Gene therapy ( 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 | 45 |
65. 原発性免疫不全症候群
臨床試験数 : 500 / 薬物数 : 614 - (DrugBank : 119) / 標的遺伝子数 : 92 - 標的パスウェイ数 : 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 | NCT05071222 (ClinicalTrials.gov) | January 2023 | 27/8/2021 | Safety and Efficacy Study of Transplantation of Autologous CD34+ Cells Transduced With the G2ARTE Lentiviral Vector Expressing the DCLRE1C cDNA in Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency Patients (ARTEGENE) | A Phase 1/2 Open Label Non Randomized Study, Multicentric, Single Arm Evaluating the Safety and Efficacy of Gene Therapy of the Severe Combined Immunodeficiency (SCID) Caused by Mutations in the Human DCLRE1C Gene (Artemis) by Transplantation of a Single Dose of Autologous CD34+ Cells Transduced ex Vivo With the G2ARTE Lentiviral Vector Expressing the DCLRE1C cDNA | Artemis (DCLRE1C ) Deficient Severe Combined Immunodeficiency | Genetic: ARTEGENE drug product | Assistance Publique - Hôpitaux de Paris | NULL | Not yet recruiting | N/A | 1 Year | All | 5 | Phase 1/Phase 2 | France |
| 2 | NCT04797260 (ClinicalTrials.gov) | July 23, 2021 | 11/3/2021 | Phase I/II Clinical Trial Stem Cell Gene Therapy in RAG1-Deficient SCID | Phase I/II Clinical Trial of Autologous Hematopoietic Stem Cell Gene Therapy in RAG1-Deficient Severe Combined Immunodeficiency | Severe Combined Immunodeficiency Due to RAG1 Deficiency | Genetic: Gene therapy | Leiden University Medical Center | ZonMw: The Netherlands Organisation for Health Research and Development;Horizon 2020 - European Commission | Recruiting | 8 Weeks | 24 Months | All | 10 | Phase 1/2 | Netherlands |
| 3 | EUCTR2019-002343-14-NL (EUCTR) | 25/09/2020 | 26/07/2019 | GENE THERAPY IN CHILDREN WITH RAG1-DEFICIENT SEVERE COMBINED IMMUNODEFICIENCY | PHASE I/II CLINICAL TRIAL OF AUTOLOGOUS HEMATOPOIETIC STEM CELL GENE THERAPY FOR RAG1-DEFICIENT SEVERE COMBINED IMMUNODEFICIENCY | Patients with severe combined immunodeficiency (SCID) based on a genetic defect in the Recombinase Activating Gene 1 (RAG1);Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: RAG1 LV CD34+ cells Product Code: RAG1 LV CD34+ cells | Leiden University Medical Center | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 5 | Phase 1;Phase 2 | Netherlands | ||
| 4 | EUCTR2020-000517-33-GB (EUCTR) | 14/07/2020 | 21/02/2020 | Gene Therapy for Leukocyte Adhesion Deficiency-I (LAD-I):A Phase I/II Clinical Trial to Evaluate the Safety and Efficacy of the Infusion of Autologous Hematopoietic Stem Cells Transduced with a Lentiviral Vector Encoding the ITGB2 Gene | Gene Therapy for Leukocyte Adhesion Deficiency-I (LAD-I):A Phase I/II Clinical Trial to Evaluate the Safety and Efficacy of the Infusion of Autologous Hematopoietic Stem Cells Transduced with a Lentiviral Vector Encoding the ITGB2 Gene | Leukocyte Adhesion Deficiency-I (LAD-I) MedDRA version: 20.0;Level: LLT;Classification code 10018137;Term: Genetic anomalies of leukocytes;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: LADICell Product Code: RP-L201 INN or Proposed INN: CD34+CELLS Other descriptive name: CD34+CELLS | Rocket Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 9 | Phase 1;Phase 2 | United States;Spain;United Kingdom | ||
| 5 | 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) | 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 | |
| 6 | NCT03812263 (ClinicalTrials.gov) | August 30, 2019 | 18/1/2019 | A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I | Gene Therapy for Leukocyte Adhesion Deficiency-I (LAD-I): A Phase I/II Clinical Trial to Evaluate the Safety and Efficacy of the Infusion of Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector Encoding the ITGB2 Gene | Leukocyte Adhesion Defect - Type I | Biological: RP-L201 | Rocket Pharmaceuticals Inc. | California Institute for Regenerative Medicine (CIRM) | Active, not recruiting | 3 Months | N/A | All | 9 | Phase 1/Phase 2 | United States;Spain;United Kingdom |
