65. Primary immunodeficiency Clinical trials / Disease details
Clinical trials : 500 / Drugs : 614 - (DrugBank : 119) / Drug target genes : 92 - Drug target pathways : 217
| 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 | EUCTR2018-000673-68-GB (EUCTR) | 09/10/2018 | 27/07/2018 | A clinical trial to study the effects of genetically modified patients' CD34+ cells in patients with X-linked Severe Combined Immunodeficiency | Phase I/II study of lentiviral gene transfer for SCID-X1 with low dose targeted busulfan - Lentiviral gene therapy for SCID-X1 | Severe combined immunodeficiency disorder (SCID) is a heterogeneous group of inherited disorders characterized by a profound reduction or absence of T lymphocyte function, resulting in lack of both cellular and humoral immunity. The most common form of SCID is an X-linked form (SCID-X1), which accounts for 30-50% of all cases. Children with SCID lack virtually all immune protection from pathogens. They are prone to repeated and persistent infections that can be very serious or life threatening. MedDRA version: 20.0;Level: LLT;Classification code 10069566;Term: Severe combined immunodeficiency syndrome;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Cryopreserved G2SCID lentiviral vector transduced patient CD34+ cells INN or Proposed INN: Cryopreserved G2SCID lentiviral vector transduced patient CD34+ cells | Great Ormond Street Hospital for Children NHS Trust | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 5 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United Kingdom | ||
| 2 | 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 | ||
| 3 | EUCTR2010-024253-36-GB (EUCTR) | 03/08/2012 | 09/05/2012 | A clinical trial to study the effects of genetically modified patients' CD34+ cells | 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 autologus CD34+ cells from ADA-deficient individuals - LV Gene Therapy for ADA 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: 20.0;Level: LLT;Classification code 10066372;Term: ADA deficiency;System Organ Class: 100000012248;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: EF1aS-ADA lentiviral vector transduced patient CD34+ cells Product Code: transduced patient CD34+ cells INN or Proposed INN: EF1aS-ADA lentiviral vector gene modified autologous CD34+ cells 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 | Not Recruiting | Female: no Male: yes | 10 | Phase 1;Phase 2 | United Kingdom |