50 completed. 50 Phase 1, 19 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 50 | Completed 50 |
| Phase 2 | 19 | Completed 19 |
Follow Primary Immunodeficiency Diseases: new trials by email Literature Review of Primary Immunodeficiency Diseases trials Research Report on Primary Immunodeficiency Diseases
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 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 |
| A Study of STAR-0215 in Healthy Adult Participants STAR-0215 (SC), Placebo (SC), STAR-0215 (IV) - Hereditary Angioedema | Phase 1 | Completed | Astria Therapeutics, Inc. | 41 | 2022-07-27 | 2024-01-11 |
| Evaluation of Safety, Tolerability and Immune Responses of Ebola-S and Marburg Vaccines in Healthy Adults cAd3-Marburg, cAd3-EBO-S - Ebola Virus Disease, Marburg Virus Disease | Phase 1 | Completed | Albert B. Sabin Vaccine Institute | 32 | 2021-01-06 | 2022-08-03 |
| Phase 1 Crossover Study in Healthy Subjects to Evaluate the PK Profile of KVD824 Following Single and Multiple Doses of Modified Release (MR) Formulations KVD824 Prototype 1 modified-release tablet, KVD824 Prototype 2 modified-release tablet, KVD824 Immediate-Release Capsule - Hereditary Angioedema | Phase 1 | Completed | KalVista Pharmaceuticals, Ltd. | 37 | 2020-05-19 | 2021-11-12 |
| Safety and Pharmacokinetics of GNR-038 in Healthy Volunteers GNR-038, 25 МЕ/kg, GNR-038, 50 МЕ/kg, GNR-038, 100 МЕ/kg - Hereditary Angioedema | Phase 1 | Completed | AO GENERIUM | 36 | 2020-03-10 | 2020-09-30 |
| Study to Test How Well Patients With Advanced Solid Tumors Respond to Treatment With the Elimusertib in Combination With Pembrolizumab, to Find the Optimal Dose for Patients, How the Drug is Tolerated and the Way the Body Absorbs, Distributes and Discharges the Drug Elimusertib (BAY1895344), Pembrolizumab (Keytruda®) - Advanced Solid Tumors | Phase 1 | Completed | Bayer | 56 | 2019-09-30 | 2024-04-02 |
| A Single and Multiple Doses Safety, Tolerability, Pharmacokinetics and Food Effect Study of KVD824 in Healthy Volunteers KVD824, Placebo to KVD824 - Hereditary Angioedema | Phase 1 | Completed | KalVista Pharmaceuticals, Ltd. | 108 | 2019-02-12 | 2022-02-17 |
| Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation Thiotepa--single daily dose, Thiotepa--escalated dose - Bone Marrow Failure Syndrome, Thalassemia | Phase 1 / Phase 2 | Completed | University of Florida | 6 | 2018-06-15 | 2023-09-15 |
| A Single Dose Safety, Tolerability, Pharmacokinetic and Food Effect Study of KVD900 (Sebetralstat) in Healthy Volunteers KVD900, Placebo to KVD900 - Hereditary Angioedema | Phase 1 | Completed | KalVista Pharmaceuticals, Ltd. | 84 | 2018-01-04 | 2025-04-29 |
| Bilateral Orthotopic Lung Transplant - Bone Marrow Transplant CD3/CD19 neg allogeneic BMT - Primary Immunodeficiency, PID | Phase 1 / Phase 2 | Completed | National Institute of Allergy and Infectious Diseases (NIAID) | 5 | 2017-12-01 | 2025-07-22 |
| Rapamycin Treatment for Activated Phosphoinositide 3-Kinase δ Syndrome Rapamycin - Activated PI3K-delta Syndrome, Immunodeficiency Primary | Phase 1 / Phase 2 | Completed | Children's Hospital of Fudan University | 30 | 2017-12-01 | 2024-07-09 |
| A Study of CDX-1140 (CD40) as Monotherapy or in Combination in Patients With Advanced Malignancies CDX-1140, CDX-301, pembrolizumab - Melanoma, Non-small Cell Lung Cancer | Phase 1 | Completed | Celldex Therapeutics | 132 | 2017-12-01 | 2024-03-28 |
| 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 |
| Magnesium Supplementation in People With XMEN Syndrome Magnesium L-threonate, Placebo, Intravenous (IV) magnesium sulfate (MgSO4) - XMEN | Phase 1 / Phase 2 | Completed | National Institute of Allergy and Infectious Diseases (NIAID) | 8 | 2016-05-17 | 2022-03-02 |
| An Open-label Drug-Drug Interaction Study to Evaluate the Effect of BCX7353 on Cytochrome P450 Enzyme Activity Using Probe Substrates BCX7353 and probes - Hereditary Angioedema | Phase 1 | Completed | BioCryst Pharmaceuticals | 20 | 2016-03-01 | 2017-01-31 |