| 7 | NCT03825783 (ClinicalTrials.gov) | April 15, 2019 | 25/1/2019 | A Gene Therapy Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I | Gene Therapy for Leukocyte Adhesion Deficiency-I (LAD-I): A Phase I Clinical Trial to Evaluate the Safety and Efficacy of the Infusion of Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector Encoding the ITGB2 Gene. | Leukocyte Adhesion Defect - Type I | Biological: RP-L201 | Rocket Pharmaceuticals Inc. | NULL | Recruiting | 3 Months | N/A | All | 2 | Phase 1 | Spain |
| 8 | NCT03837483 (ClinicalTrials.gov) | January 21, 2019 | 8/2/2019 | A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome | A Single Arm, Open-label Clinical Trial of Hematopoietic Stem Cell Gene Therapy With Cryopreserved Autologous CD34+ Cells Transduced With Lentiviral Vector Encoding WAS cDNA in Subjects With Wiskott-Aldrich Syndrome (WAS) | Wiskott-Aldrich Syndrome | Genetic: OTL-103 | Orchard Therapeutics | Ospedale San Raffaele | Active, not recruiting | N/A | 65 Years | All | 10 | Phase 3 | United States;Italy |
| 9 | EUCTR2018-003842-18-IT (EUCTR) | 08/01/2019 | 19/11/2018 | Gene therapy study using a frozen formulation of OTL-103 in patients with Wiskott-Aldrich Syndrome (WAS) | A Single Arm, Open Label Clinical Study of Haematopoietic Stem Cell Gene Therapy with Cryopreserved Autologous CD34+ Cells Transduced with Lentiviral Vector encoding WAS cDNA in Subjects with Wiskott-Aldrich Syndrome (WAS). - Clinical study using cryopreserved OTL-103 for treatment of WAS. | Wiskott-Aldrich Syndrome MedDRA version: 20.0;Level: PT;Classification code 10061598;Term: Immunodeficiency;System Organ Class: 10021428 - Immune system disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: OTL-103 Dispersion for Infusion Product Code: OTL-103 INN or Proposed INN: Other hematological Agents Other descriptive name: Autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with lentiviral vector that encodes for the human Wiskott Aldrich Syndrome (WAS) cDNA sequence Trade Name: Busilvex INN or Proposed INN: BUSULFAN Other descriptive name: NA Trade Name: Fludarabina Accord INN or Proposed INN: FLUDARABINE Other descriptive name: NA Trade Name: MabThera INN or Proposed INN: RITUXIMAB Other descriptive name: NA Trade Name: Mozobil, INN or Proposed INN: plerixafor Other descriptive name: PLERIXAFOR Trade Name: MYELOSTIM Product Name: granulocyte colony stimulating factor (G-CSF) INN or Proposed INN: | Orchard Therapeutics Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 6 | Phase 3 | Italy | ||
| 10 | 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 |
| 11 | EUCTR2018-002680-26-ES (EUCTR) | 27/11/2018 | 02/08/2018 | Gene Therapy for Leukocyte Adhesion Deficiency-I (LAD-I): A Phase I Clinical Trial to Evaluate the Safety and Efficacy of the Infusion of Autologous Hematopoietic Stem Cells Transduced with a Lentiviral Vector Encoding the ITGB2 Gene | Gene Therapy for Leukocyte Adhesion Deficiency-I (LAD-I): A Phase I Clinical Trial to Evaluate the Safety and Efficacy of the Infusion of Autologous Hematopoietic Stem Cells Transduced with a Lentiviral Vector Encoding the ITGB2 Gene | Leukocyte Adhesion Deficiency-I (LAD-I) MedDRA version: 20.0;Level: LLT;Classification code 10018137;Term: Genetic anomalies of leukocytes;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: LADICell Product Code: RP-L201 INN or Proposed INN: CD34+CELLS Other descriptive name: CD34+CELLS | Rocket Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 2 | Phase 1 | Spain | ||
