33 completed, 5 recruiting, 4 active not recruiting, 4 terminated. 51 Phase 2, 12 Phase 1, 3 Phase 3.
Follow Telangiectasis: new trials by email Literature Review of Telangiectasis trials Research Report on Telangiectasis
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 |
|---|---|---|---|---|---|---|
| Safety and Efficacy of Mutation-targeted Precision Genetic Therapy for Ataxia-Telangiectasia (A-T) Antisense oligonucleotide targeting the ATM gene - Ataxia Telangiectasia | Phase 1 / Phase 2 | Recruiting | Timothy Yu | 10 | 2026-08-01 | 2026-05-28 |
| Harmony-HHT: ATV-1601 in Participants With Hereditary Hemorrhagic Telangiectasia (HHT) ATV-1601, Placebo - Hereditary Hemorrhagic Telangiectasia (HHT), Osler Weber Rendu Disease | Phase 1 / Phase 2 | Recruiting | Atavistik Bio, Inc | 100 | 2026-07-27 | 2026-11-06 |
| 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 |
| An Phase Ib/II Clinical Trial of TCC1727 Combination Therapy in Advanced Solid Tumors TCC1727 tablet 90mg, TCC1727 tablet 120mg, TCC1727 tablet 160mg - Solid Cancers, NSCLC (Advanced Non-small Cell Lung Cancer) | Phase 1 / Phase 2 | Recruiting | Beijing Tide Pharmaceutical Co., Ltd | 266 | 2025-12-03 | 2026-01-28 |
| CLaCS Using 0.2% Aetoxysklerol Foam and 70% Glucose for the Treatment of Lower Limb Telangiectasias CLaCS - Varicose Veins of Lower Limb, Spider Veins | Phase 2 | Not yet recruiting | Charles University, Czech Republic | 240 | 2025-09-01 | 2025-07-18 |
| 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 |
| Sirolimus Treatment in Pediatric Patients With Ataxia-Telangiectasia Rapamune (sirolimus/rapamycin) - Ataxia-Telangiectasia (A-T), Ataxia-Telangiectasia | Phase 2 | Completed | Sara Elva Espinosa Padilla, MD | 6 | 2024-07-11 | 2026-09-15 |
| 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 |
| Assess Safety and Efficacy of VAD044 in HHT Patients VAD044 Part I, VAD044 Part II - Hereditary Hemorrhagic Telangiectasia (HHT) | Phase 1 / Phase 2 | Active not recruiting | Vaderis Therapeutics AG | 75 | 2022-07-18 | 2026-03-17 |
| Efficacy of Nintedanib for Treatment of Epistaxis in Hereditary Hemorrhagic Telangiectasia (HHT) Patients Nintedanib, Placebo - Telangiectasia, Hereditary Hemorrhagic | Phase 2 | Recruiting | Dr. Romain Lazor | 48 | 2022-05-05 | 2026-03-11 |
| Sirolimus for Nosebleeds in HHT Sirolimus - Hereditary Hemorrhagic Telangiectasia, Nosebleeds | Phase 2 | Completed | Unity Health Toronto | 10 | 2022-03-16 | 2026-06-04 |
| Ataxia-telangiectasia: Treating Mitochondrial Dysfunction With a Novel Form of Anaplerosis Triheptanoin - Ataxia Telangiectasia | Phase 2 | Completed | The University of Queensland | 30 | 2022-03-15 | 2023-07-20 |
| MBM-01 (Tempol) for the Treatment of Ataxia Telangiectasia MBM-01 - Ataxia Telangiectasia Louis-Bar, Ataxia Telangiectasia in Children | Phase 2 | Unknown | Matrix Biomed, Inc. | 20 | 2021-07-01 | 2021-05-17 |
| 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 |
| Efficacy of Nintedanib Per os as a Treatment for Epistaxis in HHT Disease. Nintedanib 150 mg and 100 mg soft capsules, Oral treatment of placebo soft capsule - Telangiectasia, Hereditary Hemorrhagic, Rendu Osler Disease | Phase 2 | Completed | Hospices Civils de Lyon | 61 | 2020-06-22 | 2025-08-01 |
