DDrare: Database of Drug Development for Rare Diseases

Autologous cd34+ cells transduced with lenti-d vector encoding abcd1 cdna (DrugBank: -)

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	2

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	EUCTR2011-001953-10-DE (EUCTR)		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	NA			Female: no Male: yes	32	Phase 2;Phase 3	United States;France;Germany;United Kingdom
2	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

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

EUCTR2011-001953-10-DE
(EUCTR)

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

Female: no
Male: yes

Phase 2;Phase 3

United States;France;Germany;United Kingdom

EUCTR2015-002805-13-FR
(EUCTR)

24/07/2017

Longterm Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product

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

Female: no
Male: yes

Phase 3

United States;France;Argentina;Australia;Algeria;United Kingdom