26 completed, 6 recruiting, 5 unknown, 3 active not recruiting. 16 Phase 2, 7 Phase 1, 7 Phase 3, 6 Not applicable, 1 Phase 4.
Follow Hereditary Hemorrhagic Telangiectasia: new trials by email Literature Review of Hereditary Hemorrhagic Telangiectasia trials Research Report on Hereditary Hemorrhagic Telangiectasia
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| A Trial to Assess the Efficacy and Safety of Engasertib in Participants With Moderate to Severe Hereditary Hemorrhagic Telangiectasia (HHT) Engasertib, Placebo - Hereditary Hemorrhagic Telangiectasia | Phase 3 | Recruiting | Vaderis Therapeutics AG | 240 | 2026-09-01 | 2026-09-17 |
| DIAG723 in Adults With Hereditary Hemorrhagic Telangiectasia DIAG723, Placebo - Hereditary Hemorrhagic Telangiectasia, Pulmonary Arterial Hypertension | Phase 1 / Phase 2 | Recruiting | Diagonal Therapeutics, Inc. | 93 | 2026-06-04 | 2026-09-08 |
| Body Donation in HHT body donation in HHT - Hereditary Hemorrhagic Telangiectasia | Not applicable | Recruiting | University Hospital, Essen | 5 | 2025-10-01 | 2026-09-17 |
| Cardiac Evaluation in Hereditary Hemorrhagic Telangiectasia Transthoracic echocardiography - Hereditary Hemorrhagic Telangiectasia | Not yet recruiting | Fondazione Policlinico Universitario Agostino Gemelli IRCCS | 380 | 2025-08-05 | 2025-08-14 | |
| A Study to Evaluate ALN-6400 in Healthy Volunteers and Patients With Hereditary Hemorrhagic Telangiectasia (HHT) ALN-6400, Placebo - Hereditary Hemorrhagic Telangiectasia | Phase 1 / Phase 2 | Active not recruiting | Alnylam Pharmaceuticals | 89 | 2024-11-07 | 2026-10-15 |
| Quality of Life in Patients With Hemorrhagic Telangiectasia Questionnaires - Hereditary Hemorrhagic Telangiectasia | Enrolling by invitation | University Hospital, Essen | 250 | 2024-01-22 | 2025-03-30 | |
| Comprehensive HHT Outcomes Registry of the United States (CHORUS) Hereditary Hemorrhagic Telangiectasia, Arteriovenous Malformations | Recruiting | Cure HHT | 10000 | 2023-11-13 | 2026-07-17 | |
| Stress-Echography in Hereditary Haemorrhagic Telangiectasia Patient With Hepatic Involvement Stress echocardiography - Hereditary Hemorrhagic Telangiectasia, Rendu Osler Disease | Not applicable | Completed | Hospices Civils de Lyon | 47 | 2023-08-03 | 2025-12-19 |
| Pazopanib for the Treatment of Epistaxis in Hereditary Hemorrhagic Telangiectasia Pazopanib - Hereditary Hemorrhagic Telangiectasia, Epistaxis | Phase 1 / Phase 2 | Unknown | Cure HHT | 30 | 2023-07-01 | 2022-09-08 |
| Effects of Pazopanib on Hereditary Hemorrhagic Telangiectasia Related Epistaxis and Anemia (Paz) Pazopanib, Placebo oral capsule - Hereditary Hemorrhagic Telangiectasia, Epistaxis | Phase 2 / Phase 3 | Completed | Cure HHT | 71 | 2023-05-08 | 2026-10-27 |
| Prospective Study of Antiplatelet and Anticoagulation Therapy in Hereditary Haemorrhagic Telangiectasia monitoring the use of anticoagulant and/or antiplatelet therapy in patients with osler rendering disease - Hereditary Hemorrhagic Telangiectasia, Rendu Osler Disease | Not applicable | Recruiting | University Hospital, Clermont-Ferrand | 100 | 2023-04-07 | 2026-07-10 |
| Sirolimus for Nosebleeds in HHT Sirolimus - Hereditary Hemorrhagic Telangiectasia, Nosebleeds | Phase 2 | Completed | Unity Health Toronto | 10 | 2022-03-16 | 2026-06-04 |
| Genotype-phenotype Association in Hereditary Hemorrhagic Telangiectasia Registry - Hereditary Hemorrhagic Telangiectasia, HHT | Completed | Fundacion para la Investigacion Biomedica del Hospital Universitario Ramon y Cajal | 85 | 2021-04-30 | 2025-03-17 | |
| Ultra-low-dose Chest CT for HHT Ultra-low dose chest CT - Hereditary Hemorrhagic Telangiectasia | Completed | Mayo Clinic | 3 | 2021-04-21 | 2026-04-24 | |
| Bevacizumab In Hereditary Hemorrhagic Telangiectasia Bevacizumab (Avastin®) - Hereditary Hemorrhagic Telangiectasia | Phase 2 | Active not recruiting | Hanny Al-Samkari, MD | 33 | 2020-11-23 | 2026-05-05 |
