Disease · EFO_0020983

diffuse midline glioma

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. diffuse midline glioma. Diseases, record EFO_0020983. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/EFO_0020983

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Disease

Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.

Name: diffuse midline glioma; Open Targets
Open Targets ID: EFO_0020983; Open Targets
NCI Thesaurus: C182151; NCI Thesaurus
Highest stage of its drugs: Phase 3, for 4 of its 33 drugs, from ClinicalTrials.gov; Open Targets

Drugs33

The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.

Drug	ChEMBL ID	Type	Open Targets' stage	Stage reported by	Reports	Of them trials	Trials shown here	Source
DORDAVIPRONE	CHEMBL4297310	Small molecule	Phase 3	ClinicalTrials.gov	5	5	5	Drugs and targets
TEMOZOLOMIDE	CHEMBL810	Small molecule	Phase 3	ClinicalTrials.gov	5	5	5	Drugs and targets
EVEROLIMUS	CHEMBL1908360	Small molecule	Phase 3	ClinicalTrials.gov	1	1	1	Drugs and targets
VALPROIC ACID	CHEMBL109	Small molecule	Phase 3	ClinicalTrials.gov	1	1	1	Drugs and targets
PAXALISIB	CHEMBL3813842	Small molecule	Phase 2	ClinicalTrials.gov	2	2	2	Drugs and targets
TASADENOTUREV	CHEMBL3990045		Phase 2	ClinicalTrials.gov	2	2	2	Drugs and targets
LENVATINIB	CHEMBL1289601	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
TROCULEUCEL	CHEMBL5314888	Cell	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
NIVOLUMAB	CHEMBL2108738	Antibody	Phase 1/2	ClinicalTrials.gov	2	2	2	Drugs and targets
AMXT-1501 DICAPRATE	CHEMBL6068506	Small molecule	Phase 1/2	ClinicalTrials.gov	1	1	1	Drugs and targets

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Targets51

The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.

Target	Protein	Its drugs that act on it	Mechanisms	Highest stage of those drugs	Gene in Genes	Source
FLT4	Vascular endothelial growth factor receptor	2	Vascular endothelial growth factor receptor 3 inhibitor; Vascular endothelial growth factor receptor inhibitor	Phase 2	https://conductscience.org/library/genes/2324	Drugs and targets
KDR	Vascular endothelial growth factor receptor	2	Vascular endothelial growth factor receptor 2 inhibitor; Vascular endothelial growth factor receptor inhibitor	Phase 2	https://conductscience.org/library/genes/3791	Drugs and targets
PDCD1	Programmed cell death protein 1	2	Programmed cell death protein 1 antagonist; Programmed cell death protein 1 inhibitor	Phase 1/2	https://conductscience.org/library/genes/5133	Drugs and targets
TOP2A	DNA topoisomerase 2-alpha	2	DNA topoisomerase II alpha inhibitor; DNA topoisomerase II inhibitor	Phase 1	https://conductscience.org/library/genes/7153	Drugs and targets
ALDH5A1	Succinate-semialdehyde dehydrogenase, mitochondrial	1	Succinate semialdehyde dehydrogenase inhibitor	Phase 3	https://conductscience.org/library/genes/7915	Drugs and targets
ALK	ALK tyrosine kinase receptor	1	ALK tyrosine kinase receptor inhibitor; EML4-ALK inhibitor	Early phase 1	https://conductscience.org/library/genes/238	Drugs and targets
CD274	Programmed cell death 1 ligand 1	1	Programmed cell death 1 ligand 1 inhibitor	Phase 1	https://conductscience.org/library/genes/29126	Drugs and targets
CDK4	Cyclin-dependent kinase 4	1	Cyclin-dependent kinase 4 inhibitor	Phase 1	https://conductscience.org/library/genes/1019	Drugs and targets
CDK6	Cyclin-dependent kinase 6	1	Cyclin-dependent kinase 6 inhibitor	Phase 1	https://conductscience.org/library/genes/1021	Drugs and targets
CTLA4	Cytotoxic T-lymphocyte protein 4	1	Cytotoxic T-lymphocyte protein 4 inhibitor	Phase 1	https://conductscience.org/library/genes/1493	Drugs and targets

