Childhood malignant schwannoma

Summary

Childhood malignant schwannoma (MONDO:0004345) is a disease with 3 cohort genes and 42 clinical trials. Molecularly, NF1 Loss confers sensitivity to Everolimus + Bevacizumab in Malignant Peripheral Nerve Sheath Tumor (CIViC Level B); 3 further subtype–drug associations are mapped below. Top therapeutic interventions include ifosfamide, pazopanib, and dexrazoxane.

At a glance

• Cohort genes: 3
• Clinical trials: 42
• Precision-medicine evidence (CIViC): 4 subtype–drug associations

Clinical features

No curated clinical features (Orphanet) for this disease.

Identifiers

Disease identifiers

Field	Value
Canonical name	childhood malignant schwannoma
Mondo ID	MONDO:0004345
DOID	DOID:7732
NCIT	C8094
UMLS	C0279987
MedGen	83582
GARD	0023948
Is cancer (heuristic)	no

Also known as: childhood malignant neoplasm of peripheral nerve sheath · childhood malignant neoplasm of the peripheral nerve sheath · childhood malignant neurilemmoma · childhood malignant peripheral nerve sheath neoplasm · childhood malignant peripheral nerve sheath tumor · childhood malignant peripheral nerve sheath tumour · childhood malignant schwannoma · childhood malignant tumor of peripheral nerve sheath · childhood malignant tumor of the peripheral nerve sheath · childhood malignant tumour of peripheral nerve sheath · childhood malignant tumour of the peripheral nerve sheath · childhood MPNST · childhood neurofibrosarcoma · childhood neurogenic sarcoma · malignant peripheral nerve sheath tumor · malignant peripheral nerve sheath tumour · paediatric malignant neoplasm of peripheral nerve sheath · paediatric malignant neoplasm of the peripheral nerve sheath · paediatric malignant neurilemmoma · paediatric malignant peripheral nerve sheath neoplasm (+14 more)

Data availability: 42 cell lines.

Disease family

Classification path: disease › human disease › disease by etiologic mechanism › cancer or benign tumor › neoplastic disease or syndrome › neoplasm › cancer › childhood malignant neoplasm › childhood malignant schwannoma

Related subtypes (29): childhood oligodendroglioma, pediatric osteosarcoma, pediatric fibrosarcoma, childhood choroid plexus carcinoma, childhood central nervous system primitive neuroectodermal neoplasm, childhood brain stem neoplasm, pediatric angiosarcoma, pediatric mesenchymal chondrosarcoma, pediatric liposarcoma, pediatric lymphoma, childhood malignant mesenchymoma, pediatric myxoid chondrosarcoma, childhood botryoid rhabdomyosarcoma, pediatric intraocular retinoblastoma, childhood cerebral astrocytoma, childhood epithelioid sarcoma, childhood pleomorphic rhabdomyosarcoma, pediatric infratentorial ependymoma, pediatric supratentorial ependymoma, pediatric extraocular retinoblastoma, childhood leukemia, childhood precursor T-lymphoblastic lymphoma/leukemia, malignant childhood germ cell neoplasm, pleuropulmonary blastoma, pediatric hepatocellular carcinoma, childhood malignant kidney neoplasm, childhood malignant melanoma, extrarenal rhabdoid tumor, pediatric high-grade glioma

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: 0 · Orphanet: 20 · OMIM-shared: 0 · Dual-evidence (GWAS+Mendelian): 0

Orphanet rare-disease linkage (cohort genes)

Gene	Orphanet ID	Rare disease
BRAF	Orphanet:1340	Cardiofaciocutaneous syndrome
BRAF	Orphanet:146	Differentiated thyroid carcinoma
BRAF	Orphanet:251615	Pilomyxoid astrocytoma
BRAF	Orphanet:389	Langerhans cell histiocytosis
BRAF	Orphanet:500	Noonan syndrome with multiple lentigines
BRAF	Orphanet:54595	Craniopharyngioma
BRAF	Orphanet:58017	Classic hairy cell leukemia
BRAF	Orphanet:626	Large/giant congenital melanocytic nevus
BRAF	Orphanet:648	Noonan syndrome
BRAF	Orphanet:840	Syringocystadenoma papilliferum
BRAF	Orphanet:96253	Cushing disease
NF1	Orphanet:139474	17q11.2 microduplication syndrome
NF1	Orphanet:29072	Hereditary pheochromocytoma-paraganglioma
NF1	Orphanet:293199	Pleomorphic rhabdomyosarcoma
NF1	Orphanet:363700	Neurofibromatosis type 1 due to NF1 mutation or intragenic deletion
NF1	Orphanet:638	Neurofibromatosis-Noonan syndrome
NF1	Orphanet:86834	Juvenile myelomonocytic leukemia
NF1	Orphanet:97685	17q11 microdeletion syndrome
NF1	Orphanet:99756	Alveolar rhabdomyosarcoma
NF1	Orphanet:99757	Embryonal rhabdomyosarcoma

Cohort genes → proteins

3 cohort genes, 3 distinct canonical proteins.

