31 completed, 11 recruiting, 10 unknown, 6 terminated. 29 Phase 1, 29 Phase 2, 7 Not applicable, 3 Phase 3, 1 Early Phase 1.
Follow Severe Combined Immunodeficiency: new trials by email Literature Review of Severe Combined Immunodeficiency trials Research Report on Severe Combined Immunodeficiency
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 |
| Follow-up of the Cohort of Newborns Screened at Birth Using TREC Analysis Data collection - Severe Combined Immunodeficiencies (SCID) | Not yet recruiting | Nantes University Hospital | 450 | 2026-11-01 | 2026-07-15 | |
| 25BT056: Basket Study of Viral Cytotoxic T Lymphocytes Virus-Specific Cytotoxic T Lymphocytes (vCTLs) - EBV Viremia, CMV Viremia | Early Phase 1 | Not yet recruiting | Children's Hospital of Philadelphia | 35 | 2026-11-01 | 2026-10-22 |
| 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 |
| Targeting Real World Usage In Stroke Treatment Aneurysm Treatment with a Neurovascular Flow Diverter, Dissection Treatment with a Neurovascular Flow Diverter, Perforation Treatment with a Neurovascular Flow Diverter - Saccular and Fusiform Aneurysms, Pseudoaneurysm | Recruiting | Phenox GmbH | 5000 | 2026-01-29 | 2026-03-16 | |
| 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Sirolimus Sirolimus, Busulfan, Horse -Anti-thymocyte - Severe Combined Immune Deficiency (SCID) | Phase 1 / Phase 2 | Withdrawn | National Institute of Allergy and Infectious Diseases (NIAID) | 2025-11-26 | 2025-12-01 | |
| 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 |
| Study of Populations at Risk of Developing Chronic Hepatitis Linked to Chronic Enteric Virus Infection in Patients With Primary Immunodeficiency and Secondary Humoral Deficiency Plasma, urine and stool collection - Humoral Primary Immunodeficiencies (PIDs), Secondary Form of Humoral Immunodeficiencies | Completed | Assistance Publique - Hôpitaux de Paris | 200 | 2024-10-10 | 2026-09-24 | |
| 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 |
| The Experience of Screening for SCID SCID Screening - Severe Combined Immunodeficiency | Recruiting | King's College London | 90 | 2022-11-09 | 2022-12-14 | |
| Baby Detect : Genomic Newborn Screening Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1 | Completed | Centre Hospitalier Universitaire de Liege | 6824 | 2022-09-01 | 2025-08-12 | |
| Feasibility Study to Assess an Adapted Physical Activity Program in Children, Adolescents and Young Adults Requiring Hematopoietic Stem Cell Transplantation Physical activity program - Haematological Malignancy, Acute Leukemia | Not applicable | Completed | Centre Leon Berard | 15 | 2022-05-25 | 2024-08-21 |
| 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 |
| Safety and Early Efficacy Study of TBX-1400 in Patients With Severe Combined Immunodeficiency TBX-1400 - Severe Combined Immunodeficiency | Phase 1 | Unknown | Taiga Biotechnologies, Inc. | 8 | 2021-08-01 | 2020-10-08 |
| Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC data collection - Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes | Recruiting | Paul Szabolcs | 50 | 2020-08-20 | 2026-01-13 | |
| Retroviral Insertion Site Methodology Study Strimvelis - Immune System Diseases | Phase 4 | Withdrawn | Fondazione Telethon | 2020-06-25 | 2024-01-29 | |
| Gene Therapy for X Linked Severe Combined Immunodeficiency Lentiviral Vector Gene Therapy - Gene Therapy | Not applicable | Unknown | Children's Hospital of Chongqing Medical University | 10 | 2020-05-01 | 2020-03-27 |
| Study Through Imaging of Visceral Lymphoid Organs in Patients With SCID Who Have Recieved Bone Marrow Allograft Severe Combined Immunodeficiency | Completed | Association Pour l'Aide a la Recherche en Immunologie Pediatrique | 18 | 2020-01-13 | 2021-07-22 | |
| Multi-center Clinical Study of Cord Blood Stem Cell Transplantation for SCID cord blood stem cell transplantation - Severe Combined Immunodeficiency Disease | Unknown | Children's Hospital of Fudan University | 50 | 2019-12-01 | 2020-01-28 | |
| An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV) - Adenosine Deaminase Deficiency, Severe Combined Immunodeficiency (SCID) | Enrolling by invitation | University of California, Los Angeles | 70 | 2019-09-26 | 2025-12-18 |
Showing 20 of 78. Sign in to see all of them.