| 12 | 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 | ||
| 13 | NCT03645486 (ClinicalTrials.gov) | July 1, 2018 | 24/7/2018 | Lentiviral Gene Therapy for CGD | Lentiviral Gene Therapy for Chronic Granulomatous Disease (CGD) | Chronic Granulomatous Disease | Genetic: Infusion of lentiviral TYF-CGD-modified autologous stem cells | Shenzhen Geno-Immune Medical Institute | NULL | Recruiting | N/A | N/A | All | 10 | N/A | China |
| 14 | 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 |
| 15 | JPRN-UMIN000030806 | 2018/01/17 | 15/01/2018 | A phase I/II clinical trial of hematopoietic stem cell gene therapy for Wiskott-Aldrich Syndrome | A phase I/II clinical trial of hematopoietic stem cell gene therapy for Wiskott-Aldrich Syndrome - Hematopoietic stem cell gene therapy for Wiskott-Aldrich Syndrome | Wiskott-Aldrich syndrome | WASP cDNA-transduced autologous hematopoietic stem cells are administered to patients affected by WAS after the administration of rituximab and preconditioning chemotherapy including Fludarabine and Busulfan. 1. Rituximab (day-22) 375 mg/m2 2. Preconditioning chemotherapy Fludarabine 30mg/m2 x 2 (day-3, day-2) Busulfan cumulative target AUC 48000 ng/mL*h (day-3 to -1, every 6 hours) 3. Infusion of WASP cDNA-transduced CD34 positive HSC 5 x 10^6/kg (at least 3 x 10^6/kg) | National Center for Child Heath and Development | NULL | Complete: follow-up continuing | Not applicable | Not applicable | Male | 3 | Phase 1,2 | Japan |
| 16 | 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 | ||
| 17 | NCT03478670 (ClinicalTrials.gov) | March 31, 2017 | 23/3/2018 | Strimvelis Registry Study to Follow-up Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) | Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) Registry for Patients Treated With Strimvelis (Previously GSK2696273) Gene Therapy: Long-Term Prospective, Non-Interventional Follow-up of Safety and Effectiveness | Immunologic Deficiency Syndromes | Genetic: Strimvelis | Orchard Therapeutics | NULL | Enrolling by invitation | N/A | N/A | All | 50 | Italy | |
| 18 | NCT02757911 (ClinicalTrials.gov) | March 2016 | 20/4/2016 | Gene Therapy for X-linked Chronic Granulomatous Disease | A Phase I/II, Non Randomized, Monocentric Open-label Study of Autologous CD34+ Cells Transduced With the G1XCGD Lentiviral Vector in Patients With X-Linked Chronic Granulomatous Disease | X-Linked Chronic Granulomatous Disease | Genetic: X vivo gene therapy | Genethon | NULL | Active, not recruiting | 24 Months | N/A | Male | 3 | Phase 1/Phase 2 | France |
| 19 | NCT02333760 (ClinicalTrials.gov) | September 2014 | 28/10/2014 | Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome | Long Term Safety Follow up of Patients Enrolled in the Phase I/II Clinical Trial of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome (GTG002-07 and GTG003-08). | Wiskott-Aldrich Syndrome | Genetic: Autologous CD34+ cells transduced with WASP lentiviral vector | Genethon | NULL | Active, not recruiting | N/A | N/A | Male | 10 | Phase 1/Phase 2 | France;United Kingdom |
| 20 | EUCTR2014-000274-20-GB (EUCTR) | 14/05/2014 | 12/03/2014 | Long-term follow-up of the WAS gene therapy study | LONG TERM SAFETY FOLLOW UP OF PATIENTS ENROLLED IN THE PHASE I/II CLINICAL TRIAL OF HAEMATOPOIETIC STEM CELL GENE THERAPY FOR THE WISKOTT-ALDRICH SYNDROME(GTG 002-07 AND GTG 003-08) - Long-term follow-up of the WAS gene therapy study, version 2.0 | Wiskott-Aldrich syndrome (WAS) is a rare X-linked immunodeficiencycaused by mutations in a single gene ,the Wiskott-Aldrich SyndromeProtein (WASP). WAS is characterised by micro-thrombocytopenia,recurrent infections,eczema and associated with a high incidence ofauto-immunity and of lymphoid malignancies. Over 150 uniquemutations in the WAS gene have been identified.Loss-of-functionmutations in this gene have widespread consequences on hematopoieticlineages. MedDRA version: 19.1;Level: PT;Classification code 10047992;Term: Wiskott-Aldrich syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Autologous CD34+cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector Other descriptive name: AUTOLOGOUS CD34+CELLS TRANSDUCED WITH THE W1.6_HWASP_WPRE (VSVG) LENTIVIRAL VECTOR | Genethon | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 10 | Phase 2 | United Kingdom | ||
