20. 副腎白質ジストロフィー Adrenoleukodystrophy Clinical trials / Disease details
臨床試験数 : 53 / 薬物数 : 88 - (DrugBank : 31) / 標的遺伝子数 : 23 - 標的パスウェイ数 : 123
| 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 | EUCTR2015-002805-13-NL (EUCTR) | 07/09/2021 | 30/04/2021 | Longterm Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product | Longterm Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product | Cerebral Adrenoleukodystrophy (CALD) MedDRA version: 20.0;Level: PT;Classification code 10051260;Term: Adrenoleukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Lenti-D Drug Product INN or Proposed INN: Elivaldogene autotemcel Other descriptive name: AUTOLOGOUS CD34+ CELLS TRANSDUCED WITH LENTI-D VECTOR ENCODING ABCD1 CDNA | bluebird bio, Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 60 | Phase 3 | France;United States;Argentina;Brazil;Australia;Netherlands;United Kingdom | ||
| 2 | EUCTR2018-001145-14-DE (EUCTR) | 23/01/2020 | 27/11/2018 | Clinical study to assess the efficacy and safety of gene therapy for the treatment of cerebral adrenoleukodystrophy (CALD) | A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects =17 Years of Age With Cerebral Adrenoleukodystrophy (CALD) | Cerebral Adrenoleukodystrophy (CALD) MedDRA version: 20.0;Level: PT;Classification code 10051260;Term: Adrenoleukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Lenti-D Drug Product INN or Proposed INN: elivaldogene autotemcel Other descriptive name: Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA INN or Proposed INN: plerixafor Other descriptive name: PLERIXAFOR | bluebird bio, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 35 | Phase 3 | France;United States;Netherlands;Germany;United Kingdom;Italy | ||
| 3 | EUCTR2018-001145-14-NL (EUCTR) | 24/06/2019 | 25/02/2019 | Clinical study to assess the efficacy and safety of gene therapy for the treatment of cerebral adrenoleukodystrophy (CALD) | A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects =17 Years of Age With Cerebral Adrenoleukodystrophy (CALD) | Cerebral Adrenoleukodystrophy (CALD) MedDRA version: 20.0;Level: PT;Classification code 10051260;Term: Adrenoleukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Lenti-D Drug Product Product Code: Not Applicable INN or Proposed INN: elivaldogene autotemcel Other descriptive name: Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA | bluebird bio, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 35 | Phase 3 | France;United States;Germany;Netherlands;Italy;United Kingdom | ||
| 4 | EUCTR2018-001145-14-IT (EUCTR) | 19/04/2019 | 02/08/2021 | Clinical study to assess the efficacy and safety of gene therapy for the treatment of cerebral adrenoleukodystrophy (CALD) | A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects inferior or equal to 17 Years of Age With Cerebral Adrenoleukodystrophy (CALD) - ALD-104 | Cerebral Adrenoleukodystrophy (CALD) MedDRA version: 20.0;Level: PT;Classification code 10051260;Term: Adrenoleukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Lenti-D Drug Product Product Code: [not applicable] INN or Proposed INN: elivaldogene autotemcel Other descriptive name: Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA | BLUEBIRD BIO, INC. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 35 | Phase 3 | France;United States;Netherlands;Germany;United Kingdom;Italy | ||
| 5 | EUCTR2018-001145-14-GB (EUCTR) | 17/04/2019 | 23/10/2018 | Clinical study to assess the efficacy and safety of gene therapy for the treatment of cerebral adrenoleukodystrophy (CALD). | A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects =17 Years of Age With Cerebral Adrenoleukodystrophy (CALD) | Cerebral Adrenoleukodystrophy (CALD) MedDRA version: 20.0;Level: PT;Classification code 10051260;Term: Adrenoleukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Lenti-D Drug Product INN or Proposed INN: elivaldogene autotemcel Other descriptive name: Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA | bluebird bio, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 35 | Phase 3 | United States;France;Netherlands;Germany;Italy;United Kingdom | ||
| 6 | EUCTR2018-001145-14-FR (EUCTR) | 27/03/2019 | 23/10/2018 | Clinical study to assess the efficacy and safety of gene therapy for the treatment of cerebral adrenoleukodystrophy. | A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects =17 Years of Age With Cerebral Adrenoleukodystrophy (CALD) | Cerebral Adrenoleukodystrophy (CALD) MedDRA version: 20.0;Level: PT;Classification code 10051260;Term: Adrenoleukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Lenti-D Drug Product Other descriptive name: Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA | bluebird bio, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 20 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;France;Netherlands;Germany;Italy;United Kingdom | ||
| 7 | EUCTR2011-001953-10-DE (EUCTR) | 13/07/2018 | 11/12/2017 | Clinical study to assess the efficacy and safety of gene therapy for the treatment of cerebral adrenoleukodystrophy | A phase 2/3 study of the efficacy and safety of hematopoietic stem cells transduced with Lenti D lentiviral vector for the treatment of cerebral adrenoleukodystrophy (CALD) | Cerebral Adrenoleukodystrophy (CALD) MedDRA version: 20.0;Level: PT;Classification code 10051260;Term: Adrenoleukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Lenti-D Drug Product Other descriptive name: AUTOLOGOUS CD34+ CELLS TRANSDUCED WITH LENTI-D VECTOR ENCODING ABCD1 CDNA | bluebird bio, Inc. | NULL | Not Recruiting | Female: no Male: yes | 32 | Phase 2;Phase 3 | United States;France;Germany;United Kingdom | ||
| 8 | EUCTR2011-001953-10-FR (EUCTR) | 08/07/2015 | 24/06/2015 | Clinical study to assess the efficacy and safety of gene therapy for the treatment of childhood cerebral adrenoleukodystrophy | A phase 2/3 study of the efficacy and safety of hematopoietic stem cells transduced with Lenti D lentiviral vector for the treatment of childhood cerebral adrenoleukodystrophy (CCALD) | Childhood Cerebral Adrenoleukodystrophy (CCALD) MedDRA version: 18.0;Level: PT;Classification code 10051260;Term: Adrenoleukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Lenti-D Drug Product | bluebird bio, Inc. | NULL | Not Recruiting | Female: no Male: yes | 15 | Phase 1;Phase 2;Phase 3 | United States;France;Argentina;Australia;Germany;United Kingdom | ||
| 9 | EUCTR2015-002805-13-FR (EUCTR) | 24/07/2017 | Longterm Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product | Longterm Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product | Cerebral Adrenoleukodystrophy (CALD) MedDRA version: 20.0;Level: PT;Classification code 10051260;Term: Adrenoleukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Lenti-D Drug Product INN or Proposed INN: elivaldogenum tavalentivecum Other descriptive name: AUTOLOGOUS CD34+ CELLS TRANSDUCED WITH LENTI-D VECTOR ENCODING ABCD1 CDNA | bluebird bio, Inc. | NULL | NA | Female: no Male: yes | 25 | Phase 3 | United States;France;Argentina;Australia;Algeria;United Kingdom |