Evidence partition

Subset	Genes
civic_only	3

Cohort genes (full)

Symbol	HGNC	Ensembl	UniProt	Name	Evidence
BRAF	HGNC:1097	ENSG00000157764	P15056	Serine/threonine-protein kinase B-raf	civic_evidence
PARP1	HGNC:270	ENSG00000143799	P09874	Poly [ADP-ribose] polymerase 1	civic_evidence
NF1	HGNC:7765	ENSG00000196712	P21359	Neurofibromin	civic_evidence

Cohort function summary

Lead sentence per gene, UniProt-curated.

Symbol	Protein name	Function (lead sentence)
BRAF	Serine/threonine-protein kinase B-raf	Protein kinase involved in the transduction of mitogenic signals from the cell membrane to the nucleus.
PARP1	Poly [ADP-ribose] polymerase 1	Poly-ADP-ribosyltransferase that mediates poly-ADP-ribosylation of proteins and plays a key role in DNA repair.
NF1	Neurofibromin	Stimulates the GTPase activity of Ras.

Protein-family classification

Druggable: 1 · Difficult: 1 · Unknown: 1 · Druggable fraction: 0.33

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.

Family	Genes	Fold	FDR
Kinase	1	9.2×	0.313
Transcription factor	1	2.8×	0.482
Other/Unknown	1	0.6×	0.914

Per-gene assignment

Symbol	Family	Druggable?	EC	InterPro (top 3)
BRAF	Kinase	yes	2.7.10.2	Prot_kinase_dom, Ser-Thr/Tyr_kinase_cat_dom, PKC_DAG/PE
PARP1	Transcription factor	no	2.4.2.30	BRCT_dom, Znf_PARP, Poly(ADP-ribose)pol_reg_dom
NF1	Other/Unknown	no		CRAL-TRIO_dom, RasGAP_dom, Rho_GTPase_activation_prot

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)

Bucket	Genes
narrow (1-5 tissues)	0
moderate (6-20)	0
broad (>20)	3
unknown	0

Top tissues across cohort

Tissue	Cohort genes
calcaneal tendon	2
colonic epithelium	2
buccal mucosa cell	1
ganglionic eminence	1
primordial germ cell in gonad	1
ventricular zone	1
adrenal tissue	1

Per-gene tissue summary (top 30)

Symbol	Bgee breadth	FANTOM5 breadth	SCXA	Top tissues
BRAF	265	ubiquitous	marker	buccal mucosa cell, colonic epithelium, calcaneal tendon
PARP1	292	ubiquitous	marker	ventricular zone, ganglionic eminence, primordial germ cell in gonad
NF1	283	ubiquitous	marker	colonic epithelium, calcaneal tendon, adrenal tissue

Protein interactions among cohort

Intra-cohort edges: 1.

Hub genes (top 10 by interactor count)

Symbol	Interactor count
PARP1	8,370
BRAF	7,394
NF1	5,540

Intra-cohort edges

A	B	Sources
BRAF	NF1	string_interaction

Structural data

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

Cohort genes with PDB structures (top 30)

Symbol	UniProt	PDB entries
BRAF	P15056	131
PARP1	P09874	106
NF1	P21359	26

Function

Pathway analysis

Distinct Reactome pathways touched by cohort: 49. Enrichment computed across 3 evidence-associated genes (3 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 3 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

