precursor B-cell acute lymphoblastic leukemia

Summary

precursor B-cell acute lymphoblastic leukemia (MONDO:0020511) is a cancer with 5 cohort genes (5 CIViC-evidence somatic drivers; 5 ClinVar predisposition records) and 229 clinical trials. Molecularly, BCR::PDGFRA Fusion confers sensitivity to Imatinib in B-cell Acute Lymphoblastic Leukemia (CIViC Level C); 3 further subtype–drug associations are mapped below. Top therapeutic interventions include blinatumomab, mercaptopurine anhydrous, and inotuzumab ozogamicin.

At a glance

• Classification: Cancer
• Cohort genes: 5
• ClinVar variants: 5
• Clinical trials: 229
• 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	precursor B-cell acute lymphoblastic leukemia
Mondo ID	MONDO:0020511
Orphanet	99860
DOID	DOID:0080638
ICD-11	1099674056
NCIT	C8644
UMLS	C0349636
MedGen	83896
GARD	0016920
Is cancer (heuristic)	yes

Also known as: acute B cell lymphocytic leukaemia · acute B cell lymphocytic leukemia · acute B-cell lymphocytic leukaemia · acute B-cell lymphocytic leukemia · B acute lymphoblastic leukaemia · B acute lymphoblastic leukemia · B cell acute lymphocytic leukaemia · B cell acute lymphocytic leukemia · B cell precursor type acute leukaemia · B cell precursor type acute leukemia · B-ALL · B-cell acute lymphoblastic leukaemia · B-cell acute lymphoblastic leukemia · B-cell acute lymphocytic leukaemia · B-cell acute lymphocytic leukemia · B-cell lymphoblastic leukaemia · B-cell lymphoblastic leukemia · B-cell precursor type acute leukaemia · B-cell precursor type acute leukemia · B-cell type acute leukaemia (+10 more)

Data availability: 5 ClinVar variants · 280 cell lines.

Disease family

An umbrella term covering 2 Mondo subtypes.

Classification path: human disease › disease by etiologic mechanism › cancer or benign tumor › neoplastic disease or syndrome › neoplasm › hematopoietic and lymphoid system neoplasm › hematopoietic and lymphoid cell neoplasm › lymphoid neoplasm › precursor lymphoblastic lymphoma/leukemia › acute lymphoblastic leukemia › precursor B-cell acute lymphoblastic leukemia

Related subtypes (11): childhood acute lymphoblastic leukemia, prolymphocytic leukemia, adult acute lymphoblastic leukemia, null-cell leukemia, B-cell acute lymphoblastic leukemia, B-cell chronic lymphocytic leukemia, T-cell acute lymphoblastic leukemia, lymphoblastic leukemia, acute, with lymphomatous features, plasma cell leukemia, acute biphenotypic leukemia, precursor T-cell acute lymphoblastic leukemia

Subtypes (2): B-cell adult acute lymphocytic leukemia, B-cell childhood acute lymphoblastic leukemia

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

ClinVar germline variants

5 retrieved; paginated sample, class counts are floors:

2 pathogenic, 1 conflicting classifications of pathogenicity, 1 pathogenic/likely pathogenic, 1 likely pathogenic

ClinVar	Variant (HGVS)	Gene	Classification	Review
55823	NM_005236.3(ERCC4):c.1484_1488del (p.Thr495fs)	ERCC4	Pathogenic	criteria provided, multiple submitters, no conflicts
426988	NM_001042492.3(NF1):c.6921+3A>G	NF1	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
128121	NM_024675.4(PALB2):c.1675_1676delinsTG	PALB2	Pathogenic	criteria provided, multiple submitters, no conflicts
869413	t(3;9)(q13.31;p24.1)	JAK2	Likely pathogenic	criteria provided, single submitter
265295	NM_024426.6(WT1):c.151del (p.Ala51fs)	WT1	Conflicting classifications of pathogenicity	criteria provided, conflicting classifications

Genes & proteins

Mendelian disease overlap and somatic drivers

GenCC: 0 · Orphanet: 33 · OMIM-shared: 0 · Dual-evidence (GWAS+Mendelian): 0

Somatic driver evidence (intOGen + CIViC, cohort fanout)

