12 active not recruiting. 12 Phase 2, 8 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 12 | Active not recruiting 12 |
| Phase 1 | 8 | Active not recruiting 8 |
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 Phase 2 in Adult Subjects With Hereditary Angioedema BW-20805 - Hereditary Angioedema (HAE) | Phase 2 | Active not recruiting | Shanghai Argo Biopharmaceutical Co., Ltd. | 25 | 2025-02-28 | 2026-04-17 |
| 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 |
| 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 |
| 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 |
| Haplocompatible Transplant Using TCRα/β Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID) Anti-thymocyte globulin (rabbit), Busulfan, Fludarabine - Severe Combined Immunodeficiency | Phase 1 / Phase 2 | Active not recruiting | St. Jude Children's Research Hospital | 4 | 2021-09-02 | 2026-09-16 |
| NAD Supplementation to Prevent Progressive Neurological Disease in Ataxia Telangiectasia Nicotinamide ribonucleoside - Ataxia Telangiectasia | Phase 2 | Active not recruiting | University Hospital, Akershus | 13 | 2019-06-05 | 2022-08-17 |
| Antiviral Cellular Therapy for Enhancing T-cell Reconstitution Before or After Hematopoietic Stem Cell Transplantation Virus Specific T-cell (VST) infusion - Cytomegalovirus Infections, Adenovirus Infection | Phase 1 / Phase 2 | Active not recruiting | Pediatric Transplantation & Cellular Therapy Consortium | 52 | 2018-06-20 | 2025-05-11 |
| Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome Autologous CD34+ cells transduced with WASP lentiviral vector - Wiskott-Aldrich Syndrome | Phase 1 / Phase 2 | Active not recruiting | Genethon | 10 | 2014-09-01 | 2021-06-03 |
| Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant BPX-501 T cells, Rimiducid - Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child | Phase 1 / Phase 2 | Active not recruiting | Bellicum Pharmaceuticals | 120 | 2014-04-01 | 2022-07-12 |
| CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation CD34 Stem Cell Selection Therapy - Bone Marrow Failure Syndrome, Severe Aplastic Anemia | Phase 1 / Phase 2 | Active not recruiting | Diane George | 37 | 2013-03-01 | 2025-08-14 |
| Pilot Study of Reduced-Intensity Hematopoietic Stem Cell Transplant of DOCK8 Deficiency Reduced-intensity hematopoietic stem cell, Fludarabine(Fludara, Berlex Laboratories), Cyclophosphamide(CTX, Cytoxan) - DOCK8 Deficiency | Phase 2 | Active not recruiting | National Cancer Institute (NCI) | 76 | 2010-10-05 | 2026-10-27 |
Angioedemas, Hereditary (5) · Anemia, Aplastic (2) · Acquired Immunodeficiency Syndrome (1) · Adenoviridae Infections (1) · Anemia, Diamond-Blackfan (1) · Anemia, Sickle Cell (1) · Angioedema (1) · Ataxia Telangiectasia (1) · Bone Marrow Failure Disorders (1) · Cytomegalovirus Infections (1) · Cytopenia (1) · Epstein-Barr Virus Infections (1) · Graft vs Host Disease (1) · Hematologic Neoplasms (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (12) · Genetic Diseases, Inborn (12) · Immune System Diseases (12) · Immunologic Deficiency Syndromes (11) · Cardiovascular Diseases (6) · Vascular Diseases (6) · Hereditary Complement Deficiency Diseases (5) · Hypersensitivity (5) · Hypersensitivity, Immediate (5) · Skin Diseases (5) · Skin Diseases, Vascular (5) · Skin and Connective Tissue Diseases (5) · Urticaria (5) · Angioedema (4)
Busulfan (2) · AP 1903 reagent (1) · Antilymphocyte Serum (1) · Cyclophosphamide (1) · RNA, Small Interfering (1) · Sodium Chloride (1) · Thiotepa (1) · Whole-Body Irradiation (1) · fludarabine (1) · fludarabine phosphate (1) · nicotinamide-beta-riboside (1) · thymoglobulin (1)
ADARx Pharmaceuticals, Inc. (1) · Astria Therapeutics, Inc. (1) · Bellicum Pharmaceuticals (1) · BioMarin Pharmaceutical (1) · Diane George (1) · Genethon (1) · Intellia Therapeutics (1) · National Cancer Institute (NCI) (1) · Pediatric Transplantation & Cellular Therapy Consortium (1) · Shanghai Argo Biopharmaceutical Co., Ltd. (1) · St. Jude Children's Research Hospital (1) · University Hospital, Akershus (1)
United States (8) · Australia (3) · Germany (3) · United Kingdom (3) · France (2) · Poland (2) · Spain (2) · Bulgaria (1) · Canada (1) · China (1) · Czechia (1) · Italy (1) · Netherlands (1) · New Zealand (1) · Norway (1)
Corina E Gonzalez, M.D. (1) · Diane George, MD (1) · Ewelina Mamcarz, MD (1) · Hilde L Nilsen (1) · Lauge Farnaes, MD (1) · Markus Magerl, Doctor (1) · Michael Keller, MD (1) · Michael Pulsipher, MD (1)
PMID 30720689, PMID 35075289, PMID 38294975, PMID 39445704
2023 (1)