Disease · MONDO_0000820

cerebral cavernous malformation

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. cerebral cavernous malformation. Diseases, record MONDO_0000820. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0000820

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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: cerebral cavernous malformation; Open Targets
Open Targets ID: MONDO_0000820; Open Targets
Other names: CCM, brain cavernous hemangioma; Open Targets
Disease Ontology: DOID:0060669; Disease Ontology
NCI Thesaurus: C84626; NCI Thesaurus
Orphanet: ORPHA:164; Orphanet
Highest stage of its drugs: Phase 2, for 2 of its 5 drugs, from ClinicalTrials.gov; Open Targets

Drugs5

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
PROPRANOLOL	CHEMBL27	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
SIROLIMUS	CHEMBL413	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
ATORVASTATIN	CHEMBL1487	Small molecule	Phase 1/2	ClinicalTrials.gov	1	1	1	Drugs and targets
DARATUMUMAB	CHEMBL1743007	Antibody	Early phase 1	ClinicalTrials.gov	1	1	1	Drugs and targets
SIMVASTATIN	CHEMBL1064	Small molecule	Early phase 1	ClinicalTrials.gov	1	1	1	Drugs and targets

Targets5

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
HMGCR	3-hydroxy-3-methylglutaryl-coenzyme A reductase	2	HMG-CoA reductase inhibitor	Phase 1/2	https://conductscience.org/library/genes/3156	Drugs and targets
ADRB1	Beta-1 adrenergic receptor	1	Beta-1 adrenergic receptor antagonist	Phase 2	https://conductscience.org/library/genes/153	Drugs and targets
ADRB2	Beta-2 adrenergic receptor	1	Beta-2 adrenergic receptor antagonist	Phase 2	https://conductscience.org/library/genes/154	Drugs and targets
CD38	ADP-ribosyl cyclase/cyclic ADP-ribose hydrolase 1	1	Lymphocyte differentiation antigen CD38 inhibitor	Early phase 1	https://conductscience.org/library/genes/952	Drugs and targets
FKBP1A	Peptidyl-prolyl cis-trans isomerase FKBP1A	1	FK506-binding protein 1A inhibitor	Phase 2	https://conductscience.org/library/genes/2280	Drugs and targets

Trials5

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
NCT02603328	Atorvastatin Treatment of Cavernous Angiomas With Symptomatic Hemorrhage Exploratory Proof of Concept (AT CASH EPOC) Trial	Phase 1/2	80	2025-08-22	Cerebral Cavernous Malformation	ATORVASTATIN	Its record names the disease	ClinicalTrials.gov
NCT06091332	Hemorrhagic Brainstem Cavernous Malformations Treatment With Sirolimus: aSingle Centre, Randomized, Placebo-controlled Pilot Trial	Phase 2	75		Cavernous Malformations, Intracerebral Hemorrhage, Brainstem Stroke	SIROLIMUS	Open Targets' reading of its record	ClinicalTrials.gov
NCT03589014	Treat_CCM: Propranolol in Familial Cerebral Cavernous Malformation	Phase 2	71		Cerebral Cavernous Malformation	PROPRANOLOL	Its record names the disease	ClinicalTrials.gov
NCT01764451	Permeability MRI in Cerebral Cavernous Malformations Type 1 in New Mexico: Effects of Statins	Early phase 1	12		Cavernous Angioma, Familial, Cerebral Cavernous Malformations, Cerebral Cavernous Hemangioma	SIMVASTATIN	Open Targets' reading of its record	ClinicalTrials.gov
NCT07026604	Daratumumab for Familial Cerebral Cavernous Malformations: A Single-Arm Safety and Efficacy Study	Early phase 1	10		Cavernous Malformation, Cerebral	DARATUMUMAB	Its record names the disease	ClinicalTrials.gov

Comparisons in its trials1

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
Treatment vs Placebo: Compare Rate of Drug Compliance in Atorvastatin vs Placebo Group	33 of 33	31 of 31	64	NCT02603328

Source Trial comparisons

Papers from its trials7

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
41608890	DERIVED	1	PubMed
41117517	DERIVED	1	PubMed
40570636	DERIVED	2	PubMed
40120614	DERIVED	1	PubMed
38419711	DERIVED	1	PubMed
36403580	DERIVED	1	PubMed
32398113	DERIVED	1	PubMed

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/MONDO_0000820

GET https://conductscience.org/api/diseases/v1/MONDO_0000820/drugs

API reference For AI assistants