| Tacrolimus Trial for Hereditary Hemorrhagic Telangiectasia (HHT) Tacrolimus capsule (low-dose) - Hereditary Hemorrhagic Telangiectasia, Epistaxis Nosebleed | Phase 2 | Completed | Unity Health Toronto | 10 | 2020-10-20 | 2025-04-01 |
| Influence of Hypoxic Induced Factors in Patients With Hereditary Hemorrhagic Telangiectasia hypoxic induced factors - Hereditary Hemorrhagic Telangiectasia | Recruiting | University Hospital, Essen | 100 | 2020-08-10 | 2024-12-06 | |
| Propranolol for Epistaxis in Hereditary Hemorrhagic Telangiectasia Patients Propranolol treatment, Placebo - Hereditary Hemorrhagic Telangiectasia, Osler Weber Rendu Disease | Phase 3 | Terminated | University Hospital, Bordeaux | 15 | 2020-06-23 | 2026-06-23 |
| Counseling Intervention in Hereditary Hemorrhagic Telangiectasia in the COVID Era. Counseling - Hereditary Hemorrhagic Telangiectasia | Not applicable | Completed | Fondazione Policlinico Universitario Agostino Gemelli IRCCS | 50 | 2020-05-05 | 2023-03-02 |
| Timolol Gel for Epistaxis in Hereditary Hemorrhagic Telangiectasia Timolol Gel, Placebo Gel - Hereditary Hemorrhagic Telangiectasia | Phase 2 | Completed | Washington University School of Medicine | 27 | 2019-10-20 | 2021-08-24 |
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Telangiectasia, Hereditary Hemorrhagic (44) · Epistaxis (15) · Anemia (3) · Arteriovenous Malformations (3) · Gastrointestinal Hemorrhage (3) · Genetic Diseases, Inborn (3) · Hematologic Diseases (3) · Rare Diseases (3) · Asthma (2) · Intracranial Arteriovenous Malformations (2) · Pulmonary Disease, Chronic Obstructive (2) · Telangiectasis (2) · Vascular Malformations (2) · Acquired Immunodeficiency Syndrome (1) · Alopecia Areata (1)
Cardiovascular Abnormalities (44) · Cardiovascular Diseases (44) · Congenital Abnormalities (44) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (44) · Hemic and Lymphatic Diseases (44) · Hemorrhagic Disorders (44) · Hemostatic Disorders (44) · Vascular Diseases (44) · Telangiectasis (42) · Vascular Malformations (42) · Hematologic Diseases (41) · Pathologic Processes (22) · Pathological Conditions, Signs and Symptoms (22) · Respiratory Tract Diseases (18) · Hemorrhage (17)
Bevacizumab (3) · Exercise Test (2) · Octreotide (2) · Registries (2) · Surveys and Questionnaires (2) · Thalidomide (2) · pazopanib (2) · Blood Specimen Collection (1) · Cellulose (1) · Clostridium perfringens epsilon-toxin (1) · Counseling (1) · Echocardiography (1) · Ferrosoferric Oxide (1) · Fluoridation (1) · Hematologic Tests (1)
Unity Health Toronto (5) · Hospices Civils de Lyon (4) · University Hospital, Essen (4) · Cure HHT (3) · National Center for Research Resources (NCRR) (3) · Fondazione Policlinico Universitario Agostino Gemelli IRCCS (2) · Imperial College London (2) · Alnylam Pharmaceuticals (1) · Centre Hospitalier Universitaire Dijon (1) · Diagonal Therapeutics, Inc. (1) · Fondazione IRCCS Policlinico San Matteo di Pavia (1) · Fundacion para la Investigacion Biomedica del Hospital Universitario Ramon y Cajal (1) · Hanny Al-Samkari, MD (1) · Hospital Italiano de Buenos Aires (1) · Mayo Clinic (1)
United States (14) · France (8) · Canada (7) · Germany (6) · Australia (2) · Italy (2) · Netherlands (2) · Spain (2) · Argentina (1) · Israel (1) · New Zealand (1) · United Kingdom (1)
James Gossage, MD (2) · Joshua R. Korzenik (2) · Marie E Faughnan, MD MSc FRCPC (2) · Alan Guttmacher (1) · Atul Kumar, MD (1) · Carlo Balduini, M.D. (1) · Cassi Friday, PhD (1) · Claire L Shovlin (1) · Claire L Shovlin, PhD FRCP (1) · Eitan Yaniv, MD (1) · Freya Droege, MD (1) · Gabriella Locorotondo (1)
PMID 24676541, PMID 25005464, PMID 26686256, PMID 27599328, PMID 30191360, PMID 30657990, PMID 32940653, PMID 35854330, PMID 41843464
2025 (2), U54NS065705 (2), 2016 (1)