66 completed. 66 Phase 2, 19 Phase 1, 11 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 66 | Completed 66 |
| Phase 1 | 19 | Completed 19 |
| Phase 3 | 11 | Completed 11 |
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 |
|---|---|---|---|---|---|---|
| Leniolisib for Immune Dysregulation in PIDs Leniolisib - Primary Immunodeficiency Disorders (PIDs) | Phase 2 | Completed | Pharming Technologies B.V. | 13 | 2024-10-21 | 2026-10-19 |
| 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 |
| A Study About How TAK-881 is Processed by the Body and Side Effects in People With Primary Immunodeficiency Diseases TAK-881, HYQVIA - Primary Immunodeficiency Diseases (PID) | Phase 2 / Phase 3 | Completed | Takeda | 65 | 2023-10-24 | 2026-09-30 |
| 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 |
| 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 |
| 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 |
| Effects of Vitamin B3 in Patients With Ataxia Telangiectasia Vitamin B3 - Ataxia Telangiectasia, ATM Gene Mutation | Phase 2 | Completed | Radboud University Medical Center | 24 | 2019-03-18 | 2022-07-25 |
| 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 |
| 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 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 |
| 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 |
| 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 |
| CAMB/MAT2203 in Patients With Mucocutaneous Candidiasis Amphotericin B - Candidiasis, Chronic Mucocutaneous | Phase 2 | Completed | Matinas BioPharma Nanotechnologies, Inc. | 4 | 2016-09-27 | 2024-08-07 |
| 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 |
| 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 |
| 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 |
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Angioedemas, Hereditary (21) · Severe Combined Immunodeficiency (10) · Wiskott-Aldrich Syndrome (10) · Ataxia Telangiectasia (5) · Immunologic Deficiency Syndromes (5) · Lymphohistiocytosis, Hemophagocytic (5) · Granulomatous Disease, Chronic (4) · Hyper-IgM Immunodeficiency Syndrome (4) · Infections (4) · Lymphoproliferative Disorders (4) · Anemia, Diamond-Blackfan (3) · Anemia, Sickle Cell (3) · Angioedema (3) · Bone Marrow Failure Disorders (3)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (66) · Immune System Diseases (65) · Genetic Diseases, Inborn (64) · Immunologic Deficiency Syndromes (61) · Cardiovascular Diseases (29) · Vascular Diseases (27) · Skin and Connective Tissue Diseases (26) · Skin Diseases (24) · Hereditary Complement Deficiency Diseases (21) · Hypersensitivity (21) · Hypersensitivity, Immediate (21) · Skin Diseases, Vascular (21) · Urticaria (21) · Hemic and Lymphatic Diseases (19)
Complement C1 Inhibitor Protein (7) · gamma-Globulins (6) · fludarabine (5) · Busulfan (4) · SERPING1 protein, human (4) · conestat alfa (4) · fludarabine phosphate (4) · Antilymphocyte Serum (3) · Melphalan (3) · Mycophenolic Acid (3) · Solutions (3) · Stem Cell Transplantation (3) · berotralstat (3) · thymoglobulin (3) · Alemtuzumab (2)
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PMID 36326435, PMID 36408587, PMID 25295500, PMID 30261899, PMID 30626238, PMID 31268374, PMID 34036490, PMID 35075289, PMID 12702512, PMID 16763896, PMID 16934683, PMID 17284531, PMID 18035553, PMID 18155964, PMID 18218852, PMID 18424759, PMID 18557726, PMID 18723429, PMID 19066739, PMID 19179314, PMID 19633200, PMID 19652199, PMID 19767078, PMID 19841031, PMID 20236866, PMID 20674826, PMID 20920772, PMID 20978268, PMID 21424824, PMID 22144182, PMID 22184407, PMID 22875082, PMID 22927201, PMID 23535096, PMID 23878046, PMID 23987198, PMID 24236946, PMID 24712435, PMID 25656498, PMID 26171584, PMID 26187864, PMID 26319134, PMID 26419363, PMID 26433381, PMID 26547562, PMID 26851119, PMID 27582171, PMID 27613250, PMID 27931305, PMID 28284978
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