| Status | Study |
| Recruiting |
Study Name: Autologous Cryopreserved CD34+ Hematopoietic Cells Transduced With EFS-ADA Lentivirus for ADA SCID Condition: Severe Combined Immunodeficiency Due to ADA Deficiency Date: 2016-12-19 Interventions: Biological: Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells Infusion of OTL-101 after reduce |
| Completed |
Study Name: 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 Condition: Adenosine Deaminase Deficiency ADA-SCID Date: 2013-12-20 Interventions: Genetic: Lentiviral Gene Transfer |
| Recruiting |
Study Name: Participation in a Research Registry for Immune Disorders Condition: ADA-SCID Adenosine Deaminase Deficiency Date: 2013-09-25 |
| Active, not recruiting |
Study Name: Autologous Transplant of EFS-ADA Modified Bone Marrow Cells for ADA-Deficient Severe Combined Immunodeficiency (SCID) Condition: ADA-SCID Date: 2013-05-07 Interventions: Genetic: EFS-ADA transduced CD34+ cells from the bone marrow Eligible subjects will undergo bone marrow |
| Recruiting |
Study Name: EZN-2279 in Patients With ADA-SCID Condition: ADA-SCID Adenosine Deaminase Deficiency Date: 2011-08-18 Interventions: Biological: EZN-2279 weekly ad |
| Recruiting |
Study Name: Lentiviral Gene Therapy for Adenosine Deaminase (ADA) Deficiency Condition: Adenosine Deaminase Deficiency Severe Combined Immunodeficienci Date: 2011-06-23 Interventions: Genetic: EF1αS-ADA lentiviral vector transduced patient Cd34+ cells EF1αS-ADA lentiviral vecto |
| Recruiting |
Study Name: Patients Treated for SCID (1968-2010) Condition: SCID ADA-SCID XSCID Date: 2011-04-29 |
| Completed |
Study Name: Gene Therapy ADA Deficiency Condition: Adenosine Deaminase Deficiency Date: 2011-01-18 Interventions: Biological: Intravenous infusion of transduced cells Intravenous infusion of transduced cells |
| Recruiting |
Study Name: Natural History Study of SCID Disorders Condition: SCID Leaky SCID Omenn Syndrome Date: 2010-08-20 |
| Withdrawn |
Study Name: Quality of Life and Neuropsychiatric Sequelae in Patients Treated With Gene Therapy for ADA-SCID and in Their Parents Condition: ADA-SCID Date: 2010-08-14 |