| Phase 1 Study to Assess the Safety, Tolerability, and Pharmacokinetics of Recombinant Human C1 Esterase Inhibitor in Healthy Adult Subjects Recombinant human C1 esterase inhibitor, Placebo, SHP623 - Hereditary Angioedema (HAE) | Phase 1 | Completed | Shire | 48 | 2016-02-19 | 2021-06-03 |
| Pilot Study Related to the Effect of Clopidogrel on Plasmatic Soluble CD40 Ligand During Systemic Lupus Erythematous Treatment by clopidogrel - Systemic Lupus Erythematous | Phase 1 / Phase 2 | Completed | University Hospital, Bordeaux | 18 | 2015-08-19 | 2026-05-14 |
| First-in-Human Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of BCX7353 in Healthy Western and Japanese Volunteers BCX7353, Placebo to match BCX7353 - Hereditary Angioedema | Phase 1 | Completed | BioCryst Pharmaceuticals | 122 | 2015-05-01 | 2016-01-13 |
| Study to Determine How BCX4161 is Metabolized and Eliminated by the Body BCX4161 - Hereditary Angioedema | Phase 1 | Completed | BioCryst Pharmaceuticals | 7 | 2014-08-01 | 2014-10-29 |
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Angioedemas, Hereditary (18) · Severe Combined Immunodeficiency (10) · Wiskott-Aldrich Syndrome (10) · Mucopolysaccharidosis I (6) · Immunologic Deficiency Syndromes (5) · X-Linked Combined Immunodeficiency Diseases (5) · Anemia, Diamond-Blackfan (4) · Angioedema (4) · Granulomatous Disease, Chronic (4) · Anemia, Aplastic (3) · Anemia, Sickle Cell (3) · Genetic Diseases, Inborn (3) · Hyper-IgM Immunodeficiency Syndrome (3) · Immune System Diseases (3)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (50) · Genetic Diseases, Inborn (47) · Immune System Diseases (47) · Immunologic Deficiency Syndromes (45) · Skin and Connective Tissue Diseases (27) · Skin Diseases (20) · Cardiovascular Diseases (18) · Genetic Diseases, X-Linked (18) · Hematologic Diseases (18) · Hemic and Lymphatic Diseases (18) · Hereditary Complement Deficiency Diseases (18) · Vascular Diseases (18) · Skin Diseases, Vascular (17) · Hypersensitivity (16)
berotralstat (4) · fludarabine (4) · Busulfan (3) · Alemtuzumab (2) · Cyclosporine (2) · Genetic Engineering (2) · Hematopoietic Stem Cell Transplantation (2) · Melphalan (2) · Thiotepa (2) · avoralstat (2) · lanadelumab (2) · pembrolizumab (2) · Abatacept (1) · BAY 1895344 (1) · Complement C1 Inhibitor Protein (1)
BioCryst Pharmaceuticals (6) · Shire (5) · National Institute of Allergy and Infectious Diseases (NIAID) (4) · KalVista Pharmaceuticals, Ltd. (3) · Astria Therapeutics, Inc. (2) · David Williams (2) · Emory University (2) · Genethon (2) · St. Jude Children's Research Hospital (2) · AO GENERIUM (1) · Albert B. Sabin Vaccine Institute (1) · Assistance Publique - Hôpitaux de Paris (1) · Baxalta now part of Shire (1) · Bayer (1) · Celldex Therapeutics (1)
United States (30) · United Kingdom (13) · France (3) · Germany (2) · Italy (2) · Bulgaria (1) · Canada (1) · China (1) · Czechia (1) · Jordan (1) · Russia (1) · South Korea (1) · Spain (1) · Sweden (1) · Switzerland (1)
Joanne Collier, MBChB, FFPM, Dip Stats (OU) (2) · Kimberly Kasow, DO (2) · Alain Fischer, MD, PhD (1) · Biljana Horn, MD (1) · Bordignon Claudio, MD (1) · Claire Booth, Dr (1) · David Hoover, MD (1) · Firas Almazedi, MBChB, Msc, CPI (1) · Hisham Abdel-Azim, MD (1) · Hyo Seop Ahn, Ph. D (1) · Jan Andersson, MD/PhD (1) · Jennifer Whangbo, M.D. (1) · Jennifer Whangbo, MD (1) · Jinqiao Sun, Ph.D.,M.D (1)
PMID 25295500, PMID 30261899, PMID 35075289, PMID 1538192, PMID 15577929, PMID 16375711, PMID 18218852, PMID 19179314, PMID 19633200, PMID 19652199, PMID 21725047, PMID 22184407, PMID 24296492, PMID 24980392, PMID 25426834, PMID 26319134, PMID 26419363, PMID 28225674, PMID 28716862, PMID 29635077, PMID 30981783, PMID 33152483, PMID 33974366, PMID 34655400, PMID 35086692, PMID 37244942, PMID 37478401, PMID 38446574, PMID 40158724, PMID 41794053, PMID 42777242, PMID 8237108
2009 (2), 2008 (1), 2014 (1), 2018 (1), P01HL036444 (1), P30CA015704 (1), U01AI125050 (1)