Disease · MONDO_0019180

hereditary hemorrhagic telangiectasia

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. hereditary hemorrhagic telangiectasia. Diseases, record MONDO_0019180. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0019180

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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: hereditary hemorrhagic telangiectasia; Open Targets
Open Targets ID: MONDO_0019180; Open Targets
Other names: HHT, Osler-Weber-Rendu disease, Rendu-Osler disease, Rendu-Osler-Weber disease, telangiectasia, hereditary Hemorrahagic, of Rendu, Osler, telangiectasia, hereditary hemorrhagic; Open Targets
Disease Ontology: DOID:1270; Disease Ontology
MeSH: D013683; MeSH
NCI Thesaurus: C35064; NCI Thesaurus
Orphanet: ORPHA:774; Orphanet
Highest stage of its drugs: Approval, for 1 of its 22 drugs, from TTD; Open Targets

Drugs22

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
RANIBIZUMAB	CHEMBL1201825	Antibody	Approval	TTD	2	1	1	Drugs and targets
BEVACIZUMAB	CHEMBL1201583	Antibody	Phase 3	ClinicalTrials.gov	10	9	9	Drugs and targets
TRANEXAMIC ACID	CHEMBL877	Small molecule	Phase 3	ClinicalTrials.gov	2	2	2	Drugs and targets
MUPIROCIN	CHEMBL719	Small molecule	Phase 3	ClinicalTrials.gov	1	1	1	Drugs and targets
PROPRANOLOL	CHEMBL27	Small molecule	Phase 3	ClinicalTrials.gov	1	1	1	Drugs and targets
PAZOPANIB	CHEMBL477772	Small molecule	Phase 2/3	ClinicalTrials.gov	1	1	1	Drugs and targets
POMALIDOMIDE	CHEMBL43452	Small molecule	Phase 2	ClinicalTrials.gov	3	3	3	Drugs and targets
OCTREOTIDE	CHEMBL1680	Protein	Phase 2	ClinicalTrials.gov	2	2	2	Drugs and targets
DOXYCYCLINE	CHEMBL1200699	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
ESTRIOL	CHEMBL193482	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets

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Targets35

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
ADRB1	Beta-1 adrenergic receptor	3	Beta-1 adrenergic receptor antagonist	Phase 3	https://conductscience.org/library/genes/153	Drugs and targets
ADRB2	Beta-2 adrenergic receptor	3	Beta-2 adrenergic receptor antagonist	Phase 3	https://conductscience.org/library/genes/154	Drugs and targets
IFNAR1	Interferon alpha/beta receptor	3	Interferon alpha/beta receptor agonist; Interferon alpha/beta receptor positive modulator	Phase 2	https://conductscience.org/library/genes/3454	Drugs and targets
IFNAR2	Interferon alpha/beta receptor	3	Interferon alpha/beta receptor agonist; Interferon alpha/beta receptor positive modulator	Phase 2	https://conductscience.org/library/genes/3455	Drugs and targets
CRBN	CRL4(CRBN) E3 ubiquitin ligase	2	CRL4(CRBN) E3 ubiquitin ligase inhibitor	Phase 2	https://conductscience.org/library/genes/51185	Drugs and targets
CUL4A	CRL4(CRBN) E3 ubiquitin ligase	2	CRL4(CRBN) E3 ubiquitin ligase inhibitor	Phase 2	https://conductscience.org/library/genes/8451	Drugs and targets
DDB1	CRL4(CRBN) E3 ubiquitin ligase	2	CRL4(CRBN) E3 ubiquitin ligase inhibitor	Phase 2	https://conductscience.org/library/genes/1642	Drugs and targets
ESR1	Estrogen receptor	2	Estrogen receptor alpha modulator; Estrogen receptor modulator	Phase 2	https://conductscience.org/library/genes/2099	Drugs and targets
FGFR1	Fibroblast growth factor receptor	2	Fibroblast growth factor receptor 1 inhibitor; Fibroblast growth factor receptor inhibitor	Phase 2/3	https://conductscience.org/library/genes/2260	Drugs and targets
FGFR3	Fibroblast growth factor receptor	2	Fibroblast growth factor receptor 3 inhibitor; Fibroblast growth factor receptor inhibitor	Phase 2/3	https://conductscience.org/library/genes/2261	Drugs and targets

