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 | 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 | ||
| 2 | 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 |
| 3 | 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 |
| 4 | 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 |