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


臨床試験数 : 53 薬物数 : 88 - (DrugBank : 31) / 標的遺伝子数 : 23 - 標的パスウェイ数 : 123

  
1 trial found
No.TrialIDDate_
enrollment
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agemin
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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