66 completed, 16 recruiting, 15 terminated, 12 active not recruiting. 128 Phase 2, 48 Phase 1, 15 Phase 3.
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 |
|---|---|---|---|---|---|---|
| SCID-X1 Gene Therapy Via Intravenous Lentiviral (Ivlv-X1) Injection Direct intravenous injection of ivlv-X1 lentiviral vector - SCID, X-Linked | Phase 1 / Phase 2 | Not yet recruiting | Shenzhen Geno-Immune Medical Institute | 10 | 2027-06-30 | 2026-08-27 |
| Naive T Cell Deplete Grafts for GVHD Prevention in Non-Malignant Diseases ALLOGENEIC CD34+ ENRICHED AND CD45RA- DEPLETED PBSCs - Bone Marrow Failure, Hemoglobinopathies | Phase 2 | Not yet recruiting | Fred Hutchinson Cancer Center | 40 | 2026-09-01 | 2026-06-22 |
| Safety and Efficacy of Mutation-targeted Precision Genetic Therapy for Ataxia-Telangiectasia (A-T) Antisense oligonucleotide targeting the ATM gene - Ataxia Telangiectasia | Phase 1 / Phase 2 | Recruiting | Timothy Yu | 10 | 2026-08-01 | 2026-05-28 |
| Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD) Hematopoietic stem cell transplant (HSCT) - Common Variable Immunodeficiency (CVID), Primary Immune Regulatory Disorder | Phase 2 | Recruiting | Paul Szabolcs | 25 | 2026-05-04 | 2026-05-15 |
| An Phase Ib/II Clinical Trial of TCC1727 Combination Therapy in Advanced Solid Tumors TCC1727 tablet 90mg, TCC1727 tablet 120mg, TCC1727 tablet 160mg - Solid Cancers, NSCLC (Advanced Non-small Cell Lung Cancer) | Phase 1 / Phase 2 | Recruiting | Beijing Tide Pharmaceutical Co., Ltd | 266 | 2025-12-03 | 2026-01-28 |
| Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and Sirolimus Sirolimus, Busulfan, Horse -Anti-thymocyte - Severe Combined Immune Deficiency (SCID) | Phase 1 / Phase 2 | Withdrawn | National Institute of Allergy and Infectious Diseases (NIAID) | 2025-11-26 | 2025-12-01 | |
| Improving Deceased-Donor Kidney Transplant Outcomes Via a Single Intragraft Injection of C1 Esterase Inhibitor (IMPROVE TRIAL) Berinert, Placebo for Berinert - Kidney Transplant | Phase 2 | Recruiting | National Institute of Allergy and Infectious Diseases (NIAID) | 180 | 2025-09-22 | 2026-02-27 |
| Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy Plerixafor, Filgrastim, Palifermin - X-linked Severe Combined Immunodeficiency, X-SCID | Phase 1 / Phase 2 | Enrolling by invitation | National Institute of Allergy and Infectious Diseases (NIAID) | 18 | 2025-05-09 | 2026-10-13 |
| Oral Deucrictibant for Prophylactic and Acute Treatment in Hereditary Angioedema Patients Deucrictibant XR tablet, Placebo comparator to XR tablet, Deucrictibant 20 mg capsule - Hereditary Angioedema (HAE), Angioedema | Phase 1 / Phase 2 | Recruiting | Institute for Asthma and Allergy | 10 | 2025-03-10 | 2025-07-02 |
| 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 |
| 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 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 and Efficacy Study of Transplantation of Autologous CD34+ Cells Transduced With the G2ARTE Lentiviral Vector Expressing the DCLRE1C cDNA in Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency Patients (ARTEGENE) ARTEGENE drug product - Artemis (DCLRE1C ) Deficient Severe Combined Immunodeficiency | Phase 1 / Phase 2 | Recruiting | Assistance Publique - Hôpitaux de Paris | 7 | 2023-07-19 | 2026-03-27 |
| 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 |
| Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema deucrictibant - Hereditary Angioedema, Hereditary Angioedema Type I | Phase 2 / Phase 3 | Enrolling by invitation | Pharvaris Netherlands B.V. | 150 | 2022-12-28 | 2026-07-21 |
| 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 |
| 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 |
| 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 |
Showing 20 of 128. Sign in to see all of them.