| 21 | EUCTR2012-000242-35-DE (EUCTR) | 23/12/2013 | 29/04/2013 | Gene therapy with autologous genetically-modified CD34+ cells for X-linked Chronic Granulomatous Disease (X-CGD) | A phase I/II, non randomized, multicenter, open-label study of autologous CD34+ cells transduced with the G1XCGD Lentiviral vector in patients with X-Linked Chronic Granulomatous Disease - Phase I/II G1XCGD.01 study : an ex-vivo gene therapy for X-CGD patients | X-linked Chronic Granulomatous Disease MedDRA version: 14.1;Level: PT;Classification code 10008906;Term: Chronic granulomatous disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Suspension of autologous CD34+cells transduced with the G1XCGD viral vector Product Code: G1XCGD transduced CD34+ cells Other descriptive name: AUTOLOGOUS CD34+ CELLS TRANSDUCED EX-VIVO WITH THE PCCLCHIMGP91/VSVG LENTIVIRAL VECTOR | GENETHON | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 20 | Phase 1/2 | France;Germany;United Kingdom;Switzerland | ||
| 22 | NCT01906541 (ClinicalTrials.gov) | July 2013 | 15/7/2013 | Gene Therapy for X-CGD | A Phase I/II Gene Therapy Trial for X-CGD With a SIN Gammaretroviral Vector | X-linked Chronic Granulomatous Disease | Genetic: ex-vivo gene-therapy | Hubert Serve, Prof., MD | NULL | Recruiting | 18 Years | N/A | Both | 5 | Phase 1/Phase 2 | Germany |
| 23 | NCT01855685 (ClinicalTrials.gov) | June 24, 2013 | 14/5/2013 | Gene Therapy for X-linked Chronic Granulomatous Disease (X-CGD) | A Phase I/II, Non Randomized, Multicenter, Open-label Study of Autologous CD34+ Cells Transduced With the G1XCGD Lentiviral Vector in Patients With X-linked Chronic Granulomatous Disease | X-Linked Chronic Granulomatous Disease | Genetic: X vivo gene therapy | Genethon | NULL | Active, not recruiting | 6 Months | N/A | Male | 3 | Phase 1/Phase 2 | United Kingdom;Germany;Switzerland |
| 24 | EUCTR2012-001725-26-DE (EUCTR) | 12/03/2013 | 18/12/2012 | A Phase I/II Gene Therapy trial for X-CGD with a SIN gamma retroviral vector | A Phase I/II Gene Therapy trial for X-CGD with a SIN gamma retroviral vector - gene therapy for X-CGD | Chronic granulomatous disease (CGD) is a congenital immunodeficiency, in which neutrophil granulocytes and monocytes are not capable of producing reactive oxygen species and therefore are unable to kill phagocytized bacteria or fungi. MedDRA version: 14.1;Level: PT;Classification code 10008906;Term: Chronic granulomatous disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: genetically modified autologous blood stem cells Product Code: somatic gene-therapy by X-CGD INN or Proposed INN: G1XCG Other descriptive name: Genetically modified autologous blood stem cells | Johann Wolfgang Goethe-University | NULL | Not Recruiting | Female: yes Male: yes | 5 | Phase 1;Phase 2 | Germany | ||
