Autologous CD34+ cells transduced with a lentiviral vector encoding the ARSA cDNA ( DrugBank: - )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
19ライソゾーム病1

19. ライソゾーム病


臨床試験数 : 899 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2009-017349-77-IT
(EUCTR)
15/03/201026/04/2010A Phase I/II clinical trial of hematopoietic stem cell gene therapy for the treatment of Metachromatic Leukodystrophy - TIGET-MLDA Phase I/II clinical trial of hematopoietic stem cell gene therapy for the treatment of Metachromatic Leukodystrophy - TIGET-MLD metachromatic leukodystrophy
MedDRA version: 9.1;Level: PT;Classification code 10024381
Product Name: autologous CD34+ cells transduced with a lentiviral vector encoding the ARSA cDNA
Trade Name: BUSILVEX
INN or Proposed INN: Busulfan
FONDAZIONE CENTRO S. RAFFAELE DEL MONTE TABORNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Phase 1/2Italy