Pathway	Cohort genes	Fold	FDR	Sample cohort genes
Oncogenic MAPK signaling	2	165.5×	0.002	BRAF, NF1
vRNA Synthesis	1	3806.7×	0.003	PARP1
MAPK1/MAPK3 signaling	2	87.5×	0.003	BRAF, NF1
MAPK family signaling cascades	2	68.6×	0.003	BRAF, NF1
Signaling by MRAS-complex mutants	1	951.7×	0.007	BRAF
Signalling to p38 via RIT and RIN	1	761.3×	0.007	BRAF
Negative feedback regulation of MAPK pathway	1	634.4×	0.007	BRAF
ARMS-mediated activation	1	543.8×	0.007	BRAF
RAS signaling downstream of NF1 loss-of-function variants	1	543.8×	0.007	NF1
Prolonged ERK activation events	1	475.8×	0.007	BRAF
SHOC2 M1731 mutant abolishes MRAS complex function	1	475.8×	0.007	BRAF
Gain-of-function MRAS complexes activate RAF signaling	1	475.8×	0.007	BRAF
RAF/MAP kinase cascade	2	40.7×	0.007	BRAF, NF1
Diseases of signal transduction by growth factor receptors and second messengers	2	37.9×	0.007	BRAF, NF1
POLB-Dependent Long Patch Base Excision Repair	1	423.0×	0.008	PARP1
Signaling by FGFR3	1	380.7×	0.008	BRAF
Signaling by FGFR4	1	346.1×	0.008	BRAF
Frs2-mediated activation	1	317.2×	0.009	BRAF
HDR through MMEJ (alt-NHEJ)	1	292.8×	0.009	PARP1
Signaling by FGFR1	1	271.9×	0.009	BRAF
Spry regulation of FGF signaling	1	237.9×	0.010	BRAF
Signalling to ERKs	1	200.3×	0.011	BRAF
Negative regulation of FGFR3 signaling	1	146.4×	0.014	BRAF
Signaling by RAS mutants	1	141.0×	0.014	BRAF
Negative regulation of FGFR4 signaling	1	135.9×	0.014	BRAF
Signaling by FGFR2	1	135.9×	0.014	BRAF
Downregulation of SMAD2/3:SMAD4 transcriptional activity	1	122.8×	0.014	PARP1
Negative regulation of FGFR1 signaling	1	122.8×	0.014	BRAF
Negative regulation of FGFR2 signaling	1	122.8×	0.014	BRAF
Signaling by FGFR	1	115.3×	0.014	BRAF

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 term	Cohort genes	Fold	FDR	Sample cohort genes
visual learning	2	204.3×	0.004	BRAF, NF1
long-term synaptic potentiation	2	187.2×	0.004	BRAF, NF1
positive regulation of mast cell apoptotic process	1	5617.3×	0.005	NF1
regulation of glial cell differentiation	1	5617.3×	0.005	NF1
observational learning	1	5617.3×	0.005	NF1
positive regulation of myofibroblast differentiation	1	5617.3×	0.005	PARP1
gamma-aminobutyric acid secretion, neurotransmission	1	2808.7×	0.005	NF1
regulation of base-excision repair	1	2808.7×	0.005	PARP1
negative regulation of ATP biosynthetic process	1	2808.7×	0.005	PARP1
Schwann cell proliferation	1	1872.4×	0.005	NF1
forebrain astrocyte development	1	1872.4×	0.005	NF1
DNA ADP-ribosylation	1	1872.4×	0.005	PARP1
mitochondrial DNA metabolic process	1	1872.4×	0.005	PARP1
Schwann cell migration	1	1872.4×	0.005	NF1
CD4-positive or CD8-positive, alpha-beta T cell lineage commitment	1	1872.4×	0.005	BRAF
regulation of circadian sleep/wake cycle, non-REM sleep	1	1872.4×	0.005	PARP1
glutamate secretion, neurotransmission	1	1872.4×	0.005	NF1
negative regulation of mast cell proliferation	1	1872.4×	0.005	NF1
negative regulation of Schwann cell migration	1	1872.4×	0.005	NF1
vascular associated smooth muscle cell migration	1	1872.4×	0.005	NF1
MAPK cascade	2	102.1×	0.005	BRAF, NF1
mast cell apoptotic process	1	1404.3×	0.005	NF1
negative regulation of Rac protein signal transduction	1	1404.3×	0.005	NF1
myeloid leukocyte migration	1	1404.3×	0.005	NF1
ATP generation from poly-ADP-D-ribose	1	1404.3×	0.005	PARP1
positive regulation of axon regeneration	1	1123.5×	0.006	BRAF
mast cell proliferation	1	1123.5×	0.006	NF1
replication fork reversal	1	1123.5×	0.006	PARP1
regulation of oxidative stress-induced neuron intrinsic apoptotic signaling pathway	1	1123.5×	0.006	PARP1
negative regulation of synaptic vesicle exocytosis	1	1123.5×	0.006	BRAF

Therapeutics

Drug target analysis

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

Druggability breadth: 2 of 3 evidence-associated genes (67%) 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.