Gene	intOGen role	Cancer types	CIViC
WT1	LoF	AML,MEL,PAAD	CIViC #49
PALB2	LoF	OVT	CIViC #15013
ERCC4			CIViC #1740
JAK2	Act	ALL,AML,BLADDER,BRCA,NSCLC	CIViC #28
NF1	LoF	ACC,ALL,AML,ANGS,BLCA,BRCA,CCRCC,CHOL,CLLSLL,COADREAD,GB,GBM,GIST,HCC,HNSC,LGGNOS,LMS,LUAD,LUNG,LUSC,MEL,NBL,NSCLC,OVT,PAST,PGNG,PLMESO,RMS,SKCM,SOFT_TISSUE,STAD,THYM,UCS	CIViC #3867

Orphanet rare-disease linkage (cohort genes)

Gene	Orphanet ID	Rare disease
WT1	Orphanet:220	Denys-Drash syndrome
WT1	Orphanet:242	46,XY complete gonadal dysgenesis
WT1	Orphanet:251510	46,XY partial gonadal dysgenesis
WT1	Orphanet:3097	Meacham syndrome
WT1	Orphanet:347	Frasier syndrome
WT1	Orphanet:654	Nephroblastoma
WT1	Orphanet:656	Hereditary steroid-resistant nephrotic syndrome
WT1	Orphanet:83469	Desmoplastic small round cell tumor
WT1	Orphanet:893	WAGR syndrome
PALB2	Orphanet:1333	Familial pancreatic carcinoma
PALB2	Orphanet:145	Hereditary breast and/or ovarian cancer syndrome
PALB2	Orphanet:178	Chordoma
PALB2	Orphanet:227535	Hereditary breast cancer
PALB2	Orphanet:84	Fanconi anemia
ERCC4	Orphanet:220295	Xeroderma pigmentosum-Cockayne syndrome complex
ERCC4	Orphanet:84	Fanconi anemia
ERCC4	Orphanet:90321	Cockayne syndrome type 1
ERCC4	Orphanet:910	Xeroderma pigmentosum
JAK2	Orphanet:131	Budd-Chiari syndrome
JAK2	Orphanet:3318	Essential thrombocythemia
JAK2	Orphanet:667662	Breast implant-associated anaplastic large cell lymphoma
JAK2	Orphanet:71493	Familial thrombocytosis
JAK2	Orphanet:729	Polycythemia vera
JAK2	Orphanet:824	Primary myelofibrosis
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

5 cohort genes, 5 distinct canonical proteins.

Evidence partition

Subset	Genes
multi_evidence	5

Cohort genes (full)

Symbol	HGNC	Ensembl	UniProt	Name	Evidence
WT1	HGNC:12796	ENSG00000184937	P19544	Wilms tumor protein	clinvar
PALB2	HGNC:26144	ENSG00000083093	Q86YC2	Partner and localizer of BRCA2	clinvar
ERCC4	HGNC:3436	ENSG00000175595	Q92889	DNA repair endonuclease XPF	clinvar
JAK2	HGNC:6192	ENSG00000096968	O60674	Tyrosine-protein kinase JAK2	clinvar
NF1	HGNC:7765	ENSG00000196712	P21359	Neurofibromin	clinvar

Cohort function summary

Lead sentence per gene, UniProt-curated.

Symbol	Protein name	Function (lead sentence)
WT1	Wilms tumor protein	Transcription factor that plays an important role in cellular development and cell survival.
PALB2	Partner and localizer of BRCA2	Plays a critical role in homologous recombination repair (HRR) through its ability to recruit BRCA2 and RAD51 to DNA breaks.
ERCC4	DNA repair endonuclease XPF	Catalytic component of a structure-specific DNA repair endonuclease responsible for the 5-prime incision during DNA repair, and which is essential for nucleotide excision repair (NER) and interstrand cross-link (ICL) repair.
JAK2	Tyrosine-protein kinase JAK2	Non-receptor tyrosine kinase involved in various processes such as cell growth, development, differentiation or histone modifications.
NF1	Neurofibromin	Stimulates the GTPase activity of Ras.

