15 terminated. 15 Phase 2, 5 Phase 1, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 15 | Terminated 15 |
| Phase 1 | 5 | Terminated 5 |
| Phase 3 | 1 | Terminated 1 |
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 |
|---|---|---|---|---|---|---|
| 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 |
| Conestat Alfa in the Prevention of Severe SARS-CoV-2 Infection in Hospitalized Patients With COVID-19 Conestat alfa - Coronavirus Infections | Phase 2 | Terminated | University Hospital, Basel, Switzerland | 80 | 2020-08-06 | 2021-11-09 |
| N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T) IB1001 - Ataxia Telangiectasia | Phase 2 | Terminated | IntraBio Inc | 17 | 2020-01-08 | 2026-03-25 |
| Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis - Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission | Phase 2 | Terminated | Roswell Park Cancer Institute | 31 | 2017-12-07 | 2025-07-04 |
| Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study Rimiducid, rivogenlecleucel - Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child | Phase 1 / Phase 2 | Terminated | Bellicum Pharmaceuticals | 187 | 2017-01-01 | 2023-09-26 |
| Safety, Tolerability, and Immunogenicity of One Dose of NDV 3A Vaccine in People With STAT3-Mutated Hyper-IgE Syndrome NDV-3A - Autosomal-dominant Hyper-IgE Syndrome | Phase 2 | Terminated | National Institute of Allergy and Infectious Diseases (NIAID) | 3 | 2016-11-17 | 2020-06-30 |
| Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant BPX-501 T cells, Rimiducid - Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child | Phase 1 / Phase 2 | Terminated | Bellicum Pharmaceuticals | 187 | 2014-04-01 | 2023-09-29 |
| Hematopoietic Stem Cell Transplantation (HSCT) for Children With SCID Utilizing Alemtuzumab, Plerixafor & Filgrastim Transplant Conditioning with Mobilization Only, Transplant Conditioning with Mobilization and Alemtuzumab - Severe Combined Immunodeficiency | Phase 2 | Terminated | University of California, San Francisco | 7 | 2010-08-01 | 2018-07-31 |
| Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients Promacta, blood drawing in patients with WAS, blood drawing in healthy controls - Wiskott-Aldrich Syndrome, Thrombocytopenia | Phase 2 | Terminated | Weill Medical College of Cornell University | 24 | 2009-06-01 | 2019-03-18 |
| Allogeneic SCT Of Pts With SCID And Other Primary Immunodeficiency Disorders Campath -1H, Fludarabine, Anti-CD45 - Severe Combined Immunodeficiency Disease, Severe Primary Immunodeficiency Disorder | Phase 1 / Phase 2 | Terminated | Baylor College of Medicine | 3 | 2007-10-01 | 2013-07-02 |
| Effect of Ranitidine on Hyper-IgE Recurrent Infection (Job's) Syndrome Ranitidine, Placebo - JOB's Syndrome, Hyper-IgE Recurrent Infection Syndrome | Phase 2 | Terminated | National Institute of Allergy and Infectious Diseases (NIAID) | 16 | 2007-09-01 | 2013-02-04 |
| Treatment for Growth Failure in Patients With X-Linked Severe Combined Immunodeficiency: Phase 2 Study of Insulin-Like Growth Factor-1 Increlex - Growth Failure, X-linked Severe Combined Immunodeficiency (XSCID) | Phase 1 / Phase 2 | Terminated | National Institute of Allergy and Infectious Diseases (NIAID) | 6 | 2007-06-01 | 2015-07-03 |
| Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells Cyclophosphamide Dose Level 1, Cyclophosphamide Dose Level 2, Cyclophosphamide Dose Level 3 - Stem Cell Transplantation, Bone Marrow Transplantation | Phase 2 | Terminated | Children's Hospital Los Angeles | 20 | 2007-02-01 | 2017-02-27 |
| SCID Bu/Flu/ATG Study With T Cell Depletion unrelated BM with T cell depletion, unrelated cord blood, haplo BM with T cell depletion - Severe Combined Immunodeficiency | Phase 1 / Phase 2 | Terminated | Neena Kapoor, M.D. | 9 | 2007-01-02 | 2017-09-18 |
| T-Cell Depletion and Stem Cell Transplant for Immune Deficiencies and Histiocytic Disorders Stem Cell Transplant, Myeloablative conditioning regimen - Hemophagocytic Lymphohistiocytosis, X-Linked Lymphoproliferative Disorders | Phase 2 / Phase 3 | Terminated | Masonic Cancer Center, University of Minnesota | 22 | 2000-09-01 | 2018-01-23 |
Anemia, Diamond-Blackfan (4) · Thalassemia (4) · Anemia, Aplastic (3) · Anemia, Sickle Cell (3) · Leukemia, Myeloid, Acute (3) · Lymphoma, Non-Hodgkin (3) · Myelodysplastic Syndromes (3) · Precursor Cell Lymphoblastic Leukemia-Lymphoma (3) · Wiskott-Aldrich Syndrome (3) · Anemia (2) · Angioedemas, Hereditary (2) · Cytopenia (2) · Fanconi Anemia (2) · Granulomatous Disease, Chronic (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (15) · Genetic Diseases, Inborn (14) · Immune System Diseases (14) · Immunologic Deficiency Syndromes (13) · Hemic and Lymphatic Diseases (8) · Hematologic Diseases (7) · Nutritional and Metabolic Diseases (7) · Leukocyte Disorders (6) · Metabolic Diseases (6) · DNA Repair-Deficiency Disorders (5) · Genetic Diseases, X-Linked (5) · Immunoproliferative Disorders (5) · Lymphatic Diseases (5) · Pathological Conditions, Signs and Symptoms (5)
Cyclophosphamide (3) · AP 1903 reagent (2) · Alemtuzumab (2) · fludarabine phosphate (2) · Blood Specimen Collection (1) · Busulfan (1) · Filgrastim (1) · IB1001 (1) · Peripheral Blood Stem Cell Transplantation (1) · Ranitidine (1) · Stem Cell Transplantation (1) · Transplantation Conditioning (1) · Whole-Body Irradiation (1) · acetylleucine (1) · conestat alfa (1)
National Institute of Allergy and Infectious Diseases (NIAID) (3) · Bellicum Pharmaceuticals (2) · Baylor College of Medicine (1) · Children's Hospital Los Angeles (1) · IntraBio Inc (1) · Masonic Cancer Center, University of Minnesota (1) · Neena Kapoor, M.D. (1) · Pharvaris Netherlands B.V. (1) · Roswell Park Cancer Institute (1) · University Hospital, Basel, Switzerland (1) · University of California, San Francisco (1) · Weill Medical College of Cornell University (1)
United States (11) · Italy (3) · United Kingdom (3) · Germany (2) · Austria (1) · Brazil (1) · Bulgaria (1) · Canada (1) · Ireland (1) · Mexico (1) · Poland (1) · Spain (1) · Switzerland (1)
Neena Kapoor, M.D. (2) · Alexandra Freeman, M.D. (1) · Angela Smith, MD (1) · Christopher C Dvorak, M.D. (1) · James B Bussel, MD (1) · Malcolm Brenner, MD (1) · Marc Riedl, MD (1) · Michael Osthoff, PD Dr. med. (1) · Philip McCarthy, MD (1) · Robert Krance, MD (1) · Suk S De Ravin, M.D. (1)
PMID 25459642, PMID 26224646, PMID 33397449, PMID 33482890, PMID 34349180, PMID 34620022, PMID 37965347, PMID 40739200, PMID 41865746