Symbol	Example approved molecule
BRAF	VEMURAFENIB
PARP1	NIRAPARIB

Top cohort targets by molecule count

Symbol	Molecules	Max phase
BRAF	48	4
PARP1	24	4
NF1	0	0

Drugs targeting cohort genes (top 30)

Molecule	Max phase	Targets in cohort
VEMURAFENIB	4	BRAF
PONATINIB	4	BRAF
FEDRATINIB	4	BRAF
SORAFENIB	4	BRAF
DASATINIB ANHYDROUS	4	BRAF
RUXOLITINIB	4	BRAF
INFIGRATINIB PHOSPHATE	4	BRAF
INFIGRATINIB	4	BRAF
REGORAFENIB	4	BRAF
DABRAFENIB	4	BRAF
COBIMETINIB	4	BRAF
NILOTINIB	4	BRAF
ABEMACICLIB	4	BRAF
ENCORAFENIB	4	BRAF
TOVORAFENIB	4	BRAF
PAZOPANIB	4	BRAF
DASATINIB	4	BRAF
ERLOTINIB	4	BRAF
GEFITINIB	4	BRAF
IMATINIB	4	BRAF
NIRAPARIB	4	PARP1
RUCAPARIB	4	PARP1
PALBOCICLIB	4	PARP1
TALAZOPARIB	4	PARP1
RUCAPARIB CAMSYLATE	4	PARP1
OLAPARIB	4	PARP1
AMITRIPTYLINE	4	PARP1
MASITINIB	3	BRAF
AVUTOMETINIB	3	BRAF
NAPORAFENIB	3	BRAF

Bioactivity and enzyme data

Enzyme cohort genes (≥1 EC): 2.

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

Symbol	Assays	Type breakdown
BRAF	1,442	Binding:1400, Functional:37, ADMET:5
PARP1	825	Binding:814, Functional:8, ADMET:3

Cohort enzymes (BRENDA EC)

Symbol	EC numbers	Names
BRAF	2.7.10.2, 2.7.11.1	non-specific protein-tyrosine kinase, non-specific serine/threonine protein kinase
PARP1	2.4.2.30	NAD+ ADP-ribosyltransferase

Cohort genes with high screening signal

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

Symbol	ChEMBL assays
BRAF	1,442
PARP1	825

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).

Chemical tractability of cohort targets

27 approved/phased compounds have measured bioactivity against a cohort gene (and aren’t yet in disease-level trials). This is a research / tractability signal, NOT a therapeutic recommendation — a bioactivity row often reflects off-target or screening binding (e.g. promiscuous kinase inhibitors against a cohort kinase), implying no disease mechanism.

Compound	Max phase	Cohort target (bioactivity)
VEMURAFENIB	4	BRAF
PONATINIB	4	BRAF
FEDRATINIB	4	BRAF
SORAFENIB	4	BRAF
RUXOLITINIB	4	BRAF
INFIGRATINIB PHOSPHATE	4	BRAF
INFIGRATINIB	4	BRAF
REGORAFENIB	4	BRAF
DABRAFENIB	4	BRAF
COBIMETINIB	4	BRAF
NILOTINIB	4	BRAF
ABEMACICLIB	4	BRAF
ENCORAFENIB	4	BRAF
TOVORAFENIB	4	BRAF
DASATINIB	4	BRAF
ERLOTINIB	4	BRAF
GEFITINIB	4	BRAF
NIRAPARIB	4	PARP1
RUCAPARIB	4	PARP1
PALBOCICLIB	4	PARP1
TALAZOPARIB	4	PARP1
RUCAPARIB CAMSYLATE	4	PARP1
OLAPARIB	4	PARP1
AMITRIPTYLINE	4	PARP1
MASITINIB	3	BRAF
AVUTOMETINIB	3	BRAF
NAPORAFENIB	3	BRAF

Druggability pyramid

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

Tier	Definition	Genes	Symbols
A	Approved (phase 4 drug)	2	BRAF, PARP1
B	Phased (≥1) drug, not yet approved	0
C	Druggable family + PDB, no drug	0
D	Druggable family + AlphaFold only, no drug	0
E	Difficult family or no structure, no drug	1	NF1

Undrugged target profiles

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

Symbol	ChEMBL assays	Drugged partners (top 3)
NF1	0	BRAF

Clinical trials & evidence

Clinical trials

Clinical trials: 42.

