38 completed, 11 terminated, 3 withdrawn, 1 active not recruiting. 27 Phase 2, 19 Phase 1, 15 Phase 3.
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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 Study of Avoralstat In Participants With Diabetic Macular Edema avoralstat - Diabetic Macular Edema (DME) | Phase 1 | Terminated | BioCryst Pharmaceuticals | 3 | 2025-10-02 | 2026-05-11 |
| A Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of BCX17725 BCX17725, Placebo - Netherton Syndrome | Phase 1 | Recruiting | BioCryst Pharmaceuticals | 78 | 2024-09-26 | 2026-08-27 |
| Dose Escalation of BCX10013 in Participants with Paroxysmal Nocturnal Hemoglobinuria (PNH) BCX10013 - Paroxysmal Nocturnal Hemoglobinuria | Phase 1 | Completed | BioCryst Pharmaceuticals | 8 | 2023-10-24 | 2025-01-08 |
| Long-term Safety of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria BCX9930 - Paroxysmal Nocturnal Hemoglobinuria | Phase 2 | Terminated | BioCryst Pharmaceuticals | 28 | 2023-01-18 | 2025-10-24 |
| Berotralstat Treatment in Children With Hereditary Angioedema Berotralstat - Hereditary Angioedema, Pediatric | Phase 3 | Active not recruiting | BioCryst Pharmaceuticals | 29 | 2022-10-25 | 2026-02-25 |
| Study to Evaluate the Safety, Tolerability of BCX9930 in Participants With Either Complement 3 Glomerulopathy (C3G), Immunoglobulin A Nephropathy (IgAN), or Primary Membranous Nephropathy (PMN) BCX9930 - Complement 3 Glomerulopathy, Immunoglobulin A Nephropathy | Phase 2 | Terminated | BioCryst Pharmaceuticals | 2 | 2022-02-18 | 2024-05-02 |
| BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants With Inadequate Response to C5 Inhibitor Therapy Eculizumab, Ravulizumab, BCX9930 - Paroxysmal Nocturnal Hemoglobinuria (PNH) | Phase 2 | Terminated | BioCryst Pharmaceuticals | 12 | 2021-12-06 | 2025-03-07 |
| BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants Not Receiving Other Complement Inhibitor Therapy Placebo, BCX9930 monotherapy - Paroxysmal Nocturnal Hemoglobinuria (PNH) | Phase 2 | Terminated | BioCryst Pharmaceuticals | 12 | 2021-10-26 | 2025-01-03 |
| 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 |
| A Long-Term Safety Study of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) BCX9930, Eculizumab, Ravulizumab - Paroxysmal Nocturnal Hemoglobinuria, PNH | Phase 2 | Terminated | BioCryst Pharmaceuticals | 19 | 2020-12-18 | 2025-04-13 |
| A Study to Evaluate the Safety, Pharmacokinetics and Antiviral Effects of Galidesivir in Yellow Fever or COVID-19 Galidesivir, Placebo - COVID-19, Yellow Fever | Phase 1 | Terminated | BioCryst Pharmaceuticals | 24 | 2020-04-09 | 2021-05-27 |
| First-in-Human Study of BCX9930 in Healthy Volunteers and Patients With PNH BCX9930, Placebo - Paroxysmal Nocturnal Hemoglobinuria | Phase 1 / Phase 2 | Completed | BioCryst Pharmaceuticals | 168 | 2020-03-03 | 2022-02-17 |
| Study to Evaluate the Efficacy and Safety of BCX7353 as an Oral Treatment for the Prevention of HAE Attacks in Japan BCX7353 capsules, Placebo oral capsule - Hereditary Angioedema, HAE | Phase 3 | Completed | BioCryst Pharmaceuticals | 19 | 2018-12-27 | 2024-07-19 |
| A Study to Evaluate the Single Dose Safety, Tolerability and Pharmacokinetics of IV BCX4430 galidesivir, placebo - Marburg Virus Disease | Phase 1 | Completed | BioCryst Pharmaceuticals | 32 | 2018-12-10 | 2021-07-23 |
| 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 |
| Study of BCX7353 as a Treatment for Attacks of Hereditary Angioedema BCX7353, Placebo - Hereditary Angioedema (HAE) | Phase 2 | Completed | BioCryst Pharmaceuticals | 58 | 2017-07-31 | 2021-04-01 |
| A Relative Bioavailability Study of Two Formulations of BCX7353 BCX7353 - Hereditary Angioedema | Phase 1 | Completed | BioCryst Pharmaceuticals | 24 | 2017-02-27 | 2020-01-27 |
| A Drug-Drug Interaction Study to Evaluate Drug Transporter Interactions BCX7353, Digoxin, BCX7353 + digoxin - Hereditary Angioedema | Phase 1 | Completed | BioCryst Pharmaceuticals | 54 | 2017-02-17 | 2017-10-26 |
| Efficacy and Safety of BCX7353 to Prevent Angioedema Attacks in Subjects With Hereditary Angioedema BCX7353, Placebo - Hereditary Angioedema (HAE) | Phase 2 | Completed | BioCryst Pharmaceuticals | 75 | 2016-08-01 | 2021-03-23 |
Showing 20 of 56. Sign in to see all of them.
Angioedemas, Hereditary (17) · Influenza, Human (11) · Hemoglobinuria, Paroxysmal (6) · Lymphoma (6) · Gout (4) · Hyperuricemia (4) · Leukemia (4) · Lymphoma, T-Cell, Cutaneous (4) · Mycosis Fungoides (4) · Leukemia, Lymphocytic, Chronic, B-Cell (3) · Sezary Syndrome (3) · Arthritis (2) · Burkitt Lymphoma (2) · Cough (2) · Headache (2)
berotralstat (12) · peramivir (10) · avoralstat (6) · forodesine (5) · ulodesine (4) · galidesivir (3) · Oseltamivir (2) · eculizumab (2) · ravulizumab (2) · Allopurinol (1) · Cyclosporine (1) · Digoxin (1) · Rosuvastatin Calcium (1) · peldesine (1)
United States (31) · United Kingdom (22) · France (12) · Germany (11) · South Africa (11) · Australia (9) · Canada (9) · Spain (9) · Austria (8) · Hungary (8) · Italy (8) · North Macedonia (5) · Poland (5) · Israel (4) · New Zealand (4)
Alan Hollister, MD, PhD (2) · Alex Shalaurov, MD, PhD (2) · Farhad Ravandi, MD (2) · Francesco Turturro, MD (2) · Joanne Collier, MBChB, FFPM, Dip Stats (OU) (2) · Agnes Buzin, MD (1) · Alain Martel, MD (1) · Alan B Fleischer, Jr., MD (1) · Alberto Bosi, MD (1) · Aleksander Skotnicki, MD (1) · Alexander Gaiger, MD (1) · Allan Kelly, MD (1) · Anatoli Golenkov, MD (1) · Andre Goy, MD (1) · Andrei R Shustov, MD (1)
PMID 36326435, PMID 36408587, PMID 35182042, PMID 20427701, PMID 23111657, PMID 23985625, PMID 24948692, PMID 25115871, PMID 30044938, PMID 31291057, PMID 33098856, PMID 33247955, PMID 33448731, PMID 33866032, PMID 34161665, PMID 35820771, PMID 38006972, PMID 38020288, PMID 38445295, PMID 39899371, PMID 40490218, PMID 40716738
272201300017C-18-0-1 (2), 2025 (1)