Elivaldogene autotemcel    (DrugBank: -)

1 disease
告示番号疾患名(ページ内リンク)臨床試験数
20副腎白質ジストロフィー5

20. 副腎白質ジストロフィー [臨床試験数:49,薬物数:86(DrugBank:29),標的遺伝子数:18,標的パスウェイ数:112
Searched query = "Adrenoleukodystrophy", "Adrenomyeloneuropathy", "AMN"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
5 / 49 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.NULLNot RecruitingFemale: no
Male: yes
35Phase 3United States;France;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-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
4NCT03852498
(ClinicalTrials.gov)
January 24, 201913/2/2019Clinical 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)Drug: Lenti-D; elivaldogene autotemcelbluebird bioNULLRecruitingN/A17 YearsMale35Phase 3United States;France;Germany;Italy;Netherlands;United Kingdom
5NCT02698579
(ClinicalTrials.gov)
January 201617/2/2016Long-term Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug ProductLong-term Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug ProductCerebral Adrenoleukodystrophy (CALD);Adrenoleukodystrophy (ALD);X-Linked Adrenoleukodystrophy (X-ALD)Drug: Lenti-D; elivaldogene autotemcelbluebird bioNULLEnrolling by invitationN/AN/AMale50United States;Argentina;Australia;Brazil;France;United Kingdom;Algeria