AUTOLOGOUS CD34+ CELLS TRANSDUCED WITH LENTI-D VECTOR ENCODING ABCD1 CDNA ( DrugBank: - )


1 disease
IDDisease name (Link within this page)Number of trials
20Adrenoleukodystrophy3

20. Adrenoleukodystrophy


Clinical trials : 53 Drugs : 88 - (DrugBank : 31) / Drug target genes : 23 - Drug target pathways : 123
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
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PhaseCountries
1EUCTR2015-002805-13-NL
(EUCTR)
07/09/202130/04/2021Longterm Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug ProductLongterm 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, IncNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
60Phase 3France;United States;Argentina;Brazil;Australia;Netherlands;United Kingdom
2EUCTR2011-001953-10-DE
(EUCTR)
13/07/201811/12/2017Clinical study to assess the efficacy and safety of gene therapy for the treatment of cerebral adrenoleukodystrophyA 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.NULLNot RecruitingFemale: no
Male: yes
32Phase 2;Phase 3United States;France;Germany;United Kingdom
3EUCTR2015-002805-13-FR
(EUCTR)
24/07/2017Longterm Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug ProductLongterm 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.NULLNAFemale: no
Male: yes
25Phase 3United States;France;Argentina;Australia;Algeria;United Kingdom