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Trials34

Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT04447755	A Study of Lenvatinib (MK-7902) in Pediatric Participants With Relapsed or Refractory Solid Malignancies (MK-7902-013/E7080)	Phase 2	127	2023-10-04	Relapsed or Refractory Solid Tumors	LENVATINIB	Its record names the disease	ClinicalTrials.gov
NCT02525692	Oral ONC201 in Adult Recurrent Glioblastoma	Phase 2	84	2024-12-24	Glioblastoma, Diffuse Midline Glioma, H3 K27M Glioma, Thalamic Glioma, Infratentorial Glioma, Basal Ganglia Glioma	DORDAVIPRONE	Its record names the disease	ClinicalTrials.gov
NCT03295396	ONC201 in Adults With Recurrent H3 K27M-mutant Glioma	Phase 2	73	2025-07-28	Glioma	DORDAVIPRONE	Its record names the disease	ClinicalTrials.gov
NCT02960230	H3.3K27M Peptide Vaccine With Nivolumab for Children With Newly Diagnosed DIPG and Other Gliomas	Phase 1/2	50	2025-01-22	Diffuse Intrinsic Pontine Glioma, Glioma, Diffuse Midline Glioma, H3 K27M-Mutant	HILTONOL, NIVOLUMAB	Open Targets' reading of its record	ClinicalTrials.gov
NCT04804709	Non-Invasive Focused Ultrasound (FUS) With Oral Panobinostat in Children With Progressive Diffuse Midline Glioma (DMG)	Phase 1	5	2025-06-08	Diffuse Intrinsic Pontine Glioma, Diffuse Pontine and Thalamic Gliomas, Diffuse Midline Glioma, H3 K27M-Mutant	PANOBINOSTAT	Its record names the disease	ClinicalTrials.gov
NCT05476939	Biological Medicine for Diffuse Intrinsic Pontine Glioma (DIPG) Eradication 2.0	Phase 3	433		Diffuse Intrinsic Pontine Glioma, Diffuse Midline Glioma, H3 K27M-Mutant, Diffuse Midline Glioma, H3K27-altered	DORDAVIPRONE, EVEROLIMUS	Its record names the disease	ClinicalTrials.gov
NCT05009992	Combination Therapy for the Treatment of Diffuse Midline Gliomas	Phase 2	360		Diffuse Intrinsic Pontine Glioma, Diffuse Midline Glioma, H3 K27M-Mutant, Recurrent Diffuse Intrinsic Pontine Glioma, Recurrent Diffuse Midline Glioma, H3 K27M-Mutant, Recurrent WHO Grade III Glioma, WHO Grade III Glioma	DORDAVIPRONE, PAXALISIB, TASADENOTUREV	Its record names the disease	ClinicalTrials.gov
NCT04732065	ONC206 for Treatment of Newly Diagnosed, Recurrent Diffuse Midline Gliomas, and Other Recurrent Malignant CNS Tumors	Phase 1	208		Diffuse Midline Glioma (DMG), Glioblastoma, Recurrent Ependymoma, Recurrent Malignant Central Nervous System Neoplasm, Spinal Cord Glioma, World Health Organization (WHO) Grade III Glioma, CNS Tumor, Central Nervous System Tumor	ONC-206	Its record names the disease	ClinicalTrials.gov
NCT03243461	International Cooperative Phase III Trial of the HIT-HGG Study Group (HIT-HGG-2013)	Phase 3	167		Glioblastoma WHO Grade IV, Diffuse Midline Glioma Histone 3 K27M WHO Grade IV, Anaplastic Astrocytoma WHO Grade III, Diffuse Intrinsic Pontine Glioma, Gliomatosis Cerebri	TEMOZOLOMIDE, VALPROIC ACID	Its record names the disease	ClinicalTrials.gov
NCT04196413	GD2 CAR T Cells in Diffuse Intrinsic Pontine Gliomas (DIPG) & Spinal Diffuse Midline Glioma(DMG)	Phase 1	97		Glioma of Spinal Cord, Glioma of Brainstem	CYCLOPHOSPHAMIDE, FLUDARABINE	Its record names the disease	ClinicalTrials.gov

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Comparisons in its trials3

Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.

Comparison	Group 1, with the outcome	Group 2, with the outcome	Participants	Trial	Source
Arm A vs Arm B: Number of Patients With Overall Response	5 of 43	5 of 30	73	NCT03295396	Trial comparisons
Arm A vs Arm B: deaths from any cause	33 of 43	25 of 30	73	NCT03295396	Trial comparisons
Arm A vs Arm B: serious adverse events	17 of 43	7 of 30	73	NCT03295396	Trial comparisons

Papers from its trials20

The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.

PMID	Listed as	Trials listing it	Source
36894288	RESULT	1	PubMed
33402215	RESULT	1	PubMed
30280658	RESULT	1	PubMed
29658856	RESULT	1	PubMed
29658848	RESULT	1	PubMed
25049329	RESULT	1	PubMed
19107519	RESULT	1	PubMed
17960012	RESULT	1	PubMed
16982773	RESULT	1	PubMed
41256696	DERIVED	1	PubMed

1 to 10 of 20

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Data dates

The date of each source this release read.

Open Targets: 2026-09-18; Open Targets
Drug links and their checks, from Drugs and targets: 2026-09-18; Drugs and targets
ClinicalTrials.gov, from Trials: 2026-11-12; Trials
Comparisons, from Trial comparisons: 2026-03-23; Trial comparisons
PubMed, for the checks: 2026-11-06; PubMed
MeSH, for the checks: 2026-11-10; MeSH

Sources 6

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ChEMBL, through Open TargetsCC BY-SA 3.0
ClinicalTrials.govNo license stated
NLM's MeSHNLM's terms
Open TargetsCC0 1.0
PubMedNLM's terms

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
ClinicalTrials.govnone stated
NLM's MeSHNLM's
Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
PubMedNLM's terms

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this disease from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/diseases/v1/EFO_0020983

GET https://conductscience.org/api/diseases/v1/EFO_0020983/drugs?page=2

API reference For AI assistants