Protein-family classification

Druggable: 1 · Difficult: 2 · Unknown: 2 · Druggable fraction: 0.2

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	5.5×	0.515
Scaffold/PPI	1	3.5×	0.515
Transcription factor	1	1.6×	0.634
Other/Unknown	2	0.7×	0.877

Per-gene assignment

Symbol	Family	Druggable?	EC	InterPro (top 3)
WT1	Transcription factor	no		Wilms_tumour_N, Znf_C2H2_type, Znf_C2H2_sf
PALB2	Scaffold/PPI	no		WD40/YVTN_repeat-like_dom_sf, PALB2_WD40, WD40_repeat_dom_sf
ERCC4	Other/Unknown	no		ERCC4_domain, XPF, RuvA_2-like
JAK2	Kinase	yes	2.7.10.2	FERM_domain, Prot_kinase_dom, SH2
NF1	Other/Unknown	no		CRAL-TRIO_dom, RasGAP_dom, Rho_GTPase_activation_prot

Expression context

Cohort genes with no expression data: 0.

4 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)	5
unknown	0

Top tissues across cohort

Tissue	Cohort genes
adrenal tissue	2
calcaneal tendon	2
germinal epithelium of ovary	1
metanephric glomerulus	1
renal glomerulus	1
buccal mucosa cell	1
oocyte	1
secondary oocyte	1
male germ line stem cell (sensu Vertebrata) in testis	1
sperm	1
blood vessel layer	1
monocyte	1
colonic epithelium	1

Per-gene tissue summary (top 30)

Symbol	Bgee breadth	FANTOM5 breadth	SCXA	Top tissues
WT1	168	broad	marker	germinal epithelium of ovary, renal glomerulus, metanephric glomerulus
PALB2	232	ubiquitous	yes	secondary oocyte, buccal mucosa cell, oocyte
ERCC4	242	ubiquitous	marker	male germ line stem cell (sensu Vertebrata) in testis, adrenal tissue, sperm
JAK2	272	ubiquitous	marker	calcaneal tendon, monocyte, blood vessel layer
NF1	283	ubiquitous	marker	colonic epithelium, calcaneal tendon, adrenal tissue

Protein interactions among cohort

Intra-cohort edges: 0.

Hub genes (top 10 by interactor count)

Symbol	Interactor count
JAK2	6,197
PALB2	5,641
NF1	5,540
WT1	3,938
ERCC4	2,102

Structural data

PDB: 5 · AlphaFold-only: 0 · No structure: 0

Cohort genes with PDB structures (top 30)

Symbol	UniProt	PDB entries
JAK2	O60674	164
WT1	P19544	28
NF1	P21359	26
ERCC4	Q92889	13
PALB2	Q86YC2	4

Function

Pathway analysis

Distinct Reactome pathways touched by cohort: 86. Enrichment computed across 5 evidence-associated genes (5 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 5 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	99.3×	0.014	JAK2, NF1
RAS signaling downstream of NF1 loss-of-function variants	1	326.3×	0.023	NF1
Erythropoietin activates Phospholipase C gamma (PLCG)	1	326.3×	0.023	JAK2
Erythropoietin activates STAT5	1	326.3×	0.023	JAK2
Interleukin-6 family signaling	1	285.5×	0.023	JAK2
IFNG signaling activates MAPKs	1	285.5×	0.023	JAK2
Interleukin-23 signaling	1	253.8×	0.023	JAK2
MAPK1 (ERK2) activation	1	228.4×	0.023	JAK2
Signaling by KIT in disease	1	228.4×	0.023	JAK2
MAPK3 (ERK1) activation	1	207.6×	0.023	JAK2
Signaling by Leptin	1	207.6×	0.023	JAK2
Signaling by Erythropoietin	1	207.6×	0.023	JAK2
Interleukin-27 signaling	1	207.6×	0.023	JAK2
Interleukin-6 signaling	1	190.3×	0.023	JAK2
Interleukin-35 Signalling	1	190.3×	0.023	JAK2
Erythropoietin activates Phosphoinositide-3-kinase (PI3K)	1	190.3×	0.023	JAK2
MAPK1/MAPK3 signaling	2	52.5×	0.023	JAK2, NF1
MAPK family signaling cascades	2	41.1×	0.023	JAK2, NF1
RAF/MAP kinase cascade	2	24.4×	0.023	JAK2, NF1
Diseases of signal transduction by growth factor receptors and second messengers	2	22.7×	0.023	JAK2, NF1
Nephron development	1	175.7×	0.023	WT1
Regulation of IFNG signaling	1	163.1×	0.023	JAK2
Prolactin receptor signaling	1	152.3×	0.023	JAK2
Erythropoietin activates RAS	1	152.3×	0.023	JAK2
Transcriptional regulation of testis differentiation	1	142.8×	0.024	WT1
RAF-independent MAPK1/3 activation	1	126.9×	0.024	JAK2
Interleukin-2 family signaling	1	126.9×	0.024	JAK2
IL-6-type cytokine receptor ligand interactions	1	126.9×	0.024	JAK2
Signaling by CSF3 (G-CSF)	1	114.2×	0.025	JAK2
Signaling by phosphorylated juxtamembrane, extracellular and kinase domain KIT mutants	1	103.8×	0.025	JAK2

