3 completed, 1 unknown, 1 withdrawn. 2 Phase 1, 1 Phase 2, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 2 | Completed 2 |
| Phase 2 | 1 | Completed 1 |
| Phase 3 | 1 | Completed 1 |
Follow ADA-SCID: new trials by email Literature Review of ADA-SCID trials Research Report on ADA-SCID
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 |
|---|---|---|---|---|---|---|
| EZN-2279 in Patients With ADA-SCID EZN-2279, Adagen - ADA-SCID, Adenosine Deaminase Deficiency | Phase 3 | Completed | Leadiant Biosciences, Inc. | 7 | 2014-01-24 | 2020-04-16 |
| Treatment of SCID Due to ADA Deficiency With Autologous Transplantation of Cord Blood or Hematopoietic CD 34+ Cells After Addition of a Normal Human ADA cDNA by the EFS-ADA Lentiviral Vector Lentiviral Gene Transfer - Adenosine Deaminase Deficiency, ADA-SCID | Phase 1 | Completed | National Human Genome Research Institute (NHGRI) | 1 | 2013-12-16 | 2017-09-27 |
| Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene Infusion of autologous EFS-ADA LV CD34+ (OTL-101), busulfan, PEG-ADA ERT - ADA-SCID | Phase 1 / Phase 2 | Completed | University of California, Los Angeles | 46 | 2013-08-02 | 2022-08-03 |
| 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 | |
| Quality of Life and Neuropsychiatric Sequelae in Patients Treated With Gene Therapy for ADA-SCID and in Their Parents ADA-SCID | Withdrawn | National Human Genome Research Institute (NHGRI) | 2010-08-05 | 2019-12-17 |
Severe combined immunodeficiency due to adenosine deaminase deficiency (5) · Severe Combined Immunodeficiency (3) · Reticular dysgenesis (1) · X-Linked Combined Immunodeficiency Diseases (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (3) · DNA Repair-Deficiency Disorders (3) · Genetic Diseases, Inborn (3) · Immune System Diseases (3) · Immunologic Deficiency Syndromes (3) · Infant, Newborn, Diseases (3) · Metabolic Diseases (3) · Nutritional and Metabolic Diseases (3) · Primary Immunodeficiency Diseases (3) · Genetic Diseases, X-Linked (1)
Busulfan (1) · pegademase bovine (1)
National Human Genome Research Institute (NHGRI) (2) · Leadiant Biosciences, Inc. (1) · National Institute of Allergy and Infectious Diseases (NIAID) (1) · University of California, Los Angeles (1)
United States (4) · Canada (1)
Elie Haddad, MD, PhD (2) · Donald B Kohn, MD (1) · Elizabeth K Garabedian, R.N. (1) · Morton J. Cowan, MD (1) · Richard J. O'Reilly, MD (1) · Robert A Sokolic, M.D. (1)
PMID 20004776, PMID 23818196, PMID 24139498, PMID 24290292, PMID 24331379, PMID 25075835, PMID 27262745, PMID 29728406, PMID 30154114, PMID 30193840, PMID 30194989, PMID 33974366, PMID 35671392, PMID 41289158
P01HL073104 (1), U01AI100801 (1)