8 completed, 8 unknown, 4 recruiting, 2 enrolling by invitation. 14 Phase 1, 10 Phase 2, 2 Not applicable.
Follow X-Linked Combined Immunodeficiency Diseases: new trials by email Literature Review of X-Linked Combined Immunodeficiency Diseases trials Research Report on X-Linked Combined 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 |
| 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 | |
| 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 |
| 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 |
| 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 | |
| Lentiviral Gene Therapy for X-linked Severe Combined Immunodeficiency Lentiviral vector transduced CD34+ cells - Severe Combined Immunodeficiency, X-Linked | Phase 1 | Unknown | Great Ormond Street Hospital for Children NHS Foundation Trust | 5 | 2018-12-21 | 2023-10-12 |
| Early Check: Expanded Screening in Newborns Confirmatory Testing - Spinal Muscular Atrophy, Fragile X Syndrome | Active not recruiting | RTI International | 30000 | 2018-10-15 | 2026-07-01 | |
| 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 |
| Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants CL20-i4-EF1α-hγc-OPT, Busulfan, CliniMacs - Severe Combined Immunodeficiency Disease, X-linked | Phase 1 / Phase 2 | Suspended | St. Jude Children's Research Hospital | 28 | 2016-08-17 | 2025-11-10 |
| Diversion-p64 Post Market Clinical Follow-Up Study to Assess Safety and Effectiveness of the p64 Flow Modulation Device Intracranial Aneurysm | Completed | Phenox GmbH | 450 | 2015-12-01 | 2020-11-20 | |
| Human Placental-Derived Stem Cell Transplantation Human Placental Derived Stem Cell - Mucopolysaccharidosis I, Mucopolysaccharidosis VI | Phase 1 | Completed | New York Medical College | 43 | 2013-04-01 | 2022-10-25 |
| Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID) Ex vivo culture and transduction of the patient's autologous CD34+ HSC with lentivirus vector VSV-G pseudotyped CL20- 4i-EF1alpha-hgammac-OPT vector, Busulfan, Palifermin - X-linked Severe Combined Immunodeficiency (XSCID) | Phase 1 / Phase 2 | Recruiting | National Institute of Allergy and Infectious Diseases (NIAID) | 40 | 2012-09-25 | 2026-10-22 |
| Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency CD34+ HSCs transduced with the lentivirus vector, VSV-G pseudotyped CL20-4i-EF1α-hγc-OPT, Palifermin, Busulfan - X-Linked Combined Immunodeficiency Diseases | Phase 1 / Phase 2 | Unknown | National Institute of Allergy and Infectious Diseases (NIAID) | 13 | 2012-04-01 | 2019-12-04 |
| Patients Treated for SCID (1968-Present) SCID, ADA-SCID | Unknown | National Institute of Allergy and Infectious Diseases (NIAID) | 1007 | 2011-05-15 | 2020-11-12 | |
| Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID-X1) Single infusion of autologous CD34+ cells transduced with the self-inactivating (SIN) gammaretroviral vector pSRS11.EFS.IL2RG.pre - X-linked Severe Combined Immunodeficiency | Not applicable | Unknown | Great Ormond Street Hospital for Children NHS Foundation Trust | 1 | 2011-04-01 | 2017-08-01 |
| Gene Therapy for X-linked Severe Combined Immunodeficiency Gene transfer - X-linked Severe Combined Immunodeficiency | Phase 1 / Phase 2 | Completed | Assistance Publique - Hôpitaux de Paris | 5 | 2010-12-01 | 2026-03-11 |
| Natural History Study of SCID Disorders Severe Combined Immunodeficiency (SCID), Leaky SCID | Enrolling by invitation | National Institute of Allergy and Infectious Diseases (NIAID) | 690 | 2010-09-02 | 2020-11-10 | |
| Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector Gene transfer - Severe Combined Immunodeficiency | Phase 1 / Phase 2 | Completed | David Williams | 8 | 2010-04-01 | 2026-04-21 |
| 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 |
| 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 |
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Severe Combined Immunodeficiency (7) · Severe combined immunodeficiency due to adenosine deaminase deficiency (4) · Primary Immunodeficiency Diseases (3) · Anemia, Sickle Cell (2) · Aneurysm (2) · Granulomatous Disease, Chronic (2) · Immune System Diseases (2) · Immunologic Deficiency Syndromes (2) · Infections (2) · Intracranial Aneurysm (2) · Leukemia, Myeloid, Acute (2) · Leukodystrophy, Globoid Cell (2) · Leukodystrophy, Metachromatic (2) · Metabolism, Inborn Errors (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (28) · Genetic Diseases, X-Linked (28) · Infant, Newborn, Diseases (28) · Genetic Diseases, Inborn (27) · Immune System Diseases (26) · Immunologic Deficiency Syndromes (26) · Primary Immunodeficiency Diseases (25) · Severe Combined Immunodeficiency (21) · Metabolic Diseases (10) · Nutritional and Metabolic Diseases (10) · DNA Repair-Deficiency Disorders (7) · Hematologic Diseases (5) · Hemic and Lymphatic Diseases (5) · Brain Diseases (4) · Central Nervous System Diseases (4)
Busulfan (4) · Fibroblast Growth Factor 7 (3) · Genetic Engineering (2) · Alemtuzumab (1) · Cord Blood Stem Cell Transplantation (1) · Cyclosporine (1) · Cyclosporins (1) · Filgrastim (1) · Mycophenolic Acid (1) · Whole-Body Irradiation (1) · fludarabine (1) · fludarabine phosphate (1) · mecasermin (1) · plerixafor (1) · propeptide convertase 5, rat (1)
National Institute of Allergy and Infectious Diseases (NIAID) (8) · National Human Genome Research Institute (NHGRI) (3) · David Williams (2) · Great Ormond Street Hospital for Children NHS Foundation Trust (2) · Phenox GmbH (2) · Assistance Publique - Hôpitaux de Paris (1) · Baylor College of Medicine (1) · Celgene Corporation (1) · Children's Hospital of Chongqing Medical University (1) · Children's Hospital of Fudan University (1) · Fred Hutchinson Cancer Center (1) · New York Medical College (1) · RTI International (1) · Shenzhen Geno-Immune Medical Institute (1) · St. Jude Children's Research Hospital (1)
United States (20) · China (3) · France (3) · United Kingdom (3) · Canada (2) · Germany (2) · Italy (2) · Argentina (1) · Belgium (1) · Bulgaria (1) · Poland (1) · Romania (1) · Russia (1) · Slovakia (1) · Switzerland (1)
Suk S De Ravin, M.D. (3) · Morton J. Cowan, MD (2) · Adrian Thrasher, Prof (1) · Alain Fischer, MD, PhD (1) · Christopher C. Dvorak, MD (1) · Claire Booth, Dr (1) · Curt Scharfe, MD, PhD (1) · Elie Haddad, MD, PhD (1) · Elizabeth K Garabedian, R.N. (1) · Gary Kleiner, MDPhD (1) · Harry L Malech, M.D. (1) · Jennifer Whangbo, MD (1) · Lauri Burroughs (1) · Lolie Yu, MD (1)
PMID 25075835, PMID 25295500, PMID 30261899, PMID 20004776, 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 18974988, PMID 19786112, PMID 19996402, PMID 20489056, PMID 20673987, PMID 21479529, PMID 21725047, PMID 21861757, PMID 22224700, PMID 22308292, PMID 23832379, PMID 24113871, PMID 27888015, PMID 29021228, PMID 29728406, PMID 30154114, PMID 30194989, PMID 30995372, PMID 31315600, PMID 33006109, PMID 35142618, PMID 35764638, PMID 40178760, PMID 4177932, PMID 42606118
2008 (1), P01HL036444 (1), P01HL053749 (1), P30CA015704 (1)