With Radiation Therapy for Pediatric Brain Tumors
NCT00994071	PHASE1	COMPLETED	A Phase I Study of ABT-888, an Oral Inhibitor of Poly(ADP-ribose) Polymerase and Temozolomide in Children With Recurrent/Refractory CNS Tumors
NCT01171469	PHASE1	COMPLETED	Vaccination With Dendritic Cells Loaded With Brain Tumor Stem Cells for Progressive Malignant Brain Tumor
NCT01331135	PHASE1	COMPLETED	Aflac ST0901 CHOANOME - Sirolimus in Solid Tumors
NCT01498783	PHASE1	COMPLETED	Phase I Study of 5-Fluorouracil in Children and Young Adults With Recurrent Ependymoma