GO biological processes by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 5 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

GO term	Cohort genes	Fold	FDR	Sample cohort genes
adrenal gland development	2	269.6×	0.004	WT1, NF1
mesoderm development	2	210.7×	0.004	PALB2, JAK2
positive regulation of vascular associated smooth muscle cell proliferation	2	172.8×	0.004	JAK2, NF1
nuclear receptor-mediated mineralocorticoid signaling pathway	1	3370.4×	0.007	JAK2
positive regulation of mast cell apoptotic process	1	3370.4×	0.007	NF1
symbiont-induced defense-related programmed cell death	1	3370.4×	0.007	JAK2
regulation of glial cell differentiation	1	3370.4×	0.007	NF1
interleukin-35-mediated signaling pathway	1	3370.4×	0.007	JAK2
negative regulation of metanephric glomerular mesangial cell proliferation	1	3370.4×	0.007	WT1
observational learning	1	3370.4×	0.007	NF1
regulation of animal organ formation	1	1685.2×	0.007	WT1
adrenal cortex formation	1	1685.2×	0.007	WT1
visceral serous pericardium development	1	1685.2×	0.007	WT1
gamma-aminobutyric acid secretion, neurotransmission	1	1685.2×	0.007	NF1
response to interleukin-12	1	1685.2×	0.007	JAK2
posterior mesonephric tubule development	1	1685.2×	0.007	WT1
positive regulation of growth factor dependent skeletal muscle satellite cell proliferation	1	1685.2×	0.007	JAK2
regulation of postsynapse to nucleus signaling pathway	1	1685.2×	0.007	JAK2
positive regulation of metanephric ureteric bud development	1	1685.2×	0.007	WT1
Schwann cell proliferation	1	1123.5×	0.007	NF1
forebrain astrocyte development	1	1123.5×	0.007	NF1
Schwann cell migration	1	1123.5×	0.007	NF1
positive regulation of growth hormone receptor signaling pathway	1	1123.5×	0.007	JAK2
glutamate secretion, neurotransmission	1	1123.5×	0.007	NF1
negative regulation of mast cell proliferation	1	1123.5×	0.007	NF1
negative regulation of Schwann cell migration	1	1123.5×	0.007	NF1
nucleotide-excision repair involved in interstrand cross-link repair	1	1123.5×	0.007	ERCC4
vascular associated smooth muscle cell migration	1	1123.5×	0.007	NF1
mast cell apoptotic process	1	842.6×	0.007	NF1
negative regulation of Rac protein signal transduction	1	842.6×	0.007	NF1

Therapeutics

Drug target analysis

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

Druggability breadth: 3 of 5 evidence-associated genes (60%) 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
JAK2	FEDRATINIB

Top cohort targets by molecule count

Symbol	Molecules	Max phase
JAK2	100	4
WT1	0	0
PALB2	0	0
ERCC4	0	0
NF1	0	0

Drugs targeting cohort genes (top 30)

Molecule	Max phase	Targets in cohort
FEDRATINIB	4	JAK2
RUXOLITINIB	4	JAK2
TOFACITINIB	4	JAK2
UPADACITINIB	4	JAK2
MOMELOTINIB	4	JAK2
PONATINIB	4	JAK2
AXITINIB	4	JAK2
NICLOSAMIDE	4	JAK2
RUXOLITINIB PHOSPHATE	4	JAK2
INFIGRATINIB PHOSPHATE	4	JAK2
INFIGRATINIB	4	JAK2
ENTRECTINIB	4	JAK2
DABRAFENIB	4	JAK2
PACRITINIB	4	JAK2
TOFACITINIB CITRATE	4	JAK2
BARICITINIB	4	JAK2
CERITINIB	4	JAK2
BOSUTINIB	4	JAK2
PEFICITINIB	4	JAK2
LORLATINIB	4	JAK2
FILGOTINIB	4	JAK2
BRIGATINIB	4	JAK2
ABROCITINIB	4	JAK2
REPOTRECTINIB	4	JAK2
DEUCRAVACITINIB	4	JAK2
PRALSETINIB	4	JAK2
CRAVACITINIB	4	JAK2
PAZOPANIB	4	JAK2
NINTEDANIB	4	JAK2
SUNITINIB	4	JAK2

Bioactivity and enzyme data

Enzyme cohort genes (≥1 EC): 1.

