20. 副腎白質ジストロフィー Adrenoleukodystrophy Clinical trials / Disease details


臨床試験数 : 61 薬物数 : 90 - (DrugBank : 31) / 標的遺伝子数 : 23 - 標的パスウェイ数 : 126

  
5 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2018-001145-14-DE
(EUCTR)
23/01/202027/11/2018Clinical 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
35Phase 3France;United States;Netherlands;Germany;United Kingdom;Italy
2EUCTR2018-001145-14-NL
(EUCTR)
24/06/201925/02/2019Clinical 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
35Phase 3France;United States;Germany;Netherlands;Italy;United Kingdom
3EUCTR2018-001145-14-IT
(EUCTR)
19/04/201902/08/2021Clinical 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
35Phase 3France;United States;Netherlands;Germany;United Kingdom;Italy
4EUCTR2018-001145-14-GB
(EUCTR)
17/04/201923/10/2018Clinical 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
35Phase 3United States;France;Netherlands;Germany;Italy;United Kingdom
5EUCTR2018-001145-14-FR
(EUCTR)
27/03/201923/10/2018Clinical 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.NULLAuthorised-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): noUnited States;France;Netherlands;Germany;Italy;United Kingdom