Angioedemas, Hereditary (35) · Severe Combined Immunodeficiency (20) · Wiskott-Aldrich Syndrome (18) · Anemia, Diamond-Blackfan (10) · Ataxia Telangiectasia (10) · Lymphohistiocytosis, Hemophagocytic (10) · X-Linked Combined Immunodeficiency Diseases (10) · Anemia, Sickle Cell (9) · Granulomatous Disease, Chronic (9) · Immunologic Deficiency Syndromes (9) · Anemia, Aplastic (8) · Bone Marrow Failure Disorders (7) · Leukemia, Myeloid, Acute (7) · Precursor Cell Lymphoblastic Leukemia-Lymphoma (7)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (128) · Immune System Diseases (125) · Genetic Diseases, Inborn (124) · Immunologic Deficiency Syndromes (119) · Cardiovascular Diseases (50) · Vascular Diseases (47) · Skin and Connective Tissue Diseases (43) · Skin Diseases (41) · Nutritional and Metabolic Diseases (40) · Hemic and Lymphatic Diseases (39) · Metabolic Diseases (37) · Hematologic Diseases (36) · Hereditary Complement Deficiency Diseases (35) · Hypersensitivity, Immediate (35)
Busulfan (14) · fludarabine (10) · Complement C1 Inhibitor Protein (9) · fludarabine phosphate (9) · Alemtuzumab (8) · Cyclophosphamide (7) · Melphalan (6) · Whole-Body Irradiation (6) · gamma-Globulins (6) · SERPING1 protein, human (5) · Stem Cell Transplantation (5) · conestat alfa (5) · Antilymphocyte Serum (4) · Mycophenolic Acid (4) · Sirolimus (4)
National Institute of Allergy and Infectious Diseases (NIAID) (13) · Baxalta now part of Shire (6) · Shire (6) · BioCryst Pharmaceuticals (5) · Pharming Technologies B.V. (5) · New York Medical College (4) · Bellicum Pharmaceuticals (3) · David Williams (3) · Federal Research Institute of Pediatric Hematology, Oncology and Immunology (3) · Genethon (3) · Paul Szabolcs (3) · Pharvaris Netherlands B.V. (3) · University of California, San Francisco (3) · Assistance Publique - Hôpitaux de Paris (2) · Astria Therapeutics, Inc. (2)
United States (87) · Germany (21) · United Kingdom (19) · Italy (15) · Canada (13) · France (12) · Hungary (11) · Netherlands (11) · Poland (11) · Spain (10) · Australia (9) · Czechia (9) · Austria (7) · Israel (7) · North Macedonia (7)
Paul Szabolcs, MD (4) · Suk S De Ravin, M.D. (3) · Alexei Maschan, Professor (2) · Angela Smith, MD (2) · Anurag Relan, MD (2) · Diane George, MD (2) · Mitchell Cairo, MD (2) · Mitchell S Cairo, MD (2) · Neena Kapoor, M.D. (2) · Alain Fischer, MD, PhD (1) · Alexandra Freeman, M.D. (1)
PMID 36326435, PMID 35075289, PMID 36408587, PMID 25295500, PMID 30261899, PMID 30626238, PMID 31268374, PMID 34036490, 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 25459642, PMID 25656498, PMID 26171584, PMID 26187864, PMID 26224646, PMID 26319134, PMID 26419363, PMID 26433381, PMID 26547562, PMID 26851119, PMID 27582171, PMID 27613250
2009 (2), 2019 (2), 2023 (2), 2004 (1), 2008 (1), 2014 (1), 2018 (1), K12HD043494 (1), P01HL053749 (1), P30CA015704 (1), R01AI047040 (1), R01AI054843 (1), R56AI047040 (1), U01AI125050 (1), U10HL069294 (1)