65. Primary immunodeficiency Clinical trials / Disease details
Clinical trials : 482 / Drugs : 653 - (DrugBank : 119) / Drug target genes : 92 - Drug target pathways : 212
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT01380990 (ClinicalTrials.gov) | November 15, 2012 | 23/6/2011 | Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency | Phase I/II, Historical Controlled, Open-label, Non-randomised, Single-centre Trial to Assess the Safety and Efficacy of EF1aS-ADA Lentiviral Vector Mediated Gene Modification of Autologous CD34+ Cells From ADA-deficient Individuals | Adenosine Deaminase Deficiency;Severe Combined Immunodeficiencies (SCID) | Genetic: Infusion of autologous EFS-ADA LV CD34+ cells;Other: Haematopoietic Stem Cell Transplantation (HSCT);Drug: Busulfan;Drug: Peg-Ada | Great Ormond Street Hospital for Children NHS Foundation Trust | Orchard Therapeutics | Completed | N/A | 15 Years | All | 36 | Phase 1/Phase 2 | United Kingdom |