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

Symbol	Assays	Type breakdown
JAK2	2,018	Binding:1911, Functional:51, ADMET:48, Unclassified:4, Toxicity:4
ERCC4	28	Binding:28

Cohort enzymes (BRENDA EC)

Symbol	EC numbers	Names
JAK2	2.7.10.2	non-specific protein-tyrosine kinase

Cohort genes with high screening signal

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

Symbol	ChEMBL assays
JAK2	2,018

Pharmacogenomics

Cohort genes with a PharmGKB record: 5; 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.

Compound	Max phase	Cohort target (bioactivity)
FEDRATINIB	4	JAK2
RUXOLITINIB	4	JAK2
TOFACITINIB	4	JAK2
UPADACITINIB	4	JAK2
MOMELOTINIB	4	JAK2
PONATINIB	4	JAK2
AXITINIB	4	JAK2
NICLOSAMIDE	4	JAK2
RUXOLITINIB PHOSPHATE	4	JAK2
INFIGRATINIB PHOSPHATE	4	JAK2
INFIGRATINIB	4	JAK2
ENTRECTINIB	4	JAK2
DABRAFENIB	4	JAK2
PACRITINIB	4	JAK2
TOFACITINIB CITRATE	4	JAK2
BARICITINIB	4	JAK2
CERITINIB	4	JAK2
BOSUTINIB	4	JAK2
PEFICITINIB	4	JAK2
LORLATINIB	4	JAK2
FILGOTINIB	4	JAK2
BRIGATINIB	4	JAK2
ABROCITINIB	4	JAK2
REPOTRECTINIB	4	JAK2
DEUCRAVACITINIB	4	JAK2
PRALSETINIB	4	JAK2
CRAVACITINIB	4	JAK2
PAZOPANIB	4	JAK2
NINTEDANIB	4	JAK2
SUNITINIB	4	JAK2

Druggability pyramid

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

Tier	Definition	Genes	Symbols
A	Approved (phase 4 drug)	1	JAK2
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	4	WT1, PALB2, ERCC4, NF1

Undrugged target profiles

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

Symbol	ChEMBL assays	Drugged partners (top 3)
WT1	0	—
PALB2	0	—
ERCC4	28	—
NF1	0	—

Clinical trials & evidence

Clinical trials

Clinical trials: 229.

Phase distribution (across all retrieved trials)

