16 recruiting. 16 Phase 2, 8 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 16 | Recruiting 16 |
| Phase 1 | 8 | 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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
| 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 |
| 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 |
| TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Primary Immunodeficiency and Inherited Metabolic Disorders in Children Haploidentical Hematopoietic Cell Transplantation - Primary Immune Deficiency Disorders, Metabolic Disease | Phase 2 | Recruiting | Johns Hopkins All Children's Hospital | 17 | 2020-07-22 | 2026-07-01 |
| EBV-specific Cytotoxic T-lymphocytes (CTLs) for Refractory EBV Infection cytotoxic t-lymphocytes - Epstein-Barr Virus Infections, Primary Immune Deficiency Disorder | Phase 2 | Recruiting | New York Medical College | 20 | 2020-07-07 | 2026-10-23 |
| Treatment of Refractory BK Infections With Related Donor BK Specific Cytotoxic T-cells (CTLs) BK CTL - Viral Infection, Primary Immune Deficiency Disorder | Phase 1 / Phase 2 | Recruiting | New York Medical College | 40 | 2020-07-01 | 2026-04-15 |
| Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures Alemtuzumab, Fludarabine, Melphalan - Primary Immune Deficiency Disorder, Immune Deficiency Disease | Phase 2 | Recruiting | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | 27 | 2020-02-12 | 2025-11-28 |
| Adenovirus-specific Cytotoxic T-lymphocytes for Refractory Adenovirus Infection cytotoxic t-lymphocytes - Adenovirus, Primary Immune Deficiency Disorder | Phase 2 | Recruiting | New York Medical College | 20 | 2018-11-01 | 2026-10-23 |
| Virus Specific Cytotoxic T-Lymphocytes (CTLs) for Refractory Cytomegalovirus (CMV) viral specific cytotoxic t-lymphocytes - Cytomegalovirus Infections, Primary Immune Deficiency Disorder | Phase 2 | Recruiting | New York Medical College | 20 | 2018-11-01 | 2026-10-23 |
| Autologous Gene Therapy for Artemis-Deficient SCID AProArt-CD34, CliniMACS® CD34 Reagent System cell sorter device, Busulfan - Severe Combined Immunodeficiency | Phase 1 / Phase 2 | Recruiting | University of California, San Francisco | 24 | 2018-05-31 | 2026-02-13 |
| Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning autologous CD34+ cell transduced with G2SCID vector - Severe Combined Immunodeficiency, X Linked, Gene Therapy | Phase 1 / Phase 2 | Recruiting | David Williams | 12 | 2018-02-26 | 2025-12-11 |
| Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT Hydroxyurea, Alemtuzumab, Fludarabine - Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes | Phase 2 | Recruiting | Paul Szabolcs | 100 | 2014-02-04 | 2025-12-15 |
| Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID) Campath, Palifermin, Busulfan - X-linked Severe Combined Immunodeficiency (XSCID) | Phase 1 / Phase 2 | Recruiting | National Institute of Allergy and Infectious Diseases (NIAID) | 40 | 2012-09-25 | 2026-10-27 |
Severe Combined Immunodeficiency (4) · Angioedemas, Hereditary (2) · Ataxia Telangiectasia (2) · Dyskeratosis Congenita (2) · Granulomatous Disease, Chronic (2) · Immune Dysregulation, Polyendocrinopathy, Enteropathy, X-Linked Syndrome (2) · X-Linked Combined Immunodeficiency Diseases (2) · Adenoviridae Infections (1) · Adrenoleukodystrophy (1) · Anemia, Diamond-Blackfan (1) · Anemia, Sickle Cell (1) · Angioedema (1) · Arthritis, Juvenile (1) · Autoimmune Lymphoproliferative Syndrome (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (16) · Genetic Diseases, Inborn (16) · Immune System Diseases (16) · Immunologic Deficiency Syndromes (16) · Nutritional and Metabolic Diseases (8) · DNA Repair-Deficiency Disorders (7) · Metabolic Diseases (7) · Infant, Newborn, Diseases (6) · Cardiovascular Diseases (5) · Genetic Diseases, X-Linked (5) · Skin Diseases (5) · Skin and Connective Tissue Diseases (5) · Infections (4) · Vascular Diseases (4)
Alemtuzumab (3) · Busulfan (2) · Melphalan (2) · fludarabine (2) · Complement C1 Inhibitor Protein (1) · Cyclophosphamide (1) · Fibroblast Growth Factor 7 (1) · Hydroxyurea (1) · Mycophenolic Acid (1) · Stem Cell Transplantation (1) · Tacrolimus (1) · Thiotepa (1) · Whole-Body Irradiation (1) · fludarabine phosphate (1)
New York Medical College (4) · National Institute of Allergy and Infectious Diseases (NIAID) (2) · Paul Szabolcs (2) · Assistance Publique - Hôpitaux de Paris (1) · Beijing Tide Pharmaceutical Co., Ltd (1) · David Williams (1) · Institute for Asthma and Allergy (1) · Johns Hopkins All Children's Hospital (1) · Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins (1) · Timothy Yu (1) · University of California, San Francisco (1)
United States (14) · China (1) · France (1)
Mitchell Cairo, MD (2) · Mitchell S Cairo, MD (2) · Paul Szabolcs, MD (2) · Chantal Lagresle-Peyrou, MD (1) · Christelle Achkar, MD (1) · Deepak Chellapandian, MD (1) · Heather J Symons, MD, MHS (1) · Morton Cowan, MD (1) · Peter S Heeger, MD (1) · Sindhu Chandran, MBBS, MD (1) · Stanley Jordan, MD (1) · Suk S De Ravin, M.D. (1) · Sung-Yun Pai, MD (1) · Timothy Yu, MD, PhD (1)
PMID 32634238, PMID 32750127, PMID 33159275, PMID 35764638
2019 (1)