Autologous cd34+ cells transduced with lenti-d vector encoding abcd1 cdna    (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2

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.
2 / 49 trials found
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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Inclusion_
agemin
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agemax
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PhaseCountries
1EUCTR2015-002805-13-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
2EUCTR2011-001953-10-DE
(EUCTR)
11/12/2017Clinical 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.NULLNAFemale: no
Male: yes
32Phase 2;Phase 3United States;France;Germany;United Kingdom