Lentiviral vector transduced CD34+ cells
( DrugBank: - / KEGG DRUG: - )
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 | 19 |
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
Showing 1 to 10 of 19 trials
| 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 202320230100 | 27/8/202120210827 | 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) Safety and Efficacy Study of Transplantation of Autologous CD34+ Cells TransducedWith the G2ARTE Len ... | 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 A Phase 1/2 Open Label Non Randomized Study, Multicentric, Single Arm Evaluating the Safety and Effi ... | 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 | EUCTR2020-000517-33-GB (EUCTR) | 14/07/202020200714 | 21/02/202020200221 | 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 ... | 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 ... | 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] Leukocyte Adhesion Deficiency-I (LAD-I) MedDRA version: 20.0;Level: LLT;Classification code 10018137 ... | Product Name: LADICell Product Code: RP-L201 INN or Proposed INN: CD34+CELLS Other descriptive name: CD34+CELLS Product Name: LADICell Product Code: RP-L201 INN or Proposed INN: CD34+CELLS Other descriptive name: CD ... | 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 | ||
| 3 | NCT03837483 (ClinicalTrials.gov) | January 21, 201920190121 | 8/2/201920190208 | A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wisk ... | 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) A Single Arm, Open-label Clinical Trial of Hematopoietic Stem Cell Gene Therapy With Cryopreserved A ... | 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 |
| 4 | EUCTR2018-003842-18-IT (EUCTR) | 08/01/201920190108 | 19/11/201820181119 | Gene therapy study using a frozen formulation of OTL-103 in patients with Wiskott-Aldrich Syndrome (WAS) Gene therapy study using a frozen formulation of OTL-103 in patients with Wiskott-Aldrich Syndrome ( ... | 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. A Single Arm, Open Label Clinical Study of Haematopoietic Stem Cell Gene Therapy with Cryopreserved ... | 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] Wiskott-Aldrich Syndrome MedDRA version: 20.0;Level: PT;Classification code 10061598;Term: Immunodef ... | 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: Product Name: OTL-103 Dispersion for Infusion Product Code: OTL-103 INN or Proposed INN: Other hematol ... | Orchard Therapeutics Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 6 | Phase 3 | Italy | ||
| 5 | NCT03601286 (ClinicalTrials.gov) | December 21, 201820181221 | 22/2/201820180222 | 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 |
| 6 | EUCTR2018-002680-26-ES (EUCTR) | 27/11/201820181127 | 02/08/201820180802 | 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 S ... | 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 S ... | 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] Leukocyte Adhesion Deficiency-I (LAD-I) MedDRA version: 20.0;Level: LLT;Classification code 10018137 ... | Product Name: LADICell Product Code: RP-L201 INN or Proposed INN: CD34+CELLS Other descriptive name: CD34+CELLS Product Name: LADICell Product Code: RP-L201 INN or Proposed INN: CD34+CELLS Other descriptive name: CD ... | Rocket Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 2 | Phase 1 | Spain | ||
| 7 | EUCTR2018-000673-68-GB (EUCTR) | 09/10/201820181009 | 27/07/201820180727 | A clinical trial to study the effects of genetically modified patients' CD34+ cells in patients with X-linked Severe Combined Immunodeficiency A clinical trial to study the effects of genetically modified patients' CD34+ cellsin patients with ... | Phase I/II study of lentiviral gene transfer for SCID-X1 with low dose targeted busulfan - Lentiviral gene therapy for SCID-X1 Phase I/II study of lentiviralgene transfer for SCID-X1 with low dose targeted busulfan - Lentiviral ... | 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] Severe combined immunodeficiency disorder (SCID) is a heterogeneous group of inherited disorders cha ... | Product Name: Cryopreserved G2SCID lentiviral vector transduced patient CD34+ cells INN or Proposed INN: Cryopreserved G2SCID lentiviral vector transduced patient CD34+ cells Product Name: Cryopreserved G2SCID lentiviral vector transducedpatient CD34+ cells INN or Proposed IN ... | Great Ormond Street Hospital for Children NHS Trust | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 5 | Phase 1 | United Kingdom | ||
| 8 | NCT03765632 (ClinicalTrials.gov) | January 3, 201820180103 | 8/8/201820180808 | 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 Efficacy and Safety of a Cryopreserved Formulation of Autologous CD34+Haematopoietic Stem Cells Tran ... | Severe Combined Immunodeficiency Due to ADA Deficiency | Genetic: Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101);Drug: Busulfan;Drug: Peg-Ada Genetic: Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells(OTL-101);Drug: Busulfan;Drug: P ... | Great Ormond Street Hospital for Children NHS Foundation Trust | Orchard Therapeutics | Completed | 30 Days | 17 Years | All | 13 | Phase 1/Phase 2 | United Kingdom |
| 9 | EUCTR2017-001275-23-GB (EUCTR) | 21/09/201720170921 | 03/07/201720170703 | 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 Efficacy and safety of a cryopreserved formulation of autologous CD34+haematopoietic stem cells tran ... | 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] Adenosine deaminase (ADA) deficiency is an inherited disorder that damages the immune system and cau ... | 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 Product Name: cryopreserved EFS-ADA LV transducedpatient CD34+ cells Product Code: OTL-101 INN or Prop ... | 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 | ||
| 10 | NCT02757911 (ClinicalTrials.gov) | March 201620160300 | 20/4/201620160420 | 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 A Phase I/II, Non Randomized, Monocentric Open-label Study of Autologous CD34+ Cells TransducedWith ... | 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 |