Phase	Trials
PHASE1	72
PHASE2	54
PHASE1/PHASE2	45
Not specified	24
EARLY_PHASE1	16
PHASE3	11
PHASE2/PHASE3	5
PHASE4	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT06339775	PHASE4	RECRUITING	Blinatumomab for Relapsed Acute B Lymphoblastic Leukemia After Transplantation
NCT02618109	PHASE4	TERMINATED	Identification of New Immune Factors Specific of Relapse in Childhood B Lineage Acute Lymphoblastic Leukemia
NCT02101853	PHASE3	ACTIVE_NOT_RECRUITING	Blinatumomab in Treating Younger Patients With Relapsed B-cell Acute Lymphoblastic Leukemia
NCT03007147	PHASE3	ACTIVE_NOT_RECRUITING	Imatinib Mesylate and Combination Chemotherapy in Treating Patients With Newly Diagnosed Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia
NCT03150693	PHASE3	RECRUITING	Inotuzumab Ozogamicin and Frontline Chemotherapy in Treating Young Adults With Newly Diagnosed B Acute Lymphoblastic Leukemia
NCT03914625	PHASE3	ACTIVE_NOT_RECRUITING	A Study to Investigate Blinatumomab in Combination With Chemotherapy in Patients With Newly Diagnosed B-Lymphoblastic Leukemia
NCT03937544	PHASE2/PHASE3	RECRUITING	Intravenous Autologous CD19 CAR-T Cells for R/R B-ALL
NCT03959085	PHASE3	RECRUITING	Inotuzumab Ozogamicin and Post-Induction Chemotherapy in Treating Patients With High-Risk B-ALL, Mixed Phenotype Acute Leukemia, and B-LLy
NCT04094311	PHASE3	RECRUITING	Study of Out of Specification for Tisagenlecleucel
NCT05602194	PHASE3	ACTIVE_NOT_RECRUITING	Studying the Effect of Levocarnitine in Protecting the Liver From Chemotherapy for Leukemia or Lymphoma
NCT06607419	PHASE2/PHASE3	RECRUITING	Efficacy Study of Blinatumomab Clean Up Early Residual Disease for Newly Diagnosed Pediatric B Lymphoblastic Leukemia
NCT06764238	PHASE2/PHASE3	RECRUITING	Newly-diagnosed Intermediate/High Risk Pediatric B-cell ALL Protocol
NCT06882057	PHASE2/PHASE3	RECRUITING	Newly-diagnosed Low Risk Pediatric B-cell ALL Protocol
NCT07223021	PHASE3	RECRUITING	A Study of Fludarabine Dosing in Children and Young Adults With B-cell Acute Lymphoblastic Leukemia
NCT07441291	PHASE3	RECRUITING	CD19 CAR-T vs DLI for Post-HSCT MRD in Ph- ALL: A RCT
NCT07476729	PHASE3	NOT_YET_RECRUITING	International Study for Treatment of Childhood Relapsed Precursor B-Cell ALL 2020 (IntReALL BCP 2020)
NCT07570173	PHASE2/PHASE3	RECRUITING	A Clinical Trial of MK-1045 in People With B-cell Acute Lymphoblastic Leukemia (MK-1045-005)
NCT02883049	PHASE3	COMPLETED	Combination Chemotherapy in Treating Young Patients With Newly Diagnosed High-Risk B Acute Lymphoblastic Leukemia and Ph-Like TKI Sensitive Mutations
NCT01371630	PHASE1/PHASE2	RECRUITING	Inotuzumab Ozogamicin and Combination Chemotherapy in Treating Patients With Acute Lymphoblastic Leukemia
NCT02143414	PHASE2	ACTIVE_NOT_RECRUITING	Blinatumomab and Combination Chemotherapy or Dasatinib, Prednisone, and Blinatumomab in Treating Older Patients With Acute Lymphoblastic Leukemia
NCT02877303	PHASE2	RECRUITING	Blinatumomab, Inotuzumab Ozogamicin, and Combination Chemotherapy as Frontline Therapy in Treating Patients With B Acute Lymphoblastic Leukemia
NCT02981628	PHASE2	RECRUITING	Inotuzumab Ozogamicin in Treating Younger Patients With B-Lymphoblastic Lymphoma or Relapsed or Refractory CD22 Positive B Acute Lymphoblastic Leukemia
NCT03441061	PHASE2	ACTIVE_NOT_RECRUITING	Inotuzumab Ozogamicin in Treating Patients With B-cell Acute Lymphocytic Leukemia With Positive Minimal Residual Disease
NCT03504644	PHASE1/PHASE2	ACTIVE_NOT_RECRUITING	Venetoclax and Vincristine in Treating Patients With Relapsed or Refractory T-cell or B-cell Acute Lymphoblastic Leukemia
NCT03509961	PHASE2	RECRUITING	The EndRAD Trial: Eliminating Total Body Irradiation (TBI) for NGS-MRD Negative Children, Adolescents, and Young Adults With B-ALL
NCT03739814	PHASE2	RECRUITING	Inotuzumab Ozogamicin and Blinatumomab With or Without Ponatinib in Treating Patients With Newly Diagnosed, Recurrent, or Refractory CD22-Positive B-Lineage Acute Lymphoblastic Leukemia
NCT03876769	PHASE2	ACTIVE_NOT_RECRUITING	Study of Efficacy and Safety of Tisagenlecleucel in HR B-ALL EOC MRD Positive Patients
NCT04150497	PHASE1/PHASE2	RECRUITING	Phase 1/2 Study of UCART22 in Patients With Relapsed or Refractory CD22+ B-cell Acute Lymphoblastic Leukemia (BALLI-01)
NCT04499573	PHASE1/PHASE2	ACTIVE_NOT_RECRUITING	Bispecific CD19/CD22 CAR-T for Treatment of Children and Young Adults With r/r B-ALL
NCT04544592	PHASE1/PHASE2	RECRUITING	UCD19 CarT in Treatment of Pediatric B-ALL and B-NHL
NCT04546399	PHASE2	RECRUITING	A Study to Compare Blinatumomab Alone to Blinatumomab With Nivolumab in Patients Diagnosed With First Relapse B-Cell Acute Lymphoblastic Leukemia (B-ALL)
NCT04746209	PHASE2	RECRUITING	Blinatumomab After TCR Alpha Beta/CD19 Depleted HCT
NCT04781634	PHASE1/PHASE2	RECRUITING	a Clinical Research of CD19 and CD22 Targeted Prime CAR-T Cell in Relapsed/Refractory B-ALL
NCT05082519	PHASE2	RECRUITING	Caloric Restriction and Activity to Reduce Chemoresistance in B-ALL
NCT05281809	PHASE2	RECRUITING	Local Manufacture of CAR T-Cell Products for the Treatment of B-Cell Lymphoma and B-Acute Lymphoblastic Leukemia
NCT05303792	PHASE2	ACTIVE_NOT_RECRUITING	Testing the Combination of Inotuzumab Ozogamicin and Lower Dose Chemotherapy Compared to Usual Chemotherapy for Adults With B-Cell Acute Lymphoblastic Leukemia or B-Cell Lymphoblastic Lymphoma
NCT05310591	PHASE1/PHASE2	RECRUITING	Combination of an Anti-PD1 Antibody With Tisagenlecleucel Reinfusion in Children, Adolescents and Young Adults With Acute Lymphoblastic Leukemia After Loss of Persistence
NCT05442515	PHASE1/PHASE2	RECRUITING	CD19/CD22 Bicistronic Chimeric Antigen Receptor (CAR) T Cells in Children and Young Adults With Recurrent or Refractory B Cell Malignancies
NCT05460533	PHASE2	ACTIVE_NOT_RECRUITING	A Second Infusion (Early Reinfusion) of Tisagenlecleucel in Children and Young Adults With B-Cell Acute Lymphoblastic Leukemia(B-ALL)
NCT05470777	PHASE2	ACTIVE_NOT_RECRUITING	CD22/CD19 CAR-T and Auto-HSCT Sandwich Strategy as Consolidation Therapy for B-ALL

