4 completed, 4 recruiting, 1 active not recruiting, 1 approved for marketing. 7 Phase 3, 3 Phase 2, 2 Not applicable, 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 7 | Completed 3, Recruiting 2, Enrolling by invitation 1, Terminated 1 |
| Phase 2 | 3 | Completed 2, Active not recruiting 1 |
| Not applicable | 2 | Recruiting 2 |
| Phase 1 | 1 | Active not recruiting 1 |
Follow HAE: new trials by email Literature Review of HAE trials Research Report on HAE
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 |
|---|---|---|---|---|---|---|
| Rectal Arterial Embolization vs Band Ligation for the Treatment of Internal hemOrrhoidS Hemorrhoidal Artery Embolization, Rubber Band Ligation - Hemorrhoidal Bleeding, Hemorrhoids, Internal | Not applicable | Recruiting | Jessica K. Stewart, MD | 40 | 2026-05-19 | 2026-07-10 |
| Phase 3 Extension Study of ADX-324 in Participants With Hereditary Angioedema (HAE) ADX-324 Dose Level 1, ADX-324 Dose Level 2 - Hereditary Angioedema (HAE), Hereditary Angioedema - Type 1 | Phase 3 | Recruiting | ADARx Pharmaceuticals, Inc. | 90 | 2026-04-02 | 2026-09-04 |
| STOP-HAE: A Phase 3 Study of ADX-324 in HAE ADX-324, Placebo - Hereditary Angioedema, HAE | Phase 3 | Recruiting | ADARx Pharmaceuticals, Inc. | 90 | 2025-08-28 | 2026-09-10 |
| Hemorrhoidal Artery Embolization: Longitudinal Impact On Symptoms (HELIOS) Hemorrhoidal artery embolization - Internal Hemorrhoids, HAE | Not applicable | Recruiting | University of California, Los Angeles | 22 | 2025-08-05 | 2026-11-10 |
| A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema Dose 1 of BMN 331, Dose 2 of BMN 331, Dose 3 of BMN 331 - Hereditary Angioedema, HAE | Phase 1 / Phase 2 | Active not recruiting | BioMarin Pharmaceutical | 44 | 2022-02-15 | 2024-05-16 |
| Open-label Berotralstat Access to HAE Patients Previously Enrolled in Berotralstat Studies berotralstat - Hereditary Angioedema, HAE | Phase 3 | Enrolling by invitation | BioCryst Pharmaceuticals | 139 | 2021-07-23 | 2025-12-23 |
| Biomarker for Hereditary AngioEdema Disease C1 Esterase Inhibitor Deficiency, Angio Edema | Completed | CENTOGENE GmbH Rostock | 42 | 2018-08-20 | 2022-03-24 | |
| A Long Term Safety Study of BCX7353 in Hereditary Angioedema BCX7353 - Hereditary Angioedema, HAE | Phase 2 / Phase 3 | Completed | BioCryst Pharmaceuticals | 387 | 2018-02-16 | 2023-06-18 |
| Efficacy and Safety Study of BCX7353 as an Oral Treatment for the Prevention of Attacks in HAE BCX7353 capsules, Placebo oral capsule - Hereditary Angioedema, HAE | Phase 3 | Completed | BioCryst Pharmaceuticals | 121 | 2018-02-06 | 2023-06-26 |
| Open-label, Long-term Safety Study of Avoralstat in Subjects With Hereditary Angioedema avoralstat - Hereditary Angioedema, HAE | Phase 3 | Terminated | BioCryst Pharmaceuticals | 6 | 2015-12-01 | 2016-02-17 |
| 12-Week Safety and Efficacy Study of BCX4161 as an Oral Prophylaxis Against HAE Attacks BCX4161, Placebo - Hereditary Angioedema, HAE | Phase 2 / Phase 3 | Completed | BioCryst Pharmaceuticals | 110 | 2014-12-17 | 2025-11-17 |
| Oral Berotralstat Expanded Access Program Berotralstat - Hereditary Angioedema, HAE | Approved for marketing | BioCryst Pharmaceuticals | 2020-12-11 |
Angioedemas, Hereditary (10) · Angioedema (1) · Hemorrhoids (1) · Hereditary Angioedema Type III (1) · Hereditary Angioedema Types I and II (1)
Cardiovascular Diseases (11) · Vascular Diseases (11) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (10) · Genetic Diseases, Inborn (10) · Hereditary Complement Deficiency Diseases (10) · Hypersensitivity (10) · Hypersensitivity, Immediate (10) · Immune System Diseases (10) · Immunologic Deficiency Syndromes (10) · Primary Immunodeficiency Diseases (10) · Skin Diseases (10) · Skin Diseases, Vascular (10) · Skin and Connective Tissue Diseases (10) · Urticaria (10) · Angioedema (9)
berotralstat (4) · avoralstat (2) · RNA, Small Interfering (1) · Sodium Chloride (1)
BioCryst Pharmaceuticals (6) · ADARx Pharmaceuticals, Inc. (2) · BioMarin Pharmaceutical (1) · CENTOGENE GmbH Rostock (1) · Jessica K. Stewart, MD (1) · University of California, Los Angeles (1)
United States (8) · France (7) · Germany (6) · Spain (6) · United Kingdom (6) · Canada (5) · Hungary (5) · Poland (5) · Australia (4) · Austria (4) · Belgium (4) · Czechia (4) · Hong Kong (3) · Israel (3) · Italy (3)
Lauge Farnaes, MD (2) · Bruce Zuraw, MD (1) · Dennis Jensen, MD (1) · Henriette Farkas, MD (1) · Henriette Farkas, MD, PhD, DSc (1) · Jessica Stewart, MD (1) · Lucas Cusumano, MD (1) · Marc Riedl, MD MS (1) · Peter Bauer, MD (1) · Vesna Grivcheva-Panoska, MD, PhD (1)
PMID 36326435, PMID 36408587, PMID 33098856, PMID 33866032, PMID 34161665, PMID 35820771, PMID 38006972, PMID 38020288, PMID 40490218