65. 原発性免疫不全症候群 Primary immunodeficiency Clinical trials / Disease details
臨床試験数 : 482 / 薬物数 : 653 - (DrugBank : 119) / 標的遺伝子数 : 92 - 標的パスウェイ数 : 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 | NCT04049084 (ClinicalTrials.gov) | September 26, 2019 | 6/8/2019 | An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID | An Observational Long-term Follow-up Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency (ADA-SCID) | Adenosine Deaminase Deficiency;Severe Combined Immunodeficiency (SCID) | Biological: autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV) | Orchard Therapeutics | University of California, Los Angeles;Great Ormond Street Hospital for Children NHS Foundation Trust | Enrolling by invitation | N/A | N/A | All | 70 | United States;United Kingdom | |
| 3 | NCT03878069 (ClinicalTrials.gov) | June 25, 2019 | 12/3/2019 | Registry Study of Revcovi Treatment in Patients With ADA-SCID | Single Arm, Open-Label, Multicenter, Registry Study of Revcovi (Elapegademase-lvlr) Treatment in ADA-SCID Patients Requiring Enzyme Replacement Therapy | Adenosine Deaminase Deficiency;Severe Combined Immunodeficiency | Biological: elapegademase-lvlr | Chiesi Farmaceutici S.p.A. | NULL | Recruiting | N/A | 65 Years | All | 20 | United States | |
| 4 | 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 |
| 5 | EUCTR2017-001731-39-IT (EUCTR) | 12/10/2017 | 02/07/2020 | Retroviral insertion site methodology study | Methodology study to investigate the utility of retroviral insertion site analysis in samples from subjects treated with Strimvelis™ gene therapy - Retroviral insertion site methodology study | Adenosine deaminase (ADA) deficiency severe combined immunodeficiency MedDRA version: 20.1;Level: LLT;Classification code 10066367;Term: Adenosine deaminase deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: Strimvelis Product Name: Strimvelis Product Code: [NA] | Orchard Therapeutics (Europe) Ltd | NULL | Not Recruiting | Female: yes Male: yes | 15 | Phase 4 | Turkey;Switzerland;Italy | ||
| 6 | 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 | ||
| 7 | NCT01420627 (ClinicalTrials.gov) | January 24, 2014 | 18/8/2011 | EZN-2279 in Patients With ADA-SCID | A Study of EZN-2279 (Polyethylene Glycol Recombinant Adenosine Deaminase [PEG-rADA]) Administered as a Weekly Intramuscular Injection in Patients With Adenosine Deaminase (ADA)-Deficient Combined Immunodeficiency | ADA-SCID;Adenosine Deaminase Deficiency;Severe Combined Immunodeficiency | Biological: EZN-2279;Biological: Adagen | Leadiant Biosciences, Inc. | NULL | Completed | N/A | N/A | All | 7 | Phase 3 | United States |
| 8 | 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 |
| 9 | 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 |
| 10 | 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 | ||
| 11 | NCT00794508 (ClinicalTrials.gov) | November 2008 | 19/11/2008 | MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID | MND-ADA Transduction of CD34+ Cells From the Bone Marrow Of Children With Adenosine Deaminase (ADA)-Deficient Severe Combined Immunodeficiency (SCID): Effect of Discontinuation of PEG-ADA and Marrow Cytoreduction With Busulfan | Severe Combined Immunodeficiency | Biological: ADA gene transfer | Donald B. Kohn, M.D. | FDA Office of Orphan Products Development;National Institutes of Health (NIH) | Completed | 1 Month | 18 Years | All | 10 | Phase 2 | United States |
| 12 | NCT01279720 (ClinicalTrials.gov) | October 2003 | 18/1/2011 | Gene Therapy ADA Deficiency | Phase I Gene Therapy Protocol for Adenosine Deaminase Deficiency | Adenosine Deaminase Deficiency | Biological: Intravenous infusion of transduced cells | Great Ormond Street Hospital for Children NHS Foundation Trust | NULL | Completed | N/A | 18 Years | Both | 8 | Phase 1/Phase 2 | United Kingdom |
| 13 | NCT00018018 (ClinicalTrials.gov) | June 20, 2001 | 27/6/2001 | Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency | Treatment of SCID Due to ADA Deficiency With Autologous Cord Blood or Bone Marrow CD34+ Cells Transduced With a Human ADA Gene | Severe Combined Immunodeficiency Syndrome | Drug: CD34+ cells transduced with ADA retrovir | National Human Genome Research Institute (NHGRI) | NULL | Completed | 1 Month | N/A | All | 8 | Phase 1 | United States |
| 14 | NCT00008450 (ClinicalTrials.gov) | August 11, 1997 | 6/1/2001 | Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow Transplant | Induction of Mixed Hematopoietic Chimerism in Patients With Severe Combined Immunodeficiency Disorders Using Allogeneic Bone Marrow and Post-Transplant Immunosuppression With Cyclosporine and Mycophenolate Mofetil | Adenosine Deaminase Deficiency;Autosomal Recessive Disorder;Immune System Disorder;Purine-Nucleoside Phosphorylase Deficiency;Severe Combined Immunodeficiency;Severe Combined Immunodeficiency With Absence of T and B Cells;X-Linked Severe Combined Immunodeficiency | Procedure: Allogeneic Bone Marrow Transplantation;Drug: Cyclosporine;Other: Laboratory Biomarker Analysis;Drug: Mycophenolate Mofetil;Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation;Radiation: Total-Body Irradiation | Fred Hutchinson Cancer Research Center | National Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI) | Completed | N/A | N/A | All | 6 | Phase 1 | United States |
| 15 | NCT00001255 (ClinicalTrials.gov) | September 1990 | 3/11/1999 | Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study | Treatment of Severe Combined Immunodeficiency Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency With Autologous Lymphocytes of CD34+ Cells Transduced With a Human ADA Gene: A Natural History Study | Severe Combined Immunodeficiency | Drug: ADA PBSC;Drug: ADA Umbilical Cord Blood Cells;Drug: Transduced Lymphocytes | National Human Genome Research Institute (NHGRI) | NULL | Completed | N/A | N/A | Both | 10 | N/A | United States |