| N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T) IB1001 - Ataxia Telangiectasia | Phase 2 | Terminated | IntraBio Inc | 17 | 2020-01-08 | 2026-03-25 |
| 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 |
| Pomalidomide for the Treatment of Bleeding in HHT Pomalidomide Oral Product, Placebo oral capsule - Telangiectasia, Hereditary Hemorrhagic | Phase 2 | Completed | The Cleveland Clinic | 145 | 2019-10-17 | 2024-10-23 |
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Telangiectasia, Hereditary Hemorrhagic (32) · Epistaxis (16) · Ataxia Telangiectasia (10) · Arteriovenous Malformations (3) · Gastrointestinal Hemorrhage (3) · Retinal Telangiectasis (3) · Anemia (2) · Hematologic Diseases (2) · Rosacea (2) · Vascular Malformations (2) · Acne Vulgaris (1) · Angiodysplasia (1) · Ataxia (1) · Blast Crisis (1)
Cardiovascular Diseases (50) · Vascular Diseases (50) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (43) · Congenital Abnormalities (34) · Hemic and Lymphatic Diseases (34) · Hemorrhagic Disorders (33) · Hemostatic Disorders (33) · Cardiovascular Abnormalities (32) · Hematologic Diseases (32) · Vascular Malformations (30) · Pathological Conditions, Signs and Symptoms (27) · Pathologic Processes (24) · Hemorrhage (19) · Signs and Symptoms (19)
Bevacizumab (7) · pazopanib (3) · Capsules (2) · Octreotide (2) · Ranibizumab (2) · Sirolimus (2) · Tacrolimus (2) · Thalidomide (2) · nicotinamide-beta-riboside (2) · nintedanib (2) · Argon Plasma Coagulation (1) · Buprenorphine (1) · Caspofungin (1) · Cellulose (1) · Doxycycline (1)
Hospices Civils de Lyon (5) · Unity Health Toronto (4) · Cure HHT (2) · Manhattan Eye, Ear & Throat Hospital (2) · Radboud University Medical Center (2) · University of California, San Diego (2) · Alnylam Pharmaceuticals (1) · Atavistik Bio, Inc (1) · Barretos Cancer Hospital (1) · Beijing Tide Pharmaceutical Co., Ltd (1) · BioDelivery Sciences International (1) · Charles University, Czech Republic (1) · Diagonal Therapeutics, Inc. (1) · Dr. Romain Lazor (1) · European Organisation for Research and Treatment of Cancer - EORTC (1)
United States (23) · France (11) · Canada (8) · Australia (3) · Germany (3) · Italy (3) · Netherlands (3) · Spain (3) · Switzerland (3) · Belgium (2) · Argentina (1) · Austria (1) · Brazil (1) · China (1) · Israel (1)
Marie E Faughnan, MD MSc FRCPC (3) · James Gossage, MD (2) · Lawrence A. Yannuzzi, MD (2) · Arthur Korotkin, M.D. (1) · Atul Kumar, MD (1) · Carlo Balduini, M.D. (1) · Christelle Achkar, MD (1) · Claudio Viscoli, MD (1) · Damien Picard (1) · David Coman, MBBS FRACP (1) · Eitan Yaniv, MD (1)
PMID 16763896, PMID 16934683, PMID 19841031, PMID 22144182, PMID 22396517, PMID 22875082, PMID 22927201, PMID 24236946, PMID 24595923, PMID 25656498, PMID 26433381, PMID 26547562, PMID 26686256, PMID 26851119, PMID 27599328, PMID 27599329, PMID 28652319, PMID 28871440, PMID 28969699, PMID 29110179, PMID 30191360, PMID 30586396, PMID 30796268, PMID 30948626, PMID 31427745, PMID 31910860, PMID 32179040, PMID 32739509, PMID 32778489, PMID 32940653, PMID 33437944, PMID 33482890, PMID 34349180, PMID 34620022, PMID 36344987, PMID 39292928, PMID 39510938, PMID 39718659, PMID 40739200, PMID 41297007, PMID 41719457
2017 (2), 2009 (1), 2015 (1), 2019 (1), 2025 (1), U54NS065705 (1)