X-Linked Combined Immunodeficiency Diseases (28) · Severe combined immunodeficiency due to adenosine deaminase deficiency (13) · Granulomatous Disease, Chronic (12) · Primary Immunodeficiency Diseases (11) · Wiskott-Aldrich Syndrome (11) · Immunologic Deficiency Syndromes (10) · Anemia, Diamond-Blackfan (8) · Anemia, Sickle Cell (8) · Common Variable Immunodeficiency (8) · Lymphohistiocytosis, Hemophagocytic (8) · Mucopolysaccharidosis I (8) · Chediak-Higashi Syndrome (6) · Adrenoleukodystrophy (5) · Anemia, Aplastic (5)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (78) · Infant, Newborn, Diseases (78) · Genetic Diseases, Inborn (76) · Immune System Diseases (73) · Immunologic Deficiency Syndromes (68) · Primary Immunodeficiency Diseases (67) · Nutritional and Metabolic Diseases (60) · Metabolic Diseases (59) · DNA Repair-Deficiency Disorders (56) · Genetic Diseases, X-Linked (42) · Hemic and Lymphatic Diseases (24) · Hematologic Diseases (22) · Pathological Conditions, Signs and Symptoms (16) · Anemia (15)
Busulfan (12) · Alemtuzumab (7) · fludarabine (7) · fludarabine phosphate (6) · Melphalan (5) · Stem Cell Transplantation (4) · Thiotepa (4) · Antilymphocyte Serum (3) · Cyclophosphamide (3) · Fibroblast Growth Factor 7 (3) · Filgrastim (3) · Whole-Body Irradiation (3) · Cyclosporine (2) · Data Collection (2) · Genetic Engineering (2)
National Institute of Allergy and Infectious Diseases (NIAID) (10) · National Human Genome Research Institute (NHGRI) (6) · Paul Szabolcs (4) · St. Jude Children's Research Hospital (4) · University of California, San Francisco (4) · Assistance Publique - Hôpitaux de Paris (3) · Great Ormond Street Hospital for Children NHS Foundation Trust (3) · National Heart, Lung, and Blood Institute (NHLBI) (3) · Centre Leon Berard (2) · Children's Hospital of Fudan University (2) · David Williams (2) · Nantes University Hospital (2) · Phenox GmbH (2) · University of Florida (2) · Association Pour l'Aide a la Recherche en Immunologie Pediatrique (1)
United States (51) · France (10) · United Kingdom (6) · China (5) · Canada (3) · Belgium (2) · Germany (2) · Italy (2) · Argentina (1) · Bulgaria (1) · Israel (1) · Poland (1) · Romania (1) · Russia (1) · Slovakia (1)
Paul Szabolcs, MD (4) · Suk S De Ravin, M.D. (3) · Claire Booth, Dr (2) · Donald B. Kohn, M.D. (2) · Elie Haddad, MD, PhD (2) · Morton J. Cowan, MD (2) · Robert A Sokolic, M.D. (2) · Stephen Gottschalk, MD (2) · Adrian Thrasher, Prof (1) · Alain Fischer, MD, PhD (1) · Biljana Horn, MD (1) · Bordignon Claudio, MD (1)
PMID 25075835, PMID 25295500, PMID 30261899, PMID 20004776, PMID 21725047, PMID 23818196, PMID 24139498, PMID 24290292, PMID 24331379, PMID 27262745, PMID 30193840, PMID 35671392, PMID 41289158, PMID 11222394, PMID 11520813, PMID 12598139, PMID 15696101, PMID 16219547, PMID 16449616, PMID 17697973, PMID 18023361, PMID 18162223, PMID 18218852, PMID 18723429, PMID 18974988, PMID 19179314, PMID 19633200, PMID 19652199, PMID 19786112, PMID 19996402, PMID 20489056, PMID 20673987, PMID 21479529, PMID 21861757, PMID 22184407, PMID 22224700, PMID 22308292, PMID 23832379, PMID 24113871, PMID 24296492, PMID 27888015, PMID 28346229, PMID 29021228, PMID 29728406, PMID 30154114, PMID 30194989, PMID 30251145, PMID 30946917, PMID 30995372, PMID 31315600
2008 (1), 2019 (1), P01HL036444 (1), P01HL053749 (1), P30CA015704 (1), R03HD060311 (1)