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Trials29

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
NCT03910244	Pomalidomide for the Treatment of Bleeding in HHT	Phase 2	145	2024-10-23	Telangiectasia, Hereditary Hemorrhagic	POMALIDOMIDE	Its record names the disease	ClinicalTrials.gov
NCT01408030	North American Study of Epistaxis in Hereditary Hemorrhagic Telangiectasia (HHT)	Phase 2	123	2018-10-19	Telangiectasia, Hereditary Hemorrhagic, Epistaxis	BEVACIZUMAB, ESTRIOL, TRANEXAMIC ACID	Its record names the disease	ClinicalTrials.gov
NCT04139018	Timolol Gel for Epistaxis in Hereditary Hemorrhagic Telangiectasia	Phase 2	27	2021-08-24	Hereditary Hemorrhagic Telangiectasia	TIMOLOL	Its record names the disease	ClinicalTrials.gov
NCT01408732	Office-sclerotherapy for Epistaxis Due to Hereditary Hemorrhagic Telangiectasia	Phase 1/2	18	2018-04-20	Epistaxis, Hereditary Hemorrhagic Telangiectasia	SODIUM TETRADECYL SULFATE	Its record names the disease	ClinicalTrials.gov
NCT00588146	Phase 2 Study of PEG-Intron in Hereditary Hemorrhagic Telangiectasia	Phase 2	10	2013-01-28	Anemia, Liver Disease, Hypoxemia	PEGINTERFERON ALFA-2B	Its record names the disease	ClinicalTrials.gov
NCT01402531	Submucosal Bevacizumab for the Management of Recurrent Epistaxis in Patients With Hereditary Hemorrhagic Telangiectasia (HHT)	Phase 2	10	2019-12-04	Hereditary Hemorrhagic Telangiectasia (HHT)	BEVACIZUMAB	Its record names the disease	ClinicalTrials.gov
NCT01752049	Topical Anti-angiogenic Therapy for Telangiectasia in HHT: Proof of Concept	Phase 1/2	5	2023-09-21	Hereditary Hemorrhagic Telangiectasia	TIMOLOL MALEATE	Its record names the disease	ClinicalTrials.gov
NCT07445347	Efficacy and Tolerance of Treatment With Bevacizumab for Severe Liver Involvement With High Cardiac Output in Hereditary Hemorrhagic Telangiectasia Within the French Hereditary Hemorrhagic Telangiectasia Network		111		Hereditary Hemorrhagic Telangiectasia (HHT), Pulmonary Hypertension, Bevacizumab	BEVACIZUMAB	Its record names the disease	ClinicalTrials.gov
NCT02106520	Efficacy of a Bevacizumab Nasal Spray as a Treatment for Epistaxis in Hereditary Hemorrhagic Telangiectasia (HHT)	Phase 2/3	80		Hereditary Hemorrhagic Telangiectasia, Epistaxis	BEVACIZUMAB	Its record names the disease	ClinicalTrials.gov
NCT03850964	Effects of Pazopanib on Hereditary Hemorrhagic Telangiectasia Related Epistaxis and Anemia (Paz)	Phase 2/3	71		Hereditary Hemorrhagic Telangiectasia, Epistaxis, Anemia, Nosebleed, HHT	PAZOPANIB	Its record names the disease	ClinicalTrials.gov

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Comparisons in its trials8

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
Pomalidomide vs Placebo: deaths from any cause	1 of 95	0 of 49	144	NCT03910244	Trial comparisons
Pomalidomide vs Placebo: serious adverse events	22 of 95	8 of 49	144	NCT03910244	Trial comparisons
Sclerotherapy Intervention vs Standard Treatment: serious adverse events	0 of 18	0 of 18	36	NCT01408732	Trial comparisons
Timolol Gel Arm vs Placebo Gel Arm: deaths from any cause	0 of 14	0 of 13	27	NCT04139018	Trial comparisons
Timolol Gel Arm vs Placebo Gel Arm: serious adverse events	0 of 14	0 of 13	27	NCT04139018	Trial comparisons
Pegylated Interferon Alpha-2b vs Standard Care: serious adverse events	3 of 10	0 of 10	20	NCT00588146	Trial comparisons
Topical Timolol Maleate vs Placebo: deaths from any cause	0 of 5	0 of 5	10	NCT01752049	Trial comparisons
Topical Timolol Maleate vs Placebo: serious adverse events	0 of 5	0 of 5	10	NCT01752049	Trial comparisons

Papers from its trials16

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
39510938	RESULT	1	PubMed
37592715	RESULT	1	PubMed
31910860	RESULT	1	PubMed
30191360	RESULT	1	PubMed
28652319	RESULT	1	PubMed
22396517	RESULT	1	PubMed
41719457	DERIVED	1	PubMed
41512167	DERIVED	1	PubMed
39292928	DERIVED	1	PubMed
36344987	DERIVED	1	PubMed

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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/MONDO_0019180

GET https://conductscience.org/api/diseases/v1/MONDO_0019180/drugs?page=2

API reference For AI assistants