Phase distribution (across all retrieved trials)

Phase	Trials
PHASE2	16
PHASE1	12
PHASE1/PHASE2	6
Not specified	5
PHASE2/PHASE3	2
PHASE3	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT02180867	PHASE2/PHASE3	ACTIVE_NOT_RECRUITING	Radiation Therapy With or Without Combination Chemotherapy or Pazopanib Before Surgery in Treating Patients With Newly Diagnosed Non-rhabdomyosarcoma Soft Tissue Sarcomas That Can Be Removed by Surgery
NCT00346164	PHASE3	COMPLETED	Observation, Radiation Therapy, Combination Chemotherapy, and/or Surgery in Treating Young Patients With Soft Tissue Sarcoma
NCT00427583	PHASE2/PHASE3	TERMINATED	Imatinib Mesylate Treatment of Patients With Malignant Peripheral Nerve Sheath Tumors
NCT02834013	PHASE2	ACTIVE_NOT_RECRUITING	Nivolumab and Ipilimumab in Treating Patients With Rare Tumors
NCT05642455	PHASE1/PHASE2	RECRUITING	SPEARHEAD-3 Pediatric Study
NCT05985161	PHASE2	RECRUITING	A Study of Selinexor in People With Wilms Tumors and Other Solid Tumors
NCT06277154	PHASE2	RECRUITING	MASCT-I Combined With Doxorubicin and Ifosfamide for First-line Treatment of Advanced Soft Tissue Sarcoma
NCT06735820	PHASE1/PHASE2	NOT_YET_RECRUITING	Early Phase Study Evaluating MEK and MDM2 Inhibition in Patients With NF1 and MPNST
NCT06849986	PHASE2	RECRUITING	IO Combined With AI as First-line Treatment for Patients With Soft Tissue Sarcoma(TAIS)
NCT07549022	PHASE1/PHASE2	NOT_YET_RECRUITING	Safety and Efficacy of BMS-986504 in Unresectable Malignant Peripheral Nerve Sheath Tumor
NCT00464620	PHASE2	COMPLETED	Trial of Dasatinib in Advanced Sarcomas
NCT00837148	PHASE2	COMPLETED	Sorafenib and Dacarbazine in Soft Tissue Sarcoma
NCT01418001	PHASE1/PHASE2	TERMINATED	Gemcitabine and Docetaxel in Combination With Pazopanib (Gem/Doce/Pzb) for the Neoadjuvant Treatment of Soft Tissue Sarcoma (STS)
NCT01614795	PHASE2	COMPLETED	Cixutumumab and Temsirolimus in Treating Younger Patients With Recurrent or Refractory Sarcoma
NCT01653028	PHASE2	COMPLETED	Alisertib in Treating Patients With Advanced or Metastatic Sarcoma
NCT01661283	PHASE2	COMPLETED	SARC016: Study of Everolimus With Bevacizumab to Treat Refractory Malignant Peripheral Nerve Sheath Tumors
NCT02452554	PHASE2	COMPLETED	Lorvotuzumab Mertansine in Treating Younger Patients With Relapsed or Refractory Wilms Tumor, Rhabdomyosarcoma, Neuroblastoma, Pleuropulmonary Blastoma, Malignant Peripheral Nerve Sheath Tumor, or Synovial Sarcoma
NCT02584309	PHASE2	COMPLETED	Doxorubicin With Upfront Dexrazoxane for the Treatment of Advanced or Metastatic Soft Tissue Sarcoma
NCT02584647	PHASE1/PHASE2	TERMINATED	PLX3397 Plus Sirolimus in Unresectable Sarcoma and Malignant Peripheral Nerve Sheath Tumors
NCT02601209	PHASE1/PHASE2	TERMINATED	Sapanisertib or Pazopanib Hydrochloride in Treating Patients With Locally Advanced or Metastatic Sarcoma
NCT02691026	PHASE2	TERMINATED	A Study of Pembrolizumab in Patients With Malignant Peripheral Nerve Sheath Tumor (MPNST), Not Eligible for Curative Surgery
NCT03433183	PHASE2	COMPLETED	SARC031: MEK Inhibitor Selumetinib (AZD6244) in Combination With the mTOR Inhibitor Sirolimus for Patients With Malignant Peripheral Nerve Sheath Tumors
NCT03651375	PHASE2	UNKNOWN	Hypofractionated Radiotherapy With Sequential Chemotherapy in Marginally Resectable Soft Tissue Sarcomas of Extremities or Trunk Wall
NCT03989596	PHASE2	UNKNOWN	Hypofractionated Radiotherapy With Hyperthermia in Unresectable or Marginally Resectable Soft Tissue Sarcomas
NCT04530487	PHASE2	TERMINATED	Donor Stem Cell Transplant After Chemotherapy for the Treatment of Recurrent or Refractory High-Risk Solid Tumors in Pediatric and Adolescent-Young Adults
NCT03618381	PHASE1	ACTIVE_NOT_RECRUITING	EGFR806 CAR T Cell Immunotherapy for Recurrent/Refractory Solid Tumors in Children and Young Adults
NCT04222413	PHASE1	RECRUITING	Metarrestin (ML-246) in Subjects With Metastatic Solid Tumors
NCT04420975	PHASE1	ACTIVE_NOT_RECRUITING	Nivolumab and BO-112 Before Surgery for the Treatment of Resectable Soft Tissue Sarcoma
NCT04483778	PHASE1	ACTIVE_NOT_RECRUITING	B7H3 CAR T Cell Immunotherapy for Recurrent/Refractory Solid Tumors in Children and Young Adults
NCT04897321	PHASE1	RECRUITING	B7-H3-Specific Chimeric Antigen Receptor Autologous T-Cell Therapy for Pediatric Patients With Solid Tumors (3CAR)
NCT05245500	PHASE1	RECRUITING	Phase 1 Study of MRTX1719 in Solid Tumors With MTAP Deletion
NCT00720174	PHASE1	COMPLETED	Cixutumumab and Doxorubicin Hydrochloride in Treating Patients With Unresectable, Locally Advanced, or Metastatic Soft Tissue Sarcoma
NCT00931931	PHASE1	COMPLETED	HSV1716 in Patients With Non-Central Nervous System (Non-CNS) Solid Tumors
NCT02700230	PHASE1	COMPLETED	Vaccine Therapy in Treating Patients with Malignant Peripheral Nerve Sheath Tumor That is Recurrent or Cannot Be Removed by Surgery
NCT03009201	PHASE1	COMPLETED	Ribociclib and Doxorubicin in Treating Patients With Metastatic or Advanced Soft Tissue Sarcomas That Cannot Be Removed by Surgery
NCT03880123	PHASE1	WITHDRAWN	Selinexor in Combination With Ixazomib for the Treatment of Advanced Sarcoma
NCT04811196	PHASE1	COMPLETED	A Study of Different Dosing Schedules of Selinexor in Sarcoma Patients
NCT00924196	Not specified	ACTIVE_NOT_RECRUITING	Natural History Study of Patients With Neurofibromatosis Type I
NCT03141021	Not specified	RECRUITING	Multi-Institutional Registry for Malignant Peripheral Nerve Sheath Tumors
NCT06515860	Not specified	RECRUITING	Neurofibromatosis Type 1 Tumor Early Detection Study