| 25 | EUCTR2012-000242-35-GB (EUCTR) | 10/01/2013 | 19/07/2012 | Gene therapy with autologous genetically-modified CD34+ cells for X-linked Chronic Granulomatous Disease | A phase I/II, non randomized, multicenter, open-label study of autologous CD34+ cells transduced with the G1XCGD Lentiviral vector in patients withX-Linked Chronic Granulomatous Disease - Phase I/II G1XCGD.01 study : an ex-vivo gene therapy for X-CGD patients | X-linked Chronic Granulomatous Disease MedDRA version: 19.0;Level: PT;Classification code 10008906;Term: Chronic granulomatous disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Suspension of autologous CD34+cells transduced with the G1XCGD viral vector Product Code: G1XCGD transduced CD34+ cells Other descriptive name: AUTOLOGOUS CD34+ CELLS TRANSDUCED EX-VIVO WITH THE PCCLCHIMGP91/VSVG LENTIVIRAL VECTOR | Genethon | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 11 | Phase 1;Phase 2 | France;Germany;Switzerland;United Kingdom | ||
| 26 | 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 |
| 27 | 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 | ||
| 28 | NCT01381003 (ClinicalTrials.gov) | November 2011 | 23/6/2011 | Lentiviral Gene Therapy for X-Linked Chronic Granulomatous Disease (X-CGD) | Phase I/II Gene Therapy Protocol for X-Linked Chronic Granulomatous Disease | Granulomatous Disease, Chronic, X-linked, Variant | Genetic: pCCLchimGp91s lentiviral vector transduced CD34+ cells infusion | Great Ormond Street Hospital for Children NHS Foundation Trust | NULL | Withdrawn | N/A | 30 Years | Male | 0 | Phase 1/Phase 2 | United Kingdom |
| 29 | NCT01347346 (ClinicalTrials.gov) | May 2011 | 3/5/2011 | Gene Therapy for WAS | Phase I/II Clinical Trial of Haematopoietic Stem Cell Gene Therapy for the Wiskott-Aldrich Syndrome | Wiskott-Aldrich Syndrome | Genetic: Autologous CD34 positive cells transduced with a lentiviral vector containing human WAS gene | Genethon | Hôpital Necker-Enfants Malades | Completed | N/A | N/A | Male | 5 | Phase 1/Phase 2 | France |
| 30 | NCT01175239 (ClinicalTrials.gov) | April 2011 | 29/7/2010 | Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID-X1) | Gene Therapy for SCID-X1 Using a Self-inactivating (SIN) Gammaretroviral Vector | X-linked Severe Combined Immunodeficiency | Genetic: Single infusion of autologous CD34+ cells transduced with the self-inactivating (SIN) gammaretroviral vector pSRS11.EFS.IL2RG.pre | Great Ormond Street Hospital for Children NHS Foundation Trust | NULL | Active, not recruiting | N/A | 16 Years | Male | 1 | N/A | United Kingdom |
| 31 | NCT01347242 (ClinicalTrials.gov) | March 2011 | 3/5/2011 | Gene Therapy for Wiskott-Aldrich Syndrome (WAS) | Phase I/II Clinical Trial of Haematopoietic Stem Cell Gene Therapy for the Wiskott-Aldrich Syndrome | Wiskott-Aldrich Syndrome | Genetic: Autologous CD34 positive cells transduced with a lentiviral vector containing human WAS gene | Genethon | Great Ormond Street Hospital for Children NHS Foundation Trust;Institute of Child Health | Completed | N/A | N/A | Male | 6 | Phase 1/Phase 2 | United Kingdom |
| 32 | NCT01515462 (ClinicalTrials.gov) | April 20, 2010 | 23/12/2011 | Gene Therapy for Wiskott-Aldrich Syndrome | A Phase I/II Clinical Trial of Hematopoietic Stem Cell Gene Therapy for the Wiskott-Aldrich Syndrome | Wiskott-Aldrich Syndrome (WAS) | Genetic: OTL-103 | Orchard Therapeutics | Ospedale San Raffaele | Active, not recruiting | N/A | N/A | All | 8 | Phase 1/Phase 2 | Italy |
| 33 | EUCTR2009-017346-32-IT (EUCTR) | 15/03/2010 | 26/04/2010 | HAEMATOPOIETIC STEM CELL GENE THERAPY | A PHASE I/II CLINICAL TRIAL OF HAEMATOPOIETIC STEM CELL GENE THERAPY FOR THE WISKOTT-ALDRICH SYNDROME - TIGET-WAS | Wiskott-Aldrich Syndrom MedDRA version: 20.0;Level: PT;Classification code 10061598;Term: Immunodeficiency;System Organ Class: 10021428 - Immune system disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: FLUDARABINA TEVA - 25 MG/ML CONCENTRATO PER SOLUZIONE INIETTABILE O PER INFUSIONE 1 FLACONCINO DI VETRO DA 2 ML Product Name: na Product Code: [na] INN or Proposed INN: FLUDARABINA Other descriptive name: FLUDARABINA Trade Name: MOZOBIL - 20 MG/ML - SOLUZIONE INIETTABILE - USO SOTTOCUTANEO - FLACONCINO (VETRO) - 24 MG/1.2 ML 1 FLACONCINO Product Name: PLERIXAFOR Product Code: [na] INN or Proposed INN: PLERIXAFOR Other descriptive name: Plerixafor Trade Name: MYELOSTIM - 34 1 FLACONCINO LIOFILIZZATO 33.6 MIU + SIRINGA PRERIEMPITA SOLVENTE 1 ML Product Name: na Product Code: [na] INN or Proposed INN: LENOGRASTIM Trade Name: MABTHERA - 2 FIALE 100 MG 10 ML Product Name: RITUXIMAB Product Code: [na] INN or Proposed INN: RITUXIMAB Other descriptive name: RITUXIMAB Trade Name: BUSILVEX - 6 MG/ML - CONCENTRATO PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO - 10 ML 8 FLACONCINI Product Name: na Product Code: [na] | Orchard Therapeutics (Europe) Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 8 | Phase 1;Phase 2 | Italy | ||
| 34 | EUCTR2007-000684-16-GB (EUCTR) | 21/01/2010 | 17/09/2009 | Gene therapy for SCID-X1 using a self-inactivating (SIN) gammaretroviral vector. - Gene therapy for SCID-X1 | Gene therapy for SCID-X1 using a self-inactivating (SIN) gammaretroviral vector. - Gene therapy for SCID-X1 | X-Linked severe combined Immunodeficiency (SCID-X1) MedDRA version: 9.1;Level: LLT;Classification code 10010099;Term: Combined immunodeficiency | Product Name: pSRS11.EFS.IL2RG.pre* retroviral vector Product Code: pSRS11.EFS.IL2RG.pre* retroviral vector Product Name: pSRS11.EFS.IL2RG.pre* retroviral vector transduced cells Product Code: pSRS11.EFS.IL2RG.pre* retroviral vector transduce | Great Ormond Street Hospital NHS Trust / University College London - Institute of Child Health | NULL | Not Recruiting | Female: no Male: yes | 10 | Phase 1;Phase 2 | United Kingdom | ||
| 35 | EUCTR2007-004308-11-GB (EUCTR) | 11/01/2010 | 06/07/2009 | Gene therapy for WAS | PHASE I/II CLINICAL TRIAL OF HAEMATOPOIETIC STEM CELL GENE THERAPYFOR THE WISKOTT-ALDRICH SYNDROME - Gene Therapy for WAS , version 5.0 | Wiskott-Aldrich syndrome (WAS) is a rare X-linked immunodeficiency caused by mutations in a single gene ,the Wiskott-Aldrich Syndrome Protein (WASP). WAS is characterised by micro-thrombocytopenia, recurrent infections,eczema and associated with a high incidence of auto-immunity and of lymphoid malignancies. Over 150 unique mutations in the WAS gene have been identified.Loss-of-function mutations in this gene have widespread consequences on hematopoietic lineages. MedDRA version: 19.1;Level: PT;Classification code 10047992;Term: Wiskott-Aldrich syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Autologous CD34+cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector Other descriptive name: AUTOLOGOUS CD34+CELLS TRANSDUCED WITH THE W1.6_HWASP_WPRE (VSVG) LENTIVIRAL VECTOR Product Name: Autologous CD34+cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector Other descriptive name: AUTOLOGOUS CD34+CELLS TRANSDUCED WITH THE W1.6_HWASP_WPRE (VSVG) LENTIVIRAL VECTOR Product Name: Autologous CD34+cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector Other descriptive name: AUTOLOGOUS CD34+CELLS TRANSDUCED WITH THE W1.6_HWASP_WPRE (VSVG) LENTIVIRAL VECTOR | Genethon | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | Phase 1;Phase 2 | United Kingdom | |||
| 36 | NCT00579137 (ClinicalTrials.gov) | October 2007 | 19/12/2007 | Allogeneic SCT Of Pts With SCID And Other Primary Immunodeficiency Disorders | CD45 and Alemtuzumab Monoclonal Antibody Conditioning Regimen For Allogeneic Donor Stem Cell Transplantation Of Patients With Severe Combined Immunodeficiency Disease (SCID) And Other Primary Immunodeficiency Disorders | Severe Combined Immunodeficiency Disease;Severe Primary Immunodeficiency Disorder;Undefined T Cell Deficiency Disorder;Wiskott-Aldrick Syndrome | Biological: Campath -1H;Drug: Fludarabine;Biological: Anti-CD45;Procedure: Stem cell infusion | Baylor College of Medicine | Center for Cell and Gene Therapy, Baylor College of Medicine;Texas Children's Hospital | Terminated | N/A | N/A | All | 3 | Phase 1/Phase 2 | United States |
