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 | NCT04140539 (ClinicalTrials.gov) | October 15, 2019 | 24/10/2019 | A Clinical Study to Enable Process Validation of Commercial Grade OTL-101 | A Single Arm, Open Label Clinical Study to Enable Process Validation of Commercial Grade Ex Vivo Hematopoietic Stem Cell Gene Therapy (OTL-101) in Subjects With Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency (ADA-SCID) | Severe Combined Immunodeficiency Due to ADA Deficiency | Biological: OTL-101 | Orchard Therapeutics | University of California, Los Angeles | Terminated | N/A | 17 Years | All | 3 | Phase 2/Phase 3 | United States |
| 2 | NCT03765632 (ClinicalTrials.gov) | January 3, 2018 | 8/8/2018 | Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID | Efficacy and Safety of a Cryopreserved Formulation of Autologous CD34+ Haematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1a Short Form (EFS) Lentiviral Vector Encoding for Human ADA Gene in Subjects With Severe Combined Immunodeficiency (SCID) Due to Adenosine Deaminase Deficiency | Severe Combined Immunodeficiency Due to ADA Deficiency | Genetic: Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101);Drug: Busulfan;Drug: Peg-Ada | Great Ormond Street Hospital for Children NHS Foundation Trust | Orchard Therapeutics | Active, not recruiting | N/A | 17 Years | All | 13 | Phase 1/Phase 2 | United Kingdom |
| 3 | EUCTR2017-001275-23-GB (EUCTR) | 21/09/2017 | 03/07/2017 | A clinical trial to study the effects of genetically modified patients' CD34+ cells | Efficacy and safety of a cryopreserved formulation of autologous CD34+ haematopoietic stem cells transduced ex vivo with EFS lentiviral vector encoding for human ADA gene in subjects with Severe Combined Immunodeficiency (SCID) due to Adenosine Deaminase Deficiency | Adenosine deaminase (ADA) deficiency is an inherited disorder that damages the immune system and causes severe combined immunodeficiency (SCID). Children with SCID lack virtually all immune protection from bacteria, viruses, and fungi. They are prone to repeated and persistent infections that can be very serious or life-threatening. If not treated in a way that restores immune function, children with SCID usually live only a year or two. MedDRA version: 20.1;Level: LLT;Classification code 10066372;Term: ADA deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: cryopreserved EFS-ADA LV transduced patient CD34+ cells Product Code: OTL-101 INN or Proposed INN: There is no recommended INN Other descriptive name: Autologous CD34+ HSCs transduced ex vivo with EFS lentiviral vector encoding for the human ADA gene | Great Ormond Street Hospital for Children NHS Trust | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 10 | Phase 2 | United Kingdom | ||
| 4 | NCT02999984 (ClinicalTrials.gov) | December 16, 2016 | 19/12/2016 | Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID | Efficacy and Safety of Cryopreserved Formulation of Autologous CD34+ Hematopoietic Stem Cells Transduced Ex Vivo With EFS Lentiviral Vector Encoding for Human ADA Gene in Subjects With Severe Combined Immunodeficiency Due to ADA Deficiency | Severe Combined Immunodeficiency Due to ADA Deficiency | Genetic: Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101);Drug: busulfan;Drug: PEG-ADA ERT | Orchard Therapeutics | California Institute for Regenerative Medicine (CIRM);University of California, Los Angeles | Completed | N/A | 17 Years | All | 10 | Phase 1/Phase 2 | United States |
| 5 | NCT01852071 (ClinicalTrials.gov) | August 2, 2013 | 7/5/2013 | Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With EFS Lentiviral Vector Encoding for the Human ADA Gene | Autologous Transplantation of Bone Marrow CD34+ Stem/Progenitor Cells After Addition of a Normal Human ADA cDNA by the EFS-ADA Lentiviral Vector for Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency (ADA-SCID) | ADA-SCID | Genetic: Infusion of autologous EFS-ADA LV CD34+ (OTL-101);Drug: busulfan;Drug: PEG-ADA ERT | Orchard Therapeutics | National Institute of Allergy and Infectious Diseases (NIAID);National Human Genome Research Institute (NHGRI);National Heart, Lung, and Blood Institute (NHLBI);University of California, Los Angeles | Completed | 1 Month | 17 Years | All | 20 | Phase 1/Phase 2 | United States |