Drugs tested across these trials (top 30)

Molecule	Max phase	Trials referencing
BLINATUMOMAB	4	38
MERCAPTOPURINE ANHYDROUS	4	18
INOTUZUMAB OZOGAMICIN	4	15
PEGASPARGASE	4	10
THIOGUANINE	4	9
TISAGENLECLEUCEL	4	8
CALASPARGASE PEGOL	4	7
DAUNORUBICIN	4	6
ASPARAGINASE ERWINIA CHRYSANTHEMI	4	4
HYDROCORTISONE SODIUM SUCCINATE	4	4
ACETAMINOPHEN	4	3
ASPARAGINASE	4	3
DEXRAZOXANE	4	3
IMATINIB	4	3
MITOXANTRONE	4	3
DASATINIB ANHYDROUS	4	2
LEVOLEUCOVORIN	4	2
VINCRISTINE	4	2
ACALABRUTINIB	4	1
ALLOPURINOL	4	1
ASCIMINIB	4	1
BREXUCABTAGENE AUTOLEUCEL	4	1
CLOFARABINE	4	1
DIPHENHYDRAMINE	4	1
ETOPOSIDE PHOSPHATE	4	1
FLUDARABINE PHOSPHATE	4	1
FORODESINE	4	1
HYDROCORTISONE	4	1
IFOSFAMIDE	4	1
INTERFERON BETA-1A	4	1

Precision-medicine subtype map (CIViC)

Drug × molecular subtype: 4 predictive associations from 4 curated evidence items; also 2 oncogenic, 1 diagnostic, 1 prognostic.

Molecular subtype	Therapy	Effect	Level	CIViC
BCR::PDGFRA Fusion	Imatinib	Sensitivity/Response	CIViC C	EID7990
BLK Expression	Ibrutinib	Sensitivity/Response	CIViC D	EID9288
BTK Expression	Ibrutinib	Sensitivity/Response	CIViC D	EID9287
EZH2 A692V	GSK126	Sensitivity/Response	CIViC D	EID11707