| 37 | NCT00778882 (ClinicalTrials.gov) | January 2007 | 21/10/2008 | Gene Therapy for Chronic Granulomatous Disease in Korea | An Open-label, Uncontrolled, Single Center, Phase I/II Trial to Assess the Safety and Efficacy of Autologous Hematopoietic Stem Cells Transduced With MT-gp91 Retroviral Vector in gp91 Defective Chronic Granulomatous Disease Patients | Chronic Granulomatous Disease | Drug: VM106 | Helixmith Co., Ltd. | NULL | Active, not recruiting | N/A | N/A | Male | 2 | Phase 1/Phase 2 | Korea, Republic of |
| 38 | NCT00394316 (ClinicalTrials.gov) | October 30, 2006 | 31/10/2006 | Gene Therapy for Chronic Granulomatous Disease | Autologous Transplantation of Genetically Modified Cells for the Treatment of X-Linked Chronic Granulomatous Disease | Chronic Granulomatous Disease | Drug: Phagocyte Oxidase Subunit Transduced CD34 Hematopoietic Stem Cells | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Terminated | 3 Years | 55 Years | Male | 3 | Early Phase 1 | United States |
| 39 | NCT00927134 (ClinicalTrials.gov) | June 2004 | 22/6/2009 | Gene Therapy for X-linked Chronic Granulomatous Disease (CGD) in Children | Phase I/II Gene Therapy Study for X-linked Chronic Granulomatous Disease in Children | Chronic Granulomatous Disease | Genetic: retroviral SF71-gp91phox transduced CD34+ cells | University of Zurich | Goethe University | Completed | 1 Year | 18 Years | Male | 2 | Phase 1/Phase 2 | Switzerland |
| 40 | NCT00564759 (ClinicalTrials.gov) | January 2004 | 26/11/2007 | Gene Therapy for Chronic Granulomatous Disease | Phase I/II Gene Therapy Study for X-Linked Chronic Granulomatous Disease | Granulomatous Disease, Chronic | Drug: retroviral SF71-gp91phox transduced CD34+ cells | Johann Wolfgang Goethe University Hospitals | German Federal Ministry of Education and Research | Active, not recruiting | 18 Years | N/A | Male | 2 | Phase 1/Phase 2 | Germany |
| 41 | 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 |
| 42 | 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 |
| 43 | NCT00001476 (ClinicalTrials.gov) | June 1, 1995 | 3/11/1999 | Gene Therapy for Chronic Granulomatous Diseases - Long-term Follow-up | Gene Therapy Approach for Chronic Granulomatous Disease | Chronic Granulomatous Disease;Communicable Disease | Drug: Gene Therapy Method for CGD;Device: Isolex 300i Magnetic Cell Selector | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Completed | 5 Years | N/A | All | 14 | Phase 1 | United States |
| 44 | NCT00599781 (ClinicalTrials.gov) | March 1992 | 8/1/2008 | Gene Therapy for ADA-SCID | Treatment of ADA-SCID by Gene Therapy on Somatic Cells | Severe Combined Immunodeficiency Syndrome | Genetic: gene transduced PBL and/or gene transduced HSC | IRCCS San Raffaele | Fondazione Telethon | Completed | N/A | N/A | Both | 8 | Phase 1/Phase 2 | NULL |
| 45 | EUCTR2009-011152-22-FR (EUCTR) | 09/12/2010 | Phase 1/2 clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome - Gene therapy for WAS | Phase 1/2 clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome - Gene therapy for WAS | Phase 1/2 clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome. An open labelled, non-randomised, phase I/II, cohort study involving a single infusion of autologous CD34+ cells transduced with the lentiviral vector w1.6_hWASP_WPRE (VSVg) in up to 5 patients with WAS. | Product Name: Autologous CD34+ cells transduced with the Lentiviral vector containing the human Wiskott Aldrich Sy Product Code: GTG003.08 | GENETHON | NULL | Not Recruiting | Female: no Male: yes | 10 | Phase 1;Phase 2 | France |