Gene transfer ( DrugBank: - )
1 disease
| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 65 | 原発性免疫不全症候群 | 14 |
65. 原発性免疫不全症候群
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 | NCT03601286 (ClinicalTrials.gov) | December 21, 2018 | 22/2/2018 | Lentiviral Gene Therapy for X-linked Severe Combined Immunodeficiency | Phase I/II Study of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan | Severe Combined Immunodeficiency, X-Linked | Drug: Lentiviral vector transduced CD34+ cells | Great Ormond Street Hospital for Children NHS Foundation Trust | NULL | Recruiting | 8 Weeks | 5 Years | Male | 5 | Phase 1 | United Kingdom |
| 2 | 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 | ||
| 3 | NCT03538899 (ClinicalTrials.gov) | May 31, 2018 | 3/5/2018 | Autologous Gene Therapy for Artemis-Deficient SCID | A Phase I/II Feasibility Study of Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency (ART-SCID) Using a Self-Inactivating Lentiviral Vector (AProArt) to Transduce Autologous CD34 Hematopoietic Cells | Severe Combined Immunodeficiency | Drug: AProArt;Device: CliniMACS® CD34 Reagent System cell sorter device;Drug: Busulfan | University of California, San Francisco | NULL | Recruiting | 2 Months | N/A | All | 25 | Phase 1/Phase 2 | United States |
| 4 | NCT03311503 (ClinicalTrials.gov) | January 19, 2018 | 12/10/2017 | Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning | Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning | Severe Combined Immunodeficiency, X Linked;Gene Therapy | Biological: autologous CD34+ cell transduced with G2SCID vector | David Williams | NULL | Recruiting | N/A | 5 Years | Male | 10 | Phase 1/Phase 2 | United States;United Kingdom |
| 5 | NCT03217617 (ClinicalTrials.gov) | July 15, 2017 | 3/7/2017 | Gene Transfer for SCID-X1 Using a Self-inactivating Lentiviral Vector (TYF-IL-2Rg) | Gene Transfer for X-linked Severe Combined Immunodeficiency (SCID-X1) Using a Self-inactivating Lentiviral Vector (TYF-IL-2Rg) | SCID, X Linked | Biological: TYF-IL-2Rg gene-modified autologous stem cells | Shenzhen Geno-Immune Medical Institute | NULL | Recruiting | 1 Month | 10 Years | Male | 10 | Phase 1/Phase 2 | China |
| 6 | NCT01512888 (ClinicalTrials.gov) | August 17, 2016 | 13/1/2012 | Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants | A Pilot Feasibility Study of Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants Using a Self-Inactivating Lentiviral Vector to Transduce Autologous CD34+ Hematopoietic Cells | Severe Combined Immunodeficiency Disease, X-linked | Genetic: CL20-i4-EF1a-h?c-OPT;Drug: Busulfan;Device: CliniMacs | St. Jude Children's Research Hospital | National Heart, Lung, and Blood Institute (NHLBI);Assisi Foundation;California Institute for Regenerative Medicine (CIRM) | Recruiting | N/A | 24 Months | Male | 28 | Phase 1/Phase 2 | United States |
| 7 | NCT01306019 (ClinicalTrials.gov) | September 25, 2012 | 26/2/2011 | Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID) | Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency | X-linked Severe Combined Immunodeficiency (XSCID) | Drug: Palifermin;Drug: Busulfan;Biological: Ex vivo culture and transduction of the patient's autologous CD34+ HSC with lentivirus vector VSV-G pseudotyped CL20- 4i-EF1alpha-hgammac-OPT vector | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Recruiting | 2 Years | 40 Years | Male | 30 | Phase 1/Phase 2 | United States |
| 8 | NCT03315078 (ClinicalTrials.gov) | April 2012 | 16/10/2017 | Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency | Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency | X-Linked Combined Immunodeficiency Diseases | Biological: CD34+ HSCs transduced with the lentivirus vector, VSV-G pseudotyped CL20-4i-EF1a-h?c-OPT;Drug: Palifermin;Drug: Busulfan | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Recruiting | 2 Years | 40 Years | All | 13 | Phase 1/Phase 2 | United States |
| 9 | NCT01410825 (ClinicalTrials.gov) | July 2011 | 4/8/2011 | Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome | Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome | Wiskott-Aldrich Syndrome | Biological: Retrovirus-mediated gene transfer | David Williams | NULL | Active, not recruiting | 3 Months | 35 Years | Male | 5 | Phase 1/Phase 2 | United States |
| 10 | NCT01129544 (ClinicalTrials.gov) | April 2010 | 21/5/2010 | Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector | Multi-institutional Phase I/II Trial Evaluating the Treatment of SCID-X1 Patients With Retrovirus-mediated Gene Transfer | Severe Combined Immunodeficiency | Biological: Gene transfer | David Williams | Boston Children's Hospital;Children's Hospital Medical Center, Cincinnati;University of California, Los Angeles | Active, not recruiting | N/A | N/A | Male | 8 | Phase 1/Phase 2 | United States |
| 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 | NCT00028236 (ClinicalTrials.gov) | December 10, 2001 | 17/12/2001 | Stem Cell Gene Therapy to Treat X-Linked Severe Combined Immunodeficiency (XSCID) | Ex Vivo Retroviral Gene Transfer For Treatment of X-Linked Severe Combined Immunodeficiency (XSCID) | Severe Combined Immunodeficiency | Drug: Gene-Transduced Autologous CD34+ Stem Cells | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Completed | 18 Months | 20 Years | All | 3 | Phase 1 | United States |
| 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 | 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 |