Drugs tested across these trials (top 30)

Molecule	Max phase	Trials referencing
IFOSFAMIDE	4	4
PAZOPANIB	4	4
DEXRAZOXANE	4	3
IMATINIB	4	3
DASATINIB ANHYDROUS	4	2
SELUMETINIB	4	2
AFAMITRESGENE AUTOLEUCEL	4	1
DACARBAZINE	4	1
PEXIDARTINIB	4	1
RIBOCICLIB	4	1
SELINEXOR	4	1
ALISERTIB	3	1
IXAZOMIB	3	1
CIXUTUMUMAB	2	2
LORVOTUZUMAB MERTANSINE	2	1
SAPANISERTIB	2	1
ML-246	1	1
CHEMBL4463209	0	2
CHEMBL4068768	0	1
CHEMBL4171277	0	1
CHEMBL4583196	0	1
CHEMBL1574179	0	1
CHEMBL3962632	0	1
CHEMBL3991933	0	1

Precision-medicine subtype map (CIViC)

Drug × molecular subtype: 4 predictive associations from 4 curated evidence items; also 1 diagnostic, 1 oncogenic.

Molecular subtype	Therapy	Effect	Level	CIViC
NF1 Loss	Everolimus + Bevacizumab	Sensitivity/Response	CIViC B	EID7727
BRAF V600E	Vemurafenib	Sensitivity/Response	CIViC C	EID3788
NF1 Loss	JQ1 Compound	Sensitivity/Response	CIViC D	EID1743
PARP1 OVEREXPRESSION	Olaparib	Sensitivity/Response	CIViC D	EID7016