16 completed, 4 active not recruiting, 1 enrolling by invitation, 1 terminated. 23 Phase 2, 6 Phase 3, 4 Phase 1.
Follow Hereditary Angioedema: new trials by email Literature Review of Hereditary Angioedema trials Research Report on Hereditary Angioedema
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 Long-term Study of STAR-0215 in Participants With Hereditary Angioedema STAR-0215 - Hereditary Angioedema | Phase 2 | Active not recruiting | Astria Therapeutics, Inc. | 56 | 2023-09-26 | 2025-07-23 |
| A Study of Navenibart (STAR-0215) in Participants With Hereditary Angioedema Navenibart - Hereditary Angioedema | Phase 1 / Phase 2 | Completed | Astria Therapeutics, Inc. | 29 | 2023-02-21 | 2026-05-08 |
| Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema deucrictibant - Hereditary Angioedema, Hereditary Angioedema Type I | Phase 2 / Phase 3 | Enrolling by invitation | Pharvaris Netherlands B.V. | 150 | 2022-12-28 | 2026-07-21 |
| Safety, Tolerability, PK, PD of ADX-324 in Healthy Volunteers and Hereditary Angioedema Patients ADX-324, Placebo - Hereditary Angioedema | Phase 1 / Phase 2 | Active not recruiting | ADARx Pharmaceuticals, Inc. | 53 | 2022-12-14 | 2026-06-16 |
| Dose-ranging Study of Oral PHA-022121 for Prophylaxis Against Angioedema Attacks in Patients With Hereditary Angioedema Type I or Type II Deucrictibant low dose, Deucrictibant high dose, Placebo - Hereditary Angioedema, Hereditary Angioedema Type I | Phase 2 | Terminated | Pharvaris Netherlands B.V. | 34 | 2022-04-19 | 2026-08-17 |
| 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 |
| NTLA-2002 in Adults With Hereditary Angioedema (HAE) Biological NTLA-2002, Normal Saline IV Administration - Hereditary Angioedema | Phase 1 / Phase 2 | Active not recruiting | Intellia Therapeutics | 37 | 2021-12-10 | 2026-09-25 |
| Efficacy and Safety of GNR-038 vs Berinert® in Patients With Hereditary Angioedema GNR-038, 50 МЕ/ kg, GNR-038, 100 МЕ/ kg, Berinert®, 20 МЕ/ kg - Hereditary Angioedema | Phase 2 / Phase 3 | Withdrawn | AO GENERIUM | 2021-12-01 | 2022-02-04 | |
| Dose-ranging Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema Deucrictibant, Placebo - Hereditary Angioedema, Hereditary Angioedema Type I | Phase 2 | Completed | Pharvaris Netherlands B.V. | 74 | 2021-02-03 | 2025-12-17 |
| An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema Donidalorsen - Hereditary Angioedema | Phase 2 | Completed | Ionis Pharmaceuticals, Inc. | 20 | 2020-03-31 | 2026-05-08 |
| A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary Angioedema Donidalorsen, Placebo - Hereditary Angioedema | Phase 2 | Completed | Ionis Pharmaceuticals, Inc. | 23 | 2020-01-07 | 2023-04-03 |
| A Phase II, Cross-over Clinical Trial Evaluating the Efficacy and Safety of KVD900 (Sebetralstat) in the On-demand Treatment of Angioedema Attacks in Adult Subjects With Hereditary Angioedema Type I or II KVD900, Placebo - Hereditary Angioedema | Phase 2 | Completed | KalVista Pharmaceuticals, Ltd. | 84 | 2019-07-02 | 2025-05-02 |
| A Study to Investigate CSL312 in Subjects With Hereditary Angioedema (HAE) Factor XIIa antagonist monoclonal antibody, Placebo - Hereditary Angioedema | Phase 2 | Completed | CSL Behring | 44 | 2018-10-29 | 2022-11-08 |
| 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 |
| 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 |
| A Phase 2 HAE Prophylaxis Study With Recombinant Human C1 Inhibitor Recombinant human C1 inhibitor, Placebo - Hereditary Angioedema | Phase 2 | Completed | Pharming Technologies B.V. | 32 | 2014-12-01 | 2017-12-08 |
| Safety and Efficacy of Oral BCX4161 as a Prophylactic Treatment for HAE BCX4161, Placebo - Hereditary Angioedema | Phase 2 | Completed | BioCryst Pharmaceuticals | 24 | 2013-11-01 | 2014-08-18 |
| Safety of Ruconest in 2-13 Year Old Hereditary Angioedema (HAE) Patients rhC1INH - Hereditary Angioedema | Phase 2 | Completed | Pharming Technologies B.V. | 57 | 2012-01-17 | 2024-03-29 |
| A Study to Evaluate the Safety and Pharmacology of Subcutaneous Administration of CINRYZE With Recombinant Human Hyaluronidase CINRYZE with rHuPH20 - Hereditary Angioedema | Phase 2 | Completed | Shire | 12 | 2011-09-12 | 2021-07-19 |
| A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous CINRYZE Administration CINRYZE - Hereditary Angioedema | Phase 2 | Completed | Shire | 26 | 2010-06-07 | 2021-07-09 |
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Angioedemas, Hereditary (23) · Angioedema (5) · Hereditary Angioedema Types I and II (3) · Genetic Diseases, Inborn (2) · Hereditary Angioedema Type III (1) · Hypersensitivity (1) · Immune System Diseases (1)
Cardiovascular Diseases (23) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (23) · Hereditary Complement Deficiency Diseases (23) · Hypersensitivity, Immediate (23) · Immunologic Deficiency Syndromes (23) · Primary Immunodeficiency Diseases (23) · Skin Diseases (23) · Skin Diseases, Vascular (23) · Skin and Connective Tissue Diseases (23) · Urticaria (23) · Vascular Diseases (23) · Hypersensitivity (22) · Immune System Diseases (22) · Genetic Diseases, Inborn (21) · Angioedema (18)
Complement C1 Inhibitor Protein (6) · conestat alfa (4) · Sodium Chloride (3) · IONIS-PKK-LRx (2) · SERPING1 protein, human (2) · avoralstat (2) · donidalorsen (2) · RNA, Small Interfering (1) · berotralstat (1) · sebetralstat (1)
Pharming Technologies B.V. (4) · BioCryst Pharmaceuticals (3) · Pharvaris Netherlands B.V. (3) · Astria Therapeutics, Inc. (2) · CSL Behring (2) · Ionis Pharmaceuticals, Inc. (2) · Shire (2) · ADARx Pharmaceuticals, Inc. (1) · AO GENERIUM (1) · BioMarin Pharmaceutical (1) · Intellia Therapeutics (1) · KalVista Pharmaceuticals, Ltd. (1)
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Anurag Relan, MD (2) · Bruno Giannetti, MD (1) · Henriette Farkas, MD (1) · Lauge Farnaes, MD (1) · Marc Riedl, MD (1) · Marc Riedl, MD MS (1) · Marcus Maurer, MD (1) · Marcus Maurer, Prof MD (1) · Oksana A. Markova, MD (1)
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