20. Adrenoleukodystrophy
49 clinical trials,   86 drugs   (DrugBank: 29 drugs),   18 drug target genes,   112 drug target pathways
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.
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 |
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1 | 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 | |||
2 | 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 |