RecruitingPhase 1 / Phase 2NCT07601425
Blood
Hereditary Hemorrhagic Telangiectasia
Also known as HHT, Osler-Weber-Rendu disease, Osler-Rendu-Weber syndrome
Hereditary hemorrhagic telangiectasia is a vascular dysplasia syndrome caused by heterozygous loss-of-function mutations in genes encoding components of the TGF-beta/BMP signaling pathway, most commonly ENG (endoglin, HHT type 1) or ACVRL1
ORPHA:774 ↗Gene ENGGene ACVRL1Prevalence 1 in 5,000 to 1 in 8,000Onset Telangiectasias and nosebleeds typically emerge in adolescence to adulthood; AVMs may be congenitalAutosomal dominant
13
studies recruiting now
as of 7 Sept 2026
107
studies registered in total
as of 7 Sept 2026
6
countries with a recruiting site
as of 7 Sept 2026
3 Jun 2026
most recent study posted
among recruiting studies
Recruiting trials
RecruitingPhase 1 / Phase 2NCT07623525
DIAG723 in Adults With Hereditary Hemorrhagic Telangiectasia
RecruitingNCT01761981
Institutional Registry of Haemorrhagic Hereditary Telangiectasia
RecruitingNCT06259292
Comprehensive HHT Outcomes Registry of the United States (CHORUS)
RecruitingNot applicableNCT05641142
Prospective Study of Antiplatelet and Anticoagulation Therapy in Hereditary Haemorrhagic Telangiectasia
Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.
See all 13 recruiting studiesWhere recruiting studies are running
Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.
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About Hereditary Hemorrhagic Telangiectasia
Hereditary hemorrhagic telangiectasia is a vascular dysplasia syndrome caused by heterozygous loss-of-function mutations in genes encoding components of the TGF-beta/BMP signaling pathway, most commonly ENG (endoglin, HHT type 1) or ACVRL1 (activin receptor-like kinase 1, HHT type 2), leading to abnormal arteriovenous connections in multiple organs. These arteriovenous malformations (AVMs) most frequently affect the nasal mucosa, gastrointestinal tract, lungs, liver, and brain, causing recurrent hemorrhage and in severe cases high-output cardiac failure or paradoxical embolism. The diagnosis is clinical using the Curacao criteria and confirmed by genetic testing.
Common clinical features
Recurrent spontaneous epistaxis (nosebleeds) as the hallmark symptomMultiple mucocutaneous telangiectasias on lips, tongue, fingers, and faceGastrointestinal bleeding from intestinal telangiectasiasPulmonary arteriovenous malformations causing hypoxemia and risk of strokeCerebral AVMs with risk of hemorrhagic stroke or brain abscessHepatic AVMs potentially causing high-output heart failure or portal hypertensionIron deficiency anemia from chronic blood lossMigraine headaches
From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.
Treatments being studied
1 approved treatment and 13 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.
Approved: Ranibizumab (Byooviz)
Phase 3Bevacizumab (Abevmy)
Phase 3Propranolol
Phase 3Tranexamic Acid (Cyklo-f heavy period relief)
Phase 3Sodium Chloride (Aqsia (balanced salt soln))
Phase 3Mupirocin (Bactroban)
Phase 2Pazopanib (Votrient)
Phase 2Tamoxifen
Phase 2Doxycycline Hyclate (Acticlate)
+ 5 more in development
Before you apply
Things trial teams commonly ask about for Hereditary Hemorrhagic Telangiectasia. Not eligibility rules; those are set by each study.
- Confirmed HHT diagnosis using Curacao criteria (at least 3 of 4: epistaxis, telangiectasias, visceral AVMs, family history) or genetic mutation identification is required for most trials; bring genetic test results and imaging of AVMs.
- Severity of epistaxis (frequency, duration, transfusion dependence, Epistaxis Severity Score) and iron deficiency status are primary outcomes in antiangiogenic therapy trials including bevacizumab and thalidomide studies.
- Screening for pulmonary and cerebral AVMs is recommended before enrollment in any study involving systemic agents, as untreated large pulmonary AVMs increase procedural risk.
Related conditions
Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).